2017's Biggest Breakthroughs in Neurology

Hans-Christoph Diener, MD, PhD


December 21, 2017

Dear colleagues, I am Christoph Diener, a neurologist from the University of Essen in Germany. This is my review of the most important developments in the field of neurology in 2017.


An interesting study was published in the New England Journal of Medicine looking at epilepsy surgery in children and adolescents with treatment-refractory epilepsy.[1] A total of 116 children were randomly assigned to undergo epilepsy surgery or receive medical treatment. After 12 months, 77% of the children who had undergone surgery were seizure free compared with 7% who received medical treatment.

A second study, also published in the New England Journal of Medicine, analyzed histopathologic findings in 9523 patients who had epilepsy surgery.[2] In adults, the most frequent diagnosis was hippocampal sclerosis; and in children and adolescents, the most frequent diagnosis was focal cortical dysplasia.

Multiple Sclerosis

2017 saw a breakthrough in the treatment of primary progressive multiple sclerosis (MS) with ocrelizumab, a drug that depletes B cells. The ORATORIO trial[3] randomly assigned 732 patients in a 2:1 ratio to receive ocrelizumab or placebo. The investigators found clearly lower rates of clinical and MRI progression in the patients treated with ocrelizumab.

Spinal Muscular Atrophy

Perhaps the greatest progress was achieved in the treatment of spinal muscular atrophy (SMA). A phase 2 open trial of nusinersin, an oligonucleotide, was published in Lancet,[4] and the full phase 3 trial was published in the New England Journal of Medicine.[5] Nusinersin is an antisense therapy. This sham-controlled study in 78 children with infantile SMA showed that treatment with nusinersin leads to a 50% reduction in deaths or early ventilation.

A second approach was also studied,[6] with a single-dose gene replacement therapy for SMA in which the SMA gene is applied with a viral vector. This study comprised 15 patients; it showed an improvement in achieving motor milestones in these children after gene therapy.

Parkinson Disease

Two interesting analyses were completed in Parkinson disease (PD).[7,8] The first, published in Science, indicates that it is possible to influence the production of synuclein by influencing the alpha-synuclein gene.[7] This was first accomplished in cell culture, then the experiment was repeated in animals, and finally in a population-based study in Sweden.

The investigators checked more than 1000 pharmaceutical compounds in cell culture and found that the beta-2 adrenoreceptor can modulate the expression of synuclein. They identified patients who took beta-blockers in a Swedish database and found that patients who take propranolol for hypertension have an elevated risk of developing PD. By contrast, the risk for PD is reduced in patients who receive salbutamol because of asthma.

Another interesting finding is related to vagotomy. Multiple studies[8,9] now show that patients who underwent vagotomy for ulcers of the stomach 20-50 years ago have a lower risk of developing PD than do patients who did not have vagotomy. This would indicate that some pathogen actually travels via the vagus nerve into the brain and leads to the development of PD.


This year, we have two particularly interesting neuro-oncology studies; the first was published in Lancet.[10] This trial showed that patients with 1p/19q non-codeleted anaplastic glioma receive benefit when temozolomide is added to standard therapy.

The second study[11] investigated whether patients with progressive glioblastoma benefit from bevacizumab added to lomustine. Unfortunately, no survival benefit was seen compared with lomustine alone.

Pain and Headache

An Australian study compared pregabalin and placebo in 209 patients with acute or chronic sciatica.[12] Pregabalin was not effective at improving pain or other outcomes.

In the field of headache, we have an entirely new class of drugs for the prevention of migraine: antibodies against calcitonin gene-related peptide (CGRP) or the CGRP receptor. Now all phase 2 trials and the initial part of a phase 3 trial have been published.[13] The results show that these drugs are as effective as traditional preventive medication in both episodic and chronic migraine. The biggest advantage is that they work very quickly; it is possible to know whether the drug works within 4 weeks, and they have a very good side-effect profile. The downside will be the high cost of treatment.


The DAWN trial[14] investigated the efficacy of thrombectomy during a 6- to 24-hour time window in patients who were carefully selected when imaging showed a penumbra. This trial was positive in favor of thrombectomy.

Another trial investigated head positioning in patients with mild strokes and found no impact of head position.[15] This is not a surprise because these patients with mild strokes do not develop brain edema. A trial using routine low-dose oxygen supplementation in patients with acute stroke was also negative[16]; again, this was no surprise.

Perhaps the most important progress in stroke involved three trials that compared antithrombotic therapy and patent foramen ovale (PFO) closure in patients under age 60 years with cryptogenic stroke.[17,18,19] PFO closure clearly was superior to antithrombotic therapy.


Several studies now indicate that all anticoagulation in patients with atrial fibrillation may prevent long-term dementia, the most recent from Sweden.[20]

Finally, we now have two reversal agents for novel oral anticoagulants. Idarucizumab is already approved by the US Food and Drug Administration; and the other, andexanet alfa, will be approved shortly. Both agents result in a very quick reversal of the activity of dabigatran and factor Xa inhibitors.[21,22]

Ladies and gentlemen, 2017 was a great year for neurology, with a great deal of progress. My subjective feeling is that the two most important breakthroughs are in the treatment of SMA and the introduction of a totally new class of drugs for the prevention of migraine.

I am Christoph Diener, from the University of Essen in Germany. Thank you for listening. I hope we will see each other next year on Medscape.


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