December 17, 2019
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Patient-derived stem cell therapy trial for geographic atrophy launches

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A clinical trial testing the safety of a patient-specific stem cell-based therapy for the treatment of geographic atrophy has been launched by the National Institutes of Health, according to a press release.

National Eye Institute researchers will convert a patient’s blood cells into induced pluripotent stem cells, which can potentially transform into any type of cell in the body, and program them to become retinal pigment epithelial cells, the release said. The cells will be grown in sheets that are one cell thick, replicating the structure within the eye, before they are transplanted into one eye of each of the 12 study patients.

The phase 1/2a study will closely monitor the patients for at least 1 year to establish safety.

“The protocol, which prevented blindness in animal models, is the first clinical trial in the U.S. to use replacement tissues from patient-derived induced pluripotent stem cells,” Kapil Bharti, PhD, a senior investigator and head of the NEI Ocular and Stem Cell Translational Research Section, said in the release.

If safety is confirmed, further study phases to assess efficacy will be planned, the release said.