The ISSCR, California Stem Cell Financing and Silence

It comes as no surprise that the largest organization of stem cell scientists in the world is in favor of "rigorous funding" for stem cell research and warns of the perils of decreased financial support. 

That organization is International Society for Stem Cell Research (ISSCR), which is meeting in Los Angeles later this week and expects 4,000 persons to attend. 

California's stem cell agency, known formally as the California Institute for Regenerative Medicine (CIRM), has long been a supporter of the ISSCR. In 2009 it contributed $200,000 to help out with the ISSCR's annual conference. 

Times have changed, however, since those halycon days 10 years ago. It is running out of cash for new awards. This year CIRM contributed "only" $50,000 to help stage the group's annual meeting. A few days ago it cut off applications for new research awards beginning next Monday. It needs support for $200 million in private "bridge" funding to continue its program while it awaits what it hopes will be voter approval in November 2020 of re-funding the agency. 

What does the ISSCR have to say about the state of the California stem cell agency?

Anne Nicholas, director of communications for ISSCR, was asked about the situation last week by the California Stem Cell Report. She replied,

 "We don’t have anything to add to your story at this point."

Turning Off the California Stem Cell Spigot: Will Private Donors Step Up?

Benchmarks are important to the $3 billion California stem cell research program. When scientists fail to achieve them, the flow of cash from the agency disappears. 

Last week, the stem cell agency quietly announced something of a funding benchmark for its own, 14-year-old efforts.

The bad news? In just six days, the agency will shut off  applications from California stem cell scientists and companies for multimillion dollar awards.

The agency, formally known as the California Institute for Regenerative Medicine (CIRM), last Thursday said that its dwindling finances forced the closure. 

CIRM has only $33 million left for new awards and already has requests in the pipeline for $88 million. Private funding is a possibility, but major donors have not yet surfaced publicly.

Immediate reaction to the announcement was muted but ranged from dismay to tributes to the agency and the work it has financed. 

One scientist, Jeanne Loring, chief scientific officer at Aspen Neuroscience in San Diego, said the action was like "a rug being pulled out from under you." Loring also said in an email that the agency has built an "enormous resource in stem cell expertise in California" and played an important role in her own work at Scripps Research. 

Loring's work has received $17.4 million from the agency since 2005. (See the full text of her remarks here.)

Steve Peckman, deputy director of the Broad Stem Cell Research Center at UCLA, said the agency has chalked up "impressive success" and made California an international leader in the field. (See the full text of his remarks here.)

UCLA has received $289 million from the agency. 

Robert Klein, who led the ballot campaign in 2004 that created the agency, said the application shutdown will create a gap that will hold back development of critical therapies. Klein is expected to lead another ballot initiative in November 2020 to provide $5.5 billion for CIRM. (See the full text of his remarks here.)

Klein is chairman and founder of Americans for Cures of Palo Alto, Ca. He was also the first chairman of CIRM. 

CIRM has provided funds to about 600 researchers and 128 institutions and companies. The researchers run labs that vary in the number of employees, but the total would include  hundreds more stem cell workers. 

The agency is currently trying to raise $200 million privately to continue its awards programs between now and the fall of 2020. No philanthropic gifts have been announced. Queried last week by the California Stem Cell Report, the agency said that it had nothing new to report in the fundraising effort that began last year.  

It is unclear how the application shutdown will affect the fundraising effort. It may serve as a prod, however, for some potential donors and help to crystalize decision-making as CIRM executives stress the importance of the agency.

CIRM's announcement left open the possibility of re-opening applications come September. The agency expects to have a better handle then on how much cash might be returning to CIRM from awards that have missed benchmarks. The amount is not expected to be huge. 

The agency has reported that it has enough money to sustain a wind-down of the agency and to administer remaining multi-year grants, should the yet-to-written ballot measure fail. 

Klein is optimistic, however, regarding the prospects for a bond measure 16 months from now.  He told the California Stem Cell Report that unspecified polls show that 70 percent of voters support re-funding the agency when they learn of the "remarkable progress" that has been achieved as a result of CIRM-backed research. 

CIRM Application Shutdown: Text of Robert Klein's Comments

Here is the text of comments by Robert Klein, chairman of Americans for Cures, on the shutdown of applications at the California stem cell agency. Klein was responding to questions from the California Stem Cell Report.

