Yes, still playing golf with ALS

Today I  posted on facebook (which I don’t do often) that I spent my 60th birthday playing golf on a beautiful fall day. I got a lot of nice birthday greetings in response, a couple of which marveled that I was still able to play golf despite having ALS now for four years.  The confusion is understandable – our messaging about ALS is that median life expectancy is three years. If, statistically, I shouldn’t even be alive, how am I well enough to play golf?

The answer is that I appear to have a “slow progression” version of ALS. No one knows why, but a fraction of people with ALS (about 10%) live 10 or even 20 years. The fact that my ALS symptoms are still confined to my legs, and my leg strength has not really changed much in 18 months, is a clear indication I have slow progression. That is no guarantee it will continue that way – my condition could change for the worse at any time. But for now it has allowed me to proceed with my life with only minor adjustments. I am still working full time; I can walk a mile or more at a time, slowly,  using only an ankle brace (though for long walks I use a roller just to be safe); and I can play golf as well as I could before (which is to say, still not very good). The AFO (foot ankle brace) is a huge help in keeping my weak left foot and ankle stable while I hit and walk   In some ways golf is the perfect sport for me now, as it involves walking a lot, but for short distances, and then sitting in the golf cart, which gives my legs a quick rest.  

Health Update September 2020

I had my first clinical consultation since January (delays due to COVID-19 disruptions) and the results were excellent. No notable disease progression, and the symptoms remain confined to my feet and legs. The docs were very pleased and intrigued by the very slow progression. I can still walk a mile using a foot ankle brace and either a cane or walker, and I can still play a round of golf. I do tire easily, especially if I have to stand for a long time. But overall the report is very positive. Thanks to all of you who have asked!

Fundraising season for ALS!

Dear Friends and Family,

Because of COVID restrictions, this fall the NC Chapter of the ALS Association is having to hold a “virtual” Walk to Defeat ALS. Karin, the kids, and I are again sponsoring a fundraising team, “Kens Caucus,” and are reaching out to ask for help, If your family financial situation in this difficult time permits, we would welcome a donation, large or small! And please spread the word to your contacts too.

The link to the site where you can contribute is here:

“Ken’s Caucus” — Ken’s fundraising page: http://web.alsa.org/goto/kmenkhaus

Thanks so much!

Who should our patient advocacy organizations represent?

The answer to this question is, at first glance, blindingly obvious. The patients, of course.

But which patients? The ones who have the condition or disease and know it? Or the many many more who don’t have it yet but will?  

There are an estimated 25,000 people in the United States who  are currently living with ALS. I’m one of them.

There are an estimated 650,000 or more Americans alive today who don’t have ALS yet but who  will get the devastating diagnosis. Maybe in one year, maybe in five, maybe in 40.

Do our US-based ALS organizations have a responsibility to protect and advance the interests of the 650,000 too, or only those of us with the disease now?

It is a fundamental question, laced with moral and policy implications. Each of us volunteering or working in the ALS advocacy, research, and care service space has to answer that question for ourselves as well as demand an answer from our organizations.

If your answer is that our only task is to serve those with ALS now, then policies designed to advance short-term needs and goals should prevail.  

If your answer is that we have an obligation both to those of us diagnosed with ALS and the 650,000 Americans who will get the disease, then the calculus changes. Then the difficult challenge is to balance serving the interests of those with ALS now and the interests of the “next gen” of people with ALS. That balance can and must be achieved, but it requires us to be mindful of the many voices we can’t yet hear, all 650,000 of them.

We have to do better than this

What happened this month in the ALS advocacy space must never happen again. It’s up to those of us living with ALS and our supporters to insist that our ALS organizations do better.

What went wrong? Two of our most active ALS advocacy organizations, the ALS Association and IAMALS, were only partially in sync on two bills introduced in Congress designed to improve early access to experimental therapies.  One bill, S3872 (“Promising Pathway Act”), was enthusiastically endorsed by both organizations. But on a second bill, HR7071 (“Accelerating Access to Critical Therapies for ALS Act”), they were not in full agreement. IAMALS fully endorsed HR 7071; the ALS Association expressed reservations about the bill in its current form while expressing hope that  discussions can be held with the co-sponsors, IAMALS and other stakeholders to address those concerns and strengthen the bill. 

