tag:blogger.com,1999:blog-61890040281420854382024-03-16T00:08:27.023-07:00CIRM Stem Cell Research UpdatesUpdates by the California Institute for Regenerative Medicine about news and events in stem cell research.Anonymoushttp://www.blogger.com/profile/06993943447696166426noreply@blogger.comBlogger1185125tag:blogger.com,1999:blog-6189004028142085438.post-30150112219853941542014-07-14T09:33:00.000-07:002014-07-14T09:33:23.359-07:00Clever Stem Cells Withstand Chemo Drug’s Harmful Side Effects<div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiX1RrE9jD7iFb7oGh_CbemZ_0UgBo0ox9U3wmxmy8U2QQQhqmWCv47TjWLznLpMB5pV3KyshOGwg3ifr7_oYmOskJFJlMjZLUx68rIsYhqy8PkU6Qpma257NdSWKY27ZZOuaVPBwGUj_Ku/s1600/shutterstock_154905602.jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiX1RrE9jD7iFb7oGh_CbemZ_0UgBo0ox9U3wmxmy8U2QQQhqmWCv47TjWLznLpMB5pV3KyshOGwg3ifr7_oYmOskJFJlMjZLUx68rIsYhqy8PkU6Qpma257NdSWKY27ZZOuaVPBwGUj_Ku/s320/shutterstock_154905602.jpg" /></a></div>
<p>For some conditions, it seem that the treatment can cause almost as many problems as than the disease itself. That’s often the case with some forms of cancer, such as acute lymphoblastic leukemia.</p>
<p>The most common type of cancer to affect children, treatment usually involves chemotherapy with the drug methotrexate (MTX). And, while effective at destroying the deadly cancer cells circulating in the patients’ blood, it also does significant damage to another part of the body: the bone.</p>
<p>But new research from Brown University’s Dr. Eric Darling and his team has found that not all types of bone cells are equally at risk of being damaged by MTX. In fact, one type may actually be impervious to the drug’s negative effects. These findings, published last week in the journal <i><a href="http://www.sciencedirect.com/science/article/pii/S0014482714002626">Experimental Cell Research</a></i>, are especially important as doctors look to ways that help the youngest, most vulnerable cancer patients heal faster after treatment—regaining bone strength that can take them into a healthy adulthood.</p>
<p>As Olivia Beane, a graduate student in the Darling Lab and the lead author of this paper, explained in a <a href="http://www.sciencedaily.com/releases/2014/07/140702122537.htm">news release</a>:</p>
<blockquote>“Kids undergo chemotherapy at such an important time when they should be growing, but instead they are introduced to this very harsh environment where bone cells are damaged with these drugs. If we found a stem cell that was resistant to the chemotherapeutic agent and could promote bone growth by becoming bone itself, then maybe they wouldn’t have these issues.”</blockquote>
<p>The cell type Beane is referring to are called adipose-derived stem cells, or ASCs, which normally mature from this early, stem cell state into several types of mature cells, including bone tissue. Initially, Beane had been researching the basic properties of ASCs. But during her experiments she discovered that ASCs, unlike other stem cell types that mature into bone, appear to survive MTX. Now they just needed to understand why.</p>
<p>Further experiments revealed the underlying strengths of ASCs in resisting MTX’s effects. Normally, MTX works by binding to and shutting down a protein in the cell called dihydrofolate reductase, which is normally involved in synthesizing DNA. With DNA production shut down, cells can’t divide and multiply—which is great for killing harmful cancer cells, but potentially harmful as it can also destroy cells it shouldn’t.</p>
<p>However, ASCs are a little bit different. When coming into contact with MTX, these cells ramp up the DNA-promoting dihydrofolate reductase, producing more than enough to overcome a normal dose of MTX.</p>
<p>This discovery has raised some intriguing possibilities for treating MTX’s side effects. As Darling explained:</p>
<blockquote>“Chemotherapies do a great job of killing cells and killing the cancer, and that’s what you want. But then there is a stage after that where you need to do recovery and regeneration.”</blockquote>
<p>And while the results of this study are preliminary, the researchers are cautiously optimistic that the MTX-resistant properties of ASCs could be the key to fast tracking recovery times.</p>
<p>The first step, Darling adds, is to save a life. And MTX has done that for countless children afflicted by cancer. But the cost of saving that life should also be taken into account—so that these children who have already been through so much may one day not need to worry about long, healthy lives as they mature into adults.</p>
<p>Want to learn more about how CIRM-funded researchers are developing new tools to fight all types of leukemia? Check out our <a href="http://www.cirm.ca.gov/our-progress/leukemia-fact-sheet">Leukemia Fact Sheet</a>. </p>
Anne Holden
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/07099628927281143255noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-51485450109773329922014-07-11T13:57:00.000-07:002014-07-11T13:57:37.531-07:00Stem cell stories that caught our eye: gene Rx, new and rejuvenated blood stem cells and budget cuts<div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEih5ufDQwqLIvBGFG-eIODqOO27TSUeGgbfijWNs9YnXLtDNF95Tai2Q7firH_DIM6hspvLxrrXErzKkVkZsKZnqILA-fnLlHOn4xUdNkbcY1EAVEU8NKlyNHCtq-_cI6noHq2DQYYwz2cs/s1600/Blood+cells.jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEih5ufDQwqLIvBGFG-eIODqOO27TSUeGgbfijWNs9YnXLtDNF95Tai2Q7firH_DIM6hspvLxrrXErzKkVkZsKZnqILA-fnLlHOn4xUdNkbcY1EAVEU8NKlyNHCtq-_cI6noHq2DQYYwz2cs/s320/Blood+cells.jpg" /></a></div><i>Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, othe<b>rs are of personal interest to us, and still others are just fun.</i> <br/><br/>
Tinkering with stem cell genes safe. </b> Research at the Salk Institute provides some reassurance that using gene-editing techniques to correct disease-causing mutations in stem cells is safe. This type of intervention aims to give people a corrected version of a gene that can produce a functional protein to replace the bad one they were born with, such as the hemoglobin gene in sickle cell disease. The CIRM-funded Salk team made gene corrections with both of the two most common gene-editing techniques: using a virus to carry the correct gene into the cell, and using an enzyme to cut and splice the genes. The fear, the lead researcher said in <a href="http://www.spacedaily.com/reports/No_extra_mutations_in_modified_stem_cells_999.html"><i>Space Daily</i></a>, has been that this gene manipulation would cause unwanted mutations. Instead the team found that the very small number of mutations in the edited cells did not exceed the number in normal cells growing in the lab for the same length of time. <br/><br/>
This is great news for CIRM, since eight of our Disease Teams—all of which have the goal of moving therapies into the clinic—use gene modification techniques. These include efforts to correct the genetic mutation that causes <a href="http://www.cirm.ca.gov/our-progress/awards/clinical-trial-stem-cell-gene-therapy-sickle-cell-disease">sickle cell disease</a> and <a href="http://www.cirm.ca.gov/our-progress/awards/treatment-beta-thalassemia-high-efficiency-targeted-genome-editing-hematopoietic">beta thalassemia</a>.
<br/><br/>
<b>Interview with Nobelist on stem cell potential.</b> <a href="http://www.rawstory.com/rs/2014/07/04/nobel-laureate-were-still-scraping-the-surface-of-the-full-potential-of-stem-cell-therapy/"><i>The Raw Story</i></a> ran an interview with Nobel Laureate Martin Evans about the field he helped to create when he first isolated mouse embryonic stem cells in 1981. He won the Nobel in 2007 for later work in which he used embryonic stem cells to create specific gene modifications in mice. He said we are “just scraping the surface” in unlocking the potential of stem cells to change medicine. He also addresses various aspects of reprogramming cells to become different types of tissue and provides a bit of advice to young scientists: “You should not believe in all that you read.” <br/><br/>
<b>Keep your blood stem cells acting young.</b> Blood stem cells, like most of the adult stem cells in our various tissues, become less adept at doing their job of replenishing our tissues as we age. A team at New York’s Mount Sinai has fingered the decrease of a specific protein in older stem cells as the culprit. That protein, SIRT1, was not a surprise as it has been implicated in other aging research. When laboratory animals eat a severely calorie restricted diet and live longer, SIRT1 is active at a higher than normal level. So, it makes some sense that low levels of SIRT1 would be associated with conditions of aging. The team now wants to see if increasing SIRT1 levels can put the kick of youth back into older blood stem cells. The web portal <a href="http://www.hospitalnewspaper.com/webpages/news/DisplayNews.aspx?PT=state&state=NY&ID=0915cf6e-92a3-47ef-9c0d-3e91e4bcc29f"><i>Hospital Newspaper</i></a> ran a story on the research that the team published in Stem Cell Reports. <br/><br/>
<b>Or use a new way to create blood cells.</b> If you can’t get your own blood stem cells to behave like vigorous youthful cells another option is to get some new one. The problem is many folks cannot find a matching donor and previous attempts to grow them from earlier stage stem cells have not worked. Using either embryonic or reprogrammed iPS type stem cells to try to grow large quantities of blood-forming stem cells has always resulted in immature cells that cannot make all the blood cells and don’t readily take up residence—engraft—in the patient. Researchers at Cornell Medical College may have solved this problem by growing the stem cells in a more natural environment. They grew them in a bed of cells like those that would have surrounded them in blood vessels in a developing fetus. The resulting cells engrafted in mice and produced nearly all the components of blood. They had a few lingering problems with creating the immune system’s T cells, but got much closer than previous work. <a href="http://www.devicespace.com/News/vascular-bed-to-bedside-engineered-blood-cells/339282"><i>Device Space</i></a> picked up the medical school’s press release. <br/><br/>
This goal of creating fully functional blood stem cells is sufficiently important but vexing to the research community that CIRM organized an international workshop on the topic. You can read the resulting whitepaper “<a href="http://www.cirm.ca.gov/sites/default/files/files/about_cirm/CIRM_HSCReportAUG2013.pdf">Breaking the Bottleneck.</a>” <br/><br/>
<b>
Donors needed to power discovery.</b> With federal support for research shrinking many institutions are relying more and more on donors to fund the research that leads to discoveries and eventually therapies. The New York based web publication <a href="http://www.capitalnewyork.com/article/magazine/2014/07/8548089/discovery-science-powered-increasingly-donors"><i>Capital Playbook</i></a> painted a picture of the deficit citing a 22 percent reduction in the inflation-adjusted budget for the National Institutes of Health since 2003. It goes on to quote senior scientists fearing the loss of a generation of scientists. A great comment came from my friend and former colleague David Scadden, co-director of the Harvard Stem Cell Institute. “They are seeing their senior mentors spending more and more time writing grants and going hat in hand. That’s not a good way to inspire the best and brightest.” <br/><br/>
Don Gibbons
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/01005036402794754897noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-62831417760600772482014-07-11T11:04:00.000-07:002014-07-15T11:07:39.769-07:00Gene Rx, new and rejuvenated blood stem cells and budget cuts: Stem cell stories that caught our eye<div class="separator" style="clear: both; text-align: center;">
<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEih5ufDQwqLIvBGFG-eIODqOO27TSUeGgbfijWNs9YnXLtDNF95Tai2Q7firH_DIM6hspvLxrrXErzKkVkZsKZnqILA-fnLlHOn4xUdNkbcY1EAVEU8NKlyNHCtq-_cI6noHq2DQYYwz2cs/s1600/Blood+cells.jpg" style="margin-left: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEih5ufDQwqLIvBGFG-eIODqOO27TSUeGgbfijWNs9YnXLtDNF95Tai2Q7firH_DIM6hspvLxrrXErzKkVkZsKZnqILA-fnLlHOn4xUdNkbcY1EAVEU8NKlyNHCtq-_cI6noHq2DQYYwz2cs/s320/Blood+cells.jpg" /></a></div>
<i>Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, othe<b>rs are of personal interest to us, and still others are just fun.</b></i> <br />
<br />
Tinkering
with stem cell genes safe. Research at the Salk Institute provides
some reassurance that using gene-editing techniques to correct
disease-causing mutations in stem cells is safe. This type of
intervention aims to give people a corrected version of a gene that can
produce a functional protein to replace the bad one they were born with,
such as the hemoglobin gene in sickle cell disease. The CIRM-funded
Salk team made gene corrections with both of the two most common
gene-editing techniques: using a virus to carry the correct gene into
the cell, and using an enzyme to cut and splice the genes. The fear, the
lead researcher said in <a href="http://www.spacedaily.com/reports/No_extra_mutations_in_modified_stem_cells_999.html"><i>Space Daily</i></a>,
has been that this gene manipulation would cause unwanted mutations.
Instead the team found that the very small number of mutations in the
edited cells did not exceed the number in normal cells growing in the
lab for the same length of time. <br />
<br />
This is great news
for CIRM, since eight of our Disease Teams—all of which have the goal of
moving therapies into the clinic—use gene modification techniques.
These include efforts to correct the genetic mutation that causes <a href="http://www.cirm.ca.gov/our-progress/awards/clinical-trial-stem-cell-gene-therapy-sickle-cell-disease">sickle cell disease</a> and <a href="http://www.cirm.ca.gov/our-progress/awards/treatment-beta-thalassemia-high-efficiency-targeted-genome-editing-hematopoietic">beta thalassemia</a>.
