David H. Perlmutter, MD, executive vice chancellor for medical affairs and the George and Carol Bauer Dean of Washington University School of Medicine in St. Louis, has received the Christopher Hobler Spirit of Hope Award in recognition of his advocacy for neurological research. The award was presented April 25 by the nonprofit organization Hope Happens.
The nonprofit is dedicated to improving the lives of people with neurodegenerative disorders by promoting collaborative, translational research with the potential to fast-track cures. In 2004, Hope Happens teamed up with the School of Medicine to open the Hope Center for Neurological Disorders, a basic science research center dedicated to finding cures for debilitating nervous system diseases such as amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and multiple sclerosis.
Perlmutter, also the Spencer T. and Ann W. Olin Distinguished Professor, has long promoted basic science research. His success in growing financial support for biomedical research has helped advance efforts at the School of Medicine and the Hope Center to find root causes of neurological diseases.
Perlmutter is recognized for his work on alpha-1 antitrypsin deficiency (ATD), a genetic disorder that causes liver damage. His research led to advances in understanding how cells dispose of misfolded proteins that are toxic and cause cellular dysfunction. This process, known as autophagy, turned out to be critical for the functioning of all cells. A decline in autophagy has been linked to degenerative disease in many organs – and is a key feature of neurodegenerative diseases such as Alzheimer’s and ALS.
His work contributed to the development of novel drug strategies that stimulate autophagy for treatment of ATD. One such drug is being evaluated in a clinical trial. Because the decline in autophagy function during aging has been linked to degenerative diseases in many organs, drugs targeting the process represent exciting candidates for prevention of cognitive decline and other degenerative diseases.