David H. Perlmutter

DAVID H. PERLMUTTER, MD, is the Spencer T. and Ann W. Olin Distinguished Professor, Executive Vice Chancellor for Medical Affairs and George and Carol Bauer Endowed Dean of the School of Medicine at Washington University in St. Louis.

Perlmutter is internationally recognized for his research on alpha-1 antitrypsin deficiency (ATD), a genetic disorder in which accumulations of a misfolded protein can cause severe liver damage. His work has led to advances in the understanding of how cells dispose of misfolded proteins that are toxic and cause cellular dysfunction. To evaluate potential treatments for ATD, Perlmutter and colleagues have developed a pipeline of drugs that includes one drug in phase II trials. The goal is to eliminate the need for liver transplantation, the only treatment option for patients with progressive liver disease due to ATD.

Because drugs in this pipeline target and enhance a cellular degradation pathway termed autophagy, which is critical for the functioning of all cells and because the decline in the function of the pathway during aging has been linked to degenerative diseases, these drugs represent exciting candidates for prevention of cognitive decline and other degenerative diseases of aging. Some of the candidate drugs are already FDA-approved for other indications, allowing this work to move into clinical trials much more rapidly, perhaps even within five to seven years. Perlmutter and his collaborators recently discovered that a drug currently used for Type 2 diabetes may slow age-dependent degeneration in the nervous system and in the liver.

Perlmutter was trained at Children’s Hospital of Philadelphia and Boston Children’s Hospital and has been on the faculties of Harvard Medical School, University of Pittsburgh and Washington University. Prior to his current position, he was distinguished professor and chair of the Department of Pediatrics at University of Pittsburgh as well as physician-in-chief and scientific director of the Children’s Hospital of Pittsburgh of the University of Pittsburgh Medical Center.

Perlmutter is a member of the National Academy of Medicine and the American Academy of Arts and Sciences, past president of the Society for Pediatric Research and past member of the Advisory Council of the National Institute of Diabetes and Digestive and Kidney Diseases. He has been honored with numerous awards throughout his career, including the E. Mead Johnson Award for Research in Pediatrics from the American Pediatric Society and the Sass-Kortsak Award for Pediatric Liver Research from the Canadian Liver Association. He has authored more than 200 scientific publications and holds ten U.S. patents or patent applications.