News & Views in 2016

As yet, no clear strategy has been developed for discontinuation of disease-modifying therapy in multiple sclerosis. A recent observational study adds new information, but the most informative patient groups are still to be assessed comprehensively, and several questions need to be addressed in prospective studies.

Guide RNA-mediated CRISPR–Cas nucleases are a powerful technology for the engineering of mammalian genomes. CRISPR–Cas9-dependent editing of mutated genes that cause Huntington disease and fragile X syndrome was recently achieved in cell-based models, heralding the first step towards developing this technology into viable therapeutics for neurological diseases.

A new systematic review detected strong evidence of unreliable results via use of statistical and logical analyses of 33 randomized clinical trials. Our outrage at these rare occurrences of potential fraud could be rooted in our disdain at our failures in peer review, given that this special effort was required to detect long-running potential fraud.

A cross-sectional community-based study shows that strictly lobar cerebral microbleeds are associated with cognitive impairment, and might be a surrogate marker for early small-vessel injury. The anatomical distribution of cerebral microbleeds suggests that cerebral amyloid angiopathy (with or without Alzheimer disease) might have a key role in early cognitive impairment.

A randomized placebo-controlled phase III trial of adjuvanted herpes zoster subunit vaccine has shown a substantial improvement in vaccine efficacy in adults aged ≥70 years compared with the currently used, live attenuated vaccine. The finding has profound implications for reducing illness burden, although the duration of vaccine protection needs further evaluation.

In a randomized, sham-controlled trial, transcranial focused ultrasound thalamotomy was shown to benefit patients with medication-refractory essential tremor, although the technique has limitations and the long-term benefits are unclear. Nevertheless, the work suggests that focused ultrasound thalamotomy will become an alternative to other surgical procedures for refractory essential tremor.

Analysis of data from a large number of deep brain stimulation (DBS) recipients in North America has revealed a higher rate of revisions and removals of DBS leads than was previously reported. Hardware malfunction, lead infection and malpositioning were the most frequent reasons for revision or removal.

Studies of preclinical Alzheimer disease (AD) have unexpectedly shown amyloid-β deposition and/or AD-like neurodegenerative changes in the brains of a high proportion of clinically normal elderly individuals. As two recent reports illustrate, imaging and fluid biomarker studies in these individuals are yielding new insights into the pathophysiology of cognitive ageing.

A recent study proposed a modified version of the current diagnostic criteria for neurocysticercosis, but the value of the modifications is unclear and the study design limits conclusions about the validity of the new criteria. Instead, the suggested changes might contribute to future revision of the existing diagnostic criteria.

According to new research, oestrogen therapy in postmenopausal women is associated with ventricular enlargement and increased white matter hyperintensities in the brain, but not with cognitive decline. This disconnect between structural and functional effects suggests that brain-derived lipids can be harnessed to meet the bioenergetic demand imposed by normal cognition.

According to a recent study, a high percentage of children with epilepsy show comorbid somatic, neurological, and developmental or psychiatric disorders. To provide comprehensive care for paediatric patients with epilepsy, all their needs must be evaluated and managed, including careful consideration of comorbid disorders.

Recently published American Headache Society evidence-based guidelines for cluster headache management provide updated guidance on which therapies are superior to placebo in randomized controlled trials. These valuable recommendations do not always translate to real-world settings, however, and other criteria should be taken into account when attempting to treat cluster headache.

A recent observational study showed that patients with stroke who participated in clinical research received better care and had lower mortality than patients who did not participate. However, the study has several limitations, and the available evidence suggests that patients should not be advised that participation in research improves outcomes.

In a powerful display of international collaboration, a new genome-wide association study has mapped susceptibility loci for migraine on the basis of genetic data from 375,000 individuals. The study implicates several new loci in migraine — many of them suggestive of vascular pathophysiology — but the functional relevance of many now-detected risk alleles remains elusive.

A new study has shown that dietary supplementation with a serotonin precursor reduces seizure-induced respiratory arrest (S-IRA) and death in mouse models. Development of strategies to prevent S-IRA is paramount, because S-IRA is thought to be associated with a high risk of sudden unexpected death in epilepsy (SUDEP).

Recently published findings from the ENDORSE study provide 5 years of randomized safety and efficacy data for two doses of dimethyl fumarate (DMF) in multiple sclerosis. The report complements results from the pivotal CONFIRM and DEFINE trials; however, postmarketing data from clinical practice is needed to complete the risk–benefit profile of DMF.

A recent study demonstrates that amyloid-β (Aβ) can function as an antimicrobial peptide, and additional data show that bacteria and yeast can seed Aβ deposition into amyloid. These data suggest a complex interplay between the normal function of Aβ, its accumulation in the brain, and host immune defence.

A new meta-analysis, which evaluated individual patient data from randomized trials of aspirin in patients with non-disabling acute brain ischaemia, found this drug to be much more efficacious at reducing early stroke recurrence than was previously estimated.

Alteplase is widely used for acute stroke at a dose of 0.6 mg/kg in Asian patients, whereas the standard in Europe and the USA is 0.9 mg/kg. The ENCHANTED study did not show that 0.6 mg/kg alteplase is noninferior to 0.9 mg/kg, as previously suggested, raising questions about standard practice in Asia.

The American Academy of Neurology (AAN) has updated its guidelines on the use of botulinum neurotoxins in neurological disorders. The new guidelines provide individual recommendations for the four products that are commercially available in the USA; however, the clinical differences between the products are still not clear.