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ALS Registry

January 2013

In this Issue . . .  

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

Dr. Kasarskis

An Update on Potential New Drugs for ALS

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

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

By the way, the drug you’re taking, Rilutek (riluzole) has been around for about 20 years now.  You should know that the American Academy of Neurology’s 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 it’s 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. Here’s a quick update:

  • Ceftiraxone: We just learned a few of months ago that a clinical trial was halted because the drug proved to be ineffective in a pivotal Phase 3 trial.  This was a complicated and very long study, and was particularly disappointing since there was a lot of good scientific support for this approach.  I know I speak for all the members of the study teams in thanking the patients and their families for their devotion to getting an answer. It was truly an amazing effort lead by Drs. Cudkowicz and Shefner, and supported by the National Institutes of Health.
  • CK2017357 (Tirasemtiv) by Cytokinetics: This drug increases a muscle’s strength when it contracts and might decrease fatigue. It has the greatest impact on what is described as mid-level exertion, such as walking, reaching and talking. CK2017357 appears to reduce fatigue and improve function in people with ALS. This drug, now in a Phase 2 trial, would also be used with other medications. It looks pretty promising.
  • NP001, by Neuraltus Pharmaceuticals: Results from a Phase 2 clinical study of NP001 were released this fall (on the eve of Superstorm Sandy, as it turns out, so you probably didn't read about it) and just reported at the International ALS/MND Symposium in Chicago last week. The study showed that 27% of patients who received this drug had no progression of their disease during the six-month period they were taking the medication. That means that twice as many people experienced no disease progression as compared to those who were taking a placebo (an inactive drug). The trials were conducted at 17 sites across the country, involving a total of 136 patients. People were randomly assigned to get a low dose, a high dose or a placebo. We participated in both the Phase 1 and Phase 2 trials of NP001 at the University of Kentucky.

    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 you’re interested in participating in a clinical trial in your area, visit  There is much work to be done.  

If you would like to submit questions for a future Q & A, please send your questions to Please understand that we won’t be able to address all questions and we won’t be able to respond to individuals personally.

Edward Kasarskis, M.D., Ph.D. is Director of the University of Kentucky ALS Multidisciplinary Clinic 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.  

Dealing with Tough Transitions in Care

Dealing with the healthcare system and managing critical periods of transition can be challenging to people with ALS and their caregivers. Here are two organizations that may be helpful:

  • CarePlanners
    • A fee-based service that provides advice on evolving needs, including assessment, 24/7 access to care planning experts, resources, medical billing and Medicare charge experts
    • Founded by Dr. Nancy Snyderman, a surgeon and chief medical expert on NBC News, and Alan Blaustein, a cancer survivor and investor
    • Staffed by nurses, nurse practitioners, social workers, Medicare specialists, insurance billing and claims experts
    • Call 800-989-3588 or visit for more information.
  • National Transitional Care Coalition
    • A nonprofit organization that includes organizations and individuals committed to addressing problems inherent with transitions in care, such as being discharged from a hospital or needing a new level of care
    • Founded in 2006 by the Case Management Society of America
    • Provides information and resources for consumers and health professionals, including a “Patient Bill of Rights During Transition of Care,” a toolkit for hospital discharge and guidelines for a successful hospital stay
    • Visit for more information.


Deciding When to Quit a Job


It’s one of the most challenging questions people with ALS face: how do I know when it’s time for me to resign from my job because of my condition?

For some, perhaps those near retirement or already thinking of making a change, the decision may not be too difficult. But for many, leaving a work situation can trigger fears about their financial future and a deep sense of loss as they contemplate exiting the work world due to their disability.

The decision often depends on an individual’s occupation. For someone with a desk job, such as an office worker or a writer, it may be possible to continue for quite some time. But for a person who does work that requires travel, prolonged standing, or manual work, it can be difficult and even dangerous to have even slight muscle weakness.

Experts suggest you first talk with your physician about your questions and concerns and get feedback about your best options. The first step might be to talk with your employer to explore whether job modifications or accommodations might help you stay in your role, at least for the time being. You should know that the Americans with Disabilities Act provides certain protections if you decide to stay in your job.

Thinking ahead – and around the corner – is important, especially since the process of getting disability takes time.

Ultimately, deciding when to stop working is a personal decision that depends on a host of factors, including the benefits you get from working (both psychological and financial), the impact and progression of your disease, and the nature of the work you do.

Here are some resources to help make the decision and any transition easier:

                                                                                                      By Barbara Bronson Gray, RN, MN


Participate in these Educational Web Calls

Research Webinar

Hosted by The ALS Association’s Research Department  
January 15, at 1:00 p.m. Pacific Time, 3:00 p.m. Central, 4:00 p.m. Eastern
Click here to view this webinar

Communication Options for Persons Diagnosed with ALS and FTD

Monday, January 28, 2013 at 11 a.m. Pacific, 1 p.m. Central, 2 p.m. Eastern
Speaker:  Alisa Brownlee, ATP, The ALS Association Greater Philadelphia Chapter
Hosted and organized by The ALS Association’s Care Services Department

Call-in toll-free number (US/Canada): 877.668.4490
Session number:    825 686 842
Session password:    Atcall2013

Click here to access this webinar.


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