Monday, November 04, 2019

California Stem Cell Agency Chalks Up its 60th Clinical Trial as Funds Dwindle

OAKLAND, Ca. -- The California stem cell agency last week awarded about $54 million for research into afflictions ranging  from eye disease to epilepsy as it edged increasingly closer to running out of cash.

Nine applications were approved including more clinical trials -- the last stage before therapies are approved for widespread use. The actions bring to 60 the number of clinical trials that the 15-year-old agency is helping to finance.

The California Institute for Regenerative Medicine (CIRM), as the agency is formally known,  has about $27 million left, plus any additional funds that the agency might recover when awards are cut short as research fails to meet required milestones. The $27 million, however, is earmarked for sickle cell research in a partnership with the federal government.

In some past years, CIRM handed out as much as $300 million a year.

The agency's sole source of significant funding has been $3 billion in state bonds. It is pinning its hopes for the future on voter approval of a $5.5 billion ballot initiative in November 2020.  Playing a role in that effort is likely to be a CIRM-financed, economic study from USC that bolsters the argument that the economic benefits of the agency more than justify renewing its funding.

Dana Goldman, a professor of public policy, pharmacy and economics at USC and who directed the study, said that the private sector is not going to make the types of investments that CIRM has made in developing new treatments. He told the board last week that it would take only small improvement in success rates in such areas as stroke therapy to more than cover the cost of CIRM's activities. (Here is a link to his slides for the presentation.)

CIRM was created in 2004 by voters but has yet to invest in research that has resulted in a widely available therapy.

Maria Millan, CEO of CIRM. said in a news release:
“Programs, such as those funded today, that were novel stem cell or gene therapy approaches addressing a small number of patients, often have difficulty attracting early investment and funding. CIRM’s role is to de-risk these novel regenerative medicine approaches that are based on rigorous science and have the potential to address unmet medical needs. By de-risking programs, CIRM has enabled our portfolio programs to gain significant downstream industry funding and partnership."
Below is a list of the awards with links to the official summaries of the review of the applications, which can be found by clicking on the application number. Letters by applicants to the board can be found by clicking on the applicant's name. Additional letters of support for the research can be found on the agenda for the meeting. 

Here is the UCLA news release on the awards to three UCLA researchers.

Application Number 
(in millions)
UC Irvine/jCyte
Retinitis pigmentosa

Retinitis pigmentosa
Brain Neurotherapy Bio
Parkinson’s Disease
Limbal stem cell deficiency 
Immune deficiency 
City of Hope
Ovarian cancer
Neurona Therapeutics
Nicholas Corey
(no letter posted)

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