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takyon writes:

A biotech company will attempt to use gene therapy in order to improve responses to a Parkinson's disease treatment:

Parkinson's patients who take the drug levodopa, or L-Dopa, are inevitably disappointed. At first, during a "honeymoon" period, their symptoms (which include tremors and balance problems) are brought under control. But over time the drug becomes less effective. They may also need ultrahigh doses, and some start spending hours a day in a state of near-frozen paralysis.

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, based in Cambridge, Massachusetts, is testing the idea in Parkinson's patients who've agreed to undergo brain surgery and an injection of new DNA.

[...] The cause of Parkinson's isn't well understood, but the reason the drug wears off is. It's because the brain also starts losing an enzyme known as aromatic L-amino acid decarboxylase, or AADC, that is needed to convert L-Dopa into dopamine. Voyager's strategy, which it has begun trying on patients in a small study, 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.

Reprinted at NextBigFuture.

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