Benjamin M. Greenberg:
It’s amazing when dealing with rare disorders. The first response is always, “What is that?” Because nobody has heard of these conditions and patients feel like they’re isolated, they’re alone, and there’s an immense sense of despair being diagnosed with something rare, because by definition, rare means incurable or hopeless.
Benjamin M. Greenberg:
One of the things I’d like to do with you is just really sit down with a calendar, because what I want to do is differentiate in this progression. Anytime it was a worsening of a symptom that had already been there versus something brand new, because one of the things to do for us … If we turn back the clock 5 to 10 years ago, the horror of these conditions was knowing what you were going to go through as a patient. Having a physician tell you the grueling statistics of how many people would be paralyzed, how many people would be blind, and how many people would die of neuromyelitis optica. We’ve moved that goalpost tremendously and as we’ve been sitting around the table here in LA this year, the numbers we’re seeing are spectacular in terms of our ability with medicines to keep people healthy, more than we’ve ever seen before.
Michael Levy:
One thing I tell the patients is, “Here’s my phone number, here’s my email address. The moment you think something is going wrong, please, please call me. We can treat you right away. You don’t have to go blind, you don’t have to go paralyzed.” And I just make myself much more available to my NMO patients than to anybody else, because I feel like I can’t wait with them. I can’t call them back six hours later. I need to bring them in right away.
Claudia Luccinetti:
I see patients regularly. Right now I’m on hospital service at Mayo, and I just admitted a patient with NMO about three days before I come in this meeting, who presented with nausea, vomiting hiccups, a big lesion in his spinal cord. He’s doing great. He’s stable. We treated him with steroids, with plasma exchange, and he never heard about this disease. And not only was I able to tell him about the disease, what we did, where we were at, at Mayo, knowing a lot about it, but also he was very excited about the fact that I was coming to this meeting. And I directly him right away to the foundation. And so he could immediately personalize and realize there was a lot going on in the fields. But in the clinic, what I see as plenty of patients with mild disease that I can keep under decent control and remind them that it’s not as bad as it necessarily looks in the literature. I think we’re really making progress.
Dean Wingerchk:
There’s no doubt that there is still many people who are devastated by the disease, but there are others who have milder forms of it, or who respond very well to treatment. And if we can prevent attacks, and do that early, we think we manage to preserve function for these individuals in the longterm.
Sean Pittock:
In NMO, the way to prevent disability is to stop attacks. And how do we stop attacks? Well, we try and immunosuppress the patients, and those drugs do work, but they’re not wonder drugs. They don’t stop the attacks 100%.
Brian Weinshenker:
When we did that first study at Mayo Clinic, and looked at our experience in some 80 patients, the outcome was pretty dire, 30% of patients had died within five years. All of them have respiratory failure, all of them in the context of a severe attack of spinal cord inflammation, or myelitis, and very high rate of blindness, and so forth. But again, I have to emphasize the key factor is that most of those patients were not diagnosed, or at least not till very late as having neuromyelitis optica. Very few of them had effective treatment until very late in their course.
Brian Weinshenker:
If you look at the effects comparing before treatment and after treatment, we see about an 80% reduction with several of our immune suppressive treatments in the rate of attacks. We see on a daily basis from treating individual patients, now how we can take those patients who have lost all vision in an eye, or have severe myelitis, and have failed standard treatment. If we give them plasma exchange, we see them recover, and recover to a major extent. So I have little doubt that we’ve made substantial gains with our current treatment, but we still need to improve things a great deal more.
