Facial Angio - rapamycin gel

Hello, everyone. I couldn't tell from the Wake Forest paper posted the other day, but my guess is that the 2 cases in Wake Forest was some sort of clinical trial?? The results are amazing!

There are no TS clinics here in Japan, but there is an active advocate who happens to be a dermatologist (specializing in TS) called Dr. Mari Kaneda. According to Dr. Kaneda, topical rapamycin is not on the market yet anywhere in the world, so you can only get it through perhaps some sort of clinical trial. Dr. Kaneda is also running a small-scale topical rapamycin trial (face only) in Osaka, Japan, and I have decided to have my son (7 w/TS) participate in it.

She already had about 18 patients try it out, and so far the topical agent works very well. It doesn't interfere with other medication (oral, etc), and clears up the angios, unless they are heavily clustered and like a plaque. She said she also has hopes that it will have some effect on the white ash leaf. The only thing is that this trial is only 3 months long, so we only get to use the topical rapamycin for the limited 3 month period and not afterwards. (unless there's another trial set up that we can participate in) She mentioned the angios do come back in about 1 month once you stop the topical agent, but wouldn't this be so much better than laser??

I wonder what we can all do to make this topical agent become available in the near future.

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We also are excited about the possibility of using a topical form of rapamycin. Hopefully, there is (or will soon be) a clinical trial somewhere in New England. Angiofibromas may not be the most medically serious symptom of TS, but they are a constant unwelcome visual reminder of the disease. My daughter's last experience with laser was painful and did not provide satisfactory results, so we're excited about the prospect of a better option. I don't have a clue as to what we can do to make this medication available sooner, but if there is anything, I'm on board!

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Thank you for the post. I didn't know about the clinical trial in New England, but that's definitely good news!! I hope more trials take place in the US and the need of the topical agent surfaces. Yes, angiofibromas are not the most serious medical issues of TS, but as you said they are very visible and a constant reminder. My son's angios aren't so bad yet, but he is being teased at school. I didn't know about this until he told me "I want to get rid of the bumps" and started crying. It was heartbreaking to hear from a 7 year-old...

I don't know if this applies to the US, but Dr. Kaneda heading the clinical trial here mentioned that the best way to make the rapamycin gel available in Japan would be organized pressure by patient groups towards the pharmaceutical company and our Ministry of Health, Labor and Welfare (equivalent of your FDA??). Maybe this idea is a little aggressive? I'm not sure. Right now, I'm trying to gather more info on the overall situation of rapamycin in Japan. It's only been approved for use on coronary stents here, and nothing else so far.

At any rate, my son will start using the gel from late October. I'll keep you posted on how it works :)

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My son was prescribed the topical rapamycin cream in Canada by or dermatologist. We have been using it for a month. The pharmacist had to compound 300 oral tablets into cream and the cost was $3000 which was paid by our extended medical. There is also a liquid version which we will try next. There has been some discussions about this medication elsewhere within this group on Inspire. There's also a link to a medical journal with a before and after picture that was inspiring. I think the post was "facial angiofibromas".

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Here's the article (I think you'll have to cut and paste it)...

http://informahealthcare.com/eprint/V5VVtxdxAu5HNMCGqrFt/full?tokenKey=

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Thank you for the post, leanneinvancouver!
Yes, I did read the article.
Wow, you can get a prescription in Canada? Does that mean the rapamycin cream is already approved for use there?? If so, that's great news. How is it working for your son?

I hope it becomes openly available here, too.
I read there is a clinical trial being run in Houston, TX.

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Shimon is also taking part in the trial. I initially had mixed feelings. What good was it to have a treatment for three months without the option of continuing it, even (especially) if it proved effective? On the other hand, I'm looking forward to seeing how much of an improvement there is and I want to support the research here in Japan. Shimon's had one CO2 laser treatment, and though I'm grateful for this option, the topical rapamycin is so much easier, especially because of his disability and difficulty understanding things. I too hope it becomes available soon and we parents can work together make our voices heard.

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Thank you for the post, nokidding.
I know what you mean by mixed feelings about the three-month treatment period, but I'm glad I got to know Dr. Kaneda through this trial. (and you and Shimon, of course!) I think she may help us in opening new doors in Japan.

Her group also seems interested in less-invasive laser treatment which would be less burden for the patients, even if they go through laser treatment. So I'm hoping we'll be in good hands while we wait for the development of the cream.

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At a dermatologist appointment yesterday I took the opportunity to quiz the doctor about his experience with topical rapamycin. He was very positive about it, said he wanted to use it with several patients but had encountered a very problematic response from the insurance carriers. He has treated many patients with TSC angiofibromas using various lasers often under general anesthesia - and insurance has always covered the cost. BUT to treat the same patients with a far less invasive topical cream - the insurance companies balked at the price and declared it "cosmetic" and not medically necessary! More wok to do in education for sure.

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First of all, your son's a cutie! Very nice picture.

