A new oral medication has shown significant potential to help restore the vision of patients suffering from Leber congenital amaurosis (LCA). There has been no known cure for the disease up until now. LCA causes a range of vision problems, from reduced vision to total blindness.

According to the American Association for Pediatric Ophthalmology and Strabismus, about 10 percent of all cases of congenital blindness or reduced vision in childhood are caused by LCA.

Researchers led by the Research Institute of the McGill University Health Centre (RI-MUHC) in Montreal analyzed 14 participants from all over the world who were suffering from LCA. Their blindness was caused by mutations in the genes RPE65 or LRAT, which lead to a damaging defect in the retinoid cycle, which is among the most crucial cycles in the retina. This is because this cycle is responsible for producing 11-cis retinal, which helps humans capture light that’s needed to begin the vision process. People with LCA can’t produce this molecule, causing the retinal cells to eventually die off.

When an oral retinoid intermediate (QLT091001) was administered to these participants, 10 out of the 14 were able to expand their visual horizons, while others experienced an improved visual acuity.

The study’s lead author, Dr. Robert Koenekoop, who is a Professor of Human Genetics, Paediatric Surgery and Ophthalmology at McGill University and director of the McGill Ocular Genetics Laboratory at The Montreal Children’s Hospital of the MUHC, is optimistic about the potential for oral retinoid intermediate to help restore vision in patients with LCA.

“This is the first time that an oral drug has improved the visual function of blind patients with LCA. It is giving hope to many patients who suffer from this devastating retinal degeneration,” said Koenekoop in a statement.

The findings of the study are published in The Lancet.