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.

Wednesday, December 31, 2014

The "Me" in SoMe isn't Me

It's the last day of of an incredible year for ALS in the social media.

So, let's think about what we have learned about the power of social media and the fight against ALS.  What's the first thing that comes to your mind?  Really, what's the first thing that comes to your mind?


Naw, you were thinking Fundraising, weren't you?

Has the idea of buckets of money made us myopic about the higher usefulness of social media to defeat ALS?

Has brand management overshadowed the power of ideas and interaction and accessibility that social media provide to a diverse population with physical challenges?

It shouldn't be all about the brand and fundraising.  It's not all about the organizational "Me."  It's about a very important "You" -- people with ALS and their caregivers.

Yesterday an apparently helpful tweet from an organization offered people newly diagnosed with ALS a link to information.  Upon clicking they were greeted with the year-end popup to donate.  Whoops.

Paul Wicks of PatientsLikeMe recently wrote an interesting piece on the Ice Bucket Challenge and ALS --

There's a lot to consider there.  The idea of organizational curation of Wikipedia information about ALS jumped out at me.  What a way to step forward and help maintain some quality in information that people find every day!  Some organizations don't have time?  Have we completely lost our vision?  You can be darned sure they would have time to take care of a Wikipedia entry on the organization itself.

The fight against ALS needs to turn itself inside-out.  It's simply going to take a major attitude change about the "Me" in SoMe.

Tuesday, December 16, 2014

Should This Disease Be Forgot?

A few weeks ago on a national NPR program, a caller mentioned last summer's dumping of buckets of ice and said, "Uh, what was that disease?"  Time passes on.  So do memories.  The disease that was on the world's mind and lips in August is fast fading back to its shadows.

There are year-end memories of the entertaining ice and the videos.  There are year-end memories of the buckets of money that were donated.  Who is talking about the outrageous disease that motivated it all -- ALS?

Picture a football stadium full of people.  Imagine that many people being killed by ALS.  Yes, that many people have been killed by ALS in the few months since we dumped ice on our heads.

During the heat of the ice bucket challenge, I made a series of very modest online gifts to a number of ALS charities (the kind at add up to hundreds of millions of dollars when people everywhere give them).  I got the autoresponders.  I got a few nice letters.  I got my tax receipts.

I've not gotten any insights into ALS.  I've not gotten any year-end "thanks and here's what we did with your contribution" messaging.  Were I not previously involved with ALS, I've not been given any reasons to stay committed to the disease.

ALS charities have email addresses from all of those who gave spontaneously and generously online in August.  Today's donors don't want constant requests for more money, but perhaps they would like some reasons to continue an interest in ALS.  Perhaps they need to be thanked again and again so that they know that the disease persists and must not be forgotten.

Awareness is critical.  It's a long-term proposition.  The success isn't in hundreds of millions of dollars. The success isn't in the entertaining viral videos. The success isn't in the media coverage that talks about dollars but not the disease.  True success will be in the awareness and relationships that will finally give the world the resolve to deal with this outrageous disease.  That awareness needs to be cultivated.

And today we already have people saying, "Uh, what was that disease?"

Thursday, December 11, 2014

The Ears Can Be Deceiving

If you've ever been owned by a Basset Hound, you know that the big ears can be deceiving.  Bassets aren't always the greatest listeners.  Why the big ears?  They're more about stirring up the scent for their noses to track things than actually to listen (especially to listen to their human beings who sometimes have things to say that are inconvenient to hear).  I love my hound but am realistic that's she's smart when she wants to be.  And listening isn't her forte.

We're at that time of year when the ALS Association is conducting its "listening tour" for public policy priorities for next year.  It consists of an email sent to chapters that has not changed a lot over the years. Chapters have a short due date to reply to the email with their three priority picks.  That's it.  Some chapters reach out to people with ALS and caregivers and their chapter benefactors.  Others don't.  They pick three and reply.  It's traditional.

Below is some text from this year's email.  In 2014 we have some wonderful social media tools that would cast the nets more broadly and generate some energy and ideas beyond pick-three.  They would make it much easier for everyone truly to listen.  Ah, the same tools that were so effective for engaging people to dump ice and be interested in ALS in August would also work for them to hear and be heard in December.


