James Cassidy, MD, PhD, Chief Medical Officer at SpringWorks Therapeutics, discusses how Desmoid tumors affect children compared to adults.

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. Unlike typical cancers, these tumors do not metastasize, though they are locally aggressive.

As Dr. Cassidy explains, the tumors themselves as well as their presentation are similar regardless of age. The average age of onset for this disease is 30-40 years and, in adulthood, it predominantly affects women. But it can also occur in children, although no sex difference is observed. Since recurrence is common in Desmoid tumors and children start treatment at an earlier age, there is higher chance of having multiple rounds of recurrence in their lives. With each recurrence, there is also the increased likelihood the tumors will be more aggressive.

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

Dr. Ghanouni discusses FUS treatment of pediatric desmoid tumors at Stanford Medical Center.

Clinical Study Promotion: "Desmoid Fibromatosis during pregnancy: the effect of pregnancy on disease control and the effect of diagnosis on pregnancy histories." - Dott. Gronchi
A retrospective international observational multicenter study by Desmoid Foundation Italy

Desmoid tumors are unique tumors. Prognosis and recurrence rates are variable depends on gender, hormonal status, genetic factors, locations and types of treatments etc. For more detail, please watch out our new video.
Dr. Metin Çevener
Medicana International Hospital
Istanbul/Turkey

Followed by Chest Reconstruction Using the Stratos™ System and PTFE Patch.

There have previously been no randomized trials assessing systemic therapy activity for progressive desmoid tumors. Here, Maud Toulmonde, MD, of the Institut Bergonié, Bordeaux, France, discusses the aims, methods and results of the DESMOPAZ trial (NCT01876082). This French Sarcoma Group trial investigated the use of pazopanib vs. IV methotrexate/vinblastine in desmoid tumors. This interview was recorded at the American Society of Oncology (ASCO) 2018 Annual Meeting, held in Chicago, IL.

Bernd Kasper, MD, PhD, University of Heidelberg, Mannheim Cancer Center, Mannheim, Germany and Principal Investigator of the phase 3 DeFi clinical trial, discusses the impressive results of the trial, which evaluated nirogacestat in the treatment of desmoid tumors.

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.

As Dr. Kasper explains, nirogacestat is an oral, selective, small molecule, gamma-secretase inhibitor. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which may contribute to desmoid tumor growth.

Data from the DeFi trial were recently presented as a late-breaking oral presentation during a Presidential Symposium at the European Society for Medical Oncology (ESMO) Congress 2022. As Dr. Kasper states, the DeFi trial is a global, randomized (1:1), double-blind, placebo-controlled phase 3 trial evaluating the efficacy, safety and tolerability of nirogacestat in adult patients with progressing desmoid tumors.

The trial met its primary endpoint of improving progression-free survival (PFS), as assessed by blinded independent central review, demonstrating a statistically significant improvement for nirogacestat over placebo, with a 71% reduction in the risk of disease progression. The median Kaplan-Meier estimate of PFS was not reached in the nirogacestat arm and was 15.1 months in the placebo arm. Confirmed objective response rate (complete response + partial response) based on RECIST v1.1 was 41% with nirogacestat versus 8% with placebo. The complete response rate was 7% in the nirogacestat arm and 0% in the placebo arm. Nirogacestat demonstrated statistically significant and clinically meaningful improvements in patient-reported outcomes as well, which were key secondary endpoints of the study. Specifically, nirogacestat significantly reduced pain and other DT-specific symptoms, and also significantly improved physical/role functioning and overall health-related quality of life..

SpringWorks Therapeutics plans to submit a New Drug Application (NDA) to the FDA in the second half of 2022, which will be submitted for review under the FDA’s Real-Time Oncology Review (RTOR) program.