<br />
<br />
<b>Interview with Nobelist on stem cell potential.</b> <a href="http://www.rawstory.com/rs/2014/07/04/nobel-laureate-were-still-scraping-the-surface-of-the-full-potential-of-stem-cell-therapy/"><i>The Raw Story</i></a>
ran an interview with Nobel Laureate Martin Evans about the field he
helped to create when he first isolated mouse embryonic stem cells in
1981. He won the Nobel in 2007 for later work in which he used embryonic
stem cells to create specific gene modifications in mice. He said we
are “just scraping the surface” in unlocking the potential of stem cells
to change medicine. He also addresses various aspects of reprogramming
cells to become different types of tissue and provides a bit of advice
to young scientists: “You should not believe in all that you read.” <br />
<br />
<b>Keep your blood stem cells acting young.</b>
Blood stem cells, like most of the adult stem cells in our various
tissues, become less adept at doing their job of replenishing our
tissues as we age. A team at New York’s Mount Sinai has fingered the
decrease of a specific protein in older stem cells as the culprit. That
protein, SIRT1, was not a surprise as it has been implicated in other
aging research. When laboratory animals eat a severely calorie
restricted diet and live longer, SIRT1 is active at a higher than normal
level. So, it makes some sense that low levels of SIRT1 would be
associated with conditions of aging. The team now wants to see if
increasing SIRT1 levels can put the kick of youth back into older blood
stem cells. The web portal <a href="http://www.hospitalnewspaper.com/webpages/news/DisplayNews.aspx?PT=state&state=NY&ID=0915cf6e-92a3-47ef-9c0d-3e91e4bcc29f"><i>Hospital Newspaper</i></a> ran a story on the research that the team published in Stem Cell Reports. <br />
<br />
<b>Or use a new way to create blood cells.</b>
If you can’t get your own blood stem cells to behave like vigorous
youthful cells another option is to get some new one. The problem is
many folks cannot find a matching donor and previous attempts to grow
them from earlier stage stem cells have not worked. Using either
embryonic or reprogrammed iPS type stem cells to try to grow large
quantities of blood-forming stem cells has always resulted in immature
cells that cannot make all the blood cells and don’t readily take up
residence—engraft—in the patient. Researchers at Cornell Medical College
may have solved this problem by growing the stem cells in a more
natural environment. They grew them in a bed of cells like those that
would have surrounded them in blood vessels in a developing fetus. The
resulting cells engrafted in mice and produced nearly all the components
of blood. They had a few lingering problems with creating the immune
system’s T cells, but got much closer than previous work. <a href="http://www.devicespace.com/News/vascular-bed-to-bedside-engineered-blood-cells/339282"><i>Device Space</i></a> picked up the medical school’s press release. <br />
<br />
This
goal of creating fully functional blood stem cells is sufficiently
important but vexing to the research community that CIRM organized an
international workshop on the topic. You can read the resulting
whitepaper “<a href="http://www.cirm.ca.gov/sites/default/files/files/about_cirm/CIRM_HSCReportAUG2013.pdf">Breaking the Bottleneck.</a>” <br />
<br />
<b>
Donors needed to power discovery.</b> With federal support for research
shrinking many institutions are relying more and more on donors to fund
the research that leads to discoveries and eventually therapies. The
New York based web publication <a href="http://www.capitalnewyork.com/article/magazine/2014/07/8548089/discovery-science-powered-increasingly-donors"><i>Capital Playbook</i></a>
painted a picture of the deficit citing a 22 percent reduction in the
inflation-adjusted budget for the National Institutes of Health since
2003. It goes on to quote senior scientists fearing the loss of a
generation of scientists. A great comment came from my friend and former
colleague David Scadden, co-director of the Harvard Stem Cell
Institute. “They are seeing their senior mentors spending more and more
time writing grants and going hat in hand. That’s not a good way to
inspire the best and brightest.” <br />
<br />
Don Gibbons
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>niccoloxhttp://www.blogger.com/profile/05636854896244615674noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-86129797707858573292014-07-11T08:42:00.001-07:002014-07-11T08:42:43.728-07:00Argentina soccer star pins his World Cup final hopes on stem cells <table align="center" cellpadding="0" cellspacing="0" class="tr-caption-container" style="margin-left: auto; margin-right: auto; text-align: center;"><tbody>
<tr><td style="text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEg1oQxEpGcA_g34xHb1-Z6HeWEuFhAIk_Cp8ykdYSttszMY46IGw3cqZAZUSlXJfB5YeBppfSaUICJMA-60IYIZLwSXxUagdWshbGNd9ZVBuvHNmL9c2n37VyPvFOksv5xlOAXA9zxNzbS7/s1600/Angel+di+Maria.jpg" imageanchor="1" style="margin-left: auto; margin-right: auto;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEg1oQxEpGcA_g34xHb1-Z6HeWEuFhAIk_Cp8ykdYSttszMY46IGw3cqZAZUSlXJfB5YeBppfSaUICJMA-60IYIZLwSXxUagdWshbGNd9ZVBuvHNmL9c2n37VyPvFOksv5xlOAXA9zxNzbS7/s320/Angel+di+Maria.jpg" /></a></td></tr>
<tr><td class="tr-caption" style="text-align: center;">Angel Di Maria: Photo courtesy Fanny Schertzer</td></tr>
</tbody></table>
I suppose we should have expected it. Every time there is a big sporting event stem cells seem to come into the conversation. So it’s not surprising that the World Cup in Brazil, the biggest sporting event on the planet, was bound to somehow, in some way, involve stem cells. And it has.<br />
<br />
Argentina’s speedy attacker, Angel Di Maria, suffered a torn hamstring in the game against Belgium. He was initially ruled out for the rest of the tournament but then came news that he was hoping to be able to play in the final – if his team made it, which they have – by getting a stem cell therapy.<br />
<br />
Now, as often happens in instances like this, the reports have been light on specifics although there are some hints in the media that it might involve the use of stem cells taken from Di Maria’s own fat tissue or from his blood.<br />
<br />
The web site <a href="http://www.insidespanishfootball.com/115276/angel-di-maria-to-undergo-stem-cell-treatment-to-play-the-2014-world-cup-final/">Inside Spanish Football</a> mentioned that another player, Atletico Madrid’s Diego Costa, underwent a similar procedure to try and recover from an injury before a recent championship game. The web site described it this way:
<br />
<blockquote>
“The medical procedure is used to regenerate damaged cells using the patient’s own healthy cells, with the primary object being to reduce inflammation and repair the torn muscle tissue.”
</blockquote>
Not surprisingly, because famous athletes are involved, the therapy is getting a lot of exposure in the media. The same thing occurred when <a href="http://cirmresearch.blogspot.com/2014/01/nfl-quarterbacks-super-bowl-bid-doesnt.html">Peyton Manning</a>, the quarterback for another kind of football team, the Denver Broncos, got a stem cell treatment for a neck injury; and when Yankee’s baseball pitcher <a href="http://cirmresearch.blogspot.com/2014/05/kim-kardashian-and-cc-sabathia-stem.html">C. C. Sabathia</a> underwent a stem cell treatment for a knee injury. We blogged about both of these instances.<br />
<br />
The problem with the coverage is that the media typically does a good job of explaining what the therapy is designed to do, but then fails to mention that none of these therapies have been tested or proven to work in a clinical trial. It gives the impression that this is a routine therapy for an injury. It’s not. It is, in every sense, experimental. And therein lies the problem. While the treatment may be safe there’s also a chance it is not. While it may be effective to some extent, we really have no way of knowing.<br />
<br />
Another confounding factor in all this is that alongside the stem cell therapy, Di Maria is also getting intensive traditional therapy - ice, kinesiology, electro pulse stimulation and some rehabilitation exercises in the swimming pool. So even if Di Maria does beat the odds and return in time for the World Cup Final, we really won’t know if it was the stem cells, the traditional therapy, or both that worked.<br />
<br />
And that’s the real problem here. It’s not that a professional athlete is doing everything he can to be ready for the biggest game of his life – that’s to be expected – but that the media doesn’t dig a little deeper to see if there’s any evidence this approach could work. By failing to do that they leave the playing field open to other “clinics” to offer this same kind of therapy to anyone; clinics who will promote their treatment “as used by” and give the impression that if it helped Argentina win the World Cup, or at least come in second, then it can certainly help you bounce back from your injury.<br />
<br />
So next time you read about a superstar athlete turning to stem cells for a miracle cure don’t assume that it will help them. The odds are it won’t. Sports are fun. But your health is nothing to play around with.<br />
<br />
Before considering any stem cell treatment, we highly suggest looking at educational information for patients provided by the International Society for Stem Cell Research, the world’s leading stem cell research organization. Their printable, take-it-along <a href="http://www.closerlookatstemcells.org/">Patient Handbook </a>identifies questions any patient should ask. It would be a good idea to review answers with a physician you trust.<br />
<br />
kevin mccormack<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/06993943447696166426noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-66291183149055755742014-07-10T10:38:00.000-07:002014-07-10T10:38:46.326-07:00CIRM Creativity Day Program: Training Tomorrow’s Stem Cell Scientists<div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhW5jbLa8gOL-67azjieiGCBOcotdvShED1TFlgj8unnQt97nBVJaGXNhU3-fztzQBiQXj41Z_4lm9kVhOXylvEZG3xDUA2_MY9Sp4uUghu6IERSeheXG_COiVcVTWzO7lk41D6y_LmVYkh/s1600/photo+1.PNG" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhW5jbLa8gOL-67azjieiGCBOcotdvShED1TFlgj8unnQt97nBVJaGXNhU3-fztzQBiQXj41Z_4lm9kVhOXylvEZG3xDUA2_MY9Sp4uUghu6IERSeheXG_COiVcVTWzO7lk41D6y_LmVYkh/s320/photo+1.PNG" /></a></div>
<p>It’s that time of year once again, when some of the brightest and most motivated high school students across California are given the opportunity to see first-hand what it’s like to perform cutting-edge stem cell research.</p>
<p>Called the <a href="http://www.cirm.ca.gov/our-events/creativity-day">CIRM Creativity Day Program</a>, this is a fundamental part of our mission to train the next generation of Californian stem cell scientists. Scientists who we hope will one day advance stem cell-based therapies that relieve human suffering from chronic disease and injury.</p>
<p>Offered to high-caliber students of all backgrounds, one of the program’s main goals is to give those who otherwise would not have had the opportunity to participate in cutting-edge biomedical research a taste of what it is like. The eight-to-ten week internship culminates with Creativity Day, an all-day event where each student showcases the results of his or her research project to senior scientists and CIRM staff.</p>
<p>But simply awarding these internships is not enough—just as important is communicating their value to you: policymakers, patient advocates and the public.</p>
<p>So who better to give an inside look into these internships than the students themselves?</p>
<p>Throughout the summer, students will write, photograph and/or film their experiences. You can follow along right here on the CIRM blog as we select occasional posts to share. The students are also already sharing their experiences on Instagram—log in and check out the official hashtag: #CIRMCreativityLab as they document their research progress.</p>
<p>And some students are busily filming creative, fun and informative videos documenting their experiences. When those are complete you’ll be able to view and share them right here on our blog! Want to see what kind of videos last year's students created? Check out our <a href="http://www.cirm.ca.gov/our-progress/stem-cell-videos?field_video_topic_value[]=8">compilation</a> from 2013.</p>
<p>So stay tuned in the coming days and weeks as we discover up close and personal what it’s like to be a stem-cell scientist and to see the faces of the next generation of researchers who could one day change the world.</p>
<i><p>[Photo Courtesy: Scott Voulgaris, 2014 CIRM Creativity Lab Student]</p></i>
Anne Holden
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/07099628927281143255noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-23693843683011762652014-07-09T12:42:00.000-07:002014-07-09T12:42:24.442-07:00Statement regarding former CIRM president's new position on a stem cell company's board<i>We learned this week that our former President, Dr. Alan Trounson, has joined the Board of Stem Cells, Inc., a company we are funding to develop a therapy for Alzheimer's disease. Because this raises serious concerns on a number of fronts our President and CEO, Dr. C. Randal Mills, issued the following statement:</i><br />
<br />
"CIRM was created by the people of California to help accelerate stem cell treatments to patients with unmet medical needs. Our responsibility is to them. So it is essential that we conduct these efforts with fairness and integrity.<br/><br/>
We take even the appearance of conflicts of interest very seriously. We learned of Dr. Trounson’s appointment to the Board of Stem Cells, Inc. through a press release. We understand that the appointment of CIRM’s former president to the board of directors of a CIRM loan recipient creates a risk of a conflict of interest. To mitigate a potential conflict of interest, we are taking the following steps.<br />
<br />
First, we will be sending Dr. Trounson a letter reminding him and the Chairman of the Board of Stem Cells, Inc. of the legal limitations that apply to Dr. Trounson under state law. Although it is permissible for Dr. Trounson to accept employment with a CIRM-funded company, state law prohibits him from:<br />
<br />
1. Communicating with Board members and CIRM employees on behalf of Stem Cells, Inc. for the purposes of influencing any administrative action, including the award or revocation of a grant or loan, involving Stem Cells, Inc. for one year following the termination of his employment with CIRM; and<br />
<br />
2. Assisting Stem Cells, Inc. in responding to a Request for Applications in which Dr. Trounson was involved as a CIRM employee or assisting Stem Cells Inc. with its existing loan.<br />
<br />
Second, to avoid any violation of these laws, we have also advised Board members and CIRM employees that they must refrain from communicating with Dr. Trounson regarding any matter involving Stem Cells, Inc.<br />
<br />
Finally, in the interests of transparency and good governance we will be conducting a full review of all CIRM activities relating to Stem Cells Inc."<br />
<br />
C. Randal Mills<br/><br/>
President and CEO of CIRM
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/01005036402794754897noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-43498526165939124932014-07-09T10:51:00.000-07:002014-07-09T10:51:52.363-07:00In an auditorium with 200 high school students CIRM grantee connected with one who is now heading to UCLA to study stem cells<table align="center" cellpadding="0" cellspacing="0" class="tr-caption-container" style="margin-left: auto; margin-right: auto; text-align: center;"><tbody>
<tr><td style="text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjR4S1S84RBVAyGNqn7sMbPKX7sz_jgAo3Tj8VqjGbp6jPdLGAZEwP8hZQz0V01A2sE9EgnIbyu5a-UCQM9fwmsqzfoeavBIhYenrnwcznkKucDU2LHTGr8pDXjfVDhkoZluGmH1UZUqlH-/s1600/tommy.jpg" imageanchor="1" style="margin-left: auto; margin-right: auto;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjR4S1S84RBVAyGNqn7sMbPKX7sz_jgAo3Tj8VqjGbp6jPdLGAZEwP8hZQz0V01A2sE9EgnIbyu5a-UCQM9fwmsqzfoeavBIhYenrnwcznkKucDU2LHTGr8pDXjfVDhkoZluGmH1UZUqlH-/s320/tommy.jpg" /></a></td></tr>
