Wednesday, October 31, 2012

On stem cells, athletics and the world series (Go Giants!)

Today the San Francisco Giants will be celebrating their World Series victory with a parade that will draw hundreds of thousands of fans to AT&T park, directly across the street from CIRM headquarters.

With sports so much on our minds here at CIRM, this seems like a good day to talk about stem cells in athletics. The topic made news recently thanks to a story written by Timothy Caulfield in The Atlantic. Caulfield, who is a professor at the Faculty of Law and School of Public Health, University of Alberta, is a long-time follower of stem cell research, particularly the trend of stem cell tourism where people travel overseas for unproven stem cell injections.

One point I was glad to see him make is the question of what, exactly, is a “stem cell treatment”? It’s a pretty generic term for a wide range possible treatments. A bone marrow transplant is by far the most common stem cell therapy because it replaces a person’s own blood-forming stem cells with donor stem cells that recreate the person’s blood system. This type of stem cell transplant is commonly used to treat blood disorders like leukemia. Other types of stem cell therapies in development involve transplanting neural stem cells to treat brain disorders and transplanting cells derived from embyonic or iPS cells to treat a wide range of diseases. There is no one "stem cell treatment".

In the case of sports, most so-called stem cell treatments are really just a matter of taking tissue from one part of the body (fat, for example) and injecting it somewhere else (like a shoulder, knee or elbow). Whether or not stem cells are actually involved is unclear. As Caulfield writes:
In fact, it is an open question in the research community whether this work should truly be considered "stem cell" therapy. As noted by colleague Mick Bhatia, Director and Senior Scientist McMaster Stem Cell and Cancer Research Institute, "these injury therapies lack any evidence to indicate 'stem cells' by any definition or means are being used. Any therapeutic effects noted are most likely from any cell type being injected, including cell lines, that would cause local anti-inflammatory response that is both transient (days) and does not involve any stem cell biology." His blunt conclusion: "Lots of stem cell conclusions here are bogus all the way around ... The treatment fetches a lot of money by claiming a stem cell therapy is being used."
He goes on to blame the media for talking up the athlete’s treatments as “stem cell therapies”, suggesting that these stories help market unproven, unregulated clinics overseas. For those who haven’t been following stem cell tourism, we have more information on our website and recently shot this short video with stem cell scientist Larry Goldstein at UCSD:

Despite his concerns about overblown claims among athletes, Caulfield says he’s optimistic about the field of stem cell research.
To be clear, there is great promise in this area. I firmly believe that stem cell treatments will, one day, help athletes, both professional and recreational, recover from injury. Indeed, there are teams of researchers all over the world, some funded by the NFL, working on this right now. But we aren't there yet.
It's good to see Peyton Manning back on the field leading Nobel-Prize-worthy no-huddle comebacks. But can we thank stem cell research? Unlikely.
And on that note, we’re enjoying the giants victory and the cheering crowds outside our offices. And we’re looking forward to the day when stem cell therapies are available for both critical conditions like diabetes, blindness, and Altzheimer’s disease as well as for athletic injuries.


Tuesday, October 30, 2012

Relationship building in San Diego: Bringing investors into regenerative medicine

CIRM is cosponsoring the Investor and Partnering Forum at the Stem Cell Meeting on the Mesa in San Diego. It's an effort to build relationships between investors, companies, and entrepreneurial academics.

Stem cells have been in the news a lot lately thanks to Nobel prizes and scientific discoveries. But for many biotech companies all the good news is scant consolation if they can’t get the money they need to do the research that's required to bring promising therapies to market.

That message was driven home on the first day of the annual Stem Cells Meeting on the Mesa  at the Sanford Consortium for Regenerative Medicine in San Diego. It’s a gathering of some of the top scientific researchers, industry pioneers and investors. The goal is to bring together the leaders in the field to create new partnerships and advance stem cell science into cures. But when Robert Parlay, Chairman and CEO of stem cell manufacturing company Cellular Dynamics International, got up to talk to the audience and asked for a show of hands from all the investors in the room, only a few people in a room of a few hundred raised their hands.

It was a powerful visual indicator of just how difficult it is for even leading biotech and other companies to attract the investment they need. It’s also a reminder of just how important the stem cell agency is in filling that gap. Without our funding the picture would be even more grim.

Just last week the CIRM governing board approved almost $20 million in funding two biotech companies – ViaCyte, Inc. and bluebird bio – in the first round of our Strategic Partnership Award initiative (here's our press release). It’s an effort to attract more industry engagement and investment into stem cell research.

Ron Leuty of the San Francisco Business Times recently interviewed Bluebird Bio chief medical officer David Davidson about the company's CIRM funding. He said:
Partnering with California and our investigators in California is essential for the success of this study. California has more patients with beta-thalassemia than any other state. There are world-renowned experts in the field there who we are working with. This is really essential for the success of the study.
Stem Cells on the Mesa shows that there are many companies who would love to be more engaged, who would love to be able to do more, all they need is the money.

Many speakers at the meeting spoke out loudly in praise of CIRM and the funding we provide to help drive the most promising science towards clinical trials. Jonathan Thomas, CIRM Chairman, echoed those sentiments saying: “One of the things we talk a lot about at CIRM is how we can work even more closely with industry, helping companies advance the ball.”

That doesn’t just mean with funding from us but also with advice on how to navigate the regulatory process, how to work with the FDA, and how to partner with the big pharmaceutical companies who have access to enormous resources

“We are placing real emphasis on trying to get Big Pharma to work with companies earlier on in the process,” Thomas said. “We have Big Pharma coming to us, looking for introductions to the most promising work. We are happy to do that because it gives them access to good projects and gives the researchers and industry access to the funds they need to take their products to market.”

It’s exciting being at a meeting like this, filled with so many talented people who are passionate about the work they do and believe in the promise of stem cell research to deliver therapies and cures.

As Jonathan Thomas pointed out, events like this are a reminder why the people of California are so important to the future of stem cell research. Their support makes it possible for meetings like Stem Cells on the Mesa to take place, and possible for everyone involved to talk in terms of cures instead of lost opportunities.


Friday, October 26, 2012

Diabetes stem cell therapy moving toward clinical trials

Yesterday, at a meeting of our governing board – the Independent Citizens Oversight Committee (ICOC) – we were delighted to see a project we have helped nurture from a cool theory into a truly promising therapy head towards clinical trials in people. The ICOC approved funding for a ViaCyte stem cell therapy for type 1 diabetes as part of our new Strategic Partnership Award initiative.

