Rapamycin is a small molecule originally isolated from nature. It has antibiotic and immunosuppressive properties. It also allows two proteins which do not normally interact to bind together in the cell, which causes problems in the nutrient-sensing pathway.
Mutations in key genes can lay waste to the nervous system. By studying large families predisposed to developing these genetic disorders, scientists can identify the responsible altered gene.
Understanding that cancer is caused by mutations in genes that regulate cell proliferation has led to the development of targeted drug therapies.