The Pediatric & SDH-Deficient GIST Consortium

Our Commitment to the Biden Cancer Initiative

We are excited to launch an innovative initiative to strengthen and advance research for effective treatments of a rare subset of Gastrointestinal Stromal Tumor (GIST) patients, who have been unresponsive to the current standard treatments.

Historically, most researchers with an interest in Pediatric and SDH-Deficient GIST worked independently, with each seeing very few of these rare disease patients. There is a critical need for a model of collaboration for sharing data, tissue and resources with patient advocacy groups acting as the linchpin.

To incubate progress for this rare disease population, we have formed the Pediatric & SDH-Deficient GIST Consortium, a collaboration of experts with complementary skill sets to team with patients to discover targeted solutions. We aim to identify at least one effective treatment within three years as demonstrated by the initiation of clinical trials for the SDH population.

The initial focus will include: creation of a platform for shared data to answer key questions, establishment of a tissue bank and guidelines for clinical stakeholders, and the generation of cell lines. Expansion and initiation of studies and clinical trials will accelerate results. Access and outreach strategies will include training for community oncologists and educational webinars for patients and families worldwide.

Anyone can bring together a consortium of people. The trick is to make the consortium work, and I think what helps in a situation is that a patient advocacy group like us, we become the honest brokers. We enable researchers who used to compete to share information.
– Norman J. Scherzer