A potentially groundbreaking treatment for a rare form of blindness moved one step closer to U.S. approval last week, as federal health advisers endorsed the experimental gene therapy for patients with an inherited condition that gradually destroys eyesight.

(Image: iStick/XiXinXing)

The panel of experts to the FDA voted unanimously in favor of Spark Therapeutics’ injectable therapy, Luxturna, which aims to improve vision by replacing a defective gene needed to process light.

Inherited retinal diseases (IRDs) are a group of rare blinding conditions caused by one of more than 220 different genes. Some people living with IRDs experience a gradual loss of vision, eventually leading to complete blindness. Others may be born with or experience vision loss in infancy or early childhood.

If approved, Luxturna would be the first gene therapy in the United States for an inherited disease and the first in which a corrective gene is given directly to patients.

Doctors deliver the therapy with an injection in each eye that inserts a replacement gene into the retina via a modified virus.

In their experimental genetic study, one year after treatment 18 out 20 patients who received the injection showed the maximum improvement in completing a study-designed obstacle course, and 13 completed the task at the lowest level of light needed showing an improvement in vision. None of the patients in the non-therapy comparison group did.

The FDA has until mid-January 2018,to make a decision and does not have to follow the recommendation of the panel experts, although often it does.

What do you foresee the FDA deciding? Is curing blindness enough reason to move forward with experimental genetic therapy? Or will the lack of research and knowledge about the side effects of genetic therapy blindside their decision? What are your questions or comments? Let me know. You can post them on Twitter or Facebook using #AskNurseAlice or email them to info@AskNurseAlice.com.