CIRM Exaggerates Role in San Diego Clinical Trials Research, Scientist Says

The headline last week from the California stem cell agency read: "First Clinical Trial Begins for a Therapy Enabled By CIRM Funding."

The news was big enough that California Gov. Arnold Schwarzenegger issued a statement on April 7, hailing the research as a world-leading effort. The governor said,

"Stem cell research, funded by the California Institute for Regenerative Medicine (CIRM), led to the discovery of this new treatment."

CIRM Chairman Robert Klein touted "the CIRM model designed to accelerate the quest for cures." The news also received attention in some newspapers, including the San Diego Union-Tribune.

But at least one well-regarded, California stem cell researcher thinks the agency engaged in exaggeration – perhaps even "fraud" – in making the claims about the role of its funding.

The researcher, who asked not to be identified, told the California Stem Cell Report:

"It appears as though the CIRM has wrongly taken credit for supporting work published by one of its funded grantees and/or the funded grantee has wrongly given credit to CIRM for supporting their research. In either case, the CIRM’s press release on the subject is self-serving and misleading, at best. The press and hESC stem cell research supporters have already latched on to this false claim by CIRM and, as a result, are mistakenly touting CIRM’s effectiveness in the field."

CIRM has defended its statement, which said,

"Researchers at the University of California, San Diego, used a (CIRM) SEED grant from the California Institute for Regenerative Medicine to conduct stem-cell research that verified a suspect gene mutation was by itself necessary and sufficient to cause a class of severe blood diseases called myeloproliferative disorders."

The CIRM statement, which is carried under the press release section of its web site, was modified following queries by the California Stem Cell Report. The release now says that a CIRM training grant was also involved. The current version of the news release, however, does not mention that it was altered from the original.

The initial version of the CIRM statement reportedly triggered concerns among some stem cell scientists and led to one contacting the California Stem Cell Report.

The researcher said in an email:

"The press release 'First Clinical Trial Begins for a Therapy Enabled By CIRM Funding' states that 'Researchers at the University of California, San Diego, used a SEED grant from the California Institute for Regenerative Medicine to conduct stem-cell research …'. The quoted UCSD press release describes the work as involving 'human cord blood stem cells … to find whether over-expression of a single gene could drive, or initiate, the disease … that looked like PV' and says that the '…work [was] supported in part by grants from the California Institute for Regenerative Medicine…'. Both items in the UCSD press release are corroborated by inspection of the Cancer Stem article cited.

"However, the SEED grant mentioned - RS1-00228-1: Derivation and Characterization of Cancer Stem Cells from Human ES Cells – has nothing to do with cord blood stem cells or PV. The SEED grant involves hESCs and another disease altogether, CML: 'To provide a robust model system for screening novel anti-CSC therapies, we propose to generate and characterize CSC from hESC (10-18). We will investigate the role of genes that are essential for initiation of CML such as BCR-ABL and additional mutations such as b-catenin implicated in CSC propagation (19-30).'

"Thus, any claim that the SEED grant monies were used to support the published research is either in error, an over-exaggeration, or, worse, fraud.

"CIRM needs to investigate which of these possibilities is the correct one, issue a retraction of its claim, and ensure that erroneous claims are no longer proffered by CIRM or its grantees."

The research in question was conducted at the University of California, San Diego, by a team led by Catriona Jamieson. Her article in Cancer Cell said in the acknowledgments section that:

"This work was supported in part by grants from the California Institute for Regenerative Medicine and the Mizrahi Family Foundation to E.K., C.B., and C.H.M.J., the National Institutes of Health (K23HL04409) to J.G., and an unrestricted gift from Targe-Gen Inc."

When queried, Don Gibbons, chief communications officer for CIRM, said,

"Jamieson has had a post doc funded by a CIRM training grant since July 2006, so our funds helped every stage of this work through his salary. The SEED grant to Jamieson was approved in February (2007) and the Notice of Grant Award went to the institution in July. It is typical for institutions to forward fund after an NGA pending arrival of the first check, which happened in September. The journal asked for additional experiments after the initial submission of the paper, and the Seed grant helped complete those added experiments that resulted in final publication.

