A Rare Occurence: Marlene Portnoy and Dr. Mrinal Grounder discuss how DTRF’s patient advocacy led to the breakthrough clinical trial for Sorafenib

DTRF Co-Founder, Marlene Portnoy, and DTRF Scientific Director Dr. Mrinal Grounder recorded a conversation last year (2019) for ASCO’s Behind the Breakthroughs: The Your Stories Podcast.

This candid conversation uncovers the joint impact of Marlene and DTRF’s patient advocacy and Dr. Grounder’s research that led to the breakthrough clinical trial of sorafenib. “Partnership is key”, says Dr. Grounder. 

This Podcast episode is now available to listen to on ASCO’s ConquerCancer Website, Apple Podcasts and Google Play.

First-Of-Its-Kind Global Consensus Paper Published

By evaluating existing evidence from prior scientific publications, and incorporating the input of desmoid tumour experts worldwide, the paper is the first international evidence-based consensus of its kind on the treatment of desmoid tumours. It answers critical questions regarding approach to treatment recommendations, types of treatments, and pain management.

Genetically Targeted Mouse Models Enable Continued Learning of Desmoid Tumours

“The DTRF Scientific Advisory Board identified the development of a mouse model that more closely matches human tumours as critical to the advancement in therapeutic drug development for desmoid tumours.  Prior to the project’s launch, there were no genetically modified mice that recapitulated the genetics of the disease involving the two most common CTNNB1 mutations. 

We are pleased to report that these mouse models have been generated, and have been tested to verify that the CTNNB1 mutations can be activated.”

The DFC was pleased to help fund the above mentioned study. 

Learn more about the Broad Institute’s Rare Cancer Dependency Map, which Includes Desmoid Tumours!

“The project aims first to produce a collection of desmoid tumour laboratory models with full genomic characterization to begin to address the genetic diversity seen in patients. Using these well-characterized disease models, the team will screen over 6,000 drugs to look for evidence of selective desmoid tumour cell killing.”


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