Trials to Test Gene Therapy for Parkinson's Disease Underway

Scientists at Michigan hospital have launched a trial to test gene transfer therapy that would help restore better mobility in Parkinson's patients who have lost responsiveness to drug therapy.

When brain neurons in brain begin to die, the cells can no longer manufacture the molecule dopamine, a chemical critical for controlling movement.

Parkinson’s Disease - Causes, Symptoms, Diagnosis, Treatment
Parkinson’s disease is a neurodegenerative disease caused by progressive dopamine brain cells loss. Symptoms of Parkinson’s disease are correctable to an extent.

Most current therapies and research approaches target dopamine to treat motor symptoms associated with Parkinson's disease.

"The start of this clinical trial provides hope to a Parkinson's disease patient population that has had a long-standing need for better treatment options," said Dr Henry Ford West Bloomfield Hospital neurologist Peter LeWitt, who is internationally known for his research on Parkinson's disease.

In the current study, the researchers are using gene therapy strategy to increase GABA, a brain neurotransmitter that regulates movement.

Parkinson's Disease - Surgical Treatment
Slide animation on surgery for Parkinson disease. Surgery is only indicated where drugs fail to control the symptoms of this progressive movement disorder. Surgery cannot cure the disease.

In Parkinson's disease, GABA is reduced in an area of the brain called the subthalamic nucleus, causing it to be overactive.

The researchers feel this might be a better way to help advanced Parkinson's disease.

During the clinical trial, the gene therapy product rAAV-GAD will be placed into the subthalamic nucleus by a surgical procedure.

The gene transfer is done through a catheter that is removed shortly after its placement.

Normalizing Brain Function in Parkinson's Patient by Gene Therapy
A gene therapy experiment that has shown to normalize brain function in patients with Parkinson’s disease.

As an alternative to this experimental treatment, patients with advanced Parkinson's disease have the option of deep brain stimulation.

This also involves a surgical procedure in which a pacemaker-like device is placed in the brain to help in control of Parkinson's disease symptoms.

Source-ANI
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