Topical Rapamycin in the treatment of Tuberous Sclerosis

If you’re one of the estimated one million people worldwide who suffer from Tuberous Sclerosis you’ll be familiar with facial angiofibromas – those small, but noticeable peppercorn bumps that cluster around the face – however you may not be familiar with a highly effective treatment available right now called topical Rapamycin.

Clinical trials of topical Rapamycin

Limited trials and a large body of anecdotal evidence point to the effectiveness of topical Rapamycin. On the back of this, clinical trials using large population samples are currently being conducted by Tuberous Sclerosis Australia (TSA) at the Sydney Children’s Hospital.

However while trials are currently underway, it is still safe and legal to obtain topical Rapamycin through a compounding chemist with a script from your treating doctor or dermatologist. Compounding chemists differ from regular pharmacies in that they have the strictly regulated facilities and the specialist training to make up medications from scratch.

The clinical trial currently underway at the Sydney Children’s Hospital is part of TSA’s efforts to have topical Rapamycin approved by the Therapeutic Goods Administration (TGA). TSA’s long term goal is to have topical Rapamycin added to the Pharmaceutical Benefits Scheme (PBS) so that sufferers of TS can have cost-effective access to the treatment. In the mean time Australia’s can access the treatment through a small number of specialist compounding pharmacies.

Is it safe and is it effective?

Early results from the Sydney Children’s Hospital trial show that the active ingredients in topical Rapamycin do not enter the blood stream, meaning the risk of adverse side effects is very low.

Authors of a paper published in the Australian Journal of Dermatology said, “Although larger studies are needed, this treatment shows a potential to be a first-line management for facial angiofibromas in TSC and appears safe to start in early childhood.”

The authors also claimed that, “Younger patients with smaller angiofibromas had the best response with near complete clearance.”

Where can I get topical Rapamycin?

National Custom Compounding regularly makes up orders of topical Rapamycin for Tuberous Sclerosis sufferers around the country. We’re based in Queensland however we have systems in place that allow us to deliver anywhere in Australia. We also have a large network of Partner Pharmacies across the country who can arrange to have your script filled by us on your behalf. Check the online list to see if there’s a partner pharmacy near you.

Just like all pharmacies around Australia, we can only supply medication on presentation of a script from a GP, paediatrician, dermatologist or other suitably qualified medical practitioner. Talk to your health care provider about your interest in topical Rapamycin. Alternatively we regularly work with integrative doctors around the country who understand the services offered by compounding pharmacies. Our list of integrative doctors is also available on our website.