Dry AMD is a leading cause of vision loss among older Americans and as of this writing, has no treatment.
In a potentially breakthrough study, the first U.S.-based AMD patient has been treated in the phase 1/2a clinical trial (NCT04339764) of an autologous induced pluripotent stem cell (iPSC) therapy for the treatment of dry age-related macular degeneration (AMD) by the NIH. The surgery was performed by Amir H. Kashani, MD, PhD, associate professor of ophthalmology, Wilmer Eye Institute, Johns Hopkins School of Medicine with assistance by Shilpa Kodati, MD, staff clinician, NEI.
iPS cells derived from the patients blood were programmed to become retinal pigment epithelial (RPE) cells, which were then surgically implanted as a patch of tissue. From there, the RPE cells will nourish and support light-sensing photoreceptors in the retina. The loss of these RPE cells and resulting degredation of photoreceptors is what causes vision loss in AMD patients.
According to Ophthalmology Times, the open-label clinical trial is estimated to enroll up to 20 participants aged 55 years and older who have been diagnosed with AMD, which is defined as the presence of both at least 1 druse greater than or equal to 63 micrometers in diameter in the macula in at least 1 eye and the presence of geographic atrophy (GA) in at least 1 eye. The trial will initially treat 5 participants (cohort 1) in a single eye. Pending the approval of the NEI Data and Safety Monitoring Committee (DSMC) based on a review of the data from cohort 1, up to 7 additional patients may then receive treatment in a single eye (cohort 2).
The study is being conducted at the National Institutes of Health Clinical Center in Bethesda, Maryland. Patients will be followed for 5 years after surgery. The study’s estimated completion date is May 31, 2029.