Clinical Advances in Hematology & OncologyThe Approval of Lisocabtagene Maraleucel in Chronic Lymphocytic Leukemia

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Lisocabtagene maraleucel (liso-cel) has received accelerated FDA approval for chronic lymphocytic leukemia (CLL) and small lymphocytic leukemia (SLL) after at least two prior treatments. This approval marks the first chimeric antigen receptor (CAR) T-cell therapy for CLL, offering a new option for patients with limited alternatives. The TRANSCEND CLL 004 trial demonstrated promising efficacy and manageable safety, positioning liso-cel as an important advancement in CLL treatment.

Key Points:

• Liso-cel received accelerated FDA approval on March 14, 2023 for CLL/SLL after ≥2 prior treatments, including a BTK inhibitor and BCL2 inhibitor
• In the TRANSCEND CLL 004 trial, liso-cel showed a 20% complete remission rate and 45% overall response rate in heavily pretreated CLL patients
• Median progression-free survival was 12 months overall and 26 months in partial responders
• Common adverse events included cytokine release syndrome (83% any grade, 9% grade ≥3) and immune effector cell-associated neurotoxicity syndrome (46% any grade, 20% grade ≥3)
• Liso-cel must be administered at specialized centers with experience managing CAR T-cell therapy complications
• Ongoing research is examining liso-cel in earlier treatment lines and in combination with other agents like BTK inhibitors
• Future studies may explore liso-cel’s potential in high-risk CLL patients and its ability to produce long-term remission

“We have been very successful in developing targeted small-molecule inhibitor–based therapy for patients who have CLL, but we still see resistance to both Bruton tyrosine kinase (BTK) inhibition and BCL2 inhibition.”
– William G. Wierda, MD, PhD, Professor of Medicine, Department of Leukemia, Division of Cancer Medicine,The University of Texas MD Anderson Cancer Center

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