"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
http://www.washingtonpost.com/politics/als-patients-face-loss-of-medicare-coverage-for-devices-used-to-help-speech/2014/10/26/bb8f07ac-5d40-11e4-8b9e-2ccdac31a031_story.html

"But unless CMS changes its interpretation by December, the delay doesn't make much difference, Wildman said."

So?

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"
http://www.reuters.com/article/2014/10/13/us-health-ebola-drugtrials-idUSKCN0I20W520141013 

"Use of Placebos in Ebola Drug Trials Unethical: Experts"
http://medicalxpress.com/news/2014-10-placebos-ebola-drug-trials-unethical.html 

"Issues Continue To Dog the Testing of Ebola Drugs and Vaccines"
http://news.sciencemag.org/health/2014/10/issues-continue-dog-testing-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?

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.

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.

http://www.neurology.org/content/82/10_Supplement/S46.007

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

https://ndar.nih.gov/ndarpublicweb/standards.go

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.

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.

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.

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.