Researchers inadvertantly discover potential ALS drug

IOWA CITY, Iowa A drug has been identified University of Iowa researchers that, nearly doubles the life span of mice with inherited ALS, a fatal neurodegenerative disease that destroys motor nerve cells in the brain and spinal cord, also known as Lou Gehrig's disease. The drug was discovered after an unexpected reaction between proteins, according to the Washington Post.

The researchers were studying the biology of cell signaling when they made the unexpected discovery that superoxide dismutase-1, a protein that’s mutated in inherited forms of ALS, interacts with Rac1, a protein that regulates production of reaction oxygen species by the Nox2 protein complex. Reaction oxygen species is essential for normal cell function but abnormal production is a suspected cause of ALS and other neurodegenerative diseases.

The researchers first found that deletion of the Nox2 protein almost doubled the life span of mice with inherited ALS. This provided further evidence that Nox-2 generated reaction oxygen species may play a role in ALS progression. The researchers then found that a drug called apocynin, which blocks Nox2, slows ALS progression and increases life span of mice with inherited ALS.

The researchers also noted that extensive safety and efficacy testing in pre-clinical trials must be conducted to determine if apocynin is effective in people.