Readers hold on to your hats because there has not been a time in medicine as exciting as this since the discovery of the germ theory of disease. Imagine those days in the 1860s when Lois Pasteur proved that microbes not miasma from foul air caused infectious diseases.
Now we are entering a new and exciting time in medicine when many hereditary diseases and many cancers will be cured by gene therapy The FDA has recently approved the gene therapy Luxturna to treat patients with a hereditary retinal disease that can lead to blindness.
This is the first gene therapy approved in the U.S. that targets a disease caused by a specific gene mutation. Luxturna uses a virus as a vehicle to insert a normal copy of the defective gene directly into the patient’s retinal cells. According to the FDA patients who received Luxturna in a trial “demonstrated significant improvements in their ability to complete an obstacle course at low light levels.”
Gene therapy has also shown promise in early trials for other disorders caused by a single gene mutation such as hemophilia, and sickle cell disease. Companies are investing billions of dollars to develop treatments for diseases caused by multiple gene mutations. The FDA recently approved two CAR--T therapies for blood cancers, which in trials produced nearly miraculous results for patients who had failed to respond to other treatments.
This is the dawn of a new age in medicine. Within the next five years we will experience modified gene therapy used to treat and cure many other diseases that have not responded to conventional treatments. Best of all this will be done with fewer side effects.