Revolutionizing Treatment for Hairy Cell Leukemia Patients
October 6, 2010 Leave a comment
Hairy cell leukemia is a rare malignancy characterized by spleen enlargement and progressive anemia and thrombocytopenia (low platelets). Bone marrow aspirations characteristically reveal a “packed marrow.” In the past, patients were often managed with splenectomy and oral chlorambucil. Response rates were low and complications, including infection and bleeding, often ensued.
The introduction of alpha interferon into the management of this disease by Gutterman and associates at M.D. Anderson, provided a meaningful advance during the 1980s. It was at this time that I was conducting a fellowship at the Scripps Clinic in La Jolla, Calif. I had the good fortune to work with Dennis Carson, MD, and his associate Bruce Wasson. They reasoned that the accumulation of deoxyadenosine, which occurred in children with Severe Combined Immunodeficiency (SCID) and was associated with the virtual annihilation of functional lymphocytes in affected patients, could be mimicked pharmacologically. By synthesizing a 2-chloro derivative of deoxyadenosine, they created 2-CDA.
Reasoning that the drug would be T-cell specific, they began early clinical trials in T-cell lymphoma patients. Dr. Carson kindly provided me with a small aliquot of 2-CDA for study in my laboratory. The activity observed in CLL, ALL and even AML, has since been confirmed. However, what was most interesting was the activity observed in the cells removed from the spleen of a hairy cell leukemia patient. The favorable dose response curve suggested to me that 2-CDA would be an active drug in this otherwise refractory malignancy. After my departure from Scripps Clinic, a junior fellow tested this response clinically, providing curative therapy in more than 90 percent of hairy cell leukemia patients. Today, a single cycle of 2-CDA is the treatment of choice for this disease, providing durable benefit in the majority of patients with minimal toxicity.
At Rational Therapeutics we continues to study novel drugs and combinations to examine their effectiveness in treating disease. Our unique ability to test malignant cells in their native state has enabled us to garner some of the most comprehensive results of any group. We will continue to identify new treatments as we move closer to finding cures for patients.