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Research News

argenx announces positive topline results from Phase 3 ADAPT trial for gMG

ADAPT met its primary endpoint defined as percentage of responders on the Myasthenia Gravis Activities of Daily Living (MG-ADL) score among acetylcholine receptor (AChR)-antibody positive generalized myasthenia gravis (gMG) patients. Responders are defined as having at least a two-point improvement on the MG-ADL score for at least four consecutive weeks.

 

Full press release available here.

 

Research survey for rare disease patients and their families about impacts of COVID-19

How is the novel coronavirus pandemic impacting people with rare diseases and their families? Complete the 20-minute research survey from home or learn more at this link.

 

The Rare Diseases Clinical Research Network (RDCRN) is conducting this study. The network is funded by the National Institutes of Health. It includes 23 research teams working to advance diagnosis and treatment of groups of rare diseases. The Myasthenia Gravis Foundation of America and Conquer MG partners with RDCRN through its work with the Myasthenia Gravis Rare Disease Network (MGNet).

 

Guidance for the management of Myasthenia Gravis (MG) and Lambert-Eaton Myasthenic Syndrome (LEMS) during the COVID-19 pandemic

March 23, 2020 
International MG/COVID Working Group* 

International MG/COVID-19 Working Group1SaijuJacoba2SrikanthMuppidib2AmandaGuidoncJeffreyGuptilldMichaelHehireJames F.HowardJrfIsabelIllagRenatoMantegazzahHiroyukiMuraiiKimiakiUtsugisawajJohnVissingkHeinzWiendllRichard J.Nowakm2
Corona Virus Disease 2019 (COVID-19) is a new illness caused by a novel coronavirus, severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). Symptoms are variable but typically include fever, cough, respiratory symptoms, diarrhea, reduction of smell and taste sensation. Severity ranges from mild to severe and the virus may lead to pneumonia, acute respiratory distress syndrome and death, in some patients. Nearly every country in the world has been affected by this virus and is currently defined as a pandemic, by the World Health Organization. There are no known proven therapies for treating this virus and no vaccine to prevent the infection at this time. 

 

The full guidance is available here

 

MGFA Releases Call for Abstracts for 2020 Scientific Session

The 2020 Scientific Session will be held in conjunction with the 2020 AANEM Annual Meeting on October 7, at the JW Marriott Grande Lakes in Orlando, FL.  Our call for abstracts for the 2020 Scientific Session was released on March 16.

To view the call for abstracts, see here

 

Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia GravisResults of a Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Trial

Many patients with generalized myasthenia gravis (gMG) have substantial clinical disability, persistent disease burden, and adverse effects attributable to chronic immunosuppression. Therefore, there is a significant need for targeted, well-tolerated therapies with the potential to improve disease control and enhance quality of life.

 

The findings of the above-mentioned study, published by JAMA Neurology, support a potential therapeutic role for zilucoplan in generalized myasthenia gravis and further evaluation in a phase 3 study.

 

MGFA announces 2020 Research Funding Opportunities- Deadline Extended to 5/23/2020!

We are pleased to announce the release of our Request for Applications for our High Impact Clinical Research and Scientific Pilot Projects on Myasthenia Gravis and Related Neuromuscular Junction Disorders.

 

We are also very pleased to announce our first targeted research opportunity to benefit the Seronegative MG community

 

To learn about our research agenda and complete funding opportunities, see here

 

MGFA announces 2019 Pilot Grant Awards for 2020

We are pleased to announce our pilot grant awards for the upcoming year. MGFA pilot grants recognize promising research in clinical and basic sciences for myasthenia gravis. Research grants are awarded based on our Research Agenda, which is focused around five priorities: biomarkers, diseases mechanisms, targeted therapies, patient outcomes and pediatric treatment. To learn more about our research priorities and funding, see here.
 

