Credit: Philip Patenall/Macmillan Publishers Limited

Los Angeles provided the venue for this year’s American Academy of Neurology Annual Meeting (AAN 2018). The programme featured presentations from big names and rising stars in the neurology field and highlighted an array of promising new therapies.

Advances in migraine treatment came under the spotlight at AAN 2018, with a particular focus on therapies that target calcitonin gene-related peptide (CGRP) — a neurotransmitter that is released from sensory neurons during migraine attacks. Joel Trugman reported on ACHIEVE I, a phase II trial of the CGRP receptor antagonist ubrogepant for the acute treatment of migraine. The drug provided significant benefits with regard to pain and migraine-associated symptoms.

Monoclonal antibodies against CGRP are also proving beneficial in individuals with migraine. Uwe Reuter presented evidence that erenumab can provide pain relief in patients with difficult-to-treat migraine. In addition, Richard Lipton reported that patients with chronic migraine experienced a significant reduction in monthly migraine days in response to treatment with eptinezumab (8.2-day reduction, compared with 5.6 days in placebo-treated patients).

Antisense oligonucleotide (ASO) therapy received top billing at AAN 2018. Sarah Tabrizi reported on a phase I/IIa trial of IONIS-HTTRx in patients with early-stage Huntington disease. The treatment was well tolerated and produced dose-dependent reductions in levels of mutant huntingtin (mHTT) in the cerebrospinal fluid. Although the trial was not designed to measure efficacy, mHTT lowering was found to correlate with improvements in motor scores and cognitive function.

Richard Finkel provided an update on emerging therapies for spinal muscular atrophy (SMA), including the FDA-approved ASO nusinersen, and gene-replacement strategies. Small-molecule drugs could also have a starring role in the treatment of SMA in the future. Giovanni Baranelli presented data from the FIREFISH Part 1 trial, which tested the small molecule RG7916 in babies with SMA type 2. The drug was found to increase blood levels of survival motor neuron protein (SMN) by modulating SMN2 gene splicing. Trials are now underway to determine the clinical efficacy of RG7916.

Summarizing the recent therapeutic advances in SMA and other neuromuscular diseases in the final plenary session of the meeting, Ericka Simpson concluded that neuromuscular medicine is reaching a “tipping point”, catalysed by a “perfect storm of collaboration”.