By Richard J. Nowak, MD, as told to Sonya Collins
Nowak is director of the Myasthenia Gravis Clinic at the Yale School of Medicine and chief medical adviser at the Myasthenia Gravis Foundation of America.
The last decade has been transformative for the treatment of myasthenia gravis (MG).
A dozen-plus years ago, there wasn't much research into new treatments for MG. But in the last five to 10 years, there's been a tremendous focus on targeted therapies for the treatment of MG. Over the last five years or so, that's led to FDA approval of new medications we didn't have before.
Fifteen years ago, people debated whether one would even be able to get enough people with MG enrolled to run a clinical trial to test a new drug, due to the fact it's a rare disease. But the move to conducting studies globally, rather than limiting participation to the U.S., has really allowed us to advance the field by definitively evaluating new therapies in large international trials.
When I was a medical student, and then a neurology resident, we didn't have many options for patients, especially for those with moderate to severe disease. Now we have an overwhelming number of options. It's truly been transformative.
There are two main classes of targeted medications that are FDA-approved for MG – C5 complement inhibitors and FcRn antagonists. Overall, these have led to improved patient outcomes and a shift in the treatment landscape in a number of ways.
First, according to clinical trial data and what we've seen firsthand, people who respond to these drugs start to see clinical benefits within a few weeks. Some of the older medications take longer, some of them as many as several months after people start taking them.
These new drugs also mean we have many more options for the people we treat. We can tailor treatment plans to each person based on any other health issues they may have, where they are in life, whether they're younger or older, and other factors.
Some treatment options have potential long-term risks or may not be suitable for certain populations. For example, corticosteroids can come with a number of long-term side effects. And nonsteroidal immunosuppressive therapies may not be ideal for certain populations, such as women who may have children and people who have cancer or who are high-risk for cancer.
Approval of new medications has also brought more attention and awareness to MG, which has led to more emphasis on dedicated MG care.
For the longest time, MG fell under the muscular dystrophy umbrella. A lot of these people were seen in muscular dystrophy clinics in the US. But this is clearly not a muscular dystrophy or genetic-based disorder.
While we certainly approach the evaluation and diagnosis from a neurology and neuromuscular perspective, MG is an autoimmune condition where there's a need to be intensely focused on neuromuscular immunology from a management and treatment perspective.
Over the last five years, and likely in part due to advances in targeted therapies, we've started seeing more dedicated MG clinics and delivery of more expert, up-to-date care. And, to that end, the Myasthenia Gravis Foundation of America has established Partners in MG Care, which helps doctors and other health care professionals stay up to date on MG care and research. And it provides critical resources that doctors can use for the people they treat.
But the new targeted therapies haven't solved every problem in MG, in that we still have patients with inadequate response. As such, there's still room for improvement and unmet need for people living with MG.
A challenge that comes with having many treatment choices is that we don't have a way to predict which people will respond to which treatments. That might've been OK when we had fewer options, but now that there are a half-dozen or so, it's a significant knowledge gap and something researchers are discussing how to best address. This will undoubtedly need further study and, importantly, research support to do so.
We also don't have a way to predict how MG changes over time, which could also help inform treatment decisions. Why are some patients milder, others quite severe, and others somewhere in the middle? The course of the disease and what affects the course over time are areas in need of more research.
Of course, the new targeted therapies, while they've improved care and outcomes for many people living with MG, they aren't a cure.
The Holy Grail of treatment for MG, as for any autoimmune condition, is immune tolerance. That's the state where your immune system doesn't attack itself. There are efforts underway to learn how we can create a state of tolerance in the immune system so it no longer mistakenly attacks. But we're not there just yet. That's one of the many next steps toward improved patient care and outcomes as well as a potential long-lasting treatment and/or cure.
SOURCE:
Richard J. Nowak, MD.
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