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.

Thursday, June 29, 2017

Dear Contractors, Please Come Prepared

Dear MDA, ALS Association, Les Turner ALS Foundation,

It's nearing time for the CDC's Annual ALS Registry Meeting (at least we think it is).  You know the date for sure because you are the only organizations allowed to nominate participants for the meeting.

You are also well paid as education-and-outreach contractors on the project.

Please prepare some very simple data for the meeting so that we all can measure success.
1. How many people living with ALS were in your clinic system at the end of 2016?
2. How many new people with ALS entered your clinic system during 2016?
3. How many of those new people with ALS in your clinic system also self-enrolled in the CDC's ALS Registry in 2016?
That's it.  Three simple measures will make that meeting a lot more productive.  Please be prepared.

Thank you.

Sincerely,

@ALSadvocacy

p.s. For inquiring minds, the most recent annual payments to the three per usaspending.gov were:
Les Turner ALS Foundation $100,521
ALS Association $376,852
MDA $548,658

Tuesday, June 27, 2017

Watch You Don't Break Your Arm Patting Yourself On The Back

That was Dad's standard line whenever we got braggy at the dinner table or a sporting event or a school activity.  Hubris was never a problem at our dinner table.  Humility was valued by my parents' generation, and we learned lessons about the important distinction between arrogance and self-confidence.

Every time I go to an ALS fundraising event or a scientific meeting, Dad's words go through my mind.

"We have failed" does not make a good fundraising slogan or justification for the next big research grant.  ALS events are celebrations of accomplishments that will give the public the confidence to invest.  But, Dad's words ring true to me when I hear all the "accomplishments" and "promise."

We have failed.  Individually and collectively, we have failed.  Sure, progress has been made, but people are still dying from ALS, and the more we discover, the more complicated we realize the problem is.

The first time I went to an ALS walk many years ago, I followed a man who clearly had ALS.  He struggled walking, but he walked.  As I trailed him, I wondered what he had been doing a year before.  Playing golf?  Skiing?  Running marathons?  And I thought that it was unlikely that he would be at the walk the next year.  That's a stark, ugly reality of ALS.  That gentleman walking directly in front of me helped me understand that we really had failed him despite all the fundraising and balloons.

Some folks on the forum at als.net have been giving me grief about my encouraging people with ALS to apply for the fellowships for the International ALS MND Symposium in December.  What difference does the symposium really make?  What have all those scientific accomplishments that are celebrated every year really achieved?  I understand what my friends there are saying.  I understand 100%.

Sometimes it seems like scientists need my Dad to tell them to watch they don't break their arms patting themselves on the back.

People living the reality of ALS at the symposium could convey that, too, simply by their presence and participation.  Their reality in the midst of the celebrating scientists could add a much-needed dose of realism and humility and perspective.

I think Dad would encourage them to be there.

https://alsmndpatientfellows.wordpress.com






Sunday, June 25, 2017

This Small Step Is A Giant Leap

Over the years I've been critical of the International ALS MND Symposium not embracing patient attendance and participation.  Today, we see a small step forward in a direction to fix that problem.

The Symposium is an old-school scientific meeting.  The ALS topics discussed are broad and very
deep.  https://www.mndassociation.org/research/international-symposium/

In the past, if anyone with ALS has chosen to attend, it has required payment of the substantial registration fee (plus a separate fee for an accompanying caregiver).  Add travel and hotel expenses, and there has been a large financial barrier between people with ALS and the Symposium.

This year there is a new program to help mitigate some of the financial barriers --


This is a giant leap toward having people with ALS in the midst of some important discussions.

Anyone with ALS who is interested in the science now has a chance to be in the center of the newest science in Boston in December.  This is a big meeting typically filled with big players in ALS research and with big announcements.

People with ALS are notoriously bright and high achievers.  Finally, a step has been taken that can make them part of the scientific discourse.

Please apply if you have ALS and are interested in the science.

Saturday, June 3, 2017

The Race To Eureka Needs To Start Now

And people with ALS need to be in charge of the race.

20-20 Hindsight

  • How helpful would it have been if we could have seen individual trajectories for people with ALS who have used riluzole over a number of years?
  • How helpful would it have been if we could have observed data on Americans who have been using generic edaravone with or without riluzole for the past several years?
  • How helpful would it have been to have access to data on ethnicities and genetics and experiences of people with ALS who try any number of things in their journeys?

That's water over the dam, but the massive information loss need not continue. There are a lot of good reasons to start the race to Eureka now!

To Get To Eureka, You Need Data

The promise of precision medicine is to know which potential treatments will work for which people.

And in recent days with the approval of a new ALS treatment, we have seen clinicians suggest that combinations of drugs may hold the most promise for effective treatment for people with ALS.

And there are more promising (yet not blockbusting) drug candidates that may be approved in the next several years.

We're in a world of promise without the data to take us to true Eureka moments that we need.

You Own Your Own Data

There has never been a better time in history to get ALS data out in the sunshine for all kinds of smart people to look for insights.  People with ALS own their own data, the most powerful factor in the race to Eureka.

The old public lithium spreadsheet proved that people with ALS can contribute data that will tell a story in a hurry.  We have better tools now, e.g. www.patientslikeme.com , but the tools only work if people contribute their precious data.

There are several individual patient tracking portals (e.g. ALSTDI PMP, AnswerALS) that could release selected data to a common public place like patientslikeme.com to keep people from ALS from entering data in several portals.

And there are patient organizations that could encourage information sharing but have traditionally avoided it like the plague for some reason.

Who really has the incentives and motivation to figure out what specific subgroups of people respond to an ALS treatment? Think about that. It's the people with ALS.

Do you want to go to Eureka?  The race can start now with people with ALS in command.