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CAR T-cell cancer therapy shows promise in phase 1 rheumatoid arthritis trial

A small clinical study found that an experimental immune cell infusion led to medication-free remission in three of six patients with severe, treatment-refractory disease.

The short version

  • Researchers at Charité - Universitätsmedizin Berlin conducted a phase 1 trial using CD19 CAR T-cell therapy on six patients with severe rheumatoid arthritis that had not responded to other treatments.
  • Disease activity decreased in all six participants during up to one year of follow-up, with three patients achieving medication-free remission.
  • Safety outcomes were encouraging, showing only temporary, mild-to-moderate cytokine release syndrome with no severe neurological complications or serious adverse events.
  • Researchers plan to enroll ten additional patients in a second phase to compare the CAR T-cell therapy against an approved B-cell-targeting rheumatoid arthritis drug.

Key facts

  • The phase 1 COMPARE trial evaluated CD19 CAR T-cell therapy in six patients aged 31 to 69 with severe, treatment-refractory rheumatoid arthritis.[ScienceDaily]
  • The treatment process involves collecting a patient's T cells, modifying them to target the CD19 molecule on B cells, administering short preparatory chemotherapy, and delivering the engineered cells in a single infusion.[ScienceDaily]
  • All six participants experienced a marked drop in disease activity during up to one year of follow-up, and three entered sustained remission without arthritis medication.[ScienceDaily]
  • The engineered cells successfully reached deeper tissues, including joint tissue, bone marrow, and lymph nodes, eliminating disease-promoting B cells while largely preserving protective antibody memory from earlier vaccinations.[ScienceDaily]
  • Reported side effects were limited to temporary, mild-to-moderate cytokine release syndrome, with no severe neurological complications reported.[ScienceDaily]

What remains uncertain

  • The long-term safety profile and durability of immune system resets remain unknown due to the limited size and one-year follow-up of the initial cohort.[ScienceDaily]
  • Treatment responses varied among participants, with some not achieving a complete response and one patient experiencing a disease return after initial medication-free remission.[ScienceDaily]

Sources