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Liso-cel, a CAR-T Therapy for Double Refractory CLL

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Medically reviewed by Dr. Brian Koffman

The Bottom Line:

Liso-cel produced durable, complete remissions in a subgroup of double refractory CLL / SLL patients (about 1 in 5 patients). Side effects were common and typical of CAR-T therapy.

Who Performed the Research and Where Was it Presented:

Dr. William Wierda from MD Anderson Cancer Center and colleagues presented the results at the American Society for Hematology (ASH) Annual Meeting 2023.


Patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who are double refractory (meaning treatment has failed with both BTK inhibitors and venetoclax) have few treatment options and poor outcomes. Liso-cel (Lisocabtagene Maraleucel) is a CAR-T therapy being tested for the treatment of relapsed or refractory CLL / SLL. CAR-T therapies re-engineer the body’s own immune cells to fight the cancer.

Methods and Participants:

The TRANSCEND CLL 004 study is a phase 1/2 clinical trial of liso-cel, a CD19 CAR-T therapy, for patients with relapsed or refractory CLL / SLL. While the study had three different arms, the results discussed here are for the group that received liso-cel only. This analysis looked at double refractory patients, meaning that they had relapsed on both a BTK inhibitor and venetoclax and who received the higher of two dose levels used in the trial (100 million CAR-T cells).


  • In total, 50 patients were double refractory and received the higher dose level of liso-cel.
  • Activity was modest, with an overall response rate of 44% and a complete remission rate of 20%. However, it is important to remember that this is a subgroup of patients who have few treatment options.
  • Of the patients who achieved complete remission, all have undetectable MRD, and none have yet progressed after a median follow-up time of two years.
  • Twelve patients achieved partial remission; their median progression-free survival time was about two years.
  • CAR-T therapy is associated with some well-known side effects, including cytokine release syndrome, which usually produces flu-like symptoms, including fever, and neurotoxicity, which generally produces mental confusion symptoms.
  • The majority of patients (85%) experienced some level of cytokine release syndrome symptoms, but only 8% experienced severe cytokine release syndrome symptoms.
  • Neurological events occurred in 45% of patients, and 18% experienced severe neurotoxicity symptoms.
  • In general, the cytokine release syndrome and neurotoxicity symptoms were temporary and resolved.


Liso-cel produced durable, complete remissions in a subgroup of double refractory CLL / SLL patients (about 1 in 5 patients). Side effects were common and typical of CAR-T therapy.

Liso-cel is currently under review by the FDA for use in relapsed or refractory CLL / SLL. If it does receive approval in the future, its availability may be more limited than oral drugs because CAR-T therapies are more challenging to administer and are best given at specialized centers with CAR-T experience.

Links and Resources:

Watch the interview on the abstract here:

Liso-cel – A CAR-T Therapy for Double Refractory CLL

You can read the actual ASH abstract here: Lisocabtagene Maraleucel (liso-cel) in R/R CLL / SLL: 24-Month Median Follow-up of TRANSCEND CLL 004

Take care of yourself first.

Ann Liu, PhD