Drug may Slow ALS

A compound closely related to a drug already used to treat sepsis shows promise in slowing the progression of ALS, a deadly condition for which there is currently no cure.

In a study examining the effects of an enzyme called activated protein C (APC) on cell death caused by ALS, researchers found the compound extended the lifespan of mice with an aggressive form of the disease by about 25 percent — a significant amount. The compound also lengthened the amount of time mice were able to function well despite showing symptoms of ALS and reduced the pace of muscle wasting.

Researchers say they are encouraged the treatment is a cousin to one already proven safe and hope to test the compound in human patients within five years.

“The success of this research projects has been very gratifying, and we are hopeful that a form of APC will ultimately be useful as a treatment for this disease,” corresponding author Berislav Zlokovic, M.D., Ph.D., of the University of Rochester Medical Center, was quoted as saying.

The compound worked by slowing the activity of a genetic mutation called SOD1, which weakens the natural barrier between the blood and spinal cord and causes nerve cell death. The drug also eliminated the flow of dangerous byproducts of hemoglobin into the spinal cord, which protected neurons from damage.

One barrier to treatment of ALS patients with the enzyme is a potential risk of increased bleeding, a side effect of the current form of APC used to treat sepsis. – Journal of Clinical Investigation