Scientists tout promising results from new ALS animal study

An enzyme known as activated protein C, or APC, was able to significantly prolong the lives of mice involved in a study of amyotrophic lateral sclerosis, better known as ALS or Lou Gehrig's disease. And researchers say they eventually want to find out if the same approach could help human patients with the fatal ailment.

Researchers say that mice treated with APC experienced a 25 percent increase in lifespan, extended the time they were able to function effectively and reduced the rate of muscle wasting. And while they say it could be five years before a treatment like this could be tested in humans, they were encouraged by the fact that they were testing a compound already in use for another disease.

The compound was tested in mice engineered with a mutation in a gene known as superoxide dismutase 1, which keeps cells safe from free radicals. SOD1 is believed to play a role in triggering at least a small percentage of ALS cases. "The success of this research project has been very gratifying, and we are hopeful that a form of APC will ultimately be useful as a treatment for this disease," said Zlokovic, who is professor of Neurosurgery and Neurology and director of the Center for Neurodegenerative and Vascular Brain Disorders at Rochester.

- check out the report on the project

Suggested Articles

Compass' CD137 agonist cleared large tumors in mice that other I-O agents had failed to treat. It's advancing the drug into phase 1 human trials.

UPMC researchers are planning clinical trials of a COVID-19 vaccine that uses pieces of the virus' spike protein to create immunity.

Treating mice with niacin increased the number of immune cells in glioblastomas, reducing tumor size and extending survival.