Friday, December 21, 2012

Neural stem cells show signs of treating ALS (Lou Gehrig's disease) in mice

A consortium of researchers around the U.S. used transplanted neural stem cells (shown here) to treat a mouse model of ALS.
CIRM grantees at Sanford-Burnham were part of a consortium that found transplanted neural stem cells slows the progress of a form of Lou Gehrig’s disease (amyotrophic lateral sclerosis or ALS) in mice.

It’s still too early to celebrate – mice are quite different than humans, and treating a disease in one doesn’t necessarily translate into a therapy for the other – but it’s nice to see signs of hope for a disease that’s desperately in need of a therapy. ALS is a fatal disease in which the nerve cells that control movement die.

Sanford-Burnham described the study, which was published in Science Translational Medicine, on their blog:
In this study, researchers at multiple institutions conducted 11 independent studies to test neural stem cell transplantation in a well-established mouse model of ALS. They all found that this cell therapy reduced the symptoms and course of the ALS-like disease. They observed improved motor performance and respiratory function in treated mice. Neural stem cell transplant also slowed the disease’s progression. What’s more, 25 percent of the treated ALS mice in this study survived for one year or more—roughly three to four times longer than untreated mice.
They went on to describe how the cells helped slow the disease:
Transplanted neural stem cells helped the ALS mice, but not for the obvious reason—not because they became nerve cells, replacing those missing in the ALS spinal cord. The biggest impact actually came from a series of other beneficial neural stem cell activities. It turns out neural stem cells produce protective molecules. They also trigger host cells to produce their own protective molecules. In turn, these factors help spare host nerve cells from further destruction.
We have more information on our website about our stem cell funding for ALS.


Thursday, December 20, 2012

Most popular stem cell stories of 2012: diabetes, spinal cord injury, and what the future holds

This is the time of year when everybody is posting their year end best-of list. Not wanting to be left out, I went through to find our top blog entries of the year. What I learned is that readers are eager to know about progress toward therapies, which makes perfect sense. As an agency, we exist because voters wanted to accelerate the path to new therapies and that’s just what we’ve been trying to do.

1. Our top blog post is about a video we produced to explain an exciting project working toward a therapy for diabetes. I think this post is so popular because it not only explains the science, it includes two people who are living with diabetes explaining why it’s so important to find a cure.

2011 Annual Report: Toward a stem cell therapy for diabetes

2. In June, CIRM grantee Irv Weissman at Stanford University wrote about the barriers that stand in the way of developing new therapies. That publication includes perhaps my all time favorite line of any scientific paper: “So, whom have I failed to annoy here?” He’s outspoken, but only because he cares. Here’s an excerpt from the paper, which appeared in Cell Stem Cell:
Remember, right now our patients, friends, and families are contracting diseases that have a very short window of opportunity in which regenerative therapies can save them, and each delay removes a cohort of them from possible cures. We should not fail them.
Irv Weissman on the many barriers to stem cell therapies and why they matter

3. Next on our list of most popular blogs is one that describes a conversation I had with Katie Sharify, the fifth person to participate in Geron’s now-closed trial for spinal cord injury. In includes a link to a video of our conversation, in which she talks about how she made the decision to participate and what she hoped to get out of the trial. “I was part of something that was bigger than me, and bigger than all of you.”

Fifth Geron stem cell trial participant discusses her experience

4. Earlier this year we filmed an Ask the Expert video in which we talked to CIRM grantee Lawrence Goldstein about stem cell therapies for Alzheimer’s disease. As part of that conversation, we discussed what he saw in the future for stem cell science. In our blog entry about that video we quote Goldstein, "If the public continues to adequately fund research with stem cells we will see breakthroughs that are absolutely unexpected and that will change the way that we deliver medicine."

Looking into the stem cell crystal ball: what's next?

5. In September of this year, StemCells Inc reported that two patients in their clinical trial for spinal cord injury were recovering well and seemed to have gained some sensation. We weren’t funding the work at the time, though the trial is based on work by our grantees at UC Irvine. The company recently received a disease team award to further fund this trial.

CIRM grantees show preliminary signs of success in spinal cord injury trial

Our YouTube site shows a similar focus on stem cell therapies, with videos about Parkinson’s disease, Diabetes, ALS, and macular degeneration in the top five. The only top video that didn’t describe a CIRM team working toward therapies was one about a talk given by Craig Venter about the power of genomic research.


