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

Sunday, November 30, 2014

What Should I Talk About?

That's every speaker's quandry.

There is a wonderful old story about the young, new preacher who arrives in a small town in Indiana. He is replacing a long-time fixture and is worried about his first sermon. He goes around to the barber and the banker and the grocer and asks, "What should I talk about?" He gets much guidance. Then he goes to the little old lady who is known to be the most opinionated person in town. "What should I talk about?" She gives him the wisest advice of all, "You should talk about God, and you should talk about five minutes."

So often at ALS MND meetings speakers prepare all kinds of intricate material when there are hundreds of questions that those with ALS and their caregivers would like to ask and have discussed.  There is never enough time for Q and A at the end of presentations, and many questions go unanswered.  Maybe they should just talk about ALS and talk about five minutes and let the questions lead the way.

This week prior to the big ALS MND Symposium in Brussels, there will be a two-hour "Ask the Experts" session for patients and caregivers to ask questions.  We asked, and they are accepting submitted questions via .  Some patients and caregivers who participate in the research forum at asked me to submit the following list of varied questions:

  1. Do you have any ideas why the developers of Generveron are not participating in the Symposium? As a most exciting announcement this year of halting or slowing progression in 7 of 8 PALS in a trial, it seems incredible that the subject does not appear to be anywhere on the agenda.
  2. The idea of a GUID for de-identified patient-level data in ALS data repositories seems like the ultimate common sense to me, especially with the proliferation of new, similar iPS projects. Please share your thoughts on what it will take to implement a Global Unique Identifier in ALS project databases.
  3. What can we learn about speeding up drug development from the Ebola crisis? Bill Gates recently made some comments about needing better global disease surveillance. He and others have suggested that there are ways to gather powerful clinical evidence without placebo arms. It seems to bother people a lot to suggest a placebo for Ebola patients but yet placebo is just fine for someone with ALS. Your thoughts, please?
  4. Has it become customary practice in clinical trials to sequence the DNA of the participants? If so, whole genome or just for specific known and suspected genetic markers? And if so, what is the customary practice regarding providing participants with those results? And what is the customary practice with respect to releasing the data to ALS genetic databases?
  5. Regarding the Cytokinetics… It is frustrating to me when a trial fails and then gets redesigned for a costly and time-consuming do-over. Have any other trials used SVC as a primary endpoint? Are you allowed to go into a trial with a premise that a drug could positively A or B (alternative primary endpoints)?
  6. If standard-of-care were not an issue, should every clinical trial have a branch without riluzole? Is it possible or likely that riluzole adversely effects efficacy of other treatments?
  7. If you had ALS, what would you be trying?
  8. For years ALSA has cited multiple studies of a much higher incidence of ALS among those who have served in the US military.  Have there been such studies of those who have served in other countries’ military?
  9. What ideas do you have for the next year’s equivalent of this year’s IBC?
  10. If an MND patient is getting a balanced diet, are vitamin and mineral supplements a waste of time and money?
  11. How important are large-animal MND studies. Have any large animals with ALS-related conditions ever been successfully treated?
  12. How important will bioinformatics become? Will Terry Speed be helping with MND-related research in the near future?
  13. Why is it so difficult for any research lab to win the $1 million Prize4Life prize for extending the lives of SOD1mice by 25%
  14. A $1 million Prize4Life prize was awarded for a Biomarker Tool that does not seem to be very useful. Do you have any updates on this tool or other biomarkers in the pipeline?
  15. Do you think that there’s any future in exoskeletons (and 3D printers to make them cheaply) for MND patients?
  16. In the panel of experts suddenly developed MND, what would be the one main question that they would ask? And how would the others respond?

We look forward to the discussion of the answers.

Saturday, November 22, 2014

We Need An Air-Quotes Dictionary For The Fight Against ALS

From the day a neurologist made air-quotes with his hands when he used the word promising to
describe experimental clinical trials for ALS, I knew we were in trouble.

Over many years we hear words over and over used to generate promise and hope and donations.  The words have become devalued.  They don't mean diddle without specifics.  They all need air-quotes.

We have to press for what they really mean.  Words are cheap.  We need substance.

Here are some that set off bells after you've been around the fight against ALS for a few years --
• Breakthrough
• Collaboration
• Consortium
• Exciting
• Game-changing
• Groundbreaking
• Hopeful
• Leading
• Partnership
• Promising
• Transformative

They make us all feel good. We need to press for details from those who use them.