This initiative is an effort to attract more engagement and investment from industry in stem cell research (we blogged about that initiative here). ViaCyte got more than $10 million. Bluebird Bio got $9.3 million for research into Beta-thalassemia, a potentially deadly blood disorder.

We have worked with ViaCyte on this therapy over several different funding awards and at different stages of development of the product. Now, it’s going into a critical phase, finishing off pre-clinical and starting clinical testing. You can see summaries of all the awards we've given to ViaCyte to-date, plus a summary of the Bluebird Bio award on our website.

After the governing board voted to approve the award, Paul Laikind, PhD, President and CEO of ViaCyte thanked the board and the people of California for making this possible, saying:
“You have allowed us to carry on our ground breaking research which we hope will transform the treatment of diabetes in California and around the world. Thanks to CIRM this could lead to therapies curing type 1 and helping those with type 2 diabetes.”
CIRM had Dr. Laikind said CIRM funding helped ViaCyte greatly increase the size of its workforce in California, and to use our support as leverage to attract even more funding from other organizations, including the European union. That means more money coming into the state, and more jobs for Californians.

What was also gratifying was to hear from Jason Gardner, PhD, head of Regenerative Medicine at global health care company GlaxoSmithKline (GSK). They are in negotiations with ViaCyte to help take this therapy through the rest of its clinical trial phase and, hopefully, through to FDA approval.

This would be the first time that one of our grantees has partnered with a global pharmaceutical company of the size of GSK on a product we have helped support. It is potentially a hugely important step. Dr. Gardner reflected on the value of those alliances when he said:
“We are aligned on three levels; that cell-based regenerative medicine can be transformative, that this path is complex and high risk, and that this is best shared by those who have shared goals.”
CIRM President Alan Trounson, PhD echoed those feelings when he said
“This is one of the most important steps we have taken here in terms of bringing these therapies to patients. To have a company, a major organization like GSK consider being a partner with us on a program we have shepherded through basic science into the clinic is validation of our program. I think this will resound throughout California and the US. I hope this will be one of those moments we can look back on and say this made Proposition 71 worthwhile not just for California but, I would say, for the whole world.”
That’s pretty heady stuff. But when you consider that worldwide almost 350 million people have diabetes, and those numbers are rising each year, then a therapy like this has the potential to change the world around us.

Last year we produced this video describing ViaCyte's diabetes project.


Thursday, October 25, 2012

UCLA scientist shares stem cell research with the next generation

CIRM grantee at UCLA Michael Teitell spoke with high school students on Stem Cell Awareness Day

Michael Teitell working with one of his lab students
When speaking with high school students one learns to always expect the unexpected. Such visits can be exhilarating and surprising and are never boring. I was asked to write a guest blog for CIRM because of a wonderful interaction I had recently with a biology class at Crescenta Valley High School in La Crescenta, California, as part of the CIRM sponsored Stem Cell Awareness Day. It is inspiring to talk to young people about stem cell research and the work we do at the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. Yet, this visit was special because of the very open and interactive discussion the students and I had that day.

The students shared with me their knowledge of the basics of stem cell biology, so we skipped over the usual introductory material and instead we engaged in an in-depth discussion of the cutting edge work supported by CIRM, including research at UCLA’s Broad Stem Cell Research Center (those awards are listed here), and the potential real-world application of stem cell research in the development of treatments and cures for a wide range of diseases. Among the topics that particularly energized the students was that of induced pluripotent stem cells, by coincidence the discovery that resulted in the 2012 Nobel Prize for medicine a few days later.

One student wrote to me afterwards saying:
‘The thing that interested me most was {that Dr. Yamanaka} discovered how to take mature adult cells and turn them into stem cells. I find that backwards process fascinating. This presentation me consider an occupation in stem cell research.”
The students were surprised and fascinated to learn that tissue-specific cells such as skin or blood could be reprogrammed to be pluripotent stem cells. Their questions were sharp and thoughtful and I found them very curious about the subject. Some of the students said that our discussion inspired them to possibly seek careers in molecular biology and stem cell research. One wrote:
“Before today I had very little intuition about stem cells and was not really interested in that field of study. However, your lectures totally changed my attitude towards the topic. When I got home, I spent three hours on my computer researching about stem cells. Every website I opened just increased my interest about stem cells.”
Other questions dealt with life in college, getting into college, and the students asked about doing research, such as pursuing a career in biology. As a member of a college admissions board, I noted that getting into a college they wanted and doing well there depended upon more than just studying and getting good grades. The college admissions reviewers will want to see demonstrated leadership in extracurricular activities inside and outside of high school to help assess whether the students will be productive members of the academic community. For some of the students it was important to have an opportunity to see inside this college experience:
“I found your background information and advice about college very inspirational. I gained a better understanding about what college was like and what I need to do to get where I want.”
I received a flood of emails afterward, one from nearly every student. I was impressed by the maturity, thoughtfulness and sincerity of their responses.

As the students discussed the amazing advances we are making in stem cell research it was clear that they are inspired by science. In turn, I was inspired by these students as they potentially represent the next generation of scientists who will build on our many current discoveries and transform stem cell research into the discoveries and treatments of the future.

Michael Teitell, MD, PhD
Eli & Edythe Broad Center of Regenerative Medicine and Stem Cell Research
Department of Pathology and Laboratory Medicine
University of California, Los Angeles

Wednesday, October 24, 2012

Women in science get a boost from women in online media

Astronomer Caroline Herschel was one of those to get full entry on Wikipedia.
Tielemann, 1829/W. J. Herschel/The Royal Society
As a former woman in science and a current woman in online media, a story from Nature this week struck home. They write about an edit-a-thon held by the Royal Society in London, which brought in 15 female editors to expand Wikipedia’s representation of women in science. It turns out that women are underrepresented both places – only 15% of Wikipedia editors are women.


Father of bone marrow transplant -- first stem cell therapy -- dies

By far the predominant stem cell therapy today is a bone marrow transplant. The 60,000 people who receive a transplant this year have Nobel Laureate E. Donnall Thomas to thank for the treatment.

Thomas, who died this week, began working on bone marrow transplants back in the 1950s, when the prevailing wisdom held that no human organs could be transplanted, much less bone marrow. An obituary in the Seattle Times quoted Thomas:
"In the 1960s in particular and even into the 1970s, there were very responsible physicians who said this would never work. Some suggested it shouldn't go on as an experimental thing."
When the millionth person receives a transplant this year, I’m sure that person will be grateful that Thomas ignored the conventional wisdom and continued those experiments.

