Stem cell therapy shows promise for neurodegenerative disease

A new study using mouse embryonic stem cells shows promise in the fight against neurological diseases known as neuronal ceroid lipofuscinoses (NCL). NCL diseases are inherited neurological disorders that have no cure. One of the earliest symptoms is vision loss.

University of Missouri-Columbia researchers injected neuralized mouse embryonic stem cells into the eyes of affected mice. The stem cells not only survived, but also incorporated into the retina on almost every layer.

"The donor stem cells migrated into the retina, differentiated into various types of retinal nerve cells and started acting as those cells by becoming a part of the host network," said Mark Kirk, professor of Biological Sciences.

Four months after the transplant, the stem cells had incorporated into the retinal tissue and prevented retinal degeneration in the diseased mice. The primary type of cell lost due to the degenerative process is the photoreceptor. The results of the study showed that not only did the donor cells replace lost or injured cells, including photoreceptor cells, they also protected the host cells, including photoreceptors, from degeneration and supported their survival.

"Just having the donor stem cells present actually led to a reduction in the size and number of the lysosomal storage bodies, thought to be the cause of the degeneration in the retina," Kirk said. "This work supports the idea that stem cells can be used to deliver useful therapies for neurodegenerative diseases."

The stem cells were then injected into the vitreous behind the lens of the eyes of five-week-old mutant mice. The mice were in the early stage of an inherited lysosomal storage disease characterized by retinal and central nervous system degeneration. Since the retina is formed from the same part of the embryo that forms the brain, researchers consider it an easily accessible model for studying stem cell transplantation into the central nervous system.

The MU study was published in the February 2006 issue of Stem Cells [article abstract]. Jason Meyer, Biological Sciences doctoral candidate; Martin Katz, professor of Ophthalmology; Joel Maruniak, associate professor Biological Sciences; and Kirk conducted the research. Financial support for this research was provided by the National Institutes of Health, The Rockefeller Brothers Fund, and The Batten Disease Research and Support Foundation.

Links:

Kirk Lab
Joel Maruniak
Division of Biological Sciences

Martin Katz
Department of Ophthalmology