1. bb2121, a chimeric antigen receptor (CAR) T-cell therapy that targets B-cell maturation antigen (BCMA) proteins, was shown to achieve objective response rates amongst a majority of 33 patients with refractory or relapsed multiple myeloma participating in a phase 1 safety trial.
2. A majority of patients in this study were noted to have grade 3 hematologic adverse events. Non-hematologic adverse events were not as common and were often grade 2 or lower in severity.
Evidence Rating Level: 2 (Good)
Study Rundown: Multiple myeloma is an incurable plasma-cell cancer associated with the accumulation of immunoglobulin proteins. Previously proposed treatments for this disease have included monoclonal antibodies and immunomodulatory drugs, however these have not shown to be successful treatment methods and have eventually led to relapses amongst this patient group. Chimeric antigen receptor (CAR) T-cell therapy and anti-CD19 CAR T-cell therapy have proven to be promising agents for patients with leukemia and lymphoma. In this phase 1 study, researchers studied the safety of bb2121, an engineered compound which uses BCMA protein and autologous T-cell lentiviral vectors. The therapy was associated with high levels of hematologic-related adverse events including neutropenia, leukopenia, anemia, and thrombocytopenia, although most patients recovered neutrophil counts within 1 month. However, while other types of adverse events also occurred, the severity of non-hematologic adverse events were often grade 2 or below. Additionally, many patients showed an objective response to treatment, with almost half of patients experiencing a complete response.
While the sample size of this study was low and more safety and efficacy analyses are needed, these results show promise that bb2121 can be used to achieve the needed response efficacy rates for multiple myeloma treatment. Strengths of this study include its novel treatment approach and thorough evaluation of the treatment safety profile.
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