The Scientist : NewsBlog Print: Right on Geron
The Scientist: NewsBlog:
Right on Geron
Posted by Elie Dolgin
[Entry posted at 23rd January 2009 09:43 PM GMT]

The US Food and Drug Administration's decision to green light a human embryonic stem cell-based therapy for spinal cord injury isn't just good news for the biotech in question, Geron Corp., analysts say -- instead, they argue, the move opens the door to an entire market that has heretofore been stymied.

"It's the first clear evidence of the FDA's willingness to permit cells derived from pluripotent stem cells to go into human trials," Kenneth Aldrich, CEO of Oceanside, Calif.-based International Stem Cell Corporation, told The Scientist. "It'll make it easier for us and everyone else, because we now have a roadmap. We know what the FDA wants."

"What this essentially says is that the FDA is comfortable with trials involving human embryonic stem cells," said Robert Lanza, chief scientific officer of Advanced Cell Technology (ACT), a biotech company in Cambridge, Mass. "Now, others moving forward don't have as high a hurdle to jump."

"It's an exciting sign that the FDA is satisfied with the initial safety of stem cell therapy," said Stephen Dunn, director of research at Boca Raton, Fla., securities firm Dawson James. "At some point the FDA has decided that the benefits outweigh the risks."

"The bottom line: Now we're going to get some eff-ing data," said Robin Young, a private analyst who runs the annual Stem Cell summit and was the author of Stem Cell Analysis and Market Forecasts 2006-2016.

Nearly all stem cell companies saw their stock prices surge today following Geron's announcement. Investors don't generally differentiate between embryonic and adult stem cells, so "everyone has benefited from this news event," said Reni Benjamin, managing director and a senior biotechnology analyst at Rodman & Renshaw in New York. "Once one [company] is approved, it paves the road for cellular therapies for other companies to be approved," he added. "This is really a trailblazer."

Geron's role in pushing the application forward should not be understated, noted Young. "This was the result of a significant amount of very hard work on the part of Geron. They deserve huge pats on the back for getting this done," he told The Scientist.

"All eyes are now going to be on the functionality [of the stem cell-derived cells]," said Lanza, who noted that ACT has already consulted with the FDA about using human embryonic stem cell-derived retinal cells to treat blindness, and he expects to file an investigational new drug (IND) application by the summer, "if not before then."

Lanza said that the timing of today's decision sends a clear political signal to the industry. "It's not an accident that just a few days after the inauguration of the president this thing moves ahead," he said. "The Republicans always had an antagonistic relationship with the FDA." Most analysts, however, said that the wheels had been turning long before Tuesday's change of leadership. "I don't necessarily believe that there was a political influence," said Benjamin. "This process was started last year when Bush was firmly in office." Karen Riley, an FDA spokesperson, echoed this sentiment. "The timing of the FDA's action was driven by the date the company submitted its application," she told The Scientist. "It has absolutely nothing to do with [the inauguration of President Obama]."

Benjamin said he was surprised by today's announcement, as most analysts did not expect the FDA's stamp of approval to come this soon. "It's a monumental point in a time," he told The Scientist. But the trials will be slow going, he warned. Clinical protocols still need to be approved and staff have to be trained at the seven clinical sites that are participating in the trial, which is expected to enroll around ten patients at a rate of one patient per month. Only one site -- Emory University -- has so far been approved, Benjamin noted, adding that it should take around six months before the trials start, and at least a year before the first data become available. "It's no small feat and a very great achievement, but it's early days and clinical development will still take some time," he said.


Related stories:
  • FDA OKs stem cell trial
    [23rd January 2009]
  • Stem cell trial nearly a go?
    [17th October 2008]
  • First embryonic stem cell trial stalled
    [15th May 2008]

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    A little-known California Law...
    by Don Reed

    [Comment posted 2009-01-26 14:38:36]
    Pride compels me to note that a little-known California law, the Roman Reed Spinal Cord Injury Research Act of 1999, formerly Assemby Bill 750, was the source of the initial funding for Dr. Hans Keirstead's great work, soon to become the world's first human trials for an embryonic stem cell therapy.

    I will never forget March 1, 2002, when I held a formerly paralyzed laboratory rat in my hand, and felt the little muscles working again-- while my paralysed son Roman Reed sat in his wheelchair a few feet away.

    This was at the Reeve-Irvine Research Institute at UC Irvine, on the opening day of the Roman Reed Lab, where much of the groundbreaking research took place.

    Christopher Reeve joined the celebration, calling us on the telephone-- and we got so excited to hear his voice-- we photographed the telephone!

    Who would have thought it would take seven years more work on Dr.Keirstead's part, and 21,500 pages of Geron correspondence with the FDA and the huge financial courage and sheer foresight on behalf of Geron's Thomas Okarma to bring us where we are today?

    As my son always says, everybody involved "took a stand-- so one day, everybody can."

    best,

    Don C. Reed


    Restoring science to its rightful place!
    by Paul Browne

    [Comment posted 2009-01-26 05:20:02]
    I doubt that we'll ever know for sure, and at least for some time, if the FDA deliberately waited until Bush was out of office before approving the Geron trial, but the timing seems too much of a coincidence. Even if the Bush administration did not seek to delay approval of this particular clinical trial the fact that the FDA waited until they were out of the way before announcing their approval of the trial speaks volumes about the nature of the relationship between the FDA and the Bush administration. Hopefully the Obama administration will restore scients to its rightful place at the heart of science policy, and indeed all policy!

    I've blogged about Geron's work here LINK and I'll confess that I'm quite optimistic about this one, as Geron seem to have been pretty thorough in their pre-clinical work and conservative in their clinical trial design.

    Finally I'll add that whether we're talking about ESC, iPS, or adult stem cells it's worth repembering that the threat does not only come from religious conservatives, animal rights activists are also campaiging to stop much of the research that underpins the whole stem cell field.


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