SUMMARY
Tanios S. Bekaii-Saab, M.D., is a medical oncologist who also conducts clinical and translational research to develop anticancer agents, particularly for gastrointestinal cancers.
Dr. Bekaii-Saab's research focuses on agents that target the multiple facets of cancer. These targets include the genetic and epigenetic drivers of cancer, the immune milieu, and the feeding microenvironment, which is also known as the tumor microenvironment. Dr. Bekaii-Saab conducts numerous clinical trials on a variety of gastrointestinal cancers, including colorectal cancer, biliary tract cancer, gallbladder cancer, liver cancer, gastric cancer and pancreatic ductal adenocarcinoma.
His work includes two discoveries as co-inventor of a pan-histone deacetylase (pan-HDAC) inhibitor called AR-42 and an anti-PD-1 vaccine called PD-Vaxx. Both are being studied in clinical trials and have resulted in licensing opportunities under multiple patents. Pan-HDAC inhibitors are a class of drugs that inhibit all types of HDACs. Anti-PD-1 vaccines use a vaccine to stimulate the immune system to produce antibodies that block the PD-1 protein.
Dr. Bekaii-Saab also is a leading clinician-scientist in the Alliance for Clinical Trials in Oncology, which is a National Cancer Institute (NCI)-supported cooperative research group.
Focus areas
Practice-changing clinical trials. Dr. Bekaii-Saab collaborates extensively with other scientists and industry partners to design and execute innovative clinical trials, including many first-in-human studies. His work in the MOUNTAINEER study on tucatinib and trastuzumab in HER2-positive colorectal cancer helped lead to the first U.S. Food and Drug Administration (FDA)-approved anti-HER2 regimen for metastatic colorectal cancer, representing an important new treatment option. He also contributed to the pivotal study that led to regulatory approval of liposomal irinotecan for pancreatic cancer and fruquintinib for refractory metastatic colorectal cancer. He conducted several major studies that led to incorporating new agents into treatment guidelines of the National Comprehensive Cancer Network (NCCN). These include adagrasib in pancreatic cancer and adagrasib with cetuximab in colorectal cancers that bear a KRAS G12C mutation.
Dr. Bekaii-Saab's work on optimizing the dose of regorafenib in refractory metastatic colorectal cancer led to changes in guidelines at both the NCCN and the European Society for Medical Oncology (ESMO). In addition, Dr. Bekaii-Saab's research on bile duct cancer (cholangiocarcinoma) includes major efforts in molecularly targeted therapy and drug discovery. Dr. Bekaii-Saab is especially interested in fibroblast growth factor receptor (FGFR) fusions, which are found in intrahepatic cholangiocarcinoma. His work with other Mayo Clinic investigators and industry partners is advancing clinical development of FGFR inhibitors, and his work has contributed to regulatory approval of infigratinib and pemigatinib.
- Novel therapeutics in pancreatic cancer. Dr. Bekaii-Saab's research on pancreatic cancer focuses on cancer stem cells, DNA repair and the immune milieu. He conducted a clinical and translational trial of rucaparib, which belongs to a class of agents known as poly-ADP ribose polymerase (PARP) inhibitors, in sequential combination with liposomal irinotecan. This trial examined tumors with DNA repair defects. Dr. Bekaii-Saab also played instrumental roles in developing strategies to optimize the use of immune-based therapies in pancreatic cancer.
- Personalized targeted strategies in colorectal cancer. Dr. Bekaii-Saab studies noninvasive liquid biopsies to determine the molecular makeup of colorectal cancer cells. One significant clinical trial of a liquid biopsy is COLOMATE, which stands for Colorectal Cancer and Liquid Biopsy Screening Protocol for Molecularly Assigned Therapy. This liquid biopsy platform screens for genetic mutations in people with colorectal cancer that is metastatic or that can't be removed with surgery and provides recommendations for molecularly assigned therapies.
Significance to patient care
Dr. Bekaii-Saab hopes that his research will lead to better tools to tackle the highly complex and heterogeneous nature of gastrointestinal cancers. Through a large emphasis on precision medicine and individualized care, many novel anticancer drugs are proving to be effective in subsets of cancer. Dr. Bekaii-Saab's collaborative work has the potential to accelerate the availability of new therapies for people with many types of gastrointestinal cancer.
Professional highlights
- National Cancer Institute:
- Co-chair, Hepatobiliary Task Force, GI Steering Committee, 2020-present.
- Clinical Investigator Team Leadership Award, 2011-2013.
- Editorial board member, Journal of the National Cancer Institute, 2017-present.
- David F. and Margaret T. Grohne Professor of Novel Therapeutics for Cancer Research I, Mayo Clinic, 2024.
- Editorial board member, Journal of the National Comprehensive Cancer Network, 2013-2016.
- Participant, Leadership Development Program, American Society of Clinical Oncology, 2012-2013.