A phase 2 trial of liposomal mitoxantrone — a reformulated version of an older chemotherapy drug — showed an acceptable safety profile and a trend toward fewer relapses in patients with neuromyelitis optica spectrum disorder (NMOSD). The liposomal formulation is designed to reduce the heart damage that has long limited conventional mitoxantrone's use. Results were presented at MSToronto2026.
An old drug gets a new look — and early results are encouraging. A phase 2 trial of liposomal mitoxantrone hydrochloride, a reformulated version of a decades-old chemotherapy agent, showed an acceptable safety profile and a potential trend toward fewer relapses in patients with neuromyelitis optica spectrum disorder (NMOSD). The findings were presented at the 10th Joint ACTRIMS-ECTRIMS Meeting (MSToronto2026) in Toronto.
Conventional mitoxantrone has been used as a second-line option in NMOSD, but its utility has been hampered by cumulative cardiotoxicity and a risk of treatment-related leukemia. The liposomal formulation encapsulates the drug to limit cardiac exposure while keeping it active in immune-related tissue longer — essentially trying to preserve the benefits while cutting the risks.
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Why it matters: NMOSD is a rare, debilitating autoimmune disease of the central nervous system with limited treatment options. A safer reformulation of mitoxantrone could expand the therapeutic toolkit — especially for patients who have relapsed on other therapies — though larger trials are needed to confirm efficacy.