Wednesday, December 19, 2012

Stem cell who's who: 2012 Person of the Year

Earlier this year CIRM grantee and stem cell blogger Paul Knoepfler requested nominations for his stem cell person of the year competition. In a video that accompanies his request, Knoepfler says he was hoping to honor someone other than the usual stodgy scientists—people who have gone out of their way to propel the field through legislation, advocacy, or in the case of some nominees through sheer force of personality (that’s you Roman Reed).

Knoepfler recently posted the top 16 in a veritable who’s who of stem cell notables. Some are scientists, but are nominated for their extra-scientific activities fighting stem cell tourism, others are bloggers, stem cell advocates, entrepreneurs, foundation directors, and patients fighting for cures.

It should also be noted that two CIRM folks were nominated, but Knoepfler put them out of the running due to conflicts of interest – he himself has CIRM funding. Elona Baum has worked tirelessly at CIRM to bring much-needed industry involvement into the stem cell field and Patricia Olson leads our scientific activities.

It’s fun to look down Knoepfler’s list and see familiar names, many of whom I’ve worked with while trying to tell CIRM’s story. Judy Roberson, Roman Reed, Don Reed, Jeanne Loring, Katie Sharify and Keri Kimler all grace videos on our YouTube channel (the links lead to their videos), and several of those people regularly show up to our meetings and advocate to our board for research funding.

I don’t envy Knoepfler the task of picking just one person from the list. We are lucky to work in a field that includes so many dedicated people pushing for stem cell-based therapies.


Tuesday, December 18, 2012

Old drug finds new life treating muscular dystrophy

Dr. Stanley Nelson
CIRM grantees at UCLA published a paper last week showing that a drug that’s currently being tested for other uses might also be effective in treating people with muscular dystrophy. The group got a $6 million Early Translational award from CIRM to turn this research into a clinical trial for the disease (here's a description of their project).

The researchers have a great story to tell. The lead author Stanley Nelson and his wife Carrie Miceli, who also participated in the research, have a young son with muscular dystrophy. UCLA writes about the couple, using the acronym DMD for a specific type of muscular dystrophy called Duchenne muscular dystrophy:
The research by Miceli and Nelson, who are married, is driven by more than just scientific curiosity. Their youngest son, Dylan, 11, was diagnosed with DMD in 2004. While he's still ambulatory — many DMD patients require the use of wheelchairs by about age 10 — Dylan can no longer run or climb stairs, and he can't shoot a basketball over his head like other boys his age. Despite these challenges, Miceli said Dylan remains a happy, funny and engaged boy, full of life and passion. 
"We entered into this field because of the diagnosis of our son, but we hope our research can help many others," she said. "There are drugs that can help manage the symptoms of the disease but nothing that changes its course dramatically. We're trying to correct the defect that causes DMD with highly personalized genetic medicine."
Their research was published in the journal Science Translational Medicine. Although the potential therapy involves a drug, not a stem cell, the team used patient-specific stem cells to find that drug. This type of stem cell screen is becoming an increasingly common way of finding new therapies. The group started with cells from people with Duchenne muscular dystrophy. Those cells contain the mutation that causes the muscles to waste away. They then converted those cells to embryonic-like iPS cells, which have the ability to form any cell in the body. Then, they matured those cells into muscle cells that, since they have the mutation, also show signs of the disease in a lab dish. They could then expose those cells to many thousands of different drugs, looking for ones that seem to improve the symptoms.

Over the next few years they are hoping to do longer-term studies to see if the drug combination is effective over time, and then eventually start clinical trials to see if it works in people. Right now, there is no effective treatment for this fatal disease.


ResearchBlogging.orgKendall GC, Mokhonova EI, Moran M, Sejbuk NE, Wang DW, Silva O, Wang RT, Martinez L, Lu QL, Damoiseaux R, Spencer MJ, Nelson SF, & Miceli MC (2012). Dantrolene enhances antisense-mediated exon skipping in human and mouse models of duchenne muscular dystrophy. Science translational medicine, 4 (164) PMID: 23241744

Monday, December 17, 2012

Has that stem cell treatment been tested? Better check before paying

Image: Credit: Nissim Benvenisty, via Wikimedia Commons
We’ve written quite a bit about the dangers of stem cell tourism and about unregulated treatments in the U.S. The concern is that people are paying large amounts of money to receive injections of cells that haven’t been tested for safety.

Scientific American recently published a story that’s receiving a lot of attention in stem cell circles that makes clear why these unregulated injections are a problem. It highlights the story of a woman who had received injections in her face of stem cells taken from her own fat. She ended up with bone growing in her eyelid.

