Monday, October 31, 2011

Vampires get a taste of lab-grown blood

Good news for vampires and patients alike: Scientists in Edinburgh have generated red blood cells directly from bone marrow stem cells in the lab. This alternate source of cells could help the 4.5 million people in the U.S. who need a transfusion each year (from America's Blood Centers, which has many interesting facts about how much blood is needed and how little is donated).

Wired writes:
Professor Marc Turner, who leads the team, believes the blood could be ready for trials in as little as two years, once it's married with research producing artificial haemoglobin -- the protein responsible for transporting oxygen in the blood of vertibrates.
The team are working to produce blood type O-negative, which is good news, as that's the type that 98 percent of us can accept. Because it's essentially manufactured, it would be guaranteed to be free from any viruses and diseases, like HIV, Hepatitis and vCJD (mad cow disease).
The Telegraph quotes Turner, who led the project:
"I think it will probably be two or three years before we get to clinical trials and I would think it will be a decade or so before one sees these kinds of artificial red cells or cultured red cells in routine general practice."
- A.A.

Friday, October 28, 2011

State-of-the-Art Science and Architecture combine for a World-Class Downtown

Erin Rhoades is a professional city planner and lifelong Berkeley resident. Ms. Rhoades' planning interests are focused on infill development and sustainability. Erin is a founding Board member of Livable Berkeley and the Board Chair.

The Li Ka Shing Center for Biomedical and Health Sciences/
University of California, Berkeley

The Li Ka Shing Center for Biomedical and Health Sciences, dedicated last week, stands at the nexus of downtown Berkeley and the University of California, Berkeley campus. The center is symbolic of a vision, not just for UC Berkeley, but the community as a whole. This building is the eighth of twelve stem cell research facilities funded in part by CIRM to open its doors (read more about CIRM's major facilities).

From the standpoint of design, the building incorporates a range of environmental features. The materials and energy efficiency are state-of-the-art, including a living roof. Architecturally, the center incorporates contemporary design and finish into a space surrounded by structures of historic significance and natural green space.

These design features are thoroughly consistent with the Downtown Area Plan's (DAP) goals for LEED Gold or equivalent development with outwardly visible sustainable design features that display innovation in green architecture in the context of a downtown with a predominanlty historic character.

Robert Birgeneau,
Chancellor of UC Berkeley,
at the dedication ceremony
Inside, the center will be home to student scientists at every level of education working to tackle emerging and neglected diseases of national and international significance. The flexible and open design will facilitate collaboration among these teams.

The $257 million facility, which received $20 million from CIRM, was financed through an innovative public private partnership involving state bond funds, individual donors and foundations. This model of combining public investment with private funds serves to leverage tax dollars and accelerate benefits to taxpayers.

Innovative funding approaches, like those used to construct the Li Ka Shing Center and the other CIRM major facilities, will be required in Berkeley to achieve the magnitude of investment necessary for high-density infill development and at the same time to accomplish the affordable housing component, community benefits and streetscaping called for in the DAP.

The combination of creating state-of-the-art development, supporting the knowledge (“imagined in California”) economy through public / private financing is an important model for the City of Berkeley. As we move into the 21st century, Berkeley should build on these success stories and support development that builds on this innovative model.

Development in downtown Berkeley should look for ways to showcase the best thinking in progressive environmental design and urban culture. Buildings like the Li Ka Shing Center and the David Brower Center, including their programmatic functions, exemplify Livable Berkeley's advocacy for a "world class" downtown. Our hope is that the Downtown Area Plan will result in the next building that defines Berkeley not for where it's been, but where it's going.

Erin Rhoades

Thursday, October 27, 2011

Peyton Manning and how a political football goes flat

Jonathan Moreno is senior fellow at the Center for American Progress and the David and Lyn Silfen University Professor at the University of Pennsylvania.

