What’s New in Gastrointestinal Stromal Tumor Research?

Important research on gastrointestinal stromal tumors (GISTs) is going on in many university hospitals, medical centers, and other institutions around the world. Scientists are learning more about what causes the disease and how best to treat it. There has been a great deal of progress in recent years, especially in treating GISTs. 

Targeted therapy drugs

As researchers have come to understand the genetic changes that cause these tumors, they’ve been able to use newer treatments to target these changes.

Doctors know targeted treatments like imatinib (Gleevec), sunitinib (Sutent), and regorafenib (Stivarga) often work, but they still aren’t sure exactly how and when to give them to make them most effective. For example, should these types of drugs be given after surgery to all patients, even those with very small tumors? How long should drug treatment be continued? These and other questions are now being studied in clinical trials.

Other drugs that target the KIT or PDGFRA proteins are also being studied for use against GISTs. Some of these, such as sorafenib (Nexavar), nilotinib (Tasigna), dasatinib (Sprycel), pazopanib (Votrient), and ponatinib (Iclusig), have helped some patients in early studies. Other, newer drugs that target these proteins, such as crenolanib and BLU-285, are also being studied.

Many other drugs that target different proteins involved in tumor cell growth are now being tested as well.


Immunotherapy is the use of medicines to boost the body’s own immune response to help fight the cancer.

For example, immune system cells normally have proteins that act as checkpoints to keep them from attacking other healthy cells in the body. Cancer cells sometimes take advantage of these checkpoints to avoid being attacked by the immune system.

Newer drugs called immune checkpoint inhibitors work by blocking these checkpoint proteins, which can boost the immune response against cancer cells in the body. These drugs have been shown to be helpful against many types of cancer in recent years. Some of these drugs, such as such as nivolumab (Opdivo) and ipilimumab (Yervoy), are now being studied for use against GISTs.

Other types of immunotherapy are now being explored as well.

People with GISTs who are no longer responding to standard treatments may want to ask their doctor about clinical trials of these newer types of treatments.

The American Cancer Society medical and editorial content team

Our team is made up of doctors and oncology certified nurses with deep knowledge of cancer care as well as journalists, editors, and translators with extensive experience in medical writing.

National Comprehensive Cancer Network (NCCN). NCCN Clinical Practice Guidelines in Oncology: Soft Tissue Sarcoma. V.2.2017. Accessed at  www.nccn.org/professionals/physician_gls/pdf/sarcoma.pdf on April 17, 2017.

Szucs Z, Thway K, Fisher C, et al. Promising novel therapeutic approaches in the management of gastrointestinal stromal tumors. Future Oncol. 2017;13:185-194.

Last Medical Review: May 17, 2017 Last Revised: May 17, 2017

American Cancer Society medical information is copyrighted material. For reprint requests, please see our Content Usage Policy.