If a drug was developed to block a key protein linked to the onset of Huntington's disease, it could have a clear path to the part of the brain most affected by the disease, while not bothering other parts of the brain and body, said distinguished neuroscientist Solomon H. Snyder, MD, to an audience of about 300 students and faculty at the University of Maryland School of Pharmacy.
Snyder said, "even though there is a market out there of only 100,000 patients," his laboratory at the Johns Hopkins University School of Medicine is currently partnering with big pharmaceutical companies to develop such a drug, one to block binding of the Rhes protein to mutant huntingtin protein, a genetically altered protein in Huntington's disease which kills cells in the brain's corpus striatum.
Snyder gave his remarks at the School of Pharmacy's ýannual Ellis S. Grollman Lecture in the Pharmaceutical Sciences, established at the School by his sister, Mrs. Evelyn Grollman Glick, as an endowed memorial. Grollman was a 1926 graduate of the School who practiced as a hospital pharmacist at Johns Hopkins and Sinai Hospitals in Baltimore City and as a community pharmacist in Frederick, Ocean City, Gaithersburg, and Annapolis. He died in 1982.
Natalie D. Eddington, PhD, dean of the School of Pharmacy, said that the annual lecture focuses on distinguished biomedical research with an emphasis on biological systems.
Snyder, the world's most cited biomedical researcher, began the lecture by "walking" the audience, he said, through the laborious and sometimes frustrating pathway to scientific discovery. He explained results of many "great papers in good journals" since 1975 from his and other laboratories that provided insights into the biochemistry of Huntington's. But each paper failed to pinpoint why the disease shrinks the brain's corpus striatum drastically and very selectively. "It was still a big mystery, though we do know that it is caused by a single gene mutation," said Snyder.
Huntington's disease is caused by a mutation in the protein huntingtin (Htt) that is expressed throughout the body and all the brain regions. But the pathology of Huntington's is mostly confined to the corpus striatum, said Snyder.
"It was an honor for the Department of Pharmaceutical Sciences (PSC) and the School of Pharmacy to host Dr. Snyder," said Andrew Coop, PhD, professor and chair of PSC. "The impact of his groundbreaking research in the area of neuroscience cannot be overstated, and has led to major advances in the biomedical sciences directly improving public health."
Snyder, a native of Washington, D.C., trained at the National Institutes of Health with future Nobel Laureate Julius Axelrod. He is the recipient of numerous professional honors, including the Albert Lasker Award for Basic Biomedical Research (1978), the National Medal of Science (2005), the Albany Medical Prize (2007), the Wolf Foundation Prize in Medicine (1983), the Bristol-Myers Squibb Award for Distinguished Achievement in Neuroscience Research (1996), and the Gerard Prize of the Society for Neuroscience (2000). His books include Uses of Marijuana (1971), Madness and the Brain (1974), The Troubled Mind (1976), Biological Aspects of Abnormal Behavior (1980), Drugs and the Brain (1986), and Brainstorming (1989).