Mount Sinai is a leading research center for hematology and medical oncology. The close association between clinicians working in the Derald H. Ruttenberg Treatment Center, many of whom are physician-scientists who also conduct research, and basic scientists keep all research focused on one goal, improving outcomes for patients with cancer and hematological disorders. The division is particularly well known for its leadership in mylodysplastic syndrome and for the development of 5-azacytidine, the first drug approved by the FDA for this disorder.
Sickle cell anemia and hemophilia have been major areas of focus for the division for many years. Several new therapies for these disorders were developed at Mount Sinai. Ongoing studies are focused on understanding the molecular basis of these and other blood diseases.
Mount Sinai researchers were involved in development of the first gene therapy for blood disorders and they continue to be leaders in the development of this treatment modality. Using recombinant adeno-associated virus vectors, new gene therapies, including use of suicide gene therapy, are in both basic and clinical phases of development. A Mount Sinai developed immunotherapy using gene transfer therapy for advanced colorectal and breast cancers is currently in clinical trials. Gene therapy is also being pursued as a new approach to treating pain associated with cancer.
In both clinical and basic research, investigators continue to seek means of improving therapeutics for a variety of disorders including breast, liver, and lung cancers, myelodysplastic syndrome leukemia, myelosuppression, and severe chronic neutropenia.
A new technique for bone marrow transplantation, known as mini-bone marrow transplantation was refined at Mount Sinai. Because this technique does not require intensive preparative conditioning that leads to the destruction of the patient's existing bone marrow, so it makes this life saving treatment available to those with complicating conditions and those too frail to tolerate traditional bone marrow transplantation.
In the laboratory, researchers are studying molecular pathways to understand the steps that are necessary for the transformation of a normal cell to a cancer cell. They are then using this knowledge to identify ways to interfere with the ability of the cancer cell to survive and divide. In addition, researchers are looking at cancer cells to discover what traits allow them to metastasize in the host.