By Ed White
Fredericksburg, Texas

ALS: OBSERVATIONS & REFLECTIONS

I am now in my 11th year of ALS, and I thought it would be interesting and possibly worthwhile to reflect on some of my experiences, frustrations, and thoughts concerning ALS that have been developed over this time. I should emphasize up front that these are simply my personal feelings and do not reflect every patient's experiences, nor do I expect everyone to agree with some of my conclusions. I should also point out that although I have experienced many frustrations, the glass is still half full, and I will continue to try to live in day-tight compartments and orient toward the future.

I should first report on my current status. I am a quadraplegic and am now in my 8th year of nasal ventilation (NIV) out of a total of 101/2 years with ALS, and 6 of these years have been on a 24/7 basis of respiratory assistance via NIV. Also, earlier this year I had a PEG tube inserted.

My comments, arranged by subject, are as follows:

INCIDENCE & PREVALENCE:

The current prevalence numbers for ALS in the U.S. are about 30,000 patients. However, demographics equal destiny, and short of a cure, we are about to witness an epidemic of major proportions in the incidence and prevalence of ALS in the U.S. This is due to the fact that there are now 78 million Baby Boomers moving into the "age of ALS". Unless we find a cure quickly, this is simply unavoidable.

Currently, if one includes all the variations of ALS (a lumper vs. a splitter), I believe our current prevalence number of 30,000 is low. I have simply witnessed too many patients, particularly older patients, who have been diagnosed and died within a year. These patients never show up on any radar screen. It has been told to me by a number of neurologists that the only way we will ever get an accurate count of ALS patients is to go door to door in a multi-regional, multi-year epidemiological study, and then the expense cannot be justified by the degree of accuracy obtained. It is most distressing to me that I still frequently see ALS prevalence numbers of 15,000-20,000 quoted in Washington by members of Congress and various government agencies. This has a seriously detrimental effect on our ability to compete for scarce resources.

I do not believe we have done a very good job of publicizing the ALS prevalence numbers or the epidemic we are about to experience in this decade. This coming explosion in ALS cases will overwhelm the limited resources to deal with patients' needs. Our current numbers are really academic. I would like to see an epidemiological study based on what we can expect in the next decade. In other words, take our current base of 30,000-plus; factor in the higher incidence of ALS occurring between the ages of 40 & 70; and super-impose on this the 78 million Baby Boomers entering this age over the next decade.

Other diseases are utilizing this strategy now; why shouldn't we do this with ALS also? Forget old epidemiological studies that are inaccurate and based on a few isolated counties in some state. These are not only inaccurate, but also handicap our ability to compete for scarce resources in Washington. These old studies should be relegated to the round file, and we should orient to the future.

RESEARCH:

When I was diagnosed over 10 years ago, there were at least 8 drug companies actively working on ALS. There was a great deal of enthusiasm concerning neurotrophic factors, and expectations were high. I see much less participation by drug companies today. This is unfortunate since it is the drug companies which have the resources to conduct expensive research and trials in bringing a drug to fruition. Over the last 10 years, ALS research has been very unremarkable at the patient level. If somehow we could plot the diversity of ALS research projects on a graph, it would certainly form a good duck pattern.

In other words, we have spread small amounts of money across a great number of projects. I believe it is time to change direction, and concentrate and focus our meager resources. If it were up to me, which it is not, I would allocate 50% of any research budget to therapeutic embryonic stem cell research. I feel this way, not because I know that stem cells will show efficacy for ALS, but for 3 reasons:

1) Therapeutic embryonic stem cell research has great potential for a quantum leap forward.

2) it is an area where we need a lot of questions answered quickly.

3) The political window is partially open, and I don't know how long this will exist.

I, like many other patients, have read hundreds of ALS research reports and abstracts. It's ironic, but now I find I follow people like Michael J. Fox and Christopher Reeve more closely than ALS research. I guess I'm hoping that ALS can prosper by piggybacking on the work surrounding them and their concentrated efforts concerning stem cells. In the meantime, the only thing that we patients have at our disposal concerning pharmacology is to establish an aggressive drug/supplement regimen early in our diagnoses; I will discuss
this later.

