When Former Caregivers Stay Engaged

A note of thanks to share during National Caregiver Month

Former caregivers of loved ones lost to ALS have every reason and every right to want to get as far away from that illness as they can. Every time they hear about ALS must bring back painful memories. No one can blame them if they walk away once the caregiving is done.

Which is why I am so impressed by and grateful for the huge number of former ALS caregivers who commit themselves to volunteer positions in our many ALS organizations. They make up a sizable percentage of the fundraising teams and board members in our groups, at both the local and national level.   And they devote time on support forums to provide help and advice to current caregivers. 

It’s not hard to understand why they do it – to honor the memory of their lost loved one, to support people going through what they went through, and to work for a cure to spare others of what they and their pALS went through.

What is harder to understand is how they do it – how they cope with the memories and emotions they must feel every time they encounter one of us with ALS, every time they are part of a conversation about support to current caregivers and pALS. These are people with special courage.

I know a thing or two about this. My younger sister, Janie, the second of four children, was born with severe developmental disabilities and required intensive, full-time care throughout her short life. My young parents, especially my mother, had to take on the very difficult job as a  24/7 caregiver for Janie with few resources and while trying to raise a family and pay bills. Luckily my grandmother was there to help.  Looking back, I don’t know how they did it.  It was a difficult, exhausting time for all of us. It made those years of my childhood less carefree than childhoods are supposed to be.

After Janie passed, I walked away. I avoided any volunteer or other engagement for causes involving children with severe developmental disabilities. It was too close to the bone, too difficult to revisit those hard times. I just wanted to close that chapter of my life and move on.

As I watch so many former ALS caregivers — now my colleagues —  throw their energies into our ALS organizations, I look back with regret that I did not have the strength to explore ways to support organizations dedicated to my sister’s condition.  But it puts me in a better position to fully appreciate how much emotional strength it must take our former caregivers who remain engaged in the fight to defeat ALS.

To all of you former ALS caregivers still helping us fight the good fight, you have our deepest thanks and admiration for all you continue to do for us. You are honoring the memory of the loved one you lost in a very powerful way.

ALS future scenarios: Anticipating the impact of new therapies

Summary:

The possibility of new treatments in coming years that could, slow, halt, or reverse the progression of ALS demands advance planning across a wide range of issue areas and stakeholder groups.  This paper reviews three of the most likely scenarios that new therapies could produce. It anticipates the impact of each scenario on ALS demographics, early detection, physician caseload, clinics and care services, fundraising, clinical trials, insurers and other sources of coverage, and on  persons living with ALS (pALS), their caregivers (cALS), and their family finances.

 It concludes that therapies that slow or halt the disease will produce major but manageable challenges to organizations dedicated to ALS care services, fundraising, research, and advocacy. Some of these challenges will require effective advanced planning to mitigate. Two of the most important emerging issues will include the need to develop much better early detection capacities, and the need for much higher levels of fundraising to cope with the dramatic increase in the number of people living with ALS.   

Impact of therapies that slow or halt the progression of ALS on pALS, their caregivers, and their family finances will be highly variable, resulting in very positive outcomes and options for some, and very difficult choices for others about whether the new therapies’ benefits outweigh the costs.   

A scenario in which a therapy reverses the symptoms of ALS for most or all pALS is obviously the most unambiguously positive and least complicated outcome. It will highlight the need to expand access, via international aid programs, to make the therapy affordable and accessible for the large and growing caseload of pALS in poorer countries. Advanced stages of the battle to defeat ALS will be waged in the global south.

Impact inventory

With several drugs and treatments now in Phase 3 clinical trials, and more in the research pipeline, medical researchers are increasingly expressing hopes that we will soon see one or more new ALS therapies approved and available, possibly within the next two to three years.

This optimism comes with lots of anxieties and uncertainties – about which therapies or combinations of therapies will prove to be effective, when they will be available, what percentage of people with ALS (pALS) will respond well to them, how much they will cost, whether insurance companies will cover them, and what kind of impact they will have on disease progression.

The latter question is especially critical. Will the new therapies slow ALS progression? Halt it? Reverse it?

Whatever the case, more effective therapies are going to have wide-ranging impact — on pALS, on pALS’ families, and on the wider ALS support, care, advocacy, research, and coverage ecosystems. It is not too soon to start planning and preparing for all of the opportunities and challenges that new therapies will bring, even if the first new therapies are several years away.

These need to be frank discussions, because not all of the impacts of new therapies are going to be uniformly positive. We have to be ready to handle both the good and the bad.

The good news is that organizations involved in ALS work are already thinking ahead about some of these issues.  The ALS Association convened a meeting in late October to bring together representatives from pharmaceutical companies, the FDA, and insurance companies – with some pALS representatives actively involved — to jumpstart discussions on questions of coverage of potential new therapies. This kind of forward thinking is crucial to help us get ahead of the curve on all of the issues that a new therapy will raise.

This paper is intended to anticipate the impacts of new therapies, based on three different scenarios. All of them over-simplify what will likely be a much messier reality. First, they assume uniform impact of new therapies, even though we know that variability of impact is much more likely, given the heterogeneity of the disease. Second, all refer to a single new therapy when we know that a more likely outcome will be an effective “cocktail of therapies” that will emerge gradually rather than all at once.  And all are based on impacts in the US setting. In other countries with different health care systems and income levels, aspects of these scenarios could play out quite differently.

It should be noted that though these are presented as separate scenarios, they could, and probably will, occur sequentially.

The author welcomes suggestions for amending this first cut at an “impact inventory” and hopes to update the paper using reader feedback to improve the analysis.    

Scenario 1: Slowing of progression

All of us living with ALS are hoping for new therapies that reverse the disease’s progression or even cure it. But we have to be prepared for more modest advances.

One possible outcome of this current round of new therapies could be a drug, a combination of drugs, or a gene therapy, that does not reverse the disease for most of us, but that significantly slows it down.

Let’s assume, for the sake of argument, that a therapy is approved by 2021 that on average halves the speed of progression.  That will double the median, post-diagnosis life expectancy of people with ALS from 3 to 6 years.  For pALS with slow progression, they could see their life expectancy leap from 7 to 14 years, or 10 to 20 years, or even more.

What are the implications?

ALS demographics. This will potentially double the number of people living with ALS. In the US, our numbers will quickly grow from 30,000 to 60,000, assuming most everyone has access to and uses the new therapy.  

In fact, this could actually be a significant underestimation of the total number of cases. How so? The aging of the US (and global) population is expected to naturally increase the number of ALS cases in coming decades, even without any new therapies to slow the disease down. One recent study estimates a 69% increase in the number of people with ALS globally from 2016 to 2040 (from 223,000 to 337,000), solely based on aging of the population.  Preparing for a sizable increase in the number of pALS seems prudent even if no new therapies are developed in coming years.

