Roscoe Brady
Dr. Brady received his M.D. from Harvard Medical School in 1947. He joined the NIH where he and his colleagues identified the enzymatic defects in Gaucher disease, Neimann-Pick disease, Fabry disease and others. His team developed diagnostic tests and effective enzyme replacement therapy for patients with Gaucher disease and Fabry disease. He is considered a leader and pioneer in enzyme replacement therapy. He is currently exploring molecular chaperone and gene therapy for Fabry disease and Gaucher disease and carrying out basic research and clinical investigations in additional hereditary disorders.
Interviews:
History
Stated work at NIH with glucocerebroside.
Found glucocerebroside normal in Gaucher patients-- so we looked at the breakdown.
Early test to diagnose Gaucher patients.
Treatment premise: Can you put enzyme in patients and help them?
Working with enzyme-- first patients.
Learning to change sugar configuration, need for dose response curve, success.
Gene isolated by two teams.
Satisfaction with NIH
First real success.
First patients-- lucky we got a response.
If I hadn't been at NIH.
My first Gaucher experiment-- a galactose tolerance test.
What was the single most important realization?
Basic research-- we put enzyme in people--no animal trials.
The worst day-- before we learned about sugar targeting.
Nature of Gaucher
The 5 signs of Gaucher disease.
Current test Gaucher disease.
Other tests for Gaucher disease.
Chaperone molecule-- how it works.
Treating bone involvement.