MALS vs. PALS:

The acronym, MALS, or manager of ALS, is a very accurate description of the task facing the ALS patient and caregivers. I know of no other disease that involves so many dimensions and requires such extensive management skills to successfully cope.

Managing your way through the ALS maze is analogous to those skills required in managing a small business. Whether it is equipment purchases, implementing respiratory protocols, organizing caregivers, or trying to find the financial resources to cope, ALS requires above-average management abilities.

An example of this involves equipment. My home is full of ALS-related equipment. This includes respiratory equipment, computers, augmentative communication devices, a specially equipped van, lifts and slings, a generator for backup power, signaling devices, and on and on. Maintaining this equipment is a real challenge and sometimes very frustrating.

For example, about 2 years ago, I purchased a piece of ALS equipment and the service representative left his cell phone number if I needed service. In about 6 months I needed service and contacted him via his cell phone. I reached him in Tennessee driving an 18-wheeler (I live in Texas). He had left the equipment company and was now a truck driver; we had a pleasant conversation while he was trucking down the interstate in Tennessee, and I never got the equipment serviced. The turnover with equipment suppliers and their reps is incredibly high, and will test the patience and management skills of a healthy individual, let alone an ALS patient.

Personnel turnover in every category of ALS that I deal with is one of the biggest and most frustrating problems I have. I have so much equipment I could easily justify a part time maintenance man, but there is no such person because of the diversity of the equipment. These problems must be dealt with the best they can, and I don't have an easy answer for it.

I might add that in many instances, the ALSA and MDA clinics and chapters have equipment loan banks to offer some relief.

In summary, MALS is a very appropriate term for the ALS patient. The patient and caregivers must develop a level of management skill to cope with ALS and anticipate the inevitable changes while staying ahead of the curve. Staying ahead of the curve and planning ahead are extremely important. Management, after all, is about managing change, and that is what ALS is all about.

RESPIRATORY MANAGEMENT:

One very bright spot in managing ALS is the advancement made in respiratory equipment over the last 10 years. A good respiratory management program is, by far, the best "drug" we have available at our disposal at this time.

It is estimated that only about 20% of ALS patients in the U.S. have access to a good respiratory management program; this is very unfortunate.

Every week for years I have received e-mails from patients around the U.S. desperate for assistance in the area of respiratory management. They face problems such as not being able to get a doctor's order for equipment; the wrong pressure settings on the equipment; poorly fitted masks; and having oxygen ordered for straightforward neuro-muscular problems, which can have very serious consequences. It is simply a jungle out there when it comes to proper respiratory care for the majority of ALS patients.

As I mentioned earlier, I am now in my 11th year of ALS. I am currently in my 8th year of nasal ventilation (non-invasive ventilation, or NIV), with 6 of these years being on a 24/7 basis. There is a right side to the bell curve, and sometimes people forget this.

In other words, NIV is a viable long-term option for respiratory care for a certain percentage of patients, and anyone who says it isn't simply does not understand the finer points of NIV as it applies to a neuromuscular patient.

Early on, I used a volume ventilator in addition to a BiPap for respiratory assistance. The volume ventilator had a number of advantages for me, and I believe contributed significantly to my long-term success with NIV. I now utilize the volume ventilator about two thirds of the day, and the BiPap at night for sleep. It is the best of both worlds.

One of the frustrations many patients have is finding someone to help them establish a good program. The ALSA and MDA clinics can be of assistance here initially. But the longer-term program must be managed by the patient and his or her caregivers. In my case, the turnover in respiratory therapists was unmanageable, and most were of little assistance.

Early on I decided that if I was going to survive ALS through a respiratory management program, I would have to do it myself with the help of my caregivers. I doubt that I would be here today if I had not made that decision.

It is very difficult to find experts in this area that have hands-on, in-home experience and understand all of the important idiosyncrasies that accompany a personal respiratory management program.

It is impossible to manage such a program on a long-distance basis. ALS is local, and that has never been truer than when it applies to respiratory management. I have not had anyone in my home for years who has assisted me with my respiratory care except for my caregivers.

