The Guthy-Jackson Charitable Foundation 2009 NMO Roundtable Conference

Katja Van Herle:

I’d like to start by welcoming you all to the home of NMO.

Victoria Jackson:

A lot of what happens in research is people work in all their independent little silos and they never really talk. But I know that if you’re actually going to really cure something and make a difference, you have to work through collaboration. And a symposium seemed a perfect vehicle to do that. So that’s what really bringing all of you together is about, turning up the volume on NMO, really creating some critical mass.

Victoria Jackson:

Sometimes when people have a foundation, they have to spend so much of their time fundraising, and I’ve been so fortunate that this is not about fundraising. This is about science, this is about collaboration. And this is about getting the best and smartest people together in a room to find the cure for NMO. And every time we have this meeting, it’s turning on more lights. It’s finding out more information, more science, and it’s amazing and I feel privileged to be able to bring you all together.

Victoria Jackson:

They say in the world of science, people don’t want to share, but I disagree. You need to share your great information with this one over here so that we can do this. We have to do it together collectively.

Katja Van Herle:

We have put together six leading centers, about eight to nine different researchers. As well as their academic counterparts overseas to help figure out not only what do we do for the patients here and now, but what’s the real answer to this? The empowerment of understanding clinical care and I’m a clinician is based in the understanding of how do molecules actually work? And what goes wrong in this wonderful miracle of life?

Michael R. Yeaman:

We can’t know exactly where we’re going in terms of understanding this disease. It’s so new that we’re all learning at the same time. Bringing people who have experience in it and insight from other autoimmune conditions but have a focus on NMO is the best chance to discover and develop new medicines and potentially a cure for NMO as efficiently as possible.

Victoria Jackson:

On one of the days in the symposium we had little breakout sessions where everybody gathered at a round table and we had assigned questions and then it was really just free-form exchange of ideas. And it’s such a great way to just bring out that one idea or thought that somebody hadn’t thought of before.

Claudia Lucchinetti:

Question four, targeting upstream or downstream. I think that’s a critical question because just my bias is that there’s some very critical immunoregulatory events at the get go. If you can define those and dissect that, then you could maybe get in before you’ve gone down that irreversible path. I just think it’s an amazing time to bring these minds together, this work together, these opportunities for collaboration. And I tell NMO patients all the time, we’re closer to curing NMO than we are to MS. we know what the target antigen is. We have ideas about the disease in a unique way, we can define it.

Benjamin Greenberg:

NMO’s on the fast track in terms of our ability to really intervene at the earliest steps of the disease. If we were going to do one clinical study now, I’d say it’s the observational study. Without an inherent need to compare [inaudible], more of an inherent need to define who’s failing each of them and why and move them on to whatever the next drug in the pipeline is going to be.

Sean Pittock:

As we do these studies, we will begin to learn how common is NMO and also begin to get a sense of how often is NMO misdiagnosed as MS? Because remember about 70% of patients that present with NMO were at some point diagnosed with multiple sclerosis.

Candace Coffee:

This is probably the first time that any really legitimate focus has been put on NMO. People who are diagnosed around the time I was, there was so little known about it. And now they’re getting diagnosed sooner, they’re not going to end up with permanent disabilities. It gives me hope in that sense because I think the education and the treatments and just the management of the condition itself, I’ve already seen so much improvement.

Dean Wingerchuk:

But if we had an organization where you could develop standardized protocols and funnel people into different… You could test hypotheses. I guess that’s what’s missing in eculizumab.

Victoria Jackson:

Is it one specific drug you’re meaning getting down to whether it’s the Mayo protocol, which is azathioprine and steroids?

Michael R. Yeaman:

For decades, steroids were used, which are very broad, almost hammers that suppress the immune system. If you compare the use of a steroid to a hammer, now what we’re talking about are monoclonal antibodies that are as finally sharped forceps as you can imagine.

Brian Weinshenker:

We can suppress about 80% of attacks with CellCept, with rituximab, with a variety of different treatments. The key thing is making the diagnosis accurately and early. That’s the single most important thing as far as treatment.

Claudia Lucchinetti:

I see reversibility in the disease. That means something’s happening very early that can reverse. It doesn’t need to go all the way to compliment damaged tissue death.

Victoria Jackson:

Failure’s not an option. So it’s to just keep moving along and really bringing together the best minds and the research to find the cure.


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