Clinical Studies and Small Molecule Inhibitors for RASopathy Treatment

A wide variety of RAS targeting agents have been developed and tested for various cancers over the past 30 years. Recently, many of these agents have been tested in several of the conditions caused by germline mutations in the RAS pathway, known as RASopathies. The mitogen-activated protein kinase kinase (MEK) inhibitor was the first medication approved to treat inoperable tumors in people with the RASopathy Neurofibromatosis Type 1 (NF1). In this chapter, we review the challenges and successes that led to the approval of a targeted therapy in NF1, as well as the current status of development of these treatments for non-NF1 RASopathies.