The ALS Association

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.

Q: Im taking Rilutek now but am wondering what else might be on the horizon in terms of potential drugs for ALS?

A: Thats a great question. There are several high-potential drugs being tested right now through clinical trials.

By the way, the drug youre taking, Rilutek (riluzole) has been around for about 20 years now. You should know that the American Academy of Neurologys 2009 Practice Parameters say that the drug is effective in slowing the rate of progression of ALS. Some physicians may downplay the benefits and potential impact of Rilutek, but its important to know that it does play a role in treating ALS. It is not a perfect drug, and so the quest for additional drug treatment continues.

When thinking about the future of drug therapy for ALS, most experts agree that the most effective treatment will likely be a drug cocktail, combining two or more medications, rather than taking just one. This may ultimately be our best strategy in treating the disease.

There are a large number of clinical trials in progress right now testing drugs, stem cells, and gene therapy that could change the course of the disease and preserve or improve muscle strength. Heres a quick update:

The drug is a three-dimensional mirror image of a current treatment for the symptoms of Parkinsons disease (Pramipexole). The chemical difference in structure may make "Dex" less likely to have unwanted side effects that some people with Parkinsons experience with the parent drug. A small Phase 2 trial of the drug in 102 newly-diagnosed people with ALS showed improvement in functional capability and survival.

A Phase 3 trial with almost 1,000 people enrolled should be completed very soon.

If, and this is always a BIG IF, this drug proves successful and is approved, its likely that it would be taken along with Rilutek.

Dr. Bob Miller, the principal investigator of the study, described the study results as "encouraging" in a Neuraltus press release: Halting the rate of disease progression, in a subset of patients, as this study suggests, would translate into a clear clinical benefit for these patients, he said. I agree completely. Unfortunately, rather than being an oral drug, the medication is given intravenously over several days at a time, for a period of months. The next step: a more definitive Phase 3 trial is likely to be started in 2013.

If youre interested in participating in a clinical trial in your area, visit http://www.clinicaltrials.gov. There is much work to be done.

This update is regarding one of the medications I discussed above, Dexpramipexole, by Biogen Idec. It looked very promising and I mentioned that a Phase 3 trial was expected to be completed very soon. I wanted you to know that, unfortunately, the trial was not successful. There was great hope that the drug would be effective in slowing ALS based on the preliminary studies. But in the end it did not turn out to be the case. This serves to remind us all that a clinical drug trial is, at the end of the day, an experiment. Some drugs succeed and others will fail. But the research effort must continue!

If you would like to submit questions for a future Q & A, please send your questions to theexchange@alsa-national.org. Please understand that we wont be able to address all questions and we wont be able to respond to individuals personally.

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The ALS Association