ALS Advocacy

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

Friday, December 30, 2016

Which Dots Did They Collect?

For over a decade, some of us have worked hard to get a good census of people with ALS.

My theme was, "We'll never connect the dots if we don't start collecting the dots."  I started the website www.alscounts.com in 2008 to help make the point.

And I worked hard with others to enable the ALS Association and the CDC to deliver what was to have been a population-based US Registry.

So many years and over $70,000,000 later, here we are.  

But did it count all the noses?  No.
Does it know how many noses it missed?  No.
Does it know which ones it missed?  No.
Does it make broad demographic generalizations based on the ones it did locate?  Yes.

Which dots did they collect?
Which dots are missing?
Is the epidemiology of the missed dots the same as of the located dots?

As an ALS neuroscientist so aptly said to me, "We don't know what we don't know."

And that's a problem -- a very expensive problem.

Tuesday, December 20, 2016

We Got Big Fluffy Slippers For Christmas

A long-awaited report has finally emerged from this year's blacked out CDC ALS Registry Annual meeting: https://wwwn.cdc.gov/als/Download/2016%20ALS%20Annual%20Meeting%20Summary%20Report%20Executive%20Summary.pdf

Please take a look.  It's easy reading.

It's a beginner's primer on the basics of the CDC's ALS Registry project.  It contains no specifics on any critical issues that may have been discussed at that meeting. There is no information on any decisions or advice from the participants.  Executive Summary?  Hardly.

We deserve specifics. We deserve metrics. We deserve accountability. We deserve transparency. We deserve results.

We don't deserve fluff.

Wednesday, December 14, 2016

There Are Big Lessons In Small Places

Last week was the big 27th International Symposium on ALS/MND in Dublin.  It is an intense few days.  There were 1100 people registered, mostly neuroscientists, clinical specialists, organizational employees, and a scant number of unaffiliated people with ALS and caregivers.

I learned.  I learned a lot there.

Less than a five-minute walk from the gorgeous Dublin Convention Centre on the Liffey was the small, old church of Saint Laurence O'Toole.

I learned there, too, in the shadow of the grand symposium.

When I arrived at the quiet little church, there were a few elderly folks already waiting for Mass to
start.  A couple of rolling walkers were stored behind their pews. Some younger adults trickled in.  Finally a line of very young lads and their teachers streamed in and sat in the front pews. The priest welcomed the boys studying for their first communions and prepared to start Mass.

I was relieved.  The homily wouldn't be overly long and wouldn't be all about Saint Augustine's or Kierkegaard's arcane philosophy lessons.  This day we were going to be geared for the kids.  It's not that I can't grasp complicated philosophy and theology.  It's that sometimes we need to take the shortest, clearest route from theory to practice, and that's the route that children's liturgies usually take.

And it takes a smart person to distill complicated lessons into those clear, direct paths.

As Mass began, Father also welcomed the shut-ins who were watching and praying with us on the internet.  This little church had a web stream for those who couldn't be there.

Yes, I learned at a tiny, old church in the shadow of the grand symposium of esteemed and ambitious scientists.

We could all learn.

Saturday, December 3, 2016

For the "Ask the Experts" Session Prior To the 27th International ALS MND Symposium

1. At my last count, there are at least a half dozen completely separate "precision medicine" projects for people with ALS.
     a. Has anyone actually established protocols and shared data between any of these silos?
     b. Is anyone concerned about the duplication of resources and infrastructure?

2. Are there any specific novel clinical trial design features that you expect to see implemented in the next 12 months as a result of the Airlie House guidelines or the ALSA FDA draft guidance work?

Thank you.

__________________________

Information about "Ask the Experts" can be found at
http://www.mndassociation.org/research/international-symposium/symposium-live-2016/ask-the-experts-session/

We are grateful for the live stream this year and hope that more of the symposium activities will be live streamed in the future for those who are interested in the newest and best science but cannot travel.



Thursday, December 1, 2016

Stop, Look, Listen!


After a lively day of sausage-making, a 21st Century Cures Act passed the House yesterday and now it's the Senate's turn to make some things happen. I think that this can provide some good opportunities to advance the fight against ALS.  Increased NIH funding, FDA resources, and the Precision Medicine Initiative are all very important in my opinion.

But one item gave me pause when I saw the list of ingredients left in the House version:


Sure, a neurological disease surveillance system makes ultimate sense when there are so many devastating and unsolved neurological diseases.  But wait... "a neurological disease surveillance system coordinated by the CDC..." Been there, done that, and it has not gone well.

Please, stop, look, and listen to us.  We have an opportunity to do something great or to be part of a train wreck.

Today we have a tremendously expensive ALS Registry -- a neurological disease surveillance system coordinated by the CDC.  It has failed to live up to its potential, and it has cost us taxpayers over $70 million to date.  It has let us down on many levels.  It has generated scant reports whose numbers nobody wants to  use.  It has been fraught with scope creep and gingerbread of questionable value and unrelated to delivering accurate and timely disease surveillance.  And perhaps the most revealing problem of all has been its lack of transparency.  This year the invitation-only annual meeting was blacked out and we have not see a  transcript yet.  The CDC doesn't not seem to want to talk about it except to the voluntary health agencies that lobby annually for its funding (and in a cozy arrangement receive hundreds of thousands of dollars in no-bid contracts).

So please, stop, look, and listen to us.  Let's fix the CDC ALS Registry problem and move forward with something well-designed, nimble, transparent, and focused to provide valuable information on all neurological diseases.  Please learn from our journey and don't be part of a 21st Century train wreck.