Waisman Center
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Scientists infuse rat spinal cords with brain-derived human stem cells

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Paroma Basu, University Communications

Clive Svendsen and Sandy Klein
Anatomy professor and Stem Cell Research Program director Clive Svendsen and research assistant specialist Sandy Klein work with computer image data in the program’s research lab in the Waisman Center.
©UW-Madison University Communications 608/262-0067
Photo by: Jeff Miller
Date: 10/01 File#: color slide

Unveiling a delivery method that may one day help surgeons treat the deadly neurodegenerative disease amyotrophic lateral sclerosis (ALS), researchers at the UW-Madison have inserted engineered human stem cells into the spinal cords of ALS-afflicted rats.

Reporting their work today (April 19) in the journal Human Gene Therapy, the scientists directed certain types of neural stem cells to secrete a neuron-protecting protein before injecting them into the rat spinal cord where motor neurons reside. Motor neurons dictate muscle movement by relaying messages from the spinal cord and brain to the rest of the body. ALS causes the neurons to progressively decay and die.

Notably, the UW-Madison stem cell researchers did not work with human embryonic stem cells, blank-slate cells that arise during the earliest stages of development and can develop into any of the 220 tissue and cell types in humans. Scientists have long regarded these cells as a crucial ingredient in the quest to cure spinal injuries and neurodegenerative disease.

Rather, the scientists worked with more specialized neural stem cells – known as neural progenitor cells – that arise from primitive stem cells during the first few weeks of human brain development. Unlike embryonic stem cells, they can only develop into neural tissue and they are incapable of living forever, as embryonic stem cells can. But the neural progenitor cells are much more appropriate for clinical use because, unlike embryonic stem cells, they can grow in the absence of animal derivatives that are considered a potential source of contamination, says co-author Clive Svendsen, a professor of anatomy based at the university’s Waisman Center, and a leading authority on neural progenitor cells.

“This is the first study that shows that certain types of stem cells can survive and release powerful protective proteins in the spinal cord of rats with a genetic form of ALS,” says Svendsen.

Once inside the brain or spinal cord, neural progenitor cells grow into neuron-supporting stem cells called astrocytes. Some researchers believe that ALS causes astrocyte malfunction, which in turn causes motor neurons to degenerate and eventually die.

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