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.

Wednesday, September 25, 2013

It's Time To Dump The "Pick A Leader And Get In Line" Fight Against ALS

Occasionally a big light bulb turns on in my dim mind.  This week I experienced a lot of lumens as I worked in an online group trying to plant some new seeds in the fight against ALS.

People waste a lot of time and energy vying for so-called "leadership" in this fight.

When people experience ALS, it is overwhelming.  You have to pick your battles and you have to learn to trust people who can help you.   You also have the urge to make a difference.  It's a delicate equation -- trying to find your leader who has blazed trails before and trying to help the next person.

Add the whole instinct to survive which drives you to those leaders with "hope" written on their backs.

And there is a constant stream of rookie recruits.

And the disease awareness is so poor and the disease demands are so high that resources are scarce.


For years we've operated under a pick-your-leader-and-march mentality.  It hasn't exactly worked well.  Organizations and individuals vie for followers, and people in lines aren't exactly using their creative juices well.

We need to smarten up.  We need to recognize strengths and weaknesses in organizations and people.  We need to break up the lines and learn how gang up on problems and not each other.  We need to stop meandering behind leaders.

Oh, and those who think they were born to lead us need to stop spinning stories to attract followers.  Let the data speak.  We'll figure out when and where and whether to follow today.

And sometimes the one who appears to be the least among us at the back of that line has the idea or the common sense that we really need to follow today.

Tuesday, September 24, 2013

Let's Have The Most Painful Meeting Ever

Since this seems to be the year of meetings to defeat ALS, maybe this concept of two serial meetings would actually move us forward together.

Let's start with a virtual room full of people with ALS, caregivers, some retired pharmaceutical executives, and some retired FDA officials.

Why the retirees?  They know their businesses, yet they have no horses in this race.  They don't have to worry about walking on eggshells around people who are dying.  They can be brutally honest.  They can be informative and candid.

Stakeholders need to stop trying to assume that they know what the others want or need.  They need to talk.

Meeting 1:

OK, let the first meeting begin.  Let's talk about investment in ALS drug development.  Let's talk about the business of bringing ALS drugs to market.  Let's talk about the short-term and long-term financial incentives.  Let's talk about the approval process as it really is.  Skip the Ph I,I,III basics. We hear those over and over but seldom get past them.  Let's talk about breakthrough status and expanded access programs and markets for drugs. Let's talk about how drugs are paid for. Let's talk about how a program that is good for a rare cancer drug may be not so good for an ALS drug.  Let's hear the heartburn that each special program brings to each person in that room.  Let's learn from one another.  Let's tell it like it is.

That's a pretty painful meeting for all parties, but it would be tremendously constructive.  Today patients dream dreams about programs that they think could bring drugs to them.  Are they even viable?  Are they conducive to industry investment in ALS?  Are they products of an FDA that today doesn't have good "special" programs designed for an ALS?  Are there paths we've not considered.  What do patients really want?  What do drug developers really want?  What do investors want, today and tomorrow?  What does the FDA need? What does everyone need?  

All the cards and reality need to be on the table.  We need to be candid with one another.  That's a constructive, uncomfortable, painful meeting that has never happened before.

Meeting 2:

Ah, now we're ready to design some solutions.  Now that we've stopped assuming and mind-reading, we can start the creative process.  Let's break through the maze and find a path to get therapy candidates to patients more quickly.  Let's not try to bend existing programs to fit ALS if they don't really serve all the stakeholders well.  Let's move the fight forward creatively on solid ground.


We've had far too many variations of Meeting 2 already this year.  We need a Meeting 1.


Saturday, September 21, 2013

Just The Facts, Please

History is an essential asset, yet in the fight against ALS, we do a very poor job of retaining it.

The nature of ALS is that the people dealing with it don't have memories of what happened five or ten or twenty years ago.  They are slapped with a diagnosis and are largely on their own to find information and to figure out whom to trust.

We need to do a better job of retaining what really happened two or five or ten or twenty years ago.  We need to make the fact-finding easier for those with ALS who seek a ray of hope.

Occasionally those of us who have been paying attention to matters ALS for more than a decade see or hear someone spouting revisionist history.  It may be innocent, or it may be manipulative.  In either case, it's wrong and it does not help the fight move forward.

Today's technology could give us an excellent electronic archive of historical facts.  It's time for organizations and individuals to contribute facts lest we forget or are led astray or repeat the mistakes of the past.

Saturday, September 14, 2013

Pick Your Game Of Musical Chairs

The same number of people will be added to each game continuously.  There are a lot more chairs in the blue game than in the red.

The undertaker removes people who don't have chairs when the music stops.

Which game would you rather join?


This shows the flaw in using prevalence (the number of chairs) as the measure of a disease's impact.

The small number of ALS chairs doesn't mean that it's a small problem.  It means that it's a huge problem!


Saturday, September 7, 2013

Aren't There Ever Any Scouts At The Table During Trial Design?

Envision a person with ALS who has volunteered for a clinical trial -- a trial designed in much the same way that researchers have traditionally designed trials for rashes or cholesterol control or male pattern baldness.  There is trial period with the drug or placebo, and when that's over, everyone waits while results will be evaluated and the drug company will decide if and how to proceed to another trial phase.

For ALS trials, it seems like the trial designers never seriously consider what happens to their trial volunteers if this stuff really works.  It's not like they'll just have to scratch an itch or watch their diets or comb over while they wait to see what's next.  They decline quickly. They die.

People with ALS who have volunteered for trials and who perceive improvements during the trials are left hanging out to dry.  This all could be avoided with some creative trial design that gives them options to continue on the drug (or the placebo... if it works, they really don't care) before they sign on the dotted line.

Instead we have traditional trial designs and the subsequent cry for Expanded Access Programs after the fact.  Perhaps we should get in front of the problem and have trial designers, including patients, talk about what happens to trial volunteers if this stuff really works.  Be prepared.




Wednesday, September 4, 2013

Stuck In The Middle WIth You

The organizational fight against ALS loves the middle.

At the recent Team Gleason Summit or February's FDA Hearing, who was dreaming big, daring to change the basic ways that business is done to fight ALS?

Who was dreaming small, willing to step up and deliver some simple basic changes that would tear down silos that make the armies fighting ALS so inefficient?

The battlefield against ALS is so comfy in that middle where "promising" projects become expensive and big and not game-changing... and the deliverables are poorly measured and nobody is really held accountable.

Pffft.