Unmet Need
Amyotrophic Lateral Sclerosis (ALS) affects as many as 30,000 people in the United States alone, with 5,000 new cases diagnosed every year. ALS is a fatal neuromuscular disease that is characterized by a progressive degeneration of the motor nerve cells and spinal cord. There are only two drugs approved by FDA for the treatment of ALS, and both treatments are designed to slow the progression of the disease. About 10% of ALS cases are inherited and over 20 mutated genes have now been recognized as markers of familial ALS. However, the other 90% of ALS cases are sporadic and caused by unknown factors. Given the huge unmet therapeutic need in this field, there is an urgent demand for effective treatments for ALS patients.
Technology Overview
Johns Hopkins researchers have identified the arachidonic acid (AA) pathway, involved in lipid metabolism, as a novel therapeutic target against ALS. They performed transcriptomics and metabolomics studies to identify common metabolic perturbations in both inherited and sporadic ALS spinal motor neurons (sMNs), and discovered that AA levels were aberrantly high in ALS sMNs. Pharmacological inhibition of AA metabolism by the treatment of 5-lipoxygenase (5-LOX) inhibitor reduced AA level, which was sufficient to reverse ALS-related phenotypes in both in vitro human sMN and in vivo Drosophila models. These findings suggest that AA metabolism may be a source of promising drug targets for both familial and sporadic ALS cases, and 5-LOX inhibitors could potentially be developed as therapeutics against ALS.
Stage of Development
The inventors are pursuing further testing of 5-LOX inhibitors as therapeutics in ALS models.
