Oklahoma's Dean McGee Eye Institute (DMEI) to begin administering LUXTURNA for treatment of Leber Congenital Amaurosis (LCA)

LUXTURNA® (voretigne neparvovec-rzyl) is the first FDA-approved gene therapy for the treatment of a mutation in the RPE65 genes, which cause an inherited retinal dystrophy often known as Leber Congenital Amaurosis. (Image Credit: ©robsonphoto - Adobe.Stock.com)

The Dean McGee Eye Institute (DMEI) will begin administering Spark Therapeutics’ LUXTURNA® (voretigne neparvovec-rzyl), the first FDA-approved gene therapy for the treatment of a mutation in the RPE65 genes. Mutation on these genes cause an inherited retinal dystrophy (IRD) often known as Leber Congenital Amaurosis (LCA).¹

LCA is among the leading causes of inherited blindness in children. Patients with mutations in both copies of the RPE65 gene – as determined through genetic testing – often start to lose vision by the time they are 5 years old. Retinal cells continue to die over time and the vision loss worsens as the patient ages, until most become legally blind.

If the treating physician determines a patient has functioning retinal cells remaining, LUXTURNA offers a chance to reverse the vision loss. Using a non-disease-causing virus, LUXTURNA delivers a normal copy of the RPE65 gene to the retinal cells. This enables these cells to make the proteins necessary to function normally. Through this process, cells become capable of preserving and improving visual function.

Razek G. Coussa, MDCM, FRCSC and Vinay A. Shah, MD, vitreoretinal surgeons at DMEI, will treat patients with RPE65 LCA in conjunction with OU Health Pharmacy and the OU Health-Outpatient Surgery Center. In the news release¹, Coussa said, “We are 1 of only 17 centers in the US certified to provide this specialized treatment. Previously, patients in our area who qualified for LUXTURNA would have had to travel to other states to receive treatment. We are excited to now be able to offer this treatment right here in Oklahoma City.”

R. Michael Siatkowski, MD, MBA, the DMEI’s chief executive officer and the Edward L. Gaylord professor and chair of the University of Oklahoma Department of Ophthalmology shared why this specialized treatment is an exciting addition to DMEI. He said, “We are proud of Dr. Coussa for leading the efforts for DMEI to become a designated treatment center, and for Dr. Shah who joined him in undergoing specific education to administer LUXTURNA. In addition, I want to thank Christian Kanady, founding partner of Echo, for his generous donation that enabled us to purchase the specialized visual function equipment necessary to provide this treatment.”¹

In the press release¹, Christian Kanady, Founding Partner and CEO of Echo also shared his view on the value of this treatment at the DMEI. He stated, “The Dean McGee Eye Institute continues to put Oklahoma City on the map as a leader in specialized vision care and cutting-edge treatments. Providing this transformational vision loss reversal therapy will only add to their incredible impact locally and nationally.”

The DMEI sees LUXTURNA certification as one step in the process of building a center that can provide much-needed medical and surgical treatments to patients with IRDs. It is also noted in the news release¹ that future efforts will include conducting major national and international clinical trials at DMEI/OU, and that the DMEI is the first of its kind in Oklahoma and is envisioned to attract researchers, clinicians, and patients from all over the world.

Reference:

1. Dean McGee Eye Institute, Together with OU Health, Offering New Gene Therapy Treatment for an Inherited Retinal Disease. September 1, 2023. Accessed September 5, 2023. https://dmei.org/dean-mcgee-eye-institute-together-with-ou-health-offering-new-gene-therapy-treatment-for-an-inherited-retinal-disease/