A bone marrow transplant works because blood-forming stem cells in the bone marrow, taken from either from a donor or from the patient, are transferred to someone with leukemia or other blood diseases, and those stem cells replace the person’s diseased blood system with a healthy one. Since the 1970s, when the first transplant between unrelated people was successful, scientists have incrementally improved the risky technique and expanded its range of use.

CIRM is part of that expansion. Our grantees are developing new ways of genetically engineering the blood-forming stem cells before transplantation to fix genetic diseases like sickle cell disease or to make cells resistant to HIV. Other scientists are working with cord blood cells, which contain the blood-forming stem cells taken from a baby’s umbilical cord at birth. These cells, like adult blood-forming stem cells, have proven widely useful in treating a range of diseases.

Today, diverse stem cell types are now beginning to enter clinical trials. Neural stem cells from the brain, mesenchymal stem cells from bone marrow or fat, and cells generated from embryonic stem cells and reprogrammed iPS cells are among those that are either in or nearing clinical trials.

When Thomas’ work entered clinical trials in the 1950s and 1960s, nobody knew how many lives that controversial therapy would save. Fifty years from now, I wonder which of today’s prospective therapies will be as commonplace. We won’t know until stem cell scientists do as Thomas did: keep trying.

Here’s a good resource if you want to learn more about bone marrow transplants.

Here are resources to learn about research by CIRM grantees working towards bone marrow transplant therapies for sickle cell disease, HIV/AIDS, and SCID (bubble boy disease).


Tuesday, October 23, 2012

Who's the boss of the brain? How stem cells repair damage

Tony Wyss-Coray
In addition to their usual job of creating new cells, stem cells in the brain turn out to be excellent managers. That’s according to CIRM-funded researchers at Stanford University, who have recently published a paper describing how stem cells in the brain control the behavior of other cells.

The work could help explain why stem cell transplants appear to improve brain function even when they don’t form connections with other brain cells.

The paper itself, which was published in the journal Nature Neuroscience, has a lot of talk about cells in the brain communicating via a variety of acronym-laden molecules. What it all comes down to is this: stem cells in the brain and the cells they create (with the cumbersome name “neural progenitor cells) are the managers of a vast army of other cells. Damage the stem cells and you disrupt how those other cells behave.

Bruce Goldman at Stanford University described the work in superhero terms in a press release. Imagine a group of cells in the brain whose job it is to swoop around cleaning up damage (these cells are called microglia). Most of the time, they are like Clark Kent, puny and inactive. But when the neural progenitor cells give the signal they puff up and go on the attack.

One thought has been that disorders such as Alzheimer’s and Parkinson’s as well as damage after stroke might result, in part, when those superhero cells aren’t properly called to action. This could explain why transplanting stem cells into brains of people with those diseases might help improve symptoms. It’s like dropping a good manager into a disorganized office. Where once other cells meandered about without purpose, the neural progenitor cells instill order and put those microglia to work.

Goldman also wrote an entry on Stanford’s blog about the work, which contains possibly the clearest description of neural stem cells and their progeny that I’ve read. He writes:
Neural stem cells get plenty of good press, and understandably so. They’re the matriarchal cells of the brain, from which spring all except one type of cell populating our most highly regarded (at least by itself) organ. They can remain in their primordial state for decades, languidly dividing just enough to replace their own numbers. Alternatively, they can spawn daughter cells that depart from the primordial state.
It’s the matriarchs’ daughters – so-called neural progenitor cells – that embark on committed differentiation pathways giving rise to nerve cells and other key brain cells. Given that lofty ambition, it’s not surprising that neural progenitor cells divide much more rapidly than their parents do, outnumbering neural stem cells probably by 1,000 to 1 or more.
It turns out that neural progenitors can do more than breed. They’re excellent managers, too.
This work was funded by a CIRM Basic Biology awards, which are intended to fund research that better explains how stem cells function. This study is a great example of why those awards are so valuable. It’s hard to convince the Food and Drug Administration to allow you to test stem cells in people if you can’t also explain how those cells are working. This research begins to get at why transplanted stem cells appear to work, and could help other researchers move closer to testing their therapies in people.

CIRM funding: Tony Wyss-Coray (RB2-01637)


Monday, October 22, 2012

Testing iPS cells in people? Not yet

This past week a story unfolded that ended with the dismissal of a researcher from the University of Tokyo who had claimed to be the first to test a therapy based on reprogrammed iPS cells in humans.

It all began last week when CIRM grantee and UC Davis scientist Paul Knoepfler blogged about research being reported by Hisashi Moriguchi. He chronicled the story on his website. New Scientist describes the situation:
In a poster presented at a meeting of the New York Stem Cell Foundation, Moriguchi – who claimed to work at Harvard Medical School and the University of Tokyo – described results from a trial in which cardiac muscle cells were grown from induced pluripotent stem (iPS) cells, and transplanted into six US patients with severe heart failure.

… This was surprising, given the safety concerns that surround iPS cells – adult cells that have been reprogrammed to an embryonic state. Support for the claim quickly disintegrated: within hours, Harvard released a statement noting that Moriguchi had no current affiliation with the university, nor any ethical approval to run a clinical trial.
The supposed results got a lot of attention at the time because Shinya Yamanaka had just shared the Nobel Prize for his role in discovering how to make those very iPS cells that Moriguchi was claiming to have tested. Yamanaka works part time at the CIRM-funded Gladstone Institutes.

The problem is that he never did inject those patients. This is a big deal in part because CIRM and other organizations have been very critical of clinics claiming to be offering stem cell “therapies” based on no clinical trial data (here's more information about stem cell tourism). In the U.S., new therapy ideas have to be tested in trials approved by the Food and Drug Administration, and those trials also have to be approved by an Institutional Review Board at the institution participating in the trial. In this case, Harvard claimed that their IRB had never approved the research.

CIRM requires IRB approval for any clinical trial that involves our funds, just like we require institutional animal review board and stem cell review board approval for animal research or research involving human embryonic stem cells. We get those assurances before we issue funding and in our yearly progress reports from grantees.

These steps – institutional reviews, Food and Drug Administration reviews, controlled clinical trials – do take time, but they also ensure that once a therapy reaches patients it is likely to be safe. While we would all love to find a cure for any number of diseases tomorrow the hard fact is that good science takes time, there are no short cuts. In science as in life, if something seems too good to be true, it usually is.