In a cleverly written but factually empty blog post, Tony Perkins of the Family Research Council tries to justify his organization's opposition to human embryonic stem cell research. This time he avers to the decision of the National Football League's Peyton Manning to resort to adult stem cell treatments in Europe because, Perkins tells us, "the U.S. is giving preferential treatment to embryonic stem cell research." This assertion is simply false, and I challenge Perkins to provide the evidence. In fact, nationally far more has been spent on adult stem cell research than embryonic.

Perkins also argues that Peyton's decision to go to Europe to have some of his own fat cells injected into his neck "has impacted the debate" on adult stem cell research. I'm not sure what impact or debate he's referring to. He's absolutely right that "[i]t's too early to tell if the treatment's had any impact on Manning's neck," but he seems far more excited about the debate than whether the "treatment" actually works. At this time there is no evidence that the treatment Manning seeks is more beneficial than potentially harmful. The injection of potent cells into the body is not necessarily a benign procedure, which is why the U.S. Food and Drug Administration is being so careful about reviewing these proposals, both for adult and embryonic stem cell procedures.

Perkins operates on a transparent double standard. It's also "too early to tell" whether embryonic stem cell research will be of medical benefit, which is precisely why the work is being done, but that criterion only seems to apply when it's consistent with Perkins' real agenda. Intellectual honestly requires Perkins to acknowledge that he is opposed to research involving human embryos, regardless of potential benefit. That is a respectable position that shouldn¹t be painted over by sophistry. So is Manning's decision to seek relief where he can, but that decision should be accompanied by an awareness of the risks and the lack of evidence of benefit.

Procedures like the one Manning wants should be approached as carefully controlled research studies with full informed consent and prior review by an independent ethics board. Then perhaps we will know if future patients can expect some relief, or if they are just writing a check and exposing themselves to unwarranted risks.

What is striking about debates like that over human embryonic stem cell research is the way that they continue to be fodder for cultural division. This is something new in the role of science in America, and seems to be especially prevalent in biology. I write about the "new biopolitics" in my latest book, "The Body Politic: The Battle Over Science in America" (Bellevue Literary Press, 2011). Esoteric bioethical and scientific questions like stem cells and cloning have become part of the political process. There is no reason to think that these issues will recede from the political debate; indeed, they have already surfaced in the current presidential election cycle.

But they also create somewhat unpredictable responses from those who would normally identify themselves as on the left or right, as worries about the impressive results or at least the symbolism of experimental biology cause understandable concern about the implications of powerful new science. There are good reasons to think about these issues in fresh new ways. But the same old predictable rhetoric doesn't help us do that.

Jonathan Moreno

Wednesday, October 26, 2011

Purified heart cells from human embryonic stem cells

Earlier this week, a team from South San Fransisco-based VistaGen and Toronto's McEwen Centre published a paper in the October 23, 2011 Nature Biotechnology that could have important consequences for efforts to repair heart attack damage with stem cells. VistaGen has a CIRM Tools & Technologies award, though that award did not fund the work published this week.

The team, which was led by Gordon Keller at the University Health Network’s McEwen Centre for Regenerative Medicine in Toronto, developed a way of maturing embryonic stem cells into early heart cells and purifying them. They did this by first discovering a protien that's on the surface of early heart cells. They could then use antibodies to keep only those cells that have the protein and eliminate all cells without the protein. Creating pure populations of cells is critical for developig therapies, which need to be free of the original embryonic stem cells that can form tumors.

A press release from VistaGen described the technology:
These findings provide, for the first time, a simple method for isolating some of the earliest populations of cardiac precursors and mature cardiomyocytes from human pluripotent stem cell cultures. This readily adaptable technology offers a viable approach for generating large numbers of enriched, non-genetically modified, cardiomyocytes for numerous therapeutic applications.
Creating pure populations of cells is just the first step. Before the cells are shown to be therapeutically useful they must be able to integrate into the complex three-dimensional structure of the human heart and beat in time with the surrounding tissue.


Tuesday, October 25, 2011

The European embryonic stem cell patent ban: The Other Side of the Coin?

