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On June 11, 2026, investigators at Mass General Brigham Cancer Institute and University of Chicago Medicine Comprehensive Cancer Center published phase 1 results in The New England Journal of Medicine for anito-cel, a BCMA-targeted CAR-T cell therapy tested in relapsed/refractory multiple myeloma. The trial enrolled 38 patients who had already been through multiple lines of treatment, making this a particularly hard-to-treat group.
Every treated patient responded to anito-cel, and nearly 80 percent achieved a complete response. More than half of participants remained progression-free two years after treatment, while 65 percent were still alive at three years. For a phase 1 study, the depth and durability of those remissions are striking.
The safety data also stood out. Existing BCMA-directed CAR-T therapies can cause cytokine release syndrome, immune effector cell-associated neurotoxicity syndrome, and rare but serious delayed neurological problems. In this study, serious immune-related and neurological side effects were uncommon, and no delayed neurological complications were reported.
That matters because neurological toxicity can be one of the hardest barriers to CAR-T treatment. If anito-cel continues to show a milder side-effect profile in larger trials, it could widen the pool of patients who are willing to try this approach.
Anito-cel was built with a D-domain binding domain, an engineered protein fragment designed to recognize BCMA on myeloma cells. Most CAR-Ts use more conventional antibody-derived binding domains, and the choice of binding domain can change how strongly the therapy activates T cells.
The research team’s laboratory analyses suggest that anito-cel retains strong tumor-killing activity while avoiding excessive immune activation. That balance may explain both the clinical efficacy and the low rate of inflammatory complications. The investigators described this as a plausible reason why the therapy appeared to behave differently from other BCMA-targeted CAR-T products.
Michael R. Bishop, MD, director of cellular therapy at UChicago Medicine and senior author of the study, noted that the response pattern was unusually strong for a phase 1 trial. Even at lower doses, most patients had complete responses, and none developed severe toxicities. That prompted a deeper examination of the biology behind the therapy.
Matthew Frigault, MD, of Mass General Brigham Cancer Institute and first author, emphasized the clinical relevance of the safety findings. He pointed out that neurotoxic and inflammatory complications can have serious, long-lasting effects, so the absence of those events in this phase 1 study is encouraging. He also credited the novel D-domain binder as a likely contributor to the profile.
Anito-cel is already being evaluated in phase 2 and phase 3 clinical trials designed to confirm these findings in larger groups of patients. If those studies hold up, the therapy could provide another option for patients who relapse after standard treatments such as proteasome inhibitors, immunomodulatory drugs, and anti-CD38 antibodies.
The study was supported by Arcellx and Kite, a Gilead company. The full findings, titled “Anito-cel, a D-Domain BCMA CAR-T: Phase 1 Results and Binder Characterization,” appear in The New England Journal of Medicine.
UChicago Medicine, meanwhile, continues to invest in cancer care across the region. The institution is scheduled to open the AbbVie Foundation Cancer Pavilion in April 2027, a freestanding cancer center designed to bring advanced diagnostics and new therapies to patients.
