Dr Jonathan Trent comments on the use of nirogacestat alone or in combination in desmoid tumor at the 2022 ESMO Florida conference

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/

Modernizing the Treatment of Desmoid Tumors With Novel Gamma Secretase Inhibitors

Chair, Bernd Kasper, MD, PhD, Mrinal Gounder, MD, and Alessandro Gronchi, MD, discuss desmoid tumors in this CME activity titled “On the Cusp of Change: Modernizing the Treatment of Desmoid Tumors With Novel Gamma Secretase Inhibitors.” For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/JST865. CME credit will be available until December 29, 2023.

Bernd Kasper, MD, PhD, University of Heidelberg, Mannheim Cancer Center, Mannheim, Germany, gives an overview of desmoid tumors and how to diagnose them.

As Dr. Kasper explains, desmoid tumors 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. These benign tumors are notoriously recurrent, especially with surgical removal, and each recurrence tends to be more aggressive than the previous tumor.

Currently, there is no approved targeted therapy for desmoid tumors. However, positive data from the DeFi trial, a phase 3 trial evaluating the efficacy, safety and tolerability of nirogacestat in adult patients with progressing desmoid tumors were recently presented as a late-breaking oral presentation during a Presidential Symposium at the European Society for Medical Oncology (ESMO) Congress 2022. Additionally, SpringWorks Therapeutics plans to submit a New Drug Application (NDA) to the FDA in the second half of 2022.