The Sant Pau Research Institute has initiated a Phase I/IIB clinical trial (AFAN trial) to evaluate the efficacy and safety of afatinib in patients with Fanconi anemia who have developed head and neck squamous cell carcinoma. This pioneering study addresses a critical gap in treatment options for this vulnerable population, who cannot tolerate standard chemotherapy.
Addressing Unmet Needs in Fanconi Anemia
Fanconi anemia, a rare genetic disorder characterized by defective DNA repair, predisposes individuals to early-onset head and neck cancers. Traditional chemotherapy is often not an option due to the genetic defect, leaving surgery as the primary treatment. However, surgery alone is often insufficient, making head and neck cancer the leading cause of mortality in adult Fanconi anemia patients.
Afatinib: A Repurposed Therapeutic Approach
The AFAN trial is based on the discovery of a new therapeutic application for afatinib, a drug already approved for treating certain types of lung cancer. Researchers from the Sant Pau Research Institute, the Universitat Autònoma de Barcelona (UAB), and the Centre for Biomedical Research Network (CIBER) identified its potential in treating head and neck tumors in Fanconi anemia patients. This drug repositioning strategy offers a promising alternative for a patient group with limited therapeutic options. The new use of afatinib has resulted in a patent recently granted in Europe and pending in the United States. The EMA has granted afatinib Orphan Drug designation for this indication, potentially accelerating its development.
Trial Design and Collaboration
The AFAN trial (EUCT No.: 2024-511477-29-00) is a Phase I/IIB study that will enroll patients with Fanconi anemia and locally advanced, unresectable, or metastatic squamous cell carcinoma of the oral cavity, oropharynx, hypopharynx, or larynx. The trial aims to assess the safety and efficacy of afatinib in this specific patient population. Recruitment began in October and is expected to last approximately four years.
Boehringer Ingelheim, the manufacturer of afatinib, is a key collaborator, providing the drug for the trial. The study will be conducted at Hospital Sant Pau and Hannover University Hospital, with plans to expand to other sites pending EMA approval. The research team is led by Dr. Jordi Surrallés and includes oncologists, pharmacists, and researchers from various institutions.
Significance and Future Implications
This clinical trial represents a significant step forward in the treatment of head and neck cancer in Fanconi anemia patients. If successful, afatinib could become the first effective therapy for this high-risk population, offering a much-needed alternative to surgery and improving survival rates. The project has received financial support from the Instituto de Salud Carlos III and the Fanconi Cancer Foundation.
