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First Patient Treated in Dry AMD iPSC Therapy Clinical Trial

The study’s primary end points are change in visual acuity and the occurrence of adverse events.

The first patient has been treated in the National Institutes of Health’s (NIH) 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).1

The therapy was derived from the patient’s blood by converting blood cells to iPS cells which were then programmed to become retinal pigment epithelial (RPE) cells, which were surgically implanted as a patch of tissue. The treatment was developed in collaboration with FUJIFILM Cellular Dynamics Inc. and Opsis Therapeutics, and tested for safety and efficacy in preclinical studies carried out by a team at the NIH’s National Eye Institute (NEI). The first patient’s surgery was carried out by Amir H. Kashani, MD, PhD, associate professor of ophthalmology, Wilmer Eye Institute, Johns Hopkins School of Medicine, and assisted by Shilpa Kodati, MD, staff clinician, NEI.

“The protocol, which prevented blindness in animal models, is the first clinical trial in the United States to use replacement tissues from patient-derived iPSC,” Kapil Bharti, PhD, a senior investigator and head of the NEI Ocular and Stem Cell Translational Research Section, said in a statement regarding the clinical trial’s announcement in December 2019.2

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).

Participants in cohort 1 must have an ETDRS best-corrected visual acuity (BCVA) letter score in the study eye between 14 and 53 (inclusive) and participants in cohort 2 must have a score between 14 and 58 (inclusive) in the study eye. Additionally, patients’ fellow eyes must have a score no more than 5 letters worse than the study eye. Patients who have been diagnosed with a malignancy expected to affect 2-year survival, patients with a family history of a retinal degeneration other than AMD which is judged by the investigator to play a role in their ocular phenotype, and patients who have taken within the past year or are currently taking medications with known potential toxicity to the retina, optic nerve, or lens will be excluded from the study.

The study’s primary end points are the change in visual acuity and the occurrence of adverse events. Secondary end points include retinal structure as examined via optical coherence tomography, color and autofluorescence imaging, and fluorescein angiography, as well as retinal sensitivity and fixation examined via microperimetry, and multifocal electroretinography responses. All listed end points will be measured at the 12, 24, and 60-month marks.

The study is being conducted at the NIH Clinical Center in Bethesda, Maryland. Patients will be followed for 5 years after surgery. The study’s estimated completion date is May 31, 2029.

1. First U.S. patient receives autologous stem cell therapy to treat dry AMD. News release. National Institutes of Health. August 31, 2022. https://www.nih.gov/news-events/news-releases/first-us-patient-receives-autologous-stem-cell-therapy-treat-dry-amd 
2. NIH launches first U.S. clinical trial of patient-derived stem cell therapy to replace and repair dying cells in retina. News release. National Institutes of Health.December 16, 2019. https://www.nei.nih.gov/about/news-and-events/news/nih-launches-first-us-clinical-trial-patient-derived-stem-cell-therapy-replace-and-repair-dying