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

Mortalityrates are high in relapsed/refractory T-ALL, necessitating novel treatments toimprove survival. CD7, a T-cell antigen expressed in more than 95% of diseasesamples, represents a plausible target in this setting. GC027, which targetsCD7 and was developed using using lentivirus and CRISPR/Cas9, showed promise inmurine 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.

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

All5 patients had a complete response or complete response with incomplete hematologicrecovery, and 4 of the 5 patients were minimal residual diseasenegative.

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

Witha single infusion of GC027, 80% of the patients had robust CAR-T cell expansionand achieved persistent [minimal residual diseasenegative complete response]without using any biologics as part of the preconditioning therapy or bridgingto [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.

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GC027 Shows Promise as Therapy for Adult Relapsed, Refractory T-Cell ALL - Hematology Advisor