Experimental Medicine and Clinical Trials

We are concerned with exploring the efficacy and safety of new interventions (pharmaceutical, dietary, behavioural) in humans. These are sometimes known as ‘proof-of-concept’ and ‘proof of mechanism’ studies. They are typically conducted in a small number of subjects, with the aim of discerning dose-response, effect size and tolerability. The studies are data-rich, often employing the latest tools, such as novel imaging, biomarkers, and genomics. The results inform the design of follow-on Clinical Trials, where the primary goal is to define the true value of new therapy and perhaps change clinical practice.

Professor Wilkins' group uses pulmonary hypertension as a paradigm for experimental medicine. This condition is not common but is in need of new treatments and novel ways of monitoring the disease and response to treatment. The group investigate potential drug targets and then develop a preclinical portfolio of data to address the possible use in patients. A ‘proof-of-concept’ study is then designed and conducted around the most promising. The group have focused on the cyclic GMP signalling pathway and recent successes have included work with phosphodiesterase type 5 inhibitors, such as sildenafil.

Wellcome Trust McMichael Centre for Clinical Research Facility

Professor Wilkins is Director of the Wellcome Trust McMichael Centre for Clinical Research Facility. The Centre is dedicated for Experimental Medicine and is multi-discipline. It has a team of research staff that includes nurses, research governance and administrators that conduct the studies under the guidance of each Principal Investigator.

The group work closely with the Toxicology group in the Department. It is also working to establish a formally recognised Clinical Trials Unit that will support the design and conduct of large scale clinical trials, as well as engage in pharmacoepidemiology.