There is no reason that these mixed messages needed to happen after the bill was introduced.  This was avoidable, and should have been handled long before the bill saw the light of day. What was missing was communication, coordination, and trust, exacerbated by misperceptions.  I have heard enough to believe that there is plenty of blame to go around and both organizations could have done better.  I am uninterested in a blame game. What I am interested in is seeing that we do better moving forward.

Doing better means routinizing communication and coordination on public policy ideas, initiatives, and draft legislation; creating publicly accessible  forums for debates and discussion about possible advocacy ideas, so proposals get a full airing and a wide range of patient voices can weigh in on their merits or shortcomings; and building trust, without which progress on other fronts is impossible.

The good news is that in my conversations with both leaders and members of these two organizations (including many of us who are members of both!) this desire for improved coordination is  widely and deeply shared. And I strongly suspect that leaders in the many other ALS care services, research, and advocacy groups feel the same. Everyone understands that better coordination is essential for success, and is an ethical obligation to the community we serve. I am optimistic that we can make this happen. But we need lots of voices in the ALS community to keep insisting on it. As I settle into the role of Chair of the Association’s Public Policy Committee, I promise you this will be a top priority, and I will hold myself accountable to that goal.

I am also optimistic because, as I have gotten to know both volunteers and staff in our many ALS organizations, I am deeply impressed at how dedicated, smart, and hard-working they all are, and how similar our goals and values are. These are good people. They are having to work in a highly fractious ALS organizational environment, but that is something we can change, if we want to.

All this is not to say that ALS organizations must agree on everything. We have to expect differences in policy preferences, strategies, and principles. In fact, robust exchanges of views on public policy and other matters make for better policies. The key is ensuring that those exchanges occur early and often in the process, and that if organizations end up embracing different positions it can be managed in ways that are respectful and don’t overshadow all the many, many other policies, principles, and positions on which we all agree.

The problem with HR 7071

I write this in my own capacity, I am not speaking on behalf of the ALS Association.

A key provision in HR 7071 (“Accelerating Access to Critical Therapies for ALS Act”) authorizes $450 million over four years to underwrite the costs of early access to experimental therapies. This is not a simple ask.  It raises fundamental questions of principles and priorities for the ALS community, questions that deserve a full and open discussion – one that has not happened yet, but needs to happen now.

The goal of the $450 million request is straightforward. It seeks to solve one of the most frustrating impediments people with ALS face in their attempts to gain early access to therapies still in clinical trial – namely, cost. Pharmaceutical companies running clinical trials on therapies demand very high fees for access to their experimental therapies, if they agree to allow early access at all. Depending on the type of therapy, the expenses for early access can be prohibitively expensive for all but the very wealthiest patients, some reaching as high as $1 million per treatment – and they typically require recurring treatments.  The $450 million request – covering $75 million per year in years one and two, and thereafter $150 million annually in years 3 and 4 – is meant to cover costs of early access for people with ALS.

Several legitimate concerns have been raised about this proposal.

  1. It does not solve the problem it presumably sets out to solve — making early access affordable for all people with ALS. Instead, it would only allow a small fraction of Americans living with ALS to gain early access to experimental therapies. This is the case no matter how one works the math. If the most promising therapy in clinical trial happens to be one which runs close to $1 million per treatment, that would enable only 75 people, out of a US population of 25,000 with ALS, to gain access. That’s 1 in 333 of us – roughly the same odds of being diagnosed with ALS. If the treatments cost less, say $300,000 per round, that would still only cover 250 of us, or 1% of the US population of people with ALS.  Even if the cost of early access fell to as little as $10,000 – a figure some argue might be a reasonable estimate for access to a pill-form therapy in a platform trial —  that would cover 7,500 people with ALS, or less than one-third of us in the US. And such a low-ball estimate only raises another objection – if the cost of early access drops to $10,000 for some experimental therapies, why are we asking taxpayers to cover that when middle class and wealthy households could find a way to cover the costs and poorer households could be assisted by other means? 
  2. It risks creating major rifts within the ALS community. Who will choose what constitutes an eligible “promising therapy” and who decides which people with ALS get covered by the subsidy for early access?  How will the vast majority of people with ALS feel when they learn this massive appropriation of funds provides nothing for them?   Some who follow ALS research closer than I do suggest that the most promising therapies in the pipeline in coming years are those targeting familial ALS. Will that result in most or all of the $450 million covering  the 10% of the ALS population that has familial ALS, and how will the 90% with sporadic ALS react?
  3. Given the very poor track record of ALS phase three clinical trials to date, the odds of early access resulting in meaningful outcomes for the lucky few who get it are slim, making this a very high cost, high risk of failure gambit for a small number of people with ALS.  Of the dozens of ALS therapies that have made it to phase three clinical trials, only two have led to approved therapies, and both have only modest, uneven impact in slowly disease progression. This raises a legitimate cost-benefit question about the fund. Could that $450 million be put to better use than this?
  4. If the ALS community can convince Congress to devote $450 million to our disease, earmarking it for research for a cure would be a far better way to make use of the funding.   The bill stipulates that the $450 million be a separate appropriation from research funding, as a way to deflect criticism that this will divert money away from research. But that is a distinction without a difference. The reality is that interest group pressure that is able to secure this kind of funding for early access could certainly be able to do the same for research for a cure. The authorization for early access reflects a choice we as a community are making – that we prioritize gaining subsidized access to experimental therapies for a few of us, now, over seeking a cure for all. Is that what our community wants?

