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Parkinson's Disease Patients Get Long-Term Benefits From Experimental Gene Therapy

By Cory Renauer – Updated Sep 11, 2020 at 3:28PM

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Three years after treatment with NBIb-1817, Parkinson's disease patients still are still showing improvements.

Collaboration partners, Neurocrine Biosciences (NBIX -0.40%) and Voyager Therapeutics (VYGR 1.09%) announced data from an early stage clinical trial with Parkinson's disease patients and NBIb-1817, an experimental gene therapy. Three years after a single administration, 14 out of 15 patients are still reporting motor function improvements.

At the moment, NBIb-1817 is in the middle of a phase 2 study that was put on hold in April due to the COVID-19 pandemic. If allowed to restart, the phase 2 RESTORE-1 trial will randomize patients to receive NBIb-1817 or a placebo, then measure for a change in "On" time without troublesome dyskinesia. 

In the background, a medical professional looks at a DNA double helix model in the foreground.

Image source: Getty Images.

This potential new treatment option uses a viral vector to deliver a gene encoding an enzyme that helps Parkinson's disease patients convert levodopa into the dopamine they need. 

Administering NBIb-1817 requires magnetic resonance imaging (MRI) to guide a pair of infusions that deliver the therapy directly into the striatum, a structure deep in the center of the brain. Needles through the skull aren't anybody's idea of a good time, but the side effects that come with daily doses of levodopa aren't any fun either.

For patients with severe Parkinson's disease that has a diminishing response to levodopa, the temporary discomfort that comes with NBIb-1817 treatment seems like a trade-off most will be willing to accept. Three years after a single treatment with NBIb-1817, patients were able to reduce their daily levodopa doses from a baseline of 1500.9 milligrams per day to 1061.4 milligrams per day. Despite reducing their levadopa intake, patients receiving three different dosage strengths of NBIb-1817 improved average "On" time by up to 2.23 hours.

Cory Renauer has no position in any of the stocks mentioned. The Motley Fool recommends Neurocrine Biosciences. The Motley Fool has a disclosure policy.

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