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Pomalidomide + melphalan + dexamethasone is a combination chemotherapy regimen investigated for the treatment of systemic light-chain (AL) amyloidosis. The regimen consists of pomalidomide, a third-generation immunomodulatory imide drug (IMiD) that targets the cereblon E3 ubiquitin ligase complex; melphalan, a nitrogen mustard alkylating agent that causes DNA cross-linking; and dexamethasone, a potent synthetic glucocorticoid. This triplet therapy was evaluated in a Phase I/II clinical trial led by the M.D. Anderson Cancer Center to determine the maximum tolerated dose and safety in newly diagnosed patients. The combination aims to synergistically target the underlying plasma cell dyscrasia responsible for the production of amyloidogenic light chains. However, the specific trial (NCT01700010) was terminated early during the Phase I portion.
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