To borrow from the terminology of epidemiologists, ALS incidence rate (rate of occurrence of new cases, often expressed as the percentage of new cases of a disease per year per 100,000 people) will increase simply because of the aging population. The ALS prevalence rate (proportion of total cases in a population at a given time) will leap both because of the impact of the therapies that extend life expectancy, and because an aging population will produce more cases of ALS.

It is worth noting that this level of increase in the population of people with ALS in the US would not change the FDA and NIH’s categorization of ALS as a rare disease (defined as fewer than 200,000 affected by the disease), so ALS therapy development would continue to benefit from the provisions of the 1983 Orphan Drug Act.

Early detection. Drugs that significantly slow disease progression will have much more positive impact if paired with early detection, which has been a chronic problem with ALS diagnosis. The need for earlier detection will be urgent in this scenario. This will require primary physicians and neurologists to be better trained on ALS and made aware of the need for speed in diagnosis.   If delayed diagnosis remains a problem, drugs that slow progression down will have limited impact, as they will simply arrive too late to extend life expectancy as much as they could. They will also miss the opportunity to extend the initial period of symptom onset, when symptoms are more mild and when pALS are most physically functional.

ALS physician caseload. For ALS neurologists, this scenario will, within four to five years, double the number of cases they have to carry. They could cope by reducing the number of annual consultations with pALS from quarterly to bi-annually. But how would that impact data collection, and quality of support to pALS, especially those who may not respond as well to the treatment? Another response could be policies to promote expansion of the number of newly trained neurologists who specialize in ALS.  In the short term, do we have enough ALS specialists in the medical school pipeline to respond to heightened demand?  Recent reports suggest that in the coming decade the US will actually be facing a serious shortage of doctors, so the odds of increasing the number of neurologists specializing in ALS may not be good. The physician caseload in this scenario looks to be a potentially serious bottleneck.

ALS inter-disciplinary clinics, and care and support services. For ALS interdisciplinary care and support work, this scenario will significantly increase demands on their services, doubling the requests for equipment, counseling, respiratory therapy, in-home care-giving, and other support. Rationing existing resources and care support would constitute a major drop in quality of care. Responding fully to new demands would require a major ratcheting up of fund-raising efforts, as well as an expansion of hiring of specialists and expansion of new clinics. The possibilities of harnessing new technologies to reach new pALS with telemedicine and virtual care could partially offset the spike in demand for care and support services, but would be no panacea.

Fundraising. The need to increase fund-raising for a doubling of care and support services will be acute. This will very likely exacerbate tensions between those who want to prioritize funding for research for a cure and those prioritizing fundraising for care services.  Even though care services and research are much more closely interconnected than most of the public realizes, tensions over allocations of funds for research versus care services are real, and will be aggravated by a doubling in need for care services. 

The solution — a successful ratcheting up of fundraising to fully meet all needs — is easier said than done. ALS competes with many other worthy charitable causes, and in the US total charitable giving has been flat despite a long period of economic expansion.  Powerful public awareness campaigns will be required to explain why advances in ALS therapies are actually increasing the need for more funding.

Insurance companies, Medicare, Veteran’s Health Administration. For insurance companies, Medicare, and the Veteran’s Health Administration, the cost of new therapies and the doubling of patient life expectancy will create additional payouts, but given the relatively small number of cases this will not be a significant problem.  The high cost per patient of ALS cases could, however, create pressure for cost control or create access problems.  Some insurance companies could decline to cover expensive new “orphan drugs” if they have doubts about evidence of effectiveness.

Research and clinical trials. If most of the population of pALS is taking drugs that significantly slow down disease progression, how will this effect eligibility criteria for new clinical trials? Will it make it harder to discern positive impact of drugs in clinical trials?  We know that slow progression cases tend to confound clinical trial results. In this scenario pretty much all pALS would become, to varying degrees, slow progression cases. This author is not expert on clinical trial design issues, so cedes the floor to others on this question. But it seems to be a question worth raising now.

The heightened importance for the need for early detection and screening will likely lead to calls for more research to advance that capacity. 

Social Security disability insurance program.  Our longer life expectancy could potentially raise questions as to whether newly diagnosed pALS should continue to receive expedited access to disability benefits, since at least some pALS will have the capacity to continue to work for a longer period of time (depending on their line of work and disease progression).

People with ALS, their caregivers, and their families.  This is by far the biggest issue to consider.

First, the impact of this scenario on pALS, caregivers, and families will clearly be highly variable.

For some pALS in advanced stages of the disease, this scenario may be perceived to bring minimal benefits, as it will only prolong the most difficult, final phase of the disease when quality of life calculations can come into play.  Some might have good reasons to want to extend their lives nonetheless — Stephen Hawking serves as a reminder that pALS who are entirely paralyzed can continue to pursue meaningful and productive lives. But some in late stages might forego treatment completely.

For most pALS and their caregivers, the benefit of living twice as long with the disease will be weighed against the doubling of the period of time of intensive, expensive home care for the pALS. This will put huge additional demands on caregivers, and huge additional expenses on family finances. A therapy that only slows the disease down could inadvertently produce a spike in ALS-related bankruptcies and could push some caregivers to the breaking point.  Here we could expect to see a big split in choices based on household wealth. Those with ample resources will be in a better position to choose to extend their lives than those with modest resources.

The pALS who will stand to gain the most from this scenario are those in early stages of the disease, and those with slow progression. They will potentially be able to live many more years than they expected, and could remain in possession of most of their physical capacities for much longer. Some could live long enough to end up dying of other causes. Importantly, this scenario will also buy them more time to benefit from future medical breakthroughs that could reverse or cure ALS. But the flip side of the coin is the prospect of a very long period of partial or total incapacitation, with all of the difficulties that entails for the patient, the caregivers, and family finances.  Some might opt for a two-track approach — taking the therapy during the period when they have retained most of their physical capacities, but then dropping it once they reach advanced stages of the disease.

Assessing Scenario #1. This scenario produces sizable but mostly manageable challenges for organizations involved in care, support, research on, and financial coverage of ALS. Impact on pALS and their caregivers will be more problematic and variable. The possibility of new therapies that significantly slow but do not halt or reverse ALS will be a positive development for some pALS, but a Faustian bargain for others. PALS and their caregivers and families already face very difficult and emotionally wrenching decisions on whether to use or forego procedures that extend life. This scenario will significantly increase the difficulty of weighing the costs and benefits of using therapies that extend life expectancy.

Scenario 2: Halting of progression

In this scenario, a new drug or combination of drugs effectively stops further disease progression, but does little or nothing to reverse damage already done. Everyone with ALS who gets this treatment must live with symptoms that are present at the time they start the new therapy. ALS is transformed from a fatal to a chronic disease.

What are the implications?

