A biological treatment targeting the protein CD40L has a therapeutic effect on mice with amyotrophic lateral sclerosis (ALS), reports a new study published in Nature Genetics. The study suggests that ALS progression may be amenable to treatments that target the immune response.
ALS is a progressive neurodegenerative disease that leads to neuron loss in the brain and spinal cord and eventual paralysis. ALS affects approximately 2 per 100,000 individuals worldwide and is typically fatal within 5 years of diagnosis.
Steven Perrin and colleagues administered a monoclonal antibody to CD40L to mice with a mutation in SOD1 ― a commonly used mouse model for ALS. They found that the anti-CD40L antibody delayed onset of paralysis and on average, led to a 40% extension of life duration after the first signs of paralysis.