The MCARTY study is for patients with a type of blood cancer called multiple myeloma, where standard treatments have not been successful, and their cancer has relapsed. The study tests new CAR T cells which are made using patient’s own immune cells that are genetically modified to target a protein called BCMA, present on the cancerous cells in multiple myeloma.

Similar CAR T cells targeting BCMA have shown efficacy in treating patients with relapsed multiple myeloma, however the long-term disease control may be limited due to lack of persistence of those CAR T cells or issues with targeting the BCMA protein. MCARTY aims to overcome this by testing CAR T cells which are very-sensitive to BCMA, and by targeting a second protein found in multiple myeloma and called CD19.

The initial data obtained from the study, presented by Dr Lydia Lee from UCLH, showed that:

• 11 patients with relapsed, refractory multiple myeloma were treated with the CAR T cells targeting BCMA alone (6 patients) or both BCMA and CD19 (5 patients)
• Severe cases of ICANS (Immune effector cell-associated neurotoxicity syndrome) and/or CRS (Cytokine Release Syndrome) were not observed in any of the 11 treated patients
• Both types of CAR T cells showed promising overall response rate of 100% (7 CR/sCR, 3 PR, 1 VGPR)
• While it is too early to assess  persistence, excellent initial expansion of these CAR T was observed
• Dr Lee said: ‘We are really pleased with these initial results. Although it is early days, we hope that this trial will show that this approach will allow myeloma patients to both respond to CAR T cell therapy and also maintain that response for longer. We also expect that the lessons learnt may also contribute to the next generation of CAR T cells. Afterall, the ultimate aim remains cure!’

These results are exciting and continue to demonstrate the promise of CAR T therapy to help treat multiple myeloma.