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Duke professor wins $75,000+ grant to study molecules for treating neurodegenerative disease

NEW YORK — Kenneth Matthew Scaglione, assistant professor at Duke University’s Department of Molecular Genetics and Microbiology at the Center for Neurodegeneration and Neurotherapeutics, has won a grant totaling more than $75,000 from CurePSP, the foundation for prime of life neurodegeneration.

He is among four researchers to be awarded venture grants totaling $300,000 for research in progressive supranuclear palsy (PSP) and the related disease, corticobasal degeneration (CBD). The studies will investigate the mechanisms of toxic tau protein aggregation in the brain.

Tau is a normal brain protein that, when folded on itself in an abnormal way, forms clumps called neurofibrillary tangles that are toxic to some types of brain cells. In the cases of PSP and CBD, the brain cells involved are important in the control of movement, behavior, and thinking. Unlike many other disorders of tau aggregation, PSP and CBD are pure tauopathies, which means that no other proteins are clumping along with tau. This makes these disorders good subjects for studying the pathology involved in many other and often more common neurodegenerative conditions, including Alzheimer’s disease, which afflicts some six million people in the U.S. alone.

Scaglione is researching the small-molecule regulation of a protein quality-control E3 to treat progressive supranuclear palsy (PSP).

The protein Hsc70, or CHIP, accelerates the removal of tau from the brain. This project intends to identify compounds that stimulate CHIP functions. One important such function is as an E3 enzyme, which is an important part of one of the brain cells’ “garbage disposal” mechanisms called the ubiquitin-proteasome system (UPS). E3 allows the UPS to recognize specific proteins for appropriate disposal. Finding new compounds to stimulate this function is an important first step toward developing small (that is, orally dosable) molecules to slow or prevent the progression of PSP and CBD.

CurePSP’s Venture Grants program provides seed funding for early-career investigators who want to test their innovative ideas. Grant applications are reviewed by CurePSP’s eminent international Scientific Advisory Board (SAB) chaired by Dr. Golbe. CurePSP’s Venture Grants program is one of the few sources of funding for early stage research into PSP and CBD.

CurePSP is the nonprofit organization for prime of life neurodegenerative diseases, a spectrum of fatal brain disorders that often strike during a person’s most productive and rewarding years. Since it was founded in 1990, CurePSP has funded more than 180 research studies and is a leading source of support and advocacy for patients, families, and other caregivers and education and information for doctors and allied healthcare professionals.

Source: WRAL TechWire