Trending Clinical Topic: CAR T-cell Therapy

Ryan Syrek


December 24, 2021

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Potential practice-changing information presented at the American Society of Hematology 2021 Annual Meeting, along with other promising recent findings, resulted in chimeric antigen receptor (CAR) T-cell therapy becoming this week's top trending clinical topic. Initially, the results of two phase 3 trials suggest that CAR T-cell therapy has the potential to replace chemoimmunotherapy for some patients with large B-cell lymphoma, according to Frederick L. Locke, MD, from the Moffitt Cancer Center in Tampa, Florida, who presented findings at ASH 2021 (see Infographic).

The ZUMA-7 trial had a median follow-up of 24.9 months. Patients were randomly assigned to receive CAR T-cell therapy with axicabtagene ciloleucel, or axi-cel (Yescarta). The TRANSFORM study compared the CAR T construct lisocabtagene maraleucel, or liso-cel (Breyanzi), with standard-of-care second-line chemotherapy. The trials differed slightly in eligibility criteria and other details, but their overall results show great promise for improving second-line therapy for patients with relapsed or refractory large B-cell lymphomas, according to researchers.

In an interview with Medscape Medical News, Locke said that, based on the findings of ZUMA-7 trial that he presented, it's likely that chemotherapy in the second-line setting for relapsed/refractory large B-cell lymphoma will largely fall by the wayside. This belief was echoed by Manali Kamdar, MD, from the University of Colorado Cancer Center in Aurora, who presented the TRANSFORM trial results at ASH 2021. She remarked during the briefing that "in my opinion, this is a breakthrough therapy, which has shown superiority over standard of care, in terms of not just efficacy but also an extremely favorable safety profile."

Beyond lymphoma, recent studies have shown that CAR T-cell therapy may be effective against neuroblastoma. Peptide-centric chimeric antigen receptor (PC-CAR) cells have eliminated the tumors in mice and may be ready for testing in the clinic as soon as next year. "Our next steps are to move the PHOX2B PC-CAR into a clinical trial in late 2022, develop additional PC-CARs for neuroblastoma, and discover PC-CAR targets in other important childhood cancers," John Maris, MD, of Children's Hospital of Philadelphia, told Reuters.

This fall, the US Food and Drug Administration (FDA) approved the CAR T-cell treatment brexucabtagene autoleucel (Tecartus) for the use in adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). This is a "meaningful advance" because "roughly half of all adults with B-ALL will relapse on currently available therapies," said Bijal Shah, MD, of Moffitt Cancer Center, Tampa, Florida, in a press statement from the manufacturer, Kite. The FDA approval, which was the second indication for brexucabtagene autoleucel, was based on results from ZUMA-3, a multicenter, single-arm study of 71 patients, with 54 efficacy-evaluable patients.

T-cell therapy was also reportedly effective in treating refractory systemic lupus erythematosus (SLE). A 20-year-old woman with severe, refractory SLE; active lupus nephritis; pericarditis; and other symptoms had serologic and clinical remission after receiving a CAR T-cell product directed against the B-cell surface antigen CD19, reported Georg Schett, MD, and colleagues from the German Center for Immunotherapy at Friedrich Alexander University Erlangen-Nuremberg in Erlangen, Germany. "Given the role of B cells in a variety of severe autoimmune diseases, CAR T-cell therapy that targets B-cell antigens may have wider application," they wrote in a letter to the editor of The New England Journal of Medicine. Schett said in an email response to an interview request that the patient has remained healthy and asymptomatic without further treatment after 6 months of follow-up.

This latest round of positive news surrounding CAR T-cell therapy caps off an encouraging year. Earlier in 2021, CAR T-cell therapy showed survival improvement in patients with relapsed/refractory follicular lymphoma. It was also approved by the FDA to treat relapsed/refractory multiple myeloma in adult patients who had received four or more lines of therapy. In addition, patients who underwent CAR T-cell therapy were found to have improved responses to COVID-19 vaccination, with overall cellular and humoral responses ≥80%. Given the wealth of promising news regarding the "breakthrough" treatment, it's no wonder that CAR T-cell therapy became this week's top trending clinical topic.

Learn more about CAR T-cell therapy.


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