Desmoid Tumor: Latest Research

Approved by the Cancer.Net Editorial Board, 11/2022

ON THIS PAGE: You will read about the scientific research being done to learn more about desmoid tumors and how to treat them. Use the menu to see other pages.

Doctors are working to learn more about desmoid tumors, ways to prevent them, how to best treat them, and how to provide the best care to people diagnosed with them. The following areas of research may include new options for patients through clinical trials. Always talk with your doctor about the best diagnostic and treatment options for you.

  • Targeted therapy. As explained in Types of Treatment, certain targeted therapies, such as sorafenib and pazopanib, are being used to treat desmoid tumors. This is an active area of research for desmoid tumors. One promising targeted therapy starting to be researched for the treatment of adults with aggressive desmoid tumors is a small molecule, gamma-secretase inhibitor called nirogacestat. Drugs called “small molecule drugs” can block the process that helps tumor cells multiply and spread.

    Another targeted therapy beginning to be researched is tegavivint. This drug may bind to a protein called TBL1. TBL1 binds to beta-catenin, which the CTNNB1 gene provides instructions for making. The goal is to inhibit excess beta-catenin caused by a CTNNB1 gene mutation.

  • New hormone therapies. Because estrogen may play a role in desmoid tumors, 1 study is exploring the use of endoxifen (Z-Endoxifen HCl) for treatment in tumors that have not responded to standard hormone treatments. This experimental drug has been shown to slow or stop the growth of other estrogen-linked cancers.

  • High-frequency ultrasound. This emerging, noninvasive therapy uses focused ultrasound beams to target and destroy a desmoid tumor without harming nearby tissue. It could offer an alternative to surgery in some patients. Although high-frequency ultrasound is being used to treat desmoid tumors in a few medical centers, clinical trial research on its effectiveness and long-term effects is just beginning.

  • Palliative and supportive care. Clinical trials are underway to find better ways of reducing symptoms and side effects of current desmoid tumor treatments to improve comfort and quality of life for patients.

Looking for More About the Latest Research?

If you would like more information about the latest areas of research in desmoid tumors, explore these related items that take you outside of this guide:

The next section in this guide is Coping with Treatment. It offers some guidance on how to cope with the physical, emotional, social, and financial changes that a tumor and its treatment can bring. Use the menu to choose a different section to read in this guide.