Pediatric & SDH-Deficient Research Symposium Founders at Life Fest 2018

The Pediatric& SDH-Deficient GIST Consortium is committed to:

  • Build a Consortium

  • Accelerate Research

  • Create Guidelines

  • Create and Expand Clinical Trials

  • Involve Patient Advocacy Groups

Why a Pediatric & SDH-Deficient GIST Consortium?

There is a subset of GIST which specifically affects children and young adults with a resistant form of GIST for which the current battery of drugs are not particularly effective. Since the number of these types of patients is relatively small and the nature of this subset comparatively complex, progress finding effective treatment has been difficult.

Historically, most researchers with an interest in Pediatric & SDH-Deficient GIST worked independently from other GIST researchers, with each seeing very few of these rare disease patients. The National Institutes of Health (NIH) Pediatric and Wildtype GIST Clinic was established in 2008 with the support of advocacy organizations, to bring together clinicians and patients, to begin to better understand this rare disease. After 10 successful years, this program is at a crossroads,  just at a time when no standard of care for this rare disease has been defined.

Based on this need, the Life Raft Group created and convened the inaugural Pediatric & SDH-Deficient GIST Research Symposium on July 14-15th at Life Fest 2018 in Miami. The goal of the symposium was to gather key clinicians/researchers to identify KIT-independent therapies for targeting SDH-Deficient tumors in order to improve patient outcomes.

An outgrowth of the symposium was the formation of the Pediatric & SDH-Deficient GIST Consortium. The aim of the consortium is to extend and sustain the work of the NIH Clinic and other research efforts, with the ultimate goal of finding a cure through global collaboration. As patients’ survival and quality of life are dramatically impacted by this disease, it is critically urgent that the leading Pediatric & SDH-Deficient GIST experts collaborate and share data and tissue, with the goal of finding successful targeted therapies that can help keep these patients alive.

Members of The Pediatric & SDH-Deficient GIST Consortium Include Clinical/Research Partners:

  • Christopher B. Weldon,* MD, PhD, Boston Children’s Hospital
  • Venkata Ramesh Bulusu,* MD, Cambridge University Hospitals, UK
  • Ruth Casey,* PhD, Cambridge University Hospitals, UK
  • Olivier Giger,* MD, Cambridge University Hospital, UK
  • Lee Helman,* MD, Children’s Hospital Los Angeles
  • Suzanne George,*MD, Dana-Farber Cancer Institute
  • Katherine Janeway,* MD, Dana-Farber Cancer Institute
  • Jonathan Keith Killian,* MD, PhD, Foundation Medicine
  • Margaret von Mehren* MD, Fox Chase Cancer Center
  • Joshua Schiffman,* MD, Huntsman Cancer Institute
  • Michael P. LaQuaglia,* MD, Memorial Sloan Kettering Cancer Center
  • Jason Sicklick,* MD, Moores Cancer Center, University of California, San Diego
  • Fernanda Arnaldez,*MD, MacroGenics
  • Markku Martti Miettinen,* MD, National Cancer Institute
  • Karel Pacak,* D.Sc., MD, PhD, National Institutes of Health
  • Constantine Stratakis,* MD, National Institutes of Health
  • Michael Heinrich,* MD, Oregon Health and Science University
  • Alberto S. Pappo,* MD, St. Jude Children’s Research Hospital
  • Jonathan Trent,* MD, Sylvester Comprehensive Cancer Center
  • Eyal Gottlieb,* MSc, PhD, Technion-Israel Institute of Technology
  • Maria Pantaleo,* MD, University of Bologna, Italy
  • Sosipatros Boikos,* MD, VCU Massey Cancer Center
  • Anthony Gill, MD, Professor of Surgical Pathology, University of Sydney, Australia
  • John Glod, MD, Clinical Director, Pediatric Oncology Branch, National Cancer Institute, National Institutes of Health
  • Paul Meltzer, MD, Chief, Genetics Branch, Center for Cancer Research, National Cancer Institute
  • Eamonn Maher, MD, Head, Department of Medical Genetics, University of Cambridge, UK
  • Sebastian Bauer, MD, Oncologist, West German Cancer Center, Germany
  • William Timmer, PhD, National Cancer Institute

Patient Advocacy Partners Include:

  • Phyllis Gay,* GIST Support International
  • Dr. Victoria Bassett,* GIST Support UK
  • Jayne Bressington,* GIST Support UK
  • Becky Owens,* SDH-RA Cancer Research Advocates
  • Norman Scherzer,* The Life Raft Group
  • Sara Rothschild,* MPH, The Life Raft Group

* Founding members