NEWYORK, NY — The Michael J. Fox Foundation announced a $5.6-million award to drive a Phase 2 clinical trial to investigate the potential of inosine — a naturally occurring chemical that gives rise to urate in the body — to slow or stop the progression of Parkinson’s disease.
The work is being funded under the Foundation’s LEAPS (Linked Efforts to Accelerate Parkinson’s Solutions) 2007 initiative. LEAPS 2007 was funded with a lead gift from the Edmond J. Safra Foundation, one of the most steadfast supporters of The Michael J. Fox Foundation since its inception.
Urate is a natural metabolite and major antioxidant in humans. Previous studies found that healthy people with higher urate levels in the blood had a reduced risk of developing PD. More recent work, including a study published today in the journal Archives of Neurology authored by two of the principal investigators on this LEAPS award, have linked higher urate levels to a possible slower progression of the disease.
The goals of this LEAPS award are to determine the safety of using inosine to raise urate levels, and to assess optimal dosage for therapeutic effect. Ninety people recently diagnosed with Parkinson’s disease will be enrolled in a randomized, double-blind clinical trial to determine whether and at what dose inosine can safely elevate levels of urate in cerebrospinal fluid. Three months after enrollment, cerebrospinal fluid will be tested for urate levels. If a tolerable dose of inosine adequately increases urate in the cerebrospinal fluid, subjects will continue on treatment for up to two years to assess long-term safety.
“This project is precisely the sort of work that The Michael J. Fox Foundation exists to identify and drive forward for patients’ benefit,” said Katie Hood, the Foundation’s CEO. “The study is based on interesting epidemiological observations, but because inosine is a publicly available compound, no corporate entity has the financial incentive to fund clinical research to definitively assess its potential. We believe it is our obligation to the PD community to step in where other funders may be unwilling to go, and ensure that innovative approaches like this one do not stall for lack of resources.”
Inosine is widely available to consumers in dietary supplement form. The researchers emphasize that people with Parkinson’s should not take inosine except in the context of a closely monitored clinical trial in which potential benefits and risks are carefully balanced. The evidence to date surrounding inosine and PD does not prove a cause-effect relationship. Additionally, elevated urate levels are known to carry certain health risks, only some of which have been definitively characterized to date. Kidney stones and gout are known risks; cardiovascular disease is a possible risk. (In the clinical trial, safety measures will be in place to help avoid these conditions, and to detect and treat them should they arise.)
LEAPS awards are multi-year, multi-million-dollar awards to teams of key experts who focus on answering major questions that can improve the diagnosis and treatment of Parkinson’s disease. This third and final 2007 LEAPS award is the Foundation’s single largest award to date. It brings the total funding under the 2007 LEAPS program to $13.8 million.
The principal investigators of this LEAPS award are:
- Coordinating principal investigator Michael A. Schwarzschild, MD, PhD, of the MassGeneral Institute for Neurodegenerative Disease, MassachusettsGeneralHospital, will oversee all elements of the trial.
- Co-principal investigator Alberto Ascherio, MD, DrPH, of the Harvard School of Public Health, will assist in all aspects of trial design and data analysis and head the main biostatistician team.
- Co-principal investigator Karl Kieburtz, MD, MPH, of the University of Rochester School of Medicine and Dentistry, heads the ClinicalTrialCoordinatingCenter there and is chair of the Parkinson Study Group, a network of top researchers conducting clinical research in PD.