Off-target drug repurposing for rare and ultra-rare cancers: towards a scalable therapeutic framework.

Off-target drug repurposing offers a promising therapeutic strategy for rare cancers, with evidence supporting its potential in 20-50% of anticancer agents.

• Why it matters: Rare cancers lack well-characterized targets and timely treatment options, creating a critical need for alternative approaches that can be rapidly integrated into clinical practice.
• What they did: The authors review emerging pharmacological evidence and criticisms of low-evidence genomic matching, advocating for prospective clinical trials that incorporate off-target candidates within adaptable, safety-focused frameworks.
• The result: Implementing rigorous, longitudinal, and adaptive trial designs can validate off-target therapies, ultimately expanding treatment options and improving outcomes for patients with rare and ultra-rare cancers.