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.

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 años 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.

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.

It's A Two-Way Street

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.

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?

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.