The MG-001 trial is evaluating Descartes-08 CAR-T cells from Cartesian Therapeutics in the United States. These are autologous T lymphocytes genetically modified, not by DNA but by RNA, to target the B lymphocyte maturation antigen (BCMA) expressed on the surface of plasma cells.
The first two parts of the trial, in 14 adults with a generalised form of autoimmune myasthenia with an MG-ADL score of 6 or more despite treatment, showed after a median of five months following six infusions of Descartes-08 cells on an outpatient basis:
- the feasibility of producing autologous RNA-modified CAR-T cells in patients undergoing immunosuppressive treatment, without the need for lymphodepleting chemotherapy prior to their administration,
- their good safety profile, in particular the absence of the usual undesirable effects of DNA CAR-T cells (cytokine release syndrome, neurotoxicity, etc.),
- a significant clinical improvement in the evaluation criteria (MG-ADL, QMG, MGC, MG-QoL-15r scores).
The third part of the trial, currently underway, is evaluating Descartes-08 cells against placebo. For the record, results for CAR-T cells in lupus and antisynthetase syndrome have also been published in recent months.
Are CAR T cells the answer to myasthenia gravis therapy? Meisel A. Lancet Neurol. 2023 Jul.
