Home / News / Alcohol abuse drug disulfiram can be repurposed to treat a blinding disorder

Alcohol abuse drug disulfiram can be repurposed to treat a blinding disorder

New research from Moorfields Eye Hospital and the UCL Institute of Ophthalmology

4 August 2016

The cutting-edge research has identified a gene that causes scarring, together with a repurposed treatment therapy, for the UK’s most common cause of blinding conjunctivitis. The results demonstrate that human scar making cells, from patients with scarring conjunctivitis, are returned to normal by the drug disulfiram, which is licensed to treat alcohol abuse.

In the current study, the research team screened for genetic activity linked to scarring in conjunctival tissue and in scar making cells. The aim was to identify potential therapeutic target molecules and provide a test bed for treatment. The new research published in the Journal of Clinical Investigation Insight discovered that disulfiram can prevent scars forming in a mouse model of scarring conjunctivitis by blocking the pathway that generates Vitamin A.

Scarring conjunctivitis is a major cause of chronic pain and sight loss. The conjunctiva is the membrane that lines the eyelid and covers the eye and helps lubricate and protect it. In conditions such as ocular pemphigoid and severe eye allergy it can trigger rapid scarring, which can destroy the protective functions of the conjunctiva. Ocular pemphigoid is the most common immune mediated scarring conjunctival disease in the UK. Standard treatment is to suppress the immune system. This controls inflammation when it works, but there are unpleasant side effects and it has little effect on scarring. Approximately 1 in 5 people with the condition go blind.

The results of the study

Commenting on the study Professor John Dart, consultant ophthalmologist at Moorfields Eye Hospital and joint research lead said: “These findings suggest that the repurposing of disulfiram, for the topical treatment of mucosal scarring in disorders such as severe eye allergy, may result in effective anti-scarring therapy. They also provide justification for a randomised controlled trial of disulfiram therapy for scarring in ocular pemphigold”.

Breakthrough offers new hope

Professor Phil Luthert, Director of the UCL Institute of Ophthalmology said: “Scarring remains a major problem in eye disease, and in many other conditions, and uncontrolled conjunctival fibrosis is terrible to live with. This breakthrough offers new hope and is a great example of how discovery science can come together with smart repurposing of existing drugs to reach a solution for patients”.

Fight for Sight, UCL Business, and Moorfields Eye Charity funded the study, which is published in the Journal of Clinical Investigation Insight on 4 August.

For further information

Contact: George Allen - 020 7566 2628​

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