Press Release" American Academy of Ophthalmology | October 20, 2024
Two-year study finds patients with advanced disease can recover some vision
CHICAGO, Ill. — New research to be presented this week at AAO 2024, the 128th annual meeting of the American Academy of Ophthalmology, suggests that a new kind of gene therapy can improve vision in people who have lost nearly all sight to retinitis pigmentosa, an inherited condition for which there is no cure and no way to stop it from advancing. While not all patients enrolled in the study responded to treatment, up to 50 percent gained three lines of vision on a standard eye chart. Researchers believe the treatment could also help patients suffering from other types of retinal degeneration.
“We are finally on the brink of an impactful therapy for people with severe vision loss,” said lead researcher Allen C. Ho, MD, director of Retina Research and co-director of the Retina Service of Wills Eye Hospital. “These findings finally deliver hope to patients and ophthalmologists that something is close to being able to help them.”
An estimated 1.5 million people worldwide have retinitis pigmentosa, a group of rare eye diseases that affect the retina, the light-sensitive layer of tissue in the back of the eye. The condition causes the cells responsible for sight, called photoreceptors, to break down over time, causing vision loss. It starts with loss of night vision and usually progresses to loss of color, side, and central vision.
Dr. Ho and his colleagues are investigating a technique called optogenetics to target cells in the retina that don’t normally sense light but often survive after photoreceptors die. It's a one-time intravitreal injection into the eye that uses a harmless virus to deliver copies of light-sensing molecules (Multi-Characteristic Opsin gene) to the surviving cells, turning them into new light-sensing cells to replace lost photoreceptors.
Unlike earlier optogenetics therapies that combined an eye injection with the use of high-tech goggles, this experimental treatment (MCO-010) doesn't require any external device or high intensity light stimulation to coax the cells to respond to light.
MCO-010 is not the first gene therapy to improve vision in patients with retinitis pigmentosa. Luxturna (voretigene neparvovec) is the first FDA-approved gene therapy for retinitis pigmentosa. It works by replacing a faulty gene with a healthy one. But it only works in people who have a specific gene mutation, RPE65, which represents 0.3 to 1 percent of all retinitis pigmentosa cases.
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