Setting the Pace for the Future
In July of 2018 at Life Fest, The Life Raft Group brought together a team of pediatric & SDH-deficient GIST experts for a symposium focused on this rare subset of GIST patients. This gathering culminated in the creation of a collaboration of US and international specialists and patient advocates committed to accelerating research, creating guidelines, developing and expanding clinical trials, and involving patient advocacy groups in this effort to find a cure and improve the quality of life for these patients.
At the Biden Cancer Summit in September 2018, as one of ten featured speakers, LRG Executive Director Norman Scherzer presented the mission and goals of the newly formed Pediatric & SDH-Deficient GIST Consortium. Read more: liferaftgroup.org/pediatric-sdh-deficient-gist-consortium/.
Activities Post-Summit
• Consortium members have met monthly since its inception and discussed global research on SDH-related tumors.
• Members agreed to share tissue and have worked on tissue sharing with several patients to date in the USA and even more in the UK.
• Information from our Patient Registry provoked the creation of an algorithm, which has been fine-tuned by leading pathologists, and will soon be shared (and hopefully) adopted by pathology societies. Once adopted, this can help influence diagnostic decision-making for this population around the world, which we hope will then also influence clinical decision making.
• The Consortium is working on a grant to fund future activities of the group.
• A webinar was presented for patients to educate them about SDH.
• Three Virtual GIST Tumor Boards were held to present challenging cases.
• Patient Advocacy groups are collaborating with the sharing of information and supporting each other’s efforts.
• Two clinical trials have been announced as well as one site expansion.
Some Specific Highlights
From abroad, Dr. Maria Pantaleo, University of Bologna, Italy, presented to the Consortium in October, on three research SDH-related projects she is currently conducting. Though the results are not yet published, the team is optimistic about early results and they are engaging pathologists in discussion regarding their decision-making tree.
In the U.S., Dr. Jonathan Trent has opened a HIF2a trial at Sylvester Comprehensive Cancer Center, sharing details with the Consortium in order to
evaluate the possibility of SDH-deficient GIST patients as trial candidates.
Dr. Joshua Schiffman, Huntsman Cancer Institute, is focusing on a project for patients with PGL/PCC tumors (plus any SDH-driven tumors). With the Paraganglioma Preclinical Project, his team is working to test therapies and combinations of drugs to identify what can effectively stop these tumors from growing in mice. Read more: liferaftgroup.org/2018/12/new-in-sdh-deficient-research-the-paraganglioma-preclinical-project/.
Dr. Jason Sicklick, UCSD, is focusing on laboratory research with SDH-deficient GIST tissue. Drs.Schiffman and Sicklick are actively searching for tissue from recent surgeries and have appealed to GIST patients through the Consortium members and the resources of the LRG.
The Consortium will continue to meet monthly and future updates on the work of these experts and patient advocacy group will be posted.
Other Posts Relating to the Pediatric & SDH-Deficient Consortium
New Studies Published on SDH Models and GIST Research
Two new studies were published in July and August 2021 focusing on SDH-deficient tumor models and how location can affect GIST tumor profiles and drug sensitivity.
NIH Pediatric & Wildtype Clinic’s ‘Virtual Tumor Board’ Hosted by the LRG
The LRG worked with the NIH to create a collaborative model to serve the needs of Pediatric & SDH patients, hosting a virtual tumor board on June 25th to replace the in-person Pediatric & Wildtype Clinic which the NIH holds annually.
It’s Time: The Life Raft Group Biomarker Testing Campaign – Identify Subsets & Clinical Trials
Identifying rare subsets of GIST and starting new clinical trials is critical. Read here about one researcher's clinical trial progress.
Partnership for Survival – Treating the Rarest of the Rare
Our Rare Disease Day offering is a whitepaper titled 'Partnership for Survival – Treating the Rarest of the Rare' detailing how a collaboration with the NIH has become a model for other advocacy groups and researchers.
New Horizons – Pediatric & SDH-Deficient GIST Consortium Update: A Review with Experts & Advocates
A Pediatric & SDH-Deficient GIST Consortium Update: A Review with Experts & Advocates presented by Jayne Bressington, Becky Owens, and Dr. Jason Sicklick at New Horizons 2019 in Wayne, NJ.
SDH in Action!
“You're off to Great Places! Today is your day! Your mountain is waiting, So... get on your way!” ― Dr. Seuss, Oh, The Places You'll Go! Dear SDH-deficient GIST [...]
