While having a needle placed into the eye doesn’t sound like a cause for excitement, researchers at Cedars-Sinai are enthusiastic nonetheless, based on results from a new study. The current findings could help reverse the effects of age-related macular degeneration (AMD) in the early stages of the disease.    

Currently, there is no treatment to slow the progression of AMD, which is the leading cause of vision loss for individuals age 50 or older and effects up to 50 million people globally. AMD causes damage to the macula, a small pigmented spot of cells near the center of the retina. A blurred area near the center of vison is typically the most common symptom.

“This is the first study to show preservation of vision after a single injection of adult-derived human cells into a rat model with age-related macular degeneration,” said Shaomei Wang, M.D., Ph.D., research scientist at Cedars-Sinai Board of Governors Regenerative Medicine Institute and senior author on the current study.

The findings from this study were published recently in Stem Cells through an article entitled “Human iPSC-Derived Neural Progenitors Preserve Vision in an AMD-like Model.”

Dr. Wang and her colleagues utilized a preclinical rat model for AMD, injecting them with induced neural progenitor stem cells derived from induced pluripotent cells. What the team observed was that the injected cells began to proliferate and migrate around the retina, forming a protective layer. This new cell layer halted the ongoing degeneration within the retina for up to 130 days in the treated rats. Extrapolation tables for relative animal age equate that to roughly 16 years of preserved vision in humans.  

“These induced neural progenitor stem cells are a novel source of adult-derived cells which should have powerful effects on slowing down vision loss associated with macular degeneration,” said Clive Svendsen, Ph.D., director of the Board of Governors Regenerative Medicine Institute and contributing author to the study. “Though additional pre-clinical data is needed, our institute is close to a time when we can offer adult stem cells as a promising source for personalized therapies for this and other human diseases.”

The Cedars-Sinai team was very excited by their findings, but did warn against over interpretation of the data, as the experiments were preliminary and more research needs to be completed. Accordingly, Dr. Wang and her colleagues are designing assays to test the safety and efficacy of the stem cell injections in preclinical studies. If the results are as good as the team has seen in the current study then their application for an investigational new drug should follow shortly thereafter.    

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