This transcript has been edited for clarity.
Dear colleagues, I'm Christoph Diener, from the medical faculty at the University of Duisburg-Essen. Today I would like to inform you about six studies that were published in five different indications in January 2021.
Let me start with an extremely exciting paper that came out in Science. It comes from a group of researchers led by Dr Ugur Sahin, who is one of the inventors of the new BioNTech genetic vaccine against COVID-19. In this study, they developed a messenger RNA vaccination for experimental autoimmune encephalomyelitis in mice, which is the equivalent of multiple sclerosis in humans. This vaccination had a therapeutic effect on autoimmune encephalomyelitis but no impact on other immune functions.
This would be a real breakthrough if it worked in humans. We could treat multiple sclerosis and have no additional impact on the immune system in terms of immunosuppression or immunomodulation. We will have to wait a few years to see whether this concept is really valid.
The second paper, which came out in Annals of Neurology, is about the treatment of Friedreich ataxia. Omaveloxolone has been shown in animal experiments of Friedreich ataxia to have a positive effect on mitochondrial function, and it suppresses inflammatory processes. This drug was therefore tested in a double-blind, randomized, placebo-controlled study in 103 patients with Friedreich ataxia.
Omaveloxolone had a significant positive effect on the Friedreich ataxia rating scale. The downside is that some patients developed increases in liver enzymes, but this was transient. Typical side effects included headache, nausea, and fatigue. The absolute treatment effect was not very high, but at least the drug had a significant benefit compared with placebo.
The DEVT study was performed in China with 234 patients, with the primary endpoint of a modified Rankin Scale score between 0 and 2. A good outcome was achieved in 54% of patients with monotherapy and in 46% with the combination of thrombectomy and thrombolysis, which was not a statistically significant difference.
A second study performed in Japan — the SKIP study — randomized 204 patients. The endpoint, again, was modified Rankin of 0 to 2 at 90 days. And good outcome was seen in 59% in the thrombectomy alone group vs 57% in the combined intravenous thrombolysis plus mechanical thrombectomy group. There were no differences in secondary endpoints between the groups.
Both of these randomized controlled studies were relatively small and didn't address the clinically truly relevant question. If a patient comes in with an acute stroke and large-vessel disease, and the angiography suite is empty, I think there is no doubt that they should go to thrombectomy immediately. But I also think that in cases where a patient has to wait for thrombectomy, or has to be transported to another stroke unit, they should receive thrombolysis and then thrombectomy is performed afterwards.
The next study was published in The Lancet. Researchers in the Netherlands asked whether it would make sense to give tranexamic acid in people with subarachnoid hemorrhage as soon as the diagnosis is made on CT. They randomized 955 patients to compare immediate tranexamic acid after diagnosis with placebo, using a primary endpoint of modified Rankin Scale score at 6 months.
Unfortunately, there was no difference between active treatment and placebo. So, obviously, giving tranexamic acid immediately in patients with subarachnoid hemorrhage has no benefit.
The last study was published in Lancet Neurology and deals with patients who have to undergo neurosurgery for cervical myelopathy. Researchers investigated whether riluzole is of benefit in these patients. As you all likely remember, riluzole is effective in amyotrophic lateral sclerosis. It's an NMDA antagonist that has anti-inflammatory properties. One group of patients received 50 mg riluzole twice daily for 2 weeks before and then for 4 weeks after surgery, whereas the other received placebo.
Unfortunately, again, there was no difference between the two treatment groups. This makes it quite obvious that riluzole is not helpful in people who have to undergo surgery for cervical myelopathy.
Dear colleagues, these were, I think, the most exciting studies in January 2021. I am Christoph Diener from the University of Duisburg-Essen in Germany. Thank you very much for listening and watching.
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Cite this: Hans-Christoph Diener. 6 Neurology Updates to Know - Medscape - Mar 22, 2021.