As an ALS advocate once said, "If something is called 'exciting' in a press release, it probably isn't."

Or maybe there's a drinking game here...

Thursday, November 20, 2014

Please Speak Up So That People With ALS Are Not Silenced

Medicare CMS has a public comments period that ends on December 6.  It involves their rules on so-called Speech Generating Devices and related technology.  We must submit comments on behalf of all those who can't.  We must make it clear that technology that enables people with ALS to communicate in any manner and, therefore, participate in their healthcare and lives is not a luxury.  It is a necessity.

Here is the issue we need to address as stated by CMS:

Effective January 1, 2001, speech generating devices are considered to fall within the durable medical equipment (DME) benefit category defined in section 1861(n) of the Social Security Act (the Act).   Speech generating devices are covered if the patient suffers from severe speech impairment and the medical condition warrants the use of a device. Speech generating devices are defined for Medicare coverage purposes to include dedicated speech devices used solely by the individual who has severe speech impairment.   Speech generating devices also include software that allows a laptop computer, desktop computer or personal digital assistant (PDA) to function as a speech generating device.  Devices that are not considered speech generating devices include devices that are not dedicated speech devices, or devices that are capable of running software for purposes other than for speech generation.  The national coverage determination (NCD) is available in section 50.1 of the Medicare NCD Manual (Pub. No. 100-3, Ch. 1, Part 1).
Since 2001, the technology of devices that generate speech and the ways in which the devices are used by patients to meet their medical needs has changed significantly.  For example, patients now use speech devices to generate text and email messages for the purpose of communicating with their caregivers and physicians.  Therefore, in light of the changes in technology and use of devices that generate speech, we are internally generating a reconsideration of this NCD to address coverage of devices that generate speech as well as other forms of communication.

Here are the comments that I just submitted.

When Mom was dealing with ALS in 1996, there was a huge lesson in our lives -- we take so much for granted. 
This morning I sat down in a comfortable recliner and am typing on a laptop to participate as a citizen in communicating with my government.  I can reach to the left and turn on a light switch.  I can get up and turn up the thermostat when the room gets cold.  If I need someone else in the house, I can yell (or better yet, text).  If, God forbid, I have an emergency, I can dial 911 and get some help. 
It was not so easy for Mom.  We worried about her needing to call 911 in an emergency and not being understood or being dismissed as a drunk. There was some technology that helped.  She had a little Franklin talking dictionary that we programmed with some emergency information.  The Clapper let her triumphantly control the lights... until she could no longer clap.  Communicating with her healthcare providers was difficult to impossible.  
Fortunately technology has leapt forward from the days of talking dictionaries and The Clapper.  There has been a remarkable convergence of technologies that can let people with ALS lead fuller lives more safely and be in charge of their own healthcare decisions.  Thank heavens, they can be enabled to communicate clearly and directly with healthcare providers from home without relying on speech.  Technology today can let them turn on the lights even when they can't clap.  They can control the temperature in the room or beckon a caregiver downstairs doing laundry.  They can tell a power-wheelchair to adjust to avoid the dreaded pressure sores.  They can even advise a commode when it's time to start cleansing.  Yes, we healthy people take so much for granted that technology can now assist.  Consumer devices are getting amazingly smart thanks to two-way communications.  
I feel strongly that CMS needs rules that will let CMS make smart decisions as technologies advance and converge.  We saw Steve Gleason in a Super Bowl commercial demonstrating some empowering software that is pretty mainstream today.  As consumer technology gets better and more affordable, CMS needs to be able to adapt its policies to take advantage of that.  It's good for those with ALS and it's good for the taxpayers.  As our world gets smarter, we need to make smarter policy decisions.  Communications today are so much more than speech. 
Thank you.  We take so much for granted, and technology can help give back some of life's basics that are stolen from those with ALS.

Please add yours.  We must be heard so that people with ALS are not silenced.  Thank you.

Click here for the link to submit your comments.  There are only 51 comments posted as of today.  Please spread the word so that there will be hundreds or thousands!  We must be heard.

Friday, November 14, 2014

Please, Let The Conversations Begin

The abstracts for the big annual ALS MND Symposium have been posted --

There's something for everyone interested in defeating ALS.

I hope that next year they will post those abstracts in a framework that would permit some public
conversations.  Every study is extremely important both to the authors and to all dealing with ALS.  How energizing it would be if patients, caregivers, authors, and others could comment and ask questions on the important "so what" that goes with every one of those research findings!

