A phase 1/2 study of donor-derived anti-CD33 CAR T-cell therapy (VCAR33) for relapsed/refractory AML after allogeneic HCT

Blood 2026

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Authors: Muhammad Umair Mushtaq, John F. DiPersio, Jacques Azzi, BW Cooper, Guenther Koehne, Divya Koura, Joseph E. Maakaron, John Magenau, Brian McClune, Joseph C Rimando, Nirali N Shah, Hyung Chae Suh
+23 more Kelly Beuka, John Sturrock, Mugdha Harshakumar Nikam, Eric Berglund, Jianxin Hu, Yonina Keschner, Julia Etchin, John R. Lydeard, Michele Vasquez, David O'Donnell, Guy Mundelboim, Sanjana Thosar, Giacomo Canesin, Juliana M. Xavier-Ferrucio, Sharon L. Hyzy, Deborah Lloyd, Kristin Spink, Diana Hummel, Melissa M. Lee-Sundlov, Julian Scherer, Michelle I. Lin, Jennifer Whangbo, Lori Muffly
DOI: 10.1182/blood.2025031053
Cited by: 9
References: 44

Abstract

ABSTRACT: VCAR33, a donor-derived CD33-directed chimeric antigen receptor T-cell (CAR T) product, was developed to decrease relapse of high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) after allogeneic hematopoietic cell transplantation (alloHCT). We describe preclinical characterization of the VCAR33 construct, which was optimized for long-term antitumor surveillance based on killing and persistence assays. Prior to its use in post-alloHCT maintenance, we evaluated safety and efficacy of VCAR33 in a phase 1/2 clinical study for adults with relapsed or measurable residual disease (MRD)-positive CD33+ AML/MDS after alloHCT. Fifteen patients received VCAR33 across 2 arms stratified by disease burden: 7 patients in arm A (bone marrow blasts ≥5%) at dose level 1 (DL1; 1 × 106 CAR+ Ts per kg) and 8 patients in arm B (bone marrow blasts

Fields of Study & Subjects

CAR-T cell therapy research Acute Lymphoblastic Leukemia research Hematopoietic Stem Cell Transplantation

Publication details

Journal / source
Blood
Publisher
American Society of Hematology
Volume
147
Issue
17
Pages
1914–1927
Publication date
4 February 2026
Language
EN