COMMENTARY

Top Studies in Neurology From 2023

Hans-Christoph Diener, MD, PhD

Disclosures

January 04, 2024

This transcript has been edited for clarity.

Dear colleagues, I'm Christoph Diener from the medical faculty of University Duisburg-Essen in Germany. Today I want to report the 10 most important studies published in 2023.

New Drugs in Alzheimer's and ALS

Number one: monoclonal antibodies against beta amyloid for the treatment of early Alzheimer's disease. For two monoclonal antibodies, lecanemab and donanemab, there was efficacy in slowing the disease progression. In practical terms, these two treatments pose significant problems. First, for diagnosis, you need PET-CT. The therapy has to be done via IV regularly, every 2 weeks or every 4 weeks. There are a number of side effects, which need MRI control, and the treatment is very expensive. We have, at present, no infrastructure for treating these patients. We also have a number of monoclonal antibodies that were not effective.

Number two: amyotrophic lateral sclerosis. There is a small subgroup, 1%-2% of patients, who have mutations in the SOD1 gene. There is now a new antisense oligonucleotide called tofersen, which reduces the concentration of SOD1 [protein] in the cerebrospinal fluid. There is a clinical phase 2 trial ongoing for efficacy.

Epilepsy, MS, and Gliomas

Number three: modern antiepileptic drugs taken during pregnancy in females with epilepsy. There is a study that showed that modern antiepileptic drugs like lamotrigine and levetiracetam have no effect on cognitive function in children aged 3 years. Remember, valproic acid and topiramate are clearly contraindicated in females with epilepsy during pregnancy.

Number four: There is an interesting disease called radiologically isolated syndrome. These are patients who get an MRI for whatever reason and they have signs of multiple sclerosis. We have two placebo-controlled studies for dimethyl fumarate and teriflunomide showing that you can delay the onset of the first episode of the disease compared with placebo.

The fifth study is a study in low-grade gliomas. There is a dual inhibitor of the mutated enzymes, IDH1 and IDH2, and this is called vorasidenib. This drug clearly improves the prognosis of patients with low-grade gliomas, and this is the first time that there is an effective treatment.

Migraine, Back Pain, and Stroke Treatments

Number six is the issue of triptan nonresponders in acute migraine attacks. The companies that produce the gepants and lasmiditan claim that up to 30% or 40% of patients are nonresponders to triptans. A study from the German Migraine Registry showed that triptan nonresponders do not exist if patients are given the opportunity to try three different triptans.

Number seven: We have very effective monoclonal antibodies against CGRP for the prevention of migraine, but 30% of patients will not respond. There is hope because there is another molecule involved in migraine, which is called PACAP. At the International Headache Congress, there was a placebo-controlled study that showed that a monoclonal antibody against PACAP is superior to placebo. We hope that this might be effective in people who do not respond to monoclonal antibodies against CGRP.

Number eight: acute back and neck pain. A large study from Australia clearly showed that opioids are not superior to placebo, and they should not be used also to avoid addiction.

Number nine: thrombectomy for large ischemic strokes. We were afraid to treat these patients out of fear of an increased risk for intracerebral hemorrhage, but this is not the case. There is a tension study with 253 patients that clearly shows that thrombectomy is superior to standard of care. We now have three studies showing that thrombectomy is also effective for large ischemic strokes, with only a small increase in intracerebral hemorrhage.

Remote Ischemic Conditioning

Finally, in 2022, there was a large study from China showing that remote ischemic conditioning in acute stroke might be effective. This is done by intermittent ischemia in the upper extremities. Now there is a new study in Europe with 1500 patients, which showed no benefit of remote ischemic conditioning, even if the treatment was started on the way to the hospital.

Dear colleagues, I've discussed 10 important publications and studies from 2023 which have impact for the treatment of our patients in neurology. I'm Christoph Diener from the medical faculty of University Duisburg-Essen. Thank you very much for listening and watching.

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