The story quotes CIRM grantee Paul Knoepfler, who is a stem cell researcher at UC Davis. He has blogged frequently about his concerns regarding stem cell clinics offering unregulated treatments. In the story he says:
"Many of us are super excited about stem cells, but at same time we have to be really careful. These aren't your typical drugs. You can stop taking a pill and the chemicals go away. But if you get stem cells, most likely you will have some of those cells or their effects for the rest of your life. And we simply don't know everything they are going to do."
CIRM is working with our grantees and with the FDA to help move promising therapies through clinical trials and into doctor’s offices. That process takes time, but through those trials we get some assurance that the cells aren’t going to cause any harm. This page has more information about our initiatives to bring therapies through clinical trials to patients.


Friday, December 14, 2012

CIRM Research in a Global Perspective

Sixteen years ago the first edition of The Global Burden of Disease was released in hard cover. I remember lugging the 1,022 page edition, complete with tables of data, around campus as a masters of public health student at UC Berkeley. Professor Kirk Smith made us use the tables to calculate how disease patterns vary around the world, and perhaps most importantly how such data can be used to guide health policy decisions.

Fast-forward to 2012, where a tool from the Institute for Health Metrics Evaluation saves the need for lugging and data entry. It also makes it much easier to see the worldwide importance of finding therapies for some of the major diseases that are a focus of CIRM research such as HIV/AIDS, cancer and diabetes, among others.

You can play with this online tool here. It incorporates yesterday’s release of the 2010 Global Burden of Disease data into a fabulous interactive visualization display.

The tool allows one to analyze disease patterns by age, region of the world and disease type and to compare the patterns to those seen in 1990. I spent last night showing my wife and son how important some of the diseases CIRM is focused on developing treatments for are. We examined diabetes, HIV/AIDS and cancer to discoverer the following:
  • North America ranks as the third highest region in the world for disability attributed to diabetes (note this figure include both Type I and II);
  • HIV/AIDS is the leading killer of 30-39 year olds globally
  • Cancer is the leading cause of death of 5-9 year old children in North America
I was pleased to see that the 2010 results were widely publicized and reporting good news on the health front. Specifically, that fewer children are dying or being disabled from preventable diseases. The Lancet reports:
Although 52·8 million deaths occurred in 2010 (in 1990, the figure was 46·5 million deaths), great progress is being made in population health. Life expectancies for men and women are increasing. A greater proportion of deaths are taking place among people older than 70 years. The burdens of HIV and malaria are falling. Far fewer children younger than 5 years are dying. Infectious diseases are increasingly being controlled. In some parts of the world, there has been substantial progress in preventing premature deaths from heart disease and cancer.
One can gain endless insights into world health and health policy decisions with this visualization tool. Best of all, no more late nights having to manually enter data from my text book.


Tuesday, December 11, 2012

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

Alan Trounson, CIRM President
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.

The full report this month includes a couple important advances in understanding the underlying nature of stem cells that result from reprogramming adult cells to become like embryonic stem cells, induced pluripotent stem cells (iPS). Although the science that revealed that this feat could be accomplished won the Nobel prize this year, much remains unknown about the resulting cells.

You can read that full report here, but I want to focus this blog on another paper that appeared in November, one that I frankly never expected to read. A team at Stanford placed embryonic stem cells directly into animal patients and they did not see wide spread formation of tumors. The very hallmark of pluripotent stem cells, whether embryonic or iPS cells, is that when they are transplanted directly they uniformly form a type of multi-tissue tumor called a teratoma. That is why the conventional wisdom is that pluripotent cells would always be matured at least part way to the desired final adult cell before ever being transplanted into patients.

The Stanford team only saw tumors in two out of 42 mice that received the transplants. In taking the leap to try this experiment, the researchers were building on knowledge built up in recent years about the key roll of the environment around stem cells in determining their fate; that is determining what type of adult cell they mature into. In this case they wanted the cells to become bone to replace a hole they had created in the animals’ skulls. They placed the cells on a synthetic scaffold to make the cells array as in bone and laced the scaffold with a protein known to enhance bone formation. The team attributed an additional push to form bone to signals the stem cells received from the neighboring bone at the graft site.

Two tumors out of 42 is still two too many for any regulatory approval of the process. But the team wrote that they hope to enhance the technique to yield completely tumor-free recipients. One thing they are suggesting is adding other cell types to the scaffold to act as “chaperones” for the stem cells.

My full report is available online, along with links to my reports from previous months.


