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Mount Sinai scientists have become the first to report a potentially serious side effect related to a new form of immunotherapy known as CAR-T cell therapy, which was recently approved for the treatment of multiple myeloma.
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April 10, 2022 8:42 PM
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Multiple myeloma is a complex and incurable type of blood plasma cancer that often requires multiple treatments as the disease progresses and becomes resistant to previous treatments, often resulting in chronic disease with periods of acute illness. CAR-T cells are used to target a protein known as B-cell maturation antigen
(BCMA) that is commonly found in multiple myeloma. This therapy has shown impressive response rates in people with particularly complex and treatment-resistant multiple myeloma. However, according to scientists at Icahn Mount Sinai, 3 months after injection of CAR-T cells, 1 patient began to show progressive neurological features of Parkinson's-like symptoms. The patient died as a result of the treatment. Researchers found BCMA in the basal ganglia of his brain and scarring in that area. This serious side effect could be due to the therapy targeting BCMA in the brain. Thus, this study shows that BCMA-targeted CAR-T cell therapy can cross the blood-brain barrier in at least a subset of patients and can cause progressive neurocognitive and movement impairment. In addition, the scientists also found BCMA expression in the brains of healthy individuals. CAR-T cell therapies, while effective in multiple myeloma, warrant close monitoring for neurotoxicity.