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.

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.




Wednesday, February 25, 2015

We Need To Face Some Facts

It has been a good week for ALS awareness.  Or has it?

During the broadcast of the Oscars we heard ALS mentioned twice, but did we have the difficult conversations that needed to follow?

While we're grateful for Eddie Redmayne and his honoring of those dealing with ALS, what do we tell the child who wonders why Mom died while Stephen Hawking keeps on ticking?  Are we willing to admit how much we don't know about ALS and MND?  Are we being clear with the public that ALS usually deals a far too swift demise to people who were pictures of health a year or two ago?  Are we being honest with one another that so much of your life expectancy depends on which nasty variety of ALS or MND picks you?

Are we talking about why Richard Glatzer, director of "Still Alice," wasn't at the ceremony?  Or where two of our biggest leaders in the fight against ALS, Steve Gleason and Pete Frates, were while we ate popcorn and watched the show?

Life for the healthiest is fragile.  For those with ALS, it is incredibly fragile.


During the course of that broadcast Sunday night, someone died from ALS (yes, died) for every statue that was handed out.

That's an outrageous fact.  It's not a movie.  It's a brutal reality.  We need to face it and fix it.

Monday, February 16, 2015

Their Words Give Meaning To Now

Today Genervon made a statement about their FDA request for accelerated approval for GM604 --

http://www.genervon.com/genervon/about_pressreleasestxt.php

I share two reactions from people with ALS.  They speak to the meaning of action.  They speak to the meaning of urgency.  They speak to the meaning of time.  They speak to the meaning of now.

From a tweet this evening --
"Gracias por este movimiento.. Fui diagnosticada con ALS a mis 31 aƱos y deseo una cura pronto.."
From a participant in the als.net forum where the Genervon petition started --
"I really don't know if I have 30 to 60 days left, then how long to ramp up production, might as well be a 100 years. it would still be nice to know this awful thing is beat."
When their now is gone, it is gone forever.

And we must not let our understanding of that fade like slogans on t-shirts.

Sunday, February 15, 2015

Is This A Guiding Message?

In one of the many mission-statement sessions I've participated in, a wise gentleman said that we needed to write a guiding message to tape over everyone's desk.  It should affect every decision, every phone conversation, every email.  Before you talk or hit send, look at that guiding message.

I realize that this screen shot is a statement of purpose from a tax form, but it has bothered me a lot, and words matter.

http://www.alsa.org/assets/pdfs/form-20990-20-20f2014-20irs-20sgd-2006-11-2014.pdf



I hope that words like sympathy might be replaced with respect.  

And I am not a victim.  That's exactly why I do my best to fight back against ALS and related problems.  It's about fight, not plight.

Moving forward I hope for a different attitude reflected by a powerful guiding statement of purpose.  We are more than sad, pitiful stories.  We have brains and ideas and a special rage.  And skip the sympathy.  Every person dealing with or who has dealt with ALS deserves respect.

Sunday, February 8, 2015

Is It Silly To Dream?

I didn't win the big Powerball jackpot last night, but I will admit that I thought about it. Don't we all dream about such things sometimes?

I realize that those windfalls can be curses, but I have a plan.  I think I could handle it. No new car.  I like the old one.  No new house.  I like the old one.  No lavish vacations.  I would miss finding the bargain travel.  But there would be some very new things --

Clinical Trials
Two ALS drug candidates that are stalled between trials for lack of funds would get moving.  The trials would be unlike any you have ever seen for ALS.  People with ALS and their caregivers would be part of the trial designs and it would be a very public process.  The trials would be accessible for people from Pittsburgh to Poughkeepsie and from Portland to Portland and from Poway to Pocatello.  These would be the trials that the world will watch unfold with the project status being very visible every step of the way.

Caregiver Care
Primary care for caregivers would become available at an ALS clinic.  Caregivers could get blood pressure checks, lab work, immunizations, and the basic health checks that they don't have time for today.  There might even be a partnership with a dental school to take care of that aspect of caregiver health that is so often neglected.

