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News from the 4th NIH Clinic

The fourth biannual Pediatric & Wildtype GIST clinic at the National Institutes of Health in Bethesda, Maryland was held on January 20 – 21, 2010, and the NIH is now starting to get needed information from the patients who have been involved in all four clinics. With more data continuing to be analyzed and put into a format that can help find a cure for this disease, there is excitement and hope.

The lecture presented by Su Young Kim, MD, the coordinator for this NIH clinic, is on video webcast and can be watched at this link.  The lecture discussed the findings from the first three clinics.

The experts attending the 4th clinic are shown in this photo, together with other members of the Consortium for Pediatric and wildtype GIST Research (CPGR).


Eleven patients were seen at the 4th clinic; there are already 12 patients scheduled for the 5th clinic in June 2010 (six new patients and six patients who are being seen for a 2nd time). It was mentioned that, while adult GISTpatients are being seen, pediatric patients receive priority, as the main original intent was to study pediatric wild type GIST.  The age range of patients seen so far at the NIH wild type GIST clinics is from 12 to 60 years of age. 80% of patients who have been seen are female. Due to this high female ratio, the NIH is also giving priority to male patients in an effort to study any differences between the genders in GIST.  The breakdown of the 39 patients seen in the first three NIH clinics is as follows: 10 children; 8 teens; 14 young adults; and 7 adults.

Tumor samples needed: Another focus was the continued need for tumor samples for further study.  Dr. Su Young Kim stated that he was eager and willing to go anywhere east of the Mississippi River to pick up fresh samples (distance limitation due to time restrictions in getting tumor sample back to NIH in needed timeframe).

Guidelines and Tumor Board: The NIH consortium board has grown to include more specialists from throughout the entire United States including eight different major medical centers.  Dr. Margaret von Mehren, a member of the NIH GIST consortium team, mentioned that they are indeed currently writing up pediatric GIST patient treatment guidelines with communications occurring with Dr. Carney for including the Carney triad GIST patients as a subset. The NIH is now in the process of developing a plan for a weekly tumor board. While this is still in the future, this weekly tumor board will give great benefits to current and newly diagnosed wild type GIST patients. The hometown doctor will actually be able to discuss and have the input from the experts via technology with scans, and other documents being available for viewing to all of the experts at the same time.

New publication imminent: Dr. Constantine Stratakis, who is on staff at the NIH and specializes in genetics, is very excited about a new discovery that has been made in his lab that will be published in the very near future. To read more about Dr.Stratakis and his study concentrations, please visit:

A few facts: Data are still being analyzed due to the small numbers; however, recurrence does seem to be lower for the younger patients. 73% of the 39 patients seen at the NIH’s first 3 clinics have shown recurrence with a range of 3 to 57 months before recurrence. Outside of the NIH clinic, there have actually been patients who had a recurrence from 8 to even 26 years later.  Wildtype GIST patients, at this point, can never stop getting scanned or receiving medical follow-up throughout their entire lifetime.

After surgery NED (no evidence of disease) status varies but tends to depend on age at diagnosis, mitotic rate, size, and histology status, (most pediatric GIST tend to be epitheliod). 80% of patients seen at clinic so far have had the stomach as the primary site for GIST, 56 % have had multifocal disease at time of presentation.

One very interesting and surprising finding has been that 60% of patients seen at the NIH clinic have had issues with keloid formations.  This is significant because keloid formation is very rare in Caucasian and Asian populations, yet most of the patients have been from these groups.

Clinical research on keloid significance will continue. Other clinical findings still being studied as to their significance (in regards to the wildtype GIST patients) include levels of MET (a cell surface tyrosine kinase receptor), B-RAF levels, EGFR levels, HIF-1-Alpha levels, and presentation with nevi on patients' skin.

Studies continue on which medicines /drug therapies might work best for this specific wild type GIST patient population. There is a recent interest in the drug R1507 which is an antibody to IG1R  (Roche recently decided to stop production/research on this targeted drug therapy). The NIH is in the process of trying to get this drug available to initiate a trial of their own. The hope is that this drug will work on targeting IGF1R, which is found in high numbers on the cell surface in wild type GIST patients. The theory is that the R1507 would block the formation of the new tumors.

Increasing hope!  While no discovery has been made to show a definite cure for wildtype/pediatric GIST, the study at the NIH gives us all great hope with such an intense ongoing study in place!

   Phyllis J. Gay, PT
   Pediatric Coordinator, GIST Support International
   member of the Consortium for Pediatric and wildtype GIST Research (CPGR)




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