New Gene Therapy for Vision Loss Comes to Oklahoma

The Dean McGee Eye Institute (DMEI), in collaboration with OU Health, became the 15^th location in the nation to offer a new gene therapy to help treat an eye disorder that often results in severe vision loss, meaning Oklahomans with certain types of inherited retinal dystrophies no longer need to leave the state for this breakthrough care.

Sparks Therapeutics LUXTURNA is the first FDA-approved gene therapy for the treatment of a mutation that causes vision impairment and blindness. The mutation in the RPE65 gene causes an inherited retinal dystrophy (IRD) often known as Leber Congenital Amaurosis (LCA), which results in severe vision loss at an early age. People with this disorder are born with severe visual impairment beginning at birth or develop it shortly afterward, and often, that impairment worsens over time.

Ultimately, without treatment like LUXTURNA, children and adults with the disorder often go completely blind. Before LUXTURNA, the most common treatments for LCA were simply trying to improve the remaining vision a person has.

“We are one of fewer than 20 centers in the U.S. certified to provide this specialized treatment,” said vitreoretinal surgeon, Razek G. Coussa, MDCM, FRCSC. “Previously, patients in our area who qualified for LUXTURNA needed to travel to other states to receive treatment. We are excited to now be able to offer this novel and unique treatment right here in Oklahoma City.”

The treatment is administered as an injection into both eyes. The procedure takes place while the patient is under anesthesia in the operating room. Patients undergo two separate procedures (one for each eye), at least one week apart. At OU Health, the procedure is done at the OU Health University of Oklahoma Medical Center Outpatient Surgery Center so patients can go home following each procedure.

Using a non-disease-causing virus, LUXTURNA delivers a normal “copy” of the mutated non-functioning gene to the retinal cells. These retinal cells then produce the normal protein that helps convert light to an electrical signal to improve vision loss. This enables the retinal cells to make the proteins necessary to function properly so they can preserve and improve vision for young Oklahomans and those who have lived with poor vision throughout their life.

This one-time gene therapy for an inherited disease represents a first-of-its kind breakthrough for treating the disorder and may lay the groundwork for the development of future gene therapies for other complex conditions.

“Providing a whole new world of vision experience is the goal,” said Coussa. “Some patients recently treated went from seeing only darkness or light to walking without a cane and seeing objects and human silhouettes. This is a big deal.”

OU Health’s pharmacy is one of a handful of pharmacies in the country that the manufacturer has allowed to be able to compound and dispense the medication. OU Health’s comprehensive pharmacy infrastructure allows the pharmacy team to access the treatment, which then allows Dean McGee to provide the administration of the medication to patients.

“OU Health is the only health system in Oklahoma that can provide a number of treatments such as LUXTURNA due to the resources and expertise of an academic environment,” said OU Health Chief Administrative Officer of Clinical Support Services Jigar Thakkar, PharmD, MBA, MHCDS, FACHE.“OU Health’s integrated health care delivery system is necessary to put together all our resources and leverage our assets to impact care across Oklahoma. That is our academic health system promise and obligation to the state.”

For more information about LUXTURNA treatments, call Dean McGee Eye Institute at (405) 271-1092 or (800) 787-9014.