Stuart A. Kornfeld, MD, the David C. and Betty Farrell Distinguished Professor of Medicine at Washington University School of Medicine in St. Louis, has received the 2022 Roscoe O. Brady Award for Innovation and Accomplishment from the WORLDSymposium (WORLD is an acronym for We’re Organizing Research on Lysosomal Diseases), a research conference dedicated to lysosomal diseases.
The award is named for Roscoe O. Brady, a pioneer in lysosomal research. Kornfeld received the award at the annual WORLDSymposium meeting last month, in San Diego.
Lysosomal diseases are disorders of the lysosome, important compartments in cells responsible for recycling and disposing of cellular waste and helping rid the body of viruses and bacteria. Kornfeld’s research has led to new understanding of these inherited disorders, including Gaucher disease, Pompe disease, Tay Sachs disease and mucolipidosis. Kornfeld’s lab led important work that defined the mannose 6-phosphate pathway, which ensures delivery of important proteins to the lysosomes. Defects in this pathway give rise to the lysosomal diseases mucolipidosis II and III. The uptake of important enzymes into the lysosome is the basis for cross-correction, the mechanism that underpins all lysosomal enzyme replacement therapies.