Dr. Roboz Leads New Trials for MDS: First-Time Use of AQD Drug for MDS in a Clinical Trial


Dr. Gail Roboz

Dr. Gail Roboz, Associate Professor of Medicine and Director of the Leukemia Program at NYP/WC, has been named lead investigator for a Sunesis Pharmaceuticals-funded clinical trial for the treatment of myelodysplastic syndrome (MDS). A Phase I/II (open-label, dose escalating) trial, it is set to enroll approximately 40 patients with MDS who previously experienced failed treatment using hypomethylating agent-based therapy. Using an AQD (anti-cancer quinolone derivative) drug (Vosaroxin) for the first time to treat MDS in a clinical trial, the study will seek to establish the maximum tolerated dose and then reach research endpoints that include rate of complete remission, partial remission, hematologic improvement, and blood transfusion requirements.

MDS patients often develop anemia requiring frequent blood transfusions, and the disease may worsen developing into progressive bone marrow failure. In about one third of patients with MDS, the disease leads to AML (acute myeloid leukemia). There are few proven therapies for MDS, which is seen mostly in the elderly population (those older than 60 years) at an 80-90% rate. The American Cancer Society estimates that 12,000 new cases are diagnosed each year in the U.S.