Geoff Lomax is CIRM's Senior Officer to the Standards Working Group 

Last week we posted on the initial views of scientists and policy analysts on the 10/18 European Court of Justice decision regarding the patentability of hESC products. The initial views were quite pessimistic with regard to hESC-based therapy development, although James Lawford-Davies did offer a few rays of hope for the field.

This week Nature published a piece suggesting there may be a "silver-lining" to the decision. Some commentators believe companies may focus on patents covering the tools and technologies as opposed to cells and their biological products. Further, the absence of a 'patent thicket" may speed therapy development by reducing the chance of infringement of intellectual property rights.

Nature quotes physician scientist Chris Mason of University College London:
"If anything the ruling is an opportunity. It's not the end of stem cells in Europe."
The piece goes on to explain that even a restrictive interpretation should allow companies to patent new and innovative technologies needed to turn human ES cells into treatments. The goal becomes patenting the innovative tools, rather than patenting the cells themselves.
"If the sum total of this market were some cell lines, I would be deeply, deeply worried," says Julian Hitchcock, a life-sciences lawyer at Field Fisher Waterhouse in London. Growth media, equipment and chemicals that help scientists to work with stem cells could all be patented in Europe without running afoul of the high court's ruling, he says.
In addition to debate over the ruling's long-term effects, there is some uncertainty over which cell types would fall under the ruling. Advanced Cell Technology, based in Marlborough, Mass., has developed a technique to create embryonic stem cell lines that does not destroy the embryo. They've issued a press release indicating that their cell lines would not be covered by the ban. They write:
”This is a huge setback for stem cell research in Europe,” said Robert Lanza, M.D., ACT's chief scientific officer. “However, the Court went out of its way to stress that inventions are unpatentable if they ˜necessitate' the destruction of human embryos. Therefore, this ruling does not appear to affect our single-blastomere technology, which does not require the destruction of embryos at any point. We will do whatever we can to ensure that therapies derived using these stem cells are made available to the patients in Europe who need them.”

Monday, October 24, 2011

Bakersfield residents learn about stem cell progress in aging, macular degeneration

This weekend CIRM hosted an educational event in Bakersfield to update people on the progress being made by CIRM grantees. The event featured a keynote address by board chair Jonathan Thomas plus talks by grantees working on age-related diseases including blindness.

Although CIRM holds board meetings throughout California, this is the first time people in Bakersfield have had a chance to hear directly from CIRM. If media attention in advance of the meeting is anything to judge by, the local community was excited about hosting us. The local radio station KERN spoke with CIRM patient advocate coordinator Chris Stiehl, who helped organize the event. You can listen to that interview here.

Among other things, Stiehl discussed the Geron spinal cord injury trial as one sign that the field of stem cell research is progressing.
"Geron Corporation is doing clinical trials with stem cells on people with spinal cord injuries. That's amazing. We never had anything for those people except wheelchairs and someday they may get out of their wheelchairs because of this."
The Bakerfield Californian also had a nice piece announcing the event.

As with all CIRM patient advocacy events, if you can't make the event you can follow the discussion on Twitter either by watching the stem cell conversation on #stemcells or by following the event's hash tag, which is listed on the agenda on the CIRM web site. There's another event coming up October 29 in Santa Rosa, which you can follow at #CIRMSantaRosa.

These types of public events featuring CIRM scientists and patient advocates are going to be ongoing throughout the state as a way of making sure the people of California get a chance to learn about progress being made by the institute.

You can get more information about all the awards CIRM has funded and which institutes have received funding on our website.


Wednesday, October 19, 2011

CIRM grantees at Gladstone Institute receive $5 million to expand stem cell program

The Gladstone Institutes in San Francisco are set to expand their stem cell research program with a $5 million gift from the Roddenberry Foundation.

The independent Institute, which is associated with the University of California, San Francisco, has received $24 million in funding from CIRM (you can see a complete list of those awards here). The 14 awards fund training new stem cell scientists, creating a lab facility where Gladstone scientists can share stem cell resources and awards to aid in creating reprogrammed stem cells (known as iPS cells) and treat cardiovascular diseases and HIV/AIDS, among others.

