Drug Discovery and Development Symposium 2023: Meet the speaker

Dr Alex Rothman 

FIT-PH/UK Cohort study - Deciphering the Rosetta Stone of digital endpoints 

In patients with pulmonary arterial hypertension cardiopulmonary hemodynamics, exercise capacity, and quality-of-life are used as early-phase study endpoints and for the evaluation of risk in clinical practice. Technological advances have led to the development of devices that can provide daily measurements of these parameters from the patient's home. 

We undertook two studies in patients with pulmonary arterial hypertension implanted with a pulmonary artery pressure monitor and insertable cardiac monitor to evaluate the potential of these devices to provide an early indication of clinical efficacy following the addition of an approved therapy and evaluate the physiological effects of imatinib in patients established on a minimum of dual-oral therapy. 

About Dr Rothman

Alex is an Wellcome Trust Clinical Research Career Development Fellow and Honorary Consultant Cardiologist at The University of Sheffield and Sheffield Teaching Hospitals NHS Trust in the United Kingdom. Alex has a keen interest in the development of new therapeutic strategies and in clinical trial methodologies. 

With collaborators, he undertook the first randomised trial to demonstrate the therapeutic tractability of inflammatory signalling in coronary artery disease (MRC ILA Heart) and developed SMURF1 inhibitors to augment BMP signalling as a treatment of pulmonary arterial hypertension (LTP001). His pre-clinical and early clinical studies have led to the development of medical devices to remotely monitor pulmonary artery pressure (Endotronix) and denervate the pulmonary and renal arteries (SoniVie) that are in regulatory approval studies. 

His current work seeks to use technology to provide an early indication of clinical efficacy for personalised therapy in patients with pulmonary hypertension and to facilitate novel clinical studies in the area.