New Hope for Macular Degeneration

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Scientists from the National Eye Institute (NEI) started a clinical trial testing stem cell therapy on age-related macular degeneration (AMD). There is no current treatment for this condition that impacts mostly people over 65.

 “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 (iPSC),” said Kapil Bharti, Ph.D., a senior investigator and head of the NEI  Ocular and Stem Cell Translational Research Section. The NEI is part of the National Institutes of Health.

The medical team takes the cells from a patient and handles them in the lab to become iPS cells. These cells can develop into any cell type in the human body. They directed these cells to transform into retinal pigment epithelial (RPE) cells. It is the loss of these cells that result in macular degeneration. RPE cells stimulate photoceptors, without them a person is blind.

The researchers grow the RPE in the lab on biodegradable sheets. Surgeons then place the patch of RPE cells onto the retina.

This is a phase 1/2a clinical trial on a dozen patients. The study follows research on animals where no tumor growth was observed and no mutations were present from the testing.

Using a a patient’s own cells, the autologous approach, will help prevent rejection from the patch of RPE onto the retina.

The study follows good manufacturing practice which is essential for transforming research into FDA approval for clinical use on patients. The NEI Intramural Research Program supported this research and it is being done at the NIH Clinical Center in Bethesda, MD.

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