ALS demographics. In this scenario the total number of people living with ALS in the US will steadily increase by about 6,000 per year, less the (initially) small number who die of other causes – a number that will increase as the population of pALS ages.  Moreover, as the American population ages (the total number of Americans over the age of 65 will double between now and 2060) the total number of new ALS cases is expected to rise well above 6,000 per year, increasing the number of people living with ALS even further. It would take a skilled team of demographers, epidemiologists, and statisticians to give us an accurate estimate of the total number of people living with ALS by 2040-2050 in this scenario. But we can all do a quick back-of-the-envelope rough estimate, and reach the conclusion that the number of people with ALS in the US could easily reach the hundreds of thousands.

Early detection. In this scenario early detection becomes an absolute priority. Every month of delayed diagnosis will mean permanent loss of some functionality for a person with early stage ALS, losses that could have been avoidable. Delayed diagnosis will be viewed as malpractice, not as an unfortunate reflection of the slow, “process of elimination” approach to determining if someone has ALS. Ideally, if advances in diagnostic tools are developed, detection will be done before patients even reach the diagnostic threshold, so that treatment can begin before symptoms appear or advance.  Primary care physicians and other medical professionals who are often the first point of contact with people with possible ALS will need to be given much more training and guidance on referrals of possible ALS cases to specialists. Protocols might be needed to give possible but not definitive cases of ALS access to the therapy.   

ALS physician caseload. The ALS caseload will explode in this scenario and will require a complete rethink of the current clinical support system. There is no way that the current system, which sees 20,000-25,000 cases, could shoulder several hundred thousand cases.

This potential crisis could be partially offset by the possibility that the new generation of “stabilized” pALS might not need to see an ALS specialist regularly, or even at all once diagnosed and given access to the new treatment. They could instead be monitored by their family physician and only referred to the ALS neurologist if their condition changed, if they experienced side effects from the therapy, or had other complications.  This switch in the site of care and monitoring could erode or complicate data collection on ALS cases, and could require a rethink of the role of clinics in trials of new therapies.

ALS inter-disciplinary clinics, and care and support services. For ALS interdisciplinary care and support work, this scenario will create dramatic swings in demands for services over time. Initially, demand will be steady, since the number of ALS patients with moderate to advanced stages of the disease, now with much longer life expectancies, will go largely unchanged. Over time, the profile of the total ALS population will shift, with a declining percentage of pALS in moderate to advanced stages of the disease, and a rapidly growing percentage and number of pALS stabilized in early stages of the disease. The type of care required will shift accordingly, with more support to deal with partial loss of limb or voice strength, and far less need for support associated with advanced stages of the disease.   

Fundraising. Fundraising for care services would initially need to be increased, then over time  reduced, as the needs of the pALS population shifts almost entirely toward early onset issues. Fund-raising for screening and early detection services, and training of additional specialists for this task, will become a major new priority. Fundraising for research to reverse and cure the disease will also become a dominant priority.  Whether fundraising becomes easier or harder in this scenario is hard to know, On the one hand, the success enjoyed by new therapies could create momentum for more support for research. On the other hand, it might become more difficult to argue that ALS is a top priority when the disease is transformed from a fatal to a chronic condition.    

Insurance companies, Medicare, Veteran’s Health Administration. For insurance companies, Medicare, and the Veteran’s Health Administration, the high cost of new therapies combined with the exponential increase in people with ALS will constitute a significant expense, though the price of new therapies will presumably drop over time. Since most people with ALS in this scenario will be dealing with stable, early onset symptoms, many more will continue to be able to work, so a disproportionate percentage of the costs will fall on private insurance companies, not Medicare.

Research and clinical trials. If the population of pALS is using therapies that halt disease progression, eligibility criteria for new clinical trials will have to move away from the current practice restricting participants to those with fewer than 24 months since symptom onset.  For some clinical trials, clean samples of pALS might be needed, which will pose a challenge.

Research to advance capacity for early screening and detection will be a high priority.

Social Security disability insurance program.  Once pALS have a drug or drugs that stabilize their condition, and presuming most are diagnosed in time to minimize symptom onset, a much larger percentage of pALS will be able to continue to work, though some may have to find new lines of work depending on their condition. This would likely lead to pressure to eliminate the expedited access to disability benefits for pALS.

People with ALS, their caregivers, and their families.  The impact on pALS and caregivers will vary dramatically by generation and income level. Those with advanced stages of disease progression at the time the new therapy is made available will only benefit if they have a powerful reason to want to continue to live despite severe physical constraints and if they have access to financial resources to cover costs of caregiving. For others with advanced progression, the therapy may not be an attractive option, though some might opt for it anyway in hopes of extending their lives long enough for a new therapy that reverses disease progression.

For pALS in early stages, suffering from only minor to moderate symptoms, this scenario is a life-saver, and almost all will seek access to the therapy.

PALS who are newly diagnosed after the new therapy is made available will constitute a new generation of pALS for whom the disease is chronic, and hopefully – if diagnoses are made early enough – featuring only mild and manageable symptoms such as foot drop or slight speech impediment. The entire profile of pALS will shift over time.

Assessing Scenario #2.   The halting of ALS will lead to an explosion in the total number of people with ALS, place significant new demands on sources of health coverage, require much more capacity for early detection and diagnosis, and create changing demands over time on support and care agencies. For pALS with advanced disease progression, this scenario presents them with very difficult choices. For the rest of the pALS population, the rendering of ALS from a fatal to a chronic condition will be a welcome new lease on life, tempered only by frustrations over whatever physical abilities have been lost to the disease.  Hopes and expectations that new therapies will be developed to reverse disease progression will be high.

Scenario 3. Reversal of progression

This is, of course, a happy scenario to contemplate.  For pALS and their families and friends, a therapy that reverses progression to produce a (more or less) symptom-free form of ALS will be met with tremendous joy and relief. The gradual recovery of strength and physical capacity once lost to the disease will be a tremendous emotional journal for pALS (though it is possible pALS in advanced stages of the disease could suffer some irrecoverable losses in functionality). 

Almost all US-based pALS, regardless of stages of progression or level of income, will benefit equally from the prospect of a therapy that reverses progression.  The exception will be those without insurance, Medicare, or VA coverage, who, faced with exceptionally high costs of the therapy, could face the prospect of bankruptcy and loss of their home before qualifying for Medicaid. This is a potentially solvable problem, if the right legislation were passed or policies developed, and one that will demand close attention as a matter of equity and social justice.  

What would be the wider impact of a therapy that reverses ALS progression?

ALS demographics. The population of people living with ALS in the US as a chronic, largely symptom-free condition will eventually grow to several hundred thousand.

Early detection. Early detection will no longer be as urgent, since symptoms are reversible. Delayed detection will constitute an inconvenience and annoyance for patients dealing with temporary unwanted symptoms.

ALS physician caseload. For ALS neurologists, their work will shift to confirming diagnosis and trouble-shooting cases of complications.

ALS inter-disciplinary clinics, and care and support services. Most ALS interdisciplinary care and support will gradually be rendered unnecessary. Those serving groups with other neurological conditions will simply shift their focus to those patients. The wild-card in this regard is the degree of variability of impact of new therapies. If not all pALS respond positively to the new therapies, care services will still be in demand, though at more modest levels.

