Leukemia drug gets FDA approval due, in part, to City of Hope researcher
December 18, 2014 | by Darrin Joy
A new therapy is offering hope to patients with a certain form of acute lymphoblastic leukemia (ALL). The drug recently received approval by the U.S. Food and Drug Administration (FDA), thanks in part to studies conducted by Anthony Stein, M.D., at City of Hope.
The drug Blincyto, also known by its generic name of blinatumomab, is a bispecific T cell engager, or BiTE.
An emerging class of monoclonal antibody drugs, BiTE antibodies have a unique way to activate a patient’s immune system to attack cancer cells. One section of the antibody attaches to cancer cells while the other section activates the patient’s own disease-fighting T cells and redirects them to kill the cancer cells.
Stein, a clinical professor in the Department of Hematology & Hematopoietic Cell Transplantation, oversaw clinical trials of Blincyto at City of Hope for patients with a certain form of ALL that had returned after treatment and was resistant to therapy. “The approval of Blincyto represents a significant milestone in immunotherapy research,” he said. Clinicians now have a new therapy for patients diagnosed with a highly aggressive cancer for which there are limited treatment options.
Results of the multicenter Blincyto study were strong enough to allow the FDA to grant the drug approval five months ahead of schedule.
Now Stein is leading another multicenter clinical trial comparing Blincyto with standard chemotherapy for another form of ALL in patients whose disease has relapsed and is resistant to treatment.
While overall remission rates for ALL are high, about half of patients will experience a relapse of their disease, and current therapies are not good at knocking the cancer back into remission. Stein believes Blincyto could help. He recently reported promising early results at the American Society of Hematology annual meeting in San Francisco.
Of 189 patients treated on the current study, 43 percent achieved remission within the first two treatment cycles. The drug was similarly effective among those patients who previously had a stem cell transplant and those who had not, as well as in elderly patients. Among the patients who never had transplants, more than half went on to undergo transplant using donor stem cells.
Stein is cautious but hopeful that the drug will prove to be an effective option for these ALL patients. “Our early results are very promising but we’ll need longer follow-up to be sure they bear out,” he said.
Results of the trial should be available in late 2017.
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