At the International Symposium on ALS/MND in Orlando, Florida, scientists, neurologists, and people with ALS were excited to hear about clinical progress to treat motor-neuron disease. Attendees pondered the preliminary, but positive, results of gene therapy trials in spinal muscular atrophy—results that may open the door for similar gene therapy in ALS. They heard news of completed and ongoing trials of edaravone, a drug that may slow ALS in certain people, and about therapeutics that relieve distressing problems with speech and swallowing. Basic science also made a showing. A better understanding of how pathogenic proteins travel from cell to cell emerged, and researchers debuted new genetic variants to explain some inherited ALS risk.
Trials of gene therapy for spinomuscular atrophy and stem cell treatments for ALS look promising.
Studies find that a drug in cough syrup can improve symptoms, and that radiation therapy safely shuts off a salivary gland.
The drug benefits some patients with mild amyotrophic lateral sclerosis, and is approved in Japan.
The prion concept continues to infiltrate the neurodegeneration field, with models of SOD1 propagation and a bevy of tau strains.