If our priority to to pursue treatments that stop or reverse ALS as speedily as possible for  all people with ALS, there are good reasons to question whether the $450 million for early access is a good idea, and good reasons to argue instead for lobbying for more money for research for a cure.

You may disagree with parts or all of the analysis I share above, but these are very reasonable questions and concerns, and they are being raised across the ALS community.   Fear of being attacked on social media is preventing some people with ALS from saying this publicly, but make no mistake, they are expressing these concerns in private.

What we need now, and what should have happened well before this bill was introduced, is a full, thoughtful, open discussion and debate in the ALS community about whether this bill in its current form actually reflects our priorities and principles, and if not how we can make calls to modify it to make it stronger and aligned with our principles.

Comparing recent ALS Association and MDA statements on clinical access

Sources:

  1. ALS Association, “Principles for Urgent, Patient Centered ALS Clinical Trials http://www.alsa.org/assets/pdfs/Principles-for-Clinical-Trials-FINAL.pdf (Fall 2018)
  2. Muscular Dystrophy Association, “Redoubling the Urgency for Innovative ALS Clinical Trials” (May 27 2020)  http://cqrcengage.com/mda/file/o0IMFxnubSN/MDA%20ALS%20Clinical%20Trial%20Design%20Statement.pdf

On May 27 2002, the MDA released a statement calling for rapid innovation in the design of ALS clinical trials, and calling on “all stakeholders from across the therapeutic development spectrum to commit to think more creatively on how to address these surmountable challenges, and to heighten the urgency to find solutions.”

The statement was welcomed by the ALS community. 

Some individuals on social media used the MDA statement to criticize the ALS Association for its failure to lead on, or even take a position on, this issue.

In reality, the Association has taken public positions on clinical trials. So the question is really whether the Association’s position on clinical trial innovations is lagging behind the thinking among other patient advocacy groups.

That question prompted this short analysis – I wanted to see where the MDA statement and ALS Association’s positions converged and diverged.  What follows is a very quick comparison of the May 27 2020 MDA statement on clinical trials and the most recent 2018 Statement of Principles from the ALS Association (from its website) on clinical trials.

I broke down each of the specific topics MDA addressed in its message, drew direct quotes from the MDA statement, and then searched the Association’s 2018 Statement of Principles for positions on each of those issues, taking direct quotes from that document as well. In a few instances the issue in question was discussed in more than one part of a document; in that case I included both quotes.  The results are provided in the table below.