If early on, a patient is told that nasal ventilation is not a long-term option, it is a good idea to get a second opinion. In my case, I am certainly prepared to move to a tracheostomy when and if it is needed, but switching prematurely makes no sense to me.

In summary, NIV is a long-term option for a certain percentage of patients; I don't know what that percentage is. If it is not a long-term option, it is an important intermediate step where the patient and caregivers can become familiar with all the aspects and variables involving respiratory management.

Also, I strongly recommend that the patient move to nasal ventilation when their FVC hits 75%. When I started, I waited until I reached the 50% level, and my FVC was dropping so quickly that I almost didn't make it.

Finally, there is wonderful new and compact equipment on the market which has both volume ventilator and BiPap capabilities with all the necessary alarms; these are perfect for the ALS patient whose respiratory capacity is changing, and can serve the patient in either NIV mode or with a trach.

Respiratory management is our best "drug"; NIV is a long-term option for many; I am living proof of both of these. But the road from neurology to pulmonology can be treacherous and difficult to navigate. It is full of pot holes and many delays. Unfortunately, many patients have not been able to make this transition.

My advice is to start very early in the process; pay particular attention to details; and don't assume anything. When dealing with respiratory management, pro-activity is a must. Some mistakes can be expected, but errors of commission are much preferred vs. errors of omission.

DRUG/SUPPLEMENT REGIMEN:

In my opinion, those patients who establish an aggressive drug/supplement regimen shortly after diagnosis seem to do better with ALS. This, of course, is based only on anecdotal evidence from my numerous contacts with patients over the years. I don't know if this is due to the regimen, or the patient's pro-active nature, or some combination of both.

In my case, I did establish a regimen early on based on the data I had available to me. I have adhered to this regimen for over 10 years, making alterations to it when additional information became available.

I currently take 20 drugs and supplements per day, which involves about 55 pills per day, and 2 injections per week. Obviously, I cannot state for certain that this regimen has slowed my progression of ALS. I can say for certain that it has greatly enhanced my immune system; I have not had any type of infection or illness in over 6 years, and this goes a long way in battling ALS, particularly the avoidance of any respiratory infections. In fact, in a number of ways, I am healthier today than I was before ALS.

Today there is much more information available to the patient for establishing a regimen. There is now more information available involving combination therapy of existing drugs and supplements, and the synergistic effects when taking a combination of antioxidants.

It's important for the patient to get up to speed quickly, and to get on some type of regimen. It is also important that in doing this, the patient not be discouraged by anyone who disparages it as a waste of money. As more data becomes available concerning the efficacy of certain combinations and the synergistic effects of others, the patient can factor this new information into his or her own regimen and make changes accordingly.

All I can say in summary is that I will never give up my regimen, but will only make changes along the way. Establishing an aggressive regimen along with a good respiratory management program is the only real tool we have for surviving ALS.

HOPE:

Hope is a wonderful companion but a poor guide. When fighting any serious disease, hope is an absolutely necessary companion.

Without hope, any patient is handicapping himself. And, there is no such thing as false hope; hope is simply hope. It is distressing to me when someone in the medical profession directs the term "false hope" toward an ALS patient.

It is an oxymoronic and condescending comment that should never be directed toward an ALS patient. When we don't know what causes ALS, and have no idea how to cure ALS, who in this world is to declare something to be a false hope.

There is always hope.

But, hope is a poor guide when establishing protocols, such as a drug/supplement regimen or a respiratory management program. Here, decisions should be based on the facts and data as we know them. We must simply base these protocols on the best information available to us. As I mentioned initially, hope is a wonderful and necessary companion, but a poor guide.

Finally, if you are working with someone in the medical profession who discusses mortality, life expectancy, and getting your affairs in order, I would suggest you find another doctor, since this person is not on your side. ALS is not necessarily fatal, and the patient is not well-served by someone who espouses that line of thinking.

ALS TRIALS:

In my opinion, double blind ALS drug trials utilizing a placebo or super-placebo (Rilutek), are not only archaic, but unethical; they are best relegated to the last millennium.

But more simply, these types of trials are leading to questionable results because of the increasing levels of adulteration which are occurring.