<tr><td class="tr-caption" style="text-align: center;">Tommy Nguyen in front of an image of nerve stem cells</td></tr>
</tbody></table>
When part of your job is to reach out to the community, share information and perhaps get the people you connect with excited about what they hear, it can be difficult to point to tangible examples of success. One arrived in my email inbox last week. <br />
<br />
Each year for Stem Cell Awareness Day in October we arrange for CIRM grantees and staff to go out to high schools and give guest lecture on stem cell science. Last year we reached more than 3,000 students. Probably no one reached more students than Julie Mangada of the Buck Institute for Research on Aging in Novato. She has talked to students at 15 schools in the past year. Another 21 classes have visited the Learning Center she manages at the Buck. <br />
<br />
In a wonderful turn of events, Julie’s talk at Piner High School in Santa Rosa last October caused one student in the auditorium to completely change the trajectory of his upcoming college pursuits. He went on to become class valedictorian and in his commencement speech last month mentioned Julie’s talk and his plans to now attend the University of California, Los Angeles for biological engineering and stem cell science. <br />
<br />
Someone in the graduation crowd called the Buck Institute after the talk and asked if the student could have a private tour of the stem cell facilities there. That student, Tommy Nguyen, joined Julie at the Buck last week to walk through the many stem cell projects there, several funded by CIRM. In particular, he saw how embryonic stem cells were grown into nerve stem cells that were transplanted into the brains of an animal model of Parkinson’s Disease (in photos). <br />
<br />
<table align="center" cellpadding="0" cellspacing="0" class="tr-caption-container" style="margin-left: auto; margin-right: auto; text-align: center;"><tbody>
<tr><td style="text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjcHznPDN1FCP8YWB7d_jdGfgJ7i6sh-RxHdOG4bQC0DmDRiebg1bETrSti8gOnFeVvLSJM5GoJZrC96TXqpxES36Hgc_-VHhm08n1gxieNuWBKKHbWHUEhxgqghnvopPjXvEkoBl-H3ZqJ/s1600/tommy3.jpg" imageanchor="1" style="margin-left: auto; margin-right: auto;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjcHznPDN1FCP8YWB7d_jdGfgJ7i6sh-RxHdOG4bQC0DmDRiebg1bETrSti8gOnFeVvLSJM5GoJZrC96TXqpxES36Hgc_-VHhm08n1gxieNuWBKKHbWHUEhxgqghnvopPjXvEkoBl-H3ZqJ/s1600/tommy3.jpg" height="213" width="320" /></a></td></tr>
<tr><td class="tr-caption" style="text-align: center;">Julie Mangada shows Tommy where cells would be implanted for Parkinson's Disease.</td></tr>
</tbody></table>
We believe getting young people into the stem cell career pipeline early is essential. That is why I conceived and managed the development of a five-unit high school curriculum in 2009 that is freely available at our <a href="http://www.cirm.ca.gov/our-progress/stem-cell-education-portal">Stem Cell Education Portal</a>. <br />
<br />
This story about Tommy shows early outreach to students can work. And it is fun when a colleague in the field can write as Julie did in her email last week, “I love my job.” <br />
<br />
She also conducts tours for the public at the Buck every Thursday from 10:30 to Noon. To reserve a spot, call (415)209-2245. <br />
<br />
Don Gibbons<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/01005036402794754897noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-13523919016058728472014-07-08T08:45:00.000-07:002014-07-08T08:45:03.053-07:00The Man Behind the Curtain: Protein Helps Keep Cancer Cells Alive and Kicking<div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiiYKpmkDavkhGKBdTbb6K0rLzgezik0FpGO_dPSgKHr0P-nakMrqxwUhoN7AFOAlXCIrJZbcuZxWnjsUSIqb6QEbgZLDK6ypNvSnCcjscFmn_CynGD3-9a7wyrQPqiondxilKWias0S4Nn/s1600/shutterstock_118491940.jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiiYKpmkDavkhGKBdTbb6K0rLzgezik0FpGO_dPSgKHr0P-nakMrqxwUhoN7AFOAlXCIrJZbcuZxWnjsUSIqb6QEbgZLDK6ypNvSnCcjscFmn_CynGD3-9a7wyrQPqiondxilKWias0S4Nn/s320/shutterstock_118491940.jpg" /></a></div>
<p>Being diagnosed with brain cancer comes with a sobering sentence: even with the most aggressive treatments, life expectancy for the most common form of brain cancer—called glioblastoma—is less than two years.</p>
<p>One of the key culprits, many scientists now believe, are <a href="www.cirm.ca.gov/our-progress/stem-cell-definitions#5">cancer stem cells</a>. Cancer stem cells are a subset of cancer cells that have three very unique properties: they can self-renew, they can propagate (or multiply) the cancer, and they can transform into the many types of cells that are found in a tumor.</p>
<p>Cancer stem cells are a relatively new concept, but they have generated a lot of excitement among cancer researchers because they could lead to the design of more effective therapies. And while whether or not they even existed has long been a source of debate among experts, a series of <a href="http://cirmresearch.blogspot.com/2014/04/tumor-cells-become-drug-resistant-by.html">recent</a> <a href="http://cirmresearch.blogspot.com/2014/05/immune-system-double-agent-fuels-colon.html">research</a> <a href="http://cirmresearch.blogspot.com/2014/05/getting-at-root-of-cancer-cancer-stem.html">findings</a> have bolstered the notion not only that they exist, but also that they play a significant role in the recurrence of some forms of cancer—including glioblastoma.</p>
<p>Researchers have been identifying, step by step, the many proteins and chemical pathways that form the path from cancer stem cell to tumor. Previous research had found the CDK class of proteins to be present in large quantities in mature cancer cells in patients suffering from glioblastoma. But they suspected something else was at play, helping to keep the CDK proteins switched on in mature cancer cells.</p>
<p>So scientists at McGill University in Canada, led by neurologist Dr. Anita Bellail, dug deeper. In their report, published this week in the journal <i><a href="http://www.nature.com/ncomms/2014/140623/ncomms5234/full/ncomms5234.html">Nature Communications</a></i>, the team has pinpointed a new class of proteins at play behind the scenes called SUMO.</p>
<p>Specifically, Bellail and her team observed that the SUMO1 protein in particular modifies a CDK protein called CDK6 in a process the team has dubbed ‘sumylation.’ As Bellail explained in this week’s <a href="https://www.mcgill.ca/channels-contribute/channels/news/scientists-find-important-piece-brain-tumour-puzzle-237368">news release</a>:</p>
<blockquote>“CDK6 sumylation inhibits its degradation and thus stabilizes the CDK6 protein in the cancer.”</blockquote>
<p>In other words, the CDK6 protein does not by itself maintain a presence in the cancer cells. Instead, it requires a little help from SUMO1. As Bellail continued:</p>
<blockquote>“We found that CDK6 sumylation is required for the renewal and growth of the cancer stem cells in glioblastoma.”</blockquote>
<p>It stands to reason, therefore, that shutting off SUMO1 could do the reverse—thus destabilizing CDK6 and, potentially, block the progression of the cancer.</p>
<p>And in further experiments by Bellail and her team, they found exactly that.</p>
<p>These results hold significant promise for finding new ways to treat glioblastoma because now the team has a target: SUMO1. In fact, the research team is now screening for drugs that can target SUMO1 and block it, thus reducing CDK6 levels and, as a result, cancer cells—and one day offering a more optimistic outcome for those diagnosed with glioblastoma.</p>
<p>Want to learn more about cancer stem cells? Check out our 2009 “<a href="http://www.cirm.ca.gov/our-progress/video/spotlight-cancer-stem-cells">Spotlight on Cancer Stem Cells</a>” video starring Dr. Michael Clarke, associate director of the <a href="http://stemcell.stanford.edu/">Stanford Institute for Stem Cell and Regenerative Medicine</a>.</p>
Anne Holden
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/07099628927281143255noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-57425050713349785522014-07-07T09:46:00.001-07:002014-07-07T09:46:32.065-07:00DISCUSSing iPSC Derivation<div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjYJTAoGCHcV1EBG6AQKQC-EeumAnEKSx5zjx-qC62GLnAriI4RuQ4r9Z9lE2Sv3GfIgydt2T8tFHz7BV6kextv7l_P3KQGajlq1LvLp7jDuL8TiB-Hze_vHe5aqsxOpzCdqlV3YQGO0RU3/s1600/shutterstock_182164514.jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjYJTAoGCHcV1EBG6AQKQC-EeumAnEKSx5zjx-qC62GLnAriI4RuQ4r9Z9lE2Sv3GfIgydt2T8tFHz7BV6kextv7l_P3KQGajlq1LvLp7jDuL8TiB-Hze_vHe5aqsxOpzCdqlV3YQGO0RU3/s320/shutterstock_182164514.jpg" /></a></div>
<i><p>Geoff Lomax is CIRM’s Senior Officer for Medical and Ethical Standards. He has been working in the implementation of CIRM’s iPSC Banking Program.</p></i>
<p>The ability to create high-quality stem cell lines depends, in part, on the generosity of donors. For example, CIRM is sponsoring an induced pluripotent stem cell bank (iPSC bank) that will eventually contain 9,000 stem cell lines. Each of these lines will be generated from tissue donated by 3,000 people suffering from known diseases such as Alzheimer’s disease, autism, hepatitis, blindness, heart disease—and many more. You can learn more about this important initiative <a href="http://cirmresearch.blogspot.com/2013/05/california-stem-cell-bank-will-save.html">here</a>.</p>
<p>In other countries there are similar initiatives like the one sponsored by CIRM.</p>
<p>We also believe that our donors should have accurate information about how their donated materials will be used, so CIRM has developed variety of tools designed to educate donors. For example, each donor must go through a process called “informed consent” where they are told the details of how iPSC’s are derived and preserved in a bank. We discuss this effort <a href="http://cirmresearch.blogspot.com/search?q=informed+consent&max-results=20&by-date=true">here</a>. In the context of the CIRM bank, new donors are being recruited under ethically and scientifically optimal conditions—where they can be fully informed as to how their cells will be used and how their contribution will spur stem cell research.</p>
<p>There are, however, existing libraries of cell and tissues that have inherent scientific value. For example, they may represent a rare or “orphan” disease. Or, they may be essential for tracking the progress of a patient’s disease over time. These collections have also been developed with the consent of the donor or patient, but, at the time of collection, iPSCs may not have even existed. One question that frequently arises is: can these cells be used for iPSC derivation, research and banking? It is not an abstract concern; CIRM and others often get questions about the adequacy of donor consent for precisely this purpose.</p>
<p>In 2013, CIRM, the NIH and the International Stem Cell Forum (ISCF)/McGill University formed the DISCUSS Project (Deriving Induced Stem Cells Using Stored Specimens) to engage the boarder research community on this issue. Rosario Isasi, a project collaborator from ISCF/McGill University, said that her research tells us that investigators around the world are asking the same questions about use of existing cell lines. To help inform researchers, we started by publishing <a href="http://stemcellstm.alphamedpress.org/content/2/10/727.abstract?sid=75568990-9078-4e3e-93ca-f56a7205d09f">a report</a> on this very subject. The report included nine points to consider when answering the question of whether existing cell libraries can be used for iPSC research.</p>
<p>We followed this initial effort with a series of meetings and workshops to get reactions to our proposed points to consider. The process culminated with a workshop in March where researchers from around world provided recommendations to the DISCUSS team. Sara Hull, a project collaborator from the NIH, noted that the international perspectives were key to producing a greatly improved product. A major workshop theme was the importance of having an effective management system in place, making sure that the cells are used in a way that is consistent with the donor consent. Participants described a number of specific mechanisms that should be used by the research community to ensure cells are used appropriately. Participants emphasized that having effective systems in place to manage cells and iPSC lines in accordance with donors wishes serves to build trust.</p>
<p><a href="http://www.cirm.ca.gov/sites/default/files/files/about_cirm/Final_DISCUSS_Workshop_Report_7_3_14.pdf">Our workshop report</a> elaborates on specific steps researchers and stem cell banks should take to ensure cell lines are used appropriately. The report also includes a revised set of points to consider based on comments received from meetings and workshops.</p>
<p>The DISCUSS Team looks forward to working with the research community to develop consensus for the responsible use of donated materials in stem cell research.</p>
Geoff Lomax
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/07099628927281143255noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-24763353546961320962014-07-07T08:54:00.000-07:002014-07-07T08:54:30.132-07:00Stem cell stories that caught our eye: multiple sclerosis, diabetes, cornea repair and of course, new stem cells too good to be true<table align="center" cellpadding="0" cellspacing="0" class="tr-caption-container" style="margin-left: auto; margin-right: auto; text-align: center;"><tbody>
<tr><td style="text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEh8dL-FMXNCA1NRRfKJ6mvt4qnUEHjF_A1inNyslpfvo8AvXtLhnii5POan7C6K4ABMAzkdzrepljmNvpGVHLDClOgB4I3ch78H_y3WWWHcI9d1cIvkSLKS6FOgsMDyzx_7ac9ZP14Xy7yU/s1600/MSC+with+buddy+cells.jpg" imageanchor="1" style="margin-left: auto; margin-right: auto;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEh8dL-FMXNCA1NRRfKJ6mvt4qnUEHjF_A1inNyslpfvo8AvXtLhnii5POan7C6K4ABMAzkdzrepljmNvpGVHLDClOgB4I3ch78H_y3WWWHcI9d1cIvkSLKS6FOgsMDyzx_7ac9ZP14Xy7yU/s320/MSC+with+buddy+cells.jpg" /></a></td></tr>
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<span style="font-size: small;">Fat
cells (yellow) descended from transplanted stem cells (green) inside a mouse 28
days after co-transplantation with “buddy cells” </span></div>
<div class="MsoNormal">
<span style="font-size: small;"> <i>courtesy Children’s Hospital</i></span></div>
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<i>Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun.</i> <br />
<br />
<b>
Buddy system gets stem cells to stick around.</b> The type of stem cell most likely to be used in a clinical trial today is the mesenchymal stem cell (MSC) found in fat and bone marrow. It is also the type of stem cell most likely to produce vaguely positive or downright disappointing results. In most situations they die within a few days of being transplanted, so the only impact they can have is from the various protein signals they secrete that may trigger the body's own natural healing processes. They never live up to their stem cell potential to form new adult tissue. A team at Harvard looked at their natural environment and found they most often live near a second type of cell called an endothelial colony-forming cell. When the team transplanted the two cells together they found the MSCs survived for weeks and matured into appropriate adult tissue. <a href="http://www.genengnews.com/gen-news-highlights/buddy-system-boosts-stem-cell-survival/81250062/"><i>Genetic Engineering & Biotechnology News</i></a> had a nice interview with members of the team about their work that appeared this week in the Proceedings of the National Academy of Sciences. <br />
<br />
<b>
