First RNA therapy shows improvement in man with rare motor neuron disease

A man with a slowly progressing, rare mutation-driven form of amyotrophic lateral sclerosis (ALS) has shown improved symptoms and continued working as a physician a year after becoming the first person to receive an antisense oligonucleotide therapy targeting his specific genetic mutation. The RNA-based drug uses short strands of genetic material to reduce production of the protein that builds up and kills motor neurons, differing from standard gene therapy. Results, published this week in Med, are described by University of Sydney neurologist Steve Vucic as an "exciting first step," though it remains too early to know if the treatment can halt progression or cure the disease; researchers plan to monitor the patient for another two to three years and test the drug in more people.