Cure Alveolar Soft Part Sarcoma International (iCureASPS)

I wanted to share information on my wife MJ and her diagnosis with ASPS

She was diagnosed in July 2009 with ASPS. The initial MRI revealed a 14 x 11 cm tumor in the upper right thigh. A CT scan further revealed mutiple mets in the lungs, as many as 12-13, with the largest at 3.8 cm. After consulting with an ortho oncologist and medical oncologist, surgery did not appear to be the best path. They suggested, along with a radiation oncologist, the Cediranib trial at NCI in Bethesda. We actually live a few hours on the east coast from Bethesda, so the option was pretty easy for us navigate. After several weeks of logistics, we were accepted into the trial at NCI. She began taking the medication 9-1-09. I wanted to share this information and her progress on this site. I have found over the past couple of months that this site has been very helpful for me and appreciate everyone sharing stories and information. I will keep you updated on the progress - We have our first staging scans late in October. The nurses indicate that she is the first person on the trial. It will also be her first course of treatment, as she has had no other treatments since being diagnosed.

Welcome MJ and Brian!

Lots of people very excited about Cediranib. Great that the trial's not to far away for you.

With best wishes,

'F'

Dear Brian,
Thank you for so thoughtfully sharing this important information with this Forum. As 'F' said, there are many ASPS Community members who are vey excited about this promising new teatment and they are closely following the anecdotal experience and results of those patients who are currently being treated with Cediranib. Our special thoughts and best wishes for treatment success will be with your wife, and we will be anxiously awaiting your next update. Please let me know if there are any questions which I can try to answer for you regarding Brittany's Cediranib treatment and side effects.
With special caring thoughts and continued Hope,
Bonni

Similar to Bonni's recent news on Brittany's experience with Cediranib, I bring great news as well. After the first two months on the trial drug, MJ experienced a decrease in the size of her primary tumor and reduction in the lung mets as well based upon the recent scans. The doctors were very pleased with the results, which included an approximate 20 percent reduction in size in the primary tumor and the larger lung mets. Some of the smaller lung mets have essentially disappeared and there is no new growth. The primary tumor decreased from 9.4 cm to 6.9 cm. She did have some trouble early on with side effects, and was reduced from 30 to 20 mg after only two weeks on the trial. Side effects have been minimized and she will continue on the drug. Needless to say, we are very happy with the results. We have heard that most impact is likely over the first few months of the drug, and stabilization may occur thereafter. I will keep you posted on the next results in December or any relevant updates.

Dear Brian,
Thank you for sharing this wonderful and VERY encouraging news! I am so happy for MJ and you, and I share your great joy and strengthened Hope. I am grateful that the reduced Cediranib dosage was successful in minimizing the negative side effects that MJ was experiencing apparently without reducing the effectiveness of the medication, and that she will be able to remain on the Trial.
My continued special thoughts and healing wishes are with MJ, and I will be holding very tight to Hope for continued stabilization of her disease and tumor shrinkage/necrosis. I look forward to Hopefully continued GOOD NEWS! following her four month Clinical Trial scans in December. In the meantime, please take care, give MJ a special hug from me along with my happiest congratulations and best wishes, and keep in touch with this Forum as you are able.
Sharing the great joy of your GOOD NEWS! with special caring thoughts and continued Hope,
Bonni

Congratulations and best wishes to MJ and Brittany. I hope and pray for the continued shrinkage and stabilization of your tumors.
It indeed gives hope to all of us here.

I would also like to hear how MJ and Brittany have been handling the side effects of this drug(I heard about the acid reflux, mouth sores),but are they going about as usual with all their regular work ? How much of their regular routine is disturbed by this treatment, how often do you go for follow up etc ? I know that its not the most important thing, we just want the drug to be effective, but its some information which will help us take decisions incase we get a chance to try it. Thanks.

Dear Arch,
Thank you for your very kind congratulations and good wishes. It is so nice to be able to share some good news for a change! Regarding your questions about the side effects of the treatment, Brittany certainly does suffer from many of the side effects, but with her usual very positive attitude, strong will and determination, and invincible zest for Life she determinedly tries not to allow the side effects to interfere with her very active Lifestyle. She does get fatigued ( but that is a relative term with Brittany because she has more energy even on the medication than I and most other people normally do!) The joint and muscle pain, the headaches, the increased mouth sensitivity, the hand and foot symdrome, and the gastrointenstinal problems ( diahrrea, nausea, vomiting, acid reflux) are debilitating, but thankfully thus far Brittany has been able to tolerate and cope with the problems with medications to help relieve the symptoms and dietary awareness of certain foods to avoid, and she remains very active. Brittany's Clinical Trial protocol requires that she have Clinical check-ups with blood draw and ECG monitoring in Edmonton once a month, and chest/abdominal/pelvic CT scans there every two months. Since we live in Seattle and it is a travel hardship and tremendous expense to commute 900 miles to Edmonton once a month, we asked if Brittany's Clinical check-ups and scans could be done in Seattle, but the pharmaceutical company will not allow this as they need to maintain complete control over every aspect of the Trial. I Hope that this helps to answer your questions Arch. I Hope too that Sree is doing well and that his disease has stabilized. Take care and keep in touch with the Web site as you are able.
With special caring thoughts and continued Hope,
Bonni

Arch

When MJ first started the trial, the impact was almost immediate - she had significant pain at the site of the primary tumor in her leg. She was taking some heavy pain medication to counter the pain, and it became unbearable for her to deal with at the time. This was only for about two weeks, which I think was the time of the most reduction in the size of the tumor. We could actually see the change in the size of her leg, being reduced in size. The density of the tumor also changed, from a hard mass to a much softer feel in only two weeks on the drug. After the reduction in dosage to 20mg, the pain subsided. The remaining side effects are very manageable, with fautique being the most apparent in daily life. The other effects are very manageable at this point, and do not disrupt her day that much. High bood pressure has been the most worry of late, and they are treating that with several medications. For the most part, I would say that the side effects are mild to medium, and manageable thus far.

