According to The Washington Post, on Tuesday the Food and Drug Administration, FDA, made history by approving a gene therapy for childhood blindness, which will change the lives of many who suffer from different forms of rare eye diseases and other genetic disorders.
This is a huge feat for the gene therapy field, that has faced many setbacks during clinical trials. Back in 1999, the field faced a tragedy with a teenager dying from participation in their experimental trial. Moving forward from this terrible event was difficult, but they now have broken the seal to something great. They are hoping to take the advancement success they experience with genetic eye disease therapy, and apply it to develop treatments for a multitude of other genetic diseases, such a hemophilia or sickle-cell anemia.
Spark Therapeutics, an organization in Philadelphia, created Luxturna, the newly developed treatment. This week it received approval for children and adults, and it’s price announcement is anticipated for January.
A study was conducted on individuals suffering from a deformed RPE65 gene, which is known to be genetically inherited by 1,000-2,000 Americans. When the treatment is performed, the eye is injected with the Luxturna, which uses a benign virus to successfully deliver a healthy RPE65 gene to the retina. While it is not a cure by any means, the improvement to one’s vision is a pretty remarkable accomplishment.
People suffering from genetically impaired eyesight are often diagnosed at a young age, and their sight continues to decay as they get older. Many often end up completely blind at some point. Christian Guardino, a high school student who was diagnosed with a rare genetic eye disease, was one of the first who participated in the clinical trial. While he still is far from perfect vision, his sight has improved dramatically and changed his life completely. He can now read others’ facial expressions, go outside when it’s dusk and he even got to experience seeing stars for the very first time, something we often take for granted every night.
The genetic therapy will become available in the New Year. A downside to the newly developed therapy is cost as it’s astronomical and something that will need to be reworked. Some analysts believe it could cost roughly $1 million dollars to correct both eyes. While one can never put a price to ones visual freedom, it will be a factor many will have to deal with.