I've noticed that the issue of insurance really seems to create various problems...
Luckily, our national health insurance covers most medical costs for children 0 to 12. In the case of Tokyo, children up to 15 can receive most medical care for free.

Is there any possibility that you can participate in a clinical trial and receive the topical cream? I think Leanneinvancouver mentioned "$3000 for the cost of 300 oral tablets to make the cream which was paid by extended medical. " Does the coverage greatly differ by each insurance plan?? (sorry for my lack of knowledge on the insurance situation) But it would really suck if that's the reason for not being able to use the topical cream... It's definitely not "cosmetic." It's improving QOL, I think!

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My son was prescribed topical rapamycin on Tuesday and his dermatologist informed me that we would have to pay a large amount of money for the prescription because the rapamycin may not be covered by insurance and it was not commercially available in a topical cream. I called a compounding pharmacy yesterday to get the pricing information and as it turns out, it is now made commercially in a topical cream and covered by my insurance. This was all news to my son's dermotologist when we told her and very great news for all of us!!

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Buggaboo987, thank you for the post!
The topical cream is commercially available (this is in the US?) AND covered by insurance?? That's the best news I've heard on this so far! So, that would mean some types of insurance cover this medication and others do not, I guess. If you don't mind, could you tell me what insurance plan you use? I think this would be vital info for a lot of people :) Sorry for all the questions!

And thank you so much again for posting.

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Leanneinvancover,
Thank you for posting the link to the article- very inpressive!
My son refuses to have laser for these. The dermatologist explained the procedure to him and he only heard the "feels like little bee stings" and he panicked! It doesn't help he is affraid of bees.
He has been teased by classmates/ ignorant kids for years and it is very distressing to him.
I will be bringing this up at our next appt.
Thank you!

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Hi! Yes, I'm told that my son's rescription for topical rapamycin will be covered by our insurance (Neighborhood Health Plan), but I'll find out with 100% certainity when I picked it on on Monday! I live in the Boston area and its being filled at CVS. Just so you know, when I called my insurance, it was under its drug name "Sirolimus" so that may help. Its exciting and I was very pleasantly surprised to hear this too!! Please let me know if there is any other information I can provide that would be helpful.

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Hello, buggaboo987!
Thank you for all the additional info. Rapamycin is available as Sirolimus from Wyeth (now Pfizer?), and everolimus from Novartis, I believe. I will keep my fingers crossed that it is indeed covered when you pick it up on Monday.

CVS! I used to go to college in Boston. Brings back memories.

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Hey, buggaboo987,
Would you please keep us informed on how it works out? I can't stand the thought of more laser, but am getting to the point where I also can't stand surprising myself when I happen to catch a glimpse in the mirror and realize how bumpy my skin's beginning to look - again. Luckily, for us gals, we can cover with makeup (Bare Escentuals works best for me), but as others have noted on this site, it's a constant reminder that "there's something wrong with me" when I'm usually able to ignore it.

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I am so very sad to report that when I picked up my son's prescription on Monday, it was the oral solution. :( Ugh, I should have known it was too good to be true! On a side note, I paid a $45 co-pay which is much more than any of his others, but that’s not a big deal.

Well, the prescription was the oral solution, and I called CVS. They informed me that the topical cream is not on their formulary. I called back the original compounding pharmacy that told me that it was available and she is going to look into it and get back to me. No word as of yet but I will post as soon as I know anything.

When it was prescribed, the dermatologist told us to apply the oral solution directly to his face and that it would work just as well as the topical cream. So there is still an option. My preference was to use it in a topical cream, just as they did in the studies that showed its effectiveness. How can I be sure that it will be evenly applied and just as effective if using an oral solution?

Runnergal1, you are so right. Women can use makeup as a cover (I love bare escentuals!), but my son doesn't have that luxury. I made that same point to my husband. Let's hope that the compounding pharmacy has access to the topical cream… or can get it for us at a reasonable cost…. and we are sadly back to square one.

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Let us know how you make out with applying the oral solution.

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Hi all,

The woman from the compounding pharmacy called me back. They will get the rapamycin and compound it into a topical cream (it is not commercially available as she previously thought). She contacted my insurance and all of it is covered and approved. I will pay the $45 co-pay but topical rapamycin is covered!Of course, I'll know for sure when I pick up the prescription....

The website is www.custommedicine.com (I got permission to post it). The pharmacy will ship and they bill most major insurance companies. If you have a prescription for it, it may be worth a call to see if they accept your insurance. Without insurance, I know the rapamycin alone is over $900.

Hope this helps!

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Dear Buggaboo987,
Thank you so much for all your information!
Even if the topical cream is not commercially available yet, tif it can be compound into a cream, this info should help a lot of people :) AND covered by insurance! Yeah!

I will check out the website you posted and see if they ship overseas. Maybe it'll help those of us outside the US as well.
www.custommedicine.com

Thanks again for posting!

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does anyone know if this gel would be covered by medicaid?

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