Friday, December 7, 2012

Coalition helps keep our embryonic stem cell work legal

After three days of being with a few hundred like-minded and energized folks at the World Stem Cell Summit in Florida, I was able to hop up the coast to D.C. for the annual meeting of the Coalition for the Advancement of Medicine, an event that I can usually only catch a piece of by phone. CAMR is the main organization that has fought to preserve our right to fund embryonic stem cell research and for the right of the National Institutes of Health to join us in funding that work..

The group has been instrumental in helping keep the work legal in most states. Its effort was also critical to the passage of legislation that would allow federal funding in 2005 and 2006. Unfortunately those bills were the subjects of two of President Bush’s first vetoes. But the coalition also worked to make sure Obama’s presidential order in March 2009, authorizing NIH funding for embryonic stem cell research, was one of the earliest executions of one of his campaign promises. We heard at the CAMR meeting that there are now 186 embryonic stem cell lines approved for NIH funding.

When the coalition was formed we did not expect to be spending time in litigation. (My former colleagues at Harvard helped found the group and I attended an early organizing meeting.) But when the Shelley vs. Sibelius case was filed in August 2009, we did file a brief, and have done so at each stage of this prolonged case. The CAMR board believes those briefs were influential because it was easier for us to say certain things than for the Justice Department to do so. The next key date in the case is January 4. That is the next chance for the Supreme Court to decide whether to hear it, though it may not decide then, or it could decide and not announce it at that time.

Status quo is holding on Capitol Hill. There was not any legislative activity last year and we heard today that none is expected this year unless the Supreme Court case goes the wrong way.

A member at the meeting observed, “At CAMR we always hoped we could get to the point where politics was no longer getting in the way of good science, and Sally Temple’s presentation today showed we are getting to that point. And I think CAMR has had a large role of getting us there.” (We did a short blog about that talk at the World Stem Cell Summit.)

That is at least in Washington.

Activity has moved to the states. David Chatel, of the National Multiple Sclerosis Society follows the issue at the state level for CAMR. The coalition has tracked legislation and ballot issue in 19 states in the past year. The CAMR board meets weekly to decide if action is needed at the state level. They took actions in two state sending letters to members of two legislatures: those in Virginia and Oklahoma.

CIRM is one of some 90 organizations that are members of the group ranging from major research universities to patient advocacy organizations like the Parkinson’s Action Network. I am proud to have been there at the beginning of the coalition that has certainly proven its worth over the years.


Thursday, December 6, 2012

Jonathan Thomas discussion IOM report on CIRM: "Bold social innovation"

Jonathan Thomas is chair of CIRM's governing board, the Independent Citizens' Oversight Committee

From the very beginning in 2004, when voters approved Proposition 71, creating the stem cell agency and providing us with the money to help fund research, CIRM has been a unique organization. No other state agency was ever created in this way. As we set out to start work we had no models to follow, we had to start from scratch. So we did. And I think we did a remarkable job. But there is always room for improvement and with that in mind in 2011 we asked the Institute of Medicine (IOM) to come in and take a look at everything we do from how we are run to how we fund research, and let us know what we are doing well and where they think we could do better.

This assessment has been underway since I became Chair of the agency’s governing board, the Independent Citizens Oversight Committee (ICOC), last year, and I've eagerly awaited the IOM's report on how we've been doing and how we can improve.

Why the IOM? Well they are considered the gold standard for scientific policy and health care. They set up a 13-member panel led by Harold Shapiro, PhD, a former President of Princeton University, which included experts from a wide variety of scientific and governmental fields. Over an 18-month period they held three public meetings, several briefings and asked us a lot of tough questions.

The result is a detailed report that came out this week looking at every aspect of the stem cell agency’s operations and organization. It’s a comprehensive and thoughtful look at us, and a quite complimentary one too.

Among the areas the IOM singled out for praise are:

  • CIRM’s collaborations with funders in the US and around the world saying this “substantially enhanced California’s position as one of the key international hubs of activity in regenerative medicine.”
  • Science and research - “CIRM has been highly effective in building an impressive research portfolio.”
  • Global impact - “the work of CIRM-sponsored researchers continues to enrich regenerative medicine everywhere,” and that “CIRM and those it has funded have set in motion a significant scientific enterprise.”
  • Grants management - “Given the complexity of this endeavor…..the overall success of the grant management infrastructure is impressive.”
  • Industry engagement – “CIRM has created an exemplary training program and seeded a pipeline of intellectual property and translational projects that are primed for industry involvement, outside funding, and unique therapy delivery mechanisms.”

