Top 10 Myasthenia Gravis Stories of 2021
Throughout 2021, Myasthenia Gravis News has brought you coverage on the latest scientific advancements and clinical trials related to myasthenia gravis (MG).
We look forward to continuing to be a resource to the MG community across 2022.
Here are the top 10 most-read MG news stories of 2021, along with a brief description of what made them relevant for the MG community.
A person’s genetic makeup is thought to affect their susceptibility to autoimmune diseases like MG, but the role of specific genes in MG susceptibility is poorly understood.
An international team of scientists analyzed genetic data for 1,401 people with MG and 3,508 people without the disease, all of European ancestry. In addition to known MG risk variants, the analysis also identified the rs3128125 variant in a gene called AGRN as a risk factor for MG. The AGRN gene is involved in the function of the neuromuscular junction — the place where nerve cells communicate with muscle cells — which is damaged in MG.
Zilucoplan, an investigational therapy developed by Ra Pharmaceuticals (now part of UCB), is designed to block the activity of part of the immune system called the complement cascade, which is dysregulated in MG.
Following positive results from a Phase 2 trial, Ra is conducting the global and pivotal Phase 3 study called RAISE (NCT04115293). The goal of the double-blind, placebo-controlled study is to assess the effect of treatment on participants’ ability to engage in daily activities after 12 weeks. The trial has since completed enrollment, with up to 174 participants.
Myasthenia Gravis News conducted a survey of the MG community, which was answered by 743 people, most of whom were MG patients.
Nearly three-quarters of MG patients reported being generally satisfied with their treatment plan, although nearly half were dissatisfied with their quality of life to some extent. Fatigue was the most commonly reported symptom by these MG patients, as well as the one that most impacted their quality of life.
Although it is not exactly clear how MG develops, viral infections can help set the stage for the disease — and as the pandemic has progressed, there have been reports of MG developing after COVID-19 infection.
Here, scientists described the case of a 77-year-old man who developed MG after contracting COVID-19. A sensitive cell-based blood test revealed he was positive for antibodies targeting muscle-specific kinase, one of the less common types of MG-driving antibodies.
Argenx filed for its approval in the U.S. in early 2021, based on data from a Phase 3 trial called ADAPT (NCT03669588) that showed the therapy could ease symptoms of generalized MG. In December, the U.S. Food and Drug Administration (FDA) approved the medication to treat adults with generalized MG who are positive for anti-acetylcholine receptor antibodies (anti-AChR), the most common type of MG-driving antibodies.
Vaccines against COVID-19 first gained emergency use authorization in the U.S. in late 2020, and 2o21 was the year that these life-saving vaccines first became available to the general public. This led to a lot of questions, especially in rare disease communities.
In January, the National Organization for Rare Disorders (NORD) hosted a webinar, where experts discussed these vaccines and addressed some of the concerns. Broadly, experts stressed that available COVID-19 vaccines were generally safe and effective, and their benefits for rare disease patients outweighed the risks, including for patients with compromised immune systems or those participating in clinical trials testing gene therapies.
Scientists in Japan analyzed data from 104 MG patients being followed at their institution.
Patients who had dysphagia, or difficulty swallowing, early on in the course of MG were found to be less likely to reach a minimal manifestations of disease status later on. In other words, people with swallowing difficulties were more likely to have poor clinical outcomes. Low levels of a protein called albumin in the blood also were associated with worse MG symptoms.
The human digestive system is home to billions of bacteria that have a profound impact on health. Scientists in Germany analyzed gut bacteria of 41 people with MG, as well as healthy individuals and people with other diseases. Their analyses indicated that MG patients may have less diverse gut bacteria than healthy people.
According to researchers, this indicates that targeting abnormalities in these bacteria may be a promising avenue for future MG treatments.
Scientists in New Jersey described the rare case of a 77-year-old man who had a severe worsening of MG symptoms a few days after receiving the second dose of the Moderna COVID-19 vaccine. The man required intensive care in a hospital, but eventually was stabilized with treatment.
Researchers stressed that the benefits of vaccination still outweigh the risks for patients, but noted that people with MG may require more close monitoring after being vaccinated in case of rare severe reactions.
Researchers in the U.S. analyzed data from a global registry that included 40,392 MG patients, of whom 380 contracted COVID-19.
Compared with the general population in the database, people with MG were significantly more likely to be hospitalized due to COVID-19, and were more than twice as likely to die from the disease. Since vaccination can prevent severe disease from COVID-19, these results underscore the importance of vaccination for MG patients.
We at Myasthenia Gravis News hope that all of our reporting throughout 2021 has been helpful for people affected by MG, and wish all of our readers a happy 2022.