Friday, October 19, 2012

Arlen Spector: Science policy driven by evidence, not rhetoric

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

Sen. Arlen Spector
In August 2001, I was frantically working to put the final touches on my doctoral dissertation focused on ethical and policy considerations related to the use of genetic testing for disease risk. I always viewed the ethics policy discussion as rather nuanced and academic, so it was quite a surprise to hear President Bush state in his first prime time address that: Research on embryonic stem cells raises profound ethical questions.  I knew nothing about the field of stem cell research at the time, but I decided to take notice given that research ethics had come to the forefront of the national policy discussion.

For an aspiring student of research policy, it was essential to follow how the discussion played out. I had experience in a number of state and national legislative campaigns and was keenly aware how rhetoric can trump substance in policy debates.

In 2001 -2002, the most substantive policy deliberations were taking place in the United States Senate. One senator in particular, Arlen Specter, stood out because of his cogent questioning designed to elicit answers to core policy questions. In a 2002 hearing Senator Specter focused on whether under the Bush policy there a sufficient number of stem cell lines available for the required research. It seem quite remarkable that so much attention was being given to whether particular cell lines were sufficiently robust for research on Alzheimer’s and Parkinson’s disease. One of the most striking aspects of these deliberations was Senator Specter’s effort to move past rhetoric and get a complete picture of the policy issues.

This quality in Senator Spector extended to all his work, as the Los Angeles Times noted in their obituary of Spector after his death this week. They wrote,  "he habitually asked probing questions of nominees from both parties instead of succumbing to the rhetorical approach favored by his colleagues."  

He brought this probing approach to NIH budget deliberations to document the value of disease treatment and prevention. The result was a doubling of the NIH budget over five years and Francis Collins, director of the National Institutes of Health (NIH), described Specter to The Scientist as “a towering champion for biomedical research and the mission of the NIH.”

Former CIRM Governing Board chairman Robert Klein described Spector as a man of character and conviction. I hope one legacy of Senator Spector’s career is the conviction that issues of science and research policy should be informed by substantive deliberations. Science is fundamentally driven by rules of evidence, and science policy should be no exception.


Wednesday, October 17, 2012

Getting approval for stem cell clinical trials: a step by step guide

Ellen Feigal, M.D. Senior VP for
Research & Development at CIRM
Trying to move a promising therapy from the lab into clinical trials in patients is one of the most challenging parts of any drug discovery process. But when that new therapy involves a stem cell or cell-tissue it can be doubly so. You are not only dealing with questions of whether it’s safe (always good to know) and whether it works (rather important too) but you are also dealing with a whole new area of science that adds extra questions and complications along the way.

That’s why CIRM teamed up with the Alliance for Regenerative Medicine (ARM) to come up with a new step-by-step guide on how to navigate the tough regulatory waters and get approval from the Food and Drug Administration to move a therapy into clinical trials. The result is a white paper published in the journal Stem Cells Translational Medicine.

It lays out precisely what a company needs to do, when, why and how if it wants to get FDA approval.

As Ellen Feigal, MD, CIRM’s Senior VP for Research and Development and the lead author of the article, points out in the paper:
“The field of regenerative medicine is still relatively young and involves new and novel science. The products involved might be classified as a biologic - such as human cells and tissues or gene therapy- a device, a drug or combination of those. They also span a range of areas from biology to chemistry and physics and so could come under multiple regulatory agencies. All that can create uncertainty and confusion. The goal of this paper is to help companies understand the complexity of the process and how to most effectively navigate through it.”
In our press release we quote Michael Werner, Executive Director of the Alliance for Regenerative Medicine, and a co-author of the study:
“Everyone involved in this process, whether it’s a company or the FDA, has a shared goal of bringing safe and effective therapies to the public as quickly as possible.”
The article is comprehensive but even with this information companies will face lots of challenges along the way But by helping take some of the uncertainty out of the process we hope to make it easier for companies to get their therapies where they are needed the most, in patients.


New Alzheimer’s Ask the Expert video: Of stem cells, iphones and a cellular black box

Back in July, we kicked off our new Ask the Stem Cell Expert video series by interviewing UC San Diego’s Larry Goldstein about stem cell research and Alzheimer’s disease (see our video here and previous blog here). We posed questions to him that had been sent in by followers of our Facebook, twitter, and blog pages. Dr. Goldstein is a great explainer and had so many thoughtful, easy-to-understand answers that we had more than enough material for the final edit. Because the unused footage was equally terrific, today we’re posting the extra interview clips in a second video:

It’s no wonder that Goldstein is the author of Stem Cell for Dummies because he really has a knack for simplifying complex topics. One of my favorite segments in this video is when Goldstein uses an iPhone analogy to answer the question, “why can’t you just inject stem cells directly into the brain to treat Alzheimer’s or other neurological disorders?” He points out that simply injecting the unspecialized cells into the brain would be like injecting metal into a broken iPhone and expecting it to be repaired:
Ordinarily when the brain is built is when you’re a fetus. And there are all sorts of signals that say how to build the brain that aren’t there when you’re an adult. So let’s take an example of your iPhone. So when your iPhone is being built there are all sorts of manufacturing instructions for wiring up pieces of the circuit boards but once you’ve bought the phone, those “signals” those manufacturing instructions don’t come along with the phone. That’s the “adult” phone. And so if part of it breaks just injecting metal into the phone is not necessarily going to repopulate the circuitry of the phone in a proper way even though metal is the raw material that built the phone. So we have to do something much more sophisticated than just putting the raw materials in.
When asked about his own lab’s Alzheimer’s research, he drew another intriguing analogy. This time he made the comparison between a black box data flight recorder and his lab’s recent work in human brain cells to study Alzheimer’s disease. It might seem like a no-brainer (pun intended) to study human cells for a human disease but as Goldstein explains:
that’s harder than it sounds. I can’t just take a sample of your brain out and study it and you [may not] have developed the disease yet anyway. And once somebody’s died of the disease it’s like studying a plane crash after the plane’s hit the ground. You can learn a lot of about a plane crash from studying the pattern of wreckage on the ground but what you really want is the black box. You want a record of what was happening in the cockpit when the plane was getting ready to go down or just before it did.
To get at that black box, Goldstein’s lab takes skin cells from Alzheimer’s patients and reprograms them back into a stem cell-like state. Those cells are then matured into brain cells, providing a human model of Alzheimer’s disease. This cellular reprogramming technique is the very same that won Gladstone scientist Shinya Yamanaka the 2012 Nobel Prize in medicine earlier this month (see our previous blog). With these cells in hand Goldstein is in a position to identify, at a biochemical and genetic level, abnormal behavior of the cells that may be indicative of Alzheimer’s and to test drugs that could slow down or even reserve disease progression.

