Neurologists will see groundbreaking treatment approaches this year -- notably in rare disorders like Huntington's disease, hereditary amyloidosis, and narcolepsy -- plus discoveries of novel drug delivery systems and biomarkers at the in Los Angeles.
Researchers will show the results of the first in-human clinical trial of an antisense oligonucleotide designed to target huntingtin (HTT) mRNA in patients with early manifest Huntington's disease.
"Antisense oligonucleotides [nusinersen (Spinraza)] really made a splash last year in SMA type 1, which is the most severe type of spinal muscular atrophy disorder," said Natalia Rost, MD, of Massachusetts General Hospital in Boston and chair of the AAN meeting scientific committee.
"This year, we will see data about an antisense oligonucleotide and Huntington's disease, which brings us a glimpse of hope for these patients." In the clinical trials plenary session on Tuesday, Sarah Tabrizi, MBChB, PhD, of University College London in the UK, will highlight the results of the HTT-lowering drug trial.
Another rare disease treatment breakthrough will be featured in Tuesday's clinical trials plenary session. David Adams, MD, PhD, of the French National Reference Center for Familial Amyloid Polyneuropathies, will show data from the phase III APOLLO study of patisiran, an investigational RNAi therapeutic that reduces production of mutant and wild-type TTR in patients with hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy.
Also in the clinical trials plenary session lineup Tuesday is a presentation by Michael J. Thorpy, MBChB, of Montefiore Medical Center in Bronx, New York of phase III results of solriamfetol, a selective dopamine and norepinephrine reuptake inhibitor for narcolepsy types 1 and 2. "The researchers have been able to demonstrate efficacy across almost every measure they used in narcolepsy," Rost noted.
Discoveries and Debates
On Sunday, a group from the University of Tokyo will discuss what Rost calls "a very clever design to sneak medications into the central nervous system like a Trojan horse." Hiroya Kuwahara, MD, PhD, will review novel technology for Glc-micelle, a supramolecular micelle that uses glucose to cross the blood-brain barrier to deliver high molecular drugs such as oligonucleotide and monoclonal antibody therapies.
On Monday, Gregory Van Stavern, MD, of Washington University in St. Louis will demonstrate how optical coherence tomography (OCT) and OCT angiography (OCTA) may able to identify the much sought-after group of patients with preclinical Alzheimer's disease.
And in Thursday's "Controversies in Neurology" session, neurologists will debate whether biomarkers alone should be used for Alzheimer's diagnosis, whether children should play contact sports, and whether neurologists should be responsible for taking care of patients with functional disorders.
New Stroke Guidelines
Hot topics in stroke education, including the new stroke guidelines announced at International Stroke Conference in January, will be reviewed and discussed throughout the meeting, Rost added.
"On Monday night, there will be a special session and discussion about expanding the window for stroke treatments," she said. A poster session Monday will look at arto respond to patients who could benefit from the extended stroke code window. And in Tuesday's clinical trial plenary session, Gregory Albers, MD, of Stanford University will discuss the results of the DEFUSE 3 study that were presented earlier this year.
Late-Breaking Abstracts
The Tuesday will showcase late-breaking research, including an interim analysis of nusinersen in the SHINE study of spinal muscular atrophy (SMA), a novel pre-mRNA splicing modifier treatment known as RG7916 for SMA, efficacy and safety of migraine drugs ubrogepant in the phase II ACHIEVE I study and erenumab in the phase IIIb LIBERTY study, and the following abstracts:
- INTREPID: A Prospective, Double Blinded, Multicenter Randomized Controlled Trial Evaluating Deep Brain Stimulation with a New Multiple Source, Constant Current Rechargeable System in Parkinson's Disease
- ABX-1431, A First-in-Class Endocannabinoid Modulator, Improves Tics in Adult Patients with Tourette Syndrome
- Effect of Fingolimod in Pediatric MS: Further Insights from Sensitivity, Supportive and Post-Hoc Analyses from PARADIGMS
- MRI and Relapse Results for ALKS 8700 in Patients with Relapsing Remitting Multiple Sclerosis: 1-year Interim Results from the Phase 3 EVOLVE-MS-1 Study
- Ezutromid Significantly Reduced Muscle Damage whilst Maintaining Utrophin in Patients with Duchenne Muscular Dystrophy (DMD) after 24 Weeks of Treatment