Fabulous news on MJ and thanks also for the added info on side effects at the lower dose. Cediranib goes up for FDA-approval in the 2nd half of 2010, so it may be available from the local pharmacy if approved, which many anticipate it will!

Brian - can you guys try to get an information about the number of the ASPS patients already enrolled into the NCI trial?
It has this new type of design when there is a smaller sub-group defined for the first part of the trial that is going to determine if the trial is going to proceed (from the trial's description - an optimal two-stage design to rule out an unacceptably low 5% clinical response rate (PR+CR) in favor of a modestly high response rate of 25%. The study will initially enroll 9 evaluable patients and if none of these 9 patients has a clinical response, then no further patients will be accrued. If 1 or more of the first 9 have a response, then accrual continues to a total of 24 patients. The study will accrue a maximum of 27 patients.)
I was worried that if by some reason none of the first 9 will have this response the trial will be stopped...Looks like we might be already getting this single needed response though.

Olga

Some comments made while we have been at NIH for the visits lead me to believe that there are at least 4 or more patients in the trial - I think its fairly fluid though, as comments made by the nurses seem to indicate that complications have taken some off/on again several times. I think MJ represents the first positive response, and I think that is what they needed to continue the trial to 27 maximum. I know they were very happy with the results. We were told that if 1 of the 9 showed positive results, then the trial would continue.

Arch - One more follow-up, we have to go to Bethesda every two weeks through the first four months for bloodwork, etc. Scans are done at the beginning for baseline, and then at the end of two 28-day cycles. We have scans again right before Christmas. We also are required to fax labs to them every other week from the local ongologist office. So, she is either in Bethesda or at the local Dr every Tuesday for the first four months of the trial. After two cycles (or four months), I think the visits to Bethesda are reduced to monthly.

Hello again everyone,
I agree with Brian that the Cediranib side effects seem to be manageable thus far even at the higher 30 mg. dosage which Brittany is currently on, and the side effects are certainly much less severe than most of the traditional chemo drugs. As you may remember, Brittany did experience severe nausea and vomiting with the 45 mg. dosage which required emergency hospitalization and consequently the dosage reduction to 30 mg. During the past few days she has had some nausea and vomiting, but Hopefully this will subside and will not require a further dosage reduction, but if it does it is very encouraging to know that MJ's dosage reduction to 20 mg. did not seem to negatively affect the effectiveness of the medication. According to the consent forms which Brittany signed, as well as conversations with the Clinical Trial oncologist, Dr. Sawyer, it is unknown at this time what effect the medication may have on fertility which is obviously a huge concern for Brittany at age 27 since she has been unable to have children yet due to her eight year+ ASPS battle. Was this issue discussed with MJ and you Brian, and if so, is any further information available yet on this aspect of the treatment? Again, we are so profoundly grateful for the very positive and encouraging response that both Brittany and MJ have had to the Cediranib thus far, and we now have greatly strengthened Hope with which to continue on this challenging journey.
With special caring thoughts and continued Hope,
Bonni

Dear Bonni and Brian,

Thanks for sharing this information. There might be hardships, but there is hope for a cure and that is really wonderful news.
I also hope that the trial will continue to accrue more patients with MJ's encouraging response.

Arch

Greetings again

MJ underwent the latest scans recently, and I am happy to report additional shrinkage in the size of the tumors. In total, the size reduction is near 30 percent thus far. However, the doctors have placed the drug on hold for her due to some complications with side effects, namely weight loss and liver issues. We are very happy obviously with the results thus far, but will be two weeks soon with the drug on hold due to the complications. So, its a bit frustrating right now, but hopefully she can begin again in a week. I will keep you updated, and I hope that everyone is enjoying the holiday season!

Dear Brian,
Thank you for your thoughtful update. As I wrote you in my personal e-mail to you, I am SO VERY happy and encouraged to hear the wonderful news from MJ's most recent scans which shows continued shrinkage of her tumors! I am sorry that the side effects of the Cediranib have created problems with her liver enzyme levels and weight loss which have necessitated her being taken off of the medication for two weeks, but Hopefully the break from the Cediranib has now allowed her liver enzymes and weight loss to stabilize so that she can resume taking the medication as soon as possible. It is our understanding from Brittany's Phase 1 Cediranib Clinical Trial in Edmonton that the Trial protocol only allows a two week maximum that she can be off of the medication without jeopordizing her participation in the Trial, but this may be different with the NIH Phase 2 protocol. Also, as I have told you, there is a documented risk of rebound with anti-angiogenic drugs when the medication is discontinued for too long. The following concerning information was thoughtfully shared with me by the mother of an ASPS patient who suffered tragic rapid disease progression when he was abruptly permanently taken off of Cediranib following his termination from a pediatric Cediranib Clinical Trial.
Dr. Folkman wrote, in Community Oncology, 4:5 May 2007: “Nevertheless, clinical experience reveals that it may be prudent for oncologists to continue treatment with an angiogenesis inhibitor, even in the face of apparent “drug resistance” (Daniel von Hoff, personal communication). Discontinuing anti-angiogenis therapy may lead to more rapid tumor growth.”
This is a risk which I feel that everyone taking anti-angiogenic drugs needs to be aware of, and something which should be discussed with the oncologist. My continued special thoughts and best wishes are with MJ and your family. Please take care Brian, keep this Board updated as you are able, and have a most Happy New Year filled with the strengthened Hope that Cediranib has brought to all of us in the ASPS Community.
With special caring thoughts and continued Hope,
Bonni