Advocacy
There would be some skilled and eloquent advocates for those with ALS.  Their job would be to address those who pose barriers to those with ALS and their caregivers.  And they would address them publicly, powerfully, and quickly.  Be it a government agency not knowing its own rules or a clueless entertainer making a tasteless quip, these advocates would send prompt and strong and visible messages that you don't mess with those dealing with ALS.

Obits
The ALS necrology project would be initiated.  It's simple and not expensive.  A searchable online database of obits of those with ALS would be built and continued.  It would become a vivid, continuing statement of all we lose to ALS.  Once the database is built and the process started, this would be a great student project to maintain.  No fundraising will be allowed.

Numbers
The animated infographic that we've needed for years to demonstrate how many people get ALS, how many people are living with ALS at any moment, and how many people die from ALS would be developed.  This would be done with design students and math teachers, perhaps in a contest format.  It would stop the myths and misguided use of prevalence in defining the impact of ALS.

Ratings
A Tripadvisor-like tool to evaluate and comment on those providing goods and services to people with ALS and their caregivers would be introduced.  This could raise the bar on a lot of providers, reward those who do well,  and stop much of the wheel-reinvention that characterizes the ALS caregiver's journey

Research
And with the other $100+ million, there would be investments in ALS research projects.  And those grants would be transparent.  People could look up who got what, when, and the outcome of each project would be part of that permanent, searchable record.

There.  I spent it. It didn't take long and my good old ob-la-di life goes on.  Some positive seeds are planted in the fight against ALS and some lessons are learned.  Sometimes it's not so silly to dream.

Tuesday, February 3, 2015

Sometimes A Message Just Misses The Boat


That was the case for me when I read a joint statement regarding the recent grass-roots interest in Genervon GM604.

Here's the statement:
http://www.alsa.org/news/archive/letter-to-the-als-community.html

And parts of it I read dozens of times, not believing what I was reading.

"Recent excitement about lithium in ALS and followed by no effect (or worsening) in subsequent studies underscores this point."
Lithium?  Are you sure you want to bring that one up?  Let's take a trip down memory lane. 
First, why would you choose the adjective "recent" for something that happened eight years ago?   
In 2007 there was a formal Italian study that made lithium sound like a miracle drug for ALS.  Lithium is readily available.  Every person with ALS I knew at the time had a chat with a physician and got a prescription for its off-label use.  Here's the really important part of all this.  A daughter of a  person with ALS put up a big shared spreadsheet online so that all trying lithium could track some basic data.  They did so.  Within weeks it was clear that this wasn't a wonder drug for ALS.  The excitement that a small, formal study had caused was put to rest very quickly by a broad base of grass-roots people who tried it and tracked their data.  
PatientsLikeMe stepped up to assist so that a better data repository than the spreadsheet could be used.  They published the patient-reported results far more quickly than the much more time-consuming and expensive lithium clinical trials that followed.  Here is an  article on the subject. 
Yesterday's statement mentioning lithium actually gives us a poster example for broad patient access to potential ALS therapies and patient reporting that can be much more definitive than small, slow, expensive trials.  I rest my case.
"...it is difficult to determine whether there would be any benefit to people with ALS if we were to repeat this in a larger study or the long term impact on quality of life."
Exactly!
"We do hear the voices that say, “This is exciting, let’s make sure this drug is available to all patients with ALS!”"
The voices don't stop with that sentence, though.  There have been numerous, thoughtful statements from those with ALS who want to advance the science through broader access to GM604.  It's not a selfish act of trying to survive.  It's a much more benevolent act of willingness to take risks with eyes wide open to advance the science more quickly.

Obviously you signers of yesterday's statement felt the heat of well over 100,000 signers on the change.org petition.  You clearly felt the heat of hundreds of postings and emails from people with ALS and their caregivers.  But were you even listening?

Sunday, February 1, 2015

Please Take The Shot Before Time Runs Out!

I just submitted the following to the FDA (click here for link):

In October, 2012, I participated in your Patient-Focused Drug Development meeting.  A few months later I also attended your ALS hearing.  In both meetings a message we all heard over and over from those dealing with ALS was the meaning of "risk." It's clearly different for them.