Those findings are a testament to the experience, expertise, and commitment of the staff at the stem cell agency, and of our governing board. It’s a very small organization by most standards – just over 50 employees – but our staff do an extraordinary amount of work, and high quality work at that. All that was reflected in the IOM report that called the agency “a bold social innovation.”

While it’s nice to get praise we also wanted to know where we could do better, and the report highlighted some areas and made some recommendations about where and how we might improve our performance. Those include:

  • Changes to the composition and structure of our governing board, the Independent Citizens Oversight Committee (ICOC)
  • Changes in the roles board members play in the grant application review process
  • Revisions to our Conflict of Interest policies
  • Sponsor training programs on ethical issues and stem cells
  • Establish a Scientific Advisory Board
  • Develop a sustainability platform
  • Increase industry representation on our governing board, the Independent Citizens Oversight Committee (ICOC), and other key working groups

We take the report, and all its observations, seriously and over the next few months the agency’s management team and our governing board will consider how best to respond to the report and its recommendations.


Wednesday, December 5, 2012

Short takes from patient advocates at the World Stem Cell Summit #wscs12

Posts from my colleagues who were at the World Stem Cell Summit this week reminded me about the short video interviews we did last year with patient advocates. One of the best parts of this meeting is always seeing the scientists mix with the patient advocates and meet the people who really stand to benefit from their research.

We talked with several advocates in our booth about what they were getting out of the meeting. You can see all of those short interviews on our blog entry from that meeting.

One of my favorites was my talk with Keri Kimler, Vice President for Texans for Stem Cell Research, who is a tireless advocate for stem cell research and for educating people about the value of the work. She's always bustling around that meeting, meetings scientists and other advocates and talking up Texan research. Here she is:


Sabrina Cohen honored for providing inspiration and raising awareness #wscs12

Sabrina Cohen at the World Stem Cell Summit with Chris Makos, who is an advisor to the Sabrina Cohen Foundation
Last night the World Stem Cell Summit honored Sabrina Cohen with its Inspiration award for her remarkable work in raising awareness about stem cell therapy and regenerative medicine. Sabrina suffered a severe spinal cord injury in a car accident when she was 14. She founded the Sabrina Cohen Foundation for Stem Cell Research to help educate others and raise money for research.

We asked Sabrina what she thinks the most important elements of the conference are.
"It does such a great job of bringing the community together, uniting the patient advocates and the scientists and gathering them all in the same place. That helps us all stay in touch, helps create relationships between all the different groups and collaborations that are necessary to help advance the science."

"I think the most important benefit of this summit is how it can help to inspire us all. Bernie Siegel [the organizer of the event] has done an amazing job in bringing the patient advocates into the same room as the scientists and making everyone appreciate that we are all in this together. For the scientists it helps put a human face on what they are trying to do. And once you have met the advocates, heard their stories and looked them in the eye, you can't feel the same way about your work ever again. It really does help inspire all of us."

Stem cell action awards gives nod to scientists fighting unregulated stem cell clinics #wscs12

At last night's World Stem Cell Summit awards dinner, one of the award recipients was 60 Minutes for their two-part “21st Century Snake Oil” investigation. According to the award website:
CBS News’ “60 Minutes” has provided an invaluable contribution to public awareness exposing risks and rip-offs foisted upon vulnerable patients and their families. The two-part “21st Century Snake Oil” investigation of offshore clinics making outrageous claims and luring desperate patients to undertake dubious, possibly dangerous, unproven “treatments,” has educated the public and launched investigations by both the FDA and FBI.
The issue of unregulated stem cell clinics is one that has gotten a lot of attention recently from stem cell scientists as well as the media. The concern is that people desperate for cures will pay high prices to receive unproven and in some cases unsafe "stem cell" procedures. The quotes are there because in many cases the clinics won't reveal what it is that patient actually receives. Stem cells? Maybe. Or maybe not.

During the award to 60 Minutes last night CIRM grantees Larry Goldstein of UC San Diego and Jeanne Loring of Scripps Research Institute were both mentioned for their work educating people about unregulated stem cell clinics. Here are videos we've produced with both of these scientists talking about their concerns:


I would add Paul Knoepfler to the list of those CIRM grantees who has worked hard to educate people about the risks of untested treatments. He blogs regularly about such clinics in the U.S. and internationally. You can read his blog here.