The Roddenberry gift will expand these existing areas of expertise among Gladstone scientists by creating the Roddenberry Center for Stem Cell Biology and Medicine - named after Gene Roddenberry, who created "Star Trek".

A story by Erin Allday at the San Francisco Chronicle quotes Rod Roddenberry, Gene Roddenberry's son:
"We don't fool ourselves into thinking we're going to cure Alzheimer's or heart disease overnight, but if they tell us they are breaking ground and moving forward, we definitely want to help them do it."

"It was amazing to go up to Gladstone and look through a microscope and see a sheet of beating heart cells, and know they came from skin cells," Roddenberry said. "Walking around the institute, we met scientists who were passionate and excited by what they were doing. And as corny as it sounds, a lot of them believed in the 'Star Trek' future, a beautiful future."
Allday also spoke with CIRM President Alan Trouson about the Gladstone Institutes:
"Gladstone is small, but it's incredibly effective, and it's ranked very high by scientists around this country as one of the best institutes."
A press release from the Gladstone Institutes about the gift quotes Deepak Srivastava, who directs both stem cell and cardiovascular research at Gladstone:
“Today's biggest challenge for solving disease is getting the investments required to transform our basic-science discoveries into health solutions that can alleviate human suffering.”
Investments like those from CIRM and now the Roddenberry Foundation should help accelerate the translation of basic discoveries by Gladstone scientists into real therapies. This video discusses work by Gladstone scientist Bruce Conklin, who is developing a stem cell method of screening drugs to treat a form of heart disease:


Tuesday, October 18, 2011

Experts View European Court Ruling as a Setback for Stem Cell Therapies

Geoff Lomax is CIRM's Senior Officer to the Standards Working Group

The European Court of Justice issued a ruling today that therapeutic products created from human embryonic stem cells are not patentable. A press release cites the court's decision:
Court holds that an invention is excluded from patentability where the implementation of the process requires either the prior destruction of human embryos or their prior use as base material, even if, in the patent application, the description of that process, as in the present case, does not refer to the use of human embryos.
James Lawford-Davies presented background on the case at the World Stem Cell Summit October 5 in Pasadena. He says the decision is seen as "a victory by those opposed to the use of embryos for research in the EU". Aurora Plomer, a legal scholar at the University of Sheffield, concurs:
"The ruling of the Court of Justice of the European Union could be in breach of the European Convention on Human Rights in failing to recognize the margin of discretion granted to Member States on the rights of human embryos."
Researchers are equally disappointed. An AP story quotes Pete Coffey, who received a CIRM Research Leadership Award to support his move from University College London to the University of California Santa Barbara. Coffey has been developing an embryonic stem cell-based approach to treating the most common form on blindness.
"This is a devastating decision which will stop stem cell therapies' use in medicine," Pete Coffey, a stem cell researcher at University College London, said in a statement. "The potential to treat disabling and life-threatening diseases using stem cells will not be realized in Europe."
The Guardian quotes Austin Smith, head of the Wellcome Trust Centre for Stem Cell Research at Cambridge University, who said that the decision left European scientists is a "ridiculous position".
"We are funded to do research for the public good, yet prevented from taking our discoveries to the market place where they could be developed into new medicines. One consequence is that the benefits of our research will be reaped in America and Asia."
Lawford-Davies says he still holds out hope for the field for the following reasons:
  • Inventors and patent attorneys have known about this concern for some time and have been drafting to try to take account of the court position.
  • It should be possible to obtain patents on allied technologies, such as biomarkers and diagnostics related to the particular therapy. 
  • It is still possible to obtain these types of patents in most other countries in the world, including the US.

Monday, October 17, 2011

CIRM high school curriculum a hit in the land of Disney

CIRM took our high school curriculum on the road last week to the annual meeting of the National Association of Biology Teachers in Anaheim at a hotel just down the block from Disneyland. About 800 biology educators attended the meeting. Although I did not venture into the amusement park, I suspect the exhibit hall at the conference had some equally fanciful attractions such as the 3-D video anatomy display across the aisle from the CIRM booth and the virtual tour of a Costa Rican rain forest. So, I was initially a bit concerned about how stem cells would compete for the teachers' attention. That concern was pretty quickly set aside.

