ALS Advocacy

ALS Advocacy
Lou Gehrig's Disease - Motor Neuron Disease - Amyotrophic Lateral Sclerosis
Thought it had been cured by now? Still no known cause. Still no cure. Still quickly fatal. Still outrageous.

Monday, December 8, 2014

#alssymp Days +2,+3 -- Research Symposium

Here are more stream-of-consciousness notes from the second and third days of the big research symposium.  


Attendance was inversely proportional to the time lapsed from the start of the conference.  The dropoff in attendance was pretty stunning to me, especially for a meeting with a such a substantial registration fee.  The benefit of the smaller crowd on day 2 (and the much, much smaller crowd on day 3) was that there was that people became much more congenial and interactive.

There is a definite distinction between the clinical track and the research track and I’m glad we were free to jump back and forth. 

Saturday

On the morning of day 2 I decided to give my brain a much-needed stretch by attending the sessions on in-vitro modelling rather than the care-practice sessions (which included a lead speaker on e-health, a topic that I am very interested in).  It’s the old dilemma of trying to be in two places at once, although I’m glad I did the brain-stretching.  I was able to listen and soak in a lot of the principles without getting lost in the weeds. 

Speaking of weeds, I learned a lot about iPS models and can really relate them to agriculture.  We heard from Dr. Eggan in much more intricate scientific detail that sometimes you get what you planted, sometimes you get some weeds, and sometimes you grow something a little different from what you intended.  All those possibilities have to be evaluated for the model to make sure you have what you thought you were making.

And in the iPS models, you can try things, but you don’t have a patient who can say, “That helped,” or “That makes me feel terrible.”  These models force the biomarker issue.  So the process is complicated.

He showed some work that they have done with lights that cause TMS excitability in the model, giving new significance to the flashing blue light. The activity has led them to want to test an epilepsy drug, Retigabine, in humans with ALS.  They will not only look for efficacy but also will be able to compare the activity in humans to help validate the model itself.  

I followed another talk by Dr. Bakkar on RBM45 and KEAP1 remarkably well, but I kept wondering how long all of this intricate modeling took and how much did it cost.  I believe that it’s essential that we have this kind of basic science funded to keep the pipeline full for later clinical trials, but it has to be a tremendously expensive endeavor, and nobody really talked about the prices.

And speaking of the expenses, every presenter had a wonderful slide of acknowledgements at the end of his or her Powerpoint deck.  Colleagues and funders were appreciated and thanked.  A big step in a better direction would be if every presenter had a big thank you at the very top of each acknowledgement slide thanking the people with ALS and their families who made their work possible – by volunteering for studies, by being the rainmakers who raise large amounts of funds, or by being the advocates who make sure that ALS and MND are on national agendas.  The few times I heard the people with ALS thanked at this symposium, it was almost like an afterthought. 

The next sessions I chose were on epidemiology. During the afternoon, I heard valuable human volunteers for studies called “subjects” and “controls” repeatedly. Kind of like some royal underlings and television remotes.  Those terms need to go.  They reflect an attitude that hardly reflects respect for those who are the most valuable intrinsic contributors to projects.

The presentation by Dr. Liying on the first epidemiological study on ALS MND in China was fascinating. One of the lead slides showed the vast range of prevalence numbers from studies in other countries. I think we really need to question whether those differences are real, are a result of our inabilities to count noses well, or are perhaps from diagnostic differences.  The Chinese study showed much less C9 ALS than in the US, and a younger onset.  As the genetics get more complicated, I think that the geography of the genetics gets very interesting.

Dr. Weisskopf’s study looked at military data, this time from death certificate data from veterans of various eras.   The data indicated that WW II veterans were most affected by ALS, yet something in the back of my mind says that WW II vets have also generated more death certificate than later veterans.  I’m sure more is to follow.  This study was one funded by ALS Registry funds but is separate from the Registry and did not use the Registry or the VA Registry for any of its data. I was able to ask a question that I have asked over the years, and I think I got the right answer this time – Have there been studies of military of other nations besides the US?  Yes, there was a French study, but it had some significant flaws.  Weisskopf is working on a study of Denmark’s military as we speak.  I look forward to those results.

The presentation that got the blockbuster tweets was the Fournier study on people with ALS who had experienced head trauma.  It showed no correlation between head trauma and the rate of decline in those people with ALS.  All of the people in the study already had ALS, so it was simply about decline among those already with ALS and not about head trauma causing ALS.

