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ALS - Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, is the third most prevalent, adult-onset, neurodegenerative disease (about 30,000 cases in the US)1. ALS is an age-related, paralytic disorder characterized by the progressive death of motor neurons in the brain and spinal cord. Most cases, 90-95%, have undefined etiology and are classified as sporadic (sALS). Familial ALS (fALS) describes 5-10% of cases with genetic linkages. However, oxidative stress, mitochondrial dysfunction, and neuroinflammation are core pathological components of ALS.

Sadly, the prognosis for ALS patients has not changed the disease was first diagnosed in the 1860's; initially, ALS manifests as a focal weakness but rapidly leads to respiratory failure and death within 2-5 years.  Stephen Hawking, one of mankinds greatest minds, recently died of ALS.  For unknown reasons, veterans who have served in the United States Armed Forces are more likely to be diagnosed with ALS than people who did not serve in the military. As a result, the U.S. Department of Veterans Affairs (VA) has recognized ALS as a service-connected disease.

The high socio-economic costs (>$1 billion USD/year), the severity and rapid progression, and the paucity of disease-modifying treatments highlights the high unmet medical need in ALS for a novel, mechanism-based therapies yet offers a defined clinical path for rapid commercialization. Luciole’s technology enhances the activity of a key step in DNA repair, particularly mitochondrial DNA (mtDNA), to prevent a cascade of deleterious events leading to mtDNA deletions, fragmentation, mitochondrial dysfunction, inflammation, and motor neuron death.

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