The Desmoid Tumor Research Foundation

Desmoid Tumor Awareness Month 2022 Comes To A Close

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Today is the final day of Desmoid Tumor Awareness Month 2022!

Over the last month, we have shared stories from #DesmoidSurvivors around the globe at different stages in their journeys with different perspectives on managing life with these rare tumors. Thank you to all of the patients who submitted their stories, and to all who shared them across social media. You have helped raise #DesmoidAwareness!

To mark the end of this year’s campaign, please enjoy this musical montage of the many faces of our community. If you are living with this disease, please know you are not alone in this fight. We encourage you to explore our Patient & Caregiver Resources.

In case you missed them, recordings of our special Desmoid Tumor Awareness Month Facebook LIVE stream events are available:

  •  “One Desmoid Tumor Patient’s Journey” with Jackie Kucskar  |  WATCH
  • “5 Mental Health Tools for Desmoid Patients” with Licensed Clinical Social Worker and Desmoid Patient Ashley Williams  |  WATCH
  • “Getting the Most Out of your Consultation and Treatment Plan” with Dr. Candace Haddox from Dana-Farber Cancer Institute  |  WATCH

You can help raise #DesmoidAwareness year-round by talking about your desmoid tumor journey to family and friends, as well as your physicians, and raising funds for desmoid tumor research around special occasions like birthdays, diagnosis anniversaries, holidays, and more. Start a Facebook fundraiser with one click or learn about other Fundraising options.

Stay up to date with the latest in desmoid tumor-related news from the DTRF by favoriting us on Facebook, following us on Twitter (@dtrfoundation) and Instagram (@dtrfoundation), and by signing up for our emails.

Thank you for making a difference this September!

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THANK YOU to our global desmoid tumor community of patients, care partners, researchers, and medical professionals, who attended and participated in the DTRF Together We Will Virtual Weekend. We are grateful to our platinum event sponsor, SpringWorks Therapeutics, for making the Weekend possible! #DTRFWeekend2022

Together we advanced research, connected, empowered ourselves, and fundraised for a cure, while celebrating our community and Desmoid Tumor Awareness Month!

  • 2022 DTRF International Desmoid Tumor Research Workshop: 160 dedicated physicians and researchers from more than 25 countries registered for our virtual 9th Annual DTRF International Desmoid Tumor Research Workshop! We’re honored to be able to host this important collaborative meeting each year, which helps steer the direction of desmoid tumor research and the Foundation’s funding priorities. Learn more about the annual DTRF International Desmoid Tumor Research Workshop.

If you couldn’t join us last weekend, here’s how you can access the recorded events:

  • 2022 DTRF Annual Patient Meeting Webinars 1, 2, & 3: The recordings of these educational events (including live Q&A) are available to view (along with transcripts) on our Patient Meeting Presentation/Media page.
  • 2022 DTRF RFA Virtual Challenge Ceremony: The ceremony was streamed LIVE on Facebook! Watch the recording of the entire ceremony. It’s sure to brighten your day! Thanks to the hard work of our RFA Challengers, and the generosity of other donors and sponsors, we’ve raised more than $130K, with donations still rolling in. Please note that your fundraising pages will stay open for a few months, so we encourage you to keep going! Or, start a Facebook fundraiser with one click.

The work continues to find better treatments and, one day, a cure! Once again, on behalf of the DTRF team, THANK YOU!

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*Update: Watch the 2022 DTRF Annual Patient Meeting presentations on the Nirogacestat results and the AL102 results. For all Patient Meeting recordings, visit Patient Meeting.*

BIG NEWS this Desmoid Tumor Awareness Month!

Presentations on the latest data from two promising clinical trials – both studying gamma secretase inhibitor drugs in desmoid tumors – were given this past weekend at the 2022 European Society for Medical Oncology (ESMO) conference in Paris, France.

PLEASE NOTE: Presentations tailored for the patient community on these two trials and their data will be given at the Annual Patient Meeting Webinar #2 (of 3) on Sept. 24th during the DTRF Together We Will Virtual Weekend. Register at no cost at DTRF.org/weekend, and bring your questions!

Both of these presentations have left us encouraged and hopeful. The interest in desmoid tumor research is higher than ever before and we are making significant progress in finding more effective and more manageable therapies. If you have fundraised for or donated to the DTRF, you have helped make this progress possible by supporting our patient outreach, advocacy, research, awareness, and scientific collaboration efforts. Give today.

Here are the details from the ESMO presentations:

  • NIROGACESTAT – Phase 3 DeFi Clinical Trial (SPRINGWORKS THERAPEUTICS)

On Saturday, Principal Investigator Bernd Kasper, MD, (University of Heidelberg, Maneheim) presented the latest data from the SpringWorks Therapeutics Phase 3 DeFi clinical trial for nirogacestat (a gamma secretase inhibitor) in desmoid tumor patients during the Presidential Symposium session of ESMO.

