Abbey S. Meyers
President
National Organization for Rare Disorders (NORD)
Wednesday, October 9, 2002
10:00 a.m.
I am honored to be at this tribute to Dr. Roscoe Brady. He is truly a legend in the field of biomedical research. When I think about the term "public servant," I don't think about politicians or bureaucrats; I think about Roscoe Brady. He has devoted his career to improving the lives of desperately ill people with little-known diseases that kill and cripple. Because of him, those families have hope, an industry that historically ignored rare diseases is now developing enzyme replacement therapies for the rarest genetic disorders, and young scientists are motivated to follow in his footsteps, knowing they will probably never parallel his accomplishments.
Dr. Brady has spent his 50-year career at NIH. He is responsible for breakthrough discoveries in numerous lysosomal storage diseases. He discovered the metabolic basis of Gaucher's disease, Niemann-Pick disease, Fabry's disease, and the specific biochemical defect in Tay-Sachs disease. He is responsible for enzyme replacement therapies in Gaucher's and Fabry's disease. He didn't just move his discoveries from the laboratory bench to the bedside - he developed genetic counseling procedures for these disorders because he has an extraordinary ethical sensitivity.
Roscoe Brady has set the stage for enzyme replacement therapies for countless genetic diseases. He developed the science that gave birth to an industry because he stubbornly believed in translational research while the rest of the research world ignored it.
Roscoe Brady has won numerous accolades and awards, including the Lasker Foundation's Clinical Medical Research Award, and the Kovalenko Medal from the National Academy of Sciences, to name only two. His laboratory at NINDS is leading the research effort on numerous neurogenetic diseases, such as mucolipidosis type 4, leukodystrophies of unknown causes, and novel peripheral neuropathies. In fact, his team discovered two previously unrecognized syndromes: (1) childhood ataxia with central nervous system hypomyelination, and (2) ovarioleukodystrophy.
A young scientist just entering biomedical research today can only dream of being responsible for one or two equivalent discoveries in their career. Dr. Brady's scientific accomplishments are unparalleled. The National Organization for Rare Disorders (NORD) wishes we could clone him for other groups of understudied orphan diseases; but because we cannot immortalize Dr Brady, we have done the next best thing. With a one-million dollar gift from Transkartyotic Therapies (TKT), and major contributions from Genzyme and BioMarin, we created the Roscoe Brady Research Fellowship program for lysosomal storage disease research. To date, fellowships have been awarded to young scientists from the United States, Canada, Japan, Europe, and Brazil who are studying lysosomal disorders.
It is a privilege to know Roscoe Brady; but it is even more important in the field of rare "orphan diseases" to know the patients with Gaucher's disease and Fabry's disease who are alive today because of Dr. Brady. The fact that his scientific accomplishments led to new treatments (orphan drugs) tells you something about his scientific expertise and determination; but the fact that he also recognized how important it is to properly provide genetic counseling to at-risk families, tells you something about the man. There are approximately 6,000 rare disorders affecting an estimated 25 million Americans, and they all wish they had a Roscoe Brady working on their diseases.
It is with profound gratitude that we join this tribute to an exceptional scientist and a unique human being. The truth is if Roscoe Brady had worked at an academic institution or a company, this research would never have come to fruition. Companies and academia would not have allowed him to pursue these avenues of research for 30, 40, or 50 years. Thank you NIH for sheltering Roscoe Brady because he is the Louis Pasteur of enzyme replacement therapy, and because of him people with genetic diseases can see the light at the end of the tunnel.
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