We moved on to a session that demonstrated an ALS “survival score” that included creatinine in its variables.  Later Dr. Mitsumoto did a biomarker presentation that involved good old serum creatinine.  I’m sure that all people with ALS have lots of creatinine data in their old medical records.  If only we had ourselves organized so that every person with ALS can opt in to having medical data used for research.  Also, I noticed that Dr. Mitsumoto always referred to sporadic ALS as “so-called sporadic ALS.”  Hmmm.  The plot thickens?  And Dr. Bowser talked of the huge gap between the discovery of suspected biomarkers and the validation.  And I learned about microfilaments.  There were lots of biomarker candidates that were framed as "promising."  There's that word again.

Sunday

I started the day in some nutrition sessions (although the concurrent twitter buzz told me that there was significant action in the other track with the Beckman presentation on copper chaperones). The feeding tube discussion in the nutrition track gave data but really didn’t break new ground for me.  I did appreciate the dysphagia presentation by Dr. Plowman.  She made it personal, and I think that was a refreshing entrance to her data.  I know that the clinical studies are important in proving hypotheses with data, but sometimes I think that was a lot of time and money to tell us something that we already know.

Later I moved back to the research science track and was fascinated by the non-neuronal cell presentations.  Now I know that those scientists have produced some imaging that shows stunning differences (even to my untrained eye) between those with ALS and those who do not have ALS.  There were also a very interesting so-what challenge from the audience regarding one of the imaging studies.  The questioner asked why it was important if it only showed what the clinician was able to observe with the patient anyway.  That’s when I realized that for every presentation, there should be a “what difference does it make tomorrow” closing.

I am very grateful for the recap of significant presentations that was given to us at the very end of the conference.  That helped me know about what happened in the tracks that I couldn’t attend, and it also helped me see which presentations stood out to the scientists.  We also got wind of a new ALS gene and I’m sworn to social media secrecy until it is published soon.

Some Reflections

SoMe
Social media can add a lot to the conference experience for those who choose to do so.  There were some tweeters of all backgrounds who added a lot to the understanding and tone of the meeting.  Click here for the people who made the official #alssymp tweeter list.  I was also immensely grateful for the good wifi in the facility.  It let me google at will when I heard a foreign term or wanted to look up a name.

So What
The symposium represents a huge investment of time and money.  At the end of every presentation, I think we should have the “so what” discussion.  What will we do differently tomorrow because of these findings.  If that question isn’t easy to answer, then I think it's fair to question some priorities.

Urgency 
The word came up repeatedly when I chatted with the daughter of a man with ALS who took it upon herself to attend.  Her life is built around urgency.

Back when we were dealing with ALS, I found it frustrating that people we depended on didn’t move quickly or with purpose.  I got the same sense of frustration with the feeling of pace, progress, and urgency at the symposium.

Patches, And No Way To Make A Quilt
I think that the single most significant improvement that we could make in the fight against ALS would be for the ALS MND Alliance of organizations to have a shared information policy to insure that any granted dollars come with an agreement that study data adhere to some standards.  That’s the only way there will ever be potential to put 2+2 together from these discrete study successes.

It would also be an improvement to insure that all granting organizations have data on grants, amounts, recipients, outcomes, etc. that are consistent and transparent and searchable by the public.

At the symposium we saw three days’ of scientific study accomplishments with no common threads for information synergy.  It’s the ultimate silo system.

We have an ALS information crisis and it needs to be fixed, and great neuroscientists are not necessarily great information scientists.

“We Speak A Different Language”
I had a conversation with a lovely woman who had presented at a prior symposium.  I spoke of my feeling of needing to have PALS, CALS a part of the fabric of such a symposium.  She disagreed strongly.  She said there was a person with ALS present when she spoke and she was not comfortable with that.  Besides, she said, “We speak a different language.”

Perhaps that is precisely the reason why people with ALS and caregivers need to be there!

Can you imagine the perspective and clarity that interested people with the ultimate skin in the game would have for these scientists?  Can you imagine the light that those living with the ticking clock could shed on vying priorities?  Can you imagine how much the scientists could learn from the questions and comments of people with ALS and their families?  Can you imagine how much more powerful advocates people with ALS and their caregivers could be for clinical science with more knowledge? Can you imagine? 

I can.


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