Revalesio, a US-based clinical-stage pharmaceutical company developing treatments for acute and chronic neurological disorders, announced on Wednesday new data demonstrating that the company's investigational therapeutic, RNS60, protected motor neurons in the prpTDP-43A315T-UeGFP mouse model of Amyotrophic Lateral Sclerosis (ALS).
PrpTDP-43A315T-UeGFP mice express a mutation of the human TDP43 gene and produce progressive motor neuron degeneration, resulting in gait abnormalities, muscle weakness and eventually death, imitating key features of ALS.
During this study, 30-day old prpTDP-43A315T-UeGFP mice indicating signs of degeneration were treated with RNS60 every other day for 60 days. Immunohistochemistry was conducted to quantitatively evaluate activated microglia (immune cells that patrol the central nervous system [CNS] for pathogens and damage) and activated astrocytes (cells in the CNS that perform metabolic, structural, homeostatic and neuroprotective tasks). Ultrastructural integrity of neuronal mitochondria was studied through electron microscopy. The company says that RNS60 treatment helped in improving mitochondrial ultrastructure in the motor neurons, reduced the extent of astrogliosis and microgliosis in mice, and protected health and stability of upper motor neurons and neuromuscular junctions compared to placebo-treated mice.
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