Fundraising. Fund-raising might be needed to continue to support some research on the disease – mainly devoted to finding a cure —  but that would be a lower priority compared to other medical research needs. Most of the organizations dedicated to fundraising for ALS will either close down or repurpose their mission, as the March of Dimes did once a polio vaccine was developed.

The wildcard in fundraising for ALS would be the possibility of a major shift in focus from domestic to international programming – an option considered below.  

Insurance companies, Medicare, Veteran’s Health Administration. For insurance companies, Medicare, and the Veteran’s Health Administration, the cost of a new therapy required over the course of a lifetime, for hundreds of thousands of pALS, will be a significant new expense. Over time, presumably, the cost of the drug or therapy will drop.

Research and clinical trials. Research in pursuit of an actual cure for ALS will still be conducted, but most researchers in this scenario will move on to work on other medical research endeavors, as ALS research will likely be viewed as a less urgent priority by the NIH and other funding agencies.  

Social Security disability insurance program. PALS will no longer have any need to go on long term disability, though some might require short term disability if serious symptoms presented themselves and diagnosis was slow.

Assessing Scenario #3.  A cocktail of drugs and therapies that reverses ALS will produce almost all benefits and very manageable costs, mostly related to insurance coverage.  This is a dream outcome, and is much less fraught and complicated than scenarios #1 and #2.  Even those people who lose their source of employment in ALS fundraising, advocacy, and care service organizations and have to move on to tackle other diseases will take great satisfaction that they put themselves out of business by doing what they set out to do — defeating ALS.

But many of the current team of ALS advocates and fundraisers may not have to shift to another line of work; they will simply have to shift to a different target population. ALS is a global scourge, and will require a major global effort to treat populations in the global south who are far from medical support that can diagnose and treat ALS. The energies and expertise of  people working on ALS in the American context could be put to excellent use in an expanded initiative to provide treatment globally.

Global implications

It is worth briefly anticipating the implications of new therapies globally, especially in the global south, where most of the world’s people reside and where most of the cases of ALS exist.

One study (Arthur et al 2015) estimates that 223,000 people have ALS globally at this time, and that that number will increase 69% by 2040 even in the absence of a new therapy.

Three factors will drive this trend. First,  the global population is growing at a rate of just over 1% per year, or 78 million people per year, almost exclusively in the global south. Second, life expectancy has increased dramatically in almost every country in the world, so many more people are now reaching ages when ALS is more common. Third, access to better medical care is improving globally, albeit unevenly, so we can expect that a greater percentage of people with ALS in the global south will soon be diagnosed properly.

The 2015 Arthur et al study observes that, without or without any new therapies, the weight of ALS “will shift away from the developed world towards developing countries.” It concludes that   “[t]here are currently no studies outlining the cost of ALS in developing countries, and while economic costs are most likely lower in these locations, the increase in ALS cases will place an immense burden on their healthcare systems in the coming decades.”

Now, consider what would happen if a new therapy were made available globally that halted or reversed disease progression, and that most pALS who needed the treatment could access it. The global population of people living with a managed or symptom free form of ALS could grow about tenfold, to 3-4 million, by 2040.

But how will pALS in poor countries get access to new ALS therapies? The hurdles are daunting.

First, accurate and speedy diagnosis will be very difficult given the limited number of neurologists in the global south and the general lack of familiarity with rare diseases such as ALS among primary physicians. The wealthy cohort of people in the global south will stand a much better chance of correct diagnosis, but the rest of the population will face the risk of dying of ALS without ever knowing the true cause.

Second, even those who are diagnosed will face costs for therapies far beyond what most can afford and beyond what poor national health care systems can provide.

Third, calls for funding and support for ALS therapies will compete with demands for many other more endemic and more urgent health needs in the global south, and may not be a sufficiently high priority for some governments’ public health strategies.

The potential solution to these bottlenecks could be a much smaller variation of the global health initiative to combat HIV/AIDS. Since 2003, the US Agency for International Development has devoted $80 billion through the PEPFAR program to provide affordable, accessible treatment for people in the global south living with HIV/AIDS. It has saved an estimated 17 million lives in the process, rendering what was a fatal disease into a chronic one.

ALS organizations could play a critical role in international aid to raise awareness about, provide diagnoses for, and provide affordable access to therapies for populations in poorer countries of the world. This would be a hugely challenging assignment, but one which warrants preliminary planning now.

Next Steps

This paper is intended to jumpstart a conversation about how best to anticipate and plan for the impact of different ALS therapy scenarios. It is only a first cut at the topic, and an invitation to start a much wider and more thorough conversation.

Next steps could include:

  • Collection of feedback on the draft, to revise the paper and make it into a more robust white paper on the topic for general use.
  • Development of specific action items culled from the analysis.
  • Proposals to convene stakeholders at workshops, virtual workshops, or in electronic forums, for targeted discussion of the most important action items stakeholders want to address for planning for each scenario.
  • Agreement on a division of labor among the many ALS organizations for who plays the role of convener for which prioritized topics, to maximize inter-agency coordination and eliminate redundant efforts.
  • Consideration of use of surveys as a tool to acquire better information on issue areas we feel are high priority but for which we lack solid data. This would be especially valuable in anticipating likely pALS responses to various options created by new therapies. This too would need to be a coordinated effort by ALS organizations, to avoid redundant surveys and the risk of survey fatigue among pALS and their caregivers.

Comments and suggestions are welcome – this is a living document and the author expects to update it. Send your comments to kemenkhaus@davidson.edu . The analysis presented here is the author’s alone and does not necessarily reflect the positions of any of the ALS organizations with which he is associated. Many thanks to several colleagues who agreed to review draft versions of the paper and who provided very helpful comments.

Fellowship Award!

Some great news to share!

Each year, Davidson College awards one or two faculty members with the Boswell Family Faculty Fellowship. The fellowship provides the recipient with a full year paid sabbatical (normally our paid sabbaticals are for a half year).  

This month I learned I am the recipient of the fellowship for the 2019-2020 academic year.

I can’t express in words how much this means to me, and how much freedom this gives me and my family to plan for the sabbatical year ahead. I plan to complete the book on the crisis in Somalia since 1988 that I have been wanting to do for years. And I’m hoping to do more research, writing, and advocacy on ALS matters as well. Karin and I will wait until spring to assess the state of my health before deciding whether to relocate to Kenya for part of the year, or stay back home.   We now have a wealth of options, which is a great luxury.

I am very grateful to the Boswell family for their generous support of Davidson faculty development, and to the College for its vote of confidence.

I Have ALS. Now What? A Guide for the Newly Diagnosed

By Ken Menkhaus, for the Joe Martin ALS Foundation

July 2019

This set of short essays was composed fifteen months after I was diagnosed with ALS in March 2018, and is written for others who have just learned they have been diagnosed with ALS. It tries to anticipate the most common issues faced by persons with ALS (pALS) in the first weeks and months following the diagnosis, and point you to helpful sources of support. I will update this periodically (note: original set of essays from July 2019 appear on the Joe Martin ALS Foundation website; updated versions will appear on this blog, Ken’s Caucus).  

