BRAF-Targeted Therapy Shows Promise for BRAF-Mutant Appendix Cancer

New research from MD Anderson Cancer Center, led by Dr. John Paul Shen – a member of ACPMP’s Medical Advisory Board – finds that BRAF-mutant appendix cancer responds to BRAF-targeted therapy. The study was published in npj Precision Oncology.

Why this research was needed

Appendiceal adenocarcinoma is rare, and treatment options are limited. Historically, it has been treated with the same chemotherapy used for colorectal cancer, even though there is little data showing this benefits appendix cancer patients. Oncologists have had very little evidence to guide treatment choices.

What the researchers found

The team compared the molecular profiles and clinical features of tumors from patients with BRAF-mutant appendix cancer and BRAF-mutant colorectal cancer. They found important differences:

  • Appendix cancer typically spreads to the lining of the abdomen (the peritoneum), while colorectal cancer usually spreads to the liver or lungs.
  • BRAF mutations were linked to a particularly poor prognosis in colorectal cancer, but not in appendix cancer.
  • Associations seen in colorectal cancer (with female sex, mucinous features, and higher grade) were not found in appendix cancer.

Most importantly, the study showed for the first time that BRAF-mutant appendix cancer responds to BRAF-targeted therapy, with an 80% disease control rate and a median progression-free survival of 7.1 months – better than traditional chemotherapy in these patients.

Why it matters

These findings highlight a promising treatment strategy for a rare group of patients and add to growing evidence that appendix cancer needs its own research and treatment approaches. Tumor genomic sequencing can identify whether a patient’s cancer carries a BRAF mutation; patients can ask their care team whether sequencing is right for them.

Read the study

Pattalachinti VK, et al. BRAF mutant appendiceal adenocarcinoma differs from colorectal cancer but responds to BRAF-targeted therapy. npj Precision Oncology. 2025;9:38. Read the full article.

Explore more studies in our Published Research library.

This summary is for general information only and is not medical advice. Please talk with your care team about what this research may mean for you.