“It is likely that success in identifying a therapeutic intervention, as well as a cure, will only come when the ability to suppress ALS-associated degeneration is evaluated in the context of the whole organism, with an intact nervous system and motor circuit,” Kristi Wharton said.
Kristi Wharton, professor of biology at the Brown University Institute for Brain Science in Providence, R.I., was awarded an MDA research grant totaling $103,750 over six months. In this new academic-industry partnership, Wharton is working to identify novel drug targets for ALS (amyotrophic lateral sclerosis) that might quickly be developed using the resources available at Pfizer.
Wharton and colleagues are using fruit fly and worm models of ALS to perform chemical screens for factors that alleviate motor neuron degeneration. The team aims to optimize screening design and establish feasibility for screens using the Pfizer chemogenomics library.
The proposed Brown/Pfizer collaboration takes advantage of each team’s respective strengths and expertise, Wharton said. Brown researchers contribute their experience in generating and evaluating human disease models and screening, while Pfizer researchers contribute their deep knowledge of treatment strategies, their proprietary chemogenomics library of a diverse set of compounds with known physicochemical properties, their screening expertise, and an array of molecular reagents.
If successful, this work could inform the development of therapies targeted to slow or stop neurodegeneration in ALS and, possibly, other neurodegenerative diseases.
Muscular Dystrophy Association National Office
161 N. Clark, Suite 3550
Chicago, Illinois 60601
800-572-1717 | ResourceCenter@mdausa.org
The Muscular Dystrophy Association (MDA) is a qualified 501(c)(3) tax-exempt organization.
©2019, Muscular Dystrophy Association Inc. All rights reserved.