"When Californians learn of the remarkable progress from the California funded stem cell and genetic therapies FDA approved human trials in restoring major function to paralyzed patients, to save the lives of children with severe immune diseases, to restoring sight to patients that are blind, the support for stem cell research and therapy development exceeds 70% in recent polls.

"California’s leadership in cellular and genetic therapies, through its state funded research and human trials, currently supports 51 human trials for a range of chronic disease and injuries, and 24 more human trials are in progress by biotech companies based upon California’s funded research.

"The life-changing and life-restoring work of California’s scientists and physicians will predictably earn the chance for renewed funding. Polls indicate that California voters want the opportunity to vote in 2020 on continuing this visionary California initiative, originally funded in 2004 through Proposition 71, the California Stem Cell Research and Cures Act. The gap in available state funding from the fall of 2019 through November 2020 will hold back additional critical human trials for new life saving and/or disease mitigating therapies; but, I have faith that Patient Advocates and California voters will back new funding at the polls in 2020 and continue California’s remarkable contribution to this medical revolution that effects every one of our lives.

"In advancing the research and therapies, the California research funding agency has also gained broad financial support for its portfolio of research and human trials based on the strength of more than 2850 peer reviewed published medical discoveries and the 75 human trials directly funded or separately funded supporting the California funded discoveries. The matching funds from donors, institutions, private companies and non-profit organizations have already exceeded $3,250,000,000, more than a 100% match on the $3,000,000,000 originally approved in state funding.

"This medical revolution holds the promise of restoring health and quality of life for many of California’s individuals and families suffering from chronic disease and injury. However, the last tactical mile to bring this broad spectrum of therapies to patients will require more funding and the thoughtful support of California’s public as the human trials and discoveries are refined and tested, overcome numerous obstacles or complications, and ultimately serve to improve the life and reduce the suffering of every one of us."

CIRM Application Shutdown: Text of Jeanne Loring's Comments

Here is the text of comments by Jeanne Loring on the application shutdown at the California stem cell agency. Loring was responding to questions by the California Stem Cell Report. She also filed a comment separately on the initial story. 

"CIRM has built an enormous resource of stem cell expertise in California over the last 14 years. They had a big impact on my lab; over the years I was awarded 10 grants on which I was PI or co-PI and several more as a co-investigator, we trained hundreds of researchers in pluripotent stem cell technology through CIRM’s Shared Labs and Training Center grants, I mentored 32 students through the Bridges program, and we published 71 scientific papers with CIRM support (so far). While I also obtained funding from the NIH and private donors, CIRM was by far the dominant supporter of my research.  When I obtained my first grant on human embryonic stem cells from the NIH in 2002, I could not have predicted that I would be able expand on that work so spectacularly. I was in the right place at the right time.

"While I am grateful for CIRM’’s support of my work, I have also been deeply troubled by some of  their decisions. This is not the time or place to dissect their judgments, but if they are rejuvenated by another bond measure, I hope they will call on some of us to share our experiences as they develop their strategy for the future of stem cell research and development in California."

California Application Shutdown: Text of Comments by Steve Peckman

Here is the full text of comments by Steve Peckman on the shutdown of applications by the California stem cell agency. Peckman was responding to questions from the California Stem Cell Report. 

"The inevitable announcement by CIRM serves to highlight the impressive success of Proposition 71 as a catalyst for scientific discovery and the clinical application of stem cell research and regenerative medicine.  Since 2005, CIRM’s trailblazing initiatives from science and ethics training to basic scientific research, biomedical tool development, infrastructure, pre-clinical testing, and clinical trials have made California an international leader in the field.  The pool of scientific knowledge is constantly growing because of the public’s trust and investment in the future.  The forward thinking of California voters through the passage of Proposition 71 has gifted the world with critical biomedical breakthroughs, such as a cure for ADA-SCID (developed by UCLA physician-scientist Dr. Donald Kohn and his team), whose broad impact will inform science and medicine for generations to come.

"As you noted, talk of CIRM’s death is a bit premature as it will still be awarding pre-clinical and clinical research grants in the near term and overseeing research grants and clinical trials for at least four more years."