'K' from USA - Dx 2007 at 10 years old

[Bonni Hess]

Dear 'F',
Thank you for the thoughtful update and encouraging news. I am so grateful that 'K''s four week scans since starting her PF02341066 treatment showed disease stability and some shrinkage of her lung mets, and I will be holding very tight to Hope that the treatment will continue to be effective and successful for her. I am grateful too that she seems to be tolerating the medication well thus far with only minimal side effects. Hopefully if the PF02341066 is successful in continuing to shrink her lung mets, Cryoablation won't be necessary.
Jim and I were surprised by what Dr. Littrup's nurse told you regarding being able to Cryoablate lung tumors up to six cm. since when Brittany had both of her Cryo procedures with Dr. Littrup we were told that 3 cm. was the maximum treatable size. Perhaps this has now changed, but regardless, our research and personal experience has found that it is best to have tumors treated at the smallest possible size for the best possibility of a successful response with the least risk of complications.
I Hope that 'K' is enjoying her school year and her many activities, and that she will have a wonderful summer shared together with your family and her friends. I will be anxiously awaiting your next update following her scans in two months, and sending my most positive thoughts and healing wishes. Take care 'F'.
With special caring thoughts and continued Hope,
Bonni

[Fictional]

Just thought I'd update - in general the 3rd course on PF02341066 is easier than the other ones. There have been unpleasant times - diarrhea that got better with Immodium, and a few episodes of vomiting. The hardest part of weight had been because of her migraines - she doesn't want to eat when she gets them - and we're not sure if the drug makes them worse, or if she is just getting them more because of her age. They seem to be worse around her periods.

I think you get more used to the drug (PF02341066) and side effects get easier with time, rather than the other way around. We still had weight loss issues and got warned about this and had another weigh-in yesterday. We were able to add back 1.5 lbs - and happy with this. Over-all she clocked a 7lb weight loss since starting, so now that is just 5.5 lb net loss and who knows maybe we can add back more. It might have been worse because she developed a urinary tract infection that we hadn't known about - but now that is no longer an issue. I think we didn't want to get the magical number of 10 lbs. She said some of the weight was not true weight loss because we hit the summer months and her clothing was now a lot lighter - as a result - on our weighing days she chooses to weigh wearing her jeans and belt like when she did with her initial weigh-in. We don't know if we could get kicked out of the trial if she loses too much or whether they would decrease the dose. To be on the safe side, we are having her weigh herself everyday at home so we know what's going on.

The tricky part for us was finding what we could encourage her to eat more of but wasn't necessarily so bad. We wanted to avoid concentrated simple carbohydrates (high fructose corn syrup products etc) and dairy (high levels of IGF), but that is not always that easy either especially to also put on weight. I think we've been reasonably successful with putting big cans of peanuts and dried raspberries (she likes the crunch) by her computer (she is always on Neopets) and encouraging her to exercise every day (muscle is heavier than fat). Besides treadmilling, I put very light free weights by her computer as a reminder to do her arm exercises. We also make sure she's well hydrated especially on her weigh-days and fortunately her golf classes have started back so she is not always on the computer...

We go for scans in mid July and we'll see if the stuff is doing anything (I like to think it's doing something even if it doesn't cause them to shrink) - otherwise I think we're headed for cryo on the left lung.

[Olga]

'F' - thank you for the update. I have a Q about the drug - was 'K' tested for EML4-ALK or this is a blind shot with PF-02341066 (Crizotinib its name is now). Re.migraines - I suffer from the very severe migraines from my adolescence and the pain can be very severe - I would not subject anyone to go trough it if it can be treated. The only thing that really helps is sumatriptan (Imatrex) when it is taken early, I take 1/3 of the pill and it helps, the only thing that there is a rebound effect that develops after years of taking so it should be controlled.
Re.weight issues - Omega-3 supplements are usually able to add a few pounds with the added benefit of reducing systemic inflammation overall.