 issueMDAALSA
1Date of statementMay 27 2020Fall/summer  2018
2Trial inclusion criteriaWhile trial sponsors may be concerned that expanding the inclusion criteria for the trial may jeopardize findings of efficacy, the use of multiples arms that enroll ALS patients in different stages of progression should be explored      Enrollment criteria should be based on a clear rationale and preclinical data to ensure the correct study population. Simply adopting criteria from previous trials is not acceptable. Many people with ALS want to participate in trials. Every effort should be made to be inclusive while ensuring the trial provides clear answers about treatment efficacy. Adaptive designs and other innovative practices from other disease areas should be used to conduct fast, impactful trials.
3placebosWe remain hopeful that FDA, working with sponsors, will eliminate the use of placebos in ALS clinical trials, but placebos are clearly still being employed in newly announced clinical trials. We recognize that the use of placebos will not disappear overnight, and the lack of established scientific understanding on the heterogenous progression of the disease poses challenges, but we ask that FDA and sponsors redouble their efforts to avoid placebos wherever possible (Calls for): Collaborative development of innovative clinical trial designs that employ historical controls, Bayesian statistical approaches, and cross-over designs with the FDA, the biopharmaceutical industry, and patient advocacy organizations leading the way.     Reduce the size and ratio of placebo groups and consider eliminating them completely when efficacy is not a trial outcome, and/or when additional natural history progression models, crossover studies, or other approaches provide informative comparative data.
4Continued access to therapy post -trial (open label extensions)Sponsors of ALS therapies to commit to considering expanded access and open label extensions at the outset of their clinical studies. Sponsors should meet with patients, their loved ones, and their advocates before clinical trials commence to ensure the approach is supported by the patient community.Access: Trial sponsors need to increase access to experimental treatments. Trial participants originally on placebo or who believe the treatment impacted their disease should have access receive the experimental treatment, through open-label extensions or expanded access options, provided that the additional access does not slow or reduce the impact of the trial itself.
5Funding to cover costs of expanded access programs and open label extensions Innovative financing mechanisms to overcome the financial disincentives associated with including expanded access programs and open label extensions within ALS clinical trials, including ongoing late-stage or Phase III clinical trials.   Congressional consideration of legislative interventions that would lower financial and logistical barriers to employing scientifically sound, but also patient-centric, approaches in ALS clinical studies. People with ALS and caregivers should be consulted on trial design to minimize the risks, discomfort, loss of control, hassles, financial barriers and logistical challenges of trial participation.   IRBs, the FDA, funders and other oversight groups should favorably consider trial features designed to reduce burden on people with ALS and their caregivers.   We appreciate the ethical challenges of spending resources on access to experimental treatments, when many families with ALS are already overwhelmed with the financial burden of ALS (loss of income, home modifications, copays, etc.). Providing financial support for access to experimental treatments could draw resources away from the broader community to the handful of people willing and able to take experimental treatment outside of a clinical trial. Additionally, there is the potential that trial sponsors may profit from experimental treatments by exploiting people with ALS. The Association will continue to explore this issue, while honoring the rights and wishes of people with ALS.
6Biomarker developmentGreater effort and urgency behind ALS biomarker development that can hopefully inform surrogate endpoint development (clinical trial endpoints that likely predict effective clinical outcomes) and eventual qualification to facilitate use of FDA’s accelerated approval pathway.Experimental treatments need testing to determine their effectiveness and safety using the fewest possible participants, shortest length of follow-up, and least burden on participants. Approaches include: Developing and using valid surrogate endpoints and biomarkers when possible to measure impacts quickly and precisely.
7Expanded/early accessFDA consideration of the utilization of innovative approaches towards expanded access, such as the Oncology Center of Excellence Project Facilitate, in progressive neurological diseases without satisfactory therapeutic alternatives, including ALS. –Trial sponsors should strongly consider companion studies where patients ineligible for clinical trials assessing efficacy can still participate to provide safety and tolerability information.    The entire ALS community should have access to experimental therapies under clinical supervision, provided that the additional access does not slow or reduce the impact of the trial itself.

Assessment: For the most part, the 2020 MDA statement aligns very closely with the Association 2018 Statement of Principles.

In addition, both documents use qualifying language like “should consider,” “should be explored,” “when possible” or “provided that” to express preferences without locking themselves into unconditional positions on complex issues. This diplomatic language has the virtue of allowing readers with opposing views to feel that their position was supported, without actually committing to that position in full.  

The one real divergence is on the question of funding to cover costs of expanded access programs and open label extensions  (item #5). The new MDA position embraces the creation of a government fund to cover costs associated with extended access to therapies still in clinical trials (though the MDA does soften this position a bit by asking legislators to “consider” this option). By contrast, Association takes a more cautionary tone, arguing that funds diverted for experimental therapy for a few could come at the cost of research on other promising therapies. 

