Over the past several years I have tried to gain as much information on gene therapy for Parkinson's as I could. I don't believe that gene therapy is another GDNF. I am a medical scientist and work extensively with the pharmaceutical industry. (My research is not involved with PD, but I know enough about science and clinical trials to be an informed consumer of data on gene therapy.)

There are three companies that are working hard in this area with slightly different approaches: (1) Oxfordshire Bioscience-in the UK; (2) Ceregene and (3) Neurologix in the US. (If there are others that I am not informed about please let me know.)

As far as I can tell Oxfordshire is actually behind the other two companies in their research. Ceregene's CERE 120 has completed Phase I and will complete Phase II and report results this year in Q4. (Enrollment for Phase II is now complete and patients are still being followed from Phase I as well.) Ceregene entered into a development deal with Genzyme last year that is quite substantial. In addition, Michael J Fox Foundation has contributed funds to Ceregene's research. The results from their Phase I trial are outstanding. Some patients are getting close to 3 years post surgery with no adverse events and continued improvement on standard measures at each follow-up point! This is following a single treatment. PET scans are showing cell regrowth. The cell growth is not out of control in any way.

The Neurologix approach is similar to the Ceregene approach. They have had a similar number of Phase I patients and the findings are very much like the Ceregene findings. Their study was unique in that the patients int he Neurologix study got treatment to only one side of their brains. The results were consistent with this test in that improvement generally occurred on the side of the body that would be predicted by the treatment. The Neurologix patients also showed significant improvement with continued improvement for the study participants who are about 2 years post initial treatment.

Neurologix has just initiated its Phase II study. It is also important to note that the FDA gave Neurologix a Fast Track designation to facilitate their research and potentially bring the drug to market as quickly as possible. (My company works closely with the FDA and Fast Track designation is not easy to come by.)

My wife has had Parkinson's for nearly 5 years now and I know that people with PD have been frustrated in their desire for a cure for years. I don't see gene therapy for PD as a cure, however, the impact looks to be greater than anything else that is in the pipeline at present. For people in the studies that I have read the improvements were in the range of 25%-60%+. Again, please remember that people are continuing to get better over time.

With stem cell research set back by at least a decade by an anti-science Bush administration, I believe that it is far more likely that gene therapy will affect the national and international population of people with this terrible disease. My guess is that (as long as there is no terrible unforeseen event for participants in the clinical trials--over the next year or so) we could see a commercially available gene therapy in the next 3 to 5 years.

That would be very exciting for all of us. Let's hope.