All of this is to say we enjoyed our visit with Larry and we hope people out there learn a lot from these videos. So far, the first Ask the Expert video has well over 1000 views so we take that as a good sign and we’re excited to continue this video series.

In fact, later the month we’ll interview Xianmin Zeng, a CIRM grantee who is developing stem cell-based treatments for Parkinson’s disease at the Buck Institute for Age Research. If you have questions about stem cell therapies for Parkinson’s disease, post them in the comments section here, to our Twitter feed @CIRMnews with the hashtag #askCIRM or to our Facebook page.


Tuesday, October 16, 2012

CIRM-funded researchers restore “balance” to brains disrupted by epilepsy, Alzheimer’s, Parkinsons

Arturo Alvarez-Buylla, Ph.D.
An unexpected finding by a CIRM-funded team at UC San Francisco raises new hope that neurons grown from stem cells could be transplanted into patients’ brains and survive in sufficient numbers to help the patients.

The team worked with a specific type of mouse neuron that helps control the cell-to-cell chatter that goes on constantly in our brains. These cells, called GABA-secreting interneurons, balance the actions of other neurons in the brain. Epilepsy, Alzheimer’s, Huntington’s, Parkinson’s and schizophrenia have all been linked to disruptions in this balance. Prior research has shown that implanting new interneurons in some animal models of these diseases can improve symptoms.

Despite evidence that these transplanted interneurons can improve symptoms, researchers had assumed that approach would not be an effective form of therapy. Most in the field had thought that the brain only had room for so many interneurons and transplanted healthy neurons would have to compete with the defective native neurons for space.

What the team found was quite different than expected. No matter how many new interneurons they implanted, the same proportion always survived, suggesting it will be easier than folks thought to get enough nerves to survive to impact patients’ disease. The team, led by Arturo Alvarez-Buylla, published their findings last week in Nature:

A press release from UCSF quotes Alvarez-Buylla:
“[This constant rate of survival] suggests that these cells, which other collaborative studies have shown have great therapeutic promise, can be added to cortex in significant numbers.”
CIRM funding: Derek G. Southwell and Mercedes F. Paredes (TG2-01153); Arturo Alvarez-Buylla (TR2-01749)


ResearchBlogging.orgSouthwell DG, Paredes MF, Galvao RP, Jones DL, Froemke RC, Sebe JY, Alfaro-Cervello C, Tang Y, Garcia-Verdugo JM, Rubenstein JL, Baraban SC, & Alvarez-Buylla A (2012). Intrinsically determined cell death of developing cortical interneurons. Nature PMID: 23041929

Monday, October 15, 2012

Privacy and progress in medical research: CIRM standards look forward

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

The commission that advises the President on bioethical issues recently issued a report titled Privacy and Progress in Whole Genome Sequencing. The report concludes “that to realize the enormous promise that whole genome sequencing holds for advancing clinical care and the greater public good, individual interests in privacy must be respected and secured.”

The commission’s report is a timely one. Sequencing a person’s DNA – also called whole genome sequencing – can help doctors and scientist better understand the unique qualities of a disease, and the technology is becoming more common in biomedical research. For example, National Public Radio had a good story recently about how genome sequencing helped doctors provide a pair of California twins with a highly effective treatment for a rare condition called DRD.

Despite these benefits, whole genome sequencing also runs into issues of privacy. Concerns arise over the possibility of discrimination from health insurance or jobs when a person’s entire genetic sequence becomes available.

The report make a series of recommendations designed to ensure the responsible conduct of genomic research. In a blog post, Commission chair Amy Gutmann indicated “this is a forward-looking report. It’s not a response to a crisis.”

Gutmann also indicated, “the Commission’s recommendations clump into two big categories, one is consent to whole genome sequencing and to the possible findings and how they will be treated, and the other is oversight.” Consent is the process researchers use to get permission from individuals to participate in research. Oversight typically involves a group of independent experts reviewing research studies to ensure they are being conducted safely and ethically.

Reading the Commission’s recommendations reminded me of the forward-looking nature of CIRM’s existing requirements, particularly as they apply to privacy. These requirements embodied in our Medical and Ethical Standards Regulations first we put in place in 2006. The regulations were recommended by our Scientific and Medical Accountability Standards Working Group. The Working Group placed special emphasis on the need for both comprehensive consent and effective research oversight.

CIRM has extensive requirements for consenting individuals for research, and our regulations created a new oversight body – the Stem Cell Research Oversight Committee. Shortly after the standards were enacted we published an article on some of their most innovative aspects.

The Standards Working Group has continued to build on this framework to ensure CIRM policies embody the most current thinking related to research ethics and oversight. For instance, the Presidential Commission emphasized the need for the consent process to include information about DNA sequencing and whether findings could be communicated back to research participants. The Standards Working Group took a similar position when it was discussing the informed consent process for CIRM’s Human Induced Pluripotent Stem Cell Initiative.

In May, I blogged about our efforts to develop an effective informed consent process. The model informed consent document CIRM recommends for its grantees includes information about DNA sequencing and asks cell donors whether they would like to have information communicated back to them.

The CIRM Standards Working Group will continue to look forward to support CIRMs mission of accelerating stem cell science under the highest standards. The Presidential Commission’s most recent report reassures us that our standards recognize and respond to the most pressing issues in genomic privacy.


Friday, October 12, 2012

CIRM president Alan Trounson remembers cloning pioneer Keith Campbell

Keith Campbell
This week Keith Campbell, one of the scientists responsible for cloning Dolly the sheep, died at the age of 58. The New York Times ran an extensive obituary about his contribution to science.

CIRM President Alan Trouson was a friend and scientific colleague of Campbell's. He wrote the following in celebration of his friend's life.

I first met Keith in his early days at the Roslyn Institute, Edinburgh. He was such an energetic and charismatic person immediately became a friend and colleague. He had such a profound belief in what he was doing and a simply effervescent way of drawing you into his immediate circle of friends. We talked of how to reprogram nuclei as a result of his work in amphibian and then mammals.

Given the most recent Nobel awards, he was within a whisker of being recognized as a major world figure in the science of cell reprogramming. Perhaps he felt that he didn't get all the recognition he deserved for his work in mammal cell cloning. Those of us who knew him well understood the importance of his contributions.

There were many times at frequent scientific meetings on animal reproduction, embryology and stem cell biology where I would gravitate to his company – he was such good company and genuine fellowship.

