September 28th, 2009
12:43 PM ET

Pushing ahead with multiple sclerosis therapies

By Georgiann Caruso
CNN Medical  Associate Producer

When I first heard about the possibility of a pill for multiple sclerosis (MS), I exlaimed, "No way!" As a person living with MS, I know that this would be a enormous advance for the MS community. The option of a pill instead of needles sounds incredible, making it so much easier for many people to stay on their therapies since currently they all involve needles. Delving into this further, I realized that several drugs in trials seemed to hold great promise, and they are not light years off.

But things aren't as simple as they first appeared.

First off, an explainer for those unfamiliar with the disease who might be curious, because chances are you know someone affected by MS. The National Multiple Sclerosis Society defines it as a "chronic, often disabling disease that attacks the central nervous system (CNS), which is made up of the brain, spinal cord, and optic nerves." The cause isn't yet known and there is no cure. What happens is the myelin - the covering of the nerves - is destroyed by the immune system. The result is scar tissue; therefore nerve impulses, or messages, are slowed down leaving people with any number of symptoms including balance, speech, vision and memory problems as well as fatigue and heat sensitivity, to name a few.

Currently, there are five potential first-line disease-modifying therapies on the market, which became available starting in the early 1990s. Four are injectibles, and vary from being given once a day to once a week. Then a few years ago, the drug Tysabri was introduced, offering a new option for people with MS as it is given intravenously once every four weeks. The choice is up to the patient and the provider.

The drugs are intended to reduce the frequency and severity of MS attacks and slow the progression of the disease. Depending on the therapy, clinical relapses can be reduced by between 1/3 and 2/3.

But the FDA has yet to approve a therapy that can be taken orally. That may soon change.

Scientists from the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) just wrapped up a meeting in Düsseldorf, Germany where they discussed this and other exciting developments in the MS community. So what does this mean for the more than 400,000 people in the U.S. - and the more than two million people worldwide –that have this debilitating neurological condition? And what does it mean for their families, friends and caregivers?

I posed that question to Dr. John Richert, Executive VP, Research & Clinical Programs of the National Multiple Sclerosis Society, who emphasized that this is a very exciting time in the MS community.

He brought back news of the emerging trends discussed at the conference:

– The first oral symptom-management drug designed to improve nerve conduction, Fampridine, is already before the FDA for review. Fampridine has been shown to improve walking speed, but may also prove to help with other symptoms of the disease (which are less easy to quantify.) Expected to follow soon are the oral disease-modifying therapies Cladribine and Fingolimod. It's important to note though, these new drugs may well carry different risks than some of the currently available options. So while the method of taking them may be easier, they will not come without their own set of possible side effects and, as Richert suggests, there will be some unchartered territory as more people take the drugs.

-Therapies with infrequent dosing (such as once a year) are being studied.

-Stem cells hold promise for new MS treatments. Of particular interest are adult bone marrow mesenchymal stem cells. According to Richert, it's a bit too early yet to know whether this approach will prove safe and beneficial, or how exactly the therapy will work.

-Within the next year or two, most of the common genes that predispose people to develop the disease will be identified. This is important not only for prevention, but for treatment as well. However, further research is needed about environmental triggers to see how genes and the environment interact to cause MS.

MS is a complicated disease and varies among individuals. What is your realistic hope for the disease and research? Now or 5-10 years down the road? We'd like to hear from you.

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