How can drug repurposing revolutionize rare cancer therapies

By Editorial Team
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 tumors1.

  • Pathway modulation: Tamoxifen (initially a contraceptive) blocks estrogen receptors in ER+ breast cancers1.

  • Microenvironment regulation: Non-oncology drugs such as metformin (diabetes) alter tumor metabolism and immune responses23.

Clinical Successes and Case Studies

  • DRUP Study: The Drug Rediscovery Protocol achieved a 32% clinical benefit rate (stable disease or better) across 1,350 patients with rare cancers, including off-label use of trastuzumab for gastric cancer 12 years after its breast cancer approval3.

  • Selumetinib: Originally for melanoma, this MEK inhibitor gained FDA approval for neurofibromatosis type 1 through strategic partnerships and pooled funding5.

  • Nitroxoline: A 50-year-old UTI antibiotic showing efficacy against Balamuthia meningoencephalitis in early trials4.


Challenges and Solutions

  • Economic barriers: Limited patent incentives for generics deter industry investment. Solutions include nonprofit-led trials (e.g., Children’s Tumor Foundation)5 and public-private partnerships.

  • Evidence generation: Rare cancers lack large patient cohorts. Platforms like the EDGE Tool automate EHR data extraction to identify off-label efficacy4.

  • Regulatory attrition: 21 cancer drugs lost accelerated approval since 2015 due to confirmatory trial failures6. Adaptive trial designs and real-world data integration are critical to sustain progress.

Future Directions

Emerging nanotechnologies enhance drug delivery to rare cancer sites2, while AI-driven molecular matching optimizes repurposing candidates. Collaborative models, exemplified by the ROADMAP initiative’s 94 repurposing projects5, underscore the importance of global data sharing and patient advocacy.

By prioritizing repurposing strategies, the oncology community can transform existing therapies into viable options for rare cancers, reducing development costs from $2.6B to under $300M per drug7 while saving years in approval timelines. However, sustained success requires systemic shifts in funding, regulation, and stakeholder collaboration.

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