A New Hope for a Rare Eye Disease: Repurposing an Alcohol Dependency Drug

Researchers at the University of Birmingham are leading a pioneering clinical trial funded by the Medical Research Council (UK) to explore how disulfiram, a medication in use since the 1950s, might be repurposed to treat ocular mucous membrane pemphigoid (OcMMP), a condition that causes painful blistering and scarring in the eyes.

OcMMP is a form of mucous membrane pemphigoid (MMP), which affects the delicate linings of the nose, mouth, throat, and eyes. When the eyes are involved, the disease can lead to irreversible scarring and, in severe cases, blindness.

Isobel Davies, who was diagnosed with MMP in her late-30s, has closely followed the research since disulfiram was first identified as a potential therapy by scientists at Moorfields Eye Hospital and University College London. She later joined an early-stage trial led by Professor Saaeha Rauz at the Birmingham and Midland Eye Centre, initially involving 10 patients.

'Nobody really knows what causes MMP,' Isobel explains. 'It’s an autoimmune disease that affects layers of the skin and mucous membranes. The scarring is what causes the long-term damage: especially in the eyes, where eyelids can fuse to the eyeball, leading to very painful blindness.'

The first stage of the trial focused on whether disulfiram could reduce levels of an enzyme called ALDH (aldehyde dehydrogenase), which researchers believe plays a pivotal role in the scarring process.

After successful results, the team has now launched the second stage involving a further 20 participants; that is around 30 patients in total.

Dr Kusy Suleiman, a member of the UoB research team, explains: 'Current treatments can help control inflammation, but we still lack effective therapies for the scarring itself. Alarmingly, approximately 20% of OcMMP patients lose their sight even while receiving immunosuppressive treatment.'

Given the rarity of the condition, only a few hundred people in the UK are affected. Patient involvement in the trial design was crucial. The charity PEM Friends, where Isobel serves as a trustee, worked closely with the research team to ensure the study was patient-friendly and feasible.

'Our patients helped shape the study,' says Dr Suleiman. 'They advised on the number of clinic visits, follow-up procedures, and sample collection. Their input has been invaluable.'

Disulfiram is gaining attention for its potential beyond alcohol dependency. Studies suggest it may help treat other inflammatory conditions, HIV, certain cancers, and various forms of addiction.

'Disulfiram is proving to be a remarkably versatile drug,' adds Dr Suleiman. 'Our research adds to this growing body of knowledge and opens up possibilities for treating scarring in other organs like the skin, liver, and lungs.'

To improve patient experience and reduce side effects, the team is collaborating with Professor Liam Grover (Biomaterials Science) and University spin-out company Healome Therapeutics Ltd, led by Dr Richard Williams, to develop a disulfiram-based eye gel. This topical treatment could avoid the unpleasant reactions associated with oral disulfiram, such as headaches and nausea, especially if alcohol is consumed. As this is an area of continuous active research, the team really appreciates all public contributions and support to enable its continuous efforts and endeavours to tackle this issue.

Isobel is optimistic: 'There are so few treatments that really work. The idea of an eye gel that could prevent or even reverse scarring is incredibly exciting. It could be life-changing for so many people.'

Recruitment for the trial is expected to conclude in February 2026, with early results expected by October 2026.

For more information about opthalmology research at the University of Birmingham, please contact the programme team.