Rapamycin and Autism

Does anyone have any experience in using Rapamycin in young children to treat Autism?

There are a number of studies I can find that it is beneficial but I’m looking for dosing information.

I found dosing in this format 1mg/m2/d for an 8y/o

That appears to be a dose based on the size of the patient “m2”

I know that surface area is used in converting animal does to human doses so I’m also wondering if that is what they are doing here?

https://www.researchgate.net/publication/349295206_RapamycinSirolimus_Improves_the_Behavior_of_an_8-Year-_Old_Boy_With_Nonsyndromic_Autism_Spectrum_Disorder

Hi Steve,

Welcome to the site. I’ve communicated with this guy on Twitter before and he seems like someone you should talk to. He’s into rapamycin, is a doctor, and has a daughter with autism and I think he’s done some research on the topic. Please post what you learn here for others to benefit from:

https://twitter.com/AtillaOzmumcu

Are you doing this under specialist doctor supervision?

There is thin research on sirolimus and ASD. Not only there is no high-quality data to show a definitive beneficial effect. There are also no long-term safety data and how and how much will sirolimus treatment negatively impact a developing brain. Since mTOR is important for neurogenesis and brain development in general.

What you linked is a case report from a single patient in china. It’s not a sufficient reason to start a child on a serious drug like sirolimus. And the doses used in this case report and you mentioned are the same given to transplant patients. No one should receive such intensive treatment without a specialist doctor’s supervision, preferably familiar with sirolimus. At these doses, Dangerous infections and even deadly sepsis are possible, Specially over a prolonged period. Not to mention the stunting of both physical and potentially cognitive growth.

Even if you’re convinced of the current research regarding sirolimus and ASD then try and find a trial to enroll your child in, As they’ll be able to do proper monitoring and follow-up on your child and help generate useful research. If this is not possible then at the minimum find a pediatric neurologist to monitor such off-label treatment.

Please keep in mind that thre is a high likelihood sirolimus treatment will do more harm than good. Especially given how small the research area on this has been.

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This is a good idea. Clinicaltrials.gov has some trials in this area that seem to be active:

And do lots of google searches on “mtor and autism” and “sirolimus and autism”:

Rapamycin has been used for many years in children as young as 6 months. Also, what I would consider high chronic doses, for immune suppression for transplants in children. There are a lot of studies demonstrating the safety of Rapa in children.

I only posted that one small study as it was directly related to autism. There are more :slight_smile:

There are a number other papers and studies look at ASD symptoms children with Tuberous Sclerous Complex, and several note an improvement. Go to PubMed and use Sirolimus AND TSC AND autism and you can find them. I’m not a doctor, but I’d argue that pulsed dosing of sirolimus is safer (fewer side effects) than most of the currently prescribed medications for ASD.

I received a quick reply, will review the info he provides.

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I have a collection of 35 studies now, including a few on TSC and Autism.

I’ve spent most of today reading them :slight_smile:

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Please post information on what you eventually decide to do and the rationale for your approach, for others in similar situations.

Here are a few of the reports I’ve read;

I’d need to find a trial in Canada.

I’m in touch with people at;

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I have certain limitation on what I’m willing to share but if the boys parents decide to move forward with Rapa I will relay any subjective results.

Here is a paper on dosing for Initial Dosage Recommendation for Sirolimus in Children With Tuberous Sclerosis Complex

This is a daily dose regimen, it’s may not be entirely suitable for ASD but it does show that there are lots of questions still to be ansered.

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