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The Desmoid Tumor Research Foundation

DTRF Awarded The 2021 “Abbey S. Meyers Leadership Award”, A Nord “Rare Impact Award”

News

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*Update: Watch DTRF Co-Founders accept the Abby S. Meyers Leadership award on behalf of the Foundation at NORD’s 2021 Rare Impact Award Ceremony (beginning at 55:45)

We are honored to announce that the DTRF and its Co-Founders Jeanne Whiting and Marlene Portnoy will be the recipients of the 2021 Abbey S. Meyers Leadership Award from the National Organization for Rare Disorders (NORD)! 

The DTRF is being recognized for its exceptional work benefiting the rare disease community. Jeanne and Marlene are grateful to NORD for this honor, which would not be possible without our supportive community and donors. 

Read the press release.

Marlene and Jeanne will accept this award at the Rare Impact Awards at *7pm ET on Monday, June 28th*. Celebrate by joining us virtually for this special ceremony!

The Rare Impact Awards are part of NORD’s annual “Living Rare, Living Stronger” Patient & Family Virtual Forum from June 26-27. This is an annual patient-focused conference, providing patients and families with practical tools for living their best lives with rare diseases. For more information about attending the conference and award ceremony, please visit https://rarediseases.org/living-rare-forum/.

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The DTRF is pleased to announce that our first grant recipient of 2019 is the Broad Institute of MIT and Harvard for their proposal entitled “Using genome-scale CRISPR screening to create a Desmoid Tumor Dependency Map.”

This project is directly tied to one of DTRF’s five 2019 research priorities we announced at the end of last year: “Use existing data, in addition to CRISPR data, to identify new therapies for desmoid tumors.”

While some aspects of individual desmoid tumors are shared between patients, each tumor has unique molecular and clinical characteristics. This means that selecting the right therapies for future patients with desmoid tumors requires a special type of map. This map, which would be called a “dependency map,” would enable a clinician in the future to know therapy(ies) to prescribe based on the genetic characterization of that individual patient’s tumor.

The project aims to produce freely available foundational resources and develop a pilot version of a desmoid tumor dependency map that the entire scientific community can use. They will first test a large number (>6,000) of existing drugs that might be repurposed for desmoid tumors. It’s likely that the most promising drugs for desmoid tumors likely don’t yet exist. It is for this reason that the project will focus on using new genetic tools, including a special type of molecular “scissors” called CRISPR/Cas9, to disable every gene in the genome in a systematic fashion and look for evidence of desmoid tumor cell killing in the laboratory. 

These experiments will prioritize the best targets to ensure drug companies know which targets to work on for desmoid tumors and ensure they include desmoid tumors in future drug discovery programs that might otherwise be focused on other tumor types. 

Your fundraising and donations have made this exciting grant possible! THANK YOU for helping us accelerate our journey to a cure for desmoid tumors.

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DTRF’s Efforts Enable Largest Clinical Study On Desmoid Tumors, And New England Journal Of Medicine Publishes Breakthrough Study Results

(New York, NY) – Thursday, December 20th, 2018 – The efforts of The Desmoid Tumor Research Foundation (DTRF) enabled the largest clinical study ever in desmoid tumors. The New England Journal of Medicine just released the breakthrough study results in their December 20, 2018 article, “Sorafenib for Advanced and Refractory Desmoid Tumors.”

To date, there are no approved drugs for desmoid tumors. This international study, a first of its kind, showed that patients treated with sorafenib reduced the risk of tumor growth or death by 7-fold, which are groundbreaking results.

In 2010, when the DTRF heard news of promising results from a small pilot study conducted by Dr. Mrinal Gounder at Memorial Sloan Kettering Cancer Center with 26 desmoid tumor patients responding to sorafenib, they wasted no time in encouraging him to set a Phase III clinical trial in motion. Dr. Gounder then began a series of conversations with the FDA and the National Cancer Institute to start an international trial in this rare cancer with the purpose of getting sorafenib approved for patients with desmoid tumors.

It took over three years of efforts to finally open this study as there were countless barriers to conducting a study in a rare cancer. The Phase III study, which opened in 2014 across the United States and Canada, accrued 87 patients, a remarkable number for an ultra-rare cancer. Due to the tireless efforts of the DTRF founders, Jeanne Whiting and Marlene Portnoy, the The DTRF played a significant role in the recruitment for the trial, filling it in half the time expected–
a record time of just under 17 months.

This enabled the first clinical trial of its kind on desmoid tumors, and one of the largest Phase III clinical trials on a rare disease, to take place. Prior to this study, many cancer experts had declared that conducting a trial like this would be impossible or futile in desmoid tumors. The implications of this study are immense as researchers now try to identify how research in desmoid tumors can be applicable to fighting other types of cancers, particularly colon cancer which is a leading cause of death worldwide.

The DTRF’s efforts also played a role in moving forward another investigational treatment that was “stuck” on the shelves of a large pharmaceutical company after being deprioritized for business reasons. Nirogacestat, a gamma secretase inhibitor, showed encouraging results in a Phase II study sponsored by the NCI. However, the drug’s clinical development program was halted by the pharmaceutical company. The DTRF advocated for nirogacestat because it showed such promise and patients with desmoid tumors had limited treatment options. SpringWorks Therapeutics was then conceived by Pfizer as an innovative way to advance investigational therapies that may hold significant promise for underserved patients.

In just a year since the company’s formation, nirogacestat received fast-track designation from the FDA and SpringWorks is preparing to start a Phase III study in adult patients with desmoid tumors in the first quarter 2019. Once again, when nirogacestat showed promising results in an early study, the two fearless women who co-founded the DTRF advocated to move the drug forward. They galvanized the patient community they had built and filled up the Phase II trial in record-time, enabling SpringWorks to be able to spring into action with a Phase III trial to deliver treatments to patients who are in great need and without alternatives.

With the support of the DTRF’s efforts, there also will be two additional clinical trials on desmoid tumors in 2019. For a rare disease with an incidence rate of 5-6 per million, these are remarkable accomplishments.

For more information on The Desmoid Tumor Research Foundation please visit www.dtrf.org.

About The Desmoid Tumor Research Foundation

The DTRF was founded in 2005 with the mission of facilitating and funding desmoid tumor research and supporting patients with information. The mission of The Desmoid Tumor Research Foundation is to aggressively fund research to accelerate the development of improved therapies, and ultimately find a cure for desmoid tumors. The DTRF collaborates with dedicated researchers and clinicians worldwide to improve the lives of patients through education, awareness and support. Every year the foundation has gained momentum in its progress and has seen new clinical trials, emerging therapies and important new discoveries about the disease come to fruition.

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