By Nicholas Silvestri, MD, as told to Carolyn Crist
I’ve been interested in neuroscience since I was in college, but it wasn’t until I was a neurology resident that autoimmune neuromuscular disorders, like myasthenia gravis, made an impression on me.
As a young neurologist, I’d see patients come into the hospital with certain signs and symptoms of MG and be able to make a diagnosis. I realized that starting treatment could turn people around quickly – for a disease that was potentially lethal not even 100 years ago.
Matching Patients With the Best Treatments
My particular focus is on the burden of disease and the burden of treatment. My colleagues and I think about how the more newly approved targeted medications should be incorporated into treatment. We try to understand the best treatment options for patients, not only from the standpoint of treating the disease but treating the whole patient. We want to treat the disease without burdensome side effects or burdensome costs.
In terms of treatments, we’ve been blessed in the past few years. Since 2017, we’ve had five medications approved to treat MG. Now we’re trying to understand the best way to use these medications – who to use them in, when to use them, and how to use them.
Previously, traditional treatments included corticosteroids, acetylcholinesterase inhibitors, and steroid-sparing immunosuppressive therapies for long-term management. Exacerbations and crises were managed with IVimmunoglobulin and plasma exchange.
The newer medications are more targeted to the specific processes that occur in the development and progression of myasthenia gravis. They block certain immune system proteins – complement and neonatal Fc receptor (FcRn) – that are known to promote disease progression. By zeroing in on these targets, they reduce the risk of both short-term and long-term side effects, which are major concerns when using more traditional treatment options for MG.
Importantly, though, it may not always be realistic to use medications in real life in the same way they are tested in clinical trials. So now that we have a wealth of new options that have been approved in a short period of time, we now need to understand how and when to use them in our patients. We’re hopeful about incorporating them into the current treatment paradigm.
Striving for Lower Costs and Better Access
While it’s great to have new therapies, it’s also important to make sure we’re equitable about the ways therapies are given and that any patient with the disease can get these treatments, regardless of their economic background. Patients with myasthenia gravis come from all walks of life and all different backgrounds. It truly is a disease that can affect anyone and everyone.
When selecting a newer medication for a patient, we may use a simple equation: Value = Clinical Improvement/(Cost + Side Effects + Treatment Burden). This can guide our decision-making about what’s best for a patient.
In terms of future research, we’re working to understand the total cost of the disease and the economic burden to the patient. That includes not only the different aspects of the equation above – such as medication costs and side effects – but also the lost opportunity cost, for example from being forced to quit work, and other issues surrounding the disease. These are quite important aspects to consider.
I think this research will shed light on the overall cost of the disease, raising awareness for payers and making access to newer medications easier.
Looking Ahead With Hope
I’m incredibly excited. This is a great time to be practicing in the field. We’ve had five medications approved in the past few years, and there are so many clinical trials in various stages of development. These new drugs in the pipeline target proteins and antibodies that disrupt nerve-muscle communication.
New treatments that provide better symptom control are right around the corner. I think we’re going to see these better options become available in the next couple of years.
These pipeline therapies may be just as effective as current ones but more convenient. For example, they might require fewer IV infusions, options for self-injection of medications at home, and even oral medications. Other pipeline medications look to be highly effective and may offer hope to people who have otherwise not achieved disease control with available therapies.
I think the competition generated by all these clinical trials is great because it’ll ultimately allow for better medications and treatment options for patients.
As I tell my patients, every person with myasthenia gravis is different. Every person has a different set of circumstances, from both a medical and nonmedical standpoint. The great thing about what we have available to us now and what’s coming in the future is that if treatments A and B aren’t necessarily effective in your case, then we have treatments C, D, E, and F to try.
Don’t be discouraged if the first treatment that we decide to work with is not effective. We can always come up with the next plan. My goal is for patients to be minimally symptomatic, if at all, with regard to MG – and not at the expense of side effects.
More broadly, I like to tell people that many of the disorders that we see as neurologists or neuromuscular specialists are rare. It can take time to arrive at an accurate diagnosis and treatment. However, a lot of what we have available in our treatment armamentarium is better now than even 10 years ago, and it’s only going to get better.
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SOURCES:
Nicholas Silvestri, MD, professor of neurology and associate dean for student and academic affairs, Jacobs School of Medicine and Biomedical Sciences, University at Buffalo.
Muscle & Nerve: “How should newer therapeutic agents be incorporated into the treatment of patients with myasthenia gravis?”
