1st FDA Approved Clinical Trial with Embryonic Stem Cells

Pollack, Andrew. "FDA Approves a Stem Cell Trial." The New York Times. 23 Jan 2009. The New York Times, Web. 11 Nov 2009. <http://www.nytimes.com/2009/01/23/business/23stem.html>.

Shortly after President Obama's inauguration in January of this year, the FDA approved the world's first clinical trial for a therapy derived from human embryonic stem cells. This trial will include 8 – 10 participants with severe spinal cord injuries. Stem cells are to be injected into the damaged site 1 – 2 weeks after the injury, as this is the optimal time frame believed to facilitate this type of therapy. Geron, a California based biotechnology company, announced the clearance of the clinical trial. This approval marked a research milestone. Just months before in March 2008, the trial was rejected on the basis that the proposal ‘lacked sufficient data.’ Others like to argue that there was severe ‘political overhang’ from the Bush administration.

The trial has been deemed Phase I – aimed mainly at testing the safety of the therapy. Despite the fact that Geron had identified 7 medical centers across the country that expressed interest in participating in the trial (pending approval through their Institutional Review Boards), there is still some uncertainty about whether this is an ‘appropriate’ trial to conduct. If the therapy is unsafe or does not work, there might be a public backlash against the scientific community – halting any existing embryonic stem cell research and severely crippling the field.

In defense of the notion that this first trial might not be successful, Geron argues that the purpose of the trial is safety, not efficacy. Though practical, this statement is unnerving because the scientists don’t know what results they will yield from the experiment. At best, the participants will experience some restoration of nerve tissue surrounding their spinal cord damage. At worst, the trial will be a disaster. For the sake of the scientific community, let’s hope for the former.

Geron's proposal of 22,000 pages discussed the success of the therapy in small animal models and evidence that the cells did not form tumors in these animals - one of the main concerns surrounding the use of embryonic stem cells. What about the total cost of the application and preclinical research? $45 million. For a company that has not yet been profitable, this trial is risky to say the least. But maybe it is risk and ingenuity that are necessary to get this type of research off the ground.

The approval of this trial was a remarkable benchmark set forth by the U.S. government and scientific community. Though Geron has put its company’s future in an uncertain place, it is such innovative investigation and pioneering attitude that will further the research of embryonic stem cells as a treatment therapy. I think this work is incredibly interesting and powerful. Powerful enough to change the minds of adversaries to stem cell research? This will depend on the outcome of the trial … as will the future of embryonic stem cell research, the future of Geron, the future of policy in the United States, and the future of those patients who would probably do anything for a cure.

~ Courtney Mannino