Science Highlights
Published on March 31, 2026
Study of VCAR33 for relapsed/refractory AML post-alloHCT
by Blood
Mushtaq MU, DiPersio JF, Azzi J, et al. A Phase 1/2 Study of Donor-Derived Anti-CD33 CAR T-Cell Therapy (VCAR33) for Relapsed/Refractory AML After Allogeneic HCT. Blood. 2026.
Scientists report encouraging results from a clinical trial of VCAR33, a chimeric antigen receptor (CAR) T-cell construct designed to curtail acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) relapse after allogeneic hematopoietic cell transplantation. The Phase I/II study included 15 patients with CD33-positive AML/MDS who relapsed after transplant or were positive for measurable residual disease. Researchers calculated an overall response rate to VCAR33 of 20%. Although the maximum tolerated dose could not be identified before the investigation was terminated early for non-safety reasons, the evidence collected by that point signaled an acceptable safety profile at delivery up to 3 x 106 CAR+ T cells/kg. The donor-derived anti-CD33 product could be manufactured within an acceptable window of time for use in adults with relapsed/refractory AML post-transplantation, the investigators noted.
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