Preface

First, I’m very, very sorry that you have ALS.

If you have discovered this essay online and are reading it, you are – like all of us who first got the diagnosis – scrambling to try to figure out what to do now.

You will quickly become frustrated by the fact that there are no set answers and no script to follow. This is because the symptoms and progression of the disease vary so dramatically from one person to the next, and because pALs find themselves in very different situations regarding health care access, caregiver and community support, family, and financial assets.  As a result, each of us ends up blazing our own trail – still benefiting from one another’s common experiences and collective support, to be sure, but having to customize decisions based on our own unique set of circumstances.

Much of what I share below is meant to flag common issues we all need to address, not to provide specific answers or advice. And some of the advice I do share, based on my own experience, will have more applicability to some pALS than to others. These essays are written with US-based pALS in mind – the support landscape for pALS in other countries is quite different.

There are other very good sources out there for people newly diagnosed with ALS, and I encourage you to read all of them. These are a good start: ALS Association Newly diagnosed guidanceYour ALS Guide  and I AM ALS newly diagnosed advice.

Dealing with the initial shock

You may have gone to the neurologist already prepared to hear that you have ALS. Or you may have gone in presuming that your problem was due to some other condition and were taken completely by surprise by the diagnosis. 

Regardless, all of us who get the diagnosis are initially in a state of shock.   You may be in shock now as you read this. I felt like a zombie for the first several days, like I was walking in the world but was no longer of it. It took me a few weeks before the sense of shock began to fade.

Try to avoid making major decisions immediately after the diagnosis that later will feel like rash actions. Give yourself time to let this new reality sink in. You’ll know when your mind starts to clear, and at that point you can begin to address the many, many decisions ALS forces upon us. 

And know that everyone reacts differently to the news. There is no right or wrong way to react.

Informing yourself about ALS and living with ALS

Like the rest of the general public, most of us who get the diagnosis don’t initially know a lot about the disease. You will have a million questions, and you’ll want to learn as much as you can about ALS and what steps you need to take.  Your most likely first step will be a google search of ALS. 

Be forewarned.

This can be a frustrating and somewhat traumatic experience. If you’ve already done this, you know. You can stay up late into the night scrolling through one site and forum after another, and can work yourself into quite a state of anxiety. I know I did.

First, there is the search for information about the disease itself. There are dozens of reputable websites that provide excellent basic introductions to the ALS. Most say pretty much the same things. See for example: 

Most of these explanations are meant to be pretty basic. If you’re looking for more detail, you’ll need to move on.

One option is to explore the actual research findings in scientific journals.  Here you will face two problems. First, many to most of the journals are subscription only, available to most of us only if we happen to work at a university which subscribes. You can read an abstract about the research but won’t be able to read the entire study. Second, even if you do get access, unless you are trained in the field and in statistics you will not be able to interpret the research. 

The best alternative is to follow sites devoted to providing summaries of research findings that are accessible to laypeople. One of the best is ALS News Today. Another is the NEALS monthly webinars, which treat a range of medical and nonmedical topics related to ALS in an accessible fashion.

You will also have many questions about how people live with ALS and prepare for stages of the disease – everything from dealing with social security disability insurance to handicap accessing your home to voice banks to living wills and beyond. This part of your quest for information is going to feel overwhelming at times.  

For one thing, there are so many issues to consider and decisions you and your family will have to make. It helps to make a list and prioritize so you feel at least a semblance of control. In my experience, the best source with a clear inventory of issues to prepare to address and advice on how to handle them is the ALSA’s Living with ALS Resource Guides. There are lots of other helpful guides for the newly diagnosed – see the list of sites I provided at the beginning of this essay.  But you should also rely on your  multi-disciplinary team at the ALS clinic you are going to or will go to for consultations. That team will help answer a wide range of both medical and nonmedical questions you may have. If at all possible, go to these consultations with a family member or friend who can help record and remember the conversation. It will be more information than you can take in, especially if you’re stressed (which we all are at these consultations). In some locations in the US, there exist local or regional ALS support groups like the Joe Martin ALS Foundation in Charlotte (for which this essay was originally written), which are a rich source of guidance as well.

Another, more emotionally fraught source of information are ALS on-line forums, personal blogs of pALS, and Youtube videos.  Here you will get direct, unfiltered views, advice, reflections, and complaints from fellow pALS and their caregivers (CALS).  On the positive side, you’ll learn a lot from fellow pALS, most of whom participate in these sites with great courage, compassion, and a sense of humor. A few are even so cheerful they can be annoying on a bad day.  But some entries are understandably raw with emotion – including anger, despair, and exhaustion. The difficulties they discuss can be horrifying to a newly diagnosed person with ALS, and the entries from exhausted caregivers are very painful to read.  I recommend you start with a forum that has a moderator, and stop reading if entries are too upsetting. A good place to start is the ALS Forums.  PALS on this site are very supportive of new members with questions they seek answers for.

If you explore personal blogs and Youtube videos produced by people with pALS you will encounter a huge range of content – from inspirational to helpful to angry to very emotionally wrenching. Keep in mind that blogs are written over time and so many are started by persons with ALS whose entries, over time, chronicle their declining health and impending death.  Some are inactive blogs of the now-deceased.   I have benefited from these sites, but on several instances was hit very hard by the stories and videos I read and saw. Prepare yourself before diving into these waters.

A final point about using the internet to inform yourself – beware of the unscrupulous groups trying to exploit our desperation by selling “snake oil.” Don’t be suckered in by people claiming that they were “cured” with some homegrown remedy that you can purchase from them.

Sharing the news – who and when 

Your diagnosis will force you to make some choices relatively early on, whether you are ready or not. “Not to choose is to choose” as the saying goes. One of the first issues you will confront is when to share the news with whom. This is not easy, and a number of factors should be taken into account.

Closest family members: it seems obvious that your closest family members should be told right away, since they will be dramatically impacted by the news and have a right to be part of your grieving, adjusting, and initial planning.  But there are variations on this theme. If you have children, you may want to wait until school is out or a vacation starts so you are minimizing possible disruption in their schoolwork (the ALSA has a great set of  guides for sharing the news with children, teens, and young adult children, please take advantage of these materials). This is a case by case decision that each of us has to make on our own.

I had an unusual situation that led me to hold off on sharing the news with some family members. When I got the diagnosis, my wife’s parents were visiting us from Sweden, and I did not to want to spoil that family reunion with the news I had ALS. So I kept it to myself for two weeks until my in-laws returned home, at which point I broke the news to my spouse. I can tell you that those were two of the hardest weeks of my life, and I would not recommend anyone doing that if you can avoid it.