This latter issue is especially relevant now, as legislation has been introduced in the House (“Accelerating Access to Critical Therapies for ALS Act”) that would, if passed, establish a $75 million fund to cover fees charged by pharmaceutical companies to people seeking early access to therapies still in clinical trial. The efficacy, ethics, and other aspects of this kind of government funding of expanded access to therapies still in clinical trial are complex and deserve a careful, thoughtful discussion and debate in the ALS community.

(NOTE: There are currently two bills pending in Congress on expanded/early access to ALS therapies. The House bill described above includes the $75 million fund proposal. The Senate bill, proposed by Senator Braun, focuses on creating a new pathway for expedited access to therapies still in clinical trial, does not include a government funding mechanism, but apparently creates other incentives for therapy developers to participate. More on all this soon; stay tuned!).

Sabbatical, Day 1

This evening, I completed the last grading of papers and exams, turned in final grades, and closed the book on the most exhausting and stressful semester ever – for me and every college student, professor, and administrator in the country. COVID-19 made the past two months an ordeal for all of us in higher education. And we face huge challenges ahead. Until a vaccine is developed or effective herd immunity achieved, there will be no “return to normal” at our universities. And the economic impact of COVID-1 on universities and families’ ability to pay tuition will be felt for years.  We’re sailing into uncharted waters, and face a lot of hard decisions based on fragmentary information.  

I am very fortunate not to have to be part of the collective effort to figure out how to hold classes and keep students and staff safe next year. My term as Vice Chair of the Faculty at Davidson College is drawing to a close, and with it my administrative duties. I am about to start a full year sabbatical, which I hope to devote to research and service. Research will mostly be dedicated to work on Somalia, including, I hope, completion of a book project. Our family plans to relocate to Nairobi Kenya for the year have been put on hold for obvious reasons, but we will revisit our options in January.

My service work this coming year will be mainly devoted to volunteering with the ALS Association on the Board of Trustees and various committees. Like universities, non-profits face monumental challenges due to COVID-19, even as demand for their services is spiking. I hope to be able to help navigate the challenges the Association will face this next year.

Personally, reaching Day 1 of my 2020-21 sabbatical is something of a milestone. When I was first diagnosed with ALS in March 2018, I had no way of knowing whether I had slow, typical, or fast disease progression. The sabbatical was still over two years away, and it seemed then that just getting to the sabbatical alive would be an accomplishment. Now here I am, and I’m still able to work and travel and do most of what I want to do, just with a slow gait and a limp. It might be audacious, but I’m now starting to think my next goal should be to work until retirement in 6-7 years… I love teaching,  and when you love your work, you want to do it until you can’t anymore.

May 2020 health update

It’s been a while since I shared an update on my health. If no news is good news, then this is a good news blog post. I haven’t had any significant progression in the ALS since the last posting. I sense a very gradual weakening in my leg strength, gradual deterioration in my ability to keep my balance, and slight worsening of the foot drop which slows down my walk. But it hasn’t translated into any changes in functionality. I just took a 2 mile walk with Karin. I am using an AFO (foot-ankle brace) on my left foot to reduce risk of tripping — it’s been a wonderful piece of equipment. And I’ve started to use a walker for longer walks, even though I can probably do without it for now. Anything to avoid a fall and a trip to the hospital during the COVID-19 pandemic — it pays to be careful these days.

After two months of very careful self-isolation in the house, the family and I are on a short trip to Pawley’s Island, SC, where we are social distancing in a new and nicer spot. It feels so good to get out of the house, and even better to enjoy a walk on the beach. We’re in a spot at the edge of the island so very few people are on the beach, making it super easy to keep safe distance. I will happily grade final exams and papers, and do all my zoom meetings for the college and the ALS Association, on a porch looking out over the quiet marsh here.

The walker works great on the hard sand of the SC beaches. and it doubles as a beach chair when I get tired!

My new role with the ALS Association, and a report from the trenches

Earlier this year, I began a term serving as a member of the ALS Association Board of Trustees, and also agreed to serve as Chair of the ALS Association Public Policy Committee. 

This was just before news broke of a dangerous new virus, COVID 19.

Over the past month, the Association has been in full emergency response mode as we respond to the dozens of  challenges COVID 19 creates for people with ALS, their families, the ALS clinics and ALSA chapters serving them, and for ALS research centers. Needless to say, it has been a baptism by fire for me.  

