According to a recent article written by Kiersten Boyd on 30th October 2017 and reviewed by Abdhish R Bhavsar MD, people suffering from any form of the retinal disease that causes their blindness may regain their vision with the arrival of a new gene therapy treatment.
Treatment of some inherited retinal diseases as a result of the RPE65 mutating has gotten recommended by the U.S. Food and Drug Administration (FDA). It has gotten discovered that the gene’s mutations cause early-onset blindness resulting from Leber’s congenital amaurosis and retinitis pigmentosa among others.
Gene therapy involves adding new genes to the patient’s cell replacing the genes that aren’t working correctly. Genes get carried by a virus prepared specially. It works tremendously well because naturally, viruses use the same way to introduce their genetic material into the cells.
65% of the patients treated for LUXTURNA in a clinical trial experienced meaningful vision improvements. The fact that they made their way in low to medium light levels through a maze was enough evidence.
For those patients who didn’t receive the trial therapy, it was not possible for them to navigate through the maze in up to moderate levels of light. There was no any improvement recorded in their eyesight. Amazingly, eight out of nine patients that went through the study test had their vision improve drastically. When asked to navigate through the maze in the presence of low to medium light levels, they did so very comfortably. It got reported that their sensitivity to light also improved.
In the article, Kierstan Boyd indicates that, according to the study, those who experienced the eyesight improvement has now lasted for two years. Researchers aren’t sure how long the patients will maintain these improvements.
The future of eyesight regain by use of gene therapy is something that excites the ophthalmologists. Today, there are no approved treatments or prove from medicine that can assist people suffering from inherited retinal diseases that threaten their vision. Approval of this gene therapy will be the first to open treatments for many retinal diseases.