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.

Saturday, April 29, 2017


Obviously I'm interested in ALS advocacy issues.

I read a lot and try to study them.  Our voices are important as are our ideas, and I believe that we can make a positive difference.

Every year I go the the ALSA ALS Advocacy Conference and Day on Capitol Hill.  I pay the fee and pay my own expenses.

In an email yesterday, I received a nice invitation:

I requested to join on facebook and was accepted. Nice.  I liked the idea of this new dimension to Advocacy Day preparation and discussion.

This morning I submitted four items to post.  Evidently, everything is moderated, so all were pending moderation when I signed off of facebook this morning.

Thank you for providing this area.  It’s a nice addition to let us all discuss substance that has been difficult with the format of the conference over the past few years.  We are all more than just our stories and I feel strongly that policies and our voices and thoughts matter. 
I have three topics related to the Registry that I will post and I hope they will be part of the discussion.
Thank you.

1/3  Last summer’s annual Registry meeting at the CDC was blacked out.  All we have is an “executive summary” report that is not at all substantive.  When I inquired months ago about getting a detailed report, Dr. Mehta said that there would be a recording of the proceedings posted.  When I inquired of ATSDR about an ETA for the detailed proceedings, I got the unhelpful response, “When it’s ready.”
We are being told to request another $10,000,000 for the project without any substance on who was invited to that meeting, what was discussed, what problems were (or were not) addressed, and what direction the leadership is taking the project.   How can we be responsible advocates without having that information?

2/3  We have never seen a businesslike accounting of how ATSDR spends $10,000,000 per year on the Registry.  One year after we pressed the issue, we got a vague set of percentages in some Powerpoint pie charts at the Advocacy Conference.  It’s not unreasonable for us advocates to know how that budget was or will be spent, in dollar categories (like any good budget analyst would supply).
As a friend once said in a motherly way, “I’m not giving you another $10,000,000 until you tell me what you did with the last $10,000,000.”  

3/3  What is the cumulative total that has been appropriated for the Registry over the many years, starting with the so-called building-block appropriations that happened in the years before the 2008 passage of the ALS Registry Act. 
We advocates can’t make good cost/value assessments without having a good handle on the costs. 

This evening I find myself locked out of the group.  I can only assume that my discussion points were never posted.

Perhaps the group is to stick to dinner plans and not substance?

Why, oh, why would you stifle polite, informed discussion on something this important?


Wednesday, April 26, 2017

ColLABorate, Please, Now!

A young father with ALS is in this clinical trial, on the drug protocol for more than 60 days --

He actually has experienced clinically measurable improvements.  That's huge.  Normally an ALS trial success consists of getting worse less quickly than before.  Here, we're talking improvements.

I asked him if he knew if anyone else on the drug was having similar results.  Unfortunately he was the only one he knew of on the drug protocol.  NIH had a very small sample set and found very few HERV K positive people with ALS.

Isn't this the promise of precision medicine?  To test the right therapy on the right patients?  Why aren't we finding the right patients for this clinical trial with some urgency?

There are numerous, separate precision medicine projects in the ALS space.  There are even more repositories of tissue of people with ALS.  Cmon, folks, let's screen for HERV K and find some candidates and get this trial filled and completed quickly. Now!

It's not that hard if you really mean it when you say you collaborate.

Saturday, April 22, 2017

We Need A Fact-Checker

This week I listened to several presentations on ALS.

Over many frustrating years in the fight against ALS, I've learned to listen for the devilish details. Sometimes I think we need an ALS fact-checker.

CDC Grand Rounds

First up on Tuesday was the CDC presentation for their Grand Rounds.  The topic was of particular interest to me -- "National Amyotrophic Lateral Sclerosis (ALS) Registry - Impact, Challenges, and Future Directions."

Early in the presentation there was an assertion of the applicability of this project to other conditions. But how?  The basis of using their algorithm to locate cases of ALS was based on the quick entry of people with ALS into government health coverage systems such as Medicare and the VA.  That doesn't happen with other conditions. Sure, this Registry has a mother lode of lessons learned, but how would another disease find value in its basic design?

