Parkinson's Disease

Sanford-Burnham research on Parkinson's disease

Researchers believe that Parkinson’s disease may be more amenable to stem cell therapies than many neurodegenerative conditions because the basic defect is defined. A specific group of brain cells—those in the substantia nigra region that produce dopamine—dies. Sanford-Burnham Professors Stuart Lipton and Evan Snyder are collaborating on a study designed to replace these particular cells in an animal model.

Dopamine regulates body movements, and when levels drop due to cell death, motor irregularities result—the tremors and shakes that are the hallmarks of Parkinson’s. Patients can be treated in the early stages with supplementary levodopa (L-DOPA), which helps to replace dopamine. The treatment loses efficacy over time, however. The Snyder laboratory has transferred human neural stem cells into animals with a model of Parkinson’s that most closely resembles the human disease. The cells appear to survive and produce dopamine. Perhaps even more importantly, the cells protect the animal’s own dopamine cells from degenerating, restoring their normal cellular balance and preserving the animal's own circuits. Animals receiving the stem cells display reduced symptoms. In an effort to understand more about how the stem cell therapy works—and how it might be improved—researchers in the Lipton laboratory are using electrophysiological techniques to examine how these cells might integrate into their new environment and communicate with their neighbors. They are also working with the Snyder laboratory to understand which molecules are being made by the stem cells that mediate its powerful rescue effect.

Other investigators in the Del E. Webb Center for Neuroscience, Aging, and Stem Cell Research are examining how brain cells die, in the hope they might identify ways to head off death in Parkinson’s patients.

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