Ask the Doc: Q & A with Edward Kasarskis, MD, PhD 

Dr. Kasarskis

Edward Kasarskis, MD, PhD is Director of the University of Kentucky ALS Multidisciplinary Clinic at Cardinal Hill Rehabilitation Hospital 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: How can I slow the progression of ALS? I hear there is one drug that can make some difference now and then others might be on the horizon?

Dr. Kasarskis:  

This is a question every person with ALS understandably asks. Today there is just one drug approved by the FDA to extend life for people with ALS, and that is riluzole (Rilutek®).  Here we are in the 21st century and it seems we have drugs for just about everything, so it is especially frustrating for patients and families to learn that we don’t have more pharmaceutical options when it comes to slowing the progression of ALS and extending survival.  There are many studies being conducted now and new drug approaches are being tested.

Riluzole was approved based on two survival studies from the 90’s which demonstrated that riluzole extended survival compared to placebo. Some people read or hear about the results described in riluzole’s package insert and get discouraged, asking "is that all there is?" After the initial results were submitted to the FDA for the approval of riluzole, further research showed a consistent survival benefit from taking riluzole.

In the future, we will probably be treating ALS with combination therapy in much the same way oncologists treat cancer, not relying on a single drug to cure the entire disease.  It is also important to realize that during the initial studies of riluzole, ALS physicians did not employ multi-disciplinary care as aggressively as we do now. Life can also be enhanced and even extended by getting multi-disciplinary care, which addresses the full range of issues facing a person with ALS. 

Many clinical drug trials are in the development phases or are underway.  For example, ceftriaxone, an antibiotic already approved by the FDA, is under study to determine if it (alone or in combination with riluzole) will slow the progression of the disease. (Ceftriaxone has other effects, in addition to being an antibiotic, but ALS is not infectious). This is a multicenter study at 60 sites in North America coordinated by Massachusetts General Hospital and supported by the National Institute of Neurological Disorders and Stroke (NINDS). For more information, visit http://clinicaltrials.gov/ct2/show/NCT00349622.

You should discuss participating in this or other clinical trials in your area.  As patients, we all stand on the shoulders of other patients before us who volunteered their time and their bodies to participate in a clinical drug study to prove that our blood pressure medication, or our antidepressant, or our drug for ALS is safe and effective. Clinical trials typically require regular testing and evaluation. They usually provide great psychological benefit, just from participating in something that can further our understanding of ALS and could directly help you and other ALS patients in the future.

Each issue we feature a leading neurologist specializing in ALS responding to a question. If you would like to submit questions for a future Q & A, please send your questions to Amber Walters. Please understand that we won’t be able to address all questions and we won’t be able to respond to individuals personally.