The ALS Association Public Policy Department is currently in the process of working with local chapters to identify the issues most important to the ALS community nationwide.  Feedback received will help to inform our 2015 public policy priorities.  Please take a few minutes to review the following public policy issues.  I’ve included links for ease of finding information on the topic. 
Afterwards, we ask that you IDENTIFY and RANK your top THREE public policy issues and submit your responses to us by email.  You may reply directly to this email.  We need all responses by Monday, December 15.  Thank you for your participation!
Public Policy Issues
  • MODDERN Cures Act
  • PDUFA implementation
  • Compassionate use programs
  • Drug development and approval process
  • “Right to Try”
2.      Health Care Reform Issue may include:
  • Ensure key provisions of health reform enacted in 2010 remain law, including:
    • Closing the Medicare prescription drug benefit coverage gap
    • Eliminating preexisting condition exclusions
    • Eliminating lifetime and annual caps on health insurance coverage
  • Promote respite care and respite care funding
  • Implement the CLASS Act, legislation included in health care reform enacted into law in 2010 that establishes a national program to enable people to purchase long term care services
  • Provide Medicare and private coverage of in-home custodial care services and home health aides
  • Change Medicare’s homebound requirements
  • Reimburse/health benefits to family caregivers
  • Establish caregiving tax credits separate from the deduction for medical expenses
4.      General Medicare Issue may include:
  • Eliminate therapy caps and/or extend the process to receive an exception to caps on coverage
  • Streamline and speed processing of claims
  • Improve coverage of Durable Medical Equipment
  • Provide Medicare coverage for iPads, iPhones and other similar devices
  • Oppose implementation of/repeal Competitive Bidding for DME in Medicare
5.      Medicare Prescription Drug Benefit (Medicare Part D) Issue may include:
  • Implement provisions of health reform enacted in 2010 that eliminate the coverage gap
  • Ensure Medicare coverage of specific prescription drugs (Rilutek is currently covered)
  • Ensure Medicare coverage of drugs prescribed off label
  • Limit the use of utilization management tools such cost sharing, tiering, therapeutic/generic substitutions, quantity limits and other policies of Medicare prescription drug plans
6.      National ALS Registry Issue may include:
  • National Office to continue support Chapter outreach and enrollment activities by providing materials, equipment, education and training
  • Continued funding for the National ALS Registry
  • Expand the number of surveys in the registry
  • Encourage research use of the registry
  • ­Continued funding for ALS research or policies and programs that advance ALS research at:
    • the ALS Research Program at the Department of Defense
    • the National Institutes of Health
    • the Food and Drug Administration
    • the Department of Veterans Affairs
    • the Centers for Disease Control and Prevention
  • Stem cell research including expediting human trials
8.      Speech Generating Devices Issue may include:
  • Ensure access to non-speech functions such as email, internet access and environmental controls
  • Capped rental
  • Eye-gaze technology
  • Eliminate the 5-month waiting period
  • Eliminate the “20-40 rule” under which a person with ALS must have worked five of the previous 10 years in order to qualify for SSDI
10.    Veterans Issue may include:
  • Assistance navigating/accessing VA benefits
  • Change functional criteria and other elements of the adaptive housing grant process
  • Establish a formal process to expedite claims
  • Develop partnerships with VA for clinical care
  • Increase grants for adaptive housing and accessible vehicles

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. 


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.


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

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.

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.

Saturday, December 6, 2014

#alssymp Day +1 -- Research Symposium

The first big day started in a lovely convention center.  After several introductions (including an appearance by the Prince... not the singer, but the real Prince) the presentations began.

The first general presentation was by Dr. Sandrock of Biogen IDEC.  He spoke of clinical trial design and some better principles for Phase II trials.  Some of what he said reminded me of what a gentleman with ALS suggested several years ago -- larger, more definitive Phase II trials.   He also alluded to a preliminary observation period.  I see the value in that, but why eat up more clinical trial time.  I still think that on day of diagnosis patients should have the option of opting in to having their data available for clinical trial use.  That would give any trial a trajectory of any patient's ALS before the start of any trial therapy or placebo.  And we learned at other sessions yesterday how important and variable patient trajectories are with ALS.  I really have trouble with the word "progression."  Sandrock gave a summary of the characteristics of an optimal Phase II trial.  (The slide is in my twitter stream and unfortunately I am not able to post pictures in Blogger from this connection.)  Unfortunately without reliable biomarkers, the characteristics that he described can't be delivered.  I'm pessimistic that we'll see another trial from Biogen Idec until that kind of trial can be designed and delivered.

