Questions regarding success/failure of GVAX response

[Bonni Hess]

Dear ASPS Community Friends,
I was very happy to read the entry on the Homepage from the parent of a clear cell carcinoma patient regarding his son's apparent successful response to the GVAX Immunotherapy Vaccine. It was so kind of the parent to share the information, and it is so wonderful to hear that the GVAX seems to have been effective in stabilizing the progression of his son's disease for two years, as also appears to have happened with ASPS patient Anthony Olsen. Heartbreakingly, this has not been the experience of several other ASPS patients who participated in the Clinical Trial who all experienced extensive and even aggressive post Clinical Trial disease progression, including our daughter Brittany. I don't understand why more follow-up isn't being done by the Dana Farber GVAX research team to try to determine why a few patients seem to have had a successful response, while the majority had a failed response. The Phase 1 Trial was closed, with no follow-up studies done to our knowledge since we have never received any follow-up requests from Dana Farber, and there are apparently no plans for a Phase 2 Trial, which is perplexing if the Trial was deemed to be at least successful for some patients. It would be interesting to know what type of ASPS the patients who responded had, versus those who didn't respond, since there are apparently two different types of ASPS, Type One and Type Two as identified by researcher Dr. David Vistica. I remember that during the preliminary planning four years ago for the now inexplicably abandoned Memorial Sloan Kettering Immunotherapy Peptide Vaccine, that some mention was made of the fact that eligibility for the Clinical Trial would be based on what type of ASPS the patient had. Hopefully the clear cell carcinoma patient and Anthony Olsen will continue to have disease stabilization, and some research will be done by Dana Farber to determine the cause for their significantly more successful response than that of the other GVAX Clinical Trial patients, and then a more effective vaccine can be developed which will be effective for all ASPS patients.
With special caring thoughts and continued Hope,
Bonni Hess

[Yosef Landesman]

Dear Bonni,

I am with you. I wish, for the sake of our childrens that one phase 1 clinical trial and we had a cure. The main goal of any phase I clinical trial is to assess the safety and tolerability of a drug / therapy. If a benefit for the patient is already seen in that stage it is great. But finding a benefit for the patient is not required to declare a success of a phase 1 trial. The phase 1 of the GVAX clinical trial does not recruit more patients, but the study continues. Analysis of the material that was collected during the trial continues. Medical research and additional studies demand time. Like you I want to see final conclusions of that trial and I would like to see an effective upcoming phase 2 and 3. Knowing the importance of that research, I am considering the possibility that our organization will put it a goal for 2009 to support and sponsor those studies, so we hopefully see a progress in that front. We all have children and relatives having ASPS and the time is not waiting for us.

I would also like to address three other points that you raised:
1. To my knowledge the Dana Farber is required to continue follow up the patients of this trial for at least 15 years. Follow up can vary: it may be a phone conversation once a year, or invitation of a patient to the Dana Farber for physical checkup, blood or other tests. It was promised to me that each patient continues to be followed in such a way at least once a year and if that does not happen, please let me know and I will check that.
2. Even in the few cases that we think that patients were benefited from the trial, it is impossible to attribute that to the cancer vaccine. The number of the participants was just too small.
3. Regarding the Type 1 and type 2 ASPS, this was discovered in 2001 by Marc Ladanyi from the Memorial Sloan Kettering, who was the first to describe the translocation (break and abnormal fusion) between chromosome 17 and chromosome X, which is found in all ASPS tumor cells.

With Hope for a fast coming cure for ASPS,
Yossi