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.

Sunday, September 30, 2012

Please Listen AND Hear AND Deliver

We have a lot of organizations and institutions that say that they are listening, especially to the most important stakeholders in the fight against ALS -- those with the disease.  Perhaps they are listening, but are they hearing?  Are the messages too difficult for them to handle?  Are they willing and able to deliver once they finish with the listening?

Here are two examples that make me wonder.

1. A man with ALS made some specific suggestions on how to improve the U.S. National ALS Registry.  Click here to see his presentation.  I don't agree with everything he suggested, but surely he identified some of the root problems and some simple solutions.  Since that presentation, we see a new iPad app accessible on the Registry home page for finding ALS clinics and resources.  The patient simply suggested simplification of that page.  Oh, do we recall correctly that ALS clinics were provided with non-Apple tablet devices to help patients with Registry enrollent at clinics. So much for consistency.  The stakeholder with ALS who made the helpful recommendations has since died.  And so it goes.

2. Several women with ALS who had participated in the Dexpramipexole phase II trial have reported being spurned from continuation in the open-label phase that they had counted on.  One's rejection had been because of an inability to travel to the trial site, yet the open-label trial description clearly has options for patients who can't travel.  Her travel rejection is since being reconsidered, but why do patients constantly have to step up and fight a system when they are already fighting a beast of a disease?  And so it goes.

Listening, eh?  Please give us reasons to be more confident that institutions and organizations are listening, hearing, and can deliver efficiently. Please.

Sunday, September 16, 2012

In What League Would This Quarterback Keep His Job?

Imagine a quarterback who constantly throws the ball five yards behind the receiver.  The receivers run their routes and are on their marks despite being mauled by a vicious opponent.  Still the quarterback delivers too little, too late.

Would this be tolerated in the NFL? Hardly. Fans and owners expect more of players and coaches.
How about the NCAA?  Nope.  Alumni and subway alumni and administrators expect coaches to coach and players to deliver.
Pop Warner?  Even with the kids, the expectation is for improvement (or perhaps a change to a more suitable position).

What about the "ALS League?"

Healthcare professionals constantly throw the ball five yards behind their ALS receivers.  Sometimes the play clock runs out before they even get the ball snapped. Our quarterbacks stay in the game and collect their paychecks even if they're delivering too little, too late to their patients and caregivers.

We need more accountability to raise their game... or perhaps find them more suitable positions.

Thursday, September 13, 2012

Ignore And Delay And It Will Go Away

Please read about one person with ALS and her experience with a clinical trial, an IRB, the FDA, and a very prominent ALS clinic --

http://barbarabrenner.net/?p=699


If you talk to some former ALS caregivers who have been around awhile, they'll tell you that a lot of people and institutions seem to have an unspoken "ignore and delay and it will go away" approach to solving problems.  You see, people with ALS have a very short window of life. Their problems will soon pass.  Any ruckus they raise will soon pass.

Or will it?







Friday, September 7, 2012

Do We Need A Success Drill?

Our grade school had a principal who knew the right way to hold a fire drill.
  • Procedures were clear
  • Drills were always a surprise
  • They happened randomly throughout the year
  • People moved quickly and quietly
  • At the end of each drill, the process was evaluated and improved with input from students, teachers, the principal, and the fire prevention officer
She was the ultimate Six Sigma Black Belt before anybody ever heard of Six Sigma.

It was only when I got to high school and college and the world of office buildings and hotels that I appreciated the effectiveness and value of Sister Mary Vigil's fire drills.  Theatrical drills that are staged don't really condition you for the real fire, and they certainly don't provide the information that you need to make the traffic patterns better or to remove obstacles from the paths.  Practice trumps theory when it comes to human motion.

How will human motion work when there is finally some success against ALS in a lab or clinic setting?  Do we have some continuous evaluation and improvement of the paths and barriers between a scientific success and our patients?  Isn't it smart to fix some doors and sidewalks and roads before we discover that they are blocking the evacuation from the world of ALS?

Lots of organizations talk about their plans and leading the way, but we need to see the practical process for a successful treatment. We need to walk down the fire escape to see what is confusing or blocking the way so that we can fix the path now.  Removing those barriers will involve a lot of people and organizations, but the time to face them is before the fire.  Just as some businesses or hotels never expected the big fire and didn't have effective processes in place for people to get out, is the ALS cause one that has never prepared sufficiently for the surprise of a successful treatment?

Do we really know what is on the other side of this door?

Wednesday, September 5, 2012

Nero Fiddles


This is actual information from a living human being -- a beloved father, husband, son, brother, friend.  Over the years he has posted data religiously at www.patientslikeme.com so that others might learn from his experiences.

The FRS and FVC measure the path of his ALS.

In the treatments, you can see that he participated in a Phase 2 clinical trial - Neuraltus NP001.

Once his trial treatment was completed, the waiting began while biomarkers were measured and while other patients finished their trial treatments and while the data were evaluated.  The waiting continues.

Doesn't this bother anyone else enough to change the way we handle clinical trials and risk for people with ALS?  Do we not owe clinical trial subjects more than gratitude?