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Gene Therapy Results Promising

A new gene therapy for Parkinson’s disease has achieved promising results in its first human tests, involving 15 patients.

Professor Nicholas Mazarakis, Head of Gene Therapy at the Division of Brain Sciences, Imperial College London, devised the approach while working at biopharmaceutical company Oxford BioMedica in 1997. Sixteen years later, the results of the first tests in humans have been published in The Lancet.

The treatment, called ProSavin, uses a modified virus to deliver three genes into the striatum, a part of the brain that controls movement. The genes are intended to boost the production of dopamine, a chemical that becomes deficient in patients with Parkinson’s.

Findings
15 patients received ProSavin and were followed up (three at low dose, six mid dose, six high dose). During the first 12 months of follow-up, 54 drug-related adverse events were reported (51 mild, three moderate). Most common were increased on-medication dyskinesias (20 events, 11 patients) and on—off phenomena (12 events, nine patients). No serious adverse events related to the study drug or surgical procedure were reported. A significant improvement in mean UPDRS part III motor scores off medication was recorded in all patients at 6 months (mean score 38 [SD 9] vs 26 [8], n=15, p=0·0001) and 12 months (38 vs 27 [8]; n=15, p=0·0001) compared with baseline.

Interpretation
ProSavin was safe and well tolerated in patients with advanced Parkinson’s disease. Improvement in motor behaviour was observed in all patients

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