At the very beginning of the meeting we had a CIRM hands-on workshop to walk a room full of teachers through how our five on-line modules and introductory lesson can be used. You can find them all here. Many in the room walked away after the hour session saying they planned to use the curriculum.

The next day the exhibit hall opened and we had a steady parade of teachers coming by to say their students had been asking about stem cells and that they were thrilled to find out that such a robust curriculum was available on the web and seemed a bit shocked and pleased to find out it was completely free. They liked that each module had student resources and teacher resources, glossaries and assessment tools. Many talked about dressing up their classrooms with blow-ups of the images CIRM has linked off the curriculum and on our Flickr site.

Three days later as the meeting was winding down on Saturday, one teacher who had come by to chat the first day dropped by again to say, "I just wanted to tell you, you are the best thing in this place."

With that I would like to offer kudos to the team from U.C. Berkeley and the Bay Area high school teachers that helped us build the five modules, and particularly to thank Laurel Barchas who managed the project.


Friday, October 14, 2011

Researchers fix mutation in reprogrammed stem cell, create functional liver

A group led by the Sanger Institute and the University of Cambridge, working with the Sangamo Biosciences, has shown that it's possible to fix mutations in reprogrammed cells. This work, which was published in Nature, takes two previous advances and combines them into one proof-of-concept.

Since 2007 stem cell scientists have been able to reprogram adult cells such as skin back into an embryonic-like state. These so-called iPS cells can then mature into any cell type in the body, much like embryonic stem cells.

Other groups have shown that it's possible to take adult stem cells such as those from the bone marrow, correct mutations, and create mutation-free cells that, at least in animal models, can fix diseases. That's the idea behind CIRM's two HIV/AIDS disease team awards (described here and here) and a sickle cell disease team. In fact, one of the HIV/AIDS disease teams is also working with technology developed by Sangamo to fix the mutations.

In the work reported in Nature, the team created iPS cells from a person with a genetic liver disease, fixed the mutation, then matured the iPS cells into functional liver cells. A Reuters story quotes Allan Bradley, director of the Sanger Institute:
"These are early steps, but if this technology can be taken into treatment, it will offer great possible benefits for patients," he added.
Reuters quoted David Lomas, who was part of the team from Cambridge, saying that the liver cells survived when transplanted into mice.
The researchers said it could be another five to 10 years before full clinical trials of the technique could be run using patients with liver disease. But if they succeed, liver transplants -- costly and complicated procedures where patients need a lifetime of drugs to ensure the new organ is not rejected -- could become a thing of the past.

"If we can use a patient's own skins cells to produce liver cells that we can put back into the patient, we may prevent the future need for transplantation," said Lomas.

Wednesday, October 12, 2011

Patient advocates report in on the World Stem Cell Summit

These past few days post-World Stem Cell Summit has seen a lot of activity online from patient advocates who attended the meeting. One goal of that meeting is specifically to update the advocates on what's happening in stem cell research and also empower people to speak up about the value of the research. Bernie Siegel, who organized the meeting, seems to have succeeded if the number of advocates writing blog entries about his meeting is any indication.

Don Reed wrote a summary of his experiences at the meeting on his blog Stem Cell Battles. Reed was on a panel with me, Paul Knoepfler from UC Davis, and John Hlinko of and Left Action talking about how to use social media for stem cell advocacy. Reed wrote about the need for advocates to have answers at the ready:
For example, if someone says, “We can’t afford to fund stem cell research,” we immediately answer, “We can’t afford NOT to”—and it helps to have a few basic statistics handy, such as:

In 2009, America spent $1.65 trillion on chronic (incurable) disease. This is more than all federal taxes ($1.2 trillion) put together. It even exceeds that year’s installment of the national debt ($1.60 trillion). No nation can afford such costs—and it is why the economy is going down.
You can find some quick answers to common misconceptions about stem cell research in our stem cell basics.