We heard that the Registry speeds up clinical trial recruitment and expands ethnic and geographic diversity (without any data to back up that claim).  Sending out emails to the patients you located on a small fraction of enrolling trials doesn't necessarily advance any of those noble goals (and might actually have the opposite effect on trial diversity).  Who knows?

And there was the importance of the  "partners."  It seems to me that if there is any flow of money to or from any "partners," that should be transparent.  There are some "partners" in this slide who have been paid millions of dollars in this project. And exactly for what?

There was a mention that the large national organizations cited in the partner list represent a majority of people with ALS in the US.  How do we know that?

There was a vague reference that the Registry is providing biospecimens to scientists. How many scientists are involved and how many patients' specimens have actually been distributed?

Did I hear correctly that there were 60,000 risk factor questionnaires completed for 17 separate surveys (giving an average of 3529 responses per survey)?

A woman in the audience asked what percentage of people in the US with ALS are actually in the Registry.  That most important question wasn't answered.  We got a tapdance about the challenges of completeness and ongoing statistical studies and local studies and that you'll never find them all.  But we ask again, what percentage of people in the US with ALS are actually in the Registry?

The Registry continues to make generalizations about ALS being white guys' disease based on the people with ALS who are in the Registry.  What do the people with ALS who are not in the Registry look like?  And how many are there?

One presenter who has been involved in the project annual meetings made an off-the-cuff reference to ALS as a disease of 30,000 people.  Wait.  What happened to the 15,908 number that the Registry published?

And we had a lot of interesting information from one of the clinicians who has worked with the CDC on the Registry.  He had data citations in his presentation, none of which seemed to come from the CDC Registry. Oh, well.

ALS Research Update

Thursday evening there was an ALS research update directed to some college students.  Naturally it started with some basics about ALS. Take a look at the slide on the left.   But wait, what does "affects" mean?  And "two cases per 100,000?"  Is that prevalence?  How does that jibe with the "5 cases per 100,000" on the CDC's slide?  Are global numbers that much different from US numbers?

We again saw a lot of interesting data, but none of the citations seemed to involve use of data from the obscenely expensive US CDC Registry.

Families dealing with ALS don't have time to deal with devilish details.  And they shouldn't have to.   These mixed and vague messages are not acceptable in a world where data and science are so important.  And when precious resources are poured into projects, we need to get the facts right and make sure that their value is in proportion to their cost.

Wednesday, April 5, 2017

"I'm Smarter Than You Think I Am"

A few months ago a television news show had a feature on a wonderful teacher.  Each year she had an assignment for every student -- Write down something about yourself that I don't know that I should know.

The responses were at the root of her understanding her students, and they told her of family situations or interests or hobbies or pain or joy.

And there was one child who wrote an especially important message to her -- "I'm smarter than you think I am."


On an ALS message board I just saw a link to a presentation given by a respected, world-famous
ALS clinical researcher on the importance of clinical trials.  She prefaced her message by talking about the real difficulties of doing clinical research in a world of uncurated information on the internet and fake news.  She used terms like "anti-science," "poor education," "ignorance fighting proper science."

And all I wanted to say was, "We're smarter than you think we are."

Saturday, April 1, 2017

In The Middle Of A Roundabout

"The Patient Is At The Center Of Everything We Do"

But are you really patient-centric?

The process was clever enough.
ALS Clinic Neurologist: You need to see Dr. Specialist.  Debbie will make an appointment for you on your way out. 
Caregiver to Debbie: May I make the appointment with Dr. Specialist, please?  My schedule is a little difficult, sometimes.  I have a meeting at work on Thursdays and Monday mornings are out and my loved one with ALS can't be at an appointment before 10 am, and after 2 pm he gets really weak. 
Debbie:  We'll just call them to make a first-available appointment.  That way Dr. Specialist knows that you are our referral.  Then when you get the appointment notice, you can call them and change it to whenever you want.

Clever?  Yes.

Patient-centric?  Hardly.

The patient in this case can't communicate easily.  The caregiver is trying to hold down some semblance of employment while being a more-than-full-time caregiver.  Both are dealing with a demanding disease.  Both have very reasonable preferences for all appointments that should be easily accommodated without wasted time and calls.

Sometimes a healthcare delivery system sincerely believes that it is being patient-centric, but to the patients, it's like being at the center of a roundabout with healthcare providers going around them in circles to their own destinations.