The TSA is really pleased to hear of such positive results from a recent trial of topical rapamycin for the treatment of facial angiofibromas. The study, which took place in America and Australia and more details of which you can find on the link below, found that six months of treatment with a rapamycin lotion brought about a significant improvement in the facial rash.

There are a number of further upcoming trials at differing stages of development that will seek to reinforce these results and help to understand the optimum dosage level for the treatment; however this current trial will give much encouragement for TSC patients in the UK in talking to their clinician about accessing such treatment.

Chris Kingswood, TSA Head of Research Strategy said: “This is a really encouraging result. We have known for some time that from case studies and The Exist studied that in people taking Sirolimus or Everolimus tablets for AML or SEGA many have a significant improvement in their rash (REF). 

“Small studies also showed Sirolimus applied directly to the face can work. But it takes bigger studies like this to prove the benefits outweigh the risks and to start convincing the NHS that it should be funded. We hope to extend the knowledge, proof and access by starting a study in the UK within the next 18 months.”

The full paper can be accessed: