According to a story from Parkinson’s News Today, a recent study found that treatment with an experimental gene therapy called AX0-Lenti-PD was able to improve symptoms for two patients with advanced Parkinson’s disease after just three months. This Phase I/II trial is the first ever to use gene therapy to treat Parkinson’s disease and offers new hope for patients with this progressive illness which is currently incurable and is ultimately fatal.
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.
Gene Therapy for Parkinson’s Disease
These patients received the lowest possible dose of the therapy and still saw significant benefit. At this juncture, enrollment for this clinical trial is continuing in the UK and is expected to include a total of 30 patients that were diagnosed with Parkinson’s disease in the last five years. This patient group will represent ages between 48 and 70.
While certain genetic variants have been linked to an increased risk of Parkinson’s disease, the illness still is not directly caused by a certain genetic mutation. This may make gene therapy seem like an unlikely option for treatment. The mechanism of Parkinson’s disease is linked to a lack of dopamine production in the brain. AX0-Lenti-PD works by delivering three distinct genes that are coded for certain enzymes that are critical to dopamine production. The therapy was delivered via surgery directly into the brain.
Continued research will investigate the capability of this experimental treatment in other patients at different dosage levels. AX0-Lenti-PD could represent an innovative breakthrough in the treatment of Parkinson’s.