Despite the introduction of oral drugs for the treatment of chronic lymphocytic leukemia or small cell lymphocytic lymphoma, some patients do not have the desired response to these new therapy options. 

A new CD-19 directed chimeric antigen receptor T-cell therapy (CAR T) -- lisocabtagene maraleucel (liso-cel) -- resulted in a 95% response when used to treat patients with relapsed or refractory CLL, according to recent data.

Results of the investigational study were presented at the recent American Society of Hematology meeting. The study included 23 patients with a median age of 66. Of those involved, 19 were considered to have high-risk disease and underwent an average of five prior therapies. All the patients involved had already been treated with ibrutinib, with 56.5% progressing on ibrutinib and venetoclax. 

One group received 50x106 CAR+ T cells, while the second group received 100x106. Of the 22 patients evaluated, 95% were deemed had the best possible response, while 45% reached complete remission, and 36% achieved a partial response. The average time to reach peak expansion of CAR+ T Cells was 15 days.

The most common grade 3 or 4 adverse events were thrombocytopenia (70%), anemia (96%), neutropenia (56.5%), and leukopenia (43.5%). In addition, two patients had grade 3 cytokine release syndrome, and five had grade 3 or above neurological events, including encephalopathy. 

“It’s been almost two years since the first few patients received liso-cel on this trial and at least three or four of them are still in excellent remission with no recurrent MRD, while later patients are still in follow-up at under two years” Siddiqi said in a press release about the study. “The results for this trial are very encouraging as liso-cel has so far demonstrated to be highly effective with few serious side effects in patients with fairly refractory CLL.”

In December, the drug’s manufacturer submitted a Biologics License Application to the FDA for liso-cel for the treatment of patients with diffuse large B-cell lymphoma who have progressed on at least 2 prior therapies. Liso-cel is not yet approved for any indication.