Pediatric GIST collaboration advances
Life Raft, Sloan-Kettering representatives discuss steps that are needed
Creating a center of excellence for pediatric GIST at Memorial Sloan-Kettering advanced when members of the Life Raft Group met with Sloan- Kettering representatives March 16.
The Life Raft Group now includes 10 pediatric GIST patients. For some time the LRG has been putting together a database on this group to see how these patients are doing. Although the LRG database is small, Dr. Leonard Wexler, Sloan-Kettering pediatric oncologist, noted it is nonetheless the largest pediatric GIST database in the world at this time.
From talking to pediatric GIST families and several GIST specialists, it’s clear there is no place which has acquired the experience and expertise for taking care of such young GIST patients. As a result, pediatric GIST patients have become a very rare subset of a very rare cancer and have fallen off the radar screens of both clinical practitioners and researchers
There is one ongoing phase II study of Gleevec in pediatric patients with relapsed or refractory solid tumors which includes GIST along with other cancers. It is noteworthy that this study has opened at nearly 200 sites, just to recruit 100 patients.
This is particularly important because these young patients demonstrate a different form of GIST. Most, if not all, demonstrate a wild-type mutation. That means that most, but not all, do not seem to respond well to Gleevec. On a positive note, however, GIST tumors seem to progress much more slowly in pediatric patients.
The LRG database is made up entirely of girls — something consistent with the experience of Sloan-Kettering pathologist Dr. Christina Antonescu. The youngest LRG patient was 8 at the time of diagnosis. Most have had surgery and most are on Gleevec, taking 200 mg. to 600 mg. per day. It is clear from this diversity that no one has determined the optimal dosage of Gleevec in young GIST patients.
The focus of the March 16 meeting was twofold. The first was to strengthen the current management of pediatric GIST patients so that physicians and families make better day-today decisions. The second was research, with priority to identifying downstream targets and drugs that work against those targets.
Attending from the Life Raft Group were pediatric GIST family members Dorothy and Brian McBryde, and Patricia and Chris Kastner, LRG member Dan Cunningham, Administrative Assistant Tricia McAleer and Executive Director Norman Scherzer.
Memorial Sloan-Kettering’s Department of Pediatrics was represented by Wexler, Antonescu, and pediatric oncologist Dr. Pamela Merola; Dr. Michael LaQuaglia, chief of pediatric surgery, Carol Rossetto, nursing assistant to LaQuaglia and Susanne Hershowitz, administrative manager.
Several projects
were discussed:
1. Creation of an international
registry. An example presented by Sloan-
Kettering was the pleuropulmonary
blastoma registry. Sloan-Kettering
volunteered to submit a grant proposal
for National Institutes of Health
funding to support this project.
2. Creation of a pediatric tumor bank.
3. An annual meeting bringing together
experts in the field to discuss progress
in pediatric GIST management and
research and to plan future efforts. The
LRG will seek funding.
4. The referral of patients and case
histories to Sloan-Kettering so that a
critical body of experience and
expertise can be quickly achieved.
5. An outreach program to pediatric
oncologists informing them of these
projects. Both Sloan-Kettering and the
Life Raft will link their Web sites and
explore outreach opportunities.
To quote one of the family members present: “We wanted to let you and the other parents know that this meeting brought us a sense of relief that there will be a forum for us to reference in our battle with pediatric gist. … this is the first time in the three years since our daughter … was diagnosed that we have some positive feelings that there is something out there that we can access for information, guidance and help.”