I asked him to several meetings over the years and he always came. Often he was the life of the party (meeting), always willing to present interesting data looking at his recent experiments, and neat ways to address difficulties of technology and cell biology. He was a serious intellect and genuinely nice man who you could share your time with in a productive way. I wish I knew many more people like Keith Campbell.

There are always many ways to slide down the pole of political adoration and the community’s laudatory value tree. Keith had more than his three minutes of fame. He had friends who would have walked a mile for him to lessen his burdens. He had a family he spoke about—his girls were the light of his life and he cared very much for his close colleagues.

I shall always remember the busy Keith, intent on getting it right at work. He had passionate views on what would work, why it wouldn't work and on what you were doing wrong. He loved the merriment of time down, and was a beacon for those who wanted a good companion. He will be sadly missed but I can hear his voice raised in debate of a point of order in the discussions of why a cell would behave under conditions of reversion to a basic state.

One day we will all again be able to join him in those debates and hear his laugh at the merriment of his persuasion. Now I am sad he isn’t here to jovially castigate me for being too serious about my own reflections of a man I admired.

Alan Trounson,
CIRM President

Thursday, October 11, 2012

Yamanaka: "slow and clumsy" and other tales of his path to the Nobel

Shinya Yamanaka, courtesy of CIRM science officer Arie Abo, who took the photo at the annual meeting of the International Society for Stem cell Research
It’s not often you get to use the words “stem cell researchers” and “giddy” in the same sentence but that’s the only way to describe the mood at the Gladstone Institutes in San Francisco on Monday after the Nobel prize for Physiology or Medicine was announced. Shinya Yamanaka, who works at the Gladstone, was one of the recipients of the award and the champagne and cupcakes were flowing like, well, champagne at the news.

The Gladstone Institutes is a mere stroll from CIRM's offices in San Francisco and I was able to attend the festivities where the stories flowed freely too. R. Sanders Williams, the President of the Gladstone Institutes, talked about how Yamanaka began his career as an orthopedic surgeon in Japan, but quickly came to realize he wasn’t particularly good at it (he describes himself as “slow and clumsy”) so he quit and got a PhD to pursue his true passion, research.

At that point Yamanaka applied to around 50 research facilities for a job, without success. Fortunately the Gladstone decided to take a chance on the young doctor. Unfortunately Yamanaka’s first research project didn’t turn out quite the way he hoped. He was looking for a way to reduce cholesterol and decrease the risk of heart attacks. But the method he chose not only didn’t reduce cholesterol, it also increased the risk of liver cancer (this was in mice I should point out, not people).

Rather than get discouraged by this Yamanaka decided that if he wanted to figure out what went wrong, he would have to know a lot more about how blood stem cells worked. And that set him on a course that eventually led to Stockholm and the Nobel prize.

As to how he heard he won, Yamanaka was his usual humble self when he explained that he was at home trying to repair his dishwasher when his cell phone rang. He picked it up but says he didn’t understand much of what was said other than “Stockholm” and “Nobel prize”. “That was pretty much it” is what Yamanaka says about one of the most important moments in his life.

Yamanaka shares the prize with British researcher John Gurdon for his pioneering work on cell reprogramming in 1962 using frog embryos. Yamanaka jokes that at the time Gurdon was doing his experiments, he was an embryo too. He was born a few months after Gurdon announced his breakthrough.

When asked what he was going to do to celebrate he said he wasn’t sure – though he did think he might have a beer later.


Wednesday, October 10, 2012

Nobel prize follow up: all types of stem cells needed in the search for therapies

A colony of iPS cells generated from skin (courtesy of Kathrin Plath, UCLA)
Monday’s announcement that Shinya Yamanaka had won the Nobel prize for his discovery of how to turn adult cells into a type of stem cell that can form any other kind of cell in the body has focused attention on the potential of this work for understanding and treating life-threatening diseases. But in the excitement over the news some have tried to use Yamanaka’s work in creating what he calls induced pluripotent or iPS cells as a justification for halting research using embryonic stem cells.

Groups who oppose embryonic stem cell research have argued that since both types of stem cells can form all cell types of the body, embryonic stem cells are no longer needed. Focusing entirely on iPS cells would eliminate the creation of new embryonic stem cell lines, most of which come from 4-5 day old embryos left over at in vitro fertilization clinics.

One thing to remember is that the embryos used to create embryonic stem cells were created by people using IVF procedures to start a family. When their family is complete, people have a choice to make about what to do with the excess 4-5 day old embryos being stored at the IVF clinics. Some people choose to simply discard the extra embryos. Others choose to donate those embryos to other infertile couples, and some couples choose to donate those embryos to medical research including the creation of new embryonic stem cell lines.

Those embryonic stem cells can form all the cells of the body, as can iPS cells, which are created from adult cells. However, there are some significant differences between the two cells types (as we chronicle in previous posts here and here). Right now, there are a handful of clinical trials just starting to test whether cells created from embryonic stem cells can treat two forms of blindness, one called macular degeneration and one called Stargardt’s disease. There are so far no trials testing therapies based on iPS cells, though a few are expected to begin in the next few years.

Betting on one cell type over the other is like betting on a horse before the race starts. People do it, but it’s a gamble. In the case of new therapies, betting on one cell type over another is really like betting on hundreds of races all at once – one race for blindness, one race for Parkinson’s disease, one race for Lou Gehrig’s disease, and another race for diabetes. For all we know, one cell type might be best for creating cells needed in people with diabetes, while a completely different cell type will be best for repairing hearts. We won’t know until we do the research.

Yamanaka himself has been concerned that his work is being used to argue against ongoing research using embryonic stem cells. He co-wrote a paper in Cell Stem Cells titled “New advances in iPS cell research do not obviate the need for human embryonic stem cells.”

Other experts agree. A panel of scientists brought together by the U.S. National Academy of Sciences agreed that the use of human embryonic stem cells is still necessary. As the expert panel, chaired by Richard Hynes of the Massachusetts Institute of Technology stated “It is far from clear at this point which types of cell types will prove to be the most useful for regenerative medicine, and it is likely that each will have some utility.” (Here’s a link to that report.)

We agree. That’s why we fund research using all stem cell types. You can see a break down of funding to different cell types and to different diseases on our web site. Here’s a list of all our awards, which total $190 million, to researchers creating or using iPS cells. You can also read our story about why embryonic stem cells remain the gold standard against which other stem cells are compared.

In this race, we’re betting on the best science not preconceived ideas about which approach will work. We are also betting on the patients who are waiting for the results of that research.


