Experimental and investigational drugs for the treatment of anal cancer.

INTRODUCTION: Squamous cell carcinoma of the anal canal (SCCA) is a rare malignancy, but its incidence rates have been increasing in the last decade. Studies have demonstrated that up to 47% of patients with locally advanced disease have high-risk features for treatment failure. The potential high rates of recurrence after standard chemoradiotherapy for locally advanced disease and the lack of established care for metastatic disease have created an urgent need for the evaluation of new drugs that will ultimately improve the efficacy of treatment.

AREAS COVERED: This review presents results of recent phase-I and -II clinical trials which evaluate novel therapeutic modalities. The review also describes the findings of comprehensive genomic profiling studies which provide insights for promising therapeutics.

EXPERT OPINION: HPV vaccination is underutilized in the United States and as a result, HPV-associated malignancies are likely to continue for several decades; however, pivotal breakthroughs may create a foundation for distinctive treatment approaches for other HPV-associated malignancies for which no other standard of care exists.