Treatment Options for Desmoid Tumors, Including Nirogacestat in Children

Fariba Navid, MD, Associate Professor of Clinical Pediatrics, Keck School of Medicine of USC, announces her involvement in a trial for nirogacestat as a treatment for Desmoid tumors in pediatric patients.

Desmoid tumors are benign tumors that can grow anywhere on the body into surrounding healthy tissues, including joints, muscle, and viscera. These rare tumors can cause significant pain, internal bleeding, and reduced mobility.

The most common course of action for Desmoid tumors - particularly those in non-lethal areas - is to “wait and watch” as these tumors can shrink on their own. Surgery to remove the tumors may also be used. However, as Dr. Navid says, surgery is not always ideal as it has a high recurrence rate. As Dr. Navid explains, the recommended treatment for patients with Desmoid tumors who cannot undergo surgery is the use of systemic therapy.

Dr. Navid explains in this video that a phase 3 clinical trial of nirogacestat for the treatment of adult patients with Desmoid tumors was recently completed. Official results of that trial are to come. Given the success of nirogacestat in adults, a separate trial of the drug for the treatment of children and adolescents was started. Dr. Navid serves as the Study Chair for this pediatric study, which is currently recruiting patients.

For more information about Desmoid tumors and other rare tumors, visit checkrare.com/cancers/