New safety data from a study of patients with advanced Parkinson's disease five years after gene transfer-mediated delivery of the neuroprotective factor neurturin directly to patients' brains reveal no serious adverse events related to the treatment. The encouraging long-term safety profile of the surgically administered adeno-associated virus (AAV2)-neurturin gene therapy is described in an article in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers (http://www.liebertpub.com/).
In the article "Long-Term Safety of Patients with Parkinson's Disease Receiving rAAV2-Neurturin (CERE-120) Gene Transfer (http://online.liebertpub.com/doi/full/10.1089/hum.2015.134)" William Marks, Jr., University of California, San Francisco, Tiffany Baumann, Isis Pharmaceuticals (Carlsbad, CA), and Raymand Bartus, RTBioconsultants (San Diego, CA), representing the CERE-120 Study Group, describe the study design, which greatly extended the length of follow-up of the 53 patients evaluated compared to previous studies for the purpose of identifying any safety issues that might present months or years after the treatment.
"The longevity of rAAV vector expression makes it particularly useful for treatment of chronic neurodegenerative disorders," says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worcester, MA. "These long-term data lend support to the evidence that rAAV will prove to be an important platform for treating this highly debilitating disease."
Study reveals how DNA folding drives gene activity bursts