The first comprehensive external review of the California Institute for Regenerative Medicine (CIRM) has come to overwhelmingly positive conclusions about the state stem cell agency’s progress. But some in the biotech community continue to grumble that the agency, which began operations in 2006, is focusing too much on basic research rather than fulfilling its mandate of taking stem cell therapies from bench to bedside.
In its report, posted on CIRM’s website last week, the review panel wrote that it “congratulates the State of California,” noting that CIRM’s progress so far has been “remarkable.” It also provided the agency with a list of strategic recommendations as it evolves from start-up phase to mature organization. These include moving away from a funding agency model to a more “proactive” funding strategy, expanding and better defining its relationship with industry, and engaging more closely in regulatory issues.
But some say that many of these things should already have been done. “You’ve got to remember, their whole mandate is commercialization,” says William Caldwell, CEO of Massachusetts-based stem cell company Advanced Cell Technology (ACT). What the agency should have done, he says, is identified 3-5 potential therapies that were “low-hanging fruit” in terms of clinical development. “Let’s get those through and into the clinic so that we can demonstrate to the world that the [human embryonic stem cell] platform has value,” he says.
Also, Caldwell notes, the agency should already have called for project proposals that tackle some technical areas that need work, such as techniques for the cryoprotection of cells, as well as methods to coax cells into dividing more rapidly. “These are commercial issues that can help these products move into the market,” he says.
ACT, which last week received a green light from the Food and Drug Administration (FDA) to begin the world’s second-ever clinical trial using human embryonic stem cells, had set up base in California for some time in the hope of receiving CIRM funding, but was never awarded a grant despite applying several times. While CIRM could well have had its reasons, Caldwell says, 95% of the funding was going to nonprofit institutions -– in his view a misguided strategy.
A commenter to the California Stem Cell Report blog, identified as “an executive/scientist from a California biotech firm who must remain anonymous,” echoed Caldwell’s sentiments. “If CIRM was to hold true to its promise to deliver products in 10 years, then it needed to start translational activities immediately and not building infrastructure for already rich universities like Stanford, who enjoy $10B plus endowments,” the commenter wrote.
But plenty of others agree with the panel’s conclusion that the agency is doing a fine job. One of those is Hans Keirstead, a CIRM grantee who co-directs the stem cell program at the University of California, Irvine, and whose research underlies a stem cell treatment for spinal cord injury – the first to receive FDA approval to be tested in humans — under development by California-based biotech Geron.
“My feelings is that CIRM has done an exceptional job” in building a long-term vision that involved creating infrastructure and recruiting faculty, Keirstead says. “It’s not CIRM’s responsibility to invent treatments — it’s ours as researchers. And I think when we have, CIRM has been there” with the needed support.
CORRECTION (30 Nov): This post has been updated to correct the first name of William Caldwell.