FDA Approves New Immunotherapy for Leukemia

Last week, the FDA approved a new immunotherapy called Blincyto (blinatumomab) for use in the treatment of B cell acute lymphoblastic leukemia (ALL). The drug, manufactured by Amgen, is the first of a novel class of agents known as bispecific T cell engagers (BiTE), which consist of essentially two monoclonal antibodies joined together. One end of the BiTE binds to a molecule on T cells, and the other end binds to a molecule on cancer cells; by bringing the two together, the BiTE facilitates cancer cell killing.

Blincyto (blinatumomab) is designed to treat cancers expressing a molecule called CD19—found on the surface of B cell ALL and also non-Hodgkin’s lymphoma. The FDA approval was based on a phase II clinical trial showing that, of the 185 patients evaluated, 41.6% achieved complete remission with Blincyto.

Blinctyo was a long time in the making and depended on the work of many scientists, a number of whom were funded by the Cancer Research Institute. Gert Riethmüller, M.D., a Scientific Advisory Council member and a member of the Academy of Cancer Immunology, conducted key research that led to a patent for blinatumomab. He was also involved in the clinical testing of the drug, as was Max S. Topp, M.D., a former CRI postdoctoral fellow and now a professor at Würzburg University Hospital in Germany. Topp was the first author on the phase I publication and the phase II American Society of Clinical Oncology abstract that showed that blinatumomab has anti-leukemia activity in this difficult-to-treat population.

BiTEs like Blincyto are the latest weapon in the immunotherapeutic arsenal against cancer. Along with checkpoint blockade antibodies, vaccines, and other immunotherapies, BiTEs are helping to turn our immune system into a powerful defense against this disease. One day—and one not too far away—immunotherapies will conquer cancer. Until that day comes, the Cancer Research Institute will continue to lead the way forward.