Tuesday, December 4, 2012

"If they feel the heat, they see the light": the role of the stem cell advocate #wscs12

Paul Knoepfler, a CIRM grantee at UC Davis, blogger and stem cell advocate talks about the role advocates can play in promoting stem cell research
There was a fascinating afternoon session at the World Stem Cell Summit on the power and role of the patient advocate in advancing stem cell research and particularly in overcoming the political challenges facing us at both the national and state level. Bernie Siegel, the founder of the World Stem Cell Summit, kicked the conversation off by saying that stem cells are not a Democrat or Republican issue, they are a human issue and we need to put aside political partisan feelings and work together on this.

First to speak was Paul Knoepfler, PhD, a stem cell researcher at UC Davis, a cancer survivor and, in the words of Bernie Siegel "a fabulous blogger" (you can read his blog here). Paul highlighted a number of important challenges facing advocates.
  • Funding – The level of funding for all research, including stem cell research, at the NIH has remained static or declined over recent years, this has really impacted research negatively. About 90% of grants are not funded which means that a lot of promising, quality science is going unfunded and it's very discouraging for a generation of younger scientists trying to start their careers. Paul says the Solution to this is to work as advocates to put pressure on the government to increase the NIH budget. He says advocates need to raise their expectations of and demands on President Obama to increase funding. Also need to make this a bipartisan issue so that people on both sides of the political aisle support it.
  • Big chill for human embryonic stem cell research – Funding for human embryonic stem cell research is decreasing, often not for scientific but for political reasons
  • Deregulation – Big push for deregulation from both political and non-political bodies. Paul says as the FDA budget stays fixed the number of unlicensed clinics offering unproven therapies is increasing, what he calls 'de facto' deregulation. This is posing a threat to patients and to legitimate stem cell businesses whose reputations may be harmed by these other clinics.
  • Broader approach – Paul says we need to engage stem cell scientists as advocates, do more education outreach using social media and build bridges with those we might not otherwise feel we have anything in common with. He says his blog has allowed him to meet with people he might not ordinarily meet or agree with such as Texas Gov. Rick Perry; he has also hosted a debate between Dr. Centano and Doug Sipp, two people from opposite sides of the divide. While they didn't reach agreement they did both present their feelings in a respectful way. Paul has also launched his own international education/outreach program called SCOPE. This is a 2 page white paper on stem cells that is translated into 22 languages and is part of a broader education and outreach international program
  • Scientiists and advocates need to reach out through social media to reach a wider audience and educate people.
Next up was Amy Comstock of the Parkinson's Action Network and Coalition for the Advancement of Medical Research CAMR.

Amy said the main message she has about what is happening in Washington DC around stem cell legislature is "not a lot". Up until 2009 there was a lot of activity surrounding efforts to ensure that the imposition of tight federal limits on important medical research couldn't happen again. But the filing of the Shirley vs Sebilius lawsuit put the brakes on those efforts – this lawsuit alleges that the Government is violating the law in using federal funds for embryonic stem cell research. That lawsuit has been to the DC Court of Appeals twice and the pro-stem cell side has won both times, but now it's at the Supreme Court and we are waiting to hear if the Justices will take it.

The lawsuit stalled any interest in moving stem cell legislation, so nothing has happened for more than three years as the lawmakers wait to see what the court decides.

Patient advocacy activity nowadays is focused, in DC at least, on more general funding for medicine particularly with the fears over the Fiscal Cliff and the threat of massive cuts to the NIH budget if no agreement is reached.

The third and final speaker was our good friend and long time supporter Don Reed, the VP at American's for Cures Foundation. Don's message was, as always, crisp and to the point. He says politicians respond to pressure, "if they feel the heat, they see the light"

Don said scienitsts need three things freedom, funding and friends. Activists and advocates can help ensure they get all of those.
  • Advocates can help defeat legislation in states that are attempting to shut down stem cell research. They have succeeded in many states but those battles continue.
  • Only a handful of states put up money for stem cell research, we need to try and boost that number to increase the funds available to researchers
  • Friends – people in power are essential to winning passage of bills we want or blocking approval of legislation we oppose. We need to support people competing for political office who support our aims, and then support them once they are elected.
Don had some kind words for Prop 71 and the stem cell agency calling us "amazing". Thankfully the room was quite dark so no one could see how deeply the CIRM folks were blushing.

He finished on an inspiring note, telling advocates "You can make a difference." He told the story of the time his son Roman (who broke his back in a college football accident) and he met with actor and spinal cord injury activist Christopher Reeve. "We were trying to raise money for his foundation, and he said "one day Roman and I will stand up from our wheelchairs and walk away forever." Choking back the emotion Don said "the cure didn't come in time for our champion but his light still exists and we will go forward and we will prevail."


