Nature Medicine | Spoonful of Medicine

ISSCR 2010: Will reprogrammed cells improve drug discovery?

The pharmaceutical industry has had a notoriously abysmal success rate at turning promising compounds into approved medicines. The usual number that gets tossed around is that only one in ten drug candidates will prove safe and effective. But the true success rate might be closer to one in 35, according to Corey Goodman, former president of Pfizer’s Biotherapeutics and Bioinnovation Center and now chairman of the board at iPierian, a South San Francisco-based biotech focused on using induced pluripotent stem (iPS) cells in drug discovery.

Goodman chalks up the failure of the drug pipeline to a reliance on rodent models for understanding human disease. The problem: many drugs that work in mice and rats do not prove up to scratch in people.

That’s where reprogrammed stem cells come in. “If you can create the disease in a dish… then you’ve got a model that you can manipulate dramatically,” Alan Trounson, president of the California Institute for Regenerative Medicine, said at a press conference today at the International Society for Stem Cell Research annual meeting in San Francisco.


The panel at the press conference — which also included Dolly-cloner Ian Wilmut and Children Hospital Boston’s George Daley (the subject of this month’s Q&A) — reiterated the hope that iPS cells will drastically improve the efficiency of the drug discovery process. As an example, Goodman referred to findings from iPS cells taken from people suffering from spinal muscular atrophy (SMA).

According to Goodman, iPierian scientists tested around 15 different compounds that had previously shown promise in animal models of SMA but ultimately failed in the clinic. All the drugs did not stop disease progression in the iPS cell-induced neurons, and some of the compounds even proved toxic.

Had iPS cells been around earlier, they could have saved researchers years of wasted effort, said Goodman. What’s more, with iPS cells, “you can at least start to get hits of promising compounds that do what those other compounds failed to do,” he added.

Researchers have not yet discovered any marketable compounds using iPS cell models, but the panel argued that it’s only a matter of time.

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    Mutuelle santé said:

    It seems that we should be thankful to the mice and rat communities for testing all the drug before us. It is through trial and error that many discoveries were found.