UR Collaborates on Nationwide Study for New Parkinson’s Treatment

Aug. 31, 2015
David Oakes, Ph.D.

In a 60-site clinical study to investigate whether the drug inosine can slow early Parkinson’s disease, the University of Rochester Medical Center was selected as the coordinating center for data collection and will receive an estimated $6 million research award. 

UR Professor David Oakes, Ph.D., in the Department of Biostatistics and Computational Biology, will facilitate data coordination with study chair Michael A. Schwarzschild, M.D., Ph.D., a neurobiologist at Massachusetts General Hospital. The National Institute of Neurological Disorders and Stroke (NINDS) funded the five-year, $26 million project. The UR is receiving an initial $3.5 million with more funding projected in the final years.

Parkinson’s produces body tremors, rigidity, difficulty walking and other symptoms, and is caused by the degeneration of brain cells. Although some treatments can ease symptoms, no therapy has yet been shown to relieve the loss of brain cells or slow progression of the disease.

Previous research has shown that when Parkinson’s patients have higher levels of the antioxidant urate in their blood, symptoms of the disease do not worsen as quickly as in patients who have lower urate levels. Therefore, scientists hypothesized that raising urate levels might protect against Parkinson’s progression. Inosine (a pill), is a supplement that already has been tested in a phase 2 trial, funded by The Michael J. Fox Foundation for Parkinson’s Research. It showed that inosine was safe, tolerable, and raised urate levels in people with the early stage Parkinson’s disease.

The newly funded phase 3 study is double-blinded and placebo-controlled, with the goal of learning whether inosine will boost urate levels enough to delay the progression of Parkinson’s disease over two years. Enrollment is expected to begin in mid-2016.

Schwarzschild’s research team has also discovered that the protective effects of urate in the brain might extend beyond its own antioxidant properties. Investigators reported today in the journal Neurobiology of Disease, that urate also stimulates brain cells called astrocytes to activate a major antioxidant pathway believed to have a role in several neurodegenerative disorders. While other antioxidants such as vitamin E have failed to show a benefit to patients with Parkinson’s, the new evidence of a more nuanced molecular mechanism for urate boosts the researchers’ enthusiasm, Schwarzschild said.

One potential drawback to inosine treatment is gout, an arthritic condition associated with people who have naturally high urate levels. No one with higher than normal levels of urate will be eligible to participate, Oakes said, and careful monitoring will take place during the study to look for other adverse complications. Only a few of the 75 participants in the earlier phase 2 trial developed side effects.

The phase 3 trial will closely track how patients report symptoms and outcomes, and will use brain images to support a diagnosis of Parkinson’s, which can be difficult to establish in early stages, Oakes said.

Co-investigators at the University of Rochester include Christopher Beck, Ph.D., also in the Department of Biostatistics and Computational Biology; Cynthia Casaceli, M.B.A., director of the Clinical Trials Coordination Center; and Cornelia Kamp, M.B.A., of the Clinical Materials Services Unit. The investigation is being conducted under the auspices of the Parkinson Study Group, a non-profit group of health-care professionals dedicated to finding better treatments for Parkinson’s.