NIH vs. CIRM funding of stem cell research #wscs12

Geoff Lomax took this photo during a talk by Joshua Hunsberger form the NIH Center for Regenerative Medicine during the World Stem Cell Summit. It shows the type of stem cell research funded by the NIH during 2011. By far, the largest percentage (47%) has gone to tissue-specific stem cells in animals. Human embryonic stem cell research makes up 9% of the funding and other forms of human stem cells make up 30%.

The chart below shows CIRM's total stem cell research funding (you can see more charts on our website). All of the funding shown here is for human stem cells, with embryonic stem cell research making up by far the largest percentage.
Like the NIH, CIRM funds research using all types stem cells because until we've done the research we can't know which type of cell or which approach will be most effective for different diseases.


Panel investigates unproven stem cell therapies #wscs12

A panel at the World Stem Cell Summit discussed the unproven uses of stem cells
 Tuesday morning the World Stem Cell Summit turned its attention to the use of “unproven” stem cells in clinics. This is an issue that has come to prominence in recent years, as clinics internationally and in the U.S. advertise (and charge high prices for) supposed stem cell treatments that have not been tested and shown to be effective, much less safe. (This is an issue we've blogged about quite a bit.)

In her introduction to the morning session, Ann Tsukamoto of StemCells, Inc. highlighted recent media reports concerning clinics where patient have been harmed by unproven cell treatments. Later in the session, Douglas Sipp from the RIKEN Center for Developmental Biology showed images of a stem cell clinic in the Dominican Republic that was clearly not practicing science-based medicine. The government has subsequently shut down the specific clinic.

James Guest from the University of Miami Miller School of Medicine said, “This is not something we can contain.” He felt excitement about stem cells coupled with the expansion of the internet has resulted "fast-track access" to unproven treatments. He showed examples where patents have been harmed from receiving these untested therapies. He noted that even in cases where transplanted cells are not harmful there is risk from transplant procedures that may result in damage to skin and tissue as well as infection.

The panel gave these signs of what they called a stem cell scam:
  • Claims of miracle cures for diseases
  • Single treatments or cells that can treat any type of disease
  • Lack of objective information, evidence (such as published medical reports) that a treatment is effective
  • Treatment by a doctor who is not trained or certified to treat the specific disease
  • No system exists to collect information and follow up with patients
Allan Wu from the Morrow Institute offered some ideas on what a patient can do if a university based clinical trail is not available and they want to consider a private-sector option. He indicated a consumer should ask:

  • Does the treating clinician have appropriate certificate to practice the procedure (But one need to watch out for certificates from organizations that are not authoritative)
  • Is the clinic FDA registered
  • Has the procedure they are considering been approved by an IRB, which is a panel that reviews experimental procedures
  •  Can they document results in other patients
Wu’s bottom line: You need to be able to ask your physician (1) have they done a significant number of procedures and (2) if I have a complication, will you be able to treat me? He also noted the International College of Stem Cell Medicine has formed to initiate an effort to certify doctors, laboratory directors and laboratories.

James Guest felt recent efforts to act against rogue clinics, educate patients and promote good practice has resulted in improved “adherence to accepted practices globally.” This is good news to go home on.


Monday, December 3, 2012

Legal and Policy Challenges to Stem Cell Research

An afternoon session at the World Stem Cell Summit turned its attention to legal and policy issues impacting stem cell research. Beth Roxland from the Hofstra University Bioethics Center described a lawsuit (Feminists Choosing Life v. Empire State Stem Cell Board) against New York's stem cell research program. The lawsuit had to do with New York's funding of what's commonly known as therapeutic cloning, or somatic cell nuclear reprogramming (SCNT). This is the technique that was used to create cloned animals such as Dolly the sheep, but can also be used to create embryonic stem cells in many types of animals. So far, nobody has succeeded in creating human embryonic stem cells through SCNT.

The basic argument by Feminists Choosing Life was that SCNT research indirectly supports human reproductive cloning. New York, like California, has explicit language in their law banning reproductive cloning. Feminists Choosing Life attempted to argue that SCNT research may result in knowledge that would allow others to attempt reproductive cloning in the future. Fortunately, the court found the explicit ban clear and compelling and that the reasoning of indirect support was speculative and not grounds for preventing legitimate stem cell research.

Roxland then turned her attention to Federal legal issues. Keep in mind this is a national meeting and many of the participants come from state that do not have comprehensive laws like California and New York, so federal laws have a major impact. She described August 2012 Federal Appeals court decision unanimously upholding the current NIH policy for allowing human embryonic stem cell research. She noted that the decision has been appealed to the Supreme Court so we will not know if the Appeals Court decision holds until early 2013.

