Rush University Medical Center To Conduct Phase III Clinical Trial Of Creatine For Parkinson's Disease

Parkinson's Disease Trial

Rush University Medical Center will participate in a large-scale national clinical trial to learn if the nutritional supplement creatine can slow the progression of Parkinson's disease (PD).

While creatine is not an approved therapy for Parkinson's disease or any other condition, it is widely thought to improve exercise performance.

The potential benefit of creatine for Parkinson's disease was identified by Parkinson's researchers through a new rapid method for screening potential compounds. The method is futility analysis. Rather than attempting to find out something is effective, the research is designed to see if a potential treatment is ineffective. If it can be shown to be ineffective, it is discarded as a poor drug to study in a larger more expensive study. If it can't be shown to be ineffective, it is considered a good candidate for additional study.

Creatine is marketed as a nutritional supplement. Studies have suggested that it can improve the function of mitochondria, which produce energy inside cells. It also may act as an antioxidant that prevents damage from compounds that are harmful to cells in the brain. In a mouse model of Parkinson's disease, creatine is able to prevent loss of the cells that are typically affected.

The double-blind, placebo-controlled, phase III study is one of the largest Parkinson's disease clinical trials to date. Rush is one of 51 medical centers in the United States and Canada that will be recruiting patients as part of an effort to enroll 1720 people with early-stage Parkinson's disease.

The primary investigator at Rush is neurologist Dr. Kathleen Shannon. "Rush has one of the world's largest group of experts dedicated to movement disorders, and are several Rush studies are zeroing in on preventing the progression of Parkinson's disease in its early stages. Our objective remains to halt or slow down the disease."

Currently there is no treatment that has been shown to slow the progression of Parkinson's disease.

The study will enroll people who have been diagnosed with Parkinson's disease within the past five years and who have been treated for two years or less with levodopa or other drugs that increase the levels of dopamine in the brain. Many of the symptoms of Parkinson's disease result from the loss of dopamine, a neurotransmitter that helps to control movement. Half of the participants will receive creatine and half will receive a placebo. Neither the participants nor their doctors will know which treatment they receive.

The investigators will measure disease progression using standard rating scales that measure quality of life, ability to walk, cognitive function, and the ability to carry out other activities of daily living.

The trial is the first large study in a series of NIH-sponsored clinical trials called NET-PD (NIH Exploratory Trials in Parkinson's Disease). The NIH has organized this large network of sites to allow researchers to work with PD patients over a long period of time, with a goal of finding effective and lasting treatments. NET-PD builds on a developmental research process -- from laboratory research to pilot studies in a select group of patients to the definitive phase III trial of effectiveness in people with PD.

"This study is an important step. We are pleased to have so many sites participating in this study, which may help us move more quickly toward developing a therapy that could change the course of this devastating disease," says Elias A. Zerhouni, M.D., director of the National Institutes of Health (NIH). "The goal is to improve the quality of life for people with Parkinson's for a longer period of time than is possible with existing therapies."

"This study is an example of our commitment to Parkinson's research," said Story C. Landis, PhD, director of the National Institute of Neurological Disorders and Stroke (NINDS), the NIH institute leading the study. "We are trying to explore every possible option for reducing the burden of this disease."

Parkinson's disease is a degenerative disorder of the brain in which patients develop symptoms such as progressive tremor, slowness of movements, and stiffness of muscles. It affects at least one million people in the United States. Although certain drugs, such as levodopa, can reduce the symptoms of Parkinson's disease, there are no proven treatments that can slow the progressive deterioration in function.