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.

Monday, March 30, 2015

Shoot The Ball!

I am always amazed at how different the game of basketball is in the last 30 seconds of play versus the rest of the game.

During the first 3+ quarters, well-coached teams bring the ball up the floor safely, read the defense, set up the play, and are very patient until there is a good shot.

During that last 30 seconds, the team that is trailing conserves the clock with a variety of tactics, and sometimes there are fouls and sometimes there are shots taken that aren't perfect looks, but you have to shoot the ball if you're behind.  You have to do things that you would never do in the first half.  To stand deliberately with the ball in your hands as the clock runs out will never win a game for the trailing team.

We have an FDA drug-approval system that is fantastic for a lot of diseases.  It keeps us safe.  It insists on scientific evidence and it takes the time needed to test things the right way.  It takes a lot of time.

And then we have people with ALS who are losing the game and the clock is quickly running out. They simply want to take a shot.  Sure, the methods and processes aren't as good as what they would have used during the first 3+ quarters of their lives, but sometimes you have to adapt and learn to use the clock you have.  You have to use the rules that will let you get the most out of that last 30 seconds.  Accelerated Approval comes to mind.

It may be a rim shot or it may be an airball, but if you don't take the shot, it will never go in the hoop before the buzzer.  Ask Reggie Miller.

Tuesday, March 24, 2015

A Message From A Consummate Expert In ALS

The following words of Eric Valor were read at the #FDAHope4ALS  Rally in Upper Senate Park in Washington, DC, on March 25, 2014:

Eric N. Valor, diagnosed 2005, quadriplegic and on mechanical ventilation 2008.

ALS has taken everything away from me. I would say "let that sink in" but there is no way that any of you can fully grasp what that means. I didn't either until it happened. At age 35 I was on top of the world. I was very happily married, living in a house on the shore of the Pacific, successful in my chosen career, very fit and healthy, and essentially bullet-proof. But then I was visited by random misfortune from which there is no evasion or escape. Within a few short months I was diagnosed with Amyotrophic Lateral Sclerosis. Once diagnosed there was nothing any doctor could do except chart my decline and offer relief from symptoms. There is no treatment of any kind. It's a death sentence.

Everything drifted away with the inevitability and force of a swiftly-receding tide. One by one I had to give up all those things which defined me. Then my ability to breathe was taken, and thus the last of my independence faded away. Eventually even my most treasured achievement, my marriage, was shattered under the strain.

I am now totally paralyzed (quadriplegic) and dependent on a machine for my very breath. Everything I had built, bought, saved, or enjoyed in my life was taken from me or consumed by this disease. It can happen to you and it can happen today. And without any treatment options your story will follow mine.



Right now, if you were diagnosed later today and The Cure for ALS started the usual process of clinical trials, you would not survive to see that treatment available to you. If you were lucky, you could be one of the few hundred out of over 30,000 patients to use it briefly in trial (then your access would be removed). But we have a way to give hope, and that is the near-term access to experimental treatments like GM604.

In 1992, in response to the HIV epidemic, FDA created the Accelerated Approval Program. This allowed patients facing swift death from a disease for which no treatment options existed to access drugs outside of clinical trials, provided they have passed preliminary safety and efficacy trials. Two years ago, FDA agreed with ALS patients that we should have a faster pathway to treatment options.

We believe Accelerated Approval is that pathway. GM604 has proven itself safe in a variety of trials, and the data in ALS patients suggests efficacy in a much stronger way than any previous ALS treatment ever. We, the patients living today, want access to something which can potentially save our lives today. Tomorrow is too late.

ALS can strike anyone at any time. We never thought it would touch us. But now we are literally in the fight of our lives. This is our stand. As Lou Gehrig once said, we have an awful lot to live for. We need your help.


Thank you.

Sunday, March 22, 2015

I Think They're Next

I was a small child when fast food restaurants took off.  More than once I had 40 cents in the tight grip of my right hand and politely waited my turn in line at Burger Chef.  More than once when I finally got to the counter and reached up to put my change on the cold stainless steel, the tall lad taking orders made eye contact with the tall customer behind me and ignored me completely. I was invisible to them.  I waited and waited until a kind adult moved up in the queue behind me and said, "I think she's next."

Today we have drug developers, clinical researchers, big ALS not-for-profits, and the FDA all representing their own interests. They have a kind of professional altitude that often keeps them from looking at dying people with ALS directly in the eye.

When will those with ALS and their caregivers themselves be a part of every decision and transaction?

I think they're next.





Thursday, March 19, 2015

We Have Failed

Epically.

Yesterday a young mother lost her battle (and I don't use that word lightly) with ALS.  She was a great mom.  I can think of nothing worse than for a young mother to have a disease that steals her ability to give a hug or see her young children through all the challenges and joys of growing up.  ALS is an evil disease.

