The following article features coverage from the American Society of Clinical Oncology 2020 meeting. Click here to read more of Hematology Advisor’s conference coverage.

GC027, the first humanized chimeric antigen receptor (CAR)-T cell therapy for relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL) in adults, appears to be both effective and have a manageable safety profile, according to research presented during the ASCO20 Virtual Scientific Program.

Mortality rates are high in relapsed/refractory T-ALL, necessitating novel treatments to improve survival. CD7, a T-cell antigen expressed in more than 95% of disease samples, represents a plausible target in this setting. GC027, which targets CD7 and was developed using using lentivirus and CRISPR/Cas9, showed promise in murine models for treating T-ALL.

For this single-arm, open-label study, researchers evaluated the safety and efficacy of GC027 in relapsed/refractory T-ALL. All included patients were between 18 and 70 years old, had a projected survival of more than 3 months, and had a performance status of 0 to 2. Patients with extramedullary disease or central nervous system involvement were not eligible to participate.

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Five men (median age, 24 years) were enrolled in the trial. The median number of prior lines of therapy was 5, no patients had undergone prior stem cell transplantation, and the median baseline bone marrow tumor burden was 38.2%.

All 5 patients had a complete response or complete response with incomplete hematologic recovery, and 4 of the 5 patients were minimal residual disease–negative.

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All patients also experienced grade 3 (4 patients) or 4 (1 patients) cytokine release syndrome; no grade 5 events of any kind were reported.

“With a single infusion of GC027, 80% of the patients had robust CAR-T cell expansion and achieved persistent [minimal residual disease–negative complete response] without using any biologics as part of the preconditioning therapy or bridging to [hematopoietic stem cell transplantation],” the authors wrote.


Wang X, Li S, Gao L, et al. Safety and efficacy results of GC027: The first-in-human, universal CAR-T cell therapy for adult relapsed/refractory T-cell acute lymphoblastic leukemia (r/r T-ALL). Presented at: ASCO20 Virtual Scientific Program. J Clin Oncol. 2020;38(suppl): abstr 3013.