"The SEED grant does require that Jamieson use embryonic stem cells at some point during the two year grant cycle, but it also funds many other aspects of the science. Our scientific strategic plan does not call for just funding stem cell work directly; it calls for accelerating the field as a whole."

Gibbons later said that UCSD provided no advance funding for the CIRM grant, which was not signed off by UCSD until Aug. 31, 2007, according to another source. Jamieson's article was first submitted for publication Sept. 19, 2007, according to the source.

We asked Jamieson if she had any comment on the CIRM news release issue. She simply referred us to the statement contained in the acknowledgment section of her Cancer Cell article.

The stem cell researcher who is critical of CIRM said that "...because (the agency) perceives a need to produce results rapidly (it) has, perhaps inadvertently and perhaps not, latched on without checking the facts (although in my mind the facts are quite obvious and need little checking)."

Head-to-Head on Proposition 14: 'Fatally Flawed,' Life-Saver or Both?

2012 CIRM video of Sandra Dillon (on right) and Catriona Jamieson of UC San Diego

Sandra Dillon says she owes her life to the California stem cell agency, and she wants California voters to give it $5.5 billion more to help save the lives of many more, including someone they love. 

Dillon made her comments in a piece carried last month in the San Diego Union-Tribune. Her article was paired with another commentary in the paper that strongly opposed Proposition 14 on this fall's ballot. It was written by Jeff Sheehy, a patient advocate member of the board of the California Institute for Regenerative Medicine (CIRM), the official name of the stem cell agency.  

Together, Dillon's and Sheehy's commentaries capture the essence of the arguments for and against the ballot initiative and highlight what is at stake this fall. And that is the continued existence of the stem cell agency, which was created in 2004 by another ballot initiative that provided it with $3 billion in money borrowed by the state. That funding is all but gone. If voters reject Proposition 14, CIRM will begin closing its doors this winter. 

(The actual cost to taxpayers, it should be noted, is about $4 billion in the case of the 2004 measure and an estimated $7.8 billion for Proposition 14.)

When Dillon was 28, she was diagnosed with a rare form of blood cancer called myelofibrosis. She wrote in the San Diego paper, 

"Upon learning of my cancer diagnosis, I was left feeling helpless, afraid and searching for answers about how this disease would affect my everyday life, what I could do to fight it, and how much time I had left to live....

"It is because of California’s forward-looking leadership in passing Proposition 71 in 2004 — hoping to find treatments and cures for chronic diseases and illnesses — that has allowed me to still be here today." 

Dillon concluded,

"The passage of Proposition 71 helped save my life. It is unimaginable to think that Californians would vote to discontinue this amazing effort — I don’t know where I would be or what condition I would be in if it wasn’t for the investment Californians made nearly two decades ago."

Dillon's experimental therapy involved research by Catriona Jamieson of UC San Diego, who has received $17.3 million from the stem cell agency.

In the other commentary, Sheehy, who has served on the CIRM board since 2004 and was chairman of its science subcommittee, was deeply critical. He was the only member of the CIRM to votes against endorsing the measure. 

The California Stem Cell Report carried a piece last month summarizing what Sheehy had to say in his piece, which was headlined,  "Why Prop. 14 is unaffordable, unnecessary, fatally flawed and unsupportable."

But to refresh readers, Sheehy, whose article was solicited by the newspaper, said, 

"It must seem odd that someone who has spent countless hours over the last 15 years as a member of the governing board of the California Institute for Regenerative Medicine (CIRM) would oppose Proposition 14, which seeks to provide $5.5 billion in new funding for the stem-cell agency. While I value CIRM and its work to date, Proposition 14 commits California to spending money it does not have — $7.8 billion including interest for research that is already well-funded. Plus, CIRM’s pre-existing flaws are actually exacerbated by new provisions in the measure."