Patients are increasingly reluctant to forfeit all or part of their drug/supplement regimen for the chance to receive a super-placebo for 6 - 12 months. This type of trial is best left to drugs for Acne/Laxatives/Sexual prowess, but not for ALS.

There is a better way.

In future ALS trials, we should utilize our extensive database from past trials, and create a computer-generated placebo. In addition, the patient should be given a dose level of the drug being tested that is shown to be efficacious in the Stage 2 trial. In other words, no super-placebo. If the drug is good, and is effective, we'll know it.

Now, this isn't perfect, but what we are currently doing is less perfect and is leading to results that are statistically questionable. The level of adulteration reached relatively high levels in the Myotrophin trials, and has gotten progressively worse, since patients are refusing to part with certain regimens they have established.

Each patient must make up his or her own mind concerning trial participation, and it is a very important decision to forgo one's current drug/supplement regimen for the chance to receive a placebo, or super-placebo.

In addition, trials involving existing drugs for possible off-label ALS applications present a unique problem. Here, one can simply go out and buy the drugs directly, and therefore, the only meaningful answer for these trials is to give the patient a dose level of the drug being tested that was shown to be efficacious in Stage 2.

In summary, trial participation is becoming a very dicey decision and requires considerable thought by the patient before participating. Questions such as being able to continue one's regimen and whether the participant will immediately receive a dose shown to be effective are important questions.

We are dealing here with life and death decisions, and I believe strongly that we must bring our trial technology into the 21st century and improve accuracy and attract participants. We can do better than our current trial protocols.

Finally, I hope we have seen the end of ALS trials involving sham surgery, or "placebo" surgery, to accommodate placebos when intrusive procedures are required.

It is very probable that future ALS trials will require some type of intrusive procedure. This dictates that we develop, in advance, a computer-generated, statistical placebo. The use of sham surgery is not only bad ethics, but it is also bad science and bad medicine.

COMPETITION:

Like most ALS patients, I have been in competition with something all my life. It started with high school athletics; carried through into a very competitive business environment; and I even competed with myself in numerous marathons. Now I find myself competing with ALS.

Initially I thought this was unhealthy, but now I do not feel that way. It has always been my nature to compete, so why not carry through and direct this competitiveness toward ALS. I have certainly had enough experience in this area and find it pays dividends with ALS.

In my opinion, competition with ALS is beneficial; why not use it to one's advantage?

HEALTH MAINTENANCE:

For the ALS patient, it is very important to treat the total package. In other words, one should maintain all aspects of health in addition to addressing ALS. Psychologically, this is very important. ALS is local. By that I mean it cannot be treated on a long distance basis; there are just too many day-to-day, in-home issues to consider. This also includes other aspects of the patient's over-all health. So, it is important to find local doctors and dentists who are understanding and willing to work with you.

In instances where the reputation of ALS has preceded you, and a doctor shows limited understanding, or his eyes glaze over when you talk to him, simply move on and find another doctor. They are out there and can be invaluable to the patient whether dealing with ALS-related problems, or other health issues.

If one expects to live with ALS long-term, they must maintain all aspects of their health, and not just focus on ALS alone.

CENTIMETERS:

I have often said that ALS is a disease of centimeters. By that I mean, when I ask for some movement, it is never more that a few centimeters, whether it is my position in the wheelchair, position in bed, position of my mask, position of my sip and puff, and so on. Movement is never more than a few centimeters.

One of the first things I try to emphasize with my caregivers is to think in terms of centimeters; ALS is a disease of centimeters.

FINANCIAL:

Living with ALS on a long-term basis can be one of the most expensive diseases to deal with. Expenses cover the gamut from respiratory equipment to caregivers to drugs and supplements, and so on.

The interesting thing that has struck me over the years in communicating with patients is the variation in expense levels. Some who are quads with trachs spend relatively little.

This, of course, puts a heavy demand on family and friends. Others I have spoken with are spending over $100,000 a year for equipment, caregivers, and medications. Most patients fall somewhere in between. Medicare can be of assistance on some of the necessary equipment. Unfortunately, there is no help there when it comes to outside caregivers and nurse assistants. Hopefully, the efforts made here by David Jayne and others will change Medicare's approach to ALS patients who are homebound.