Matthew Vincent of Advance Cell Technology then discussed how the Federal policy environment has impacted ACT's work on developing hESC-based treatments for people with macular degeneration. Before discussion policy issues, Vincent first described how one patient receiving the treatment has visual gains and continues to show improvement (we blogged about those findings here). Matthew described how ongoing litigation and federal policy governing hESCs has resulted in the company not using Federal funds. Vincent said, “There is a lot more we could have done” were it not for Federal policy considerations. The good news is that if the Appeals Court decision holds then the path may be cleared for more research.


Advances in stem cell research: like going from a landline to an iPhone #wscs12

Sally Temple of the Neural Stem Cell Institute showed this slide during her talk at the World Stem Cell Summit comparing telephone technology since she started in the laboratory until now. She says the same leap of technology has occurred with cell research and stem cells.


Near-term use for iPS cells: repurposing existing drugs

George Daley opened the World Stem Cell Summit in West Palm Beach, Florida
Geoff Lomax is CIRM's Senior Officer to the Standards Working Group

Dr. Daley kicked off the World Stem Cell Summit by describing the strengths and opportunities for the field. To illustrate near-term strengths, he described his research on  a disease that results in bone marrow dysfunction -- Shwachman Diamond Syndrome. They used cells taken from people with the disease and turned those into an embryonic-like stem cell called an iPS cell to study in a lab dish how this disease can result in bone marrow failure.

Based on what this research taught them about how the disease forms, they found an existing drug that could prevent the disease from taking place in those cells in the lab dish. These studies suggest that the drug may be a good therapy for Shwachman Diamond Syndrome patients. His group is now trying to learn whether the drug that works so well in the lab dish might be able to treat the disease in people.

Dr. Daley's bigger message was that the greatest near-term opportunity for iPS cell research is their ability to help us repurpose existing drugs to treat diseases.


World Stem Cell Summit kicks off: opportunities and threats to the research

A packed crowd at the World Stem Cell Summit keynote address.
The World Stem Cell Summit is taking place right now in West Palm Beach Florida, bringing together scientists and patient advocates to discuss the future of stem cell research. We'll be posting updates throughout the meeting.

The World Stem Cell Summit began in West Palm Beach this morning with a fascinating keynote address by George Daley, MD, PhD of Children's Hospital Boston. He decided to do a SWOT analysis of the field of stem cell research. For those of you, like me, who skipped statistics at school, this is a structured method to measure the Strengths, Weaknesses, Opportunities and Threats (hence SWOT) of a project.

In this case Dr. Daley said the Strength of stem cell research is that it is exciting science and has enormous potential for leading to therapies and cures.

The Weaknesses are that we have a limited understanding of how stem cells, particularly adult stem cells, work. We know a lot about them but there's so much crucial information we don't have - such as how do we consistently guide stem cells into changing into the kind of cell or tissue we want. He also pointed out that, as yet, we don't have a business model for how to deliver these cell therapies to patients

When it comes to Opportunities he highlighted a number of areas where we are already doing exciting work that is showing evidence of paying off. For instance with induced pluripotent or iPS cells, we have been able to use these to create "disease in a dish" models, turning the cells into the kind of cell found in patients with deadly diseases, then using those cells to better understand the disease and even to test what drugs might work against it. He pointed to encouraging advances in treatment of vision problems, spinal cord injury and Parkinson's disease. He cautioned that he is not talking about cures, but about small advances, benefits that while not helping someone in a wheelchair get up and walk, will help improve the quality of their life

The Threats are something we at the stem cell agency have been talking about for some time, the unproven and premature use of therapies in patients. This can be either overseas as part of the "stem cell tourism" issue, or in the US with clinics and individuals offering treatments that are not proven and may not be safe. These providers typically attract customers by magnifying the benefits and trivializing the risks.

Having done his analysis Dr. Daley said as much as we would like to say otherwise, right now the only proven stem cell therapies are for blood indications, such as bone marrow transplants and he said that in order to protect people we need to "prohibit the direct marketing of unproven therapies."

But despite the threats and problems he remains optimistic. He said we are seeing a remarkable increase in the number of stem cell therapies heading into or that are already in clinical trials, in the US and globally. And he ended by posing a single questions: Can the objectives we have set ourselves be met? And he answered with a resounding YES.

A promising start to what looks like being a fascinating couple of days.