Gene therapy breaking ground in treating Parkinson’s disease

Parkinson’s patients who take the drug levodopa, or L-Dopa, are inevitably disappointed…[when,] over time[,] the drug becomes less effective…

A biotech company called Voyager Therapeutics now thinks it can extend the effects of L-Dopa by using a surprising approach: gene therapy. The company…is testing the idea in Parkinson’s patients who’ve agreed to undergo brain surgery and an injection of new DNA.

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Voyager’s strategy…is to inject viruses carrying the gene for AADC into the brain, an approach it thinks can “turn back the clock” so that L-Dopa starts working again in advanced Parkinson’s patients as it did in their honeymoon periods.

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[There is] one edge [that] Voyager’s approach has over others. It is possible to tag AADC with a marker chemical, so doctors can actually see it working inside patients’ brains…[In] past studies of gene therapy, by contrast, doctors had to wait until patients died to find out whether the treatment had been delivered correctly.

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“I believe that previous failure of gene-therapy trials in Parkinson’s was due to suboptimal delivery,” says [Krystof] Bankiewicz.

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion and analysis. Read full, original post: Manufacturing Dopamine in the Brain with Gene Therapy