An FFB-funded research team from the University of Wisconsin and the University of Utah used human neural stem cells — cells derived from the brain — to rescue vision in a rodent model of retinal degenerative disease.
A team of investigators including David Gamm, M.D., Ph.D., Ray Lund, Ph.D., Clive Svendsen, Ph.D., and Shaomei Wang, Ph.D., says that human neural stem cells offer promise for slowing vision loss in humans affected by a variety of retinal degenerative diseases including retinitis pigmentosa and age-related macular degeneration.
Plans are underway to study human neural stem cells for treating retinal diseases in more rodent models and a larger animal model — important steps before the treatment approach can be evaluated in a human trial.
Also known as human neural progenitor cells (hNPCs), these cells express growth factors that help keep the recipient’s retina healthy. The research group investigated two types of hNPC. One type is naturally occurring and expresses insulin-like growth factor-1 (IGF-1) and fibroblast growth factor-2 (FGF-2). The other type also expresses these factors, but is engineered to express glial-derived neurotrophic factor (GDNF), as well. Numerous studies have shown that GDNF may be a particularly effective retina rescue agent.
Both the naturally occurring and GDNF-engineered cells performed well in the study. “They survived and migrated well, both beneath and within the retina, and did not show evidence of any unwanted effects,” says Gamm. “The cells display a unique distribution pattern within the retina; they migrate to areas that other cell-based drug delivery methods may not reach effectively. This seems to produce a beneficial effect.”
Gamm and Lund are collaborating on other investigations of hNPCs with Clive Svendsen, Ph.D., a researcher at the University of Wisconsin, who has studied hNPCs extensively for the treatment of neurodegenerative diseases such as Parkinson’s disease and amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease).
A human study of hNPCs is underway for treating Batten disease, a rare devastating condition that causes mental impairment, seizures, and progressive loss of sight and motor skills. The disease is fatal and often affects young children.
In addition to sharing information and results from their projects, Gamm and his colleagues will take advantage of a stem cell banking facility at the University of Wisconsin’s Waisman Center. The banking facility provides the resources for mass producing large quantities of high-quality cells for research. Gamm notes that quality control in stem cell production is critical to their safety and efficacy.
The stem cell research of Gamm, Lund and other retinal degenerative disease investigators is being funded by the FFB-Walsh Stem Cell Consortium — a nationwide collaboration established in 2004 to quickly advance emerging stem cell treatments for retinal degenerative diseases.
See this issue of InSight for another story on promising cell-based research conducted by FFB-funded investigator Ray Lund, Ph.D.
DISCLAIMER: Physicians differ in their approach to incorporating research results into their clinical practice. You should always consult with and be guided by your Physician’s advice when considering treatment based on research results.