  • 2019 High Impact Pilot Project Grant Award: Amanda C. Guidon, M.D., Massachusetts General Hospital, Neuromuscular Diagnostic Center, “Evaluation of automated techniques for decoding speech and movement abnormalities in myasthenia”  $55,000
  • 2019 High Impact Pilot Project Grant Award: Jeffrey T. Guptill, M.D., Duke University, “Metabolic Pathways of Pathogenic Th17 Cells in Myasthenia Gravis”  $55,000
  • 2019 High Impact Pilot Project Grant Award: Ricardo A. Maselli, M.D., University of California Davis, “CSF Delivery of AAV9-mediated gene therapy of congenital myasthenic syndrome due to CHAT mutations”  $55,000

We wish to extend our congratulations to these recent grantees. With this promising work in the pipeline, coupled with the efforts of MGNet, the MG research landscape has never looked brighter!   

 

To learn more about our research grants, see here.

 

University of California, San Francisco, Launches First Consortium on Pediatric MG in Partnership with MGFA and MDA

For families with children that have myasthenia gravis, we have some exciting news to share. Thanks to a generous gift from an anonymous donor to the University of California, San Francisco, the first clinical Pediatric Myasthenia Gravis Consortium is now a reality.  MGFA was honored to provide a grant to underwrite the costs for the September inaugural meeting of this exciting new project, and to participate on the advisory committee for the Consortium.

 

Response to Treatment in Pediatric Ocular MG 

In children, myasthenia gravis (MG) almost always appears in one of two forms: ocular myasthenia gravis (OMG), where weakness is evident only in the eyelids and surrounding tissue, whereas in generalized myasthenia gravis (GMG), various tissues throughout the body are affected. This study, published in Muscle & Nerve,  examines how these two disease types tend to differ from a clinical perspective, as well as patient response to treatment. 

 

National Institutes of Health (NIH) Awarded a Research Team at the George Washington University (GW) $7.8 million to Establish a Rare Disease Network for Myasthenia Gravis

The National Institutes of Health (NIH) has awarded a research team at the George Washington University (GW) $7.8 million to establish a rare disease network for myasthenia gravis. The network, which will be part of 25 established NIH Rare Diseases Clinical Research Networks, will include basic and clinical investigators, patient advocacy groups and biotechnology and pharmaceutical companies working together to enhance therapeutic development for this rare disease.  The team is led by former and current MGFA Medical and Scientific Advisory (MSAB) Chairs, Henry Kaminski, MD and Linda Kusner, PhD, and the steering committee members are all leaders of the MGFA MSAB as well. 

 

The grant will fund research into the underlying pathophysiology of the disease, provide fellowships in MG for young investigators, and fund pilot grants.  This funding will also ensure that the serum bank created by the MGFA’s transformative grant will continue.  

MGFA is proud to represent the MG Community as a member of MG Net, and has committed $250,000 of funding ($50,000 for each year) to support the project.  This commitment from MGFA, as well as that of Illinois-based Conquer MG, was instrumental in demonstrating the support of the MG Community for the project—an essential component of the criteria for funding established by the NIH. Press release available here.

 

 

Diabetes drug Metformin May Have Novel Therapeutic Potential to Treat MG

The study, "Metformin attenuates autoimmune disease of the neuromotor system in animal models of myasthenia gravis", published by International Immunopharmacology, used a mouse model to investigate the therapeutic potential of metformin on autoimmune myasthenia gravis. Researchers showed that oral administration of metformin diminished the onset, reduced clinical severity and led to a reduction in mortality in experimental autoimmune myasthenia gravis rats. 

 

Zilucoplan Receives Orphan Drug Status for Treating MG 

Ra Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to zilucoplan for the treatment of myasthenia gravis. Ra Pharma is developing zilucoplan, a self-administered macrocyclic peptide inhibitor of complement component 5 (C5), for the treatment of generalized myasthenia gravis (gMG) and other rare, tissue-based complement-mediated diseases.

