August 2020

Dear Colleagues,

I suspect that most of us have been consumed with the COVID-9 pandemic, the social justice efforts, and the political maelstrom that this country is in. I sincerely hope that you have had a chance to take some time off from work to relax and reflect. In catching up on some reading in the past few weeks I came away with a renewed optimism for recent advancements in neurology therapeutics. Our field is truly making progress, and our patients will benefit greatly from these discoveries. Organizations like the ANA will be critical to help create and maintain the productive milieu that will continue to push discovery.

These are just a few of the new therapies that caught my eye.

1. Viltolarsen Approved for Duchenne Muscular Dystrophy Treatment. The drug is indicated in the 8% of people with DMD who have confirmed mutation in the DMD gene that is amenable to exon 53 skipping. The drug, like other Duchenne therapies approved by the FDA, is not supported by proof that it can modify the course of disease, but it does increase dystrophin levels1.

2. Risdiplam Approved as First Oral Treatment for SMA. The agent, marketed under the brand name Evrysdi, was approved based on the findings of two small clinical trials, the FIREFISH and SUNFISH studies. The agent is anmRNA splicing modifier that increases systemic SMN protein concentrations by improving the efficiency of SMN2 gene transcription2.

3. Rituximab appeared to be more effective and lead to fewer treatment discontinuations in people with multiple sclerosis (MS) than Gilenya (fingolimod) and Tecfidera (dimethyl fumarate), according to real-world data based on two years of therapy. Rituximab’s effectiveness appeared to be comparable to that of Tysabri (natalizumab), but with fewer treatment discontinuations3.
 
4. Head to head comparisons in two simultaneously conducted active-controlled trials of ofatumumab, a SC anti-CD20 monoclonal antibody vs teriflunomide, an oral inhibitor of pyrimidine synthesis that reduces T- and B-cell activation. In 2 trials relapse rates were 0.11 and 0.10 vs 0.22 and 0.25. Ofatumumab was superior to terflunomide in suppressing lesion activity on MRI scans and also lowered serum concentrations of NFL, a marker of neuraxonal damage. Serious infections occurred in 2.5% and 1.8%4.

There is positive news from the field of neuroepidemiology too. The incidence rate of dementia in Europe and North America appears to have declined by 13% per decade over the past 25 years, consistently across studies. Incidence is similar for men and women, although declines were somewhat more profound in men. These observations are important because they affect the projections of the impact of dementia on public health. If the determinants of this decline could be identified and were modifiable then even greater declines might be feasible5.

Finally, don’t forget to complete the ANA Diversity Engagement Survey. This will provide data about the attitudes within the ANA about diversity, equity and inclusion, and will propel us towards action steps that we will discuss during the Social Justice Symposium on Saturday, October 3, 2020.

Warm regards,

Justin C. McArthur, MBBS, MPH
President, American Neurological Association
John W. Griffin Professor of Neurology and Director, Department of Neurology
Johns Hopkins University School of Medicine