In FY 2019, a group of investigators from CCR and DCEG successfully competed for funding for a new NCI initiative for the RASopathies. With this funding, we assembled a multi-disciplinary group of international RASopathy experts at a project kick-off meeting in February 2019. As a result of this meeting, we produced a draft of a longitudinal cohort study for patients with non-NF1 RASopathies that will be conducted through DCEG. We anticipate that this trial will be open in the Fall of 2019. The goals of this trial are to prospectively study the incidence of cancer development in these patients but also to identify specific measurable endpoints that can be used for monitoring the success or failure of interventional trials. Potential first interventional trials were identified, and appropriate industry contacts have been made, including a CRADA with Kura Oncology for the use of tipifarnib in Costello syndrome. We plan to test tipifarnib preclinically in models of Costello syndrome in the next fiscal year.
