Parkinson’s Disease Gene Therapy: Success by Design Meets Failure by Efficacy
 

Over the past decade, nine gene therapy clinical trials for Parkinson’s disease (PD) have been initiated and completed. Starting with considerable optimism at the initiation of each trial, none of the programs has yet borne sufficiently robust clinical efficacy or found a clear path toward regulatory approval. Despite the immediately disappointing nature of the efficacy outcomes in these trials, the clinical data garnered from the individual studies nonetheless represent tangible and significant progress for the gene therapy field. Collectively, the clinical trials demonstrate that we have overcome the major safety hurdles previously suppressing central nervous system (CNS) gene therapy, for none produced any evidence of untoward risk or harm after administration of various vector-delivery systems. More importantly, these studies also demonstrated controlled, highly persistent generation of biologically active proteins targeted to structures deep in the human brain. Therefore, a renewed, focused emphasis must be placed on advancing clinical efficacy by improving clinical trial design, patient selection and outcome measures, developing more predictive animal models to support clinical testing, carefully performing retrospective analyses, and most importantly moving forward—beyond our past limits...

http://scienceindex.com/stories/3809..._Efficacy.html

thanks for the heads up. not an easy read but imho more gene therapy discussion is needed, not less. kudos to those who volunteered for these trials especially when you have a 50/50 chance of getting sham surgery and from what i gather from this paper not a very good chance of getting a benefit. hopefully wrong about that in current and future trials.
reminds one how strong the placebo affect is in open label studies.