A novel gene therapy which is meant to help treat a rare form of blindness has just been approved by the Food And Drug Administration. The therapy helps patients who have the inherited blindness from their birth. The news comes as 2017 is just coming to a close, making it a good year for the medical field.
The novel gene therapy is called the Luxturna and will be able to treat retinal dystrophy. Retinal dystrophy is an inherited disease by which patients progressively get blond in their adult or adolescents age. The patient starts suffering from obscured vision at a young age, and progressively it gets worse until they become blind. The blindness is usually caused by a gene which is responsible for damage to retinal cells, thereby leading to blindness.
In the United States alone, at least close to 2,000 people are affected by the disease. Much more are affected by the disease worldwide, according to reports.
However, thanks to the new therapy, doctors will be able to restore the properties of the retinal cells. The cells will have the chance to restore the protein that usually lacks when the patient starts going blind. Luxturna essentially gives out a benign virus which is able to deliver a functional version of the gene, the RPE-65 gene. The gene will be injected underneath the retina. When the gene is delivered into the retina, it starts creating more of the missing enzyme. Ultimately, the replacement gene therapy will be able to stop the disease’s progression and in some cases even improve the condition of the patient.
Scientists did a stage three clinical trial, and subjects were given obstacles to complete at night. Some of the tested subjects were even able to do completed nighttime tasks such as reading books, playing games or even riding bicycles. After the third stage clinical trials, the FDA moved in and approved the gene therapy. This marks the third gene therapy which has been approved the FDA since August 2017.
In a statement, the FDA Commissioner, Scott Gottlieb, said that the approval of the gene therapy marked another first in the field of gene therapy. The therapy was unique in how it’s worked and the achievement showed humans potential in treating a wide range of challenging diseases.
The developer of the treatment, Spark Therapeutics has not disclosed as yet how much the drug will cost once it hits the market. However, many experts believe the firm might sell it for $1 million to every patient. Dr. Peter Bach, director of the Center for Health Policy and Outcomes at the Memorial Sloan Kettering Cancer Centre, said that he believed the pharmaceutical firm would sell the drug at an enormous price. The drug will cost at least 20-30 times more than the average American can afford, he believes.