And I also saw the farewell posting of a man with ALS.  Those happen more often than you might imagine.  The first time I saw one almost two decades ago, I said out loud, "No!"  And I've said it many times since.

So what now?

Tomorrow isn't just another day.  It is a day when families are stuck with huge holes where loved ones had lived vibrantly just a few months before.  It is a day when ALS-caused grief and immense challenges will permeate lives.

And we go on.  And our lives go on.  And we feel good about projects that are self-described as "promising" and "exciting" (those words that have been cheapened over decades of ALS failures).

This seems like the material that would have inspired a parable a couple thousand years ago.

We all need to look in the mirror today and say, "We have failed... epically."

And now do some things differently.

Wednesday, March 18, 2015

Yesterday Was The ALS Reality Challenge

It hit like a bucket of cold water to the face.  It was the ALS reality wake-up call.

First there was a twitter exchange with @gehrig38 (Curt Schilling) as he talked about the people with whom he would like to have dinner --



Then there was another set of twitter exchanges over a simple "ALS stinks" tweet --


Curt Schilling was right.  Lou Gehrig would be devastated that the disease still kills and that it is part of his memory.

And then we saw the reality of ALS in the words of a husband caring for his wife fighting the evil disease.

It's easy to talk about "hope" and "promise."  Those words raise funds and make us all feel good.

We also need to face the reality of ALS.  It is an outrageous, cruel disease, and we have failed to deliver a treatment.  Period.  We have failed.  That's reality.

We need to throw that cold water in our faces more often.  Challenge issued.


Thursday, March 12, 2015

Don't You Worry Your Pretty Little Heads

Many years ago at the big ALS advocacy conference session where advocates were being briefed on the ALS Registry legislative request, a woman at a table toward the back of the room near me asked a question on some numbers.  Instead of getting an answer, she receive a response that it wasn't necessary for her to get lost in the weeds of the details.  It was only important for advocates to know the request and some bullet points when meeting with legislators.  And just tell your stories.

I muttered to myself, "Answer the question."

She muttered loud enough for me and others to hear, "Don't you worry your pretty little head."

And so it goes in the fight against ALS.  People with the disease and their caregivers never quite break through that "don't you worry your pretty little heads" mentality to become valued members of policy and research discussions.  They are the experts on ALS, and they are often kept on the bench, only to be called into action to tell their stories or raise funds.

In the last few weeks we have seen some remarkable people with ALS who have refused to wait silently for action, and I think they have been impressive examples of how much people with ALS and their caregivers have to contribute, and quite frankly, to improve the fight.

Example 1.

Example 2.

And there are many more if we choose to pay attention.  It's a rich, diverse, talented, competent asset  of thoughtful voices who understand ALS like nobody else.

PALS and CALS have questions, ideas, and concerns.  They must be respected and valued for their questions, ideas, and concerns.

Tuesday, March 3, 2015

Welcome to Hotel Inefficiency

Welcome to our hotel.

The other desk across the lobby told you that your room isn't ready?  We'll be so glad to help.

First leave your information with Albert here.  He can inform you as soon as a room is ready, but he only knows about rooms on the fifth floor.  


Next leave your information again with Alice on the right.  She can let you know when some of the rooms  on the tenth floor are ready, and she also has access to information on a couple of rooms on the fifth floor that Albert has, too.

You say you have a room preference for a king bed?  Oh, that's too bad  We'll let you figure all that out when a room is ready.  You can decide with the other desk if you don't want a room then.  

Oh, don't expect Albert and Alice to talk to each other.  Their jobs are funded in different departments'  budgets and you know how that goes.

And the other desk across the lobby will know the status of all the rooms in the hotel.  You'll need to check in with them persistently to find the rooms we don't know about, and they can't call or email you.  They just know and you need to keep checking with them.

Enjoy the experience of trying to find a room.  We know you're tired and anxious, but it's really not about the guest here.  It's about our systems and incredibly large budgets to develop systems that are all about Albert and Alice.

________________________________________

What brought this on?  This wacky hotel is a lot like what people with ALS endure to find clinical trials.

First we had the national ALS Registry with its archaic clinical-trial "matching" tool that has a small subset of enrolling clinical trials.

Now we learn of another government registry being kicked off https://www.rarediseasesnetwork.org/CREATE/index.htm

So we'll have both Albert and Alice and still have to constantly go back to the main hotel desk at clinicaltrials.gov (not to mention all of the donor funded clinical trial sites).

Any one of these individually isn't very good.  Together they are a model of expensive inefficiency.

Welcome to the world of ALS.  It's about incredibly large budgets to develop systems that are all about Albert and Alice.