Marizomib for central nervous system‐multiple myeloma

Summary Marizomib, a natural marine product, is an irreversible proteasome inhibitor currently under investigation in relapsed‐refractory multiple myeloma (RRMM) and malignant glioma. Central nervous system‐multiple myeloma (CNS‐MM) is a rare manifestation of extra‐medullary disease with few therapeutic options, highlighting the unmet clinical need in these patients. Marizomib demonstrated encouraging activity in RRMM and has emerging clinical activity in glioma, making it a potential CNS‐MM therapeutic intervention. Herein, we present two patients with RRMM and CNS involvement who benefited from marizomib‐based therapy. These cases provide the first proof of principle for further exploring marizomib in CNS‐MM patients.