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, May 31, 2018

Here's The Deal

People with ALS hear that all the time.  Here's the deal.  Sign here.

Clinical trials have a deal.  Van purchases have a deal.  Homecare companies have a deal.  Power wheelchairs have a deal.  Pharmacies have a deal.  Hospitals have a deal.  The ramp for the front door has a deal.  The fundraiser even has a deal.

But it's never really a deal.  It's always THEIR deal.  They write the rules.  Their lawyers write the contracts. Take it or leave it.  A person with ALS has no leverage.  You sign.

The patient is at the center of everything they do except the deal.

Yesterday with the signing of Right to Try legislation in the US, we're entering the era of some important new deals.  Who is going to look out for people with ALS in these deals?

Who is going to make sure that dying people aren't handed more papers to sign that are a bad deal for them and others with ALS?

  • Who is actually going to make clinical trial deals better so that people who volunteer for clinical research aren't forced into a pay-to-play arrangement to continue an experimental therapy?  
  • Who is going to make sure that people who got stuck with placebo in a trial are at least given a free try of the therapy as part of the trial deal without having to resort to Right to Try access?
  • Who is going to keep Right to Try deals from imposing secrecy that will attempt to stifle patient rights to have and use and share their own data.
  • Who is going to make sure that Right to Try deals and monetary exchanges are transparent?
  • Who is going to stand up for the patients and make sure that they get some leverage in a deal and are not just handed somebody else's idea of a deal

Patients asserted the right to try.  They have a right to a say in the deal, too.

Code of Conduct anyone?  Now is the time.








Monday, May 28, 2018

The Corn Maze We Call ALS


I've never understood why someone would pay to enter a corn maze. Why would you want to become totally disoriented, having to learn the hard way at each decision point, receiving no clues along the way from people who navigated before you, and never sure how or when or where you will finish?  Oh, and then the next person gets to be as confused as you.  Fun?  Not for me.

Perhaps it's because I've already done the ultimate corn maze -- ALS caregiving.

Families dealing with ALS work hard and do research and discover the same things that other families discovered two years ago or two months ago or two hours ago.  They waste precious time going down disorienting wrong paths.  Sometimes they later find that others knew things that would have saved that them from the dead ends.

And the maze gets even worse when we have multiple places to look for sometimes conflicting information. Think about a registry that notifies people that they should also register in a different registry. 

Last week with the passage of the federal Right to Try legislation,, a new maze was dreadfully apparent.  Families were calling support organizations and drug companies and individuals, trying to find out what they might be able to try and how soon they could try it. Who knew fact from rumor?  Who got a real answer from someone?

You see, there is urgency here. People are dying. 

We need a central clearing house for objective, accurate, and timely information on the three paths that people with ALS now have to access experimental treatments, and we need it immediately.

For every candidate drug (having already passed a Phase 1 trial and currently in a Phase 2 or 3 trial), families need to know at a minimum --

  • Is that drug developer currently seeking clinical trial volunteers?
  • Will the company consider an Expanded Access Program?
  • Will the company consider Right to Try access?

Think of how efficient it would be for families dealing with ALS, support people, healthcare professionals, and therapy developers to know even these basics.

Maybe start with a simple public spreadsheet.  Grow to a good database as it gets traction. Add transparent information about how programs work (criteria to participate, locations, costs), and we would actually have refreshingly efficient information for people to deal with ALS and to take advantage of appropriate paths to experimental treatments.

Until then, step right up to the entrance of yet another corn maze that will waste the precious time of everyone involved in a disease that makes you pay a terrible price for wasting time.


Thursday, May 24, 2018

Give Them A Moment At The Controls

The day a family member gets an ALS diagnosis, you become a passenger on a terrible, out-of-control, speeding, downhill train.  It is a nightmare ride and there is little you can do to change what is happening.  Somebody is in the locomotive doing ineffective things, and the whole family is stuck riding in a passenger car.

"Try" is a word that has a different meaning when you are stuck in coach.  It's not particularly the transitive verb that the rest of the world uses -- try escargot, try a Chevy, try a new tennis racket, try asparagus.  It's simply that you want to try.  You want to take a swing at life.  You want to take a nasty swipe at the disease that put you on this train.  You want a moment at the controls.

Regardless of where you are on the Right to Try opinion spectrum, we all owe it to every family dealing with ALS to give them a better journey.  Yesterday there was pandemonium with families trying to decipher what concrete options may have opened up to them.  They don't need more pandemonium.  They need help.

ALS advocates and advocacy organizations owe them that help.

This isn't a moment for "I told you so."  This isn't a moment to conveniently hide behind agnosticism.  This is a moment when families need organized information on all options (including candid policies from drug developers).  This is a moment when we can encourage people with ALS to openly share data about whatever paths they find and take.  This is a moment when we can fix the problems of trial and access processes that we didn't deal with for years.

People with ALS and their families deserve a fighting moment in that locomotive.

Monday, May 14, 2018

The $88,000,000 Question



It undercounts prevalence.

It can't even do incidence.

It has burned through $78,000,000.

Do we really want to spend another $10,000,000 on this?

Read more here.

Wednesday, May 2, 2018

Every 90, 85, 80, 75 Minutes

"Every 90 Minutes" has been a tagline that organizations have used for over a decade to describe how often someone dies from ALS in the US.

Take a look at CDC death certificate data  --

The number has been steadily increasing.  Based on an average of 7000 ALS deaths per year in the US in recent years, the tagline is missing 3 American funerals every day.

It's every 75 minutes, and that number is quickly approaching one funeral per hour.

The tagline should be "Every 75 Minutes and Not Getting Any Better."