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 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.

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