Does New Drug Treat Parkinson’s Disease?

GM1 ganglioside clinical trial shows a reduction in Parkinson symptoms and progression

/ Author:  / Reviewed by: Joseph V. Madia, MD

(RxWiki News) Current available drugs for Parkinson’s disease (PD) treat symptoms and improve function but do not slow or halt the progression of the disease. A new drug recently tested in a clinical trial may be changing that.

A recent randomized control trial of an experimental drug called GM1 ganglioside for Parkinson’s disease (PD) has ended after two and a half years.

This clinical trial found that the new drug improved symptoms and slowed the progression of the disease.

"Talk to your doctor about new medications."

Jay S. Schneider, PhD, of the Department of Neurology at Thomas Jefferson University and colleagues studied 77 PD patients between the ages of 39 and 85 years.

The participants were randomly assigned to receive placebo for 24 weeks followed by GM1 for 96 weeks (delayed-start group) or GM1 for the entire 120 weeks (early-start group). Seventeen additional PD patients receiving standard care and usual PD medication were followed for up to 96 weeks for comparison.

The Unified Parkinson's Disease Rating Scale (UPDRS) was used to measure the progression of PD. Measurements for the UPDRS were taken twice a day during each evaluation. Evaluations took place at baseline, six, 12, 18, 24, 36, 48, 72, 96 and 120 weeks. Follow-up examinations took place at one and two years after GM1 was stopped.

The trial showed significant improvement in UPDRS motor scores in the early start group. Meanwhile, the delayed-start group experienced worsening of scores.

Both the early-start and delayed-start groups showed improvement in their symptoms after beginning GM1 treatment and had slower symptom progression over the following two years that the group receiving standard care.

After GM1 use ended, PD symptoms worsened.

PD symptoms are different for every individual. They usually include tremors, slowed movement, rigid muscles, difficulty walking, balance problems and impaired speech.

Symptoms are typically mild early on in the course of the disease and progress with time. Most medications used to treat PD increase or substitute dopamine in the brain to control the symptoms and improve function.

PD patients have a shortage of dopamine-producing neurons in the brain. This creates a shortage of dopamine and causes the motor symptoms PD patients experience.

GM1 is thought to work by protecting the neurons that produce dopamine from dying or losing function.

The use of GM1 has been in research since the 1980s. It has undergone several animal testings and multiple human studies. Future research of GM1 could include a larger clinical study.

This trial was funded by the National Institutes of Health (NIH) and was published in the Journal of the Neurological Sciences. Some authors report receiving funding from the NIH, connections with five different pharmaceutical companies and one author is the founder of a company that develops therapeutics for patients at different stages of Parkinson’s disease, as well as the owner of ganglioside-related patents.

Reviewed by: 
Review Date: 
December 12, 2012
Last Updated:
February 11, 2013