The Myasthenia Gravis Foundation of America was founded by a patient advocate and mother of a Myasthenia Gravis (MG) patient. A rare disease, MG in its most common form is a chronic autoimmune neuromuscular disorder characterized by fluctuating weakness of the voluntary muscle groups. Symptoms include double vision, drooping eyelids, limb weakness, difficulty swallowing, or difficulty breathing. As is so often the case with rare diseases, founder Jane Ellsworth found that the resources available to her were inadequate, and established the MGFA to support efforts to find a cure and help patients and their families understand and cope with MG.
There are approximately 60,000 to 70,000 MG cases in the United States. For many years, MGFA was a small organization, often with a staff of one. However, it’s grown over the past ten years. “We’re getting to a place where we have a full complement of staff,” said MGFA Board Chair Nancy Law.
MGFA has had a long history of involvement in research, and funds research activities as guided by a research agenda of five priorities:
Biomarkers: facilitate early diagnosis, predict clinical outcomes and immunosuppressive therapy response and utilize in clinical trials
Disease Mechanisms: understand basic mechanisms and self-tolerance loss throughout the course of the disease
Targeted Therapies: develop new therapeutic targets, prevent widespread immunosuppression and off-target side effects, optimize treatment strategies with existing therapies
Patient Outcomes: understand the full impact of the disease on daily living and patient treatment priorities, understand collateral effects of disease; related medical conditions, side effects, and financial impact
Pediatric Treatment: identify strategies, safety concerns, and long-term outcomes
“This is the most exciting time ever,” in the history of MG, Law said. There has long been an understanding of what happens in patients with MG, the functioning of antibodies against receptor sites of the neuromuscular junction. But there had not been a new FDA-approved drug for MG in over six decades until the first disease-modifying therapy for MG was approved in 2017. And, just in late May 2020, a Belgian drugmaker announced promising results from a stage three clinical trial, which will enable it to seek U.S. approval. MGFA has also become part of a rare disease clinical network funded by NIH, with several other clinical trials for MG treatments underway.
“As a small organization, we rely a lot on organizations like Research!America,” to support our advocacy goals, said Law. MGFA has a new advocacy committee to stay focused on policy and advocacy priorities such as access to health care, coverage of preexisting conditions, and costs of treatment. “Even as we’re seeing more treatment for rare diseases, we’re also seeing astonishing price tags,” Law added.
As is the case with so many rare disease organizations and patient advocacy groups, the current situation around the COVID-19 pandemic is also a pressing issue. “Our population is generally at higher risk because of the immunosuppressive effects of many current MG treatments,” Law said. Maintaining community connections has been paramount. Like many groups, MGFA made a last-minute pivot from an in-person conference in April to a virtual conference — but with surprisingly positive results. “We expected 300 participants, and ended up with nearly 1,500 from 42 different countries,” Law reported. The virtual conference “gave us a window on the world.” Overall through various virtual connections, “we’ve had three to four times the participation over the last month. People need these connections, even as they adapt to new technology,” Law added.
“It’s a testimony to the human spirit,” said Samantha Masterson, MGFA’s recently appointed Chief Executive Officer. “This community is really special, very tight-knit — patients, medical professionals, donors, volunteers, sponsors, and more — it’s very powerful.”
Another challenge on the horizon that COVID-19 may bring is the availability of plasma — which is needed to manufacture immunoglobulin — a treatment that is important for many MG patients, COVID-19 has caused a slowdown of donations of both blood and plasma due to social distancing measures.
Importantly, June is MG Awareness Month, and outreach activities will include calls to action for individuals to donate plasma.
To learn more about MGFA, visit www.myasthenia.org.