MDA Engage: Specialist urges individualized treatments for MG
New and emerging therapies offer more options for people living with disease
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- Syed Ahmed, a neuromuscular specialist, spoke at MDA Engage: Dallas.
- In MG, he said, it’s important to individualize treatment to each person’s needs.
- Clinicians should also listen to their patients to better understand those needs.
With a wide range of treatments available for myasthenia gravis (MG), it’s important to individualize treatment to each person’s needs, depending on their symptoms and what their life was like before they developed the disease, according to a speaker at a recent MDA Engage event.
“We’ve made a huge stride in the treatment of myasthenia gravis,” with improvements in keeping patients out of the hospital and improving their quality of life, said Syed Ahmed, a neuromuscular specialist and assistant professor of Neurology at UT Southwestern Medical Center in Dallas, who recently gave a talk on MG at the MDA Engage: Dallas symposium.
“[However,] I still think that we have a ways to go. There’s aspects of MG that were not treating, we’re not paying attention to,” he added, and clinicians should listen to their patients to understand those needs better.
Ahmed’s talk at the symposium, simply titled “Myasthenia Gravis,” included the treatments that are currently available for people with the disease and those on the horizon. The Oct. 23-24 event brought together people living with neuromuscular diseases and their caregivers, along with clinicians and advocates, for education and community support.
Treatments can have different targets in MG
Ahmed’s presentation began with an overview of what’s actually happening in the body of someone with the disease. MG is a chronic autoimmune neuromuscular condition that occurs when the immune system produces self-reactive antibodies that disrupt communication between nerves and muscles, resulting in symptoms of muscle weakness and fatigue.
Most commonly, these antibodies target receptors for acetylcholine, a chemical messenger released by nerve cells that triggers muscle contraction. In some people with MG, however, the antibodies target other proteins involved in nerve-muscle communication, including muscle-specific kinase (MuSK) and low-density lipoprotein receptor-related protein 4. Some people have no detectable MG-associated antibodies with currently available tests, a form of the disease known as seronegative MG.
Because multiple mechanisms can disrupt nerve-muscle communication and cause muscle weakness in MG, treatments can have different targets.
“Obviously, you ask then …, what is the most optimal pathway?” Ahmed said. But clinicians don’t know the answer to that. Instead, they have to try to individualize treatment to each person’s symptoms and MG type.
In clinical trials, researchers often aim for a treatment goal called “minimal manifestation status,” which means patients have no symptoms or at least no functional limitations, though they may still have some degree of muscle weakness.
“Ultimately, the goal is to be able to say the patient who is a runner wants to be able to go back to … running. The patient who has had to take early retirement maybe wants to go back to work,” Ahmed said. “Making them functional is the goal.”
A variety of treatments
Doctors also track patients’ symptoms with the MG Activities of Daily Living, a scale that assesses how well they can perform various activities, such as talking, chewing, swallowing, keeping their eyelids open, or rising from a chair. It’s not a perfect measure, Ahmed said, but it’s a place to start, and the goal of treatment is to achieve enough consistent control of the disease that people can return to their daily activities and social life with minimal medication side effects.
Acetylcholinesterase inhibitors like pyridostigmine bromide (sold as Mestinon and generics) are typically the first-line treatment for MG. These therapies work by inhibiting the enzyme that breaks down acetylcholine, thereby helping to increase acetylcholine levels and boost its effects. Pyridostigmine can ease symptoms for many patients, but may be less helpful for those with anti-MuSK antibodies and for some people with ocular MG, in whom symptoms of weakness and fatigue are restricted to the muscles controlling the eyes and eyelids. Pyridostigmine only takes 15 to 30 minutes to begin working and its effects can last for about four hours.
Since pyridostigmine doesn’t address the root cause of MG, the mainstay of therapy, Ahmed said, is immunosuppression. Patients who don’t respond adequately to pyridostigmine typically next try steroids, usually prednisone. It’s very effective, starting to work within a few days or weeks. However, prednisone comes with a lot of side effects, including difficulty sleeping and increases in blood pressure and blood sugar.
If prednisone is ineffective or its side effects become intolerable, other treatments may include steroid-sparing immunosuppressants, such as azathioprine (sold as Imuran and others), mycophenolate mofetil (sold as Cellcept and others), cyclosporine (sold as Neoral and others), or tacrolimus (sold as Prograf and others).
Today, however, more patients are beginning to take newer immunosuppressant or targeted therapies. These include are Soliris (eculizumab), Ultomiris (ravulizumab-cwvz), Zilbrysq (zilucoplan), Vyvgart (efgartigimod alfa-fcab), Rystiggo (rozanolixizumab-noli), Imaavy (nipocalimab-aahu), and Uplizna (inebilizumab-cdon). Before starting these treatments, patients may need screening for certain infections and review of their vaccination status, though the specific precautions vary by medication.
Many other therapies are undergoing testing in clinical trials, Ahmed said.
Hearing from patients
Ahmed also wanted to hear from those living with MG about where gaps in care are. One attendee wanted to know how to tell when a treatment is controlling the disease. Another, Lillye Buckans, who is living with MG in Saginaw, Texas, wanted to know how doctors individualize treatment for patients.
In both cases, Ahmed said he asks how close patients are to their pre-MG baseline.
“Control is … being able to go back to doing the things that you enjoy,” he said. A treatment may be controlling the disease “as long as you’re being able to be functional [and] keep moving around,” even if a person is not in full remission. What that looks like might be different for different people too, so it’s important to communicate with your doctor about your symptoms and what daily activities you’re still unable to do fully.
Control is … being able to go back to doing the things that you enjoy.
It’s also important to consider all the possible causes of a symptom, Ahmed said. Fatigue, for example, might be caused by something other than MG, so clinicians need to explore what else might be causing fatigue before adjusting medications.
Buckans told Bionews, the parent company of this site, that she found Ahmed’s discussion of different treatments the most helpful aspect of his talk. She first developed symptoms of MG in June 2022 and was diagnosed in early 2023.
“When I was … first diagnosed, all I was told was this is a snowflake disease” and that she should go online and educate herself, Buckans said. “I was not educated on MG or … the treatments or what was bad or good, per se, so it’s just amazing to see that there’s [really] so much.”
Buckans was also thrilled to meet someone else living with MG at her first MDA Engage event. Until attending Ahmed’s talk, Buckans hadn’t known anyone else with MG.
Note: The Myasthenia Gravis News team is providing coverage of the MDA Engage Symposium. Go here to see the latest stories from the conference.
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