Gene Therapy Improves Symptoms of Parkinson’s Disease
A new study published in The Lancet Neurology reports gene therapy for Parkinson’s disease (PD) has shown significant improvement in PD symptoms.
Genes are the instructions the body uses to make proteins, and proteins enable the body to carry out all of life’s daily functions. Illnesses are sometimes caused when a gene is defective and causes the body to make a protein that doesn’t work. Gene therapy is a technique used to correct the malfunctioning gene that causes disease and allows the body to make a working form of the protein.
In the phase II clinical trial conducted by Andrew Feign, MD and colleagues at the Feinstein Institute for Medical Research (Manhasset, NY), researchers injected the gene for the protein glutamic acid decarboxylase (GAD) into the brains of Parkinson’s disease patients.
The body uses GAD to synthesizes gamma-aminobutyric acid (GABA), a chemical that reduces how sensitive muscle cells are to stimulation from the brain. GABA levels in the brain are low in PD patients compared to healthy individuals. Scientists believe that this may explain the tremors typically seen with Parkinson’s disease. Without GABA, muscle cells are really sensitive to stimulation from the brain and will fire and twitch at the slightest signal.
The scientists believed that injecting the GAD gene directly into the brains of PD patients would cause them to produce more GABA and would result in improvements in Parkinson’s symptoms. Of 37 patients completing the study, 16 received surgery for gene therapy and 23 recieved surgery without injections of the gene GABA.
Before the surgery, doctors measured each patient’s physical movement using a scoring system designed to evaluate Parkinson’s disease patients. The formula measures problems such as freezing up, tremors and uncontrolled movements. Six months after the surgery, doctors scored the patients again. Half of patients receiving gene therapy achieved dramatic symptom improvements, compared with just 14 percent in the control group.
Overall, the patients who received gene therapy showed an average improvement of 23.1 percent in their scores, compared with a 12.7 percent improvement among the group that did not receive gene therapy.
Currently there is no cure for Parkinson’s disease, and current treatments only ease the symptoms. More tests are still needed to determine if gene therapy is safer and more effective than other methods for reducing the symptoms of Parkinson’s disease. If it works, it will be great news to the more than 1 million people in the US that already suffer from Parkinson’s disease, and the more than 50,000 new cases that are identified each year.
If you would like to find out about other clinical trials for Parkinson’s disease, please visit us at http://www.paidclinicaltrials.org/dir/category/by-condition/neurological-disorders/parkinsons-disease/.
Venita Gresham Watson, Ph.D.
Tags: gene therapy disease, latest news parkinson disease, medication for parkinson disease, parkinson disease cures
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