Oregon State University researchers have surprised the medical world in recent months with the announcement of a new approach to stop the progression of amyotrophic lateral sclerosis — more commonly known as Lou Gehrig’s disease after it was diagnosed in the American baseball legend in 1939.
Studies done with laboratory mice show that use of a compound called copper-ATSM essentially stopped the progression of ALS and allowed the mice to approach their normal lifespan. The work was published in the journal Neurobiology of Disease.
It’s not known yet whether the compound, after trials first for safety and then efficacy, will have the same results in humans. If it does, the work may become one of the most significant biomedical accomplishments ever to emerge from the university, and it could provide hope for people around the world who
struggle with this debilitating and fatal disease.
The discovery reflects more than a decade of work by Joe Beckman, a distinguished professor of biochemistry and biophysics in the College of Science and holder of the Burgess and Elizabeth Jamieson Chair at Oregon State’s Linus Pauling Institute. Beckman has said he was “shocked” at how well the treatment stopped the progression of disease in mice. But questions remain about how it will translate to humans.
“We have a solid understanding of why the treatment works in the mice, and we predict it should work in both familial and possibly sporadic human patients,” Beckman says. “But we won’t know until we try.”
ALS currently has no therapy, despite decades of work toward one. The new approach is based on bringing copper into specific cells in the spinal cord and mitochondria that are weakened by copper deficiency. This metal helps to stabilize superoxide dismutase, an antioxidant protein whose proper function is essential to life.
Doctors in the College of Veterinary Medicine hope to try the new approach on a similar disease in dogs, and other work to begin human trials is progressing — while people around the world watch and hope.
For more information about the Linus Pauling Institute, visit lpi.oregonstate.edu.