Gene therapy eyedrops restored a boy’s sight. Similar treatments could help millions
By Laura Ungar and Freida Frisaro, Associated Press, July 24, 2023
FDA Approves First Topical Gene Therapy for Treatment of Wounds in Patients with Dystrophic Epidermolysis Bullosa
FDA Press Release, May 19, 2023
One of the more distressing and dangerous aspects of false and disinformation is how it has persuaded too many people to reject science and one of the products of science, modern medicines, among them vaccines and gene therapies.
Vaccines are one of the great triumphs of humankind, freeing people worldwide from some of our greatest scourges. Yet they only work if we all participate, and sadly participation is dropping due to disinformation.
Gene therapy holds great promise for curing some disorders and diseases once thought incurable. Of course, this will happen only if people, religious groups, and politicians don’t throw up barriers to development and testing, which often happens with new therapies, such as germline genetic therapies.
These two articles, one from the Associated Press and the other an FDA press release, report on a drug called Vyjuvek, approved for treatment of an inherited disorder called dystrophic epidermolysis bullosa (DEB). The FDA release explains the drug and the disorder. The Associated Press article describes how the drug, developed as a topical gel, was adapted to droplet form for use in a young male patient blinded by DEB.
Some might regard restoring the boy’s eyesight as a miracle. In fact, it resulted from scientific research and oversight from the FDA, a government agency much maligned recently over public health issues related to Covid.
The next time you hear or read science and medicine attacked by those who know little of the subjects, think about this article and press release, and as importantly, how these fields of study have improved you and your families’ lives. Ask yourself, where would we be today without them? w/c
Writer / Critic 