Quebec researchers develop ALS vaccine

CHARLIE FIDLMAN, Montreal Gazette
Published: Monday, January 29, 2007

There’s glimmer of hope for people with ALS or Lou Gehrig’s disease.
A Quebec team of researchers is the first to develop a vaccine for an inherited form of amyotrophic lateral sclerosis (ALS), a progressive disease of the central nervous system that remains incurable.

Tested in mice, the study led by Laval University neuroscientist Jean-Pierre Julien, is a completely new approach to finding therapies for Lou Gehrig’s disease which affects two to five per cent of all ALS cases.

The vaccine delayed the onset of the disease, and also prolonged the life of the sick mice by about one month, or 10 per cent longer.

Scientists are cautiously optimistic about the discovery , published Monday in the online version of The Proceedings of the National Academy of Sciences journal.

The vaccine is based on mouse models of ALS and it remains to be seen whether it could work in people, said Philip Wong, professor of neuropathology and neuroscience at Johns Hopkins University.

"But it’s a very, very intriguing finding that could provide us with a novel therapeutic strategy," said Wong, a director at the university’s Robert Packard Centre for ALS, which funded some of the research.

The inherited form of ALS is caused by a mutant gene, SOD1, which produces a toxic protein.

Mice carrying the mutant SOD1 gene develop ALS and die. Those with normal SOD1 are fine, Julien said.

It’s yet not known how, but the excreted protein contributes to the death of the nerves that enable muscle movement. That idea – the protein – became an attractive research target, Julien said.

"Let’s try to have the immune system attack and neutralize the toxic protein," he said.

Animals engineered with a mutant human gene responsible for a familial form the disease but inoculated before the usual onset of symptoms survived disease-free much longer.

They also lived longer.

"It’s a terrible neurological disease," Julien said. Most people die within two to five years of diagnosis, usually of respiratory problems.

Many drugs have been tried for the treatment of ALS. Only one was found to have a modest effect, Rilutek (riluzole); it seems to prolong patients survival by about three months.

The fact that immunization makes a difference in the familial form of ALS paves the way similar approaches in other forms the illness, said Jeffrey Rothstein, professor of neurology and neuroscience at Johns Hopkins University and head of Packard.

"It gives us a different way of thinking about the disease... of trying to discover mechanisms for how the disease occurs, and that’s very important," Rothstein said.

The non-profit Robert Packard Centre for ALS is part of the Johns Hopkins School of Medicine in Baltimore, MD.

cfidelman@thegazette.canwest.com