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ALS Registry

June 2013

In this Issue . . .  

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

Dr. Kasarskis

The Nuts and Bolts of Clinical Trials

Q: What do you tell your patients when they are considering participating in a clinical trial? Is it worth the effort?

A: I can understand your concerns because clinical trials involve your willingness to be part of something without having any idea whether the study will be helpful to you. 

I always tell patients that they should understand that a clinical trial is actually an experiment. The purpose is to discover if a drug that worked on animals can be safe and effective with humans -- and work better than a placebo or the current best treatment.

When patients sign up to be part of a study, they sign a consent form that is actually a legal contract. It usually ranges from 6 to 25 pages long. Signing the "contract" can be a little overwhelming. Usually patients get the consent form to take home, and they come back and sit down with a doctor, nurse, or other member of the clinical trial team to review the form.

Patients agree to put their bodies on the line, volunteering to follow instructions related to the trial, such as faithfully taking medication or following some prescribed regimen.  They also will have blood or urine taken at regular intervals, or whatever else is spelled out in the form; provide personal information; and agree to record side effects.  They come for a study visit regularly, a process which can take up to 6 to 8 hours of poking and prodding. Patients are also informed that they are free to quit the trial at any time, no questions asked.  But for the good of the study and advancing knowledge, we hope that you stay with it to the end.  That means you should really understand what is being asked of you and make the commitment.

At the same time, the people in charge of the trial have responsibilities, too. They are expected to conduct the study to the best of their ability, exposing patients to the new treatment in the safest way possible, and by monitoring them very closely. If there's a hint of any sort of problem, the trial leaders will do whatever is necessary to resolve it. Patients won't have to pay for tests, the drug or treatment, or for exams.

So why should you do it? Everybody who participates in a study gets a great psychological benefit just by participating.  Participants are also monitored more than the average ALS patient, a factor which may contribute to better management of ALS.

Yes, it's true: any trial participant could be placed (randomly, like a coin flip) on a placebo rather than the active test drug. But sometimes people taking the placebos are the lucky ones; the non-active drug may turn out to be no less effective than is the trial medication, or the test drug may actually cause troubling side effects or be harmful.  We have seen this in some ALS drug trials.

It's helpful to know the three different types of trials involved in testing a potential new drug. Typically in what is called a "Phase 1" clinical trial, healthy humans are exposed to a single dose of the drug and monitored closely to see how quickly the drug is absorbed and how long it stays in the bloodstream.  This will develop information about how safe each dosage seems to be.

Once the nuts and bolts are tested in normal individuals, a "Phase 2" study tests the drug in the target population, with people who would actually need and will take the medication.  In our case, these are patients with ALS.  The researchers need to determine if the drug behaves similarly for these people just as it did in healthy volunteers, whether the dose is correct, and what the side effects are.
Actually the process can be sped up a bit since ALS is an uncommon illness.  Trials related to ALS may qualify for "orphan drug status," allowing a drug to be tested right away in ALS patients, bypassing normal volunteers.

Next, there's a "Phase 3" trial, which takes the drug at the now-determined dose and evaluates it with a larger number of people.  This time, the drug is compared to a placebo or current standard of care to prove that the drug is effective or an improvement over current therapy.  Phase 3 trials are the definitive trial  the outcome is critical to the drug approval process -- and have the most patients enrolled.

To get the drug approved by the Federal Drug Administration, (the FDA), the drug maker must provide the results of two independent studies that demonstrate the same results.

When does a patient get to know how the trial turns out? After all the data are gathered from the very last patient and after all the data are sealed so they can't be tampered with, the test site is notified about which of their patients got the active drug and which got placebos. A patient enrolled early on in the study process may finish the trial early, but all patients usually have to wait until the trial is completed before they'll learn whether they were taking the active drug or the placebo. (If early data shows the drug is extraordinarily effective, patients on placebo may be placed on the active drug). But typically, the first patient enrolled in a study might have to wait 2 years to know.

Many patients wonder or worry about how all this is regulated. There are actually two levels of protection for clinical trial participants:

  • First, the details of how the study will be conducted are submitted to an independent "Institutional Review Board" (IRB) at the trial site, typically a university.  Physicians, nurses, pharmacists and community members are on the boards, and they review all the design and operational details.  You must have IRB approval before the study can start.  The IRB reviews and approves the consent form document that forms the contract between the study site and the research volunteer
  • "Data Safety Monitoring Board," a small group of people not connected with the study or the drug company in any way, monitors how the trial is conducted. The members of the board are the only people who know in real time who is taking the active drug and who is on placebo, and they watch for adverse events and other issues. They have the authority to stop the study immediately at any time should that be necessary.

So, now back to your original question: Yes, participating in a clinical trial is not only worth your effort but it is essential. Without people like you, no drug will ever be approved in the U.S. Every one of us benefits from what is learned from clinical trials of drugs, whether the treatments are found safe and effective, or not. 

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.

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.   

Traveling to National Parks this Summer: What You Should Know

By Barbara Bronson Gray, RN, MN

Traveling to National Parks

It's summer! And it's a great time to see the nation's spectacular national parks. But those with mobility challenges and other disabilities may wonder just how much of the beauty they'll be able to see. Will Visitor Centers and other places of interest be accessible? What tours and other activities might be available?

Most of the national parks today offer a wide range of access and services to people with disabilities, and getting out in the great outdoors need not be daunting.
Altogether, there are about 200 million acres of national park land, including national recreation areas, national forests and other parks. No two parks are alike; each offers unique vistas and a broad range of different experiences for those with physical disabilities. Some have magnificent, historic lodges, and others have more modest facilities, but with exceptional views. And most are now accessible to everyone.
For example, at Kenai Fjords National Park, in Alaska, all cabins have wheelchair accessible doors and decks. At Grand Teton National Park, in Wyoming, the Jenny Lake shuttle boats are ADA-compliant. Great Sand Dunes National Park and Preserve in Colorado loans out sand wheelchairs that make it easy to cross the dunes. Visitors there can also camp in the backcountry at a site that is a tenth of a mile from a parking area.

