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Multiple Myeloma Responds to CAR T-Cell Therapy

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Dec. 9, 2020 -- For patients with heavily-pretreated multiple myeloma, the strong responses seen with the new CAR T-cell treatment ciltacabtagene autoleucel (cilta-cel) have also been prolonged, according to investigators in the CARTITUDE-1 trial.

Among 97 patients with multiple myeloma whose cancer got worse while on three or more prior types of treatment or following treatment with at least two types of therapy with a proteasome inhibitor and immunomodulating agent, the overall response rate was 96.9%, reported Deepu Madduri, MD, of Mount Sinai Medical Center in New York, and colleagues.

"We saw how heavily pretreated these patients were, and to see a one-time treatment get these kind of response rates is quite exceptional. What's even more impressive is that 72% of these patients were still maintaining their response at the time of data cutoff," she said in an oral abstract presented during the virtual American Society of Hematology annual meeting.

Cilta-cel is a second-generation CAR T-cell therapy.

It was granted a breakthrough therapy designation for relapsed/refractory multiple myeloma by the FDA in December 2019.

At the 2019 ASH annual meeting, Madduri reported phase 1b results from the trial, which showed that for 29 patients with heavily pretreated, relapsed/refractory multiple myeloma, the overall response rate at 6 months median follow-up was 100%, including 69% who had no signs of cancer, with 27 patients remaining free of disease progression.

Safety Data

All patients had at least one hematologic side effect, 96 of which were more severe. The events include neutropenia, anemia, thrombocytopenia, leukopenia, and lymphopenia. The median time to recovery was 2 to 4 weeks.

Infections occurred in 57.7% of patients, including pneumonia in 8.2% and sepsis in 4.1%.

Cytokine release syndrome of any type occurred in 92 patients, but only 4 had a more severe case.

Neurotoxicities occurred in 20 patients, of whom 10 had more severe cases.

Immune effector cell-associated neurotoxicity syndrome (ICANS) occurred in 16 patients, with 2 having more severe cases. The syndrome affects the central nervous system. Other neurotoxicities of any severity, many which overlapped with ICANS, occurred in 12 patients, with 9 more severe.

The median time to ICANS onset was 8 days, with a median time to recovery of 4 days. Other neurotoxicities took longer to manifest and disappear, however, with a median time to onset of 27 days, and median time to recovery of 75 days.

The study was sponsored by Janssen Research & Development and Legend Biotech. Madduri disclosed honoraria, consultancy, and speakers bureau activities for those companies and others.

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