ALS, also called Lou Gehrig’s disease, is a neurodegenerative disease, in which slow death of motor neurons causes paralysis and ultimately death, within 3-5 years of diagnosis. To date, there is no effective cure or treatment.
Two key mechanisms involved in ALS pathogenesis are neuroinflammation and dysregulation of RNA metabolism.
Neuroinflammation is increasingly recognized as a major factor that promotes disease progression and amplifies the motor neuron death-inducing processes.
Additionally, studies show that microRNAs (miRNAs) are dysregulated in motor neurons of patient’s tissues, and that reduction in miRNAs is sufficient to cause spinal motor neuron degeneration.
These two pathways are targeted simultaneously by our combination drug, PrimeC. It has shown outstanding results in animal models of ALS, both in motor movement and in morphology, of motor neurons and other cells affected by the disease.
NeuroSense Therapeutics is currently preparing for a Phase II clinical trial in ALS patients.