Comparing recent ALS Association and MDA statements on clinical access


  1. ALS Association, “Principles for Urgent, Patient Centered ALS Clinical Trials (Fall 2018)
  2. Muscular Dystrophy Association, “Redoubling the Urgency for Innovative ALS Clinical Trials” (May 27 2020)

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.

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.

My “Patients Like Me” Introductory Video

I’ve just started serving on the Patients Like Me Team of Advisors. It’s a one year term, and involves providing patient input on the Patients Like Me forums, data collection, privacy issues, other matters. We had an initial meeting in Boston where each of us was filmed for a short introductory video, which you can find here:

Music and a Fundraiser for ALS in Davidson, NC

A big thank you to the musical group Davidson Local — a fun collection of musically gifted Davidson faculty, students, and friends — for offering me a platform to do some ALS awareness-raising and fundraising during a break in their gig at Summit coffee last night. The crowd was great, and when we passed around the hat (well, actually an empty beer pitcher), patrons donated $512 to the NC chapter of the ALS Association. Thanks to all!

Our Bucket List trip to the Grand Canyon and southern Utah

My professional work has allowed me to travel much of the world, including to some extraordinarily compelling and difficult-to-access places. But I’ve been less well traveled within the US. One trip on my Bucket List has been the Grand Canyon  and southern Utah. I really wanted to experience it while still relatively mobile. So this Christmas, our family gift to one another was a trip out West.

It was even more spectacular than I imagined.

We rented an SUV in Phoenix and started off in Sedona; the red rock cliffs were beautiful, and I managed a 2-3 mile hike on up-and-down and uneven terrain in Red Rock State Park. Then came the bad weather – a major snowstorm that hit north of us, burying Flagstaff, the Grand Canyon, and most of the high altitude locations in southern Utah with anywhere from 4 to 12 or more inches of snow. We made our way through the snow to Flagstaff, and then the Grand Canyon. The roads were ok and though the view at the Grand Canyon was very limited when we first arrived, the snowfall made for beautiful landscape and appeared to scare off many tourists, as the park was not crowded. Thanks to a Christmas gift from my brother and sister in law, we stayed at the historic El Tovar lodge right at the edge of the canyon. The next day, the clouds and snow gave way to clear skies and we were treated to spectacular views in a cold, winter wonderland.

And the mind-blowing landscapes of the West continued, as we traveled to Monument Valley, Arches National Park, Capital Reef, the unbelievable route 12 up and over Boulder Mountain, Bryce Canyon, and Zion national park. All of the locations featured otherworldly combinations of impossible rock formations with soft, deep snowcaps.  We thought after a few days we’d get bored; but each new park brought more amazement. Except for Zion, which was crowded, the other parks were lightly populated with travelers, thanks to the snow, and in many cases we were alone gazing at eye-popping views.  We hiked for moderate distances at every park; the kids would hike at a normal pace and then wait for me to catch up, with Karin walking with me.  I used walking poles and that helped on the slippery snow and ice, but even so I still slipped and fell several times. The snow made for soft and harmless landings. Each time, my supportive family rushed to me and laughed uncontrollably at my “slow motion” falls before helping me to my feet…

We ended the trip in Las Vegas, where my sister and brother-in-law treated us to tickets to the Cirque du Soleil “O” performance, which was amazing.

When we first planned the trip, it was a calculated risk. It’s always hard to know if ALS symptoms will worsen quickly over the course of several months. I was prepared to have to stay in the car while my family hiked. But my legs held up fine and I was able to hoof it to everything I hoped to see, just slowly.  I’m a bit tired now, but am very, very glad we were able to pull off this trip.

Year Four with ALS

It was three years ago this month when I first noticed something was wrong. I was playing tennis, and when I tried to push off for a quick lateral move to hit a wide shot or poach it was as though my brain said “go” and the leg said “no.” There was a strange delay in messaging, and it meant that my first step was fine but the second one was dangerously late and awkward. I fell several times, to the point my doubles partners started to worry about me and I stopped playing. I initially attributed it to being out of shape after a lengthy recovery from a knee injury. But from the start it never felt like I was just tired or out of shape — it felt more like a disconnect in the messaging. And it was. It took me 12 months to mention it to my family physician, and another 3 to get the diagnosis.

So now I’m entering year four with ALS. The disease’s symptoms are, for now,  still confined to my legs, which are gradually weakening, in maddeningly asymmetrical ways. The foot that was initially weak is now stronger, the foot that was strong is now much weaker. Balance is hard — I walk very slowly and deliberately if without a cane or an ankle foot brace. Bending over to pick things up or tie a shoe is an adventure in balancing. But with the ankle foot brace (AFO) on my weak left foot, I can walk for several miles, at a reasonable pace, and work out on the reclining bike machine for an hour. Weirdly, it’s easier to hike a mile than to just stand with confidence without shoes at the kitchen sink.

This is all good news, under the circumstances, since the median life expectancy for people with ALS is only three years from symptom onset.  But as I enter year four, surpassing the terrible three year benchmark, I can feel the legs weakening, and can feel fatigue set in faster when I walk for long distances or climb stairs. So far, this has been a slow progression, and seems to suggest that I qualify as a “slow progression” case. But it’s still a disease progression, and like everyone else with ALS, I’m on the clock.

We’re all on the clock, of course. It’s just that some of us can see the sand moving in our hourglass a bit more vividly than others. When you’re “living on borrowed time,” it incentivizes setting clear priorities and making the best use of whatever time one has left.

For me, my priorities and goals for 2020 are to be a good role model to my children and students for how to handle adversity with as much grace and sense of humor as I can muster; to fight death without fearing it; to spend quality time with family and friends, celebrating everything life has to offer with them; to stay professionally active as a teacher and scholar; to redouble my volunteer work with ALS organizations to raise awareness, raise funds, provide quality care services, and  promote policies that expedite the search for therapies that halt or reverse ALS;  to work to build better collaboration, trust, and unity of purpose within an ALS community that is frustratingly and unnecessarily fractured; and to thank God every day that I have such a loving and supportive family to count on.

A Successful Santa Hustle Fundraiser

The Santa Hustle half-marathon, which partnered with the NC Chapter of the ALS Association to enable us to fundraise as part of the event, was a great success. We raised over $30,000, and had a great group of friends and family running part or all of the half marathon with me as I rolled through the 13.1 miles on a recumbent trike loaned to us by Charlotte Cycles (BIG thank you to Charlotte Cycles!). I have to be honest — triking the half marathon was actually pretty easy, much easier than working out on a recumbent bike in the gym. Those trikes are built for speed, so whenever I got to a downhill, I flew! Many thanks to everyone who supported this effort, with your contributions, your participation, your help organizing the event, and your moral support!

after the race
prepping before the race
My sister Susan, my mom, and cousin Phil.

Training for the Santa Hustle Fundraiser

I got this….

Next Saturday (Dec 14) I’m helping to fundraise for the ALS Association NC chapter as we partner with the Santa Hustle half marathon. A variety of different groups are sponsoring one of the 13 miles to raise money, and will have an escort running alongside me as I attempt to finish the 13 miles on a recumbent trike. Training so far is looking good, I can complete 10 miles in 52 minutes. But stationary bikes don’t have to go up hills! A big thanks to Santa Hustle for partnering with the ALS Association, to Charlotte Cycles for loaning me a recumbent trike, and for all of you who have already contributed to the fundraiser. I’ll post photos, hopefully of me finishing the ride in one piece. The link to the gofundme page is here: