The treatment landscape for acute leukemias is evolving, with new therapies and approaches gaining traction. A recent highlight is the FDA's approval of obecabtagene autoleucel (obe-cel; Aucatzyl) in November, marking it as the first CAR T-cell therapy approved for patients with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL). This approval brings a new option to patients who have not responded to previous treatments.
The FDA approved obe-cel based on data from the FELIX trial, which demonstrated an overall complete remission rate of 63%, with 51% of patients achieving a complete response at any time. Notably, obe-cel does not require a Risk Evaluation Mitigation Strategy program. However, it carries a boxed warning for cytokine release syndrome and neurologic toxicities, which occurred at low levels, as well as the risk of secondary hematological malignancies.
CAR T-Cell Therapy in AML
While obe-cel's approval focuses on ALL, CAR T-cell therapy is also showing promise in acute myeloid leukemia (AML). Research published in Leukemia Research indicates that CAR T-cell therapy is an effective treatment for AML patients following hematopoietic stem cell transplantation (HSCT). A review of 13 studies involving 57 AML patients treated with CAR T-cell therapy showed a complete remission rate of 48%. This is a significant finding, considering the limited treatment options available for AML patients post-transplant. Currently, there are no CAR T-cell therapies approved in the US to treat AML.
Immunotherapy Combinations in AML
Beyond CAR T-cell therapy, immunotherapy is being explored as a means to expand the treatment toolbox for AML. Research published in Biomedicine & Pharmacotherapy suggests that combination therapies may be the best approach for many AML patients. Standard therapies for AML, such as chemotherapy and allogeneic HSCT, often result in significant relapse rates. Researchers are investigating several immune checkpoint inhibitors, as well as CAR T-cell therapy and antibody-based therapies like bispecific antibodies and antibody-drug conjugates. These immunotherapies offer hope for improving outcomes in AML, though the disease remains a challenge.
Patient-Reported Outcomes
In addition to novel therapies, understanding patient-reported outcomes is crucial. Fatigue has been identified as a patient-reported outcome that could be used as a prognostic indicator to determine if a patient had a higher risk of death or another adverse outcome. These findings were presented a month earlier at the 2023 American Society of Hematology Annual Meeting and Exposition. The research analyzed patients with AML, ALL, and myelodysplastic syndromes and found that those with greater fatigue levels had significantly worse outcomes.
