Anal cancer: research and therapeutic landscape
Retifanlimab is the only FDA‑labelled drug for squamous cell carcinoma of the anal canal.
Anal cancer is a rare malignancy associated with HPV infection and other risk factors. Current therapeutic options include surgery, radiation, chemotherapy, and emerging immunotherapies/targeted agents. A comprehensive landscape of genes recurrently implicated in this disease (12 genes including CD274, PDCD1, PIK3CA, etc.) and their druggability, clinical trials, public datasets, and funding has been compiled from Open Targets, ClinicalTrials.gov, openFDA, GEO, PubMed, and NIH RePORTER. Only one drug (Retifanlimab) is FDA‑labelled for anal cancer, though many others have indirect activity through broader indications. Six HPV‑E6/E7 cell‑therapy trials are registered, four of which remain active. Funding for FY2025 is $9.0 M, primarily from NCI. Key datasets include GSE254331 (98‑sample patient cohort) and GSE253560. The burden of disease in the U.S. is estimated at ~11,270 new cases per year, with a five‑year relative survival of 70.9 %.
Its FDA label confirms that the disease has an approved therapeutic, yet the majority of genomic targets remain unaddressed or unenrolled in trials, highlighting a therapeutic gap.
Evidence level: Sejtvonalas. Laboratóriumi sejtekben vizsgálták.
Related signals
- Clinical Outcomes, Patterns of Failure, and Salvage Therapies of a Large Modern Cohort of Patients With Anal Squamous Cell Carcinoma Treated With Definitive-Intent Intensity‑Modulated Radiation Therapy
- Treatment of Anal High-Grade Squamous Intraepithelial Lesions to Prevent Anal Cancer
- Advancements in Immunotherapy for Anal Cancer Treatment
- Background and Current Treatment of Squamous Cell Carcinoma of the Anus
- Genotyping and Cytology Co-Testing for Anal Cancer Screening: Exploratory Evaluation of Cumulative HPV Genotype Burden