As for telling our three children, my wife and I opted to break the news slowly, by first telling them that I was diagnosed with a degenerative nerve condition and that I would probably need to use a wheelchair in the future, that the doctors aren’t sure what it is, and that it might be something bad like ALS, but we don’t yet know.  I held off until  8 months later, after Christmas, to tell them the bad news. I was able to do this in part because I have (so far) slow progression, and so was able to “pass” as just having a limp during that year. I don’t regret doing this, as it bought me the better part of a year to travel and spend time with the family without that cloud hanging over their heads. But for most PALS, whose progression is faster, they don’t have the luxury of time to put off sharing the news for very long.

Be prepared for a wide range of reactions from family and friends as you share the diagnosis. Everyone has their own way of absorbing this kind of bad news. Most will be amazingly supportive. Some feel compelled to give unwanted advice – just take it politely and nod, they mean well. Others may immediately start to tell you about an illness they or their relative has or had, as though you’ve just entered the “Who Has the Worst Disease” Olympics. Cut them some slack too; as one observer put it, “some people give you their heartbreak like a gift.”

One piece of advice I would share is this: do not render judgement on friends and family members based on their response to the news. There is a bromide that circulates among some with terminal illnesses that “you find out who your real friends are.” I’m not convinced that is true. I have had a number of friends  who did not initially respond to my news, who later confided that they were too upset or simply unwilling to accept it. People are complicated, especially in response to bad news like this, and you will do yourself and them a huge favor by showing them some grace if their reaction and support initially is not what you expected.

Sharing the news about your condition publicly – and make no mistake, once you start to share the diagnosis beyond immediate friends and family, word spreads fast – is a decision shaped by how private a person you are, how fast you perceive your progression to be,  and how visible your condition is. Because I have slow progression, I opted to wait for over a year from my diagnosis before sharing the news widely, mainly because I didn’t want to be a distraction at work and in the classroom (I teach for a living). If you need to mobilize help from your circle of friends and family, letting people know sooner is obviously better.

When you do share the news widely, do it during a period when you are able to cope with the flood of messages of condolences and support. Depending on how many people you tell, this can be a bit overwhelming for a few days.

I have found little advice on the internet about how to share the news with supervisors and work colleagues.  Most websites seem to assume, probably correctly, that most persons diagnosed with ALS (if not already retired) leave work immediately and go on disability. The fact that the diagnosis usually takes so long means many to most persons receiving the diagnosis are in fact getting to the point where they can longer work by the time they get the diagnosis.  For those of us who have jobs that allow us to continue working for a time despite the disease, and who have slow progression, deciding who to tell when at work is a choice that you need to consider carefully.  On the one hand, making supervisors and your HR office aware of your progressive disability will allow them to work with you to create a work environment that accommodates you, which they are legally bound to do. It also should shield you from being laid off for reasons related to your condition, as long as you are able to fulfill your work function.

Not divulging your condition in your workplace or your professional circles can make sense if you fear that clients and co-workers will cease investing in working relationships with you. I do some consulting work internationally and was worried that the phone would stop ringing once word got out of my condition, and so I made sure to reassure those work colleagues I was still fully functional to meet their needs.

How to share the news

How you deliver the news to family, friends, and colleagues is more important than you may think. I urge you to give this careful thought, because the way you frame the news will shape how people respond to it and interact to you.

There is no right way or wrong way to do this. You may choose to convey sadness, anger, hope, faith, or a thousand other themes, and on this count my only advice is to try to be true to yourself (easier said than done with so many conflicting emotions running through us). But whatever tone you choose to strike, I would urge you to put yourself in the shoes of those reading or hearing the news, and try to help guide them in terms of how you would or would not like them to respond. People hearing about a terminal diagnosis react in many different ways, and you’ll be on the receiving end of all of them. But in almost every case their common fear is saying the wrong thing to you. Some will be so afraid of this they will actively avoid interacting with you, even though they care deeply about you. If you can help them out in your communication by telling them what kind of response is or is not welcome, they will be grateful and much less anxious about reaching out to you.

I took the approach of directly asking everyone not to walk on eggshells around me or worry about saying the wrong thing – that I have a thick skin and a sense of humor and that I am good with both them asking me how I’m doing or not saying a thing, whatever works for them. Lots of friends wrote back thanking me for freeing them up with that advice, which made me think that it’s worth sharing with you all. The letter I sent out sharing my diagnosis is posted on my blog and you are welcome to borrow from whatever part of it may be useful to you. You may prefer to tell people in person, but I found that most preferred to absorb the news privately by being told via email or a letter.

Planning, the initial phase

Several of the websites I suggested above do a very good job helping us plan out next steps, and I won’t repeat what they have to say, except to add that you should try to organize all these tasks by high priority vs lower priority and by ease of completion. Otherwise you and your future caregivers will be overwhelmed trying to trouble shoot too many things at once. Among the easier things to complete (well, easier in terms of time spent, not easier psychologically) that will make you feel better about shrinking your “to do” list include:

  • Writing or reviewing your will
  • Creating a living will/advance directives
  • Starting the paperwork to gain access to medicare and social security disability insurance
  • Reviewing family finances and making a financial plan (or multiple plans, depending on circumstances) accounting for costs of equipment and homecare, and loss of income once on disability
  • Recording your voice on one of several voice bank programs.
  • Signing up on the national ALS registry
  • Working with loved ones to create a wish list of things you want to do together while you are still relatively mobile. This could be trips you’ve always wanted to take, or more time visiting with friends and family, or any of the other things we all wish we would be able to do more of (my family and I took the trip of a lifetime to show the kids where I work in East Africa, and though it was costly I have no regrets at all about it. It was an amazing collection of memories).
  • Doing preliminary exploration of costs of equipment and handicap-accessing your home (or, exploring moving out of your home and into a handicap access residence)

Depending on the speed of the progression of your ALS, you may or may not have some time to wait on big decisions like actually handicap-accessing your home and purchasing a power wheelchair. Again, the ALS support group websites are very good at giving guidance on these big and costly decisions. My sense, based on reading of hundreds of blogs and forum exchanges, and conversations with fellow PALS, is that the most common mistake is waiting too long.

Taking stock of your resources and support

You are going to need a lot of help and support in the months and years ahead. The hard and unfair reality is that pALS enter into the fight with ALS with wildly different resources. Some of us are resource rich, some of us are resource poor. We will need to draw on whatever assets we have at our disposal, so take stock now to help develop a strategy. These resources include:

  • Financial. Review your savings and fixed assets and try to calculate how much you and your household can afford when you go on disability and begin facing the increasingly large costs of care and technological support that are not covered by insurance. The main ALS websites out there have information on the costs of care. They are astounding figures. The cost of complete home care, which we will need in late stages of the disease, is estimated at $200,000-$250,000 per year, which will bankrupt most families within a year or two. But know that there are many sources of support to help offset these expenses (discussed below).
  • Family. Discuss openly with your close and extended family about what they can and cannot do. Some will be able to promise time as a caregiver, but not financial support; others may be able to write checks but won’t be in a position to help as caregivers.
  • Your social capital (extended network of friends). Most of us don’t have the level of savings needed to cover the huge caregiving costs of the disease in late stages. But many of us are rich in social capital – our network of friends, neighbors, co-workers, and fellow congregants if a member of a faith group. Take stock of this important asset, as it will eventually be a potentially powerful source of support. Ten years ago, in my town, a woman with a husband and two young children was diagnosed with ALS, and members of the community rallied to organize to raise funds for the family and help look after the children.
  • ALS Support Groups. The ALS Association local chapters, and other ALS support groups, are expert at providing a variety of assistance for PALS and their caregivers. Make full and early use of these support groups. 