Master switch for creating brain insulation.</b> Researchers know how to take a skin cell from a patient, turn it into an iPS type stem cell and then turn those cells into the type of intermediate cell that can become the myelin that insulates our nerves and is lost in Multiple Sclerosis. The problem: the process takes way too long to be a feasible therapy. To get enough of these middleman cells called oligodendrocyte progenitors for a therapy can take as much as a year. Neural stem cells naturally mature into multiple intermediate cells, but prefer to become the progenitors for neurons, which would not help an MS patient. A team at the University of Buffalo looked to see what genetic switches were active in neuron progenitors versus those for myelin. They found that just one of these switches could push the early nerve stem cells to the myelin middlemen. That genetic factor, SOX10, instantly becomes a candidate for a path to a more efficient therapy. Again, <a href="http://www.genengnews.com/gen-news-highlights/myelination-master-switch-in-brain-stem-cells-discovered/81250047/"><i>Genetic Engineering & Biotechnology News</i></a> did the best of several write-ups of this work that was published in the Proceedings of the National Academy of Sciences. <br />
<br />
You can read about CIRM’s projects working on a cure for MS on our <a href="http://www.cirm.ca.gov/our-progress/multiple-sclerosis-fact-sheet">Multiple Sclerosis Fact Sheet</a>. <br />
<br />
<b>Can gut be taught to make insulin.</b> Earlier work at Columbia University had shown that in mice you can turn off a single gene and get normal gut cells to secrete insulin and to do so in response to sugar in the bloodstream. Now the team has made the often difficult transition of moving from mouse results to humans, or in this case human gut cells in a dish. They matured human stem cells into gut tissue and then shut down the one gene. The resulting cells produced insulin in response to sugar in their environment. The research published in <b>Nature Communication got coverage on a few sites including <a href="http://consumer.healthday.com/diabetes-information-10/misc-diabetes-news-181/gut-cells-may-be-coaxed-to-make-insulin-for-people-with-type-1-diabetes-689282.html"><i>HealthDay</i></a>. <br /><br />
Early success in cornea repair poised to get even better. </b> One of the stem cell field’s early successes has been work pioneered in Italy using a type of stem cell found in the cornea of the eye. When a patient has the cornea of one eye damaged they harvest these cells, called limbal stem cells, from the healthy eye and transplant them to the damaged eye. It often works quite well, but not always and the success has been correlated with how many actual limbal stem cells are among the cells transplanted. It has been difficult to sort out and purify the stem cells until now. A team from three Harvard affiliated hospitals has found a marker that let them transplant purer human limbal stem cells into mice and they saw consistent regrowth of damaged corneas. <a href="http://www.redorbit.com/news/health/1113184412/stem-cell-corneal-regrowth-improve-vision-070314/"><i>RedOrbit</i></a> wrote up the research that was published in Nature. <br />
<br />
<b>STAP stem cell retraction everywhere.</b> When Japanese and American researchers published a new, simple method for creating stem cells in January it got way more news coverage than an unconfirmed and unconventional piece of research should have. Most of that coverage failed to include the caveat that the work needed to be replicated to confirm the findings. In less than six months, the research community quickly reported repeated failures to replicate the work and more recently found outright errors in the published papers. When the journal that published the work, Nature, formally retracted the papers this week it was good to see that this “oops-ignore-our-first-article” seemed to get equal play. To show the reach of this news, I have included the Associated Press version from the tiny Logansport <a href="http://www.pharostribune.com/bonuscontent/x1927835884/Scientists-withdraw-claim-about-making-stem-cells"><i>Pharos Tribune</i></a>, which averages about 12 pages a day and is the closest real newspaper to the tiny Indiana town where I grew up. <br />
<br />
Don Gibbons
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/01005036402794754897noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-48597612368695626832014-07-03T12:50:00.000-07:002014-07-03T12:52:04.325-07:00The Art of public service <table align="center" cellpadding="0" cellspacing="0" class="tr-caption-container" style="margin-left: auto; margin-right: auto; text-align: center;"><tbody>
<tr><td style="text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgNcCxjgvABNqVAUJ-1epTswVYcdhyphenhyphenvBmpX-2Je03Ws3BGXAoBi68y31iYS1y1dFLPpSt4um1HqOFKtXYG8a9ETEA9YAUlNC6tPwlTVL0GarxDe3eG2zF1F9fbVn2XN_f6ESL9hiCVLjBd8/s1600/Photo+Art.jpg" imageanchor="1" style="margin-left: auto; margin-right: auto;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgNcCxjgvABNqVAUJ-1epTswVYcdhyphenhyphenvBmpX-2Je03Ws3BGXAoBi68y31iYS1y1dFLPpSt4um1HqOFKtXYG8a9ETEA9YAUlNC6tPwlTVL0GarxDe3eG2zF1F9fbVn2XN_f6ESL9hiCVLjBd8/s400/Photo+Art.jpg" /></a></td></tr>
<tr><td class="tr-caption" style="text-align: center;">Our Board Vice Chair and tireless patient advocate, Art Torres</td></tr>
</tbody></table>
Usually we use this space to talk about important or interesting developments in stem cell research. Developments that help increase our understanding of how stem cells work and how we can use them to develop much-needed treatments for a wide variety of diseases and disorders. But every once in a while it’s good to stand back and take a moment to appreciate the people who work hard to make all of this possible. I don’t just mean the researchers and the scientists; I’m also talking about our tireless staff. And in this case, I’m talking about one in particular, <a href="http://www.cirm.ca.gov/board-and-meetings/board-members-art-torres">Art Torres</a>.<br />
<br />
Art is having a truly remarkable career, one that shows no sign of slowing down. He started out as a community organizer, worked with the legendary Cesar Chavez and then went on to serve 20 years in the state legislature (8 in the Assembly, 12 in the Senate) before becoming the Chairman of the California Democratic Party.<br />
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He joined the stem cell agency in 2009 as both a patient advocate (he’s a colon cancer survivor) and as the Vice-Chair of our governing Board, the Independent Citizens Oversight Committee.<br />
<br />
In 2010 he was sworn in by then Mayor Gavin Newsom to a four-year term on San Francisco’s Public Utilities Commission.<br />
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And as if all that isn’t enough, he has now agreed to be a Trustee for the <a href="http://foundation.ucsc.edu/">University of California Santa Cruz Foundation Board</a>. He was elected, unanimously, by the other Board members to serve in a leadership role to help support what the Foundation’s website calls “the university’s vision of academic excellence and its commitment to public service.”<br />
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It’s not too surprising really that Art would take on this latest challenge. UC Santa Cruz is his alma mater and he rarely wastes an opportunity to cheer on the “Slugs” as they are affectionately known.<br />
<br />
So it’s appropriate that as we celebrate the 4th of July we celebrate and honor Art for his devotion to public service. Long may it continue.<br />
<br />
kevin mccormack <div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/06993943447696166426noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-27446576373963373032014-07-03T10:02:00.000-07:002014-07-03T10:02:33.456-07:00Confining Cells within Geometric Structures Key to Replicating Embryonic Development<div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEj0glQSmdqz7pfOVOgH7mNQ_9ekci-jjby6NSbBW0XNT2amU6Kf1hGjzdd6UZrRPtKddVhWYE3q5psCp8D6Yfb9IEowPKCN0ktIAui-RI7Zxx88aaVEDvOQ3IuEDxpOtQu7aciHULQu9OLO/s1600/75564_web.jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEj0glQSmdqz7pfOVOgH7mNQ_9ekci-jjby6NSbBW0XNT2amU6Kf1hGjzdd6UZrRPtKddVhWYE3q5psCp8D6Yfb9IEowPKCN0ktIAui-RI7Zxx88aaVEDvOQ3IuEDxpOtQu7aciHULQu9OLO/s320/75564_web.jpg" /></a></div>
<p><i>Forty-two hours after they began to differentiate, embryonic cells are clearly segregating into the various layers that will one day become specific tissues and organs. Researchers say the key to achieving this patterning in culture is confining the colonies geometrically.</i></p>
<p>It’s like trying to capture, and then recreate, a moment in time: the exact instant after fertilization when a small group of dividing cells begin to organize themselves into the various cellular layers that will one day make up the skin, the heart, the liver and the brain. But for all the advances in our understanding of how an embryonic stem cell grows, matures and differentiates—scientists still can’t replicate that very important process in the lab.</p>
<p>But now, scientists at The Rockefeller University have tried something new, and in so doing have finally found a way to stimulate this organization, thus mimicking in a petri dish what happens in the human embryo. The missing ingredient, the researchers found, wasn’t a molecule or chemical compound. Rather, the team just had to use a bit of geometry.</p>
<p>Reporting in the June 29 issue of the journal <i><a href="http://www.nature.com/nmeth/journal/vaop/ncurrent/full/nmeth.3016.html">Nature Methods</a></i>, the Rockefeller team—led by Dr. Ali Brivanlou—describes how they constructed microscopic circular patterns on glass plates that confined embryonic stem cells inside, similar to a hedge maze.</p>
<p>To their amazement, the cells confined within these patterns soon began to go through gastrulation, the process by which embryonic stem cells begin to form highly organized layers that eventually mature into the body’s various organs and tissues. A second group of cells not confined within these patterns, however, did not.</p>
<p>The next question they had to figure out, according to the researchers, was why.</p>
<p>To solve this mystery, Brivanlou and his team next monitored specific chemical signals between the cells as they matured. In so doing they uncovered a delicate arrangement of chemical cues—molecular ‘on-and-off-switches’—that guided each cell down one developmental path as opposed to another. What were crucial to these cues going off without a hitch, the researchers found, were the geometric patterns.</p>
<p>As Dr. Aryeh Warmflash, one of the paper’s lead authors, stated in this week’s <a href="http://www.eurekalert.org/pub_releases/2014-06/ru-ugr063014.php">news release</a>:</p>
<blockquote>“At the fundamental level, what we have developed is a new model to explore how human embryonic stem cells first differentiate into separate populations with a very reproducible spatial order just as in an embryo. We can now follow individual cells in real time in order to find out what makes them specialize, and we can begin to ask questions about the underlying genetics of the process.”</blockquote>
<p>Added Brivanlou:</p>
<blockquote>“Understanding what happens in this moment, when individual members of this mass of embryonic stem cells begin to specialize for the very first time and organize themselves into layers, will be key to harnessing the promise of regenerative medicine.”</blockquote>
<p>Anne Holden</p>
[Image Credit: The Rockefeller University]
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/07099628927281143255noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-3489036301726367172014-07-02T11:07:00.000-07:002014-07-02T11:07:14.147-07:00More than Meets the Eye: Stem Cells Generated using Different Methods Produce Different Types of Cells <div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhuYVcLW8hSmJj1lzbdN_sDtIwyXe2HVKmuNwxSK31vH92MlpOzHXpfV1a3X7J6_6zwUGfI78Dcs6TU1FhQZ00sfdHBTThqJUQUokld6hDhHw0zsRSCjuDjffqSO8M-Vk0KYPnYobn5gPLS/s1600/75463.jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhuYVcLW8hSmJj1lzbdN_sDtIwyXe2HVKmuNwxSK31vH92MlpOzHXpfV1a3X7J6_6zwUGfI78Dcs6TU1FhQZ00sfdHBTThqJUQUokld6hDhHw0zsRSCjuDjffqSO8M-Vk0KYPnYobn5gPLS/s320/75463.jpg" /></a></div>
<p><i>Scanning electron micrograph of cultured human neuron from induced pluripotent stem cell.</i></p>
<p>What’s the best way to make a fully versatile, ‘pluripotent,’ stem cell? Three different methods each have their pluses and minuses. But now new research has found that the stem cells created by each method, while similar on the surface, show vast differences.</p>
<p>The findings, published online today in the journal Nature, reveal new insights into stem cells’ underlying cellular machinery—which is of utmost importance as researchers transform their discoveries from the lab and into much-needed therapies for patients.</p>
<p>Stem cells have held promise for regenerating tissues, or even organs, lost or damaged by injury or disease. This is due to stem cells’ ‘<a href="http://www.cirm.ca.gov/our-progress/stem-cell-definitions#2">pluripotency</a>’—their ability to transform into virtually any cell in the body. Initially, scientists used stem cells extracted from unused embryos that consenting couples had donated to research. But the use of these so-called <a href="http://www.cirm.ca.gov/our-progress/stem-cell-definitions#1">embryonic stem cells</a>, or ES cells, has since been limited due to ethical considerations and early limits to federal funding.</p>
<p>So scientists have been on the hunt for an alternative method of creating pluripotent cells. And so far, they have come up with two.</p>
<p>One, called <a href="http://www.cirm.ca.gov/our-progress/creating-new-types-stem-cells#6">somatic cell nuclear transfer (SCNT)</a> takes the genetic material of an adult cell and transplants it into an unfertilized egg. The second method transforms adult cells, such as skin or blood, back into embryonic-like stem cells—called <a href="http://www.cirm.ca.gov/our-progress/creating-new-types-stem-cells#7">induced pluripotent stem cells, or iPS cells</a>—by manipulating various genes.</p>
<p>Each of the newer methods has its pluses and minuses—but which produces cells that most closely resemble ES cells, still considered the “Gold Standard” in stem cell biology? Since the success of the SCNT technique is so recent, no one had taken a close look until now. So a collaboration of researchers from the University of California, San Diego (UCSD), The Salk Institute for Biological Sciences and Oregon Health & Science University (OHSU), compared the two methods side by side. And what they found was surprising.</p>
<p>Dr. Louise Laurent, co-senior author from UCSD, explained in <a href="http://www.eurekalert.org/pub_releases/2014-07/uoc--nrm062614.php">today’s news release</a>:</p>
<blockquote>“The nuclear transfer ES cells are much more similar to real ES cells than the iPS cells. They are more completely reprogrammed and have fewer alterations in gene expression and DNA methylation levels that are attributable to the reprogramming process itself.”</blockquote>
<p>iPS cell technology, which was pioneered in 2006 by Shinya Yamanaka, offers a series of advantages over traditional ES cells. As Laurent continued:</p>
<blockquote>“The ability to make personalized iPS cells from a patient that could be transplanted back into that patient has generated excitement because it would eliminate the need for immunosuppression.”</blockquote>
<p>iPS cells have generated so much excitement, in fact, that Yamanaka was awarded the 2012 Nobel Prize in Physiology or Medicine for developing this technique.</p>
<p>The SCNT method was developed more recently by OHSU’s Dr. Shoukhrat Mitalipov. The current researchers generated lines of cells using both methods. After confirming that each line was, in fact, pluripotent, they used advanced genomics techniques to examine the biochemical process called ‘DNA methylation’ in each line.</p>
