Efficacy study of PAK1 activating small molecules as a potential disease-modifying therapy in hypertrophic cardiomyopathy

Our past studies have shown that stimulating p21 activated kinase-1 (PAK1) in heart cells ameliorates pathological cardiac hypertrophy and fibrosis in animal models. In this project, we will evaluate a therapeutic strategy by using a small molecular {AK1 activator a disease-modifying therapy for familial hypertrophic cardiomyopathy (HCM).  The disease stage and time-dependent effects of PAK1 activators in ameliorating cardiac hypertrophy and fibrosis in HCM hearts will be examined and characterised.  The data generated will support developing drug-like small molecules targeting PAK1 as the springboard for further drug development for unmet medical needs of HCM.