How can drug repurposing revolutionize rare cancer therapies
Drug repurposing offers a promising strategy to address the critical unmet needs of rare cancers, which collectively affect millions globally but individually lack dedicated therapies due to economic and logistical barriers. By leveraging existing drugs with known safety profiles, researchers can bypass early development stages and accelerate access to potential treatments. Below, we explore the mechanisms, successes, and challenges of this approach. Mechanisms of Drug Repurposing in Rare Cancers Repurposed drugs target rare cancers through: Polypharmacology : Drugs like auranofin (originally for arthritis) exploit multitarget effects to inhibit cancer stem cells in gastrointestinal stromal tumors 1 . Pathway modulation : Tamoxifen (initially a contraceptive) blocks estrogen receptors in ER+ breast cancers 1 . Microenvironment regulation : Non-oncology drugs such as metformin (diabetes) alter tumor metabolism and immune responses 2 3 . Clinical Succe...