In December 2020, I was part of a group of people living with ALS who took part in the 31st International Symposium on ALS/MND, the largest annual gathering of researchers working on ALS or ALS-related diseases. Because of COVID-19, this year’s symposium was virtual.
Below, I share a few personal take-aways from the opportunity to listen to three intensive days of research presentations.
What role can we usefully play in ALS research design and practice?
Ever since I decided to engage on ALS advocacy after my diagnosis, I have struggled with this question: what is the appropriate role for people diagnosed with ALS in discussions and debates over ALS-related research? On the one hand, the movement for patient voice and patient-centrism across all disease groups has grown rapidly in recent years and has shown us that increasing patient engagement at all level of therapy development has real potential. On the other hand, people with ALS are a challenging group to integrate into some deliberations, in part because we generally have such a short life expectancy after symptom onset. Most of us just don’t have the time or energy left to educate ourselves on the many complexities of ALS research and policy, and then play the kind of sustained role needed to have an impact. Those of us who do are in the “slow progression” sub-group of pALS, and often possess specific professional backgrounds in medicine, public health, academic research, statistics, and other fields that equip us to follow at least some of the complex scientific issues at play. That is a small subset of the total population of people living with ALS.
After listening to sessions devoted to basic scientific research on aspects of the disease, and assessments of clinical trial results, I reached the following conclusions:
- pALS cannot and should not attempt to master the complexities of lab research, unless they happen to already possess an advanced degree in a field such as cellular biology, bio-chemistry, genomics, etc. The research is simply too complex and advanced to master through a quick self-education. We need to leave that work to the experts.
- pALS do have potentially useful roles providing input on the structure and best practices of clinical trials. We play a central role in this research; we have ample experience in clinical trials; and most of the issues and debates surrounding the structure of clinical trials (enrollment criteria; bio-markers; placebo groups; challenges of heterogeneity in disease progression on trial results; open-label extensions and expanded access options; platform trials; etc.) are, with a bit of reading and time, relatively accessible and understandable to generalists.
- pALS need to be very careful about jumping into debates about the statistical results of research. Much depends on the level of statistics at issue. If the discussion involves relatively straightforward discussions about a statistically significant correlation, yes, those of us with some basic stats background can contribute. But advanced statistical analysis requires advanced statistical knowledge, or else we risk making serious errors.
Respect the science
There is a lot of easy chatter on social media criticizing research on ALS. Some of the criticism is warranted. Some of it is misinformed, disrespectful, even arrogant. Lately, a lot of it is driven by our observations of how the world was able to mobilize massive resources and research efforts to develop vaccines for COVID-19 within a year of the pandemic outbreak. Why can’t that be done for ALS, some ask.
My overall takeaway from the proceedings is that contemporary research on ALS is well-conceived, sophisticated, and promising. It deserves our respect. Researchers have to work within some very challenging parameters. These include a disease the causes of which we do not know; a lack of reliable bio-markers; sizable heterogeneity in disease progression; and the sheer complexity of neuroscience and the human neurological system. We get understandably frustrated over the lack of progress in development of effective therapies to slow, stop, or reverse ALS. And we see weaknesses in clinical trial design that can be exasperating. But we have to remind ourselves that most other neurological diseases, including some that garner much larger amounts of research funding, have encountered the same slow progress in development of effective therapies. Alzheimer’s, Parkinson’s, Huntington’s, and MS are among the many other neurological and neuromuscular diseases which have defied the best efforts of the scientific community to date. We’re hardly alone.
The breakthroughs will come, hopefully soon, but the medical researchers working on ALS are grappling with some of the most complex and poorly understood aspects of human physiology. And unlike COVID-19, we don’t even know what causes ALS. Without that knowledge, effective therapies are going to be harder to develop.
Impact of ALS on the brain
Many of us in the pALS group attending the symposium were struck by the research linking ALS to greater risk of cognitive, emotional, and executive function impairment. This is a sensitive topic in the ALS community, but one we need to openly acknowledge and discuss. How do we self-monitor for changes in cognition or empathy, especially for those of us who continue to work full-time? What does this mean for our aspirations to play more of an active, lead role in ALS organizations?
A space for thoughtful discussion and exchange of ideas
Our group of pALS met via zoom at the end of each day to discuss the proceedings. It was very useful. It was clear to me that we need to make that kind of space for thoughtful, extended discussion and debate among pALS more widely available. Social media is not the right environment for us. I hope we can find a way to develop electronic platforms that can support and build on this sort of serious exchange of ideas among pALS. We have a lot of smart, thoughtful people among us, and we need to make sure their knowledge and insights are given plenty of exposure.
Ken's Caucus 