In summary, ALS requires a certain financial minimum for survival. But, I have talked with so many patients who have demonstrated to me, although difficult, that where there is a will, there is a way. And where there is a strong will to survive, they somehow pull the resources together to make this possible.

CAREGIVERS:

Because of the debilitating nature of ALS, good caregivers are an absolute necessity for living with the disease. I am fortunate in that my wife, Linda, is a wonderful caregiver and serves as the focal point for my care.

I am also fortunate to have good Home Care nurses who have been with me for years and come to my home every day.

I know this can be a problem as many patients continue to battle the turnover with outside caregivers. From my experience in talking with patients over the years, the inability to find good caregivers is second only to the inability to establish a good respiratory management program. This is a serious problem, and unfortunately, is getting worse.

MIND AND BODY:

Before ALS, I never gave a great deal of thought about the connection between the mind and the body. After being diagnosed over 10 years ago, I have spent a great deal of time thinking about this subject. I believe that trying to battle a disease like ALS without having your mind in gear, or in sync with your body, is analogous to having one arm tied behind your back. In other words, there must be both a physiological and a philosophical approach to any serious disease. My "philosophy" towards ALS can be summarized with 3 points.

My underlying, or basic philosophy was laid down by Descartes over 3 centuries ago when he said, "I think, therefore I am". As long as I have my cognitive abilities, I will do everything in my power to survive ALS.

Secondly, a heavy dose of stoicism is a must. One can learn to be stoic, and with ALS, we must accept those things we cannot change, and they are numerous.

Finally, I have always been a little bull-headed, and it is finally paying off with ALS. I just won't let those things I cannot do interfere with those things I still can do. Although my body is out of order physically, I believe the activities I am still capable of doing and enjoying, outweigh those I can no longer do.

So in summary, give me one part Descartes, one part stoicism, and throw in some bull-headedness, and I'll live with ALS. Epicurus said that the unplanned life is not worth living. In that regard, I will continue to plan and manage my affairs with an orientation to the future. Finally, hope does spring eternal; there is always hope.

And, then, there is stress. Excessive stress is extremely debilitating for the ALS patient. Everything about ALS is stressful. The patient and caregivers must find a way to minimize stress. Developing a philosophy for coping with ALS can help.

Stress can never be completely eliminated even when we are healthy, but for the patient to successfully live with ALS, it must be kept at a minimal level. High levels of stress with ALS adversely affect the way we breathe, speak, move, swallow, and even think. In short, stress is a direct result of ALS, but it is incompatible in living with it.

COURAGE:

Does it take more courage and personal fortitude to live with ALS or, conversely, to decide not to live with ALS? I thought about this question numerous times over roughly the first 5 years of my diagnosis. Fortunately, I don't ask myself these questions anymore.

I have many ALS friends who have chosen to live with ALS. This takes courage, particularly when the patient is facing the prospect of a locked-in status. I also have had many friends over the years who have chosen not to live with ALS, either by refusing respiratory care, or by directly taking their own life.

In my opinion, any of the above actions takes courage, and I would never be judgmental concerning a particular patient's decision. Each patient is unique, including his or her circumstances; there is no template that fits all when it comes to decisions relating to ALS or any other life-threatening disease.

On numerous occasions, people without ALS have insinuated to me either directly or indirectly that they could not exist the way I am living. I've decided that I find these comments somewhat puzzling and irritating, and I've concluded that I would not indicate my feelings to a patient if the role were reversed. I never know whether the person is saying this because they are admiring my personal fortitude to survive, or whether it appears to them that I am very high maintenance (which I certainly am), and am wasting the time of the people around me.

In short, until they are in the patient's shoes, no one is qualified to make that statement.

In summary, both the decision to live with ALS or to die with ALS is one requiring courage and personal fortitude. I will say that ALS is not necessarily fatal, but to decide to live with ALS involves many factors and is very unique to the patient and his or her circumstances.

Ed White
Fredericksburg, Texas