Stem cell therapy protects vision in preclinical retinal disease study

A cellular therapy developed by Newark, NJ-based StemCells ($STEM) preserved sight in a rat model of retinal disease, pointing to a promising new option to treat vision loss.

Researchers observed that, when treated with the company's proprietary purified human neural stem cells (HuCNS-SC), animals with retinal degeneration maintained the number of light-detecting cells called photoreceptors that would otherwise be lost to the disease. Furthermore, the surviving photoreceptors appeared to be healthy and normal, and maintained their synaptic connection to other important cells necessary for visual function, according to a study published in the journal Investigative Ophthalmology and Visual Science.

"This study demonstrates that, at the cellular and sub-cellular level, the surviving photoreceptors have all the components that characterize a healthy and normal photoreceptor, and they have the correct synaptic connections," said Nicolas Cuenca, professor in the department of physiology, genetics and microbiology at the University of Alicante, Spain, and lead author of the study. "The robust anatomical preservation of the photoreceptors and their synaptic connections most likely underlie the preservation of visual function."

StemCells is encouraged by the results, saying the study is relevant to vision loss disorders, including age-related macular degeneration, which affects about 30 million people worldwide.

HuCNS-SC is StemCells' lead therapeutic product candidate; the company is developing the cells as a potential treatment for a broad range of central nervous system disorders as well as for age-related macular degeneration.

The company's Phase I/II clinical trial in dry age-related macular degeneration is enrolling patients at two locations in California and Texas. So far, 5 patients have been dosed in the 16-patient trial. A Phase I trial involving HuCNS-SC to treat Pelizaeus-Merzbacher disease, an inherited and highly fatal condition in children involving the brain and spinal cord, is also underway. Another Phase I/II involving the stem cells to treat chronic spinal cord injury is in progress in Switzerland and Canada.

- here's the study abstract
- read the press release