"And after spending all of that money, not a single U.S. Federal and Drug Administration-approved product has materialized on which CIRM’s funding played an important role."

"Proposition 14 will add at least another $260 million a year in annual repayments. That means California taxpayers will be on the hook for $587 million a year for stem-cell research. Remember state imperatives such as education, health care and housing are not only chronically under-resourced but with a looming deficit, will be starved for funding because bonds must be repaid first. Cuts have already happened and more are likely on the way. Critical needs will go unfunded."

California's Alpha Stem Cell Clinics Garner Little News Coverage

The California stem cell agency’s $34 million Alpha clinic venture last week received minimal attention from the mainstream media.

The effort was also was subordinated by the agency itself, which issued a press release that focused heavily instead on the plan by its new president, Randy Mills, to improve operations at the agency.

News stories, however, led with the action on the Alpha clinics. Articles appeared by Bradley Fikes in the San Diego U-T, Rebecca Kheel in the Orange County Register and in The Sacramento Bee by yours truly. John Schreiber of City News Service in Los Angeles also wrote a brief article that was picked up by smaller outlets.

The occasion was approval of the first stage of the Alpha clinic plan, which was originally budgeted for $70 million by former agency president Alan Trounson and the agency board.

Last Thursday, the agency’s board handed out three awards of $8 million each, trimming the applicants’ proposals by as much as 26 percent.  Next year, the agency is expected to add a $10 million information/data center to the Alpha effort. The center was cut from $15 million earlier this year.

The winners last week were the City of Hope, Duarte, Ca.; UC San Diego and UCLA/UC Irvine. The latter two issued press releases quickly (see here and here), but the City of Hope has still not issued one.

The press releases provided a few more details about the research, including the names of the principal investigators. At UC San Diego, it is Catriona Jamieson, deputy director of the Sanford Stem Cell Clinical Center. For the UCLA/Irvine effort, it is John Adams, a member of the UCLA Broad Stem Cell Research Center and professor in the department of orthopaedic surgery. Donald Kohn and Antoni Ribas are directing the two clinical trials. Normally the stem cell agency identifies the principal investigators in a press release following board action but did not do so in this case.

(An earlier version of this incorrectly said Kohn and Aribas were the principal investigators for UCLA-Irvine.)

CIRM Moving Forward with 'Vibrant' Economic Benefit Study

Key directors of the California stem cell agency tomorrow are likely to give the go-ahead to a $300,000 proposal to study the economic impact of its $3 billion research effort.

Under the terms of the RFP, the folks doing the study must “execute a vibrant and aggressive strategy to support the goals and initiatives of CIRM.”

Five firms responded to the RFP last year. Only one, LECG of Emeryville, Ca., is being considered tomorrow at a teleconference meeting of the directors' Governance Subcommittee. LECG says Henry Miller of its Washington, D.C., office and Jose Alberro will be co-directors of the study.

We have written previously about the credibility problems associated with any economic study funded by CIRM. There is no doubt that CIRM's efforts have a beneficial economic impact. But no CIRM-financed study will convince skeptics that the program is necessarily justified.

Nonetheless, CIRM is proceeding with the effort, which will undoubtedly be a useful public relations tool. The study is also likely to be served up at some point to support the need for additional state funding. And it could serve as a marketing device should CIRM push forward with its plans to peddle state bonds privately.

If the data are public record and available in a non-proprietary format, they could be useful to other economists and interested parties. Most of CIRM's records are public, but sometimes some of its important information is tucked away from the public gaze. The RFP, the LECG bid and the staff report carry no assurances that the information gathered during the study will be available publicly or in a format that could be used by non-CIRM researchers.

We have asked CIRM whether the economic data will be a public record and will carry the agency's response when we receive it.

The RFP for the study was first posted about a year ago, but is no longer available on the CIRM Web site. A CIRM staff report said that negotiations on the contract were suspended because of concerns about CIRM's financing.

