PHA Proof of Concept Research Grant Winner 2013
Kylie M. Drake, PhD
Lerner Research Institute
The Cleveland Clinic Foundation, Cleveland
Title: “Pharmacological Correction of BMP Signaling by Read-Through in Pulmonary Arterial Hypertension”
Term: December 1, 2013 through November 30, 2014
This Proof of Concept award is made possible through a generous donation from Ms. Betty Lou Wojciechowski in honor of her late husband, Mr. Jerry Wojciechowski.
Summary of Research Project:
Despite recent advances, therapies for PH have only demonstrated modest clinical improvements in patients. In particular, patients with heritable PH due to BMPR2 mutations are less likely to respond to some treatments. The ideal therapy for PH would not only ameliorate symptoms, but also drive the reversal of pathological features such as vascular remodeling and vasoconstriction in patient lungs. Dr. Drake will assess the effectiveness of ataluren as a therapeutic molecule for the sizeable subset of PH patients with a BMPR2 mutation. This study is highly novel in that it seeks to target the underlying genetic defect in heritable pulmonary hypertension. Dr. Drake’s preliminary work suggests that this project will herald an exciting new era in which PH treatment is personalized to specific genetic causes.