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Scripps chemist wins national award for neurodegenerative disease research

Chemist Jeffery W. Kelly of Solana Beach, Calif., will be honored August 28 by the worlds largest scientific society for his advances in understanding and developing treatments for rare neurodegenerative diseases. He will receive the 2001 Arthur C. Cope Scholar Award from the American Chemical Society at its national meeting in Chicago.

What we do is design and utilize small molecules to change the course of biology, said Kelly, a chemistry professor and vice president of the Scripps Research Institute in La Jolla, Calif. In particular, we work to prevent neurodegenerative diseases similar to Alzheimers disease but more rare. Disorders that strike fewer than 100,000 people are often called orphan diseases ones too rare for pharmaceutical companies to expect sales to recoup a drugs costs in research, development, and Food and Drug Administration approval.

Thus few if any treatments exist for many of these diseases. But once we make enough progress to get people interested in it, an orphan disease may or may not remain as such, Kelly said. One type of rare disorder his team studies is familial amyloid polyneuropathy (FAP).

At its root is a misshapen version of transthyretin, a blood protein that helps regulate metabolism and nutrition. As in Alzheimers and other amyloid diseases, and for reasons yet unclear, the protein refolds into a new shape that clumps together instead of functioning properly.

What weve done over the years is understand how mutations [in the transthyretin gene] can cause the misshape, he said. Our small molecules can compensate for those mutations. They act as braces to stabilize the normal protein. Several of their molecular braces tailor-made to fit within the proteins three-dimensional structure without blocking its function are now in preclinical trials, he added.


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Contact: Rodney Pearson
r_pearson@acs.org
202-872-4400
American Chemical Society
20-Aug-2001


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