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California stem cell agency needs to study itself

The California Institute for Regenerative Medicine has downplayed the recent exit from the stem cell field by Geron, but the fiasco raises questions about how the agency does business and whether its addiction to hype does a disservice to patients and taxpayers.

December 07, 2011|Michael Hiltzik
  • Geron of Menlo Park, Calif., announced in November that it was abandoning the stem cell field, halting a clinical trial that had enrolled five patients.
Geron of Menlo Park, Calif., announced in November that it was abandoning… (Paul Sakuma, Associated…)

For years, Geron Corp. had claimed to be in the vanguard among California companies engaged in stem cell research. So it was something of a stunner when it announced Nov. 14 that it was abandoning the stem cell field completely.

Geron's shares fell 20% the next day, but that was probably nothing compared with how far spirits must have fallen at the California stem cell agency, which just a few months earlier had made its highest-profile investment ever by awarding Menlo Park-based Geron a $25-million loan to help fund the first human trial of stem cell-based spinal cord therapy.

Since the announcement, the agency has been doing its darndest to reassure everyone that Geron's decampment isn't a huge embarrassment.

Such unexpected glitches are bound to happen when you're on the cutting edge of science, says the agency, which is known formally as the California Institute for Regenerative Medicine, or CIRM. "There are going to be fits and starts," its chairman, Jonathan Thomas, told me last week. Even so, he maintained, "we remain unwavering in our commitment to pursuing the science."

Agency officials point out that since Geron promptly refunded the $6.42 million it had drawn down from the CIRM loan, plus interest, this is a case of no-blood, no-foul. That's an exceedingly tolerant view of the ramifications, however. CIRM's approval of the loan last May had been preceded by what the agency says was "robust" peer review and proclaimed by the agency's founder and then-chairman, Robert Klein, to be a "landmark step for CIRM." In bailing out of the field, Geron halted a clinical trial that had already enrolled five patients.

So we're talking at least about months of wasted effort by CIRM and Geron's researchers, crushing disappointment for those patients and conceivably a major setback for stem cell science generally. Thomas observes that Geron said it made its decision strictly on financial grounds, not because of scientific reversals. But for an R&D company financial considerations always encompass scientific judgments, and Geron plainly concluded that the prospect for profits from stem cell therapies was receding.

The Geron fiasco underscores the old questions, and raises new ones, about what CIRM is supposed to accomplish, how it does business and whether its addiction to hype does a disservice to patients and taxpayers.

These questions are only going to become more important in the future, as CIRM prepares to ask California voters for new billions to supplement its original $3-billion bond issue authorized by voters through 2004's Proposition 71, which established the agency.

CIRM loves to compare itself to the federal government's biomedical research agency, the National Institutes of Health, but the two bodies are very different. The responsibilities of NIH are broad enough for it to make disinterested judgments about programs and scientific approaches. CIRM, however, was designed from the start (by Klein, who oversaw the drafting of Proposition 71) to focus on a very narrow field of biomedical science — embryonic stem cell research — and to promote that research in California as a sort of economic development tool.

These two goals have always been ethically and scientifically incompatible, and the Geron case points to why.

To begin with, there's evidence that CIRM, anxious to show progress toward bringing stem cell therapies to market, downplayed legitimate questions about the state of Geron's science and the design of the clinical trial. CIRM's anxiety sprang from the Proposition 71 campaign itself, which snowed California voters with exaggerated promises of cures for conditions such as Alzheimer's, Parkinson's, diabetes and spinal cord paralysis.

The latter was Geron's baby. The company last year won approval from the Food and Drug Administration to start injecting patients with cells derived from stem cells. This trial was a preliminary step toward showing that the process might help regenerate critically injured spinal tissue and end the subjects' paralysis.

But Geron had been criticized in the past for over-promising results. Some researchers questioned the design of its clinical trial and even whether spinal cord injury was the best subject for the first tests of stem cell therapies on humans. Says Joan Samuelson, a Parkinson's patient advocate who cast the sole dissenting vote on the CIRM board against the Geron loan: "I felt at the time that not all my questions had been answered about the readiness of that trial by that company."

None of these issues were aired publicly in the run-up to the vote, because CIRM didn't disclose in advance that Geron was the loan applicant. Nor did it disclose that its own scientific review panel had awarded the Geron trial a scientific score of only 66 out of 100; that fact, along with other details of the board's consideration of the Geron loan, was pried out of CIRM later by David Jensen, the tireless proprietor of the indispensable California Stem Cell Report.

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