Reducing corneal scarring in Epidermolysis Bullosa with a novel factor (Martin GR000016)Ongoing
|Prof Keith Martin
|Centre for Eye Research Australia (CERA), University of Melbourne, Austrialia
|Partner organizations & collaborators
|Co-researchers: Dr Gink Yang, Prof Mark Daniell
|Funder(s) / Co-Funder(s)
|Symptom prevention & relief
Short lay summary
Epidermolysis bullosa is a rare inherited disease that causes blistering of the skin and mucous surfaces of the body, including the eye. Living with this disease is like living with third degree burns. Sufferers have restricted mobility and often must be bandaged every day to protect and medicate their painful wounds. Specific mutations in certain types of this disease also have serious impact on the health of the cornea – the outer transparent layer of the eye. The cornea is an important part of our visual system, and any erosion or scarring to the cornea can result in severe eye pain and impaired vision. Current clinical treatments for epidermolysis bullosa including the use of contact lenses, lubrication, and antibiotics to reduce symptoms, but do not offer a solution to prevent scarring in the cornea. Corneal scarring can cause serious deterioration to vision, and hence quality of life, for those already fighting unimaginable daily battles.
Aim of the study: Our research aims to develop an anti-scarring eyedrop for epidermolysis bullosa sufferers using a novel factor.
The novel factor has been shown to restrict molecular mechanisms responsible for corneal scarring and improve corneal transparency in our preliminary studies. The objective of the project is to validate its efficacy in epidermolysis bullosa models using human corneal cells. The human corneal cells will be isolated from donated corneas and transiently reprogrammed to mimic the characteristics from specific forms of epidermolysis bullosa. We will then administer this factor in cell culture following induction of fibrosis and assess its efficacy using biochemistry and microscopy.
An international patient survey on EB patients in 2020 reported patients commenting that corneal erosions ‘‘usually completely shut down my life’’ and ‘‘are one of the worst secondary issues associated with EB, if not the most painful.’’ The proposed project is of basic research nature; however, the overriding aim of the project is to develop a novel anti-scarring eyedrop formulation for EB patients experiencing corneal erosion and scarring. The proposed research may also reveal additional novel targets for EB-induced corneal scarring. This will ultimately accelerate the research and development of other drugs for EB patients in the future.
Prof Keith Martin