Geron CEO: Q&A

On the afternoon of Wednesday, January 21, Geron Corp.'s president and CEO Thomas Okarma received a phone call from the US Food and Drug Administration, notifying

him that Geron's 21,000-odd page investigational new drug (IND) application for the first-ever Phase 1 human trial of a medical treatment derived from human embryonic stem cells had been linkurl:approved.;http://www.the-scientist.com/blog/display/55353/ The decision, which lifted a hold on the trial in place since May of 2008, has been greeted by linkurl:enthusiasm;http://www.the-scientist.com/blog/display/55354/ from industry and patient advocates.Okarma spoke with __The Scientist__ today (January 26) about the decision, rumors over the political timing of the announcement, and when the much-anticipated trial is set to begin. __**The Scientist**__: How does it feel to finally be entering the clinic with a stem-cell treatment for spinal cord injuries?__**Thomas Okarma**__: It is probably my greatest professional accomplishment. It is something that I and the people that I work with have been trying to do for a long time. Finally getting permission to demonstrate safety, feasibility, and hopefully efficacy of the first embryonic stem cell-derived therapy in a devastating condition like spinal cord injury is the first page of the new era. There's no question that living cells produced this way will go way beyond the reach of pills and surgical techniques, and can provide a much higher value paradigm for clinical medicine. We don't see any disease, condition, or injury that would somehow be beyond the reach of this technology.__**TS**__: Why do you think that Geron was the first company to get to this point? __**TO**__: We spent as much on this program as the NIH spent on the [human embryonic stem cell] field for the whole country. That means, of course, that for the academic researcher in embryonic stem cells, he or she was terribly disadvantaged compared to our workers. We plan to publish elements of the IND package in the peer-reviewed literature that will speak to the extensive work that we did to prove safety. That rightly is not only the concern of the academics who haven't seen the data, but it was the concern of the FDA who did. And the clearance of the IND is really proof to the world of the safety of this new approach.__**TS**__: Why did the FDA put a hold on your clinical trial application last May?__**TO**__: They had lots of issues regarding safety. They had concerns like where do the cells go, concerns about how the cells would be prepared by the clinical trial sites, concern about how algorithms for patient inclusion would be generated. Those are examples of the kind of things that occupied the to-ing and fro-ing between the time we were put on hold and last Wednesday when the hold was released.__**TS**__: Did you expect that obtaining the FDA's approval would take this long?__**TO**__: Given the size and complexity of the application -- this is the largest IND submitted to the FDA, ever -- we knew it would be a difficult review. And it was. There was a lot of information for the agency to get their arms around, and we were not surprised that they would have issues. There's no playbook for this, either for us or the agency. There was no precedent, so we created one. And we now have a proprietary roadmap for how to address the legitimate concerns on other embryonic stem cell-like therapies.__**TS**__: The FDA's decision came the day after President Barack Obama's inauguration. Was there a political aspect to this announcement?__**TO**__: No, we have absolutely no evidence that that was the case. The timing actually was 30 days to the day after we had submitted the last written submission back in December. By law, the agency is obligated to respond within 30 days of receiving the most recent update to the application, so we were the ones who set the timeframe. Having said that, there's no question that the Bush administration made the whole process much more onerous for us and everyone else.__**TS**__: When can we expect to see the Phase 1 trial start?__**TO**__: There's a lot to do before the trial starts, unfortunately none of which could begin until now when the hold was lifted. There are IRB [institutional review board] approvals at each site. There are clinical trial committees that have to approve the protocol. We have extensive training now to do. So that's a lot. That boils down to probably initiating the trial at one or two sites in the early summer.__**TS**__: What's next in Geron's pipeline?__**TO**__: Our second IND will probably be [embryonic stem cell-derived] heart muscle cells. With these cells, one would go home with functionally restored tissue that is also responsive to standard cardiac drugs. We are currently in the last pre-IND phase -- testing these cells in large animals so that we have a better assurance that these cells don't generate arrhythmias. That's kind of the last hurdle. This is not an IND that's going to go in this year, though. It's probably a 2010 event. __ Image: Julie Jacobson, Associated Press __ **__Related stories:__** *linkurl:Right on Geron;http://www.the-scientist.com/blog/display/55354/
[23rd January 2009]*linkurl:FDA OKs stem cell trial;http://www.the-scientist.com/blog/display/55353/
[23rd January 2009]

Geron CEO: Q&A

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