Experimental Gene Therapies Still Raising Hopes

Wills Eye Hospital has announced that it has treated the first RESCUE trial patient in the United States enrolled in an FDA-approved gene therapy vision research study. A product called GS010 (GenSight Biologics), can be injected right into the eye and, in a sense, “re-wire” or lower the patient’s risk for getting the disease. The patient has Leber’s Hereditary Optic Neuropathy (LHON), a rare, genetic eye disease, passed on by the mother and affecting 35,000 patients worldwide.
Patients with vision related disorders are considered ideal candidates for gene therapy, because the product can be injected directly into the eye.
The first experimental treatment for LHON, also known as Leber’s congenital amaurosis (LCA), was injection of the gene labeled rpe65 in 2008. The procedure showed some success by researchers from the University of Pennsylvania School of Medicine and University College London. In that study, one patient improved from an acuity of 20/2000 to 20/710. Three others showed no acuity improvement, but did improve in other areas of visual function.
If successful, research such as this will open doors for future patients with other eye diseases for which no cures presently exist.
Wills Eye Institute
Effect of Gene Therapy on Visual Function in Leber’s Congenital Amaurosis (James W.B. Bainbridge, Ph.D., et al, 10.1056/NEJMoa0802268, April 27, 2008)