A new study demonstrated that targeting mutant KRAS may offer a promising new treatment option for appendix cancer. Researchers led by John Paul Shen, M.D., assistant professor, Saikat Chowdhury, Ph.D., instructor, and Ichiaki Ito, Ph.D., research scientist, all of Gastrointestinal Medical Oncology, demonstrated that KRAS inhibitors significantly reduced tumor growth, blocked key cancer signaling pathways and triggered cancer cell death in advanced preclinical models of appendiceal adenocarcinoma. In a clinical cohort of 15 patients treated with a variety of different KRAS drugs, all evaluable patients experienced a marked reduction in serum tumor markers – the most effective measure of response in appendix cancer.
“Appendiceal cancer has long lacked effective systemic treatment options, and patients urgently need new approaches,” Shen said. “I am very excited about the future prospects of KRAS inhibition. This study is just the first step.”