Monday, October 8, 2012

Stem cell pioneers Gurdon and Yamanaka win Nobel Prize

Shinya Yamanaka
This year’s Nobel Prize for Physiology or Medicine went to two stem cell pioneers: Shinya Yamanaka and John Gurdon. The two scientists showed that mature cells such as those of the skin or intestine can be coaxed back to an embryonic-like state and can then form all tissues of the body.

According to the Nobel Prize announcement:
These groundbreaking discoveries have completely changed our view of the development and cellular specialisation. We now understand that the mature cell does not have to be confined forever to its specialised state. Textbooks have been rewritten and new research fields have been established. By reprogramming human cells, scientists have created new opportunities to study diseases and develop methods for diagnosis and therapy.
Shinya Yamanaka has a co-appointment at the CIRM-funded Gladstone Institutes in San Francisco (here’s a list of all CIRM awards to Gladstone scientists). Their press release discusses the importance of Yamanaka’s work in the development of new therapies:
Six years ago, Dr. Yamanaka discovered that by adding just four genes into adult skin cells in mice, he could induce the cells to become like embryonic stem cells. He called them induced pluripotent stem cells, or iPS cells. In 2007, he announced that he had done the same with human adult skin cells.
Embryonic stem cells—which are “pluripotent” because they can develop into any type of cell—hold tremendous promise for regenerative medicine, in which damaged organs and tissues can be replaced or repaired. Many in the science community consider the use of stem cells to be key to the future treatment and eradication of a number of diseases, such as diabetes, blindness and Parkinson's disease.
Gurdon, who shared the award, carried out classic experiments in the 1960s showing that the nucleus from an adult intestinal cell could be placed into an egg, which then formed a normal tadpole. Together, the two sets of experiments overturned conventional wisdom, which held that once a cell matured into an adult type such as skin or intestine it lost the ability to form any other tissues.

CIRM president Alan Trounson said:
"There are few moments in science that are undisputed as genuine elegant creativity and simplicity. Shinya Yamanaka is responsible for one of those. The induced pluripotent stem cells he created will allow us to interrogate and understand the full extent and variation of human disease, will enable us to develop new medicines and will forever change the way science and medicine will be conducted for the benefit of mankind. An extraordinary accomplishment by a genuinely modest and brilliant scientist. He absolutely deserves a Nobel award.”
Before Yamanaka’s 2007 announcement that he had reprogrammed adult skin cells to an embryonic state most stem cell research focused on embryonic stem cells or tissue-specific stem cells. Just five years later, CIRM alone is funding almost $190 million in awards developing better ways of creating iPS cells and using those cells to develop new therapies (the full list of iPS grants is on our website).

One CIRM disease team is attempting to use iPS cells to develop a therapy for the devastating childhood skin condition epidermolysis bullosa (here’s a link to that award). Those scientists hope to be in clinical trials in the next few years.

Since Yamanaka’s discovery there has been public debate about whether the reprogrammed iPS cells rendered embryonic stem cells unnecessary. The idea was that both cells had the potential to form all cells in the body and could therefore be used in similar ways to develop therapies.

In fact, many experiments have shown some differences between the two cell types. CIRM funds research with all types of stem cells – adult, iPS and embryonic – because until it’s clear which cell type is best suited for each type of therapy we don’t want to limit the options for getting those therapies to patients.

You can read more about iPS cells in previous blog entries:

Friday, October 5, 2012

Creating eggs from stem cells opens huge window onto mysteries of fertility

This morning’s news cycle has been filled with stories about the work of a Japanese team that created eggs from both embryonic mouse stem cells and mouse reprogrammed stem cells, iPS cells made from skin. My favorite headline came from the BBC “Skin Cells become Grandparents,” noting that the eggs were fertile producing offspring who were also fertile. But perhaps the most informative is in the Nature News blog here, covering the paper their rival Science published online yesterday.

Most of the coverage has raised many good issues about the work. They are rightfully noting it is going to be hard to translate this mouse work into humans, when we do it opens up new options for fertility treatment, but before then society needs to address some pretty big ethical and regulatory questions.

CIRM grantee Amander Clark from UCLA is quoted in the widely used Associated Press article making these points:

I think it’s a pretty large advance in the next generation of reproductive technologies for women. (Discussion about policy and regulation) needs to begin now.

But it is the Nature piece that addressed why the scientific community is so excited by this work. Many of the fundamentals of fertility, in particular how eggs and sperm mature are some of the biggest mysteries in biology. How do eggs and sperm end up with one copy of each chromosome instead of two like all our other cells? How do so many genes in eggs get silenced so that the eggs can remain dormant until it is their turn for ovulation?

Understanding how this happens in humans, not just mice, could open up significant new options for women. Another CIRM grantee, Stanford’s Renee Reijo Pera is quoted in the LA Times about the potential to allow women in their 30s and 40s to turn back their biological clock:

This is a get-them-back strategy.

CIRM funds Dr. Reijo Pera to study egg development. A colleague blogged about her work here and you can watch a video of her discussing it here.


Fortune favors California's stem cell agency

Sometimes one of the hardest things to do is to take a complex subject and tell it in simple, easy to understand terms without ‘dumbing it down’. It’s particularly true of science where concepts and language are often so dense you need a Thesaurus at your side just to understand what they are talking about. That’s why an article in the latest issue of Fortune magazine about the business of stem cell research was so refreshing. It delved deep into the science without losing any of the sense of hope and wonder at what it is the researchers are trying to do.

Full disclosure: we worked closely with the author of the piece, Jeffrey M. O’Brien, to help him understand the work that we do here at the stem cell agency, how we have used our funding to help create the infrastructure for a whole new industry in California, one with a potentially enormous payoff.
Now that the infrastructure is in place, CIRM is making a beeline for the clinic with $437 million in grants aimed at bringing therapies to trial within five years. The targeted diseases are a murderers' row, including Type 1 diabetes, heart failure, leukemia, and stroke. One recent grant recipient is expected to begin a clinical trial in post-heart-attack patients within months.
(There's more information about those projects nearing therapies, and the diseases they target on our website.)

