NEWYORK, NY — Michael J. Fox Foundation-funded researchers at Vanderbilt University Medical Center have reached a milestone in the development of potential new drugs for Parkinson’s disease that may avoid the limitations of current therapy.
The team, which received an MJFF award of up to $4.4 million under the Foundation’s 2007 LEAPS (Linked Efforts to Accelerate Parkinson’s Solutions) initiative, has identified two drug-like molecules that, when given systematically in a pre-clinical model of Parkinson’s disease, reduce PD symptoms by acting on a specific glutamate receptor in the brain called the metabotropic glutamate receptor, or mGluR4.
Jeffrey Conn, PhD, director of the Vanderbilt Drug Discovery Program and leader of the research team, will detail the discovery in a presentation called “Discovery of mGluR4 Potentiators for Symptomatic and Disease‐modifying Treatment of PD” at the third annual Parkinson’s Disease Therapeutics Conference, hosted today in New York City by The Michael J. Fox Foundation and The New York Academy of Sciences.
“If we can develop a molecule that has the right properties and does not have toxicity, we really believe this has an opportunity to have a major impact for Parkinson’s patients,” said Conn. “Nothing could be more exciting than that... Nothing could be more satisfying.”
“Obviously there are no guarantees,” Dr. Conn cautioned. But if progress continues, clinical trials in patients with Parkinson’s disease could begin sometime in 2013, he said.
In previous studies, Conn and others have found that they could relieve symptoms of Parkinson’s disease in pre-clinical models by activating the mGluR4 receptor.
In their latest finding, the Vanderbilt researchers have found two compounds that act on mGluR4 and, when given systemically in a pre-clinical model of Parkinson’s disease, get to the brain and relieve motor symptoms, including rigidity and akinesia or a “freezing” of certain motor muscles. Further pre-clinical testing will be required before tests in humans can begin. But, said Conn, the identification of these molecules is “a big step forward.”
Dr. Conn’s colleagues in this effort include Colleen Niswender, PhD, Corey Hopkins, PhD, Carrie Jones, PhD, Craig Lindsley, PhD, and David Weaver, PhD, each of whom directs critical components of this large multidisciplinary effort.
More information, including a detailed Q&A with Jeff Conn, is available on The Michael J. Fox Foundation’s Web site at www.michaeljfox.org/research.