According to a story from Parkinson’s News Today, a phase 2 clinical trial that will test a gene therapy for advanced Parkinson’s disease has begun recruiting patients. The experimental gene therapy, which is being co-developed by Voyager Therapeutics and Neurocrine Biosciences, is known as VY-AADC02. Among the 17 recruitment sites is the Hackensack University Medical Center. The study is expected to include 40 patients with Parkinson’s disease that is considered advanced.
About Parkinson’s Disease
Parkinson’s disease is a type of long term, progressive, degenerative illness that affects the central nervous system. Symptoms tend to develop over a period of years and primarily affect the movement ability and mental state of the patient. The cause of Parkinson’s disease remains a mystery, although there are a number of risk factors that have been identified. These factors include head injuries, pesticide exposure, and certain genetic variants and mutations. About 15 percent of patients have a close relative with the disease, suggesting some genetic connection. Symptoms include slowed movements, poor coordination, trouble walking, shaking, stiffness, abnormal posture, depression, anxiety, inhibited thinking, hallucinations, and dementia. Treatment may involve a number of medications, rehabilitation, and surgical operations. Survival rate varies, but most patients survive around a decade after getting diagnosed. To learn more about Parkinson’s disease, click here.
Patients in the study must have been diagnosed at least four years ago and be between the ages of 40 and 75. In addition, participating patients must be dealing with unexpected motor symptoms despite the use of other treatments and must not have plans to try new medications or undergo a deep brain stimulation procedure before the trial begins.
VY-ADDC02 will be administered to patients via a surgical procedure and the study will also include a control group that will undergo a placebo ‘sham’ procedure. The gene therapy will utilize a viral vector in order to deliver a copy of the AADC gene into an area of the brain called the putamen. The gene codes for the AADC enzyme and this region of the brain is closely associated with motor functions. It also contains a significant concentration of dopamine receptors.
An earlier trial using a precursor variant of the therapy showed encouraging results.