The Medical Letter on Drugs and Therapeutics
Tisagenlecleucel (Kymriah) for ALL
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The FDA has approved tisagenlecleucel (Kymriah — Novartis), an individualized, genetically-modified cellular product, for treatment of relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) in patients ≤25 years old. The patient's own T cells are genetically modified to express chimeric antigen receptors (CAR) and then reinfused. Kymriah is the first CAR T-cell immunotherapy to become available in the US. A CAR T-cell product for B-cell non-Hodgkin's lymphoma is expected to be approved soon.

TREATMENT OF ALL — ALL is the most common cancer in children. About 80% of children respond to first-line chemotherapy, but if leukemia recurs, it is less responsive to subsequent interventions. For non-responders or patients who respond initially and then relapse, allogeneic hematopoietic cell transplantation is often required to achieve remission or cure. ... more      

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Title: Tisagenlecleucel (Kymriah) for ALL
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