Phase II study identifies potential biomarkers in HPV-positive anal cancer
MD Anderson News Release March 07, 2025
Cytotoxic chemotherapy is the standard of care for patients with unresectable and metastatic squamous cell carcinoma of the anal canal – a rare type of anal cancer often linked to human papillomavirus (HPV) infection. However, this treatment has limited efficacy, and patients have a poor prognosis. To address this, researchers led by Van Morris, M.D., conducted a Phase II trial to evaluate the combination of the anti-PD-L1 antibody atezolizumab and anti-VEGF antibody bevacizumab in 20 patients with unresectable and/or metastatic HPV-associated anal cancer. They also collected tissue biopsies from each participant before and after treatment to further analyze tumor-immune microenvironments. While the combination therapy was not superior to standard treatment, the biopsies identified chromosomal and transcriptomic markers that were linked to longer survival in patients treated with immunotherapy. The study provides critical insights into the tumor-immune microenvironment and identifies potential new biomarkers that could improve future treatment studies. Learn more in Clinical Cancer Research.
To our knowledge, this trial represents the most comprehensive translational work completed on prospective trials for patients with metastatic anal cancer receiving immunotherapy. It included analyses of paired biopsies which, historically, have not been routinely collected in previous clinical trials.