RNA-Seq reveals a novel therapeutic strategy for ALS treatment

Amyotrophic lateral sclerosis (ALS) is a fatal heterogeneous neurodegenerative disease that lacks effective treatment option. Researchers at Genervon Biopharmaceuticals hypothesized that the traditional single-target approach to treat neurodegenerative disease may not be effective due to the complexity of these conditions and their multifactorial pathogenesis.

Using a systems biology approach, they discovered that GM6, a 6-amino acid peptide drug modeled upon an endogenous embryonic stage neurotrophic factor, has multi-target effects consisted with a neurotrophic factor, leading to improved survival of neurons in neurodegenerative patients.

RNA-seq was used to determine whether ALS-associated genes exhibit unique responses to GM6 treatment in SH-SY5Y cells, which would suggest that GM6 is impacting signaling pathways linked to processes that underlie ALS onset and/or progression. Bioinformatic analyses identified GM6-regulated genes with potentially important roles in ALS development and progression. These genes were categorized with respect to 6 hypothesized mechanisms of action (MOA). This MOA posits that GM6 protects neurons by combating oxidative damage while bolstering neurogenesis, microtubule stability, immune- mediated neuroprotection, synaptic transmission and glutamate clearance.


Genervon Biopharmaceuticals presented its recent findings at the 28th national symposium on ALS/MND in Boston on Saturday, December 9, 2017.

The researchers have shown that GM6 promotes neuroprotection against toxic factors contained in CSF from patients with neurodegenerative diseases and importantly can cross the blood brain barrier. In the SOD1 mouse model of ALS, they demonstrated that GM6 delays disease onset and reduces mortality, with concomitant improvements in physiological function. Finally, the data in our Phase 2A clinical trial of only 2 weeks (6 doses) suggested a positive signal of GM6 in ALS patients with favorable shifts in disease biomarkers and functional measurements. The data supports further study. A Phase 3 study based on a longer treatment period and larger sample size is now being planned.

Source – BusinessWire

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