The Desmoid Tumor Research Foundation

DTRF Announces The First Publicly Available Desmoid Tumor Immortal Cell Line

Research

by

We are thrilled to announce that through our research at Broad Institute of MIT and Harvard and partnership with Pattern.org, the first publicly available desmoid tumor immortal cell line has been generated and is now available to researchers worldwide.

Cell lines are critical for researchers to learn more about the disease and in testing new therapeutic options. The availability of this cell line is an initial, but very significant milestone as we work to provide verified and standardized resources to our research community.
We thank all of our patients who have consented to donate tissue through Pattern.org as you are instrumental in helping expedite the development of improved therapies and ultimately find a cure for desmoid tumors. If you have an upcoming surgery and would like to donate, please visit our Donate Your Tissue page for more information.

by

SpringWorks Therapeutics has announced FDA acceptance and priority review of the New Drug Application (NDA) for nirogacestat, their investigational gamma secretase inhibitor for the treatment of adults with desmoid tumors.

“The acceptance of our NDA for nirogacestat with Priority Review represents a significant milestone in our ambition to provide the first approved therapy for patients with desmoid tumors. We look forward to working closely with the FDA during the review process and remain focused on ensuring that we are well-positioned to expeditiously serve the desmoid tumor patient and the physician communities following approval.”

Saqib Islam, CEO, SpringWorks Therapeutics

Read the press release

What This Means

This announcement brings our community one step closer to an FDA-approved therapy for desmoid tumors. The FDA now has until August 27, 2023 to complete the review process, which will lead to a decision of whether nirogacestat will receive FDA approval. This is very exciting!

Related News

The DeFi phase 3 trial results that lead to this NDA were published in The New England Journal of Medicine. The publication and NDA are remarkable accomplishments that demonstrate the power of collaboration between patient groups, researchers and industry.

Read DTRF Press Release.
Read “Nirogacestat, a γ-Secretase Inhibitor for Desmoid Tumors” on NEJM.org.

by

On the heels of another successful Desmoid Tumor Awareness Month, we are thrilled to formally announce The Desmoid Tumor Research Foundation Grant Awards for 2022! 

The proposals from these institutions offer the potential for significant advancements in desmoid tumor research and align with the research priorities set by our Scientific Advisory Board. For more information, visit DTRF-Awarded Grants.

Funding for these critical projects would not be possible without the continued support of our community and donors – YOU! We would like to extend an additional special thank you to the families who have helped fund some of these important projects. 

The awards below are contingent upon a mutually agreed upon grant agreement.

To mark this exciting announcement, please consider sharing this news and/or making a donation today. 

  • Sameer Rastogi, MD, Aiims- All India Institute of Medical Science,New Delhi, “To see the early effect of sorafenib on cognition, cardiovascular and reproductive functions in patients with fibromatosis.”
  • Cristabelle De Souza, PhD, Stanford University, “Activation of c-JUN modulates the transcriptional activity of CD63 serving as a novel target for the treatment of Desmoid Type Fibromatosis.” Jointly funded by the DTRF and Desmoid Tumour Foundation of Canada (DFC)
  • Joanna Przybyl, PhD, Research Institute – Centre universitaire de santé McGill – McGill University Health Centre, “Targeting hexosamine biosynthesis pathway for the treatment of desmoid tumors.” Jointly funded by the DTRF and Desmoid Tumour Foundation of Canada (DFC)
  • Jonathan Noujaim, MD, #CanSaRCC, “Tyrosine Kinase Inhibitors in Desmoid-type Fibromatosis- A Canadian Multicenter Retrospective CanSaRCC Study.” Jointly funded by the DTRF and Desmoid Tumour Foundation of Canada (DFC)
  • Kris Vleminckx, PhD, Universiteit Gent, “Investigating EZH2 as a druggable mediator of immune cell exclusion in desmoid tumors.” Year 2 of 2. Jointly funded by the DTRF and Desmoid Tumour Foundation of Canada (DFC)
  • Gerlinde Wernig, MD, Stanford Medicine, Department of Pathology, “Studying CD47 blockade as an immunotherapy for desmoid-type fibromatosis.” Year 2 of 2.

Thank you for your support in our mission to find a cure for desmoid tumors!

by

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!

by

*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

by

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)