Renier J. Brentjens, MD
The use of chimeric antigen receptor (CAR) T-cell therapies for the treatment of hematologic malignancies is still in its early stages, but when the FDA approved tisagenlecleucel (Kymriah) and axicabtagene ciloleucel (axi-cel; Yescarta) in 2017, this gave hope to oncologists and patients with some types of leukemia and lymphoma who have exhausted all other options. The findings and approvals for this approach were so welcome that the American Society of Clinical Oncology (ASCO) named CAR T-cell therapy as the Advance of the Year as part of the Clinical Cancer Advances 2018: ASCO’s Annual Report on Progress Against Cancer.1
The clinical trials have focused on initial clinical outcomes and long-term studies are not yet available. There also may be some questions about the reported responses being for the patients treated, rather than a percentage of the intentto-treat population. So, it is still not known how often these treatments provide complete remission, how long patients can expect to remain in remission, and how the durability of the treatment matches the high cost of treatment.
Recent Findings for CAR Therapies
Tisagenlecleucel was the first FDA-approved CAR T-cell therapy; the agency approved it in August 2017 to treat children and young adults (up to 25 years) with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.2
The therapy was approved based on outcomes among 63 patients who received a single dose of tisagenlecleucel during the phase II single-arm international ELIANA trial. Among the infused patients, 83% achieved a remission, including 63% with a complete remission (CR).
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