The National Institutes of Health (NIH) was proud to host the inaugural Pediatric GIST Clinic. The objective of this clinic was to bring together young patients with national experts in the medical and research realms, in an effort to build a foundation of knowledge upon which to build. From the NIH perspective, the clinic was very successful.

There were four aspects to this clinic. First, we asked patients to send us their medical information prior to their visit. The response we received was incredible. Patients and families went to great lengths to send medical reports, radiographic images and pathology slides to us. This allowed NIH physicians to become familiar with the medical history prior to seeing the patient.

The second objective of the clinic was to discuss the patient’s history with GIST clinicians and researchers. We were fortunate to have several doctors, in various specialties, volunteer their time and expertise in this endeavor. This included Dr. Cristina Antonescu, pathologist; Dr. George Demetri, medical oncologist; Dr. Katherine Janeway, pediatric oncologist; Dr. Michael LaQuaglia, pediatric surgeon; and Dr. Alberto Pappo, pediatric oncologist. Hosting the session was Dr. Lee Helman, pediatric oncologist at the National Cancer Institute, Dr. Constantine Stratakis, geneticist and pediatric endocrinologist at the National Institute of Child Health and Human Development, and myself. During this meeting, we were able to discuss interesting aspects of each patient’s history and to assimilate elements that were common to many cases.

The third part of the clinic was the most satisfying, in that we had the chance to meet with patients and their families. In a short period of time, we addressed the major concerns of each patient. This was made possible since we were familiar with each history, and patients came prepared with a list of questions. Patients also had the opportunity to speak with a range of specialists at the NIH, including Genetics, Pain Management, Nutrition, Psychology, Social Work, Recreation Therapy, Art Therapy and Alternative Medicine teams. Our hope is that the patients and families found these sessions as helpful to them as we found their medical information helpful to us. All of the families were extremely delightful and it was truly a pleasure interacting with all of those who attended.

The fourth aspect of the clinic involved discussion of the present state of GIST. The Office of Rare Diseases (NORD) and the National Cancer Institute (NCI) graciously provided funding for this conference. We were fortunate to be joined by Norman Scherzer and Tricia McAleer of the Life Raft Group and Phyllis Gay and Rebecca Bensenhaver of GIST Support International. This provided a great mixture of advocates, parents and patients. Based on the information that we obtained from the patient records and the cumulative knowledge of those present, we were able to comment about certain aspects of pediatric GIST. This included recommendations on treatment, imaging and research.
nih group2So what did we learn? We determined that pediatric GIST is not a single entity and that there are more aggressive forms and less aggressive forms. In most cases, we found that tumor growth was very slow and that the interval between scans could be increased. We found that treatment for patients has not been uniform and one of our goals is to try to determine the natural course of pediatric GIST and evaluate the many different regimens that patients have received. We talked about Dr. Antonescu’s and Dr. Janeway’s recent findings that levels of Insulin-like Growth Factor Receptor 1 (IGF-1R) are much higher in wildtype KIT tumor samples, compared to that of mutated KIT samples. Regardless of age, we believe that patients with wildtype GIST will have more in common than those with gene mutations. We then discussed Dr. Demetri’s and Dr. Janeway’s plan to initiate a treatment protocol for patients with pediatric or wildtype GIST. The study agent will be an IGF-1R antibody. These were some of the many issues that we addressed. Specifics details of this meeting and other aspects of the Clinic will be presented in the coming months on our website.

As a consequence of this clinic, we are proud to announce the formation of the Consortium for Pediatric and wildtype GIST Research (CPGR). Member institutes include Dana-Farber Cancer Institute, Memorial Sloan-Kettering Cancer Center, Texas Children’s Hospital and the National Institutes of Health. All clinicians and researchers with an interest in pediatric and wildtype GIST are encouraged to join.

CPGR will meet twice yearly at the NIH. The second Pediatric and Wildtype GIST Clinic is scheduled for January 21 and 22, 2009.

Updates of results from Pediatric GIST Clinics will be posted quarterly on the NIH Pediatric & wildtype GIST clinic website, which is expected to open on October 1, 2008. There will also be a parallel series of articles that addresses research. The first of these will explain what happens to a tumor sample, how it is processed and what types of experiments are performed. Every article will be written in a way that a medical dictionary will not be required to understand the document. In addition, there will be a section that contains published scientific articles, in an easy, downloadable PDF format.

The website will also contain restricted access subpages, accessible only by the patient, the patient’s doctor and CPGR members. The delay in opening this website is to ensure security and maintain confidentiality. Medical records, radiographic images and scanned pathology slides can be uploaded to this site. This will allow CPGR members the opportunity to correlate clinical care with research endeavors. As CPGR grows, the ability to help promote patient care via this web interface will also increase.

All of the above was made possible by the willingness of patients and support groups to participate in the inaugural Pediatric GIST Clinic. We would like to thank everyone who contributed time and effort to help in this endeavor. You are truly the pioneers who have established the foundation for scientific progress.