Desmoid tumors are rare tumors that tend to grow into the surrounding area and not spread to distant parts of the body. These tumors aren’t typically life-threatening but can cause significant pain and lead to a poor quality of life. Desmoid tumors can affect different areas of the body and cause various symptoms. Surgery to remove desmoid tumors is not recommended, as they tend to grow back quickly after they are removed.

Until 2023, no specific therapies were approved for treating people living with desmoid tumors. Nirogacestat (Ogsiveo) is the first medicine approved by the U.S. Food and Drug Administration (FDA) for treating desmoid tumors. Scientists continue to research several other medicines to help improve the treatment of desmoid tumors.

In this article, we’ll explain how these new medicines work and mention some of the older treatments for people with desmoid tumors. We’ll also cover why it’s important to talk to a desmoid tumor (or sarcoma) specialist before starting treatment or joining a clinical trial.

Nirogacestat

The FDA approved nirogacestat in November 2023 for people living with desmoid tumors.

Before the FDA can approve a new drug, the treatment first has to go through clinical trials. These well-regulated studies help researchers determine whether the drug is safe and effective. In a clinical trial for nirogacestat, people with quickly growing desmoid tumors were treated with either nirogacestat or a fake pill (called a placebo). People treated with nirogacestat experienced a significant improvement in progression-free survival (meaning the tumor did not become worse) and reduced pain from the desmoid tumor. In 41 percent of people receiving nirogacestat, the desmoid tumor shrank substantially, whereas only 8 percent of people on placebo had some shrinkage of the desmoid tumor.

Most of the people receiving nirogacestat experienced nausea, vomiting, and other stomach issues, which were controlled by using anti-nausea and anti-diarrhea pills. Young females receiving nirogacestat had hot flashes and stopped menstruating, but these symptoms disappeared completely when the medicine was stopped.

Treatments Currently in Clinical Trials

Researchers continue to run clinical trials to try new substances that might treat desmoid tumors. Some trials are looking at old medicines that are used to treat other cancers and may help in the treatment of desmoid tumors, while other trials look at entirely new drugs. Clinical trials need people with desmoid tumors to participate so researchers can learn more about how these medications work.

If you’re interested in joining a clinical trial, talk to your doctor or oncologist (cancer specialist). They can recommend studies near you that you may qualify for.

AL102

AL102 is a newer medicine that also blocks the enzyme gamma-secretase. It works in the same way as nirogacestat, but there are some differences in the dose and the side effects. The clinical trial for AL102 recently finished enrolling participants, and the results are currently being processed. The FDA has not yet approved this medicine for use in people with desmoid tumors but might in the future.

Tegavivint

Tegavivint is a new medicine that attempts to block a protein pathway in the body that would otherwise help the desmoid tumor to grow. This targeted therapy is being studied in a clinical trial recruiting young people up to the age of 30 years.

The trial will enroll young people who are experiencing pain or rapid growth in their desmoid tumors. The complete list of participating criteria is listed on Clinicaltrials.gov. If you’re interested in participating in this trial, talk to your oncologist.

Cryoablation With Nirogacestat

Cryoablation is a technique that uses extremely cold temperatures to freeze and destroy tumors. In this technique, a radiologist will place a wire into the tumor and then use nitrogen gas to freeze the tumor. One clinical trial is looking at the option of treating desmoid tumors where cryoablation will be done along with the administration of nirogacestat. In this study, the participants:

• Take nirogacestat for three cycles (each cycle is 28 days)
• Have a single cryoablation treatment
• Continue taking nirogacestat for up to 26 cycles (24 months total)

To learn more about this trial, you can visit Clinicaltrials.gov. People with symptomatic desmoid tumors (tumors that are painful or grow fast) should talk to their oncologists to see how they can participate.

High-Intensity Focused Ultrasound

Clinical trials are also looking at the use of high-intensity focused ultrasound (HIFU) as another way to treat people with desmoid tumors. Ultrasound uses sound waves to create images of the soft tissues and organs in your body.

HIFU uses intense, high-frequency sound waves to heat desmoid tumor tissue and burn it. Doctors can focus these sound wave beams on a specific area and prevent injury to the organs surrounding the desmoid tumor. This technique is not currently being studied in clinical trials in the United States. However, a few studies done in China showed that this technique is a possible alternative for those with desmoid tumors.

Sorafenib

The FDA has approved sorafenib (Nexavar) for the treatment of kidney, thyroid, and liver cancers. It belongs to a class of medicines called tyrosine kinase inhibitors (TKIs). Sorafenib is a pill that works by blocking proteins that help desmoid tumors grow blood vessels around themselves. Once these blood vessels are blocked, the tumor cells die due to starvation.

Sorafenib was studied in a clinical trial in people with symptomatic desmoid tumors. Those who received sorafenib had a significant improvement in pain and saw a longer period where the tumor did not progress. Overall, 33 percent of people who took sorafenib had their tumors shrink. It’s also worth noting that 20 percent of those who took the placebo experienced a shrinkage. This is an expected behavior with desmoid tumors where spontaneous shrinkage can be seen in up to 20 percent of people without any treatment.

Although the FDA has not officially approved sorafenib for desmoid tumors at this time, national guidelines and several sarcoma doctors support its use to treat desmoid tumors.

Pazopanib

Pazopanib (Votrient) is another TKI approved by the FDA for treating people with soft tissue sarcomas (cancers) and kidney cancer. It was studied in a small clinical trial for treating people with desmoid tumors. In the study, people with symptomatic desmoid tumors were either treated with chemotherapy (vinblastine and methotrexate) or with pazopanib. The results of the study showed that the use of pazopanib was not only able to prevent growth in desmoid tumors in most participants but also reduced the tumor volume in 37 percent of them.

Talk to Your Doctor About Desmoid Tumor Treatments

If you’re diagnosed with a desmoid tumor, always seek the opinion of a sarcoma specialist (a cancer doctor specializing in sarcoma and desmoid tumors). Sarcoma centers in your state can be located on Sarcoma Alliance website. A sarcoma specialist is more experienced in managing desmoid tumors and can guide you and your local oncologist on the best treatment plan for you.

Participating in clinical trials can help researchers better understand desmoid tumors and may also offer you an opportunity to receive treatment. However, remember that participation in a clinical trial does not guarantee success or personal benefits. You can weigh the pros and cons of participating in a clinical trial with your doctor.

Find Your Team

MyDesmoidTumorTeam is the social network for people with desmoid tumors and their loved ones. On MyDesmoidTumorTeam, members come together to ask questions, give advice, and share their stories with others who understand life with desmoid tumors.

Are you interested in trying a new treatment for desmoid tumors? Have you participated in or talked to your doctor about a clinical trial? Share your experience in the comments below, or start a conversation by posting on your Activities page.