In the meantime, we have been advised that people can submit their questions to . That's an appreciated resource, but if we learned one thing about ALS and buckets of ice, it's that there are millions of people interested in ALS who are active in our world of social media, and many have invested heavily in these projects.  Please give them ways to comment, ask questions, and share the good news publicly.

Please, let the conversations begin.

Monday, November 10, 2014

Finally, A Pearl

There are a few people who get a case of ALS rage after helping a loved one through the difficult journey.  Nothing is easy with ALS, and it is an outrageous disease.

One such person fights relentlessly on behalf of those with ALS.  It's not her job.  It's her passion.  She is quiet about what she does.  She knows who she is.

She's the one who first pointed out the change in Medicare CMS administration of Speech Generating Devices that was a disaster for those with ALS.  When told by organizations that they had it covered, she kept pressing.  When told by professionals that these things take time, she pushed and prodded because time is the one thing that those with ALS don't have.  When one advocacy organization sloughed her off or offered feckless statements, she pressed others.  When told to stand down, she stood up and fought.

She was an irritant to many.  Too bad.  You can't be afraid to irritate a few on the way to fixing a
problem for those with ALS.

Last week, all of that irritation resulted in a pearl.

As she said to me last week, "It's so rare to have positive news."  Amen.  Finally people with ALS got some positive news with the announcement from Medicare CMS that the Speech Generating Device rules were being fixed.  Were it not for that one advocate who pushed and prodded and irritated and found individuals who could be effective, I'm sure we would still be in the bureaucratic morass.

Here's an article on the announcement.

And you can see that there is more to do.

Special thanks to this remarkable advocate and to Team Gleason and the Center for Medicare Advocacy and the other amazing irritants who joined forces.

It was a pearl of a week.  Finally.

Sunday, November 2, 2014

T Minus 29 Days And Counting

A solution regarding the Medicare CMS Speech Generating Device problem is due by December 1.

The following message is from The Center for Medicare Advocacy.  Note that they are considering action if CMS does not reach an acceptable decision.

Please help them know of people with ALS who have been affected, and please help spread the word.

Thank you.


We Need Your Speech Generating Device (SGD) Stories!

Thank you again for taking action to preserve the voices of those in need of Speech Generating Devices. Because of your advocacy, CMS is currently reviewing its SGD policies. We are hopeful that CMS will revise these policies to allow SGD users to keep their speech when they’re admitted into health care facilities and be able to use their SGDs to communicate with the outside world.
However, in the event CMS does not reach an acceptable decision about SGDs, we are considering action to help people who will lose access to SGD communications.
If you, or someone you know, have been harmed by these policies, please contact us at SGD@MedicareAdvocacy.orgWe are gathering information and data from anyone who wants to be heard, but we are particularly interested in those who can share stories because they have been affected by any of the following:
  • They experienced problems keeping their SGD since capped rental payments went into effect.
  • They experienced problems getting or keeping non-speech communication functions on their SGD in 2014.
  • They experienced problems getting or keeping environmental control functions on their SGD in 2014.
  • CMS denied their request for eye-tracking, or other SGD attachments in 2013 or 2014.
If you or someone you know can answer "yes" to any of the questions above, please contact us at SGD@MedicareAdvocacy.orgInclude the name of the SGD user, email address, phone number, street address, and a summary of the experience.  We will not use any of the information you share without your express permission.

Every story we receive helps us advocate to maintain the voices of our most vulnerable citizens. Thank you for your continued support of all those affected by CMS’s SGD policies.

Kathy Holt
Associate Director/Attorney
Center for Medicare Advocacy

For more information see:

Monday, October 27, 2014

"I Was Hungry, And You Formed A Committee"

They say that those will be the bitterest words at final judgement.

I think that, "I lost my tools to communicate, and you signed a letter," will be right up there, too.

Washington Post
ALS Patients Face Loss Of Medicare Coverage For Devices Used To Help Speech
"But unless CMS changes its interpretation by December, the delay doesn't make much difference, Wildman said."


Thursday, October 23, 2014

Let's Talk About Ebola, Placebos, and ALS

Ebola is dreadful and quickly fatal.  The only "promising" treatments are experimental and yet unproven.

Sound familiar?

There have been a number of articles on the experimental drugs that have been provided for Ebola patients in hopes that something might help.  It bothers the human conscience to hand placebos to these dying people in urgent need of help.  It bothers the human conscience  a lot.

"Experts Question Ethics of Placebo Drug Trials in Case of Ebola" 

"Use of Placebos in Ebola Drug Trials Unethical: Experts" 

"Issues Continue To Dog the Testing of Ebola Drugs and Vaccines"

Why aren't our human consciences bothered by the idea of handing a person with ALS, another  quickly fatal disease with no effective treatment, a placebo?

It's interesting.  What's the difference?  Why is a placebo so clearly an unacceptable tactic to Ebola clinicians and at the same time the "gold standard" of good research to ALS clinicians?

Let's talk.  Seriously, let's talk.  What's the diff?

Tuesday, October 14, 2014

You Can Get Better Information On Where To Park Your Car In Chicago Than Where To Find Good Care For ALS

This is an idea whose time is way overdue.  I write today because last week I heard a story that should be the last straw.  A man with ALS who must rely on a professional care facility somehow received a painful injury attributed to a simple piece of equipment.   That is terrible.   As someone trying to help him from afar said, "The bedsores should have been a clue."  And I wondered what will keep the next person with ALS from ending up in this same place.

It's 2014.

Who doesn't look up a hotel on TripAdvisor before making a reservation?

Who doesn't look up a restaurant on Yelp or UrbanSpoon to see what people think of the food and service?

Who doesn't read the reviews of items for sale on Amazon?

Who doesn't look for online ratings of roofers or painters or butchers or bakers or candlestick makers?

There are even online tools that help us find the safest and most economical parking lots when we drive to a strange city.

(And it's always helpful to see how restaurants and hotels and merchants address bad reviews.  Yes, they pay attention.)
Yet there are no good online tools for people dealing with ALS to help share experiences to rate a nursing home or equipment dealer or pharmacy or brace fitter or an online nutrition vendor or a VA clinic or even a clinical trial site.

Almost two decades ago we needed to find a new neck brace for Mom that would work with difficult ALS neck weakness.  I found that the ALS clinic staff had no ideas.  They referred  me to an occupational therapy department that was ALS-clueless.  I spent an entire day on the phone and driving around trying to find someone who could help.  Finally we found him.  There was an excellent fitter at a large brace company who had the right brace and got the job done.  At Mom's next clinic visit I told the neurologist about the hunt and the good result and the name of the young man and the company that was so helpful.  A month later I asked the doctor if he remembered where Mom got her neck brace and the name of the brace fitter.  He didn't.  Would the next caregiver spend a day off work looking for a neck brace and not be so fortunate as we were?  ALS was and still is the disease of reinventing wheels.

We need a Yelp-like tool where people can share their experiences with those who provide goods and services to those with ALS. If someone has a good experience, others should know about it.  If the experience is bad, why should the next person blindly stumble into a disaster?

Decisions that you have to make quickly when dealing with ALS are a matter of life and death.  They are also expensive, and it's hard to undo a bad one.  They are a lot more important than my travel or dining choices where I am so well-armed with information today.

Capturing and sharing experiences could be a tangible game-changer for people with ALS and their caregivers.  Finally.

Tuesday, October 7, 2014

Who Is Leading Us To Our Promontory Summit?

And what will we find when we get there?

Thanks to all who have mentioned the use and interest in a Global Unique (or Unifying depending on what you read) Identifier, GUID, for people in research studies. We now know that the NeuroBANK project that received a big bucket of funding from the ALSA windfall will be using GUIDs to key its data.

I found the information from the NIH National Database for Autism Research to be a most helpful  backgrounder on the concept --

The words that we are getting from ALS organizations and researchers are reassuring.  Since a wise parent once said, "The best predictor of future behavior is past behavior," we have questions about how this it will really happen.  How will will ALS researchers actually deliver new repositories of ALS data containing this common thread that can potentially relate data from multiple data silos?

Here are some questions --

  • Will the GUID generator be available to all researchers?
  • It appears that it's uncommon but possible for a GUID generator to come up with a previously assigned ID, and someone needs to referee and fix that problem.  Who is doing this small but important function for ALS studies? 
  • There won't be any proprietary pieces to getting and using GUIDs, will there?  The last thing in the world we need is for anyone to "own" or charge others for this simple collaborative piece of information.
  • When the personal information that is fed into the GUID generator is not available for a patient, it looks like a researcher can assign a temporary fake GUID.  Will there be a way to insure that fake GUIDs are consistent among studies or replaced quickly?  These pseudo-GUIDs could defeat the whole purpose.
  • Will people with ALS be told what their GUIDs are?  That would make a nice safety net to make sure that their subsequent studies are using the correct GUID, and maybe some of them will wear their GUIDs proudly as study participants.
  • Who is leading to make sure GUIDs in ALS research happen now?  We are at a critical juncture for ALS research and it's important that things move quickly and smartly, but somebody has to lead!   
If we don't get this right now, we'll pay the price forever. Studies are starting and expanding at this very moment. We're not asking organizations to share data today.  We're simply asking that they don't eliminate that possibility in the future by talking about GUIDs but not delivering now in a way that works for all researchers.