<p>DNA methylation is a fundamental chemical process within each cell. It’s responsible for switching key genes on and off at precise intervals. In recent years, researchers have discovered that the order and timing of this process is vital for the correct development of the cell. As Dr. Joseph Ecker, co-senior author from the Salk Institute, explained:</p>
<blockquote>“If you believe that gene expression and DNA methylation are important, which we do, the closer you get to the patterns of embryonic cells, the better. Right now, nuclear transfer cells look closer to the embryonic stem cells than do the iPS cells.”</blockquote>
<p>However, while the scientists confirmed that SCNT cells more closely resemble ES cells, the process of producing them is far from ideal. First, the SCNT method is technically difficult. And second, federal funds still cannot be used in this procedure—representing a significant hurdle to being widely adopted.</p>
<p>On the other hand, iPS cell generation is, by comparison, a much easier process technically. So perhaps these findings can spur the development of an improved method, taking the technological ease of iPS cell generation and marrying it with the accuracy of the SCNT method. Laurent argues that this could yield a new and improved approach:</p>
<blockquote>“Our results have shown that widely used iPS cell reprogramming methods make cells that are similar to standard ES cells in broad strokes, but there are important differences when you look really closely. By using the egg cell to do the job, we can get much closer to the real thing. If we can figure out what factors in the egg drive the reprogramming process, maybe we can design a better iPS cell reprogramming method.”</blockquote>
<p>Anne Holden</p>
[Image Credit: Mark Ellisman and Thomas Deerinck, National Center for Microscopy and Imaging Research, UC San Diego]
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/07099628927281143255noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-3040016818817882972014-07-01T13:06:00.001-07:002014-07-01T13:06:47.651-07:00No Fear of Rejection? Partial Stem Cell Transplant Reverses Sickle Cell Disease—even without Immunosuppressant Drugs<div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjIEtA3a7UfX_BOKxXefxUDggildZFgyIDAmsdb4IZS6siqqGfTMO5hXZ4Ia6BlC-m9fQ4pJX9jbRmYojF_swJrx_i3o1SOkSvu9-WQ55JHXnus_QsmqYjmYPQlMmGf9ZOJYbhwAYywoQ7n/s1600/75505.png" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjIEtA3a7UfX_BOKxXefxUDggildZFgyIDAmsdb4IZS6siqqGfTMO5hXZ4Ia6BlC-m9fQ4pJX9jbRmYojF_swJrx_i3o1SOkSvu9-WQ55JHXnus_QsmqYjmYPQlMmGf9ZOJYbhwAYywoQ7n/s320/75505.png" /></a></div>
<i>Magnified blood sample of a patient with severe sickle cell disease.</i>
<p>For those who suffer from the blood disorder <a href="http://www.cirm.ca.gov/our-progress/sickle-cell-anemia-fact-sheet">sickle cell disease</a>, there is really only one cure: a full bone marrow transplant followed by a lifetime of anti-rejection, immune-suppressing drugs. But now, researchers from the National Institutes of Health are testing an attractive alternative for the sickest patients.</p>
<p>Sickle cell disease gets its name from a single genetic change, or mutation, that alters the shape of one’s red blood cells.. Unlike the round cells that can pass easily through the body’s blood vessels, the sickle-shaped cells clump together, clogging up blood vessels. This leads to a lifetime of severe joint pain and, in many cases, organ damage and stroke. In this country it affects primarily African Americans.</p>
<p>The only cure is a bone marrow transplant, in which the patient’s own bone marrow is first depleted with chemotherapy, and replaced by the donor marrow. The patient then faces a lifetime of immunosuppressant, anti-rejection medication to prevent deadly rejection or graft-versus-host disease, a potentially fatal condition where the donor cells attack the recipient’s immune system.</p>
<p>But what if, instead of replacing the entirety of the patient’s bone marrow, doctors only replaced some of it? Would this mix of sickle and non-sickle-shaped cells be enough to reverse the symptoms? A clinical trial published today from the NIH research team in the <i>Journal of the American Medical Association</i> has some encouraging results.</p>
<p>As lead author Dr. Matthew Hsieh noted in <a href="http://www.eurekalert.org/pub_releases/2014-07/niod-asa062714.php">today’s press release</a>:</p>
<blockquote>“Typically, stem-cell recipients must take immunosuppressants all their lives. That the patients who discontinued this medication were able to do so safely points to the stability of the partial transplant regimen.”</blockquote>
<p>In this study, the researchers performed partial bone marrow transplantations on 30 adults with severe sickle cell disease. After one year, they took 15 patients off the standard regimen of immunosuppressant drugs. And more than three years later, those 15 patients remain free from rejection.</p>
<p>These results are promising, in that a lifetime of immunosuppressants comes with its own set of negative side effects for the patient. According to the paper’s senior author Dr. John Tinsdale:</p>
<blockquote>“Side effects caused by immunosuppressants can endanger patients already weakened by years of organ damage from sickle cell disease. Not having to permanently rely on this medication…means that even older patients and those with severe sickle cell disease may be able to reverse their condition.”</blockquote>
<p>Indeed, the research team found that even a partial transplant—which resulted in a stable mix of both red blood cell types from donor and recipient - was sufficient to reverse the disease’s debilitating symptoms.</p>
<p>The results from this trial open the door to treating patients whose immune systems are already too weak—and are unable to tolerate the negative effects of a full stem cell transplant.</p>
<p>But even this half transplant has the risks associated with donor marrow. That is why CIRM is funding a team using a patient’s own stem cells and genetically modifying them to produce the correct version of the mutated protein. These self-transplants would be safer and open up the therapy to all patients regardless of their ability to find an immunologically matching donor. We expect a clinical trial with this approach to begin soon.</p>
<p>Want to know more about how CIRM-funded scientists are working toward this goal? Check out our “<a href="http://www.cirm.ca.gov/our-progress/stories-hope-sickle-cell-disease">Spotlight on Sickle Cell Disease</a>.”</p>
Anne Holden
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/07099628927281143255noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-15092522456453531372014-06-30T12:26:00.000-07:002014-06-30T12:26:30.962-07:00Precious Cargo: Scientists Hijack Red Blood Cells to Serve as Potential Therapeutic Delivery System<div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEh0-hT2qsjUgQhn0uyxNiX8AvHwtleAzIKFGOIKbgGR6JEf2u1YmMpUPV3On4bC174AmTo-EQyGu8rKr1RIQyQBrQBdvyYsZ6NyUiKIxtk48FO-2Ky8CbjaUKKOpaw3Pju3HlpPO2a-j3Gw/s1600/shutterstock_173237540.jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEh0-hT2qsjUgQhn0uyxNiX8AvHwtleAzIKFGOIKbgGR6JEf2u1YmMpUPV3On4bC174AmTo-EQyGu8rKr1RIQyQBrQBdvyYsZ6NyUiKIxtk48FO-2Ky8CbjaUKKOpaw3Pju3HlpPO2a-j3Gw/s320/shutterstock_173237540.jpg" /></a></div>
<p>A unique property of red blood cells is now being harnessed to help deliver microscopic cargo to sites throughout the body, according to research published today in the Proceedings of the National Academy of Sciences.</p>
<p>There are anywhere from 3 to 6 million red blood cells in the human body at any given time, and they are tasked with one main job: transport oxygen throughout the body. But researchers, led by Drs. Harvey Lodish and Hidde Ploegh from the Whitehead Institute, wondered if these cells could transport other important molecules. As Lodish explained in <a href="http://wi.mit.edu/news/archive/2014/engineered-red-blood-cells-could-carry-precious-therapeutic-cargo">today’s news release</a>:</p>
<blockquote>“We wanted to create high-value red blood cells that do more than simply carry oxygen. Here we’ve laid out the technology to make mouse and human red blood cells that…can potentially be used for therapeutic purposes.”</blockquote>
<p>Red blood cells are unusual in that, once mature, they ditch their nucleus—and the DNA housed within. This is an attractive characteristic for a potential therapy: without any genetic material, there is no risk that manipulating the DNA could result in later tumor formation.</p>
<p>So Lodish, an expert in the biology of red blood cells, and his team used this characteristic to their advantage. They introduced a set of genes into early stage red blood cells, called ‘progenitors,’ that still had their nucleus. These genes, when activated instructed the cell to produce a particular type of protein that latched itself to the surface of the cell. Then, when the cells matured and jettisoned their nuclei, the proteins remained on the cells’ surface.</p>
<p>And while this method, called ‘sortagging,’ here involved a protein sticking to the cellular surface, the researchers argue that the same method could be applied to stick virtually any type of molecule to the cell. As Ploegh explained:</p>
<blockquote>“Because the modified human red blood cells can circulate in the body for up to four months, one could envision a scenario in which the cells are used to introduce antibodies that neutralize a toxin. The result would be long-lasting reserves of antitoxin antibodies.”</blockquote>
<p>The research team envisions this approach being useful for everything from carrying proteins to break up blood clots to those that alleviate chronic inflammation. One of the most exciting possibilities, according to Ploegh, would be using this method to suppress the body’s unwanted immune response after being treated with protein-based therapies.</p>
<p>The possibilities, it would seem, are endless.</p>
Anne Holden
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/07099628927281143255noreply@blogger.com1tag:blogger.com,1999:blog-6189004028142085438.post-61456972547391847872014-06-27T13:00:00.000-07:002014-06-27T13:00:58.085-07:00Stem cell stories that caught our eye: fingering chemical cancer cause, treating leukemia and getting better ID on cells<i>Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun. <br/><br/></i>
<b>Stem cells model environmental damage.</b> Using human embryonic stem cells to generate prostate tissue in mice, a team at the University of Illinois has shown how the endocrine-disrupting chemical BPA can lead to prostate cancer. They implanted the cells, derived from human stem cells, along with supportive rodent cells in mice and then fed the mice a diet with low levels of BPA. As the cells matured into prostate tissue in the animals the chemical seemed to reprogram the cells in a manner that raises the risk for cancer. The team reported its work at the annual meeting of the Endocrine Society in Chicago this week and the association’s press release was picked up by <i><a href="http://www.bio-medicine.org/medicine-news-1/BPA-exposure-during-fetal-development-raises-risk-of-precancerous-prostate-lesions-127145-1/">Bio-Medicine</a></i>. <br/><br/>
<b>Protecting stem cell “home” may be new therapy</b>. A Spanish team has discovered that certain rare <a href="http://www.cirm.ca.gov/our-progress/leukemia-fact-sheet">leukemias</a>, known as myeloproliferative disorders, seem to be triggered by damage to the area where stem cells hang out in our bone marrow known as the stem cell niche. Normally the niche controls the behavior of blood-forming stem cells but when it gets inflamed that control breaks down the team reported in the journal Nature. But there is good news, they tested a currently available drug in an animal model and it seemed to reverse the damage to the niche and return normal stem cell controls. <i><a href="http://www.sciencedaily.com/releases/2014/06/140622142236.htm">ScienceDaily</a></i> ran a story about the work. <br/><br/>
Clever trick could make stem cell frequent fliers. Researchers often share stem cells with colleagues around the world by freezing them first. But in some uses, it would be better if the cells could be shipped in living cultures. That usually requires some sort of electrical motor to agitate the vials to keep the cells from clumping, but airlines don’t allow running electric motors in cargo on planes. So a group of engineering students at the University of California, San Diego, has taken cues from old pendulum clocks and developed a spring powered motor that can keep the cells agitated. As our field gets ready to commercialize cell products, there may be times this could help centralize mass production of cells for shipping. <i><a href="http://www.mdtmag.com/news/2014/06/clock-inspired-electronics-free-device-allows-transport-stem-cells-planes">Medical Design Technology</a></i> explained the students’ project. <br/><br/>
Getting a better ID on the “other” bone marrow stem cell. Most people know that our bone marrow has blood-forming stem cells, but it also has mesenchymal stem cells (MSCs). Those cells can form bone, cartilage and fat and release proteins that seem to direct the work of other stem cells. Those skills have led to more than 200 clinical trials investigating the potential of MSCs to treat many diseases. But those trials have often produced results that are hard to interpret, and many researchers in the field say part of the problem comes from our inability to accurately identify true MSCs resulting in most clinical trials using a mixed population of cells. Now one of the leaders in the field, Sean Morrison at the University of Texas Southwest Medical Center, reports that his team has found <a href="http://www.healthcanal.com/medical-breakthroughs/52270-children%E2%80%99s-research-institute-finds-key-to-identifying%2C-enriching-mesenchymal-stem-cells.html">a reliable marker for MSCs</a> known as leptin receptor. His current work was in mice, but if it can be duplicated with human cells, it could increase the chances for valuable data coming from MSC trials. <br/><br/>
Don Gibbons
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/07099628927281143255noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-41663001698255656752014-06-27T11:13:00.000-07:002014-07-15T11:14:05.009-07:00Fingering chemical cancer cause, treating leukemia and getting better ID on cells: Stem cell stories that caught our eye<i>Here are some stem cell stories that caught our eye this past
week. Some are groundbreaking science, others are of personal interest
to us, and still others are just fun. <br /></i>
<b>Stem cells model environmental damage.</b> Using human embryonic stem
cells to generate prostate tissue in mice, a team at the University of
Illinois has shown how the endocrine-disrupting chemical BPA can lead to
prostate cancer. They implanted the cells, derived from human stem
cells, along with supportive rodent cells in mice and then fed the mice a
diet with low levels of BPA. As the cells matured into prostate tissue
in the animals the chemical seemed to reprogram the cells in a manner
that raises the risk for cancer. The team reported its work at the
annual meeting of the Endocrine Society in Chicago this week and the
association’s press release was picked up by <i><a href="http://www.bio-medicine.org/medicine-news-1/BPA-exposure-during-fetal-development-raises-risk-of-precancerous-prostate-lesions-127145-1/">Bio-Medicine</a></i>. <br />
<br />
<b>Protecting stem cell “home” may be new therapy</b>. A Spanish team has discovered that certain rare <a href="http://www.cirm.ca.gov/our-progress/leukemia-fact-sheet">leukemias</a>,
known as myeloproliferative disorders, seem to be triggered by damage
to the area where stem cells hang out in our bone marrow known as the
stem cell niche. Normally the niche controls the behavior of
blood-forming stem cells but when it gets inflamed that control breaks
down the team reported in the journal Nature. But there is good news,
they tested a currently available drug in an animal model and it seemed
to reverse the damage to the niche and return normal stem cell controls.