COVID 19 presents unique and exceptional dangers to people with ALS. Many to most of us with ALS have, in varying degrees, weakened respiratory functions, and so are at greater risk if we contract the disease and it spreads to our lungs. We may also face difficulties accessing critical care at hospitals where triage protocols could cite either our ALS diagnosis, or our reduced respiratory capacity, or both, as exclusionary criteria (though the Association is working hard on this issue, see more below).

Even if we avoid the virus ourselves, the support systems we rely on are being disrupted. Clinics are having to shift to remote consultations (in the long run, the shift to telemedicine is a good thing, but in the short run disruptive). Some families with a person with ALS are losing jobs and income. Some family caregivers with jobs outside the home are having to calculate how to provide care without exposing the person with ALS to possible infection. Families are also having to decide how to balance social distancing with the need for professional home care givers to support people with ALS. Lower income households are no doubt under particular strain.

At the same time, the 39 chapters in the Association’s federated system are – like all non-profits in this crisis – soon to face significant financial shortfalls as their fund-raising events have been cancelled. Some are in a better position to weather the storm than others. For them, the challenge is that demand for care services is spiking even as their resources will start to dwindle. The longer and deeper the economic crisis is, the worse the financial pressures will grow on non-profits, and on everyone else. The Association leadership is actively working on strategies to ensure that chapters and the services they deliver to people with ALS will be safeguarded.

There is more, but I think you get the point. 

What you need to know, if you are a person with ALS or a caregiver reading this, is that my new perch in the Association affords me a close look at how the organization is responding to the crisis,  and I can report with confidence that it is getting very good grades at crisis response.

In big ways and small, the Association’s team of professionals and volunteers are effectively troubleshooting immediate challenges, and planning to deal with medium and long term challenges. My new colleagues are working very long hours, late into the evenings and on weekends, working to ensure that care services are able to meet new needs, that guidance to people with ALS is clear and accessible, that clinics are reaching out to patients, that disruptions to research are managed and minimized, and that the Association maximizes its impact on critical public policy issues effecting people with ALS by signing onto letters and petitions with coalitions of patient advocacy groups.  The dedication these individuals have to the well-being of people with ALS is really coming out in this crisis.

You may agree or disagree with some decisions or positions the ALS Association has taken over the years, but know this – these are very good people working very hard on our behalf, often on issues that get no visibility but which have outsized impact.  They include skilled professionals with no direct personal link to ALS, but also hundreds of volunteers on boards and task forces at the national and chapter level, most of whom are former or current caregivers of a loved one with ALS, or people living with ALS, like myself. I am proud to work with this group.

I’m working most closely with our public policy response and so can speak to that in more detail.  Over the past month the Association’s lean but very effective public policy team has signed onto over a dozen coalition letters and petitions, including some that seek to protect people with disabilities from discrimination in health care related to COVID-19, and that guarantee that an ALS diagnosis is never used as an automatic exclusionary criteria in triage. These are going to be, literally, matters of life of death for some of us if triage is required in our areas. The ALS Association leadership team has had rich and – for me – emotionally draining debates over the positions we are advocating for, the principles that should undergird these positions, and the ethical and moral responsibility we have both to people with ALS and to the greater good. I have won some of the debates and lost some. But what has impressed me is the culture of open debate and the deep commitment to ethical principles that permeates the organization. That bodes well for the many policy decisions the Association will have to make in coming months as we respond to new health and economic challenges posed by COVID 19.

I’m a political scientist by profession, and so am not naïve when it comes to the study of organizations and organizational politics. No organization is perfect. They are built by and populated by humans, and are as flawed as we are – sometimes even more so. I can see the imperfections in the Association and the areas where it can, and will, be improved.  I also see opportunities for more effective cooperation and coordination across the many ALS care service, research, and advocacy groups, and hope that is something we can all work toward.

I don’t know how long I have before ALS progression forces me to stop advocacy work, but with the time I have left I hope I can be part of a collective effort to  get us closer to therapies that halt or reverse ALS, promote public policies that advance the interests of people with ALS and related diseases, and provide the best care services possible, so that people with ALS can live longer and better lives. The better we can work together, the faster we will achieve these goals.