The first breakout I attended included Dr. Benetar speaking on diagnosis.  There is an approximate 12-month diagnosis delay for people with ALS and that has been the case for more than a decade.  That is outrageous in my opinion.  He indicated that much of the delay is attributable to a period between the primary care physician and getting the patient to an ALS neurologist.  I wish they would crowdsource this sometime and find the real reasons.  I suspect that could reveal some of the weaknesses of American healthcare delivery, including inept scheduling.  He showed data on interventional delay, too. Again, I wonder if that's not as much an indictment of American healthcare delivery as physician behavior.  Have you ever tried to get a riluzole prescription and then get it filled?  Benetar showed some disease trajectories and showed what many of us have also observed -- ALS trajectories are often not linear as neurologists often describe.  Some patients have a linear decline followed by a sudden dropoff.  They hit a wall.  It was nice to see some data that admit that.

The next presenter was interesting, but so help me, I thought I would scream when I heard clinical trial volunteers called "subjects."  Time to move on from that term.  And while we're at it, "controls" is a pretty disrespectful word for people who volunteer their data and parts of their lives for medical research.  Let's use references that respect all who volunteer for clinical trials as team members who are as valuable as those paid to do the research.  And while we're at it, every acknowledgement slide at these meetings would do well to thank the patients first.  I'm glad that at this meeting the importance of the data from those people who volunteer for the control group has been emphasized.

Dr. Schoenfeld gave an interesting presentation on trial design when the treatment being tested poses some potential harm in its delivery mechanism and therefore puts placebo patients at risk.  That's a big deal in the world where stem cells and other things are being placed in bodies.  He suggested that an add-on trial design could be a solution to some of the placebo dilemma in these cases.  And again the concept of an observation period in a trial came up and I ask why we aren't tracking all with ALS from day of diagnosis.

Dr. McGrath from Neuraltus spoke on the biomarker that they were tracking in the NP001 trial.  I was interested in this because some friends participated in this trial and crashed during the washout period when the biomarker was being tracked.  He showed the LPS+ data they tracked and how responders' data did correlate.  I asked about whether they would design their Phase II trial differently if they knew then what they know now.  That washout period with no further therapy was cruel to patients who had responded well during the therapy period.  He indicated that the trial design was based on budget.  They had not funds to do anything more than the six-month trial regardless of whether the drug worked or not.  OK, lesson learned, folks.  Every patient who volunteers for a trial needs to ask, "What happens to me if this stuff really works?"

My day moved on to some presentations on communication devices (while I watched Steve Gleason's entertaining and effective twitter stream on communication device comments to CMS).  There was a brain-interface presentation that interested me, and I learned that this kind of brain interface requires some gazing.  I wonder if some of the problems that people have with today's eye-gaze devices will carry forward to that technology.

I hope that all of the poster presenters will upload their data to the sharable site where we can view in more detail and with more energy than we had at the end of a long day.

Your best tidbits from this symposium will come from following #alssymp on twitter. 

Friday, December 5, 2014

Just Follow 37

Time is running out for the public comments period to CMS regarding speech generating devices and communication needs for people with ALS.

Communication is more than speech.

Just follow the lead of Steve Gleason.  You'll enjoy his tweets and do a good deed, too.

Thank you.

Click here for your messages from Steve Gleason.

Thursday, December 4, 2014

#alssymp Day -1 -- Ask the Experts

For the next few days I'll share impressions as I attend the big global ALS MND Symposium.

The events involve a complicated (at least for me) series of "insider" meetings for organization employees and healthcare professionals.  The culmination will be three days of research meetings for which anyone who can put up the substantial registration fee can register.

Yesterday there was also a public opportunity -- An "Ask the Experts" session held by the host ALS organization.  I knew about the concept from past symposia and asked if I could attend.  Local patients and caregivers are the main audience.  There was no webcast this year.  When I inquired, I was advised that they would accept submitted questions, and I did so on behalf of several people with ALS who participate in an online research forum.  See the blog post dated November 30 ("What Should I Talk About").

