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April 26, 2026 — A Phase 1 clinical trial led by Mass General Brigham Cancer Institute and University of Chicago Medicine Comprehensive Cancer Center found that an investigational BCMA-targeted CAR-T cell therapy, anito-cel, can produce deep and durable responses in patients with relapsed/refractory multiple myeloma. The peer-reviewed data appear in The New England Journal of Medicine.
Multiple myeloma is a blood cancer that can often be managed for years, but most patients eventually relapse or stop responding to treatment. Each additional course of therapy tends to produce shorter remissions, which is why new options are needed beyond standard drugs, transplants, and immunotherapies.
CAR-T therapy has already changed how this disease is treated. It takes a patient’s own T cells and engineers them to attack a protein on myeloma cells, usually BCMA. The challenge is balancing powerful tumor-killing activity with acceptable side effects.
Anito-cel was designed with a D-domain BCMA binder. That binder is the part of the engineered T cell that recognizes the tumor. It may interact with BCMA in a way that differs from binders used in already approved CAR-T therapies.
All 38 patients treated in the trial responded to anito-cel. Nearly 80% reached a complete response, meaning their cancer became undetectable by standard testing. More than half of patients were still alive without disease progression two years later, and 65% were alive after three years.
Those numbers are striking because the trial enrolled heavily pretreated patients. Participants had already received multiple prior lines of therapy, a group where durable responses are hard to achieve.
Serious immune-related side effects were uncommon. No delayed neurological complications were reported, and no patients developed severe toxicities at the doses tested. That safety signal matters because neurological problems after BCMA CAR-T therapy are rare but can be severe, and some complications may appear months after the infusion.
The research team’s laboratory analyses suggested that anito-cel’s engineered D-domain binder kept the ability to kill cancer cells while limiting excessive immune activation and inflammatory signaling. Too much chronic stimulation can exhaust CAR-T cells and make them less likely to persist. A binder that avoids that problem could help explain the combination of efficacy and safety seen in the clinic.
Senior author Dr. Michael R. Bishop, director of the David and Etta Jonas Center for Cellular Therapy at UChicago Medicine, said the Phase 1 results were impressive, with complete responses in most patients even at low doses and no severe toxicity. He said the team was motivated to look more closely at why the binder behaved this way.
Lead author Dr. Matthew Frigault, of Mass General Brigham Cancer Institute, focused on the safety data. Neurological and inflammatory side effects from CAR-T can be serious and long-lasting, so their absence in this small trial is encouraging. He pointed to the novel D-domain binder as a possible reason and called the results a strong foundation for continued investigation.
Anito-cel is already being evaluated in Phase 2 and Phase 3 trials in larger patient populations. Those studies will provide longer follow-up, more detailed safety information, and direct comparisons with current standards of care. They will also help researchers identify which patients are most likely to benefit from this approach.
Because this is early-phase data, cross-trial comparisons with approved CAR-T products are not reliable. Patient populations, prior treatments, and toxicity grading can differ across studies. Still, the durability of the responses supports moving ahead with larger trials and may influence how next-generation CAR-T therapies are designed.
The study, titled “Anito-cel, a D-Domain BCMA CAR-T: Phase 1 Results and Binder Characterization,” was funded by Arcellx and Kite, a Gilead company. The author team includes investigators from multiple academic centers.
UChicago Medicine is also expanding its cancer-care infrastructure. The AbbVie Foundation Cancer Pavilion is scheduled to open in April 2027 as Illinois’s first freestanding cancer pavilion, with advanced diagnostics and cellular therapy housed in a dedicated facility.
For patients with hard-to-treat multiple myeloma, anito-cel is a promising investigational option that could combine durable efficacy with a lower risk of the neurological side effects seen with some BCMA CAR-T therapies. Larger randomized trials will determine whether the Phase 1 results hold up across a broader patient population.
As of April 26, 2026, anito-cel has not been approved by the U.S. Food and Drug Administration. Patients interested in CAR-T therapy should consult an oncology team about eligibility, risks, and available alternatives.