My impression has been that the FDA isn't the stumbling block in our past failures to deliver an effective therapy for ALS.  We realize that you need for people to bring you proposals for ALS, and that just hasn't happened often enough.  We understand that Genervon is about to bring you a proposal.  I hope that you can work with them to find a path that will let people with ALS assume more risk so that the science and understanding of GM604 advance more quickly.  Please let those with ALS be a part of the solution with their eyes wide open that there are risks.  If you were to ask people with ALS to put all of their remaining chips on the table and pick between safety and attacking ALS, the chips would land on the latter every time.

I realize that you have much to consider.  I sincerely hope that you can work with a willing drug developer to come up with a plan that will give people living with ALS today a chance -- a chance to try and be part of a solution.  As you well know, people with ALS are faced with lousy choices every day.  We can't let the perfect get in the way of something that might be good.  We've done that for decades and it hasn't worked.   
Two years ago I was given six minutes to talk to the FDA about my thoughts on ALS.  I was driven to finish my words before Dr. Katz's light flashed on.  I made it.  Someone, somewhere died from ALS while I spoke.  People with ALS face a cruel, lethal clock every day.  We must find responsible ways to help them.  I hope that you can work with Genervon to do just that.  

Thank you.

Please submit your comments.  Time is quickly running out as Genervon is meeting with the FDA in early February.

It's like you've been passed the ball for a buzzer-beater three point shot for the win.  Organizations are saying, "We don't think you should shoot. Give us the ball."  I say, let it fly.



Thursday, January 29, 2015

Place Your Bet

Ask a person with ALS to put all his or her remaining chips on a bet.

Choice "A" is against ALS.

Choice "B" is for safety.

The chips land on "A" every time.

Friday, January 23, 2015

Will The ALS Ice Bucket Challenge Have A Surprising Legacy?

Perhaps the real legacy of the ALS Ice Bucket Challenge won't be the buckets of money.  Perhaps it will be that individuals found voice in the fight against ALS.

This week there have been incredible examples, all with roots in individuals who spoke and acted --

The meeting of Steve Gleason directly with HHS Secretary Burwell and key Senators and government officials. 
An online petition of the FDA that now has 85,000 signatures. 
And this eloquent piece from a man with ALS.

We are our stories and so much more.  There are thoughtful individual voices in the fight against ALS.  Yes, sometimes one voice is all it takes.

Thanks to all.

Sunday, January 18, 2015

Let's Change The Subject

Over the past few years, many interested in patient-centricity in clinical design and processes have banished the old word "subject" to describe those who volunteer as trial participants.

Words matter.  Nobody likes being called a "subject."  It conjures up visions of royal underlings or targets of police actions or objects.  It's simply not patient-centric.  It does not reflect the valuable role that people who volunteer for clinical trials have in these projects.

In the fight against ALS, many healthcare professionals and researchers question why it is so difficult to fill clinical trials.  What can we do?  Using words more respectful of the most important people in the projects might be a good start.


Friday, January 16, 2015

It's Time To Think Out Loud

When I testified to a very stoic FDA panel at the ALS hearing two years ago, I envisioned thought-clouds above a lot of heads.  When people with ALS, their caregivers, drug developers, and organizations asked for faster approvals, I imagined FDA scientists facing us thinking, "Fine. Bring us something."

There was a lot that was unspoken in that room.

It's time to speak.  It's time to get a proposal on the table and work out the good, the bad, the ugly, the impossible, and, yes, the possible.  There is a willing drug developer.  There are people with ALS who are more than willing to accept risk beyond a normal clinical trial in order to advance the science and have a chance at a possible therapy.  There is an FDA that understands the urgency of ALS within the context of its serious responsibilities.

Let's think out loud this time, candidly.

The Genervon GM6 conversation has its roots (literally grassroots) on the forum at www.als.net .  To view the forum requires registration, but it is well worth it if you are interested in the science and challenges.

In addition here are some pertinent background links --

This blog's first post on the subject including a letter from Genervon.
This blog's second post on the subject about choices.
The change.org petition started by a gentleman with ALS with 19K signatures as of this morning.
An important blog from another gentleman with ALS who has taken GM6 and is working with Genervon.

Silence is easy.  It's time for some difficult conversations.  Out loud.