Use in Myeloma

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The use of CAR T cell therapy is being explored beyond just targeting CD19. In the treatment of myeloma, researchers are using therapies engineered to target a protein on myeloma cells called B cell maturation antigen (BCMA). Preliminary results from 2 trials of BCMA-targeted therapies were presented at the 2017 ASCO Annual Meeting.

In a Chinese trial, 33 of 35 patients reached complete remission within 2 months of receiving BCMA-targeted CAR T cells.3 Nineteen patients with a minimum of at least 4 months of follow-up had a stringent complete response; 5 of these patients have been followed for at least 1 year with no signs of cancer.

In the second trial, 21 patients were treated with a BCMA-targeted CAR T cell therapy, with 18 eligible for evaluation.4 In the phase 1 trial, the dose of therapy was gradually increased. Only 1 of the 3 patients treated at the lowest dose responded. But among subsequent patients, the overall response rate was 100%, with 27% of patients having a stringent complete response and the remainder having a very good partial response.

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Other trials are also exploring CD138-specific CAR T cells.

Although these therapies show a lot of promise for patients with myeloma, Dr Savoldo cautioned that even if tested successfully and approved by the US Food and Drug Administration (FDA), a lot of work will required to develop the infrastructure and hospital training necessary for administering these therapies.

“I doubt this will be the type of therapy you can get at just any hospital,” Dr Savoldo said. “They will more likely be like bone marrow or stem cell transplantation than like a regular chemotherapy regimen.”

“These therapies are complex but promising, and it will be interesting to see how they will translate to the treatment of solid tumors,” Dr Savodo said.


  1. FDA approval brings first gene therapy to the United States [news release]. Silver Spring, MD: US Food and Drug Administration; August 30, 2017. Accessed October 10, 2017.
  2. Landoni E, Savoldo B. Treating hematological malignancies with cell therapy: where are we now? Expert Opin Biol Ther. 2017 Oct 5. doi: 10.1080/14712598.2018.1384810 [Epub ahead of print]
  3. Fan F, Zhao W, Liu J, et al. Durable remissions with BCMA specific chimeric antigen receptor (CAR)-modified T cells in patients with refractory/relapsed multiple myeloma. J Clin Oncol. 2017;35(suppl; abstr LBA3001). doi: 10.1200/JCO.2017.35.18_suppl.LBA3001
  4. Berdeja JG, Lin Y, Raje NS, et al. First-in-human multicenter study of bb2121 anti-BCMA CAR T-cell therapy for relapsed/refractory multiple myeloma: updated results. J Clin Oncol. 2017;35(suppl; abstr 3010). doi: 10.1200/JCO.2017.35.15_suppl.3010