Knoepfler has taken on the unusual role of stem cell scientist and patient advocate (he survived prostate cancer). He now has a blog about the science of stem cell research that he promotes through both Twitter and Facebook. He is a strong advocate for scientists speaking out and not being afraid to voice a public opinion about the value of research. On his blog, Knoepfler wrote:
Today I was part of a panel discussion on social media for the stem cell cause at the World Stem Cell Summit

Something that one of speakers said in the session that ended right before ours stuck with me as I was giving my talk. This person said that as part of the multi-dimensional team advocating for stem cell research that:

Scientists should just stick to the facts.

I like facts (aka data) as much as the next guy, but when it comes to advocating for stem cell research, my opinion is that scientists need to go beyond facts. We need to use our authority to voice opinions.
One point all three of us made is that whether you are a scientists, a patient or a member of the public who cares about finding new therapies it's important to get your voice heard by writing your legislators, talking to friends or family members and sharing information about stem cell research through conversation, emails, facebook, twitter or in blogs.

One advocate who is on board with that message is Karmel Allison who wrote about her experiences at the World Stem Cell Summit on the blog A Sweet Life. She said:
At the conference, I heard about amazing progress being made towards modeling neurological disease, regenerating heart tissue, growing kidneys, and even curing HIV. Even with so many stem cell applications to talk about, though, diabetes was no small player, and I was surprised by how much I didn’t know about diabetes and stem cells.

I had heard about the hope for beta cell regeneration, in which stem cells would be differentiated into beta cells and implanted into diabetics so that we could essentially regrow our defunct pancreases. This source of beta cells would be far more renewable and therefore preferable to the current source– pancreases from cadavers, which are very hard to come by.
Allison also attended a talk by CIRM grantees at ViaCyte, who have a Disease Team Award to develop a stem cell-based therapy for diabetes. She wrote:
The challenge ViaCyte is aiming to overcome is that the stem cell development process needs to be done in a reliable, industrialized fashion such that it can be profitable. So, ViaCyte is using a stable, well-characterized line of human embryonic stem cells that are cultured for two weeks, differentiated into progenitor cells, frozen for transport if necessary, encapsulated in a biocompatible envelope, and, in theory, implanted. The envelope system is designed to protect the cells from immune rejection, thereby circumventing the need for genetic matching.

Monday, October 10, 2011

Video describes life with Huntington's disease, hopes for stem cell therapies

The University of California, Irvine has begun posting video interviews with their stem cell institute director Peter Donovan (You can see all of those videos here.). Of the eight interviews, including ones with former CIRM board chair Robert Klein and leading UCI stem cell researchers, one of the most moving is with Huntington's Disease advocate Frances Saldana.

Saldana lost her husband, one daughter and two of her husband's brothers to the disease. Her two remaining kids have advanced Huntington's disease. She now has two grandchildren, both of whom have a 50/50 chance of having inherited the disease. In the UCI video Saldana says:
"This is not an illness that is going to go away once you lose your loved one. It's going to carry on through the generations. When they do inherit Huntington's Disease they will lose the ability to walk, to talk and to swallow and they will start to experience atrophy of the muscles which causes spasticity and a lot of pain. It's very painful."
Saldana spoke to the CIRM governing board about life with Huntington's disease back in 2007. Saldana is one of many of the passionate advocates for Huntington's disease who have spoken with the agency over the years and who have been tireless supporters of stem cell research.
"What I would hope for, if not a cure, is a treatment to alleviate human suffering for our families. I don't think stem cell research will provide a cure for my two other children, but I have two grandchildren and now they are vibrant and beautiful and I hope they have a full life ahead of them, but we need to have something for them if and when they might be carrying that mutant protein."
The agency currently funds five awards with a focus on the disease. You see a list of these awards, learn more about Huntington's disease, and watch a video CIRM produced about the disease on our disease fact sheet.


Friday, October 7, 2011

Stem Cell Awareness Day introduces high school students to heros

Stem Cell Awareness Day on Wednesday brought scientists to close to 60 classrooms to deliver lectures on stem cell science and inspired events throughout the world, some of which are ongoing throughout October. (You can see a full list of events here.)

