Investment in Parkinson's disease data bank yields potential therapy

October 12, 2009

Individuals with Parkinson's disease who have higher levels of a metabolite called urate in their blood and in cerebrospinal fluid (CSF) have a slower rate of disease progression, according to a study funded by the National Institutes of Health. A clinical trial is under way to examine the safety and potential benefits of supplemental urate elevation for recently diagnosed Parkinson's patients who have low urate levels.

Investigators demonstrated the link with urate by mining a repository of clinical data and tissue samples collected from Parkinson's patients more than 20 years ago as part of a pioneering study called DATATOP, funded by NIH's National Institute of Neurological Disorders and Stroke (NINDS). The new study appears in Archives of Neurology. It was funded primarily by NINDS, with additional support from the Department of Defense and private organizations.

"This study speaks to the value of saving data and biospecimens from large clinical studies, and making them available to the research community to pursue new, unanticipated ideas," said Michael Schwarzschild, M.D., Ph.D., an associate professor of neurology at Massachusetts General Hospital in Boston, who lead the study together with Alberto Ascherio, M.D., Dr.PH, a professor of epidemiology and nutrition at the Harvard School of Public Health.

Experts emphasize there is no proof that elevating urate levels will help against Parkinson's disease, and that it should not be attempted outside of a clinical trial, where physicians can closely monitor possible benefits and risks, such as gout and heart disease.

Parkinson's disease attacks cells in the brain that regulate movement by releasing a chemical called dopamine. The loss of those cells leads to progressively disabling symptoms, including involuntary shaking, slow movement, stiffened muscle tone, and impaired balance. Levodopa, a precursor of dopamine, provides some relief from those symptoms but does not alter the disease course.

"Effective treatments for Parkinson's disease have been elusive. By taking a fresh look at the repository of clinical data and stored samples from the two-decade old DATATOP trial, this study has identified urate as a biomarker for the progression of the disease and suggests a potential new pathway for targeted therapy development," said Margaret Sutherland, Ph.D., a program director at NINDS.

Urate (or uric acid) is a product of the body's metabolism. Diets high in liver, seafood, and dried beans and peas tend to cause higher levels of urate in the blood, and are also associated with gout - a painful buildup of urate crystals in the joints. Urate is a natural antioxidant, and many studies have found that antioxidants slow the course of Parkinson's disease in animal models. Also, prior research from Dr. Ascherio's epidemiology group has shown that people who have gout or who consume foods associated with high urate have a lower incidence of Parkinson's disease.

Drs. Ascherio and Schwarzschild and their collaborators in the Parkinson Study Group are the first to examine whether urate levels are related to the course of Parkinson's disease. Last year, after mining data from another large clinical trial, they reported that high levels of urate in blood were associated with a slower disease course. The new study is an expansion of that work and the first time that investigators have looked for a connection between the course of Parkinson's and levels of urate in CSF, the fluid that fills spaces in the brain and spinal cord.

The DATATOP trial began in the late 1980s, and was designed to test whether vitamin E, the drug deprenyl (selegiline), or a combination of both could slow the course of early-stage Parkinson's disease. The trial enrolled 800 patients and followed them for two years. Deprenyl, which inhibits the breakdown of dopamine, was found to provide short-term relief from symptoms. Vitamin E showed no significant benefit.

As part of the DATATOP trial, samples of blood and CSF were acquired from more than 90 percent of the participants at enrollment. In the new study, the researchers analyzed whether blood and CSF urate levels were related to the course of Parkinson's by relying on blood measurements done at the time of the DATATOP trial and by taking new measurements from the 20-year-old, frozen samples of CSF.

Looking across all of the treatment groups in the study, the researchers found that patients with the highest urate levels in their blood and CSF had a slower functional decline as measured by their need for levodopa treatment. The results suggest that urate elevation might slow the course of Parkinson's in patients with early-stage disease and low urate levels.
The Safety of URate Elevation in Parkinson Disease (SURE-PD) study, led by Dr. Schwarzschild, is a placebo-controlled trial designed to test that hypothesis. Patients in the treatment arm of the trial will take daily, oral doses of a urate precursor called inosine for up to two years. The trial is funded by the Michael J. Fox Foundation, and is recruiting recently diagnosed patients who do not yet require Parkinson's medication at 11 sites across the United States. For more information, visit and search by the identifier NCT00833690.

Reference: Ascherio A et al. "Urate as a Predictor of the Rate of Clinical Decline in Parkinson Disease." Archives of Neurology, published online October 12, 2009.

NINDS ( is the nation's primary supporter of biomedical research on the brain and nervous system.

The National Institutes of Health (NIH) -- The Nation's Medical Research Agency -- includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. It is the primary federal agency for conducting and supporting basic, clinical and translational medical research, and it investigates the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit

For more information about Parkinson's disease, visit

NIH/National Institute of Neurological Disorders and Stroke

Related Clinical Trial Articles from Brightsurf:

Clinical trial investigates gabapentin for alcohol use disorder
This randomized clinical trial investigated if gabapentin, a drug often used to treat nerve pain, would be useful in the treatment of patients with alcohol use disorder (problem drinking that becomes severe) and a history of alcohol withdrawal symptoms.

Electrical stimulation helps treat constipation in clinical trial
Electrical stimulation benefited women with constipation in a recent clinical trial published in Alimentary Pharmacology & Therapeutics.

Treatment of migraine pain in randomized clinical trial
Adults experiencing a migraine of moderate or severe severity took the drug ubrogepant or placebo and reported if after two hours they were free of pain and of their most bothersome migraine-associated symptom in this randomized clinical trial.

First entirely digital clinical trial encourages physical activity
As little as a daily ping on your phone can boost physical activity, researchers from the Stanford University School of Medicine and their collaborators report in a new study.

HIV vaccine nears clinical trial following new findings
A promising vaccine that clears an HIV-like virus from monkeys is closer to human testing after a new, weakened version of the vaccine has been shown to provide similar protection as its original version.

Sickle cell drug showing promise in clinical trial
An investigational drug for the treatment of sickle cell disease is showing early promise in clinical trials for impacting biomarkers of the disease in patients, reported UConn School of Medicine researchers.

Meditation goes digital in new clinical trial
Scientists at UC San Francisco have developed a personalized digital meditation training program that significantly improved attention and memory in healthy young adults -- a group already at the peak of brain health -- in just six weeks.

Could blockchain ensure integrity of clinical trial data?
UC San Francisco researchers have created a proof-of-concept method for ensuring the integrity of clinical trials data with blockchain.

Eating crickets can be good for your gut, according to new clinical trial
A new clinical trial shows that consuming crickets can help support the growth of beneficial gut bacteria and that eating crickets is not only safe at high doses but may also reduce inflammation in the body.

Idera Pharmaceuticals presents clinical data from the ILLUMINATE-204 trial of the combination of tilsotolimod and ipilimumab for anti-PD-1 refractory metastatic melanoma at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting
Idera Pharmaceuticals Inc., a clinical-stage biopharmaceutical company developing toll-like receptor and RNA therapeutics for patients with rare cancers and rare diseases, announced results from the ongoing ILLUMINATE-204 trial investigating tilsotolimod, Idera's intratumorally-delivered Toll-like Receptor 9 agonist, in combination with ipilimumab (Yervoy®).

Read More: Clinical Trial News and Clinical Trial Current Events is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to