The report said that contract being considered tomorrow will involve something of a pilot project. The staff said,

“The plan agreed upon between CIRM and LECG is to initially build a model around a test case, a specific disease – Polycythemia Vera – that is the target of a clinical trial under the direction of Dr. Catriona Jamieson at UCSD (the topic of a disease “spotlight” at the June 2009 ICOC meeting in San Diego). This clinical trial involves a small molecule drug therapeutic identified through studies using stem cells. The model will take into account direct benefits of CIRM funding (job creation through research and facilities awards), secondary cascades of CIRM funding (“multiplier effect” – e.g. payments to suppliers and subcontractors along with affiliated job creation and state revenues from income, sales and property taxes), potential savings in health care costs (compared to current therapies) and increased productivity for both patients and their caretakers (related to improved vitality and quality of life). Once this model is created, it will be evaluated and fine-tuned by a panel of experts selected by LECG and CIRM. The application to Polycythemia Vera will then be completed and a report prepared by December 2009.

"Subsequently, the model will be expanded and adapted for analyzing other diseases and other applications of stem cells for treating these diseases. In addition to the development of small molecules, other approaches will include cellular therapies (replacing cells, organs &/or organ functions) and the creation of normal and disease specific cell lines for screening (e.g. predictive toxicology) and early diagnostics. The entire project should be completed by the beginning of May 2010. Further, we expect the results to identify data that CIRM should collect on an on-going basis as part of its grants management process for use in future economic analyses.”

The other firms offering bids for the economic RFP included the Analysis Group, which previously performed studies for CIRM and the Prop. 71 ballot campaign. The others were RiskAnalytica, PricewaterhouseCoopers and Tripp Umbach.

The public can take part in the teleconference meeting at locations in San Francisco (2), Palo Alto, Irvine, Los Angeles and UC Davis. Specific addresses can be found on the agenda.

CIRM Makes a YouTube Move

The California stem cell agency has joined YouTube, an Internet video-sharing service used by millions worldwide.

CIRM, the world's largest source of funding for hESC research, hooked up with YouTube on Jan. 15 with videos of California scientists talking about their research. Since then, 259 videos have been watched, as of this writing. Currently CIRM appears to have 28 different videos on the site.

The service is called CIRMTV, and it is part of CIRM's expanding PR effort. On Tuesday, CIRMTV keyed off the Obama inauguration with a four-minute video of CIRM President Alan Trounson and UC San Francisco researcher Susan Fisher (see photo) discussing the likely impact of the new president.

The inaugural video currently has the highest number of views, 109, with one of Hans Kierstad of UC Irvine coming up in second with 76 views, outstripping UC San Diego's Catriona Jamieson, who had 57, and Stanford's Irv Weissman, who had 48.

As always with the Internet, the main problem is how to build traffic. CIRM needs lots of links to the site to build viewership along with comments on the material from outside sources, ranging from print publications to other Internet sites. However, stem cell research has limited general appeal considering the other matters on YouTube, and it is unrealistic to expect massive numbers.

Don Gibbons, chief communications officer for CIRM, reports that CIRMTV has "zero outside cost" and requires "minimal staff time."

The same videos can be seen on the CIRM web site. In our case, the YouTube version loaded and played smoothly while the CIRM site stalled repeatedly. There could be a host of reasons for that, ranging from server capacity to video quality to the bandwidth of our connection.

Normally the videos cannot be downloaded for viewing later when you are not connected to the Internet. But Wikipedia reports that free software can be downloaded to provide that capability.

On Tuesday, CIRM also devoted a good chunk of its home page to the inauguration, featuring both the Trounson-Fisher video and a link to a two-page statement on the impact of Obama's expected stem cell moves.

For more on California state government uses of YouTube, see this piece by Daniel Wood of the Christian Science Monitor.

The Elusive Stem Cell and the Power of Hope

It was, you might say, the best of times and the worst of times last week for the stem cell community, particularly in California. At least based on a spate of headlines and news stories.

