Determining novel SMURF1 signalling pathways and precision medicine strategies in patients with pulmonary arterial hypertension

Pulmonary arterial hypertension (PAH) is a devastating disease caused by abnormal growth of cells in the wall of the arteries that supply blood to the lungs. Altered cell function leads to narrowing of the arteries, increased blood pressure in the lung and right heart failure. Patients experience shortness of breath and premature death. Despite differences in the underlying disease process, current treatment guidelines are identical for all patients and treatment options are limited to drugs that relax muscles in the blood vessel wall. Not all patients respond and many experience side effects. An alternative would be to target the cell growth that drives disease.

I have recently demonstrated that SMURF1 levels are increased in patients with PAH and that SMURF1 is critical to cell growth and disease. I will examine how SMURF1 functions in disease using biological techniques and new SMURF1 targeted drugs. I will try to find ways to identify patients who are most likely to benefit from this approach by examining clinical samples and data from the UK National Cohort, the world’s largest cohort of patients with PAH.

My findings will help to improve treatments for patients with PAH.