Proposed Clinical Trial Of Gene Therapy For AADC Deficiency

Over the last several years the PND Association has closely followed the AADC gene therapy trial utilized for Parkinson's disease. In 2006 the PND Association reached out to Avigen the biomedical company initially involved in this project. Discussions were underway about compassionate use treatment when the project was taken over by Genzyme .

In 2008 members of the PND association board and M&S advisory committee met with Genzyme. At the time of the meeting Genzyme conveyed that once the clinical trial in Parkinson's patients was complete they would consider allowing others to utilize the vector on behalf of the AADC pediatric population.

When the clinical trial was completed in 2009 the PND Association reached out to Dr. Krystof Bankiewicz, MD, PhD, the neuroscientist who developed the procedure for vector delivery. In July 2009 Dr. Bankiewicz made the extraordinary commitment to works towards a gene therapy clinical trial on behalf of the AADC population. Since 2009 Dr. Bankiewicz and leaders in the field have been working towards determining the best direction for this project.

At this time we are pleased to share that Dr. Bankiewicz in conjunction with the National Institute of Health and clinicians from Columbia Presbyterian have committed to working towards a clinical trial of AADC gene therapy for children with AADC deficiency.

The PND Association has been involved with this project and to date has provided $40,000 in funding with an additional funding commitment of $10,000 in March 2013. The study protocol is currently under development. Please continue to check the website for updates.

For detailed information about clinical trials and how drugs/treatments are developed please visit:

http://clinicaltrials.gov/ct2/info/understand

http://www.fda.gov/drugs/resourcesforyou/ consumers/ucm143534.htm