California’s stem cell agency on Thursday charted a fresh, $890 million course that aims to make good on its unfulfilled promise to voters that it would produce stem cell treatments ready for wide-scale use on patients.
The agency proposes to help fund 50 new clinical trials for stem cell therapies on top of the 15 it already has underway. Next year, it plans to crank up a $150 million partnership with private investors to turn research into cures. And it plans to launch a site on the Internet to connect scientists with partners who can help them get their research beyond the basic level.
State bond funding for the agency will run out in 2020.
C. Randal Mills, president of the California Institute for Regenerative Medicine, as the agency is formally known, said the plan was devised to have “the greatest possible impact for our patients. We didn’t want something ‘good enough.’ We wanted something transformational.”
Directors of the Oakland-based agency approved the plan unanimously at a meeting in Los Angeles.
Asked for comment, CIRM board member Al Rowlett, chief executive officer of the Turning Point mental health program in Sacramento, said, “It’s ambitious, but then isn’t that what the people of California were when they approved Proposition 71. They wanted to create something that was going to change the face of medicine. That’s what we hope to do.”
The Proposition 71 ballot initiative created the $3 billion agency in 2004 following a campaign that suggested stem cell therapies were right around the corner. None, however, has emerged through the stem cell agency.
The agency’s plan for the next five years says it would benefit the people of California by creating “an industrial stem cell therapeutic powerhouse that expands the tax base, adds high-quality jobs and increases the likelihood of the commercialization of stem cell treatments for patients with unmet needs.”
The spending plan is not without risks for the state. The agency itself devoted three pages in the 45-page plan to its perils, ranging from foot-dragging by federal research authorities to a lack of business interest in developing stem cell cures.
Late last month, Mills, who became president of the $3 billion enterprise in May 2014, told a committee of CIRM directors, “No matter what we do, we may not be able to convince investors that stem cell therapies are a good idea.”
Mills, however, remains optimistic that the agency’s staff of about 55 people can pull it off. The proposal, an overhaul of the agency’s previous strategic plan, carries forward earlier moves by Mills. Within months of his arrival, he introduced what he called a radical overhaul of the agency, speeding money to researchers and focusing hard on financing clinical trials, the federally regulated last stage before research can move into widespread use.
The CIRM plan calls for spending $620 million on clinical work and translational research, which aims to take basic discoveries beyond the most preliminary stage. Basic research would receive $170 million. Educational programs total $50 million. Another $50 million would go for infrastructure.
One of the riskier elements of the proposal may be the agency’s plan to offer $75 million to private investors to begin a partnership in which they would have access to the best of the CIRM-funded research that doesn’t already have a private partner.
Investors, who could be big drug companies, smaller firms or venture capitalists, would have to add $75 million. The agency would also continue to support the selected research, thus minimizing the risk to the private investors.
For months, Mills has talked about how reluctant private investors are to engage in stem cell therapy development. The process is expensive and new, presenting a host of problems that conventional drugs do not. Even with conventional drugs, the vast majority of those entering clinical trials never make it to the marketplace.
Competition for the $75 million is scheduled to begin behind closed doors early next year. Gregory Bonfiglio, managing partner of Proteus Regenerative Medicine, a Portola Valley venture capital firm, said such efforts by the agency are long overdue.
“There are risks inherent in the development of new, disruptive technology,” he said. “The bigger risk is failing to deliver on (CIRM’s) underlying promise to bring new regenerative therapies to patients. … The bigger risk is not doing anything.”
Another significant element of the plan involves creation of accelerating and “translating” centers, funded at $15 million each, beginning next year. The centers would provide much of the work needed to negotiate federal rules and regulations, and win ultimate approval of a therapy.
The competitive proposals may attract interest from UC Davis, which has received $127 million so far from CIRM.
David Jensen is editor of the California Stem Cell Report, which has published more than 4,000 items since 2005 dealing with California stem cell issues.