Monday, March 24, 2014

The Long Odds Story of Stem Cell Research

It was the sort of story that Californians did not see during the ballot campaign 10 years ago that created the state's $3 billion stem cell agency.

The story line then was of hope and the imminent prospect of cures for a host of diseases that afflicted half of California's population.

Today in the San Francisco Chronicle, Stephanie Lee wrote of biotech companies that go for more than two decades without ever developing a commercial product. Lee's poster child was Geron Corp., which abandoned stem cell research in 2011 along with a $25 million loan from the stem cell agency.

Lee wrote,
“A company that goes 24 years without ever selling a product may sound unusual. But in biotechnology, it's not that uncommon. 
“Take Geron Corp. in Menlo Park, which has struggled to develop a therapy - any therapy - since its founding in 1990.”
Lee noted that Geron has an accumulated deficit of $893 million in debt since its founding and is now facing a number of lawsuits from unhappy investors.
“Faced with 10- to 15-year timelines and uncertain regulatory outcomes, companies and investors might plow hundreds of millions of dollars into therapies that will never see the light of day.”
Besides Geron, Lee also mentioned Isis Pharmaceuticals and Dendreon Corp. as examples of biotech firms with a lengthy development history.

She wrote,
“Overall, the odds of getting a drug to market aren't much better than gambling in Las Vegas. Only 1 in 5,000 to 10,000 compounds discovered in the lab gains FDA approval.”
Those odds, we should note, do not reflect development of stem cell therapies, but rather simpler substances. The expectation is that odds will go higher for stem cell therapies.

All of which is not the best news for the stem cell agency, although the information is not all that new. It was out there during the 2004 stem cell campaign, but the stem cell community did not want to talk about it, and the mainstream media largely did not report it.

The figures remained unchanged today, although a more positive aura surrounds biotech than has existed for several years. Nonetheless, the story of long odds and fruitless research is one that bedevils the stem cell agency. It will run out of funds for new awards in 2017 and is looking for ways to avoid its financial demise. Creating a new narrative, one with a brighter future, is the agency's task as it tries to develop new funding.
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8 comments:

  1. Anonymous1:57 PM

    It is misconception that the odds will go higher to develop a stem cell product than a simpler substance drug. For diseases caused by cell or tissue loss, only sth more complex like a cell can fill in to restore the function, no simpler substance can. That is why all those drug companies have searched for 20, 30, or more years, still cannot find any simpler substance drug or why Geron failed.

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  2. Anonymous2:05 PM

    It is misconception that the odds will go higher to develop a stem cell product than a simpler substance drug. For diseases caused by cell or tissue loss, only sth more complex like a cell can fill in to restore the function, no simpler substance can. That is why all those drug companies have searched for 20, 30, or more years, still cannot find any simpler substance drug or why Geron failed. The FAD regulation processes do need to be simplified. What’s the use if a stem cell product, obvious working since it’s come from human embryos, have to go through 10 or 20 years to get FDA approval while a lot of patients need it now, cannot wait for 10 or 20 years.

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  3. Great coverage as usual David. I'd like to discuss the statement that "The expectation is that odds ill go higher for stem cell therapies." I've been pushing the Alliance for Regenerative Medicine to do some analysis of the limited data we have on this to address this very question. Unfortunately this has not yet been completed so I don't have any data to help refute the notion but I'm not convinced there is a pervasive expectation that cell therapies will have a higher discovery-to-commercialization failure rate than other biopharmaceuticals. I'm curious if you have any supporting references or sources to support this theory.

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    1. My comment about the expectation odds on stem cell therapies was based on multiple remarks at several CIRM board meetings by board members, mostly from those with business or scientific expertise. The agency staff may have commented as well but I can't recall with certainty. The reasoning is basically that this is new stuff, and the FDA will be conservative, more conservative than with familiar therapies. Geron's prolonged FDA experience with its hESC trial is a good case in point, although being first is always hard. Also being new can mean that more unexpected obstacles can arise, slowing or halting the process. It seems a reasonable scenario, one that a prudent business should plan for.

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  4. This reality of biotech is exactly why CIRM needs experienced industry professionals on their advisory board and on their grant review panels to assess the real world probability of technical success, rather than strictly voting on interesting science in an unmet medical need, of which there are far too many. It's also important for outsiders to realize that 80% or more of all drugs that make it into clinical trials fail, which drives up the cost of medicines since the clinical trials are where the majority of costs associated with drug development are incurred (tens to hundreds of millions of dollars).

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  5. Error: Isis Pharmaceuticals got its first approval in 1997, not 2013. The product was Vitravene, for those with an AIDS-related eye infection. I wrote about Vitravene at the time.

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    1. Thanks for the heads up, Bradley. For readers who may not have gone to the Chronicle story, the figure you mentioned as erroneous was contained in that article. The specific year was not mentioned in the item on this blog so I am reluctant to make a correction here on something that I did not write. Nonetheless, I welcome double and triple checks on accuracy from all readers.

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  6. Barbara1:57 PM

    CIRM wouldn't need to be thinking of how to create a new narrative if they had delivered on the promises they made to voters in the first place. By now, thousands of patients could have been treated with adult stem cells and data would be available to help satisfy taxpayers that their money was not just going towards endless research for products that would not be delivered to them in decades, if ever. CIRM has let voters down. You call it a story line that they were given and I call it a tall tale.

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