The Phase 2 CAR-PRISM trial shows striking results for high-risk smoldering multiple myeloma using the CAR T-cell therapy ciltacabtagene autoleucel (Carvykti). A single infusion led to 100% of patients achieving minimal residual disease (MRD) negativity at a very deep level.
At a median follow-up of 15.3 months, all patients achieved complete or stringent complete responses, with no disease progression or deaths reported. This level of response is much higher than what is typically seen with standard multi-drug treatments.
Side effects were common but manageable. All patients experienced mild to moderate cytokine release syndrome, and about 35% had some neurological side effects. Adjustments such as lowering the dose and adding preventive steroids helped improve safety without reducing effectiveness.
These findings suggest that early use of CAR T-cell therapy could potentially stop the disease before it becomes active multiple myeloma, moving closer to a one-time treatment approach instead of long-term disease control.