 

MG Metabolic Profile May Be Used to Predict Course and Treatment for MG

"Beyond the antibodies: serum metabolimic profiling of myasthenia gravis", published by Metabolomics, uses a profiling approach as a potential strategy for identifying biomarkers unique to myasthenia gravis. Comparisons between patients with MG vs. HC (healthy controls), and RA (rheumatoid arthritis) vs. HC were made using univariate and multivariate statistics.

 

Patterns of Muscle Weakness Vary Widely for MG Patients

"Heterogeneity and shifts in distribution of muscle weakness in myasthenia gravis", published by Neuromuscular Disorders, analyzes the distribution of muscle weakness in 225 AChR MG patients over time. The study identified phenotypes: ocular, bulbar, neck/ limbs/ respiratory, or a combination. MG patients frequently shift between phenotypes. These variations found in AChR MG suggest that other factors aside from the ACHR antibody mediated immune response are important in determining the disease expression of MG. 

 

Poorer Nerve Cell Response in Diagnostic Tests May Prompt More Aggressive Treatment Recommendations for MG

Patients who initially showed greater fluctuations in the electric signal at the neuromuscular junction and lower electrical signals (reduced by 20% or more) were more frequently positive for autoantibodies and had generalized disease. These patients were also classified as having more severe disease, having higher quantitative (above 10.5) myasthenia gravis score (QMGS) at baseline, compared to patients with better electrophysiological test results. See here for the full article from the Canadian Journal of Neurological Sciences, "Baseline Decrement in Patients with Mild Myasthenia Gravis Predicts Immunomodulation Treatment".

 

MGFA Statement on Marijuana and MG

Several states have legalized the use of marijuana for medical and/or recreational use.  Can marijuana or chemicals extracted or based upon components of marijuana help people with MG?  Let’s consider what is currently known about medical benefits of marijuana. In researching this topic, members of the MGFA Medical and Scientific Advisory Board (MSAB), reviewed the scientific literature and the information available from reputable sources such as the National Institutes of Health, the American Academy of Neurology, the American Neurological Association and the American Medical Association. For more detail and our full statement, see here

 

Adverse Events in Placebo-Treated MG Patients do not Result in Higher Clinical Trial Dropout Rate

The study, "Nocebo effect in myasthenia gravis: systematic review and meta-analysis of placebo-controlled clinical trials", published in the journal Acta Neurologia Belgica, is a meta-analysis of adverse events experienced by patients with myasthenia gravis following placebo treatment. The study demonstrates a low nocebo dropout rate in MG comapted to central nervous system disorders. 

 

MG Patients with Coexisting Autoimmune Diseases do not have Higher Disease Burden 

While many MG patients were affected by fatigue, sleepiness, depression and anxiety, the study, "Impact of autoimmune comorbidity on fatigue, slepiness and mood in myasthenia gravis",  does not suggest that coexisting autoimmune diseases substantially contribute to the magnitude of these cumbersome comorbid symptoms. However, the higher frequency of steroid treatment may have counterbalanced the effects of the autoimmune comorbidity. 

 

Zilucoplan, a Subcutaneously Self-Administered Peptide Inhibitor of Complement Component 5 (C5), for the Treatment of Generalized Myasthenia Gravis: Results of Phase 2 Randomized, Double-Blind, Placebo-Controlled Trial and Open-Label Long-Term Extension 

Results of the Phase 2 study were presented at the 2019 American Academy of Neurology Annual Meeting in May: 
Presentation

Poster 

 

Retrospective Study to Determine the Efficacy of Rituximab in Treating Myasthenia Gravis

A group of scientists in Austria conducted a study to determine the efficacy of rituximab in treating myasthenia gravis. The study, titled, “High efficacy of rituximab for myasthenia gravis: a comprehensive nationwide study in Austria” looked at 56 patients and studied their response to rituximab, finding that 26.4% of the patients studied were in remission after three months of treatment. In this retrospective study on RTX for MG, the largest to date, RTX appeared safe, efficacious and fast acting. Benefit from RTX was greatest in MuSK ab + MG.

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