Standout presentations at this year’s American Academy of Neurology annual meeting range from targeting tau in Alzheimer’s disease, to new treatments for spinal muscular atrophy, to the controversial topic of allowing your child to play contact sports, but all are sure to have an impact, according to Clinical Neurology News Medical Editor Richard J. Caselli, MD.
“There are a lot of good talks and papers being presented, and it is impossible without having seen and heard them all to accurately predict what will be the real standouts, but from a purely personal perspective, and with all due apologies to any others not mentioned below, these are some of the ones I think could have large and, in some cases, almost immediate impact or potential impact,” said Dr. Caselli, professor of neurology at the Mayo Clinic Arizona in Scottsdale and also associate director and clinical core director of the Arizona Alzheimer’s Disease Center.
Targeting tau in Alzheimer’s
Among the Aging and Dementia oral abstract sessions, presentations on “the monoclonal antibody trials aimed at amyloid continue to be of interest,” said Dr. Caselli. But “I was more intrigued by the abstract by Mignon and colleagues about a tau-lowering antisense oligonucleotide in patients with Alzheimer’s disease.” This study was designed to assess the safety, tolerability, and pharmacokinetic parameters of repeated intrathecal injections of the tau-lowering antisense oligonucleotide IONIS-MAPTRx in patients with mild Alzheimer’s. It’s set to be presented during the Sunday, April 22, session S2, “Clinical Trials and Therapeutic Approaches in Neurodegenerative Diseases.”The measuring of plasma tau to detect preclinical Alzheimer’s, as is described in the abstract from Pase and colleagues, is an “intriguing” approach, Dr. Caselli said. In that study, higher plasma tau levels were observed across correlates of preclinical Alzheimer’s: poorer cognitive function and smaller hippocampal volumes on MRI. Plasma tau level was also a strong predictor of future dementia. It will be presented Friday, April 27, 1:00-3:00 in S48, “Novel Biomarkers in Aging and Dementia.”
Focus continues on SMA
More advancements continue to be made in the treatment of various forms of spinal muscular trophy. In Monday morning’s Presidential Plenary Session, Richard Finkel’s presentation in receipt of the Sidney Carter Award in Child Neurology, should chart the development, current state, and future of antisense oligonucleotide therapy for SMA.
In the Emerging Science poster program on Wednesday, April 25, attendees will get an update on trial results for a different approach to the treatment of SMA using AVXS-101 gene replacement therapy for SMA type 1. John W. Day, MD, PhD, will provide longer-term outcomes after last year’s presentation of results in 15 patients.