The Sant Pau Research Institute (IR) will launch a new Phase I/IIB clinical trial for patients with Fanconi anemia diagnosed with head and neck cancer. The study, known as the AFAN trial (EU CT No.: 2024-511477-29-00), is led by Dr. Jordi Surrallés, head of the Cancer Predisposition Syndromes Group, scientific director of the IR Sant Pau, and professor in the Department of Genetics and Microbiology at the UAB. The trial will evaluate the safety and efficacy of the drug afatinib in patients with Fanconi anemia and locally advanced, unresectable, or metastatic squamous cell carcinoma located in the oral cavity, oropharynx, hypopharynx, or larynx, making it the first clinical trial worldwide for this indication. The study, approved by the Spanish Agency for Medicines and Medical Devices (AEMPS) last August, began recruiting patients this October, with a process expected to last approximately four years.
This project is the result of joint research by the Sant Pau Research Institute (IR), the Autonomous University of Barcelona (UAB), and the Biomedical Research Networking Center (CIBER), which led to the discovery of a new therapeutic use for afatinib, a drug currently administered to treat certain types of lung cancer in the general population. This project is, therefore, a drug repurposing initiative, transferring a treatment from a common disease to a rare disease with no therapeutic alternatives.
Fanconi anemia is a rare genetic disorder caused by a defect in DNA repair. It leads to bone marrow malfunction, and patients are particularly prone to developing head and neck tumors at a very young age. Due to their genetic defect, cancer patients cannot undergo chemotherapy and therefore have no effective treatments beyond surgery, making this the leading cause of mortality among the adult population affected by this disease.
The new use of afatinib developed by researchers from these institutions has led to a patent recently granted in Europe and pending in the United States. Additionally, as a rare disease, the project has received afatinib’s designation as an Orphan Drug by the EMA, expediting the development timeline for this new drug use.
To carry out this clinical trial, the crucial collaboration of Boehringer Ingelheim, the current owner and distributor of afatinib, has been secured, and they will provide the drug to be administered to patients during the study. This advancement marks a significant step forward in the treatment of patients with Fanconi anemia, who have developed this type of cancer and currently lack therapeutic alternatives. To ensure the success of the trial, patients will be treated not only at Sant Pau Hospital but also at Hannover University Hospital in Germany, where EMA approval is also expected soon.
The AFAN research team, led by Dr. Jordi Surrallés, includes Dr. Georgia Anguera, AFAN’s principal investigator, and Dr. Oscar Gallego (oncologists from the Medical Oncology Service at Sant Pau Hospital), Drs. Núria Berga and Maria Estela Moreno from the hospital’s Pharmacy Service, with the collaboration of Dr. Javier León from the Otolaryngology Service and Dr. Ramón García-Escudero from the Research Institute at Hospital 12 de Octubre, who will perform genomic analyses associated with the trial.
The institutions wish to thank all the entities that have made possible all the preclinical research and the current AFAN clinical trial, including industry partners like Boehringer Ingelheim, who support cutting-edge R&D, as well as the various institutions and patient associations that have provided financial support for the clinical research project, such as the Carlos III Health Institute (ICI22/00076, funded by the European Union – NextGenerationEU) and the Fanconi Anemia Research Fund (https://fanconi.org/).
