The new €2.25m US-Ireland R&D partnership is bringing together a unique team of scientists and clinicians to develop a gene therapy approach with the aim of potentially reversing diabetes-linked blood vessel damage to the retina – the light-sensitive tissue at the back of the eye. Ultimately, the team hopes to prevent disease progression and restore visual function.

Diabetes-related blindness, also known as diabetic retinopathy, is caused by high blood sugar levels damaging the blood vessels in the retina. This global challenge affecting more than 90 million people is one of the leading causes of ‘new’ blindness in working-age adults in the UK.

Existing therapies, such as laser treatment and monthly injections of drugs into the eye, are not effective for all patients and carry significant side effects and cost implications for the NHS.

The research team is planning to overcome these limitations by developing a gene therapy approach based on the use of adeno-associated viruses (AAV) which insert genetic material at a specific site. This will enable long‑term delivery of a protein, called COMP-Ang1, to the retina. The researchers have previously discovered that this protein can protect the retina from damage during diabetes, but precisely how it does this remains unclear.  

The team will investigate how COMP-Ang1 prevents inflammation, leakage from blood vessels and improves the function of the retina. They will also investigate the ability of COMP-Ang1 to enhance a stem cell therapy which the team at Queen’s has been developing over several years. Ultimately, it is hoped that COMP-Ang1 will facilitate the reversal and repair of diabetic damage to the retina.

Dr Tim Curtis, from the Wellcome-Wolfson Institute for Experimental Medicine at Queen’s University, said: “During diabetes, blood flow to the retina is impaired and this is believed to trigger the development of diabetic retinopathy. Our aim is to examine whether COMP-Ang1 treatment is capable of preventing or reversing diabetic retinopathy by improving blood flow and vascular repair in the retina. Queen’s researchers are established leaders in diabetic retinopathy and we are delighted to be part of a leading international consortium tackling one of the major health challenges facing us today.”

At the University of Utah, researchers will ascertain how the drug prevents inflammation in diabetic patients, using the smallest possible drug dose to achieve optimal long-term effect while minimising side-effects of toxicity. The researchers at Dublin City University will advance our knowledge of therapeutic viruses to ensure their improved efficiency for delivery into the eye.

Research funding of €2.25m was received through the US-Ireland R&D Partnership Programme.  This research partnership is a unique arrangement involving funding agencies in the USA, the Republic of Ireland and Northern Ireland which combine resources to enable the best researchers from Ireland and the USA to work together on research to address critical issues and generate valuable discoveries that will impact on patient care.

Dr Janice Bailie, Assistant Director of the Public Health Agency’s HSC R&D Division, which is funding the Northern Ireland part of this project with support from the Medical Research Council, said: “We are delighted to be funding this project which will tackle an important problem affecting people with diabetes. We expect that the outcomes from this international research will lead to significant advances in the treatment of patients with diabetes-related blindness in the UK, Ireland and beyond.”

The five-year programme will commence in April 2016.