Amyotrophic lateral sclerosis (ALS), often called Lou Gehrig´s disease, is a fatal progressive neurodegenerative disease characterised by motor neuron degeneration. Degeneration of motor neurons results in both motor and non-motor symptoms such as muscle weakness, spasticity, emotional lability, dementia, and difficulty breathing.
The incidence of ALS is 1-2 cases a year per 100,000 people, and there are over 200,000 people living with ALS worldwide. Most cases (approximately 90%) are sporadic, meaning that there is no clear explanation or genetic component that is responsible for the development of the disease. However, familial forms of ALS accounts for 10% of all ALS cases, where mutations in genes such as SOD1, C9ORF72 and TARDBP are inherited from a family member. ALS is more common in men than women.
Currently, there are only three symptomatic treatments available in the United States, and only one in the EU. These do not offer substantially improved quality of life for ALS patients, but only prolongs their life by a few months. It is therefore imperative to develop new innovative treatments to enhance the life of these patients.
Here at 2N Pharma, we believe that mitochondrial dysfunction has a central role in the progression of ALS. ALS patients display lower fatty acid concentrations in blood, as well as decreased lipid derivates. This indicates that the metabolism has shifted from glucose to lipid metabolism. We are currently testing this hypothesis by using Mitometin to treat ALS-like symptoms in various animal models of ALS.