LHON Study Finds Double the Impact

Wednesday, January 13 2021 | 10 h 58 min | Vision Science

In a surprising finding, scientists have shown that injecting a gene therapy for Leber’s Hereditary Optic Neuropathy (LHON) into one eye can improve the vision in a patient’s other eye.

LHON is a genetic eye disease which leads to degeneration of the optic nerve, which transmits light signals to the brain. This can lead to sudden irreversible central vision loss.

In this Phase 3 LHON clinical trial, sponsored by GenSight Biologics and published in the journal Science Translational Medicine, scientists injected a gene therapy into one eye of an individual who was recently diagnosed (less than 12 months ago) with LHON. The eye that didn’t receive the gene therapy injection acted as a “control” to ensure that any vision changes or safety issues that occurred were caused by the treatment itself and not by something else. Interestingly (and unexpectedly) 78% of the 37 treated patients experienced improved vision not only in the treated eye but in both eyes.

Further research showed that the gene therapy was likely being transferred to the other eye, but importantly was not going to other parts of the body. These are exciting results and the researchers are completing a few more studies. The hope is that if these studies show that the gene therapy is consistently safe and effective, it could be a new treatment for LHON helping to preserve and even restore vision.

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