1.1 The case for basic science – Professor Alan Stitt

The case for basic science

Professor Alan Stitt,
Dean of Innovation & Impact,
McCauley Chair of Experimental Ophthalmology Centre for Experimental Medicine
The Wellcome-Wolfson Building
Queen’s University Belfast

Basic science underpins many mainstream therapeutics currently used to treat eye disease. It needs to remain clinically relevant and translatable into demonstrable benefits for patients if possible. Much of this basic research addresses fundamental questions and is necessarily long term, focusing on solving the mystery surrounding many basic molecular mechanisms driving many ophthalmic diseases. Basic research is highly clinically relevant as it can enable understanding of disease processes, identify new therapeutic targets, develop new drugs and validate their safety/efficacy prior to clinical trial.

Indeed, integrated teams of researchers and clinicians at Queen’s University (Belfast) with complementary skills are focusing on retinal diseases such as diabetic retinopathy (DR) and age-related macular degeneration (AMD) which have clear socio-economic and clinical needs, supported by input from public health, epidemiology, practicing ophthalmologists and patient groups.

Basic researchers in collaboration with clinicians are now moving into an era of so called “precision medicine”, which is accelerating positive patient outcomes. They are working tirelessly to identify and harness prevention and treatment strategies that take individual differences amongst patients into account whereby patients progress through the stages of disease at different and unpredictable rates, manifest distinct types of end-stage disease and respond very differently to drug therapy. Not one size fits all – for example, Anti-VEGF therapy is now a major therapeutic option for wet AMD and diabetic macular oedema (DMO).

However, for these sight-threatening conditions the intra-ocular injections address “late- stage” disease and we continue to treat most patients only when they have full-blown disease. So, there is a pressing need to develop new ways to identify and treat patients earlier to avoid progression to permanent tissue damage.

Tailored Therapies enabling the provision of the right drug at the right dose at the right time would be warmly welcomed by patients wrestling with DR and diabetic macular oedema (DMO) and certainly in the context of the NHS, achieving this goal would have wide-ranging impact on patient outcomes and health care costs.