<i><a href="http://www.sciencedaily.com/releases/2014/06/140622142236.htm">ScienceDaily</a></i> ran a story about the work. <br />
<br />
Clever
trick could make stem cell frequent fliers. Researchers often share
stem cells with colleagues around the world by freezing them first. But
in some uses, it would be better if the cells could be shipped in living
cultures. That usually requires some sort of electrical motor to
agitate the vials to keep the cells from clumping, but airlines don’t
allow running electric motors in cargo on planes. So a group of
engineering students at the University of California, San Diego, has
taken cues from old pendulum clocks and developed a spring powered motor
that can keep the cells agitated. As our field gets ready to
commercialize cell products, there may be times this could help
centralize mass production of cells for shipping. <i><a href="http://www.mdtmag.com/news/2014/06/clock-inspired-electronics-free-device-allows-transport-stem-cells-planes">Medical Design Technology</a></i> explained the students’ project. <br />
<br />
Getting
a better ID on the “other” bone marrow stem cell. Most people know
that our bone marrow has blood-forming stem cells, but it also has
mesenchymal stem cells (MSCs). Those cells can form bone, cartilage and
fat and release proteins that seem to direct the work of other stem
cells. Those skills have led to more than 200 clinical trials
investigating the potential of MSCs to treat many diseases. But those
trials have often produced results that are hard to interpret, and many
researchers in the field say part of the problem comes from our
inability to accurately identify true MSCs resulting in most clinical
trials using a mixed population of cells. Now one of the leaders in the
field, Sean Morrison at the University of Texas Southwest Medical
Center, reports that his team has found <a href="http://www.healthcanal.com/medical-breakthroughs/52270-children%E2%80%99s-research-institute-finds-key-to-identifying%2C-enriching-mesenchymal-stem-cells.html">a reliable marker for MSCs</a>
known as leptin receptor. His current work was in mice, but if it can
be duplicated with human cells, it could increase the chances for
valuable data coming from MSC trials. <br />
<br />
Don Gibbons
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>niccoloxhttp://www.blogger.com/profile/05636854896244615674noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-48818607884103480842014-06-26T08:52:00.000-07:002014-06-26T08:52:06.466-07:00BIO International panel showed stem cell science poised to make a difference in medical practice soon<div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhTJxs9z8bN9tYvrj_XI78YIlieWSSXUd2BGpw9I9idhO_0jWXdAmy6tJ_6D1pdQ-q71WoaddOeqk7I8Bhid0dJxSgmaazX7nO0PdXO6iY5RDYoFVkRnmAVyI5kbxHMyuCXct_9YwSaPm4i/s1600/995548_10151801308142804_405229409_n.png" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhTJxs9z8bN9tYvrj_XI78YIlieWSSXUd2BGpw9I9idhO_0jWXdAmy6tJ_6D1pdQ-q71WoaddOeqk7I8Bhid0dJxSgmaazX7nO0PdXO6iY5RDYoFVkRnmAVyI5kbxHMyuCXct_9YwSaPm4i/s320/995548_10151801308142804_405229409_n.png" /></a></div>
<p>When the biotechnology trade association began holding annual conferences in 1993, they drew 1,400 to the first event. This year <a href="http://convention.bio.org/">BIO International</a> expected nearly 20,000 here in San Diego. Among the dozens of concurrent sessions each day of this four-day scramble, <a href="http://mybio.org/event/member/105171">stem cells</a> got one track on one day this year. But listening to the progress being made by our presenters yesterday, our field is set to grow at the pace this meeting has—and could dominate the medical sessions here within the next decade.</p>
<p>After setting the scene with <a href="http://cirmresearch.blogspot.com/2014/06/at-bio2014-stem-cells-shine-as-path-for.html">our opening panel</a> yesterday, four subsequent panels confirmed the vast near-term potential painted by the opening speakers. They revealed a field maturing rapidly and starting to be a valued research tool of the bigger companies that have dominated the biotech industry, at the same time it is starting to deliver therapies to patients.</p>
<p>The second panel displayed the robust power of stem cells to model disease better than animal models ever could. These cells also let researchers dive much deeper into the genetic causes of disease, particularly diseases with multiple genes involved. Anne Bang from the Sanford-Burnham Institute mentioned her role in a consortium organized by the National Institutes of Health that is looking at the many genes involved in a type of heart weakening called left ventricular hypertrophy. Because different ethnicities tend to respond differently to drugs used for the condition, the consortium teams are creating iPS-type stem cell lines from 125 Caucasian patients and 125 African-American patients with various forms of the condition.</p>
<p>Their goal is to personalize and improve therapy across both patients groups. The way cells behave in the lab can tell the researchers much more relevant information than most animal models, so drugs developed based off their discoveries should have a better chance of success. All four panelists agreed that the field needs enough drugs developed with these tools to show that they do indeed have a better success rate. That track record should start to develop over the next few years.</p>
<p>The third panel talked about the shift in the medical mindset that will happen when genetically modified stem cells can change the care of chronic diseases from daily therapy to cures. Louis Bretton of Calimmune discussed how his company is trying to do this for HIV, which <a href="http://cirmresearch.blogspot.com/2014/06/innovative-stem-cell-therapy-for-hiv.html">we blogged about yesterday</a> when they announced promising first phase results from their first four patients. Faraz Ali of bluebird bio showed that his company has already made this life-changing shift for two patients with the blood disorder Beta Thalassemia. Like most patients with the disease they had been dependent on regular transfusions to survive, but when they received transplants of their own stem cells genetically modified to produce the correct version of a protein that is defective in the disease, they were able to live without transfusions.</p>
<p>The fourth panel provided proof that the field is maturing in that they discussed the many hurdles and pitfalls in taking those final steps to prepare a cell therapy to be a commercial product. The three big hurdles—financing, regulatory approval and reimbursement by insurers—all required creativity by the companies outlined in the two case studies. They are working through them but it is anything but a straightforward path. This is the area I hear the most hand wringing about in the halls of meetings in our field.</p>
<p>The last panel showed that one way around some of those end stage hurdles is to reach across borders. Four panelists discussed specific examples of ways international collaborations have accelerated their work toward developing therapies. CIRM has more than <a href="http://www.cirm.ca.gov/our-funding/stem-cell-research-collaborative-funding-agreements">20 collaborative agreements</a> with funding agencies around the world, many of them painstakingly nurtured by our former president Alan Trounson. He gave the final presentation of the panel talking about one of his new projects, building an international stem cell bank with enough cell lines that almost everyone could get donor cells that were immunologically matched.</p>
<p>Our board chair, Jonathan Thomas, moderated the last panel and ended with a tribute to Alan noting that his build-out of our international program would be one of his many lasting legacies.</p>
Don Gibbons
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/07099628927281143255noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-77211376251697787692014-06-25T14:32:00.001-07:002014-06-25T14:32:48.950-07:00At BIO2014 stem cells shine as path for changing how medicine is researched and practiced<div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhBGjvaL6cr6g7MCBPpcDp2jLbsKo2Z_9oxmDXUbkO992HtJLFMFDiaWDiJ2pQ3NX0daKNvwAT1bKFbMyoQ0CjYlq3lfWqeCU1vMHfd6VN817wIjIlhprh8W5nsYissjuFGwjqJF3Ctj-OZ/s1600/BIO+panel+3.jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhBGjvaL6cr6g7MCBPpcDp2jLbsKo2Z_9oxmDXUbkO992HtJLFMFDiaWDiJ2pQ3NX0daKNvwAT1bKFbMyoQ0CjYlq3lfWqeCU1vMHfd6VN817wIjIlhprh8W5nsYissjuFGwjqJF3Ctj-OZ/s320/BIO+panel+3.jpg" /></a></div>
<center><i>Panelists speak at "Regenerative Medicine: Propelling a Paradigm Shift in Medicine and Healthcare Delivery"</i></center>
<p>For the first time ever at <a href="http://convention.bio.org/2014/">BIO International</a>, the largest annual biotechnology conference in North America, stem cells and regenerative medicine are being showcased today in an all-day forum. This morning kicked off with <a href="http://mybio.org/event/member/105608">a panel</a> discussing how stem cells are shifting the paradigm in how we research disease, discover drugs and treat patients.</p>
<p>With a nearly full room, former CIRM president Alan Trounson led four panelists through a lively discussion of how stem cells are accelerating the discovery of the fundamental mechanisms of disease, while also helping to make drug discovery a much more targeted process and, most importantly, beginning to deliver life-changing therapies to patients.</p>
<p><a href="http://www.cirm.ca.gov/our-progress/people/catriona-jamieson">Catriona Jamieson</a>, a CIRM grantee from the University of California, San Diego, showed how she had uncovered how cancer cells develop stem cell-like properties in order to evade treatment. She has, in turn, found three molecular pathways the cells use to make this transformation and has drugs being tested to block those pathways. But not everyone responds to those various drugs the same way, so her goal is to analyze the genetics of each patient and deliver what she has dubbed “precision regenerative medicine.”</p>
<p>Next, Eric Michael David from <a href="http://www.organovo.com/">Organovo</a> talked about the company’s ability to 3-D print small portions of liver or kidney tissue and have it function like the real thing. The long-term goal is to create tissue of sufficient quality to implant in patients, but until that is perfected their system is being widely used to screen potential drugs to see how they affect the various tissues. Understanding this potential toxicity early stands to save drug companies millions—and potentially billions—of dollars by not taking drugs destined to fail into costly phase 3 large-scale clinical trials. Or, even worse, find out about toxicity through surveillance after the drug is on the market with the associated product liability costs.</p>
<p>David said that between 1990 and 2010 160 drugs failed in phase 3 or post marketing, and unfortunately all the drugs in a company’s development pipeline share the cost of those failures. This gives new stem cell technologies the ability to significantly drive down drug costs.</p>
<p>Then, Donna Skerret from <a href="http://www.mesoblast.com/">Mesoblast</a>, which may be the largest stem cell company in the world, made it clear that while mesenchymal stem cells, which form the basis of their products have the ability to impact several diseases, we still don’t know the full mechanism for all those effects. They can reduce scar formation and moderate an overactive immune response, but the exact method of action for each still needs to be worked out. She called them drug delivery devices because they secrete various proteins that affect the cells around them. Mesoblast currently has therapies in phase 3 clinical trials for heart disease and other therapies in late stage phase 2 trials.</p>
<p>Perry Karsen of <a href="https://www.celgene.com/">Celgene</a> talked about his company’s work with placental-derived stem cells. He too discussed ongoing and much needed work to understand why they are having some of the effects they are having in order to maximize their therapeutic potential. But even now, he said, “We see cell therapies as having the ability to completely change how medicine is practiced by the end of the decade.”</p>
<p>All the panelists talked about needing to use what Karsen called “the full research ecosystem,” forming collaborations between academic researchers and industry. Donna noted that Mesoblast is relying on academic partners to help define the mechanism of action of their cells.</p>
<p>Forming these partnerships to <a href="http://www.cirm.ca.gov/our-progress/progress-toward-therapies">accelerate getting treatments to patients</a> has always been a core part of CIRM’s function.</p>
<p>An outline of all five panels of the Regenerative Medicine Forum can be seen here. We organized the day along with the Alliance for Regenerative Medicine and the International Society for Stem Cell Research.</p>
Don Gibbons
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/07099628927281143255noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-31164613494145834352014-06-25T09:58:00.000-07:002014-06-25T09:58:27.959-07:00Innovative stem cell therapy for HIV passes milestone <div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhvVE_ur_Rhzb3zDfsIjt2YzOejx8aaQbs6CKFt_OvjP9dWqGrn4Mi1qS7ORSGHN4HSqruD6fGK0tIoG-j6Z7wd3s6j8aRygIrtnmIMPUXPb32plgLdTrILs42_eJoW14To2_vE6bAi64Ap/s1600/Bio+News+%232.jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhvVE_ur_Rhzb3zDfsIjt2YzOejx8aaQbs6CKFt_OvjP9dWqGrn4Mi1qS7ORSGHN4HSqruD6fGK0tIoG-j6Z7wd3s6j8aRygIrtnmIMPUXPb32plgLdTrILs42_eJoW14To2_vE6bAi64Ap/s320/Bio+News+%232.jpg" /></a></div>
<br />
<i>
Left to Right: CIRM President and CEO C. Randal Mills, Calimmune CEO Louis Breton, Calimmume Chief Scientific Officer Geoff Symonds</i>
<br />
<p>Milestones are useful things. They measure how far we have come on a journey, and give us a sense that we are on the right path. One of the projects we are helping fund just passed a big milestone, and it’s given the researchers the go-ahead to move on to the next, perhaps even more important stage.</p>
<p>The project is Calimmune’s stem cell gene modification study, which takes blood stem cells from people who are HIV-positive, genetically modifies them so they carry a gene that blocks the AIDS virus from infecting cells, and then re-introduces the modified cells to the patient. The hope is that those stem cells will then create a new blood system that is resistant to HIV.</p>
<p>The milestone it passed is that the Data Safety Monitoring Board (DSMB) looked at the results from the first group of four patients treated with this approach, found that there were no serious adverse events or dangerous side effects from it, and gave Calimmune the go-ahead to start treating the next group of patients.</p>
<p>In a news release we put out jointly with Calimmune, their CEO Louis Breton said this is a big step forward for them:</p>
<blockquote>“We are very excited and encouraged by this development. This recommendation from the DSMB is an important step in bringing this one-time therapy to the patients, and takes us closer to our ultimate goal of eradicating AIDS.”</blockquote>
<p>It’s a pretty big deal for us too, as our President and CEO C. Randal Mills noted in the same release:</p>
<blockquote>“The mission of CIRM is to efficiently accelerate the development of stem cell treatments for patients suffering from unmet medical conditions. While still early in clinical development this announcement demonstrates real progress towards this mission. The accomplishments of Calimmune’s team is a great example of how CIRM partnerships are working to impact patient’s lives today.”</blockquote>
<p>Now, just treating four people might not seem particularly impressive, after all HIV/AIDS has killed more than 25 million people worldwide and has infected another 25 million more – around 1.1 million here in the U.S. But every treatment has to begin with a simple premise, that whatever you do is not going to hurt the patient. Getting the green light from the Data Safety Monitoring Board, an independent panel of experts who review data and advise the researchers doing clinical trials, shows this approach appears to be safe.</p>
<p>The next step is to repeat this same process in 3 or 4 more patients but to give those patients a preconditioning regimen, treating them with a medication before returning their modified stem cells to them, to try and make the therapy more effective. This could show that the therapeutic approach, called Cal-1, is not only safe but also is working to protect patients against HIV.</p>
<p>If the safety data from that second group also looks good, then Calimmune can move on to the next group of patients. Each step, no matter how small, moves us ever closer to our end goal of developing a cure for HIV/AIDS.</p>
<p>That’s still a very distant goal right now, but with each milestone we pass it shows that we are heading in the right direction.</p>
<p>Want to know more about Calimmune's path towards clinical trial? Check out Calimmune CEO Louis Breton's <a href="http://www.cirm.ca.gov/our-progress/video/hivaids-stem-cell-clinical-trial-louis-breton-ceo-calimmune">recent video describing their progress towards a cure for HIV</a>.</p>
Kevin McCormack<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/07099628927281143255noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-64415752045110558982014-06-24T09:28:00.000-07:002014-06-24T09:28:17.967-07:00When hope runs up against reality: balancing patient optimism with medical evidence<div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjyJeEjI-qIO2BJLxXpR37kv149iZTB8B4zXR3HsnYnSVUU3AohTVLpKSqqqr-cM98n1P6A1K6xhYyJXMbpePlFQs6WqRYPR2lJOR_jLWr7op0WafT9_nBL8jO6WGi-qtiRhi01CSLgtU3A/s1600/shutterstock_126288149.jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjyJeEjI-qIO2BJLxXpR37kv149iZTB8B4zXR3HsnYnSVUU3AohTVLpKSqqqr-cM98n1P6A1K6xhYyJXMbpePlFQs6WqRYPR2lJOR_jLWr7op0WafT9_nBL8jO6WGi-qtiRhi01CSLgtU3A/s320/shutterstock_126288149.jpg" /></a></div>
One of the big concerns among scientists - including many at the <a href="http://www.isscr.org/home/2014annualmeeting">International Society for Stem Cell Research (ISSCR) conference</a> in Vancouver, Canada – is that patient expectations about stem cells are often greater than researchers are able to deliver today. That can result in patients in search of a cure heading to overseas clinics that offer unproven therapies.
