Clinical trial suggests triple gene therapy is safe

A Phase I/II trial suggests that a new triple gene therapy called ProSavin, which is based on lentiviral vectors, is tolerable and safe. In the trial, 15 refractory individuals aged 48–65 years with advanced Parkinson's disease were given low, medium or high doses of ProSavin, which was injected directly into the dopamine-depleted striatum — the region of the brain that controls movement — to provide a continuous and stable source of the neurotransmitter. Motor symptoms significantly improved after 6 and 12 months, whereas adverse effects of the treatment were mild to moderate. The authors warn about over-interpreting results regarding the efficacy of this approach; however, its safety could provide a proof of principle for future studies.