[Fictional]

Hi Olga,

No we did not test her for EML4-ALK. The drug has the same potency at the MET receptor as for ALK. That was why we opted to try it. There is no data about PF02341066 and ASPS that we know of. The only sarcoma that we know responded was an ALK sarcoma very different from ASPS.

In our molecular profiling we found both her primary and her lung mets are positive for MET. Also although there wasn't much shrinkage on ARQ197, a few of the nodules had 40-80% necrosis while she was on it (met inhibitor) and she had almost no side effects. MET is also thought to be expressed on stem cells too, so that's another reason we like those drugs, but they may need to be combined with other drugs for best effects on the course of disease.

[Fictional]

Yay. Stability!

'K''s now completing 3 months on PF 02341066. The good news is stability! The 3 largest nodules didn't budge (radiologist thought one maybe up 1 mm in one, but down 1mm in another) and most looked as if they didn't do anything.

These were not fine cuts, so not easy to make any subtle calls on the nodules, but all round good news for us. No new ones.

'K' would like to put off cryoablation or anything else as long as possible and this clears the way for a nice procedure-free summer.

We are happy with the result...her nodules grew more on the 3 weeks between coming off R1507 and starting PF02341066 (on nothing then) than the last 3 months she been on the drug, so we're pretty sure it's doing something.

Side effects from PF or crizotinib have really have gotten tons better too, she's back to running a little over a mile, golfing, and volleyball. She put back her weight.

We are hoping we can be stable on this one a long time - apparently some patients have been on the drug for as long as 1-2 years. The ways she's been feeling well, she could definitely go back for volleyball team in the Fall.

She also had a repeat pelvic MRI (now 3 years out from surgery) today - and that looks great too. Phew!

This drug might be on faster-track for approval because it has dramatic effects in ALK positive non small cell lung cancer. Apparently 85% of of those with the ALK mutation either PR or stable and it last up to 2 years (and still going).

[Amanda]

YAY! 'F'!
This is wonderful news!!!
*Cheers for 'K'*
I hope that your families summer is filled with laughter and happieness!!

[Arch]

Hi 'F',

We are very happy to know that 'K' is stable on this new drug and that she is tolerating the medicine quite well.
Good that she has also regained the weight that she lost initially...we send our best wishes for continued stability and maybe even some shrinkage.

Arch

[DottyW]

'F',
Celebrating with you the positive news! It's so good to hear that 'K' can participate in so many activities. Jordanne is now walking without crutches with her brace and has actually been able to move her toes and ankle a slight bit. This is great news for her that the nerve may be regenerating or healing.
The complications from the surgeries are always disappointing so I am praying that 'K' can continue to have success on this drug and avoid having to have more procedures or surgery on her lungs.
Praying for continued healing and strength,
Dotty

[Ivan]

Awesome news! I didn't know she played volleyball. Is she any good?

Show her our league videos, for example http://www.youtube.com/watch?v=pfajRfOXaXY

[Fictional]

Not really good, but I think she'll enjoy your league videos. She was among the top girls in her team (middle school volleyball), but slower at running and suicide drills etc. Ivan, you've really been an inspiration and I think that keeping physically active has also helped her ASPS. In the beginning we let her take it easy - and I think that was the wrong idea! There is only one team in her little school for middle school girls - but now over the summer she also practices with the high school girls (and they win district awards etc). I think she is especially good and sensing the dynamics of how kids move on the court - and even the personalities of the girls who don't go for it - so need more assists.

She is usually not interested in anything that reminds her of cancer and that is why she doesn't post here. If you are on FB (she recently set up an account) - she'd probably friend you.

: )

[jcs2007]

Just wanted to say how thrilled I am to hear that 'K' is doing so
good and able to get back to her activities like volleyball. I know
sam loves playing basketball but has a hard time with the suicide drills too
esp. in his left leg. He also likes to keep his "C" quiet which can be
challenging at times. Thanks for the updates.
Wishing her continued healing and prayers,
Cindy

[Fictional]

Yay ! Got more good scans today. Possibly only 1 budged - 1mm or so - the rest are no growth at all. No new ones as usual.

