Gene suppression significantly prolongs life in ALS: Potential for future blockbuster treatment?

Advances in the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, a fatal, rapidly progressive neurodegenerative disorder characterized by weakness, muscle atrophy and spasticity due to the selective loss of both upper and lower motor neurons, are discussed in a new report from industry experts GlobalData.

ALS is the most common motor neuron disease, affecting more than 86,000 people worldwide, and with an average life expectancy of three to five years from onset. There is currently no cure for ALS, while most treatments, such as Sanofi’s Rilutek (riluzole), only succeed in prolonging life by a moderate extent. Hence, given the great unmet need, novel treatments with improved efficacy are needed.

An important recent discovery may greatly aid the search towards a cure for ALS. Backed by funding from the ALS Therapy Alliance and CVS/Pharmacy, research led by Wim Robberecht found that suppression of EphA4, a gene involved in controlling output of motor nerve terminals, significantly extended the lifespan in worm and mouse models of ALS. Subsequently, investigators at the University of Massachusetts, USA, documented that rare defects in the same gene also promoted significant survival in human ALS patients, whereas Profilin-1 (PFN1), another gene within the same pathway as EphA4, was identified as an additional modulator of survival in ALS. Collectively, these findings highlight the potential for future ALS treatments involving the suppression of target genes such as EphA4.