Current Series: Multiple Sclerosis: A Disease of an Immune System Gone Awry

Fred D. Lublin, MD: The optimal treatment of multiple sclerosis [MS] is an inexact science. We sit and talk with someone, and we take a look at what their history has been in terms of the illness. We look at their examination, how the diseases affect their function, and the MRI [magnetic resonance imaging] scans to see how much damage there is. We try to make an assessment of what the best approach would be for them with their guidance and an understanding of what else is going on in their life, their comorbidities, if they prefer a pill to an injection or to an infusion, and how long they are going to be available for follow-up because some of our therapies require long-term follow-up.

We’ve been very productive in coming up with new therapies for multiple sclerosis, in part because we have a very good idea of how we can study patients over the short term to predict longer-term benefits. At first we had injectable agents; then pills, or oral agents; and now infusions, and we’re going back to studying some injectables again. All of our therapies right now are directed toward having an effect on the immune system, and they’ve worked very well.

Moving forward, we’re looking for better therapies for progressive disease. We have 1 approved agent for primary-progressive disease, which is really exciting because that came 24 years after we started treating relapsed-remitting disease. Treating progressive disease is more complicated, and it’s harder. We have some other agents in the pipeline with effects on progressive disease. They’re modest so far, and that suggests we need a different strategy than what we’re using now to get more robust effects. But there are earlier-phase studies on other agents that affect the innate immune system. We’re trying to develop newer protective agents. That’s our short-term goal for the future, which is to get a better handle on progressive disease.

The long-term goal for which we have studies under way already is repairing the damaged nervous system. This is probably the most exciting thing that we’re doing. When I was in medical school, we were told you could never repair damage done to the brain or spinal cord. And now we have a pretty good idea that we can. We have to work out techniques for measuring it but also the right molecules for enhancing repair, and that is the most exciting area of our field.

Early treatment is a bedrock feature of MS, and it’s one that we get asked about a lot. You could understand why. Someone has a single episode, this clinically isolated syndrome that I discussed earlier, which would be something like an optic neuritis, with lost vision in one eye or the other transiently over days to weeks. It usually improves, although not necessarily 100%. Or it could be brain stem cerebella syndrome or a partial myelitis affecting the spinal cord.

For someone who presents with those symptoms and has MRI changes that look like MS, we have really, really good data that those people should be treated, that you shouldn’t wait. What’s the risk of waiting? The risk is that they have another attack. With any given attack, there’s a 50% chance that there will be something left behind. It may be mild, but it may be severe, and we have no way to predict that. That’s why we think that people should be treated early.

It’s an interesting conversation because you’re talking about medications, all of which have some sort of adverse effects and some of which have significant safety issues. But it’s a matter of educating and explaining to people. Of course, our job is ultimately to give an individual the information they need to make a decision on how they want to move forward.