We are VERY happy with this. This clears the way for a normal start to school and volleyball team games in October (yay!)

She's finishing 5 months on PF02341066 aka Crizotinib (ALK / Met inhibitor). Side effects very manageable now (was rough the first month or so) - she takes an immodium if she has cheese, ice cream, or something greasy, otherwise she's fine. She's put her weight back on, but she's not quite at the weight she was at at the beginning of the study.

We were thinking we'd probably be heading to Germany again soon because a month ago Dr. Rolle told us that he would do a redo on the right (operated once 2 years ago)...and that if we let them grow, then he may have to resect more lung, but nothing grew at all on the right, and one that started shrinking when Kgirl was on R1507 looks like it's continuing to shrink, so we are going to let it ride for at least another 2 months. Apparently after these 2 months, we can go to every 3 months.

We are asking permission from the study to do a cryoablation of one met on the left. This is the one that might have grown 1 mm over the past 2 months, but our oncologist is very doubtful. He thinks we would have to leave the study to do it and we don't want to do that. This left one was the one met that Rolle missed with his surgery on the left - and Littrup said he can cryoablate it. It is not in so dangerous a place and a little bigger apparently is not a big deal for the cryo, but I hate it there.Who knows - may be it is not really bigger - she had a slightly different rotation from the last exam, and it clearly did not grow in the preceding 2 months.

We are hoping the Crizotinib will be approved within a year (super success rate for NSLC) - if so it would be easier doing things like cryoablation and going back on the drug in one week.

Thanks for all your prayers and best wishes everybody, 'F'

[Arch]

That is awesome....thanks for the update 'F'.
Crizotinib is also available through trials for both kids and adults at mutliple locations in the US, which is good incase someone else wants to try it, but do you think its better to try it when the tumor load is very low ? Are you aware of anyone else with ASPS on the trial?
Its also good that you have surgery/cryo plans ready just incase some tumor misbehaves.

Best wishes to 'K' for the new school year.

Arch

[Amanda]

Hello 'F'
Wonderful news!
1mm is so small and i pray that it was the scan and not growth! * I think it was the scan *
How many other ASPS paitents have been ont his trial :/

*cheers for 'K'*

[Fictional]

Don't know of any other ASPS patient on this trial so I can't say I would recommend it for everybody. The only definite amazing benefit is for non small cell lung cancer with the ALK mutation. 90% of these patients respond (amazing) and there have been some complete remissions. The only sarcoma I know to respond (by dramatic shrinkage) is an ALK-positive sarcoma - a weird inflammatory sarcoma.

We chose PF02341066 because of several factors - her primary was > 5 cm (increase risk of metastases - we really don't want brain mets or any other mets if we can help it), we knew that Kgal's fastest growing mets had 40-80% necrosis on the met inhibitor ARQ197 (but it only induced cell death in 10% of total nodules), and the drug had fairly low toxicity. Also it was open in Seattle - and she could get in fairly quickly. We also knew theoretically at least, met inhibitors are also mild antiangiogenesis factors because they are upstream from VEGF, and I've always thought that the combination or sequence of IGF inhibitor plus or followed by a met inhibitor would be a good combination in ASPS. If you read a lot about the biology of met, it makes sense that it would beneficial in metastatic disease and that it wouldn't cause too much trouble with normal development or physiology.

I don't know whether she's doing as well as she is because she took an IGF Ab followed by a met inhibitor. That is another thing that could confound results.

Kgal does not have bulky disease, but her left chugging met is now up to 2.3 cm (fortunately not in a dangerous location) - so we would like cryoablate it if it continues to grow.

In the best of all possible worlds, we would like to be on Crizotinib before and after cryoablation - in case the ablation process could cause some tumor cells to break off and potentially be metastatic.

But Dr. Rolle seem champing at the bit a little too - if he decides to operate, it seems generally he wants it sooner rather than later. But if it really hasn't changed, and there are tiny ones that we think he could miss, we think we should wait as long as possible.