James A. Shayman, M.D., to deliver Distinguished University Innovator lecture

James A. Shayman, M.D., developer of a new class of drugs for treating a group of rare but devastating congenital disorders, will deliver his 2016 Distinguished University Innovator Award lecture, “Why I Study Rare Diseases,” at 4 p.m. May 9 in Forum Hall at Palmer Commons. A reception will follow.

Sponsored by the Office of Research, the award honors faculty who have made important and lasting contributions to society by developing novel ideas and insights through their research and then translating them to practice.

Shayman is the Agnes C. and Frank D. McKay Professor of Internal Medicine and professor of pharmacology at the Medical School. He is being honored for his innovative work that has led to a simpler and more effective approach to treating lysosomal storage diseases, a group of 50 related genetic disorders. In these diseases, cells and body tissues are damaged by fatty substances that build up to toxic levels in lysosomes, structures within cells that process cellular waste.

One such disorder is Gaucher disease, which can lead to life-threatening damage to the spleen and liver. Many patients have been treated with an expensive program of intravenous replacement of the defective enzymes that produce the substance that builds up in the lysosomes. Shayman pursued a new approach, called synthesis inhibition therapy, designed to limit the production of those enzymes in the body.

After years of study, Shayman and his colleagues developed a compound that not only performed the required task, but also could be administered orally.

To bring the drug to the marketplace, Shayman worked with the Office of Technology Transfer to license it to Genzyme Corp. Collaboration with the company led to clinical trials that established the safety and effectiveness of the approach. The U.S. Food and Drug Administration approved the drug in 2014, and it was made available to patients that same year. Further efforts by Shayman and Genzyme are now underway to test the emerging promise of this approach for treating other lysomal storage diseases as well as other maladies. 

“Jim has shown exceptional dedication, patience, and perseverance as he worked his way along the long path from basic science to the marketplace,” said U-M Vice President for Research S. Jack Hu,, Ph.D. “And his innovations have not only led to a new treatment for Gaucher disease, but also laid the groundwork for further developments that could help millions of patients worldwide.”