Cure Alveolar Soft Part Sarcoma International (iCureASPS)

Our trip to NCI on the 27th of January did not work out very well for us or so we thought then. We were hoping that my son would qualify for the pazopanib trial as the NCI web page (http://clinicaltrials.gov/ct/show/NCT00674024) indicated that 4 weeks of stable brain disease was required, and we had just achieved that benchmark. The principal investigator, Dr. Shivaani Kummar, informed us that 3 months at least was required for entry into this trial. She then told us that she would inquire about getting the drug cedaranib, in a trial at NCI for prostate cancer, for my son. I was surprised and encouraged but didn't dare get my hopes up, because my son and I have been let down so many times before. He completely thought that that was Dr. Kummar's way of letting us know she couldn't help us. We were both prepared to move on. She submitted a "special exception protocol" for my son to the IRB at NCI, they approved it, and by the grace of God his labs made the cut (his liver has many tumors). He started taking the drug on Monday.

I let Dr. Kummar know that I would be sharing this information with my dear friends battling this awful disease and this is what she said -- she said that she could not possibly submit additional special exception protocols for other patients. She said that cedaranib trials for asps patients in the United States were "months away." That's all I know.

I want to share my thoughts on why she may have gone an extra mile for my son. He was diagnosed in 1997. We have been to NCI at least 4 times during the years since then, participating in one trial there in 1998, and nearly getting into one a couple of years ago that the doctors there had high expectations and hopes about for my son. I have communicated with the research nurses at NCI over the years, and I consider them to be dear friends. Importantly, my son's condition has worsened pretty awfully in the past few months. He'd undergone three bland hepatic embolizations between Oct. and Dec. last year, a brain tumor was surgically removed in Oct., several cyberknife treatments to the brain since then, cyberknife to a tumor near his spine in December, and a large tumor in has backed also removed in December. My son has compassionate and caring doctors who were working hard to help him get to a point where he could get systemic treatment. God's grace did the rest.

So we have a deep sense of relief and also I am experiencing a feeling of having crashed -- I'm wiped out.

I hope and pray for all of us that we will find a cure for this "dragon," as my dear friend, Orland H. used to call it. I know that Orland is in heaven, praying for us now, with many other dearly beloved friends who have gone before us.

Beth

Beth, it is great that this doctor went out of her way to get your son on the trial for cediranib, probably they felt guilty after a few unsuccessful attempts before and jumped on this opportunity. He will be first of ASPS patients (as far as I know) in North Am. to go on it and even though she said that other people can not get on that trial using this way, may be there will be other possibilities for them. Please, keep us posted re. side effects, we all are hoping that this drug will help to your son.

Dear Beth,
Thank you for your thoughtful sharing. I am so happy that your son has been accepted into the Cediranib Clinical Trial, and I will be holding very tight to Hope that he will have a very successful response to this encouraging treatment with stabilization of his disease and shrinkage/necrosis of his tumors. I Hope that he will tolerate the side effects well, and will be anxiously awaiting your future updates regarding his treatment experience and results. In the meantime, I Hope that you can get some well deserved rest and relaxation with the great relief and strengthened Hope and encouragement that comes from knowing that your son has been given the opportunity to receive this promising new medication. Take care Beth and keep in touch as you are able.
Sharing your happiness and Hope with special caring,
Bonni

here is the update on the scan results from the first 8 weeks --good news -- this is preliminary based upon the doctors' looking at the scans from baseline and comparing them to yesterday's -- i.e. we don't have an official report yet -- the disease is stable -- they said there is no growth and nothing new -- this is wonderful news for us since we haven't seen two months of stable disease in a row for what seems like forever ... the worst side effect for him has been weight loss, being tired and having no appetite, as he was very thin to begin with. now we are working on gaining about 5 lbs back of the 7 he lost.

here is the brief rundown on his particular experience -- he had high levels of liver enzymes when he started. AST was at the level which allowed him to squeak into the trial (I think it was 2 1/2 times upper limit of normal (ULN) but don't quote me on that). He has lots of liver tumors. After one week on 45 mg dose, AST shot up to nearly 5 times ULN, so the doctors gave him 7 days off of the drug for his AST to come back to baseline -- if after 7 days it didn't come down to baseline, he would be off the trial -- after only one week on it -- you can imagine how stressed out we were that week. By the grace of God, it came down enough, and then he started at week 3 on 30 mg dose.

my prayers are also that all of our loved ones with asps can get on this drug. one of my dear friends told me years ago when my son was diagnosed that if you can get on a drug that will work for 6 months, at the end of that 6 months there would be something new to try. it hasn't worked out quite that way for my son, but he is still fighting and holding on to that hope.

This is fabulous news, Beth! Thanks so much for sharing it. We are keeping you all in our prayers!

Dear Beth,
Thank you so much for your gracious sharing of this very encouraging news which brings so much strengthened Hope to all of us in the ASPS Community. Thank you too for the detailed treatment and side effects information which is so very helpful to those of us who are pursuing the promising Cediranib treatment. Please know how deeply I am sharing your happiness and relief, and how very much my continued special thoughts and healing wishes will be with your dear son as he continues his courageous battle and his Cediranib treatment. Will he be able to remain on the Cediranib indefinitely if his weight, his labs, and his tumor growth remain stable, and if he is tolerating the medication well with no serious debilitating side effects? When will his next scans be done, and are the scans full body scans including chest, abdominal, pelvic CTs, brain MRI, and bone scan? I will be anxiously and prayerfully awaiting your next update, and holding very tight to Hope for continued good news of disease stabilization and tumor shrinkage/necrosis. In the meantime, please take care dear Beth and know how very grateful I am for your invaluable shared treatment information.
With special caring thoughts and strengthened Hope,
Bonni

I'm sorry I can only post a brief note--my son is off the trial --I can't get into details, but his AST was increasing, and a bone met in his pelvis progressed from this most recent scan compared to his April scan, so we are going to see his radiation oncologist. We are of course devastated about this turn and doing what we can to remain positive. Please no questions. best regards,

Beth

This is a very devastating news from Beth re. her son taken off cediranib trial. She did not specify if the progression was seen only in the bone met or elsewhere and if the MRI or PET were done to verify the actual progression versus local tissue necrotic expansion/swelling as the treatment effect. So at least thanks to her people will know that bone metastases might be not susceptible to the cediranib and it might be not getting inside of the bone so for the people with the bone mets there is no time to loose waiting to see if it might have worked and the treatment needs to be done immediately - a radiosurgery or may be RFA like Brittany Hess had, as it was successful on the small size. Bone scan has to be done periodically - may be once a year - for the people with the proven metastatic ASPS to ensure an early detection and an optimal management of the bone mets. I remember the other case we had on the old web-site when the radiation treatment was completely successful to eradicate the bone met in another ASPS patient.

We're so sorry to hear this, Beth. We're keeping you all in our prayers.

Dear Beth,
As I wrote you in a private e-mail, I deeply share the profound heartache and immense disappointment of this devastating news. I am holding very tight to Hope that your son's pelvic bone met can be successfully treated with RFA, and that another systemic treatment will be found for him very soon that will be much more successful. Please know how deeply grateful all of us on this Board are for your gracious sharing of this very difficult news, take care, and know that your son and your family are held very close in heart, thought, and prayer.
Reaching out from my aching heart to yours with deepest caring and continued Hope,
Bonni