Dodging the Politicos and Delivering Billions to Stem Cell and Gene Therapy Research in California
CIRM dishes out the dough in contrast to turmoil in federal biomedical research funding
Ray Therapeutics of Berkeley, Ca., is expected to receive an $8 million award next Thursday from CIRM for a phase one trial.
While the Trump administration slashed away last week at federal medical research, California scientists flooded a little-known state department with applications for a total of $37 million to create revolutionary therapies for afflictions ranging from autism to cancer.
The state agency is the California Institute for Regenerative Medicine (CIRM), as the stem cell and gene therapy program is known. It has a bulging, $4 billion purse, which it uses to finance research. Last Thursday, 372 scientists applied for some of the cash. Only 12 applications are expected to be approved.
CIRM has been busy with its largesse recently. Just last month, at a meeting on the banks of the Sacramento River, it awarded $26.5 million to push clinical research involving epilepsy, Angelman syndrome and graft versus host disease. That same day, it also approved a new, $429 million spending plan designed to boost its impact on biomedical research. And last Friday, CIRM opened the door for applications in a $84 million round with awards up to $13 million each. CIRM said it will give preference to neuro disease proposals that deal with disorders of the brain, spinal cord and peripheral nervous system.
All this can be attributed to the voters of California, who created CIRM in 2004. They backed it with $3 billion then and another $5.5 billion in 2020 via ballot initiatives. The state borrows the money, so interest pushes the total cost to taxpayers to an estimated $12 billion. CIRM has “only” $4 billion left to spend under the terms of the initiative.
CIRM was created 20 years ago as a reaction to another federal research brouhaha -- the Bush administration restrictions on federal financing of human embryonic stem cell research. In what was likely a national first, a ballot initiative was used to create the agency and isolate it from political influences involving the legislature and the governor.
Last week’s 372 applications from businesses and academia seek funding for basic research into knowledge gaps in human biology and/or disease pathology. The awards run up to $3 million each over a three-year period, close to the end of the Trump administration.
The CIRM staff will go over the proposals and send a much shorter list to its grant reviewers, who come from out of state and perform their reviews anonymously, behind closed doors. They make the de facto decisions on the awards, which are ultimately ratified by a subcommittee of the 35-member CIRM governing board. That will occur some months down the road.
The last round of this particular program — DISC0 — drew only 110 applications. Beyond the federal turmoil, “there may be several reasons for the increase,” a CIRM spokesman said. We’ve seen a steady increase across all programs, even before the new administration came in as the field grows.
“We have also made more efforts to spread awareness among scientists, including boosting marketing through email and social media. Federal actions may have also contributed, but (are) not the sole reason.”
More rounds of funding that reflect CIRM’s new priorities are scheduled to be offered later this year. And in just seven days, CIRM directors are expected to approve an $8 million award to Paul Bresge of Ray Therapeutics, Inc., of Berkeley, Ca. The application says Ray will provide an additional $3.4 million in co-funding, a requirement in this particular round, for a phase one clinical trial.
Bresge has already received $8 million from CIRM for his past work (see video above). Next week’s award is aimed at improving the visual function of people with retinitis pigmentosa by “delivering a light sensitive gene to retinal cells, allowing the surviving cells to detect light and transmit vision signals to the brain.”
CIRM said, “This approach is independent of the underlying genetic mutation and therefore could target a broad patient population.”
The award will bring to 116 the number of clinical trials that CIRM has helped to finance.
(For more about the sweeping scope of CIRM’s operations, see this overview on the California Stem Cell Report, the only independent, regular source of news and information about CIRM for the last 20 years.)