More MS news articles for Nov 2001

Spinal cell transplants hint promise in ALS mouse model

Tuesday Nov 13th, 2001
Investigator: Svitlana Garbuzova-Davis
by Dan Ferber

Two types of cell transplants hold off muscle atrophy in mouse models of amyotrophic lateral sclerosis (ALS). These results offer hope that cell transplants could one day be used to treat this devastating and incurable disease, two research groups announced here this week.

ALS, also known as Lou Gehrig's disease, causes spinal-cord neurons that communicate with muscles to waste away, leading within five years to muscle weakness, then paralysis and death. Despite the efforts of researchers, there's little physicians can do for ALS patients; the only FDA-approved drug, sriluzole, prolongs life just six months.

As an alternative to drugs, Svitlana Garbuzova-Davis and Paul Sanberg of the University of South Florida in Tampa and their colleagues tried transplanting cells called hNT neurons into the spinal cords of mice that had a disease model of ALS. These hNT neurons, which are stable neuron-like cells derived from a human cancer line, are the only cell line approved by the FDA for neural transplants into humans. So far, they've shown promising results in the clinical trials for stroke patients, and they also seem to help animals with Parkinson's disease, Huntington's disease, and spinal cord injury.

In earlier work, which is in press at the Brain Research Bulletin, the researchers showed the hNT tranplants survived in the spinal cord of mutant mice that would soon have developed symptoms of ALS. The treatment prevented degeneration of spinal cord neurons, which helped ALS-afflicted mice live two weeks longer. (Since the diseased mice normally live only about four months, two weeks represented a significant increase in life expectancy.)

In the current work, the hNT neurons did not extend the lifespan of the ALS model mice, but the treatment did slow their loss of movement. To quantitate how well the mice could move, the Florida researchers injected cells into seven of the mutant mice with signs of advanced ALS. Then, they measured the ability of each mouse to walk across a beam without falling, to maintain their balance on a rotating rod, or to extend their hind legs when suspended by the tail.

Although some of the transplanted animals died soon after the operation, the one that survived kept its ability to perform all of these tasks for three weeks longer than mice injected only with an innocuous solution and another ALS mouse that received no treatment. What's more, the hNT neurons survived in the animal's spinal cord.

The work was partially funded by Layton Bioscience of Sunnyvale, California, which owns rights to sell hNT cells. Sanberg is a scientific advisor to the company.

Although the results are "very preliminary," the work is "important because it provides hope that alternative procedures could give rise to therapeutics," says Jay Wang of the Carolinas Medical Center in Charlotte, North Carolina.

In another recent study, also presented here this week, a team led by Jeffrey Rothstein of Johns Hopkins University showed that stem cells derived from human embryos incorporated themselves into the spinal cord of African green monkeys with ALS-like symptoms. The work was partly funded by the Geron Corporation, which owns sole rights to sell therapies based on human embryonic stem cells.

Since earlier work demonstrated that the cells helped rats with ALS symptoms regain strength in their hind legs, the researchers are keeping an eye on the monkeys to see if the cells help the monkeys regain motor skills.
SFN 2001 - Society for Neuroscience

See also:

Of mice, men and motor neurons [Review]
Helen J. Newbery and Catherine M. Abbott
Trends in Genetics, 2001, 17:10:S2-S6

Recombinant proteins for neurodegenerative diseases: the delivery issue [Review]
Patrick Aebischer and Jean-Luc Ridet
Trends in Neurosciences, 2001, 24:9:533-540

Novel drug development for amyotrophic lateral sclerosis [Review article]
Orest Hurko and Frank S. Walsh
Journal of the Neurological Sciences, 2000, 180:1-2:21-28
© Elsevier Science Limited 2000