NU 2011-071

Inventors

Teepu Siddique*

Thomas Lukas

Hasan Arrat

Short Description

Discovery of ALS compounds that decrease SOD1 expression with diminished toxic side effects

Abstract

Northwestern researchers have discovered compounds that target and downregulate Cu, Zn-superoxide dismutase (SOD1) for potential treatment of patients with familial ALS. After screening compounds using a cell-based in vitro assay, they focused their drug discovery on those that reduced mutant SOD1 protein expression in vitro and in transgenic ALS model mice. One compound was identified as potent with neuroprotective properties. It effectively reduced mutant SOD1 expression in brain and spinal cord without toxic side effects. The investigators believe this compound may now be exploited to develop orally active drugs to treat SOD1-linked ALS.

Application

• Therapeutics: Amyotrophic Lateral Sclerosis (ALS)

Advantages

• Dose-dependent reduction
• Potential for oral drug development
• Decreased cell toxicity
• Longer in vivo half-life
• Successfully tested its efficacy in ALS mouse model

IP Status

Issued US Patent No. 9,669,014