The Daily Pilot had a nice story on an event at University of California, Irvine, which brought more than 100 high school students into UCI's Sue and Bill Gross Hall. CIRM contributed $27.2 million to the construction of the building. In addition to stem cell labs, the building houses equipment to help people with spinal cord injuries regain strength. The story quotes one of the students who attended:
"It's nice to see our tax dollars go to something that actually helps people," said Brittany Jackson, a Trabuco Hills High School senior. "There's so much technology. You don't realize there's so much that goes into helping those less fortunate."
The story goes on:
Standing between a computer monitor showing color dyed stem cells under a microscope and a refrigerator growing stem cells, Christina Tu talked about why she and researchers do what they do.

One scientist is working with a bioengineer to generate cells that can help a baby recover from heart failure at birth. Tu, the stem cell core facility coordinator, moved into stem cell research from another field because of the possibilities.

"They must have this passion," she said. "A lot of times, outcome is negative. You have to overcome that to see what you want to accomplish."

Nancy Luke, a lawyer stricken with multiple sclerosis, encouraged the students to move science forward.

"What I have is the hope that [what UCI is studying] can cure MS," she said. "Researchers rock. And you guys will be the heroes."
That's what Stem Cell Awareness Day is all about: Exposing people to those heroes, and encouraging students to become heroes themselves.


Thursday, October 6, 2011

New cells lines made using “cloning” technique valuable for research

Yesterday a New York Stem Cell Foundation team reported for the first time that they had created two new embryonic stem cell lines through a technique known as somatic cell nuclear transfer (SCNT), which is sometimes called therapeutic cloning. They reported their findings in the journal Nature.

SCNT is a third avenue for creating cell lines able to form all tissues in the adult body – called being “pluripotent.” Interestingly, SCNT borrows from the two other techniques used to date.

Pluripotent cell lines were first created by extracting them from 5-6 day old human embryos left over after in vitro fertilization – hence their name human embryonic stem cell lines.

Pluripotent cell lines were later created by reverting skin cells to a pluripotent state through a process called “reprogramming” – commonly referred to as induced pluripotent cells.

SCNT is a reprogramming method that involves the creation of an embryo as a first step.  In this case, scientists took DNA from a human skin cell and placed it in a human egg, which they then stimulated to form a 5-6 day old embryo. In this environment, the DNA was reprogrammed to an earlier state and the resulting cells were extracted to create human embryonic stem cell lines.

The fact that SCNT-derived stem cell lines have so much in common with other forms of pluripotent stem cells has some opponents of the research asking why bother? Here’s why. CIRM held a conference in June 2010 to discuss the value of pursuing SCNT and posted a report on the findings in November, 2010. 
That report suggests three areas where embryonic stem cell lines generated through SCNT would clearly be valuable in three ways:

  • Understanding how you reprogram any cell to become pluripotent could help us optimize the creation of iPS cells, which are so far inefficient to create in addition to being incompletely reprogrammed.
  • Understanding and treating the rare diseases that are passed on from those few genes that reside outside the nucleus in the cellular organ called the mitochondria.
  • Studying the very early stages of human development, which are poorly understood now, and which is when some human diseases are thought to originate.

The fact that the New York team got the technique to work in humans is a significant advance that has value for all three of those potential areas of research. However, the two cell lines reported yesterday aren’t exactly ready for therapies. Rather than having two copies of each gene, as all of us do, these cells have three copies of every gene and are therefore biologically abnormal. The Wall Street Journal described the problem like this:

While such cloning experiments have been successful in various mammals, the "de-nucleated" egg approach hasn't worked so far in humans. Now, Dr. Egli and his colleagues have—partially—achieved it via a simple move: They didn't remove the egg's own nucleus.

Not removing the egg’s nucleus resulted in the triple copy of chromosomes (one from the egg and two from the donor’s nucleus) that left the cells as “research only” cell lines. Many news stories about the work have referred to the new lines as coming from “cloned embryos”. However, because the cell lines contain more chromosomes than the donor cell they are not truly clones.