I'm sure that we'll have more questions.  Thanks to all who have enlightened us so far.

Saturday, October 4, 2014

Be Prepared

So far we have some "don't worry" and "we have it covered" and a "we'll encourage" reactions from ALS organizations regarding using a common, universal patient identifier among their newly funded information repositories.

I do worry, and I think that all involved can do more than encourage.

Will ALSA, each of it's IBC grantees, ALSTDI, AnswerALS, Project ALS, and others pledge to use a common, universal patient identifier in their IBC-funded information repositories?

We're not asking that they share data.  We're simply asking that they establish the groundwork so that if anyone would ever decide to share data, we would have a prayer that they would be meaningful. It's collaborate 101. Be prepared.

We've devalued so many words in the fight against ALS over the years -- "promising," "breakthrough," "exciting."  Please don't let "collaborate" be the next.

Thursday, October 2, 2014

NOW Is The Time To Get This Right! Collaborate.

The ALS Ice Bucket Challenge has primed the pump and we're starting to see some projects leave the starting gates.

At least three of the new ALS research initiatives involve people with ALS having their genomes mapped and a wealth of information assembled on each participant for researchers to mine.

Will it be business as usual?

Picture this --

Joe Doe has ALS.  He volunteers for Project Purple and is assigned a de-identified code 13579.  Joe leaves tissue and his genome is mapped. He leaves all kinds of health data that will be mined by researchers.  Joe gets a device that will measure his movements and the disease trajectory at periodic intervals.  Joe's tissue can be used as a lab model of ALS.  Project Purple even promises that other researchers will have access to its data.  Cool. 
Joe hears about Project Black and is assigned a code of 24680 when he is accepted into Project Black.  Joe leaves tissue and his genome is mapped.  He leaves all kinds of health data that will be mined by researchers, with many of the types of information different from what he gave Project Purple.  Joe will be contacted by Project Black researchers from time to time for more data and perhaps to be approached as a candidate for trials needing his variety of ALS, and again his tissue can become a lab model. Great. 
Wanting to give back as much as possible to the fight against ALS, Joe asks to be a part of Project Red that just got started in the U.S. as a result of some new funding.  Joe is given research patient  code ABCD12 and gives some tissue and his genome is mapped.  Joe becomes part of a huge data global bank of genetic mapping of people with ALS.  Awesome.

OK, we know that organizations and researchers love to say that they collaborate but they seldom really do.  Business as usual gives us three silos that don't even know whether Joe is in one, two, or three projects' gold mines of data.  And did they duplicate expensive screening processes that they could have shared?  Who knows?

Now's the time to put a very easy collaboration concept to good use.  Let's get this right. Now.

If all three projects would simply agree to share a research identifier for a person with ALS, think of the potential that we would have for the future.  If Joe were patient 265371 in all three projects, that would be one small step that would turn into a giant leap in the fight against ALS.  It would finally be possible to get inter-silo insights.

This is about as easy as collaboration gets.  This is about as valuable as collaboration gets.

We must get this right.  We must do it now.

Wednesday, October 1, 2014

We Learn From Questions AND Answers

Recently there have been some public online events on ALS organizations, their work, and the
#ALSicebucketchallenge windfall.  All have welcomed questions.

All have left my questions submitted online unasked publicly and unanswered.

While there is never enough time to get to every question during these events, there is surely time afterward to answer the unanswered questions in a public milieu.

We learn from questions and answers.

Burying questions isn't a healthy sign.

Friday, September 26, 2014

There Are Better Ways To Keep Track Of A Portfolio

For years we've asked for an orderly, searchable portfolio of ALS research grants.  Trying to keep track of press releases with no accounting of outcomes is a poor way for donors and people with ALS to understand and evaluate the research portfolio.

We recently got a Word document with new research grants.

There are better ways, and they're not rocket science.  Try this.

Search and find.  Leave fields empty to find all.

And wouldn't it be nice if the projects were soon to be filled in with grant amounts and outcomes?

Online Database by Caspio

Click here to load this Caspio Online Database.