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The results were astonishing. All 11 subjects experienced improvement in motor skills and cognitive function. One participant who had previously been confi ned to a wheelchair was able to walk again, and three others regained the ability to speak. Researchers observed that nilotinib penetrated the blood- brain barrier better than other medications designed to treat the decline in dopamine production that occurs in patients with Lewy body complications. Some patients were able to reduce or completely stop the use of dopamine-sparing medications. Participants with earlier stages of the disease responded best to treatment. "To my knowledge, this study represents the fi rst time a therapy appears to reverse — to a greater or lesser degree depending on stage of disease — cognitive and motor decline in patients with these neurodegenerative disorders," Dr. Pagan said in a GUMC news release. "But it is critical to conduct larger and more comprehensive studies before determining the drug's true impact." UNDERSTANDING THE LIMITATIONS Although the initial fi ndings are exciting, questions remain about the long-term safety and effi cacy of using nilotinib to treat Parkinson's disease. Study limitations included a small patient pool and no control group. "There is controversy about what the mechanism is of this medication," says James Beck, PhD, Vice President of Scientifi c Aff airs for the Parkinson's Disease Foundation. "Another issue is regarding the dose. The Georgetown group used 25 percent of the dose used for leukemia. That's because the drug can cause sudden death. There are other inhibitors of alpha-synuclein [the protein that makes up Lewy bodies] that may be safer." Determining the safety of nilotinib to treat patients with Parkinson's disease was the primary goal of the GUMC group's initial trial. The researchers noted that the regimen was well- tolerated by participants, even though the medication in its use to treat leukemia is linked to a wide variety of complications, several of which aff ect more than 10 percent of patients treated for leukemia using this medication. Beck points to the promising fi eld of immunotherapy as a potentially safer approach to halting the progression of Lewy body development. While Parkinson's disease is not infectious, the abnormal cell development responsible for the disease can spread from cell to cell, aff ecting additional portions of the brain and speeding the progress of the disease. Immunotherapy seeks to combat that process using antibodies. A vaccine and passive therapies are currently being researched. In the wake of the GUMC research, the Parkinson's Disease Foundation released a statement recommending against the use of nilotinib in a clinical setting at this time while endorsing additional research into the drug's safety and benefi ts. WHAT'S AHEAD Participants in the GUMC study regressed when they stopped taking nilotinib. Once the initial trial closed, patients were able to continue the use of the medication as part of an expanded access trial. A larger clinical trial is slated to begin in 2016. Researchers are optimistic about the drug's potential to help the 7 million to 10 million people living with Parkinson's disease worldwide. "A lot of institutions talk about expediting the translation of research from the lab to the bedside, but it doesn't happen quickly very often," Dr. Moussa says. "This is a solid example of how that is possible and why it is so important." n In Development According to a recent report by the advocacy organization Pharmaceutical Research and Manufacturers of America, 37 new medications designed to benefi t people with Parkinson's disease are in development and testing. SPECIAL CLINICAL SECTION: GERIATRICS M D N E W S . C O M /// M D N E W S L O N G I S L A N D ■ 2 016 2 3

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