- Researchers from the National Institutes of Health (NIH) presented interim trial data showing positive responses from an engineered T-cell therapy in patients with multiple myeloma. Presented at this year's American Society of Hematology (ASH) meeting, the positive data has important implications for Bluebird Bio and Celgene, who are partnering on development of a T-cell therapy using the same method as the NIH researchers.
- The therapy, CAR-BCMA (B-cell maturation antigen), was tested on 12 patients with multiple myeloma who had already received multiple therapies. The overall response rate was 33 percent.
- Other data presented at ASH may support expansion of the label for AbbVie/Johnson & Johnson's oral therapy Imbruvica (ibrutinib) as a first-line therapy in patients with chronic lymphocytic leukemia (CLL).
Bluebird/Celgene's new therapy is exciting not only because of the high response rates and the potential for remission in certain patients, but also because BCMA is a newly targeted protein found on both healthy and malignant plasma cells. This therapy is the first CAR-T that completely eradicated or decreased measurable multiple myeloma, according to government researcher, Dr. James Kochenderfer of the National Cancer Institute, as reported by The Street.
One of the two patients receiving the highest dose of CAR-BCMA went into complete remission. The other had undetectable symptoms of myeloma in bone marrow plasma cells, although that patient had not yet gone into complete remission.
New data on Imbruvica, which is currently used as second-line treatment for CLL, showed amazing results as first-line treatment—with an 85% decreased risk of death, based on a treatment population of 269 patients with CLL aged 65 and older.
Although there have already been three newly approved treatments for multiple myeloma this year, moving Imbruvica to first-line treatment status could greatly enhance treatment options. In the study, there were three deaths among Imbruvica patients and 17 among patients treated with standard of care (chlorambucil). Overall median progression-free survival (PFS) was 18.9 months in chlorambucil-treated patients. There are no PFS results for Imbruvica-treated patients yet, because at the 18-month mark most of them had not yet experienced disease progression.