Researchers from McMaster University and the University of Alberta have, for the first time, found a way to make mice with Huntington’s disease better.
Dr. Ray Truant’s lab worked with scientists from Alberta and discovered pumping measured amounts of ganglioside GM1, a lipid, into the sick rodents’ brains restored the mice to their normal motor functions.
While researchers expected to halt the disease’s progression or minimize its neurodegenerative effects, they were surprised to see the mice back to “completely normal,” Truant said.
“Not only did we halt the disease, but somehow the brain had managed to repair itself.”
Their groundbreaking paper has been published in the current edition of the journal Proceedings of the National Academy of Sciences and opens a door for research directed at finding a treatment for the genetic disorder.
GM1 is almost nonexistent in people with Huntington’s and other neurodegenerative diseases. So after the researchers pumped dosages of the compound into the animals for 28 days, the mice performed completely better at tests that measured how long the rodents could remain on a rotating rod or balance and walk on a beam.
With their paper out, they are now at the phase where they are looking for drug compounds that can mimic the effects of GM1, Truant said.
“I’m optimistic in that I think there’s going to be, in the three-to-five-year timeframe, a major advancement in clinical trials as a result of this,” he said. “It may not be GM1, but it may be a drug that acts like GM1.”
And the only way to test whether this research applies to human therapy is to conduct small-level clinical trials, he said.
Pharmaceutical companies have encountered safety and efficacy issues with administering the lipid in humans because it is difficult to synthesize and the alternative option was to purify it from sheep brain, Truant said.
But interest in a possible drug has been recently resurrected because there are now apparently ways to make part of it synthetically and part of it by purifying from “super clean” sheep, he said.
“It is important to understand that some treatments may work in laboratory models, but not in people. The applicability of the treatment discovered by Drs. Sipione and Truant to HD patients will be determined in clinical trials,” the Hungtinton Society of Canada’s CEO Bev Heim-Myers said in an email to The Spectator.
“We are optimistic that this research demonstrates real potential for an HD therapy.”
Truant is just as cautiously optimistic. His fear is that people will start using that dreaded “C-word,” the McMaster expert said.
“There are almost no cures in medicine, really. There are a lot of successful treatments. There are a lot of things that will improve the quality of life in people. And I think there’s a real hope in this in that respect.”