Friday, December 20, 2013

CIRM, the stem cell bandwagon, and the hard sell

Biomedical science moves from one hot fashion to the next. A new technology emerges, investigators jump on the bandwagon and pitch it to the general public as the next great thing: a way to cure this, that or the other disease. I don't think the public understands just how extremely difficult it is to advance even the best ideas to a point where they have any hope of being transformed into a therapy, let alone the enormous difficulty of developing a therapeutic agent. We work with immensely complex systems (cells, tissues, the entire body) and, frequently, do not even have a halfway decent blueprint of the systems we're trying to manipulate.

Back when I started, the flavor of the month was gene therapy. Later, antisense, siRNA and genomics all had their moment in the spotlight. The beautiful thing is that these approaches sometimes pay off: it just takes a lot of time and slow, painful, grinding research. Often, they don't. Monoclonal antibodies were the hot new thing for drug development in the early 1980s, making the covers of Newsweek and Fortune in 1985. They faded from the public limelight, but investigators and biotech companies kept plugging away and, in 1997, important new therapies for cancer and autoimmune diseases based on humanized monoclonal antibodies began to emerge. It has been exciting and gratifying to watch the steady roll-out of important antibody-based therapies. Gene therapy, on the other hand: well, that's been a different story.

Which brings me to to stem cells. In 2001, the Bush administration severely limited research using human-embryo-derived stem cells. In response, in 2004, a group of activists sold California voters on the promise of the sexy new technology of "regenerative" medicine. California voters--perhaps a bit too trusting--jumped on the bandwagon, passing proposition 71 to fund the research. Scientists were heavily involved in making the pitch, most notably scientists who would directly benefit from this source of new grant funds (though they didn't make any great efforts--not any effort at all really--to point this out). Oh, by the way, how many lines of text in print advertisements or in scripts for TV and radio commercials did the proponents devote to conveying how challenging it would be to actually develop therapies? Or informing the lay public that stem cell-based therapies might not pan out at all?  Here's one hint: a central topic of one of the first stem-cell planning meetings held at my institution after Prop 71 passed was how to dampen down the very expectations that the campaign had raised way, way too high.

In 2009, restrictions on federal funding of human embryonic stem cell funding were dropped. Also, new sources of stem cells not involving human embryos have been developed. The California agency doling out the stem cell research funds (CIRM, California Institute for Regenerative Medicine), has finite access to borrowed money and will award its last grants in 2017.

The good news is that the federal restrictions are gone: now stem cell researchers can compete on equal footing with researchers taking other approaches to finding cures. Since their approach is so promising, they should feel confident that their proposals will fare well in the grant application review scrum. Oh, wait, surprise! They are already making noises about wanting the good people of California--already some of the most highly taxed in the nation--to sell  more bonds to support more research. Whose research? Theirs of course! Why compete for funding with investigators trying to cure cancer, nerve injury, diabetes etc with different approaches when you can pitch your story right to the well-meaning voters, going through an agency that is rife with conflicts of interest? If the stem cell folks decide to go ahead and ask the people of California for more money for research, will the proposition they write be more at aimed at curing diseases (in which case research using the most promising approaches, whether stem cell-based or not, will be considered for funding) or will it be equally aimed at funding their own labs and thus promoting their own careers (including salaries)?

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