“Results showed a statistically significant improvement in progression-free survival in patients randomised to nirogacestat compared to the placebo group, with a 71% lower risk of disease progression on average. The response rate was also much higher – 41% in the nirogacestat arm and only 8% in the placebo arm; nearly one in ten patients (7%) showed a complete response with the agent.” 

– Bernd Kasper, MD, PhD, University of Heidelberg, Principal Investigator of the DeFi trial

For more information, see the SpringWorks’ press release

…and the ESMO article.

  • AL102 – RINGSIDE Phase 2/3 Clinical Trial (AYALA PHARMACEUTICALS)

On Monday, Prof. Robin Jones, MD, (The Royal Marsden) presented the latest data from the Ayala Pharmaceuticals RINGSIDE Phase 2/3 clinical trial for AL102 (a gamma secretase inhibitor) in desmoid tumor patients.

“Efficacy was demonstrated across all cohorts of Part A of the study, with early responses that deepened over time. The results also showed that AL102 was well-tolerated across all doses.” 

– Roni Mamluk, PhD, CEO of Ayala


For more information, read Ayala’s press release

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*Update: Watch the CTOS webinar on Desmoid Tumors*

The Connective Tissue Oncology Society (CTOS) – a prestigious organization of multidisciplinary sarcoma medical professionals – has named desmoid fibromatosis as the “Sarcoma of the Year” for their 2022 annual meeting this fall.

As part of this honor, the DTRF’s Executive Director Jeanne Whiting will present “Patients’ perspective: navigating the desmoid tumor journey” during a special webinar that will be aired by CTOS on Thursday, October 20th from 10am – 12pm ET.

The webinar will feature pre-recorded contributions from a patient advocate and 15-minute educational sessions on the pathobiology, pathology, and localized and systemic treatment options currently available for desmoid tumors. The webinar will finish with a dialogue between experts on current challenges.

The aim of the webinar is to bring all members to a good basic level of understanding of the “Sarcoma of the Year” in preparation for the CTOS Annual Meeting taking place a few weeks later in November.

Stay tuned for information about how to access this webinar.

We look forward to this opportunity to increase awareness about desmoid tumors and the patient experience with the global sarcoma community this fall.

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Registration is *NOW OPEN* at DTRF.org/weekend for the 2022 DTRF Together We Will Virtual Weekend (Sept. 23-25), kicking off our summer-long RFA Virtual Challenge virtual fundraiser!

As a reminder, the primary events of our signature September weekend will be held *virtually* once again. However, we also hope to host a special, outdoor, in-person community event at Fairmount Park in Philadelphia on the morning of Sunday, September 25th.

Plan to join your community this fall, and TOGETHER WE WILL…

  • CONNECT patients and care partners from across the globe at our International Virtual Community Networking Event.
  • EMPOWER OURSELVES with information from top physicians and researchers studying and treating desmoid tumors at the signature DTRF Annual Patient Meeting Webinars.
  • FUND A CURE all summer long with the #RFAVirtualChallenge community fundraiser! Go the #Distance4Desmoids and we’ll celebrate with a LIVE virtual ceremony to close the Weekend. Learn more.
  • ADVANCE RESEARCH by hosting some of the brightest researchers and medical professionals from across the globe at the DTRF International Desmoid Tumor Research Workshop. (*Researchers and medical professionals ONLY*)

Stay informed by adding us to your Favorites on Facebook and signing up to receive our emails.

Learn more about the Weekend events.

(#DTRFWeekend2022)

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On May 24, 2022, SpringWorks Therapeutics announced topline results from their Phase 3, double-blind, placebo-controlled trial called the DeFi trial. This trial evaluates the gamma secretase inhibitor, nirogacestat, for efficacy, safety and tolerability in adult patients with progressing desmoid tumors.

The DeFi trial’s primary endpoint was met by improving progression-free survival (PFS) for usage of nirogacestat compared to placebo, with a 71% reduction in the risk of desmoid tumor progression.

SpringWorks Therapeutics expects to present additional data at an upcoming medical conference as well as submit a New Drug Application (NDA) to the U.S. Food and Drug Administration in the second half of 2022.

From SpringWorks Therapeutics’ press release:

“‘Desmoid tumors are aggressive soft-tissue tumors that can lead to severe negative outcomes for patients, including long-lasting pain, disfigurement, and amputation. In rare cases, when vital organs are impacted, desmoid tumors can also be life-threatening,’ said Saqib Islam, Chief Executive Officer of SpringWorks. ‘Today’s announcement represents a significant milestone towards our goal of bringing the first approved therapy to the desmoid tumor community. We look forward to sharing the DeFi trial data with the FDA and to presenting detailed study results at a medical meeting later this year.’”

The DTRF would like to express our gratitude to members of the community who have participated in and are currently enrolled in clinical trials, including the DeFi trial. Your participation is advancing our understanding of desmoid tumors and bringing us closer to improved treatments for all desmoid tumor patients.