<p>Megan Munsie - head of the Education, Ethics, Law and Community Awareness Unit at the University of Melbourne in Australia – wanted to find out what happens when patients’ hopes for new treatments come into conflict with scientific views on medical evidence. So she started with a small survey of 16 Australians, patients and patient-caretakers, who had travelled outside Australia for stem cell treatments for a variety of diseases including MS and cerebral palsy.</p>
<p>She says there were a number of interesting findings:</p>
<ul><li>They all considered themselves pro-active and well-informed</li>
<li>They rejected advice from their own doctor but instead relied on the overseas doctor selling them the treatment for advice</li>
<li>They felt they had no choice but to travel overseas because they were running out of time and options in Australia</li>
<li>They didn’t consider the health risks, believing that the worst that would happen is that the “treatment” wouldn’t work and they would have spent a lot of money for nothing</li></ul>
<p>Perhaps the most surprising finding was that all of them talked about the “benefits” they gained from going abroad for the treatment, that it gave them a sense of hope even if there was no evidence of medical benefit.</p>
<p>This led to a bigger study where Munsie surveyed patients and patient advocates but also stem cell scientists and physicians. Not surprisingly the researchers had a very different view of the subject than the patients.</p>
<p>Researchers/doctors said they felt that patients don’t understand science and don’t appreciate the subtleties of clinical trials</p>
<ul><li>They said patients were basing their decisions not on science but desperation</li>
<li>They considered overseas providers as dubious, selling hope and taking advantage of a vulnerable patient population</li></ul>
<p>What was interesting, however, is that many doctors said they didn’t try to persuade their patients not to go, instead they chose to respect their autonomy but did at least try to give them the facts so that they could make a decision based on knowledge not ignorance.</p>
<p>When asked why they didn’t tell patients not to go, they said they respected the patients’ need for hope and didn’t want to take that away from them because they had nothing they could offer to replace it.</p>
<p>Munsie says recently some doctors have started offering these kinds of unproven therapies in Australia. She talked to four of them asking how they could justify it. All four said there is a huge unmet medical need and it was better to offer these therapies in Australia than have patients travel to other countries for them. They also said that they felt competent to provide treatment because they had undergone some kind of training or had a license to use equipment needed for the therapy.</p>
<p>Ironically while they all considered themselves legitimate providers of a needed medical therapy – albeit an unproven one – and only interested in the science, they regarded others doing the same as “cowboys” and only interested in the money.</p>
<p>When asked if they would support more regulation of the kinds of therapies they were already offering they said yes, saying that the other doctors who claimed they were “self-regulating” is like “giving the keys to the asylum to the lunatics.”</p>
<p>Munsie says it’s clear that it’s not just patients who could benefit from some guidance on expectations about stem cell therapies.</p>
<p>She says we need to do a better job of managing patient expectations without robbing them of a sense of hope, perhaps by offering them information that is more tailored to their particular needs.</p>
<p>We also need to manage what she called “the unbridled enthusiasm of providers” who are offering speculative treatments as “medical practice”. That might take regulatory change by the government.</p>
<p>She says it’s difficult to strike a balance between hope and scientific evidence, in maintaining a patient’s sense of optimism while acknowledging the reality of the science and the risks posed by unproven treatments.</p>
Kevin McCormack
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/07099628927281143255noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-44251949492225817132014-06-20T15:02:00.000-07:002014-06-23T09:01:46.811-07:00Stem Cell Stories that Caught our Eye: Speeding Stroke Recovery, HIV Clinical Trial, New Method for Growing Heart Cells<i><p>Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun.</p></i>
<p><b>Transplanting cells to speed stroke recovery</b>. Stroke remains one of the most common forms of death and disability, yet utilization of therapies that can break down the blood clots that cause most forms of stroke lags; these therapies are only effective when used within 3 to 4 hours of the stroke but most patients arrive at the hospital too late. Now scientists from Shanghai Jiao Tong University may have a different solution that can repair damage already done.</p>
<p>Scientists have recently been looking to stem cell transplantation as a way to restore blood vessels or brain tissue destroyed by a stroke, but early experiments revealed limited effectiveness. In this study, which was published this week in <i><a href="http://www.cell.com/stem-cell-reports/abstract/S2213-6711%2814%2900153-2">Stem Cell Reports</a></i>, the researchers coaxed embryonic stem cells further along in the development process before implanting them—which appears to have done the trick.</p>
<p>Using animal models, the team—led by Dr. Wei-Qiang Gao—transplanted two different types of so-called ‘precursor cells’ which have the ability to turn into the major types of brain and blood-vessel cells, the types of cells that are lost during a stroke.</p>
<p>Gao argues that this kind of transplantation is superior to previous methods because the two types of precursor cells can actually support each other in order to promote cell growth, and thus lays the foundation for new <a href="http://www.eurekalert.org/pub_releases/2014-06/cp-sct061214.php">stem cell-based therapies</a> to speed up recovery for stroke survivors.</p>
<p><b>CIRM-Funded Clinical Trial to Treat HIV</b>. A team comprised of the City of Hope in Los Angeles, Sangamo Biosciences and the University of Southern California have developed an innovative approach to eradicating HIV.<p>
<p>With support from a <a href="http://www.cirm.ca.gov/our-progress/awards/ziinc-finger-nuclease-based-stem-cell-therapy-aids">CIRM grant</a>, the researchers are developing a combination stem cell and gene therapy approach that is based on the success of the so-called “Berlin patient,” an HIV-positive man who was essentially cured after a bone-marrow transplant to treat his leukemia. In this instance, the bone marrow donor had a unique HIV-resistant mutation. The transplant transferred this mutation to the Berlin patient, and scientists have since been looking for a way to replicate this mutation on a larger scale. As explained in this week’s <a href="breakthroughs.cityofhope.org/hiv-clinical-trial-cirm-grant">news release</a>:</p>
<blockquote>“Using an enzyme called a zinc-finger nuclease (ZFN), the research team can …“edit” the HIV patient’s stem cell genes so that, like the Berlin patient’s donor, they can no longer produce the protein. No protein, no HIV infection. The virus might then disappear from the body.<br /><br />
This study will be the first trial of ZFN technology in human stem cells. Earlier clinical studies in HIV-positive patients show that the ZFN method is generally safe when used with white blood cells called lymphocytes. And in one patient, the therapy was associated with temporary control of HIV without antiviral medication.”</blockquote>
<p>The team hopes to begin testing this approach by the fall of 2014 on HIV patients who have not responded well to traditional therapies. CIRM funds a team that uses a different approach to gene editing that began a clinical trial last summer. You can read about both on our <a href="http://www.cirm.ca.gov/our-progress/hivaids-fact-sheet">HIV fact sheet</a>.</p>
<p><b>Building a Better Heart Cell</b>. Stanford stem cell scientist Dr. Joseph Wu and his team have devised an improved method for generating large batches of heart muscle cells, known as cardiomyocytes, faster and cheaper than ever before. This new technique, described in the latest issue of <i><a href="http://www.nature.com/nmeth/journal/vaop/ncurrent/full/nmeth.2999.html">Nature Methods</a></i>, solves a long-standing problem in the field of regenerative medicine. As Wu explained in the Stanford University School of Medicine’s blog <i><a href="http://scopeblog.stanford.edu/2014/06/17/a-new-era-for-stem-cells-in-cardiac-medicine-a-simple-effective-way-to-generate-patient-specific-heart-muscle-cells/">Scope</a></i>:</p>
<blockquote>“In order to fully realize the potential of these cells in drug screening and cell therapy, it’s necessary to be able to reliably generate large numbers at low cost….[Our] system is highly reproducible, massively scalable and substantially reduces costs to allow the production of billions of cardiomyocytes.”</blockquote>
<p>This research, which was supported by a grant from CIRM, stands to improve scientists’ ability to use patient-derived cells not only to better understand how a heart becomes a heart, but also to test drugs that treat various types of heart disease.</p>
Anne Holden
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/07099628927281143255noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-64632898120413881622014-06-20T13:55:00.000-07:002014-06-20T13:55:06.299-07:00ISSCR 2014: Tony Atala, Jason Burdick and the Power of Tissue Engineering<div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjN8LAMHj_eq8dT5VAzcf_W2CJvAnS8amWsZjjM_p7FqUxK6IIFYdHOd07XRA6OXjSkEZyMOIXlbolMjAOQa8pgYDOosvx2cRDv5KHfCwFHrS8wGqaDxKCHG31aTqeGOFwC81AZEPVfl7xe/s1600/1280px-Anthony_Atala,_Printing_a_Human_Kidney_on_Stage_(5507356887).jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjN8LAMHj_eq8dT5VAzcf_W2CJvAnS8amWsZjjM_p7FqUxK6IIFYdHOd07XRA6OXjSkEZyMOIXlbolMjAOQa8pgYDOosvx2cRDv5KHfCwFHrS8wGqaDxKCHG31aTqeGOFwC81AZEPVfl7xe/s320/1280px-Anthony_Atala,_Printing_a_Human_Kidney_on_Stage_(5507356887).jpg" /></a></div>
<i><p><center>Tony Atala speaks about tissue engineering in a 2011 TED talk (credit: Wikipedia)</center></p></i>
<p>The progress in tissue engineering in just the past two decades has been like the construction industry moving from simple lean-to structures to homes with plumbing, heating and cooling systems. We are not yet ready to build a high-rise—think of a beating functioning heart—but we are making major strides toward that goal.</p>
<p>One of the founders of the field, Wake Forest’s Dr. Tony Atala, led off this morning plenary session at the annual meeting of the International Society for Stem Cell Research. He started trying to build simple organs in 1990. His talk nicely mapped his progress through four levels of complexity of structure.</p>
<p>The first level, accomplished by a few teams, was our largest organ, skin, which is relatively simple because it is flat. Next, came simple hollow organs like blood vessels and the urethra that carries urine from the bladder. He followed that with more complex hollow organs, first the bladder and more recently the vagina. Last up were complex solid organs: the heart and the penis. He expects to begin clinical trials with the latter soon, which is eagerly anticipated by our military dealing with the aftermath IED explosive injuries from the wars in Iraq and Afghanistan.</p>
<p>He noted that researchers in the field quickly learned that just throwing cells on scaffolds and hoping they knew what to do was not enough in most cases. They need to grow blood vessels so they can get nourishment and communicate with their surroundings and they often have to make multiple cell types. His own work here benefited from a bit of geographic serendipity. His lab at the time was on the same floor as Judah Folkman’s at Harvard affiliated Children’s Hospital. Folkman is the father of the field of angiogenesis, the art of growing blood vessels.</p>
<p>Atala showed slides comparing injecting cells where you need new muscle, to cells plus scaffold, and finally to the two combined with a vessel growth factor. The three-way combo far outperformed the others. He published his first study using this technique for a hollow simple organ, the urethra, in 2011. At that point his patients had been living with the functional new organ for six years. They work and last.</p>
<p>Researchers almost always place a cell-scaffold complex in a soup of nutrients and growth factors called a bioreactor before implanting it. But at the time of implant, the organ is not mature. Atala said the body acts like a “finishing bioreactor” to fill out and strengthen the organ, which becomes fully mature around six months after implant. He showed images of this in-body growth in his first patients who had been born without a complete vagina and were given a fully functioning organ. He just published that study two months ago, eight years after the implants in order to make sure they stayed functional over time.</p>
<p>He then showed his animal model work creating a penis in rabbits. Being a highly vascular organ it required much more structure. He used a donor organ that had all its cells chemically washed away to leave just the intracellular scaffold. This structure helped guide the blood vessel growth and the rabbits succeeded in mating and having offspring.</p>
<p>His lab has begun early stage work for both liver and heart. They have created miniature livers about the size of a half dollar that are able to produce the appropriate proteins and metabolize drugs. They have used a 3-D printer to build two chambers of a heart that are able to beat in a dish, but their structure has not been stable. So, he noted much more work lies ahead for complex organs.</p>
<p>The second speaker, Jason Burdick from the University of Pennsylvania, concentrated on making better scaffolds for the stem cells, which can have three enhanced properties:</p>
<ul><li>they can be instructive, they can tell cells what to do;</li>
<li>they can be dynamic, they can react to their environment and the cells around them;</li>
<li>they can lead to heterogeneity, they can provide varied instructions so you get the different cell types that you need for a complex tissue.</li></ul>