The most important thing to keep in mind is, regardless of your circumstances,  you are not alone. Make full use of any and all help you can get. Some of us are proud and hate to ask for help. This is not the time for that mindset.

Dealing with your own thoughts and emotions

No matter how strong you think you are, you are human, and you will need time to cope with the diagnosis even after the initial shock has worn off.   The quiet of late evenings, and the first minutes when your head hits the pillow at night, when you are not preoccupied with day-to-day issues, can be a time for useful reflection or a time when your mind wanders to dark places.

Depression is a common symptom for persons with ALS, for obvious reasons. Get counseling if you need it.

No matter how centered you are, ALS-induced anxiety will be a periodic and chronic part of your life.  This can come from worries about dying; about health care costs and family finances; about the impact of your condition on your children and spouse; and about signs of progression of the disease.

Since I’m not a psychologist I’ll stay far away from offering advice on this subject, except to say that occasional bouts of anxiety are to be expected, and that it’s important not to overreact to good days and bad days. It’s easy to panic at every indication of a loss of muscle strength, but often it’s just that you’ve overworked your muscles too much and they are tired or you’ve sat all day and they are extra stiff.

If you need help with anxiety or depression, don’t “self-medicate” with excessive alcohol consumption or drugs, that could expose you to much greater risks of falling and injuring yourself. Your physician, professional counselors, and psychologists can help you manage anxiety and depression.

Depending on how advanced your ALS condition is, you may already have noticed that you have less energy than before. If it hasn’t happened to you yet, it will, and you need to be mindful of how much you can and should realistically ask of your body each day. Over-tiredness can compound anxiety.

One daily mental and emotional aspect of the disease that will surprise you is the fact that you will suddenly hear and see constant references to ALS, other neurological diseases, disability, and death in conversations and in the media.  I was shocked at how prevalent these and other reminders of my condition pop up all the time. In the past, I simply was not sensitized to these references and so they made little impact. Now they do.

You will also be surprised at how many of your friends’ and colleagues’ lives have been touched by another ALS case. The disease is considered rare, but if one in every 300 people die of it, that means almost every person knows of at least one case. To put it in perspective, that’s roughly one to two members of every high school graduating class.

Another emotionally challenging aspect of day to day life with ALS is dealing with the fact that so many of our conversations are about the future – planning vacations, career changes, kids’ school changes, retirement, you name it. I never realized how future-focused my conversations with others were until I suddenly was told I no longer had one. I came across a fine essay on how to talk with others when diagnosed with a potentially terminal illness. The author penned this memorable line (I paraphrase slightly), “the future is a language I no longer speak.” I think of that line often, even as I try to stay engaged in conversations with friends who are musing on the best retirement location. I am slightly envious of their casual use of the future tense, but happy for them that they can do it.

Always remember the impact on your closest family members

As you are no doubt realizing, your closest family members (some combination of spouse, children, siblings, if you have them) are likely to shoulder the heaviest caregiving and financial burdens. They may be at least as stressed as you at the thought of losing you, and at trying to work out what to do next.  Do what you can to help them through these first months of the diagnosis, and remind them of the support groups for caregivers.

Start keeping a medical journal, and a personal journal too if you wish

Record changes or perceived changes in your muscle strength, pain levels, and general health.  Monitor how you are doing with whatever exercises and weight lifting you can do. This is more useful than you may think. Our memories can play tricks on us when it comes to accurate recollection of when you first felt foot drop, how many minutes you could go on the bike machine, etc. Your doctor will appreciate getting these close observations from you too.

Dealing with your first quarterly consultation at the ALS clinic

Not all pALS have easy access to an ALS clinic, and so you may not be scheduled to go in for quarterly consultations with an ALS specialist (but you should try to arrange this, even if living in a remote rural area – your ALS support groups may be able to help you get to these meetings). But for those of you who are scheduled to go in for your first and second quarterly visits, know that these are by their very nature anxiety-producing affairs, which is why I am always amused that they take our blood pressure right at the outset of the consultation (mine turned out to be high. Who knew?). You’ll worry about test results that show decline in one or another indicator; you’ll share a visiting room with pALS in more advanced stages of the disease, which can be unsettling at first; and you’ll be engaged in talking about a range of topics that force you to confront issues you may have wanted to ignore. It’s all for the best, and the teams at the ALS centers are amazing people who are there to help – but just know that if you walk through the door with a sense of dread, you’re not alone.

Support Group Meetings

ALS support groups hold monthly meetings for PALS and their caregivers. It is a chance to exchange ideas and advice on all kinds of ALS related matters, and to build relations with another layer of local support. If you are up to it, consider attending one of these. With caregivers.

Coming to grips with the likely but unknowable trajectory of your ALS

This is a tricky but important subject, and one that you will be preoccupied with over the next six months and beyond.

As you may know if you’ve done some basic research on the disease, life expectancy after diagnosis varies a great deal among pALS. The median life expectancy is 3 years. Of those who live longer than 3 years, 20% live five years or longer. Ten percent of all ALS patients are true “slow progression” cases who live ten years or longer. Some PALS pass away within a year of diagnosis. The sister of a friend of mine died only two months after the diagnosis; I know of others living 15 plus years since they were diagnosed. It’s very unpredictable. And that makes planning for the time we have left complicated.

The “typical” rate of progression, as measured by the ALS Functional Rating Scale, which you will get to know, is a loss of one point per month on a scale of 0 to 48, which assumes a steady deterioration. But that is not how ALS always progresses.  PALS can stay on a plateau for months, or even years, and then experience a significant progression of the disease. Some pALS report an early fast progression followed by a lengthy period of stasis.  Despite this maddening variability, the general, very imperfect rule of thumb is that slow progression at outset is the best indicator of continued slow progression.  Much depends on where your symptoms first appear (limb onset is more closely associated with slower progression than bulbar onset).

Over the coming months, you will find yourself monitoring your condition closely, maybe even a bit neurotically, to try to determine if the disease is progressing at a typical or a slower rate. In six months, you may have a somewhat better sense of your trajectory.

Why does this matter? In the short term it may help you plan and prioritize, though it is important that you not delay important decisions just because you feel (or are told) that you are a slow progression case. For pALS with typical progression, the main preoccupation will be pretty immediate issues – how to get all essential paperwork in order,  handicap access the house, and secure support technology like power wheelchairs. For those with slower progression, concerns are a bit different. I recommend to those of you who appear to have slower progression to create multiple scenarios, based on best case, middle case, worst case progression, so that you have a plan you can adopt as circumstances unfold.