An email advised me that Ask the Experts would start at 2pm yesterday.  When I got there shortly before 2, the crowd was sparse.  A few families were there with members who appeared to have ALS.  People trickled in and the session itself didn't start until 2:50.  Fortunately my phone battery was good and I could still tweet.  They provided video and audio translations from the presenters' English to Dutch and French.  My crowd-size guess is around 50 people with around a dozen people with ALS.

The format was one that they had used in the past.  There were three scientists --
Dr. Hardiman (Ireland)
Dr. Van Den Berg (Belgium)
Dr. Bruijn (US)

Each gave a presentation followed by a very short question and answer period.

Dr. Hardiman
Presentation was around the mouse model.  Not much new. She emphasized the cost of human trials, mentioning that the recent Dexpramipexole trial that plopped cost $100 million.  A rather discouraging note was that good drugs fail because of flawed trial design. The solutions cited were cleverer subgrouping and subtyping making more personalized trials.  Tricals is an ENCALS group which is working on better trial matching to patients.  She had a really nice bubble chart showing the various known genetic variations in ALS.

Questions -- Why not have individual trials?  What about compassionate use?
A (VDB) -- If you have compassionate use, then you can't do trials.  Without a biomarker we can't measure efficacy in a very small trial.  If in a small trial everybody got better, then compassionate use wouldn't be a problem.
A (Hardiman) -- Lithium was an example of something that people thought might help and the trial proved it hurt.
My thought cloud -- Lithium was actually a great example of public data showing that the drug didn't work for ALS far before the formal clinical trial was finished.

Van Den Berg
He spoke of Project MinE and identifying the spectrum of disease variants and associated risks of having genes vs risks of getting the disease.  The goal for Project MinE is to sequence 15000 people with ALS and 7500 controls.  He made some very bold statements about combining all ALS biobanks in the world, using standard formats, etc.  I hope he's right, but I wonder if it's wishful thinking.  The lure of volume pricing isn't going to be enough to get some researchers out of their silos in my opinion.
Question -- What are you doing with all this information.   Are you just collecting it?
A (several) bridged the conversation to iPS models and the ability to screen drugs against a lot of variations of ALS.

The four main IBC research grants were described.  There was a reference to "ALS Association Research Institute" which sounds grand but I think is just a title to get all their "prongs" to sound more like research.  The big news I heard was that the four IBC granted projects will use a GUID as a patient-level identifier.
Question (me) -- Is anybody using GUID for control groups to make them more rich and versatile?
A (several) -- Got a lot of feedback on the importance of control groups and heard a foggy from Bruijn that they would look at that.  Hardiman thought it would be unethical since you have to deidentify control data.  I think that GUID is considered to be deidentified.
Followup Question (me) -- Is anybody tracking the control group members to see if they develop ALS?  (I think I detected an eyeroll from VDB?).

Question -- Nobody has discussed stem cells.  Why not?
A (Bruijn) -- That wasn't within the scope of her prepared presentation.  It's not because they are not important.
The discussion livened up a little and there were some references to concerns in Europe, are stem cells the answer, etc.  This was a topic that patients obviously wanted to hear about and discuss.

And time ran out.

There was no indication that the experts got the questions that were presubmitted.

Maybe next year the host organization will make this session more productive and patient-focused and accessible.  Ask PALS, CALS what format works for them.  Get the questions that they want answered and then have some lively discussions (like the one that was just starting on stem cells when time ran out). Presenters were all fighting the clock trying to use presentations that were too long and not much discussion was stimulated. There are better ways to do this.

The patients and caregivers really are the experts.

Tuesday, December 2, 2014

They Pitched A #GivingTuesday Shutout Today

Today I received eight (yes, eight) different emails from ALS organizations asking me to make a monetary #GivingTuesday donation.

Today I received zero (yes, zero) messages from any of them asking me to contribute my comments in the soon-closing public comments period on CMS coverage of technology related to communication and speech-generating devices.

Score:  8-0




Please make your comments using this link and ask your friends and family to comment, also.   We need to give CMS an earful before #GiveEmAnEarfulSaturday .

Thank you.

Monday, December 1, 2014