Largely ebullient rhetoric rolled out of San Diego, only to be tempered or contradicted elsewhere. The World Stem Cell Summit was underway in Southern California, generating a small wave of coverage.

On Tuesday, Bradley Fikes of the San Diego UT reported,

“Stem cell research has already yielded historic breakthroughs against incurable diseases, a panel of top stem cell researchers said at a public forum Tuesday evening. And that's just the start....”

On Wednesday, the $3 billion California stem cell agency carried on with the theme and blogged about “breakthroughs.”

On Thursday, GEN News quoted Bernard Siegel, the major domo of the summit, as saying,

 "The field is a true scientific revolution and reflects the transformative power of hope...."

But then there was this midweek story, also by Fikes,

“Stem cell research faces a budget crunch -- The cash-strapped federal government’s ability to fund stem cell research has become severely limited....”

On Saturday, Bloomberg headlined a discouraging story about research based in Boston,

“HIV Returns in Two Men Thought Cured After Stem Cell Transplant."

And earlier in the week, a consultant to the $3 billion California stem cell agency made it clear that after 2017, it will no longer be handing out $300 million a year in research grants. His report also noted that it would take 10 years or so for stem cell research to emerge from early clinical trials to become real, commercial therapies.

The diverging stories and comments reflect the ebb and flow of a young technology. They also reflect the inveterate optimism of many researchers and patient advocates, who publicly see few barriers to stem cell therapies. Catriona Jamieson, a scientist at UC San Diego who appears to be enjoying remarkable success in her work, last week commented,

"The biggest barrier is nihilism."

Cash is short, however, not only in the federal government and at the California agency, but also in the private sector, which has basically stiff-armed stem cell research for the last several years or longer.

Despite some recent, promising signs of interest from some companies, Siegel's comment may be the most on the money about the current state of affairs: The stem cell field continues to reflect the “transformative power of hope.” And widely available, safe, effective and reasonably priced stem cell therapies remain elusive.

California Stem Cell Agency Pushes $23 Million into Clinical Trials for Leukemia, Bone Marrow Transplants

California's 12-year-old stem cell research program this morning popped for $23 million to help finance clinical trials to "confuse and kill" leukemia cells and to develop a better alternative to bone marrow transplants.

The two awards are part of the stem cell agency's effort to produce a widely available stem cell therapy before its money for new awards runs out in less than three years. So far, the agency, known formally as the California Institute for Regenerative Medicine (CIRM), has not been successful in producing such a therapy.

CIRM has now funded 35 clinical trials, which are the final stage of testing before a therapy is approved by the federal government for widespread use.  Thirty-one of the trials are still active.

Today's largest award, $18.3 million, will go to Thomas Kipps of UC San Diego. An additional Oncternal Therapeutics, also of San Diego.  Kipps is a scientific advisor to the firm. The other award, $5 million,  goes to Angiocrine Bioscience, Inc., of San Diego. The company will add $2.7 million to the effort.

Thomas Kipps, Oncternal photo

$13.3 million will be provided by

The Kipps project targets the most common form of lymphoma, chronic lymphocytic leukemia (CLL) the agency said in a news release. The agency said Kipps is "using a combination strategy to kill the cancer stem cells that help CLL – and other cancers – survive traditional therapies, such as chemotherapy, and cause a relapse."

Every year, about 20,000 people nationwide are diagnosed with CLL.

The Angiocrine research is aimed at increasing the supply of cord blood for bone marrow transplants, when no matched donor is available.  Its product is called AB-110.

Maria Millan, interim CEO of the stem cell agency, said,

 "This (Phase 1) clinical trial will be the first step in determining if AB-110 is able to provide a way to improve the results of cord blood transplants."

Today's award appears to advance work previously funded by CIRM at a cost of $4 million.  Paul Finnegan was listed as the principal investigator on that work.

Angiocrine's key technology  is licensed from Weill Cornell Medical College and was invented and developed by Shahin Rafii.

