More Good News From Ethical Biotechnology: Potential Gene Therapy Treatment for Parkinson’s

Why don’t the American media report these stories (as if I didn’t know)? A new experimental gene therapy treatment seems to alleviate symptoms in Parkinson’s disease. From the Telegraph story:

Evidence that a breakthrough has been achieved in gene therapy for serious brain diseases has come with the release of the hard evidence that it works in Parkinson’s disease. Patients were given injections of billions of copies of genetically altered viruses into parts of the brain

The world’s first gene therapy for a brain disease brought about significant improvements in the mobility of Parkinson’s sufferers. American doctors said it could also herald a landmark in the treatment of other neurological disorders, such as Alzheimer’s or epilepsy but there was a lingering doubt that the reports by a dozen patients of improvements of up to 65 per cent in mobility could be anecdotal or due to the placebo effect.

Today, Prof David Eidelberg of the of The Feinstein Institute for Medical Research in Manhasset, New York, and colleagues report in the Proceedings of the National Academy of Sciences evidence that the brain chemistry of the patients has been altered by gene therapy, ending concerns that the evidence that it worked depended too much on what the patients said and not enough on objective measures. “It is the first solid evidence of benefit from gene therapy. It is objective,” Prof Eidelberg told The Daily Telegraph.

The new approach is going into Phase II human trials, meaning the tests will be to determine safety and efficacy:

The researchers are about to start a larger Phase 2 study in Parkinson’s disease this year and a preliminary trial with epilepsy sufferers. The success of this trial lays the foundation for the use of gene therapy against neurological diseases generally, notably Alzheimer’s and epilepsy.

Warning: We have had high hopes for gene therapy before, only to have them crushed. Moreover, if new treatment approaches fail, it is usually in this phase of the clinical trial. But this seems like the real deal. And it is entirely ethical.