Tuberous Sclerosis Association

Sirolimus Clinical Trials

Sirolimus Trials - This exciting 'life-saving' project offers the first potential treatment for TSC patients in the reduction of tumour growth.

history Campaign has now closed

It ran from to

Registered Charity in England and Wales (1039549)





  • Health/WellbeingHealth/Wellbeing
  • Medical ResearchMedical Research


  • Children (3-18)Children (3-18)
  • Women & GirlsWomen & Girls
  • Young People (18-30)Young People (18-30)
  • OtherOther



Professor Julian Sampson, Cardiff, says: 'When we came to understand what the Tuberous Sclerosis genes did we realised that the job they do is very similar to the job that Sirolimus (also known as Rapamycin) does in cells and so we believe that treating TSC patients with Sirolimus will effectively replace the gene's function. We will offer them treatment with the drug and then monitor the size of the growths in their kidneys and lungs hoping to shrink those growths. We anticipate that this will be the very first of many findings of this kind that will enable us for the first time to treat these previously untreatable inherited diseases.