Parkinson's disease a major focus of HSCI program

The first signs are often so subtle that they escape notice or are chalked up to normal aging: a slight trembling of the hands, a weakened voice, difficulty rising from a chair. But over time, the symptoms—tremors, muscle rigidity, slowed movements, and impaired balance—become more pronounced, pointing inexorably toward a diagnosis of Parkinson’s disease.

Named for the early 19th century British physician who first described it, Parkinson’s disease is a chronic, progressive disease that affects at least a half million Americans. Medications and surgical procedures often improve patients’ lives, at least for a while. But there is no cure for Parkinson’s disease, and patients invariably become increasingly disabled.

Parkinson’s disease occurs when a significant percentage of the nerve cells in a region of the brain called the substantia nigra die or become impaired. These nerve cells, or neurons, produce dopamine, a chemical messenger that permits smooth, purposeful movement. Although genetics and environment play a role, precisely what causes dopaminergic (dopamine-producing) neurons to die or stop functioning has not yet been determined.

One of the goals of HSCI’s Nervous System Diseases Program, led by Massachusetts General Hospital neuroscientist Jeffrey D. Macklis, MD, DHST, is to harness the enormous resources of HSCI to focus on the unsolved riddles of Parkinson’s disease and, in the process, find new, effective therapies as quickly as possible. Writing the molecular biography According to Macklis, a high priority of this collaborative effort is to “write the molecular biography” of the dopaminergic neurons responsible for Parkinson’s disease.

“An understanding of the developmental biology of these neurons and the genetic and environmental influences that help determine their fate will yield important insights that could become the cornerstone of new therapies aimed at slowing down or preventing the neuronal death that causes Parkinson’s disease,” says Macklis.

Macklis emphasizes that this basic knowledge could also be applied to advance other active areas of Parkinson’s disease research. These include repair via precursor/stem cell-transplantation therapies, which have shown promise in preclinical and early clinical trials, and the induction of adult neurogenesis (the birth of new neurons) from within the brain itself.

Macklis points out that since many disorders of the nervous system share much of the same biology, Parkinson’s disease research will also be highly relevant to HSCI investigators working in other areas of the Nervous System Diseases Program.

Think tank fosters collaboration

HSCI’s Parkinson’s disease initiative, which is led by Macklis and McLean Hospital’s Ole Isacson, MD, involves dozens of investigators spanning multiple disciplines and disease programs within and outside of HSCI, in collaboration with experts around the world.

In June, with generous support from the Sharon and Leon Golub Fund for Parkinson’s Disease (see below), HSCI’s Nervous System Diseases Program convened a Parkinson’s disease think tank, in which a group of eight leading neuroscientists from the U.S. and abroad gathered in Cambridge with Macklis, Isacson, and new HSCI faculty member, Paola Arlotta, PhD, of Massachusetts General Hospital.

The goal of this intensive two-day meeting was to share current knowledge regarding the development and survival controls over the neurons of interest and state-of-the-art approaches to study them; define the most promising areas of research; and, through collaboration, accelerate the pace of discovery. According to Macklis, the think tank was so fruitful that all participants urged HSCI to convene another in the summer of 2007.

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