In the longer run, the speed at which the disease is progressing in each of us is now part of a simultaneously hopeful and terrifying calculation about possible survivability – the topic of the next section of this set of essays.

Surviving ALS? The prospects of new treatments

You have been diagnosed with ALS at a singular moment in the history of the disease. In the past, a diagnosis of ALS was followed by advice to “get your things in order” because the disease is 100% fatal. Today, we are part of a unique generation of pALS who have a chance – maybe a pretty good chance, maybe only a “puncher’s chance” —  of surviving the disease. Thanks in part to the big funding push for ALS research that the 2014 Ice Bucket Challenge made possible, a number of potential therapies have now made their way through the (unavoidably slow) research pipeline to clinical trials. Medical scientists working on ALS are now voicing optimism that a therapy will be developed in the next five years that will either dramatically slow, halt, or even reverse the disease.  

To use an over-worn cliché in the ALS community, “we’re in a race against time.”

If in fact we are getting closer to discovery of a therapy that will render ALS a chronic and not fatal disease – and it is impossible to know when this will occur – those of us with ALS today are either going to be the last cohort of PALS to die of the disease, or the first to survive it. I know which one I prefer.

The closest approximation to our situation is the discovery of a cocktail of drugs in 1995 that effectively treated HIV/AIDs. HIV/AIDs patients faced 100% mortality rates up to 1995; thereafter mortality rates plummeted, and today people with HIV/AIDs are able to live with the disease as a chronic condition.

Two factors, both outside our control, will determine if we will be able to benefit from effective therapies in time. First is the length of time it will take for these therapies to be discovered and approved, and the second is the speed of progression of the disease each of us is dealing with. One of the reasons many of us have joined ALS support and advocacy groups is to lobby hard for a big push by the National Institutes of Health (NIH) to increase funding for ALS research at this critical moment. Tens of thousands of lives potentially hang in the balance.

This is not meant to build up false hopes on our part, but it is to say we have a reasonable hope.  I have found it psychologically very helpful to keep telling myself that I will be part of the first generation of pALS who survive this disease.

Participating in Clinical Trials, ALS Surveys, National Registry

As a newly diagnosed person with ALS, you will be asked if you are willing to participate in clinical trials of drugs and other therapies that might slow or stop ALS. You will also be asked if you are willing to participate in a number of other studies on ALS (some may ask you for a saliva sample, for instance). And you will be asked if you are willing to fill out surveys, and submit your information to an ALS national registry.  

Please consider participating! We pALS are not just patients, we are essential, active partners with medical researchers in a quest to understand the causes of ALS and find a cure. Researchers cannot find a cure without our participation. The survey research and national registry are essential to helping researchers look for combinations of environmental and other factors that might offer clues as to what triggers ALS. Please fill them out. As for clinical trials, newly diagnosed pALS are essential participants – most trials will not accept pALS who have had symptoms for more than 24 months, so your role in trials of new therapies will be limited to the first year or so (depending on how long it took for your diagnosis) after your diagnosis.

Participating in a clinical trial is a sacrifice we pALS make for one another. It may involve frequent travel to clinics, or scheduling that is inconvenient, and you may end up getting a placebo rather than the actual drug or treatment (there are important scientific reasons for this; be aware there is an ongoing debate about use of placebos for diseases like ALS).  But you may also get the opportunity for early access to a new treatment that slows or stops the disease. And even if the trial does not result in significant results, you have still helped advance our knowledge of the disease and brought us closer to the day when we have a cure.

Be aware that there are more than one registry projects for ALS, so joining one does not guarantee you have joined all. This is an unnecessary confusion that ALS researchers and organizations are trying to better coordinate. The most important one is the National Registry, which you can access here. The others are legitimate and you certainly should register with them as well if you choose.

When you join the National Registry, you have the option of  receiving notices about research studies related to ALS, which will come to you by email.  This is a convenient way to monitor at least some clinical trials. A more comprehensive listing is apparently available at the ALS Therapy Development Institute website’s listing of clinical trials.  Or you can follow all active ALS research projects and see which are recruiting volunteers for trials at this National Institutes of Health (NIH) clinical trials website.

Activism on ALS?

Many pALS and their caregivers are too preoccupied with responding to all of the changes ALS forces on them to devote much energy to lobbying the government for legislation related to ALS or fundraising for research on a cure for ALS. That is totally understandable. But if you and your friends and family feel energized to try to help with fundraising for ALS research or get critical laws passed in support of ALS medical coverage, support, and research, welcome! You will find yourself in good company. Thousands of PALS are trying to make a difference in the time we have.

One warning: when you search the internet and social media to see who is doing what on ALS advocacy, you will discover that the ALS community is very fractured, with a few flagship organizations (like the ALS Association and the Muscular Dystrophy Association) and many, many smaller fund-raising, support, and advocacy groups.  For the most part, these groups work toward a common goal, but there is a distressing amount of badmouthing on social media, lack of coordination, and redundancy of effort. One exasperated PALS concluded that “ALS is the most fractious fundraising group in the country.” You are likely to feel just as frustrated as he did once you survey the ALS landscape.

Many of us are quietly working to address this problem. For now, don’t be put off by the back-biting, and don’t let the angriest voices on social media sway you. The spats in social media only end up hurting all of us with ALS, by eroding public confidence in all of our organizations. What pALS need is unity of purpose and a collective commitment to a “do no harm” principle. Meanwhile, please explore ways to join our fundraising, awareness-raising, and advocacy efforts if you can.

Finding a purpose in your new reality

You’ll likely have plenty of conversations with yourself about this in the first months after the diagnosis. Do you refocus time and energy on doing all the things on your “bucket list” while you can? Do you devote more time to being with family and friends? Do you jump into your work (if your work has real meaning and value to you and others)? Do you engage in advocacy or volunteerism? Do you use your response to the illness to try to set a good example for your children and others, and if so how? Do you deepen or rediscover your spirituality or connection with God? Do you try to do a bit of all of the above?

PALS forums and blogs are rich with discussion on this topic. Whether we have only a little time  or seven years or (if treatments are developed in time) a full lifetime left to live, the diagnosis compels each of us to think hard about how we want to spend the rest of our lives. For as much as I hate having this diagnosis, I have to concede that it has had a clarifying effect on my sense of purpose in life. I’m spending more time with family and friends, and replacing some of the more pointless work I’ve shouldered with advocacy work on ALS. It has also made me a better human being — I am a more empathetic person than before.   I hope you will find this is true for you.  We don’t control the disease, but we can control how we respond to it and live with it. 

If you remember nothing else from this essay, I hope you retain these two messages:

You are not alone.

There is hope for a cure, so don’t stop fighting to be part of the first generation of pALS who survive this disease.

I wish you well.