<p>He discussed two examples, the first was growing better cartilage (as he joked, for injured World Cup soccer players). One problem with early gels used as scaffold was they held the cells individually apart from each other limiting their ability to communicate with each other. This cell-to-cell cross talk is key to tissue maturation. He showed how you could chemically alter the gel to enhance this communication. He also showed how you could implant the gels with microspheres loaded with growth factors to deliver instructions to the cells.</p>
<p>Burdick’s second example focused on minimizing injury after an induced heart attack in rodents. But instead of loading the gel with cells, they loaded it with microspheres that release chemicals that summons the stem cells waiting quietly in reservoirs in all of us. They saw sustained release of the chemicals for 21 days and significant improvement in heart function.</p>
<p>But he closed with a fun twist. The first heart experiment used a strict time-release formulation. He said it would be much better if the chemicals were released at the points the heart needs it the most. So, he is working on a system that releases the chemical based on the levels of an enzyme the heart makes when it is injured. He is hoping this right-amount-at-the-right-time formula will be even better.</p>
<p>We have a short video of the highlights of <a href="http://www.cirm.ca.gov/our-progress/video/tissue-engineering-and-stem-cell-research-cirm-workshop">a workshop we held on tissue engineering</a> that you can watch to get a better feel for where the field is going.</p>
Don Gibbons
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/07099628927281143255noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-70778417452978496382014-06-20T11:52:00.000-07:002014-07-10T11:55:48.432-07:00Speeding Stroke Recovery, HIV Clinical Trial, New Method for Growing Heart Cells<i>Here are some stem cell stories that caught our eye
this past week. Some are groundbreaking science, others are of personal
interest to us, and still others are just fun.</i><br />
<b>Transplanting cells to speed stroke recovery</b>. Stroke
remains one of the most common forms of death and disability, yet
utilization of therapies that can break down the blood clots that cause
most forms of stroke lags; these therapies are only effective when used
within 3 to 4 hours of the stroke but most patients arrive at the
hospital too late. Now scientists from Shanghai Jiao Tong University may
have a different solution that can repair damage already done.<br />
Scientists
have recently been looking to stem cell transplantation as a way to
restore blood vessels or brain tissue destroyed by a stroke, but early
experiments revealed limited effectiveness. In this study, which was
published this week in <i><a href="http://www.cell.com/stem-cell-reports/abstract/S2213-6711%2814%2900153-2">Stem Cell Reports</a></i>,
the researchers coaxed embryonic stem cells further along in the
development process before implanting them—which appears to have done
the trick.<br />
Using animal models, the team—led by Dr. Wei-Qiang
Gao—transplanted two different types of so-called ‘precursor cells’
which have the ability to turn into the major types of brain and
blood-vessel cells, the types of cells that are lost during a stroke.<br />
Gao
argues that this kind of transplantation is superior to previous
methods because the two types of precursor cells can actually support
each other in order to promote cell growth, and thus lays the foundation
for new <a href="http://www.eurekalert.org/pub_releases/2014-06/cp-sct061214.php">stem cell-based therapies</a> to speed up recovery for stroke survivors.<br />
<b>CIRM-Funded Clinical Trial to Treat HIV</b>.
A team comprised of the City of Hope in Los Angeles, Sangamo
Biosciences and the University of Southern California have developed an
innovative approach to eradicating HIV.<br />
With support from a <a href="http://www.cirm.ca.gov/our-progress/awards/ziinc-finger-nuclease-based-stem-cell-therapy-aids">CIRM grant</a>,
the researchers are developing a combination stem cell and gene therapy
approach that is based on the success of the so-called “Berlin
patient,” an HIV-positive man who was essentially cured after a
bone-marrow transplant to treat his leukemia. In this instance, the bone
marrow donor had a unique HIV-resistant mutation. The transplant
transferred this mutation to the Berlin patient, and scientists have
since been looking for a way to replicate this mutation on a larger
scale. As explained in this week’s <a href="https://www.blogger.com/breakthroughs.cityofhope.org/hiv-clinical-trial-cirm-grant">news release</a>:<br />
<blockquote>
“Using an enzyme called a zinc-finger nuclease (ZFN), the
research team can …“edit” the HIV patient’s stem cell genes so that,
like the Berlin patient’s donor, they can no longer produce the protein.
No protein, no HIV infection. The virus might then disappear from the
body.<br />
<br />
This study will be the first trial of ZFN
technology in human stem cells. Earlier clinical studies in HIV-positive
patients show that the ZFN method is generally safe when used with
white blood cells called lymphocytes. And in one patient, the therapy
was associated with temporary control of HIV without antiviral
medication.”</blockquote>
The team hopes to begin testing this approach by the fall of 2014 on
HIV patients who have not responded well to traditional therapies. CIRM
funds a team that uses a different approach to gene editing that began a
clinical trial last summer. You can read about both on our <a href="http://www.cirm.ca.gov/our-progress/hivaids-fact-sheet">HIV fact sheet</a>.<br />
<b>Building a Better Heart Cell</b>. Stanford stem cell scientist
Dr. Joseph Wu and his team have devised an improved method for
generating large batches of heart muscle cells, known as cardiomyocytes,
faster and cheaper than ever before. This new technique, described in
the latest issue of <i><a href="http://www.nature.com/nmeth/journal/vaop/ncurrent/full/nmeth.2999.html">Nature Methods</a></i>,
solves a long-standing problem in the field of regenerative medicine.
As Wu explained in the Stanford University School of Medicine’s blog <i><a href="http://scopeblog.stanford.edu/2014/06/17/a-new-era-for-stem-cells-in-cardiac-medicine-a-simple-effective-way-to-generate-patient-specific-heart-muscle-cells/">Scope</a></i>:<br />
<blockquote>
“In order to fully realize the potential of these cells
in drug screening and cell therapy, it’s necessary to be able to
reliably generate large numbers at low cost….[Our] system is highly
reproducible, massively scalable and substantially reduces costs to
allow the production of billions of cardiomyocytes.”</blockquote>
This research, which was supported by a grant from CIRM, stands to
improve scientists’ ability to use patient-derived cells not only to
better understand how a heart becomes a heart, but also to test drugs
that treat various types of heart disease.<br />
Anne Holden
<br /><div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>niccoloxhttp://www.blogger.com/profile/05636854896244615674noreply@blogger.com0tag:blogger.com,1999:blog-6189004028142085438.post-36915473827116492622014-06-20T11:47:00.001-07:002014-06-20T14:00:16.116-07:00ISSCR 2014: Lorenz Studer talks Parkinson’s cells <div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEi08_ha7XqOTdBKsupShyphenhyphenxMPa8IU0Rtz3YMAMsw7AKElYXAy3UKYTSjtWPonZD5Ul8qTIM-xNZJLMmHsrIAno_LbnqdHoQjHRBsQoJhCm9CuXte5FLv6t_ghmsonD3MfxypVcOOcHcAN_aU/s1600/shutterstock_165718637.jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEi08_ha7XqOTdBKsupShyphenhyphenxMPa8IU0Rtz3YMAMsw7AKElYXAy3UKYTSjtWPonZD5Ul8qTIM-xNZJLMmHsrIAno_LbnqdHoQjHRBsQoJhCm9CuXte5FLv6t_ghmsonD3MfxypVcOOcHcAN_aU/s320/shutterstock_165718637.jpg" /></a></div>
<p>Two presentations at the International Society for Stem Cell (ISSCR) conference, from two different sides of the pond, looked at ways to get stem cell therapies out of the lab and into patients. They both focused on the problems that need to be overcome, but came to the positive conclusion that this could be done.</p>
<p>Lorenz Studer, from the Sloan Kettering Institute for Cancer Research, has been working since 1995 to try and find a renewable source of cells to treat Parkinson’s Disease. He thinks he’s finally found it.</p>
<p>Let’s back up a little. Studer says the key movement problems seen in people with Parkinson’s (tremors, rigidity, difficulty moving) are caused by a loss of the dopamine-producing neurons in their brain. The good news is that this creates a great target for researchers to try and find a replacement. The bad news is it’s devilishly difficult producing the right kind of cell to survive and function in the brain.</p>
<p>In the 1980s fetal tissue transplants were tried to treat the disease and while these tissues seemed to engraft into the brain and have survived, in some cases, for more than 30 years, they only benefitted a small number of patients and had some unexpected side effects in others. So Studer focused his approach using dopamine-producing neurons (the kind that are destroyed by Parkinson’s disease) that derived from human embryonic stem cells (hESC).</p>
<p>He found that these hESC dopamine neurons worked well in animal models, surviving term and mirroring the normal development of a human neuron.</p>
<p>Studer says new MRI technology means we can be much more precise in where we place these cells in the brain, ensuring that they go exactly where we want them.</p>
<p>So Studer feels he has the right cells in the right number and the ability to place them in the right location. But that still left a number of questions: how do we know they are engrafting into the brain and producing dopamine, and is that producing any impact on behavior?</p>
<p>Studer turned to optogenetics, the use of light to control neurons, to assess and measure what was happening in the brain with these transplanted cells. He put markers into the neurons that were being transplanted and then used pulses of light to switch them on and off. Turning the cells off stopped the dopamine production; turning them back on increased it. They found that the cells were indeed functioning and producing the dopamine.</p>
<p>That still left the question of whether that actually changed behavior. So he devised a study comparing mice with healthy brains to those with Parkinson’s-like lesions on one side of the brain. He put the mice in a tunnel with food pellets on either side of it. The mice with a healthy brain went along the tunnel and ate food from both sides. The other mice ate food almost completely from just one side: the side opposite where the lesion in their brain was.</p>
<p>Then Studer transplanted the dopamine-producing neurons into the study mice and repeated the experiment. This time they ate from both sides of the tunnel suggesting the transplanted cells were producing dopamine, affecting behavior in a positive way.</p>
<p>He hopes to be in clinical trails in patients in late 2016 or early 2017.</p>
<p>For Roger Barker of the University of Cambridge, UK, finding the right cells was only one of four basic questions that need to be considered when trying to take stem cell therapies into clinical trials:</p>
<ol><li>What is the evidence that cell therapies work in replacement</li>
<li>Can you make an authentic, effective cell replacement</li>
<li>How can you test such therapies in patients</li>
<li>Are these competitive to existing therapies</li></ol>
<p><b>Question 1</b><br />
Baker says numerous studies in animals over the years have shown that using dopamine-producing stem cells to replace the damaged cells can increase dopamine levels.</p>
<p>A European contingent called TransEuro is about to start a clinical trial to see if this also works well in people. This consortium is using fetal tissue and will treat patients with more early stage disease when, at least in theory, it’s more likely to respond to the therapy. They hope to transplant their first patient in the next four weeks.</p>
<p><b>Question 2 </b><br />
Can you make an authentic dopamine producing neuron? Baker said Studer’s work suggests you can, as long as it is a form of the cell called an A9 NIGRAL dopaminergic neuron. Barker says even these cells are not perfect cells but they have enough qualities to suggest they are worth trying.</p>
<p><b>Question 3</b><br />
Barker says many therapies have been tested in early stage clinical trials in the past that, based on preclinical evidence, weren’t good candidates. When they failed they set the field back by creating the impression that stem cells wouldn’t work for this kind of approach when the real lesson is that stem cells may well work, but they have to be the right ones, used in the right way.</p>
<p>He says GFORCE—a consortium featuring CIRM, and groups in New York, the UK and Japan—is now working as a group to set common standards and agreed upon best practices, so future trials can be compared to each other rather than stand alone.</p>
<p>Here at the stem cell agency we have also created a <a href="http://www.cirm.ca.gov/our-funding/regenerative-medicine-consortium ">Regenerative Medicine Consortium</a> to bring together leading companies, academic and funding institutions to share best practices and resources, and to help speed up this process and make it more consistent and efficient.</p>
<p><b>Question 4</b><br />
Many existing therapies today work very well in helping control some of the symptoms, at least in the early stages. To be effective these new stem cell therapies have to be at least as good—and at least as affordable—as existing treatments. Whether that proves to be the case will determine whether, even if they show they are effective, they become widely available.</p>
<p>Both scientists acknowledge we have come a long way in recent years. Both also acknowledge we still have a long way to go. But at least now we seem to all be asking the same questions and that is a clear sign of progress.</p>
<p>At the stem cell agency <a href="http://www.cirm.ca.gov/our-progress/parkinsons-disease-fact-sheet ">we have invested more than $43 million in 23 different research projects</a> aimed at finding new treatments for Parkinson’s.</p>
Kevin McCormack
<div class="blogger-post-footer"><p></p>
<p>Read more stem cell research news from the California Institute for Regenerative Medicine by visiting our blog at <a href="http://cirmresearch.blogspot.com" target="_blank">cirmresearch.blogspot.com</a>.</p></div>Anonymoushttp://www.blogger.com/profile/07099628927281143255noreply@blogger.com0