A European research consortium has begun recruiting patients for the first human trial of eye drops designed to silence a gene signal linked to abnormal blood vessel growth on the cornea, a complication that affects people with aniridia and those undergoing high-risk corneal transplants.
The trials, run out of the Department of Ophthalmology at University of Cologne in Germany, are testing an antisense oligonucleotide treatment known as Olisens. The drops work by blocking expression of Insulin-Receptor Substrate 1 (IRS-1), a protein that triggers blood and lymphatic vessels to invade the cornea. Left unchecked, this vessel growth clouds vision and, in transplant recipients, raises the risk of graft rejection.
Two studies have opened under the European Union-funded Restore Vision project: Olisens-Aniridia, recruiting 20 patients with aniridia-associated keratopathy, and Olisens-Precon, recruiting 10 patients with corneal neovascularisation linked to high-risk transplantation.
The Olisens eye drops already hold orphan drug designation. The current studies are early-phase trials assessing safety and efficacy, with a 12-month recruitment period. Treatment duration is 12 weeks for the aniridia study and four weeks for the transplant study.
the first human trial of eye drops designed to silence a gene signal linked to abnormal blood vessel growth on the cornea
Aniridia is a rare genetic condition affecting around one in 100,000 people, in which the surface of the eye deteriorates progressively as abnormal vessels spread across the cornea. There is currently no targeted treatment for the condition. The same vessel growth is also one of the leading causes of rejection following corneal transplant, and University of Cologne carries out more corneal transplants than any other centre in Europe.
Study physician Dr Mert Mestanoglu said the lack of targeted treatment options was particularly acute for aniridia-associated keratopathy, given the complexity and rarity of the condition.
