I have worked for many years in the area of translational and clinical investigation focused on the Myelodysplastic Syndrome and leukemia. This has involved drug development for an orphan disease in a multi-institutional national Cooperative Group, The Cancer and Leukemia Group B. I served as Principal Investigator for three successive trials; two phase I/II studies which demonstrated clinical activity of azacitidine in MDS and then a randomized phase III study of azacitidine compared to supportive care demonstrating azacitidine superiority. These three trials led to the approval of azacitidine by the FDA as the first drug approved for this disease. The treatment I helped pioneer, develop and test at Mount Sinai and in the Cancer Cooperative Groups and an International Consortium has changed the face of treatment of the Myelodysplastic Syndrome. It has improved the outcomes for tens of thousands of patients and their families around the globe. Based on our studies, azacitidine has become the standard of care for patients with higher-risk MDS and represents an important treatment option for older patients with Acute Myeloid Leukemia. These studies have established Mount Sinai as a center of expertise for MDS, and we are currently studying new therapeutic approaches to MDS including the development of novel agents. The approach we pioneered demonstrated that epigenetic therapy, one that can reprogram cells, could be clinically effective in MDS and some forms of AML. Cell reprogramming is now being tested in other diseases to modulate gene expression and cellular behavior.

Languages Spoken: English