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Innovation Observatory > Reports > Drugs > Ciltacabtagene autoleucel for relapsed and refractory multiple myeloma

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Ciltacabtagene autoleucel for relapsed and refractory multiple myeloma


Cancer and Palliative Care

September 2021

Ciltacabtagene autoleucel is in clinical development for the treatment of adults with relapsed and lenalidomide-refractory multiple myeloma (MM). MM is a rare, incurable cancer of the plasma cells in the bone marrow. Abnormal plasma cells interfere with the production of red and white blood cells as well as platelets, causing symptoms such as bone pain and fragility, weakness, unusual bleeding (from the gums and nose, as well as heavy periods) and eventually kidney damage. In the early stages, MM is symptomless, then, as disease progresses, patients experience periods of time without symptoms followed by periods where the symptoms return (relapsed MM). Eventually the periods without symptoms will shorten and the illness will become immune to the treatment (refractory MM). As most patients experience serial relapse to existing treatments, there is a need for new treatment options.
Ciltacabtagene autoleucel is a type of gene and cell therapy that targets a protein called BCMA (B cell maturation antigen) which is expressed on B-cells (a type of immune cell). Binding of ciltacabtagene autoleucel to BCMA prevents B-cell maturation and differentiation into plasma cells. It is administered as an intravenous infusion and aims to kill cancer cells by harnessing the power of a patient’s own immune system. If licenced, ciltacabtagene autoleucel would be the first BCMA targeted CAR-T cell therapy with two distinct antigen binding domains for MM.

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