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Fox Foundation Funding Parkinson Drug Discovery Research

Ana Maria Cuervo

Ana Maria Cuervo (Yeshiva University)

25 July 2014. Researchers from Albert Einstein School of Medicine at Yeshiva University in New York are testing compounds for drugs that take on underlying causes of Parkinson’s disease. A $165,000 grant from the Michael J. Fox Foundation for Parkinson’s Research is funding the one year project led by Ana Maria Cuervo and Evripidis Gavathiotis, professors of neuroscience and biochemistry respectively at Einstein.

Parkinson’s disease is  a neurodegenerative  disorder resulting from the loss of cells in the brain that produce dopamine, a chemical that helps control movement. The disease is marked by trembling in the hands, arms, and face, stiffness in limbs and trunk, impaired balance and coordination, and slowness in movements, but can be compounded by depression and difficulty in swallowing and speaking. National Parkinson’s Foundation estimates Parkinson’s disease affects some 4 to 6 million people worldwide, including 1 million people in the U.S.

Treatments for Parkinson’s disease today include drugs and deep-brain stimulation designed to relieve symptoms of the disorder resulting from the lack of dopamine. The Einstein researchers, however, are aiming for the underlying causes of the disease. “While current therapies for Parkinson’s help many people manage their symptoms” says Cuervo in a university statement, “we are eager to stop or even reverse the disorder itself.”

The Einstein team plans to build on a study from Cuervo’s lab published last year identifying a chemical process that interferes with the normal cleaning and recycling of waste products in the brain, leading to a build up of  a toxin called alpha-synuclein, which damages neurons or nerve cells in the brain. The study also identified a cell-signaling pathway that enhances the cleaning and recycling processes in neurons, offering a potential target for therapies.

Gavathiotis’s lab focuses on the structure and function of protein interactions that regulate cell death, and the translation of these protein activities into therapies that address medical needs. In another study published last year, Cuervo and Gavathiotis identified potential compounds for blocking the process that interferes with cell cleaning in the brain that are derived from retinoic acid, made naturally in the body from vitamin A.

In the project funded by the Fox Foundation, Cuervo and Gavathiotis plan to test at least 30 similar compounds, to find 1 or 2 leading candidates for boosting the brain’s waste cleaning and recycling processes. The top candidates will be evaluated in lab mice for potency, delivery to the brain, and possible side effects, leading to a drug that can tested in human clinical trials.

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