“Increasing Focus on Targeted and mTOR Inhibitor-Based Therapies”
- Targeted therapies, especially mTOR inhibitors, are gaining popularity due to their effectiveness in treating symptoms such as brain tumors and kidney lesions
- For instance, everolimus is commonly used to manage subependymal giant cell astrocytomas
- The adoption of everolimus has increased significantly as it directly addresses abnormal cell growth caused by the genetic mutation in tuberous sclerosis
- Pharmaceutical firms are heavily investing in enhancing mTOR-based drugs, such as Novartis’s Afinitor, which has shown positive results in controlling disease progression
- Research is ongoing to improve the safety profile and delivery methods of these treatments, making them more accessible and tolerable for long-term use
- This shift reflects a broader movement toward precision medicine, which focuses on personalized care strategies tailored to the genetic and clinical profile of individual patients with tuberous sclerosis



