After a mere 4 years to get through the approval process, the Food and Drug Administration (FDA) has given the green light to the first clinical trial of stem cell therapy on human subjects. Biotech company Geron will use the trial to test spinal cord damage treatment by injection of precursor cells.
Being a Phase One trial, the test will only examine the safety of the therapy, not actual effectiveness. According to Wise Young, director of the WM Keck Center for Collaborative Neuroscience at Ruters University,
This is an important trial, there's a lot of hope riding on it. With this trial, we need to lower the expectations a bit. People are expecting miracles from a first trial, but it was only designed to test safety.
The approval was expected to be given last May, but last minute FDA objections required Geron to work closely with the FDA until final approval could be given, which happened Monday. Young is calling the trial the "first shot across the bow", and believes that it compares well with development efforts of antibiotics in the 1940s.
The trial also has its concerned pundits, and given the effect this can have on future clinical trials this is certainly understandable. Evan Snyder, Director of Stem Cells and Regenerative Medicine Program at Burnham Institute for Medical Research cautions about a mis-step at this stage of the game,
I think everyone in the field is taking their collective deep breath and hoping nothing bad happens. What would happen then would set the field back dramatically. I don't think it was ready for clinical trials.
One concern that experts in the field are expressing is that there lies a slight possibility of implanted cells creating a tumor known as a teratoma.Young hopes to avoid these problems by using precursor cells, which are less prone to cause tumors than the more potent stem cells.
While it is huge in its significance of the future of stem cell applications, the outcome of the trial itself is limited in its applications. If the trial can make it through Phases two and three, the target group remain those with recent spinal cord injuries, not those already in a wheel chair. While that alone is nothing to consider minor, it does show the nature of the focused trial.
The importance of the trial's impact, success or failure, really cannot be understated. Mark Noble, a professor in the Biomedical Genetics department at the University of Rochester, expresses this sentiment and the impact it could have on those suffering from spinal cord injuries,
In this early stage of stem cell medicine, its already clear that there will be some spectacular successes, but there will also be some failures. Even if this particular trial did not provide benefit or actually caused problems, this should not cause spinal injury patients to lose hopes, there are other technologies that are moving forward very rapidly.
