Bright Future for Gene Therapy in Hematologic Malignancies

Christina Loguidice
Published: Tuesday, Feb 27, 2018
Michael Pulsipher, MD

Michael Pulsipher, MD
Chimeric antigen receptor (CAR) T-cell therapy has been named the Advance of the Year in the American Society of Clinical Oncology’s (ASCO’s) Clinical Cancer Advances 2018. 1 It’s not hard to understand why. CAR T-cell therapy is unique among treatments because it simultaneously serves as a cell therapy, gene therapy, and immunotherapy. Typically, T cells are extracted from the patient, genetically modified to express a CAR, expanded in vivo, and then reinfused, enabling a patient’s immune cells to fight the cancer. Thus far, remarkable results have been observed in young patients with acute lymphoblastic leukemia (ALL) and adults with lymphoma and multiple myeloma.

Where We Currently Stand

So far, 2 CAR T-cell therapies have been approved by the FDA: tisagenlecleucel (Kymriah) and axicabtagene ciloleucel (axi-cel; Yescarta).3,4 Tisagenlecleucel was approved in late August 2017 for patients 25 years and younger with B-cell precursor ALL that is refractory to treatment or in its second or later relapse.3 Approval was based on the results of the ELIANA trial, which showed a 3-month complete remission rate of 83% and a 6-month probability of survival of 89%.5

Current Treatment Challenges

A key challenge with CAR T-cell therapy is the adverse event (AE) profile, with cytokine release syndrome (CRS) and neurological toxicity being common, particularly among adults. In the longer term ZUMA-1 data, the incidence of grade ≥3 AEs was 12% for CRS and 31% for neurological toxicity among 101 patients.8 “Almost half of the patients required some type of intervention with either an IL-6 receptor antibody blocker or steroids,” Maloney said.
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TitleExpiration DateCME Credits
Advances in™ Multiple Myeloma: Changing Treatment Paradigms and the Emerging Potential of CAR T-Cell TherapyAug 30, 20191.5
Clinical Practice Connections™: From Diagnosis to Emerging Immunotherapeutic Options: Understanding the Burden and Risks in Peanut AllergySep 28, 20191.0
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