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News in Brief
Results from Phase I Gene Therapy Study Released
The first gene therapy trial for Parkinson’s was recently reported in the June 23 issue of The Lancet. Researchers at New York-Presbyterian Hospital – Weill Cornell Medical Center described the results of a long-awaited “safety and tolerability” study involving the insertion of a human gene into a deep brain structure in 12 patients with moderately advanced Parkinson’s disease.
The gene therapy method employed a virus that was genetically engineered to carry billions of copies of the human gene for glutamic acid decarboxylase, or GAD. The GAD enzyme breaks down a brain chemical called glutamic acid, which is involved in an important brain circuit in Parkinson’s patients. The gene-carrying virus was injected into one side of the subthalamic nucleus (an area that is overactive in Parkinson’s). The goal of the gene therapy injection was to reduce the activity of the subthalamic nucleus, and improve the symptoms of Parkinson’s.
Gene therapy is potentially risky, and previous efforts to inject gene-carrying viruses have led to serious consequences, including death. In this study, there were no complications or significant adverse effects after one year of follow-up.
The study was an unblinded open-label trial, in which all 12 patients received the gene therapy treatment. The rating scales were done by personnel who were aware of the treatment, and so a placebo effect could not be excluded. Given these limitations, the investigators reported improvements in patients’ motor skills after surgery, mainly on the side of the body opposite the gene therapy injection. The patients did not report statistically significant improvements in their ability to accomplish tasks of daily living, but there was a trend towards improvement.
The investigators emphasized that the purpose of their study was to establish safety, and not demonstrate effectiveness — though modest improvements were found. Nonetheless, the demonstrated safety of the technique in this small study opens the door for future innovations in the treatment of Parkinson’s.
Neupro® Skin Patch Approved for Treatment of early Parkinson’s
On May 9, the US Food and Drug Administration (FDA) announced the approval of Neupro®, a silicone patch that delivers rotigotine, a dopamine agonist (in the same family of drugs as Mirapex® and Requip®), through the skin as a treatment for early-stage Parkinson’s disease.
Neupro, manufactured by Schwarz Pharma, comes in strengths of 2 mg, 4 mg or 6 mg over 24 hours. A single patch is applied to the skin, and the medication releases into the body in a steady manner. The transdermal approach may be helpful for those patients who have difficulty taking oral medication or experience food interference problems.
Common side effects of Neupro use include skin reactions at the site of the patch, as well as dizziness, nausea, vomiting, drowsiness and insomnia. Less common side effects include sudden onset of sleep during daily activities, hallucinations and postural hypotension.
The FDA approval was based on several studies with patients with early Parkinson’s who were not taking other drugs. European studies have also tested the patch in patients with advanced Parkinson’s disease who are taking levodopa and experiencing wearing-off spells.