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Potential Treatment for Parkinson's Disease

Jeff M. Bronstein, M.D., Ph.D., Aswani Kotagiri, Ph.D., Arthur G. Fitzmaurice, Gal Bitan, Ph.D.
UCLA David Geffen School of Medicine
NIEHS Grants R21ES016446 and P01ES016732


NIEHS grantees report that the compound CLR01 successfully prevented the aggregation of alpha-synuclein in zebrafish without any toxic effects to healthy brain cells. The protein alpha-synuclein is thought to lead to Parkinson’s disease when it aggregates in the brain and kills neurons, and CLR01 might offer a treatment for slowing or stopping the progression of the disease.


Since alpha-synuclein is found throughout the brain, it is challenging to find a drug that targets only the aggregates without destroying the protein’s normal function. The researchers tested a novel molecular tweezer known as CLR01, which has been shown to inhibit the assembly and toxicity of proteins that have some similarities to alpha-synuclein.


They first experimented with CLR01 in cell cultures, finding that it kept alpha-synuclein from forming aggregates, prevented alpha-synuclein toxicity, and broke up existing aggregates. They also used fluorescent proteins to track CLR01’s effect on the aggregations in a transgenic zebrafish model that expressed human alpha-synuclein in neurons and found that the molecular tweezer prevented alpha-synuclein aggregation and neuronal death.


Citation: Prabhudesai S, Sinha S, Attar A, Kotagiri A, Fitzmaurice AG, Lakshmanan R, Ivanova MI, Loo JA, Klärner FG, Schrader T, Stahl M, Bitan G, Bronstein JM. 2012. A novel "molecular tweezer" inhibitor of alpha-synuclein neurotoxicity in vitro and in vivo. Neurotherapeutics 9(2): 464-476.

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