Understanding what factors in the nucleus aided in getting SCNT to work could provide clues about factors that might aid in making iPS cells more efficiently, and also provide clues as to how to create SCNT-derived lines with normal numbers of chromosomes.

The Wall Street Journal story quotes George Daly, a stem-cell researcher at Children's Hospital Boston , who summarizes the findings as “a landmark even if it isn’t a complete victory.”

In coming months we should watch for advances that turn this landmark into a victory for people hoping to use the SCNT-derived stem cells to study the earliest stages of development, understand and treat mitochondrial diseases, and learn how to create better iPS cells.


Wednesday, October 5, 2011

Guest blogger Alan Trounson — September’s stem cell research highlights

Each month CIRM President Alan Trounson gives his perspective on recently published papers he thinks will be valuable in moving the field of stem cell research forward. This month’s report, along with an archive of past reports, is available on the CIRM website.

Much good science and important science does not qualify as a “breakthrough.” Turning stem cells into heart cells is nothing new. Many teams have done it with both embryonic stem cells and reprogrammed, or induced Pluripotent Stem Cells (iPSC).

However, most the methods for steering stem cells down the path toward heart tissue are not very efficient or don’t produce uniform heart cells. You often end up with a mixed batch of cell types. This month’s literature produced the type of incremental advance that does not wow you, but is exactly the type of thing we need to make the field more practical for patients looking forward.

A team at Kyoto University reported an efficient way to make heart progenitor cells from both embryonic and iPSCs. But more important they found a cell surface marker on those progenitor cells that let them develop an efficient and scalable purification method to get and end product that was just the cells they wanted. But even this step was not easy. They screened 242 antibodies to find this one marker.

My full report of this month’s highlights has some similar incremental advances in improving bone marrow transplant as well as a few other papers I hope you will find interesting.

Tuesday, October 4, 2011

CIRM video journal of the World Stem Cell Summit

The World Stem Cell Summit is going on now in Pasadena. This is the primary meeting for pulling together scientists and patient advocates to talk about progress in the field.

CIRM has transformed our booth into a mini recording studio to film interviews with advocates and scientists at the meeting. They talk about the importance of advocates spreading the word, what they hope to learn from the meeting and what they think all people should know about the field. Here's the first batch of videos:

Jonathan Thomas, Chair of the CIRM governing board: What do you want all people at this meeting to know about CIRM?
I don't think there's a real comprehensive feel for the fact that we are engaged in research going after 26 different diseases.

Keri Kimler, Vice President for Texans for Stem Cell Research: What is the reception to your message about the power of stem cell research in Texas?
There's a lot of misconception. I think that we do everything we can to educate on the true purpose of the research and that's to find solutions to unmet needs and to find cures and to find new treatments and I think that that usually works very well.

Roman Reed, Roman Reed Spinal Cord Injury Research Act: Is there something all advocates should learn and take home from the meeting?
It's incumbent on all of us to get the message out there because when you really know about stem cell research you know it's the right thing to do. What's wrong with trying to help the suffering?

Laurel Barchas, PhD candidate at UC Berkeley: Is there a point about stem cell research that you think all people should take away from this meeting?
What's striking about this conference is that I've met people who are working in law, working to educate people, working to educate patients, patient advocates. All of these people are coming together with the common goal of advancing stem cell research. No matter what you are interested in you can use your talents to push this research forward.

Don Reed, Vice President for the Americans for Cures Foundation: Is there a message that all patient advocates should go home and tell their friends and family members?
Get involved. There's so many opportunities for patient advocates to get involved. Everybody knows somebody with a disease or disability for whom the doctors have said there's no chance, there's no hope.

Judy Roberson, past president of the Huntington's Disease Society of America: Is there some important role you think advocates play at this meeting?
I feel like patient advocates get a lot from listening to the hopeful messages and I think they get encouraged by hearing our personal stories about how Huntington's disease as effected my family and that we are very hopeful about stem cell research.