CAR T-cell Therapy Shows Promise for Refractory/Relapsed Multiple Myeloma
Key findings
- In this multicenter, open-label, phase 1 trial, 33 patients with refractory/relapsed multiple myeloma received an infusion of idecabtagene vicleucel, a CAR T-cell therapy that targets B-cell maturation antigen
- Cytokine release syndrome was mostly of grade 1 or 2; the two grade 3 events (6%) resolved within 24 hours
- The overall frequency of grade 3/4 neurologic toxic effects was also low (3%), with no fatal events
- The objective response rate was 85%, including complete responses in 45% of patients, and the median duration of response was 10.9 months without any ongoing myeloma therapy
Chimeric antigen receptor (CAR) T-cell therapy has proven to benefit some patients with leukemia or lymphoma, and now it's being investigated for refractory/relapsed multiple myeloma. Idecabtagene vicleucel (ide-cel, formerly known as bb2121) is a CAR T-cell therapy that targets B-cell maturation antigen (BCMA), which is principally expressed by plasma cells and some mature B cells.
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In The New England Journal of Medicine, Noopur Raje, MD, director of the Multiple Myeloma Center at the Massachusetts General Hospital Cancer Center, and colleagues published promising results from a multicenter, open-label, phase 1 trial of ide-cel in U.S. patients with multiple myeloma.
Study Methods
Eligible participants had received at least three previous lines of therapy, including a proteasome inhibitor and an immunomodulatory agent, or were refractory to both drug classes. 36 patients underwent leukapheresis between January 31, 2016, and April 30, 2018. Manufacture of ide-cel was successful for all of them, but three discontinued before infusion because of disease progression. The remaining patients received one infusion of ide-cel (doses varied).
Safety
- All 33 patients had adverse events (AEs), including grade 3/4 in 97% (one patient who had a grade 3 event during the first eight weeks later died of cardiorespiratory arrest)
- The most common grade 3/4 AEs were hematologic, as expected with lymphodepleting chemotherapy
- Cytokine release syndrome—Grade 1/2 in 70%; grade 3 in 6% (grade 3 resolved within 24 hours)
- Neurologic toxic effects—Grade 1/2 in 39%; grade 4 in 3% (grade 4 resolved within one month)
- Infection—Grade 1/2 in 36%; grade 3 in 6%
Efficacy
- Objective response rate—85%, including 45% with complete responses
- Median duration of response—10.9 months without any ongoing myeloma therapy
- Median progression-free survival—11.8 months
- 1-year progression-free survival rate—40%
- Durable persistence of CAR+ T cells—57% of patients at six months; 20% at 12 months
Next Steps
The participants were transferred to a follow-up safety study that will last up to 15 years.
In September 2020, the FDA granted priority review to ide-cel for treatment of relapsed/refractory multiple myeloma.
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