Chemist Jeffery W. Kelly of Solana Beach, Calif., will be honored August 28 by the world’s 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 Alzheimer’s 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 drug’s 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 Alzheimer’s and other amyloid diseases, and for reasons yet unclear, the protein refolds into a new shape that clumps together instead of functioning properly.
“What we’ve 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 protein’s three-dimensional structure without blocking its function — are now in preclinical trials, he added.
Kelly received his undergraduate degree from the State University of New York College at Fredonia in 1982 and his Ph.D. from the University of North Carolina in 1986. He is a member of the ACS divisions of biological and organic chemistry.
The ACS Board of Directors established the Arthur C. Cope Scholar Award in 1984 to recognize excellence in organic chemistry. Under the MIT chemist’s will, each of 10 such awards consists of a $5,000 prize and $40,000 research grant.