MDNews - Long Island

PRELIMINARY FINDINGS FROM A SMALL-SCALE STUDY BY GEORGE TOWN UNIVERSIT Y MEDICAL CENTER (GUMC) RESE ARCHERS INDICATE THAT A MEDICATION APPROVED TO TRE AT LEUKEMIA MAY HALT OR RE VERSE THE PROGRESS OF PARKINSON'S DISE A SE AND LE W Y BODY DEMENTIA. BY VALERIE LAUER THE S T UDY, W HICH examined the effi cacy of the chemo- therapy medication nilotinib, was the work of the Director of Georgetown's Laboratory of Dementia and Parkinsonism, Charbel Moussa, MD, PhD, and Fernando Pagan, MD, Director of the Movement Disorders Program at MedStar Georgetown University Hospital. "Treating people with neurodegenerative diseases and limited access to drugs or treatments made me feel the need to pursue more translational and lab work — to basically identify new drugs or new compounds that would potentially halt or reverse the neurodegenerative nature of Parkinson's and Alzheimer's and other neurodegenerative diseases," Dr. Moussa said in an interview for GUMC's news outlet. "Our work has been focused on repositioning this already-FDA-approved drug, that we now have a good safety profi le to use, for the treatment of neurodegenerative diseases — to stop or halt cell death in the brain and reverse the progression of the disease." SMALL BUT PROMISING During preliminary experiments, Dr. Moussa and his team found that exposing brain cells in a petri dish to nilotinib eff ectively cleared cellular accumulations of Lewy bodies — protein deposits thought to be responsible for the disruption of normal cell function and dopamine production, and thus for the declining function of those with Parkinson's disease. Two years after this discovery, the phase 1 GUMC study began. The study enrolled 12 participants, 11 of whom completed the trial. Each received either 150mg or 300mg of nilotinib daily, signifi cantly lower doses than those prescribed for the treatment of leukemia — up to 800mg. Yields Hope for Reversing Effects of Parkinson's DRUG REPURPOSING SPECIAL CLINICAL SECTION: GERIATRICS 2 2

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