What Would You Risk to Fight Severe Myasthenia Gravis?

What Would You Risk to Fight Severe Myasthenia Gravis?

As anyone with Myasthenia Gravis will tell you, this disease is no picnic.

In most cases it’s treatable with immunosuppressive therapies and people can live full—and fulfilling—lives with MG. But where there’s a “most,” there’s also a “some.” And for some MG patients, those therapies just don’t work; the disease continually breaks through, causing severe or even life-threatening symptoms. Now, there may be a possible new weapon in these patients’ MG-fighting arsenal… but it’s a pretty big gun, and the kickback is a real bitch.

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This particular big gun is called an autologous hematopoietic stem cell transplant, and a group of doctors in Canada have spent the last several years following eight MG patients who underwent the procedure.

The transplant involves destroying the faulty immune system and replacing it with the patient’s own stem cells, harvested before they’ve developed into the faulty autoimmune cells. If that sounds complicated, it is—I’m still trying to figure out how they know the cells aren’t faulty. And if you were thinking it’s risky, you’d be right. Between the heavy-duty doses of chemo needed to wipe out the malfunctioning immune system and all the associated side effects and risks (up to and including death), this really isn’t an option for people who can control their MG with standard therapies.

For those who had the procedure though, the rewards were just as amazing as the risks were grave. The eight patients all went into a full, sustained remission and have been symptom free for several years. The researchers hesitate to use the term cure—that’s a pretty loaded word—but they did describe it as “encouraging.” Which in research-speak qualifies as “OMFG YOU GUYS, CAN YOU EVEN BELIEVE THIS?!”

The findings were recently published in JAMA Neurology, and as you’d expect all the patients experienced some type of side effects. But considering they were facing the very real risk of death from severe and uncontrolled MG, those side effects have to pale in comparison. And today, these patients have made medical history and offer an avenue of hope for those living with the worst this disease has to offer.

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