The awards were approved in less than 10 minutes this morning with no discussion by the CIRM governing board, which simply ratifies the decisions of its out-of-state, grant reviewers, who considered the applications in July behind closed doors.

A summary of the reviewers' comments can be found here for Kipps' application, CLIN2 -101, and here for Angiocrine's, CLIN2-10386. Information on the Kipps trial can be found here on clinicaltrials.gov. Catriona Jamieson of UC San Diego is listed as the principal investigator on the Kipps' award.

The Angiocrine trial was not yet listed on clinicaltrials.gov at the time of this writing.

California Researchers Score $16 Million Plus in State Stem Cell Awards

The California stem cell agency today handed out $16.4 million in research grants seeking therapies for afflictions ranging from gum disease and cancer to vision loss and Parkinson's Disease.

The award for Parkinson's was relatively tiny -- only $150,000 -- but represented a rare case in which the agency's governing board overturned its reviewers, who make the de facto decisions on awards.

The reversal came after one board member, David Higgins, of San Diego, who has Parkinson's, noted that the most common drug that Parkinson's patients take is 70 years old. He told the board.

David Higgins, CIRM photo

“I’m a fourth generation Parkinson’s patient and I’m taking the same medicine that my grandmother took. They work but not for everyone and not for long. People with Parkinson’s need new treatment options and we need them now. That’s why this project is worth supporting. It has the potential to identify some promising candidates that might one day lead to new treatments.”

The award went to Zenobia Therapeutics, Inc., of San Diego, whose president and co-founder, Vicki Nienaber, had filed an appeal on the reviewers' decision.  Another applicant rejected by reviewers, Toshio Miki of USC, also filed an appeal with the board. His appeal was not discussed. Miki's application sought $5.9 million for research involving metabolic disorders.

The largest award today, $5.6 million, went to Anthony Oro of Stanford, who will be testing a therapy to treat an affliction that creates wounds that will not heal. Dan Kaufman of UC San Diego received $5.5 million to produce "killer cells" to help people with a form of leukemia. Catriona Jamieson, also of UC San Diego, received $2.7 million for leukemia research.

Here is a link to summaries of reviewer remarks, including scores, on the three winners and the 11 other applications that were rejected in the translational awards category. (Scroll down on the page to see the reviews.)

In addition to the "discovery" award for Parkinson's, here are the names of the other winners in that category:

•  DISC1-10603, Ngan F Huang, iPSC-Derived Smooth Muscle Progenitors for Treatment of Abdominal Aortic Aneurysm, Palo Alto Veterans Institute for Research, $172,621
• DISC1-10475, Semil P Choksi, Generation of human airway stem cells by direct transcriptional reprogramming for disease modeling and regeneration, U.C. San Francisco, $238,408
• DISC1-10643, Dmitriy Sheyn, IVD rejuvenation using iPSC-derived notochordal cells, Cedars-Sinai, $241,992 
• DISC1-10598, Alice F. Tarantal, Enhanced Branching Morphogenesis and Pluripotent Cell Lineage Differentiation for Pediatric Regenerative Therapies, U.C. Davis, $235,80
• DISC1-10583, John R Cashman, Human Pancreatic Cancer Stem Cells: Developing a Novel Drug for Cancer Eradication, Human BioMolecular Research Institute, $303,894 
• DISC1-10555, Hiromitsu Nakauchi, Optimizing self-renewal signaling kinetics to stabilize ex vivo hematopoietic stem cell expansion, Stanford, $235,836 
• DISC1-10620, David J. Baylink, Bone Marrow Targeting of Hematopoietic Stem Cells Engineered to Overexpress 25-OH-VD3 1-α- hydroxylase for Acute Myeloid Leukemia Therapy, Loma Linda University, $178,967 
• DISC1-10513, Guillem Pratx, Novel metabolic labeling method for tracking stem cells to irradiated salivary glands using PET, Stanford, $235,613 
• DISC1-10522, Gerald P Morris, Identification of antigenic neo-epitopes from in vitro reprogrammed human tissue precursors for regenerative therapy, U.C. San Diego, $193,500
• DISC1-10588, Julia J. Unternaehrer-Hamm, Targeting cancer stem cells with nanoparticle RNAi delivery to prevent recurrence and metastasis of ovarian cancer, Loma Linda University, $172,870 
• DISC1-10721, Karl J. Wahlin, An IPSC cell based model of macular degeneration for drug discovery, U.C. San Diego, $232,200 
• DISC1-10516, Alyssa Panitch, Development of treatments to improve healing of ischemic wounds, U.C. Davis, $235,800 
• DISC1-10718, Alireza Moshaverinia, Gingival mesenchymal stem cells as a novel treatment modality for periodontal tissue regeneration, U.C. Los Angeles, $194,483

