Amyotrophic lateral sclerosis (ALS) is a late onset neurodegenerative disease characterized by a progressive loss of motoneurons in the motor cortex, brain stem and spinal cord. Most ALS patients have the sporadic form of the disease, while 5-10% of ALS cases are of the familial type caused by autosomal dominant mutations of the human Cu-Zn superoxide dismutase 1 (hSOD1) gene. ALS symptoms are characterized by muscle weakness, atrophy, spasticity, and paralysis. Most people with ALS die of respiratory failure or pneumonia. Death usually occurs within two to five years of diagnosis.
There is no cure for ALS. Despite the improvement in symptomatic treatment of ALS, a disease-modifying therapy is still unavailable, in part due to the lack of knowledge on its causative mechanisms. Riluzole therapy has been shown to only improve the survival of ALS patients by a few months and has several side-effects, including asthenia, nausea, anorexia, and diarrhoea.
Neurotec Pharma is developing new therapeutic approaches to treat effectively and safely the progressive degeneration of motor neuron systems that occurs in ALS. Our treatments can significantly reduce the cascade of reactions that culminate in apoptotic cell death in order to delay or decrease the accumulation of neurological disability of patients. The proposed treatments inhibit microglial reactivity, thus preventing the release of a proinflammatory signal that may damage the spinal motor neurons.