The National Parks offer a free, lifetime special access pass for those with a permanent disability (defined as a permanent disability that limits one or more major life activities, such as caring for oneself, performing manual tasks, walking, breathing, or working, among others). The pass allows you to visit federal parks at no charge, waiving entrance and camping charges. It also offers discounts on certain other fees, including swimming, boat launching and guided tours. A pass can be obtained in person from a participating federal recreation site or office, or by mail.

Once you've decided on a park (or parks) to visit, it's smart to check out the park's website to learn more about the facilities and get up to date on any closures, weather conditions or other issues. Make sure you have reservations for camping, lodging and any activities you're interested in, if possible. Talk directly to a ranger. He or she will be able to answer all your questions and give you a better idea of what experiences are available.

Even if you have reservations at a park, it's always a good idea to have a back-up plan. Bring information about nearby hotels with you.

Travel experts warn everyone who visits a national park to be careful not to try to do too much. It's important for everyone  whether you have a physical disability or not  to pace yourself realistically, and take the time to sit back and take in the amazing beauty.


  • For specific information about the accessibility options available at a National Park you're interested in visiting, see
  • For a one-stop shop for trip planning and reservations brought to you by 12 federal agencies (including the Army Corps of Engineers, Forest Service, National Park Service, Bureau of Land Management, Bureau of Reclamation, Fish and Wildlife Service, and the National Archives), and for advance reservations at 2,500 federal areas for over 60,000 facilities and activities, visit
  • If you're interested in camp sites, visit to access extensive lists of camping facilities available in all 50 states.   

Tips for Using Urgent Care Centers

By Barbara Bronson Gray, RN, MN

6 01 2013

What do you do when you can't get an appointment with your physician or have an immediate need on a weekend or after office hours? Many people turn to urgent care centers.

For those with ALS and their families, it's especially important to know how to use these resources most effectively.

Urgent care began in the late 1970's and grew slowly as the concept of seeing a physician you didn't know for a non-emergency began to become more acceptable.There are now about 9,000 urgent care centers in the U.S., and approximately 600 new centers are opening every year, according to the Urgent Care Association of America. The trend is being fueled now by a wide range of factors: longer emergency room waiting times, reduced availability of primary care physicians, and fewer truly "on call" physicians who are willing to see patients on evenings, weekends and holidays.

Urgent Care facilities are designed to help fill the gap between primary care physicians and emergency rooms. They should not be used if there is any question in your mind whether the situation is an emergency. If you're not sure, it is best to go straight to the emergency room for evaluation or call 911.
Most urgent care centers will give immunizations and treat eye and ear infections, colds and flu, minor burns, cuts, and minor sprain. Some also provide x-rays, vaccinations, physical and occupational therapy, and other services. As for laboratory testing, some only provide the basics, while others have a complete range of services, approaching the level of an emergency department.

In addition to free-standing urgent care centers, there are more than 1,000 retail clinics in many chain stores such as Walgreens, CVS Caremark and Walmart. These typically offer convenient care for minor medical conditions and provide immunizations.

It's wise to scope out your community's urgent care centers before you need one. You can ask your primary care physician if there is a facility he or she would recommend. You can also visit them, or ask a friend or family member to do so, to find out whether the center seems to run efficiently, the names and credentials of the physicians and staff, what insurance coverage they accept, and what hours they are open. Visit the center's website to learn more about them. Once you find a place that meets your needs, keep the facility's phone number, address and hours handy.
One of the downsides of getting care from someone who doesn't know you is that the physician and staff will be unfamiliar with the drugs and treatments you are taking. While that is true for all of us, it can pose particular challenges for the person with ALS.

It's a good idea to have a folder or even a large, zipped plastic bag  anything that's easy to bring with you  ready at all times, just in case you need any type of emergency care. Be sure it includes your medical history, a list of your prescription drugs (and their dosages), and any other medications you are taking, a description of any respiratory assistance you use at night or during the day, and the names and phone numbers of key family or friends to contact if necessary. You should also include a brief description of ALS, since some healthcare staff may not fully understand the disease and its impact. If you have difficulty communicating, such information can be especially important.

Remember, too, that your primary physician or neurologist most likely won't be automatically informed of your visit to an urgent care center, (or even an emergency department). So ask for a copy of any lab work, x-ray analysis or work-ups you get, and give a copy to your physicians the next time you see them so they are up to date on your health status. Be sure to keep copies of those reports for yourself, too, since you may want to refer to them in the future.

If you're planning to travel, it's smart to scope out the urgent care centers in the cities and towns you'll be visiting, so you'll be prepared, just in case. 


Participate in these Educational Web Calls

Turn your Existing Home into a Smart Home 

Monday, June 17, 2013, 11 a.m. Pacific Daylight Time, 2 p.m. Eastern Daylight Time

Call-in toll-free number (US/Canada): 877.668.4490
Session number: 826 081 122
Session password: ATcalljune2013

6 01 2013

MuSK Agonists as a Treatment for ALS

Hosted and organized by The ALS Association's Research Department
Learn how "MuSK agonists" have the potential to improve motor function in ALS.
Tuesday, June 18, 2013: 1 p.m. Pacific Daylight Time, 4 p.m. Eastern Daylight Time
Speaker: Steve Burden, Skirball Institute, New York University, New York
Click here for information on how to register for this call:

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