Here is a link to all the review summaries and scores in the round, including the 28 rejected applications.  Here is a link to the CIRM press release. 

California Stem Cell Agency Pulls $19 Million Grant

The California stem cell agency has terminated a $19 million grant to a UC San Francisco researcher involved in the agency's ambitious attempts to push stem cell therapies into clinics.

The agency said the research effort led by Mitchel Berger, chairman of the department of neurological surgery at UCSF, "did not meet a go/no-go milestone" stipulated in the grant. His research was funded in 2010 to treat brain tumors with genetically modified neural brain cells. No further explanation for the termination was provided by CIRM in a report prepared for tomorrow's meeting of the CIRM governing board. The agency estimated the cancellation would save $13 million.

The California Stem Cell Report has asked Berger and his co-PIs for comment on the CIRM action. The other researchers are Evan Snyder of Sanford-Burnham and Webster Cavanee of the Ludwig Cancer Institute. Their remarks will be carried verbatim when they are received.

The CIRM action was disclosed in the progress report on the $230 million disease team effort launched by the agency in 2009. The amount climbed to more than $250 million with contributions from partnering countries. Three of the 14 funded applicants – Irv Weissman and Gary Steinberg, both of Stanford, and Karen Aboody of the City of Hope – were approved only after they appealed to the CIRM board to overturn rejections by grant reviewers. (See  here , here and here for their written appeals. See here and here for coverage of the 2009 board action.)

One other disease team grant was modified to limit its scope and revise its funding. No savings were announced by CIRM. The PI on the $20 million project is Dennis Carson of UC San Diego. Co-PIs are Catriona Jamieson, also of UC San Diego, and John Dick of the University Health Network of Canada. The research is aimed at leukemia.

The actions on the disease team grants were not entirely unexpected. From their inception, CIRM directors have been told not to expect all the grants to finish successfully.

Ellen Feigal, senior vice president for research and development at CIRM, prepared the 19-page update on the disease team efforts. The grants are aimed at generating an investigational new drug application with the FDA within the four-year term of the grant.

She said that the funding decisions were made following evaluation of the projects by panels of clinical development advisors. Their recommendations were then considered by CIRM staff.

Feigal's report laid out accomplishments of the research so far and discussed changes in direction.

She said two companies have been formed since the grants were awarded to commercialize the hoped-for products. She said that in June 2011 Aboody founded TheraBiologics Inc., Newport Beach, Ca., of which she is chief scientific officer and director. Another company, Regenerative Patch Technologies, Glendale, Ca., was created by the team working on an hESC treatment for age-related macular degeneration. That $16 million grant involves Mark Humayan and David Hinton of USC, Dennis Clegg of UC Santa Barbara and Peter Coffey, formerly with University College, London, but now at UC Santa Barbara. The effort has generated seven patent filings.

The Feigal update also discussed the efforts of companies involved in other disease team grants. The lack of CIRM funding for biotech firms has been a bone of contention with industry and troublesome for some CIRM directors.

CIRM indicated the projects involving the firms were moving on schedule with no major difficulties reported. The companies involved are ViaCyte of San Diego, Calimmune of Tucson, Az., and Sangamo Inc. of Richmond, Ca.