The cytoskeleton is one of a few biological areas with broad potential for drug discovery and development and has been scientifically and commercially validated in a wide variety of human diseases.
In heart failure the cytoskeleton plays a fundamental role in cardiac muscle contraction and has been linked to the origins of heart failure, a disease of impaired cardiac function. Certain commonly used heart failure drugs that work by indirectly modulating cytoskeletal function have limited therapeutic value due to their clinical side effects.
In cancer, the cytoskeleton also plays a fundamental role in the cell proliferation process and cancer is a disease of unregulated cell proliferation. A number of commonly used cancer drugs inhibit cell proliferation by disrupting aspects of cytoskeletal function. However, these drugs also interrupt cytoskeletal functions unrelated to cell proliferation. This limits their clinical benefit and results in dose-limiting toxicities.
Our understanding of the cytoskeleton allows us to develop potentially safer and more effective drugs for heart failure, cancer and other diseases.