O’Brien quotes our President Alan Trounson, PhD, and Chairman Jonathan Thomas, PhD, JD, in talking about the progress being made in California in pursuing promising therapies for deadly diseases.
Trounson has high-profile targets in mind as well. "The science stands up that we can cure HIV. My feeling is, let's get that done," he says.
(Here's more about the HIV disease team that's nearing clinical trials.)The author also points out the many challenges that stem cell scientists face, not just in delivering those therapies but also in creating a business model that will make this kind of research viable over the long term. But ultimately he remains convinced that there is no turning back from this path, that we are heading in the right direction. From our perspective, it’s hard to argue with that.
It's easy to imagine the benefits of stem cell therapies reaching even further. Scientists are doing their part, and there are signs that the government and the citizenry are increasingly excited. Will the money follow? The citizens of California have spoken. If my grandmother and I had the power to get the rest of the country to follow, we would.

Wednesday, October 3, 2012

Spreading the word on Stem Cell Awareness Day #stemcellday

Today is the fifth annual Stem Cell Awareness Day, being celebrated this year with events throughout California and in four additional states and five countries. CIRM is also coordinating an effort to teach high school students about stem cell research by having scientists visit classrooms. We expect to reach more than 100 classes in California today.

It has been fun to watch this event take off over the past five years. This year, in addition to public lectures and tours of stem cell facilities, we have the Irish Stem Cell Foundation launching a new website and kicking off a national media campaign, and EuroStemCell is promoting resources and activities for people planning stem cell events. Another event that stands out is one at Monash University in Australia, where in addition to live lectures they’ll be answering stem cell questions in real time on their blog.

The full list of worldwide events is available on the Stem Cell Awareness Day website.

Another new feature this year is a Stem Cell Awareness Day twitter hashtag #stemcellday that has been busy with organizations throughout the world promoting their events and resources. If you have a favorite resource for following stem cell research (and access to a Twitter account) post your links under that hashtag for others to use.

For those who are looking to get up to speed on stem cell research and CIRM’s role in accelerating the path to new cures, here’s some background information:

CIRM’s stem cell basics – Background information about the different types of stem cells and areas of research

Disease information – Fact sheets about CIRM-funded research in 22 disease areas

Stories of Hope – Learn about stem cell research from the patients who stand to benefit

Search all CIRM grants – See all grants funded by CIRM, and learn more about the research goals

Progress toward therapies – Learn about the steps involved in going from a good idea in the lab to a new therapy, and learn how CIRM is working to accelerate the research

Funding charts – See how CIRM’s funding has been allocated to different types of stem cells and research areas


Tuesday, October 2, 2012

Guest blogger Alan Trounson — September’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.

This month I want to focus my blog on a paper that I think is a major advance in spinal cord injury repair. A CIRM-funded UC San Diego team mixed stem cell science with some classic tissue engineering to achieve the greatest degree of spine repair in rats that I have ever seen reported (you can read a summary of their award here). They embedded neural stem cells in fibrin, a protein found in blood, and added several growth factors to this gel.

When you use neural stem cells is it is not good enough for them to just mature into neurons. Those neurons need to produce lengthy axons that can reach out and make connections with the existing neurons in the host. The UCSD team placed the gel at the site of complete spinal cord separations in rats and after six weeks found that the number of axons growing from the injury site exceeded what other teams had seen by 200-fold. Those axons also grew10 times the length seen in earlier studies.

They found this when they implanted rat neural stem cells as well as with two different human neural stem cell lines. One of those human lines is already approved for use in humans and is being tested in a clinical trial for ALS (Lou Gerhig’s disease). This could greatly reduce the time needed to move the UCSD process from animal testing to human trials.

My colleagues wrote about this work when it first appeared in the journal Cell here.

This month’s report also includes two articles showing that the field continues to make major strides in understanding and refining the process of reprogramming adult cells to become cells that behave like embryonic stem cells, that is iPS cells. We blogged about one of these papers here. My report also discusses a paper that firms up the role of cancer stem cells in leukemia.

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


Monday, October 1, 2012

Europe not Competitive in hESC Research: Patently False

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

The European Parliament is currently considering its next research funding program called Horizon 2020. Under consideration in that program is the continued level of support for human embryonic stem cell (hESC) research.

The current European research program is similar to U.S. NIH policy, where research involving the use of hESC lines that conform to certain ethical standard is permitted, but the EU does not fund the derivation of new hESC lines. Any changes to that current standard in the 2020 program must be approved by the European Parliament and the Council of Ministers.

Nature recently ran a story pointing out that funding levels for hESC research in the Horizon 2020 program might be at risk. Here’s how they explain the reasoning of those who oppose the funding:
The Legal Affairs Committee of the European Parliament voted last week for its exclusion, referring back to the controversial decision of the European Court of Justice last year that the use of hES cell lines in research was immoral – and therefore unpatentable – because it had once involved destruction of an embryo.
The Legal Affairs Committee argued that since such research was unpatentable in Europe, it could not contribute to European competitiveness. And since the main aim of Horizon 2020 is to stimulate European competitiveness, such research should not be funded by the programme.
Prof. Aurora Plomer University of Sheffield (UK) tracks EU funding policy. I asked for her assessment of the situation. She indicated:
So far, the majority of delegations at the Council have expressed their wish to maintain the status quo. But at the last meeting of the Research Working Party of the Council, on May 16th 2012, proposals were introduced to restrict funding to adult stem cells.
Professor Plomer blogged previously about how the Court’s decision to render hESC research unpatentable in the EU trumped a variety of national and EU-wide policies and showed “complete disregard for the diversity of moral and legal cultures on the human embryo in Europe.” Now it appears the ruling will have the effect of penalizing researchers striving to develop therapies for patients. The problem is this: a researcher in the UK might develop a breakthrough therapy for blindness or deafness, but that groundbreaking research might not receive funding from the EU because it cannot be patented.

It is sad and simultaneously bizarre that the perceived inability to patent therapies would become a sticking point for funding. For years during the development of CIRM’s Intellectual Property Policy, concerns were raised over the potential for patents to hinder access to therapies. (We’ve written an FAQ about our IP policy, which is available here.)

EU members are carrying out highly innovative hESC research. This is why CIRM has formed collaborative funding arrangements with Germany, UK, Spain, and France (here’s more about that program). CIRM values these collaborations and sees them as vital for the advancement of patient therapies.

In our effort to support our EU partners, CIRM recently submitted comments to the House of Lords Select Committee on Science and Technology Call for Evidence. The Committee is considering recommendations for the UK’s research programs. We suggested that the ruling preventing patentability of hESC research could impact commercial development of new therapies in addition to reducing or eliminating hESC funding in the Horizon 2020 research program. In light of that, we recommended that the House of Lords evaluate how the patent ruling could effect national research programs in the UK and consider policies to avoid conditions would impede the development of new therapies.