Edward Kasarskis, M.D., Ph.D. is Director of the multidisciplinary ALS Center at the University of Kentucky Neuroscience Center in Lexington, Kentucky, professor in the Department of Neurology at the University of Kentucky, and Chief of Neurology at the VA Medical Center in Lexington KY. 

Dr. Kasarskis

Q: What is your opinion of Rilutek^®? My husband was diagnosed with ALS three months ago. We’ve talked to two ALS specialists; one doesn’t endorse the drug and one does. My husband just started taking it, but we aren’t sure it will help him.
-- Andrea, New York City

A:  Good question. It’s true that, as you have experienced, ALS experts disagree about its relative value. Basically, this is viewing the glass as either half-full or half-empty. Riluzole (Rilutek^®) is frustrating because it’s not a perfect drug, and does not stop ALS in its tracks. But, in short, I recommend it.  I am in the half-full camp. The evidence-based guidelines for the treatment of ALS developed by the American Academy of Neurology (AAN) conclude that riluzole is safe and effective for slowing disease progression and should be offered to patients.

When we look at the original studies from the 1990’s to get FDA approval for the drug and subsequent studies since then, it appears that the advantages of taking riluzole are consistent.  The package insert, which is always conservative, says the drug provides a three month survival advantage -- meaning you are likely to live three more months than if you didn’t take the drug.  But several studies done in the last few years indicate that the survival advantage of taking riluzole may actually be greater.  It also seems that beginning the drug as soon after diagnosis as possible may offer advantages.

In addition to the modest extension of lifespan riluzole can offer, some people swear it makes them actually feel better. Some say they experience more restful sleep and others feel it controls fasciculations. We estimate that about 50% to 75% of people with ALS are taking riluzole.

There are a few downsides to the drug for some individuals.  Some just can’t tolerate it; they feel tired and run down. Of course, it’s hard to know if those symptoms are side effects of riluzole or simply part of what people with ALS often experience. Sometimes people who are older seem more prone to these side effects than are others.

It’s important to monitor liver function enzymes for the first few months (your family doctor can easily do this), but there is usually no permanent liver damage. And I recommend people start with an initial dose of just 50 mg a day, and then after a week take 50 mg twice a day. Studies that have looked at what dose is ideal have found that 100 mg is better than is 50 mg, but upping the dose to 200 mg conferred no additional benefit.

And riluzole is expensive. Without insurance or third-party coverage, the out-of-pocket cost can be about $1,200 a month. With coverage, co-pays tend to range from $20 to $200 a month. Medicare and the VA cover it, and the National Organization for Rare Diseases can help support the cost for the indigent.

No single drug or treatment should be viewed in isolation when dealing with ALS. The best bet is to tackle the disease with a comprehensive approach, including nutrition, respiratory therapy and support, physical therapy, occupational therapy, social support, and, yes, riluzole.

If you would like to submit questions for a future Q & A, please send your questions to Amber Walters at awalters@alsa-national.org. Please understand that we won't be able to address all questions and we won’t be able to respond to individuals personally.