The continuing significant results from small research projects caught the attention of advisors at Novartis, a pharmaceutical company. At the time, the company was looking into cancers that everolimus, an mTOR inhibitor they owned, could treat. They sent representatives to an international TSC research conference in Rome and asked if they could collaborate in a large phase II randomised international trial.
What is everolimus?
Everolimus is a type of mTOR inhibitor. It is marketed under several names, including Afinitor and Votubia in oncology.
History of Everolimus
In the early 1970s, a group of scientists from Canada were searching for new antibiotics. They stumbled across a curious molecule that they named rapamycin. A Canadian pharmaceutical company patented it as an antifungal compound in 1974.
Rapamycin was later found to have immunosuppressant functions. It was acquired by Pfizer, a pharmaceutical company, who developed it as a drug to stop transplants being rejected by the body’s immune system. It has been marketed as an immunosuppressant after being approved by the US FDA since 2001.
Everolimus is a derivative of Sirolumus developed by Novartis. Novartis was able to change Sirolimus’s structure enough to get a new patent while retaining its therapeutic properties. Everolimus is primarily used as an immunosuppressant to prevent the rejection of organ transplants, but it is also being investigated as therapy for a number of cancers.
Clinical research into everolimus
In 2007, Novartis funded a phase II trial which investigated the effects of Afinitor (another name for everolimus) on TSC in 30 American patients. Then in 2008, another phase II trial investigated the long-term effects of using Afinitor in treating angiomyolipoma (a type of kidney tumour TSC patients can get) in 10 patients. They later launched a phase II clinical trial to investigate the effects of everolimus on TSC epileptic seizures using a sample of 20 patients.
The results from all three trials were positive. The regulators approved the use of Everolimus for SEGA (a type of brain tumour), angiomyolipoma and epilepsy. The TSA, however, had to do a lot of lobbying to get the drug funded by the NHS. Through the TSA being assertive from the outset, the NHS has agreed to fund these drugs for the given indications.