24th  April 2014 - New research

CLINICAL TRIAL OF GENE THERAPY FOR PARKINSON'S DISEASE

Lancet [2014] 383 (9923) : 1138-1146 (S.Palfi, J.M.Gurruchaga, G.S.Ralph, H.Lepetit, S.Lavisse, P.C.Buttery, C. Watts, J.Miskin, M.Kelleher, S.Deeley, et al)  Complete abstract

ProSavin (NLX-P101) uses LentiVector gene delivery technology to deliver genes for three enzymes they suggest are required for the formation of dopamine. The product is administered locally to the relevant region of the brain in order to increase the brain's own capacity for the formation of dopamine. For more information go to Prosavin

In a clinical trial of Prosavin, 15 patients received ProSavin, with three people taking a low dose, six taking a mid dose, and six taking a high dose. During the first 12 months 54 drug-related adverse events were reported (51 mild and 3 moderate). The most reported were increased dyskinesias, and on-off phenomena. No serious adverse events related to the drug or the surgical procedure were reported. A moderate improvement in mean Parkinson's Disease symptom scores was recorded in all patients tested at 6 months and 12 months.

In a previous clinical trial the degree of efficacy was quite moderate, with an average 27% improvement after 3 months, peaking at 31% after 6 months, and declining to 23% after 2 years. In the long term stimulating gene and enzyme levels artificially reduces a person's own formation of those genes and enzymes. For more news go to Parkinson's Disease News.