Having been closely involved with the project from inception, the TSA is delighted to report that the TESSTAL clinical trial of Sirolimus has been published in the academic press*.

As a phase 2 trial, it was designed to investigate how effective and safe the drug was in treating kidney angiomyolipoma (AML) in relatively mildly affected patients, paving the way for more widespread phase 3 trials of this type of drug (mTOR inhibitors)

Over the two years of the trial, all patients were found to have some reduction in the size of their AMLs, with most of the shrinkage occurring in the first year. Half of the patients showed a formal response by internationally accepted clinical criteria, which is a very high success rate for a single, relatively safe drug. Side effects were as expected from earlier experience of Sirolimus in other (non-TSC) usage.

Although the main focus of the trial was on the kidney lesions, neurocognition and pulmonary function were also monitored in the study, with researchers recommending that more investigation is needed on these aspects.

*Sirolimus Therapy for Angiomyolipoma in Tuberous Sclerosis and Sporadic Lymphangioleiomyomatosis: A Phase 2 Trial D. Mark Davies, Petrus J. de Vries, Simon R. Johnson, et al. Clin Cancer Res; 17(12); 4071–81

Posted on 6th July, 2011