Hunting down new clues to the causes of brain degeneration is leading Mass General neuroscientists to exciting new possible therapies. Until recently, no one suspected that the immune system had any role in amyotrophic lateral sclerosis (ALS), a debilitating disease that causes progressive death of brain cells controlling movement. Nor that a natural antioxidant in the body called urate might help those with Parkinson’s disease. But today at Mass General, researchers are exploring these approaches to find new treatments for these devastating, incurable neurodegenerative diseases.
Mass General’s Michael Schwarzschild, MD, PhD, and his collaborators have been investigating clues related to urate since its role was first suspected. They discovered that urate helps protect brain cells and that people with higher levels of urate have lower rates of Parkinson’s. People who already have Parkinson’s fare better if they have higher levels of urate, they also found. Dr. Schwarzschild is director of the Molecular Neurobiology Lab at MassGeneral Institute for Neurodegenerative Disease and head of the Parkinson’s Study Group, a national research group that conducts multicenter clinical trials.
“Not only are we excited about urate as a potential therapeutic target for Parkinson’s, but work in our department shows that it might also be a target for other neurodegenerative diseases.”
Those findings led his team to investigate whether urate levels could be boosted by giving inosine, a chemical compound in the body that plays a role in making urate, to patients with Parkinson’s disease. Dr. Schwarzschild’s team has determined that inosine is safe and effective in raising urate levels. The team is now developing a larger clinical trial to find out whether inosine slows progression of the disease.
Full Court Press for Treatment
“Not only are we excited about urate as a potential therapeutic target for Parkinson’s, but work in our department shows that it might also be a target for other neurodegenerative diseases, particularly Alzheimer’s disease and ALS,” Dr. Schwarzschild explains.
Mass General ALS researchers are pursuing a full-court press for treatments, including the use of gene therapy, monoclonal antibodies and stem cells. “And now with better tools to investigate the immune system, we understand that the immune system is driving the disease progression of ALS, at least in some patients,” says James Berry, MD, MPH, unit chief of the ALS Clinic and associate medical director of the MGH Neurological Research Institute.
He and his colleagues are experimenting with ways to target immune system involvement, including a drug called Gilenya, already approved for multiple sclerosis. Gilenya blocks key immune cells from entering the brain and spinal cord.
Dr. Berry also points out that coordinated, multidisciplinary treatment itself—like what Mass General provides—improves quality of life and survival. Research trials can be an important part of patient care, he adds, “It makes us a team, driven by hope.”
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