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, May 25, 2015

Meet Two Jimmy Clarks

Meet Jimmy Clark

Yesterday at the Indy 500 they celebrated the 50th anniversary of Jimmy Clark's victory with a pre-race lap of his beautiful winning car.  Jimmy Clark was special.  We called him "The Flying Scotsman." He wasn't flashy.  He was just fast. He looked more like an accountant than a race car driver, but he could drive. He used new technology to chase his dreams of winning.  He assumed risk with eyes wide open and he achieved great success in all kinds of racing.  We applauded his many victories and mourned when we lost this great talent and gentleman in a racing accident in 1968.

Meet Jimmy Clark

In March at the first Rally for Faster Drug Approval I met another Jimmy Clark.  He is special, too.  He is loved by his family and loves life.  He also has ALS.  That puts a bad wrench in things.  All he asks is that he be allowed to assume some risk with eyes wide open so that he might chase his dreams, too.  He wants to be allowed to take risks that are minuscule compared to those that are taken every weekend by those who participate in auto racing.  He wants to advance technology and science just as race car drivers are allowed to do.  He also wants to live for a family that is far more important than a race or a trophy.  This gentleman simply wants us to let him try an unproven, investigational drug with a good safety profile.

Why not, I ask.  Why not?  Where are our priorities?

Friday, May 22, 2015

Your Hands Aren't Really Clean

You don't want blood on your hands.  Nobody does.

If someone were to die because of an allowed, yet unproven ALS treatment, that would leave blood on the hands of the FDA.  Nobody at the FDA wants that.

IF someone were to die from untreated ALS, that's an act of God, right?  No blood ends up on anyone's hands, right?

Wrong!  It's on all of our hands.

Not acting doesn't wash our hands of responsibility to try to stop deaths from ALS.

Finding excuses and reasons to delay does not mean our hands are clean.

Regulators, scientists, advocates, physicians, and bloggers share a new permanent stain with every life lost to ALS.  Our hands are not cleaned by inaction.  You can't wash it off by letting ALS take its course.

Saturday, May 16, 2015

Just Read The Transcript

On February 25, 2013, dozens of people with ALS, caregivers, scientists, drug developers, ALS
organization employees, and clinicians made a trip (many at their own expense and with a great deal of difficulty) to testify to the FDA.

They gave a gold mine of ideas for speeding up drug development and approval.  It's a big gold mine because people testified all day in six-minute segments.

They talked about risk, biomarkers and endpoints, trial design, FDA processes, natural history, controls, and on and on.

Today we hear that the FDA guidance document that was indicated at that meeting is only now being started and that dozens of people with ALS, caregivers, scientists, drug developers, ALS organization employees, and clinicians will soon be convened to give their input.

Ok, everybody.  How about just reading the transcript from that FDA hearing?  You have the materials to start a draft FDA guidance document right under your noses.  Let's get moving.  Spending buckets of money on unnecessary meetings isn't a sign of an understanding of urgency.  Nearly 300,000 more people have died from ALS since that hearing.  Urgency?

You can watch.

You can read the whole thing.!documentDetail;D=FDA-2013-N-0035-0271

And you can read the FDA's summary and reaction.!documentDetail;D=FDA-2013-N-0035-0272

As an example, one of the ideas presented that day is that people with ALS have a right to know who is doing what during the clinical trial and FDA back-and-forth in the approval process.  Are we waiting for statisticians?  Are we waiting for an FDA decision?  Are we waiting for more data to be submitted?  What's going on?  Are we waiting for somebody on vacation?  Think about how much more clearly the Accelerated Approval application for GM604 might be perceived if we simply had this kind of transparency policy.  Any successful modern business has project teams that know what's going on in the scrum today, yet the veil of secrecy continues to obstruct the view of the most important people in the drug development and approval process -- those with ALS and their caregivers.

And there are hundreds of more examples of ideas that can have a direct impact on speeding up drug development and approval.

Just read the transcript.

Thursday, May 14, 2015

Some Stream-of-Consciousness Thoughts From #ALSAdvocacyDay2015

In no particular order, I reflect on my short trip to Washington, DC, this week.

  • For the first time ever, I'm grateful for being held captive on a plane, waiting for a jetway.  My Senator across the aisle could not have been more gracious and concerned as we talked about ALS, drug approvals, and the FDA.  Thanks, USAirways.
  • The Rally for Faster Drug Approvals held by did not disappoint -- enlightening speeches, thoughtful PALS and CALS, good information on FDA and drug approvals.  It was hot, but the PALS, CALS there did not wilt.  And there were signs and cameras, and heads on the street turned.
  • A sign taught me that "We are the change that we have been waiting for." I love it.
  • The evil ALS continues to pick some remarkably talented individuals and families.
  • Double-blind, placebo-controlled trials don't need to be the "gold standard."  Shoot, the gold standard is no longer the "gold standard," a PhD economist with ALS explained.
  • You listen to the SGD, but you look at the PALS delivering a speech, and it's amazing.
  • Open and diverse discourse can teach us all new things, and God knows, we need new ideas.
  • The status quo is simply unacceptable.  Same old same old isn't good enough.
  • Denial of hugs is one of the big evils of ALS.
  • Bragging rights should not be an unspoken priority.
  • PALS, CALS are their stories and so much more. So much more.
  • The Abigail Alliance has a the most refreshing IRS Form 990 that I've ever read.
  • The ship's photographer can be annoying in a legislative meeting.
  • Advocacy is year-round work.  Many of us take that seriously. All the more reason to give advocates a heads-up when a HR number is changed and prior co-sponsors need to re-up on the new HR. 
  • Legislators are very frustrated with the FDA, too.
  • I love Hoosiers.
  • Dormant Therapies provision is important in 21st Century Cures, but there is a lot more than needs to be in there that will have a direct impact on getting therapies to PALS faster.  Is anyone looking out for PALS?  Really looking out?
  • We all don't have to agree.
  • What do those ALSA people who randomly morph into your legislative meetings really do?
  • Money doesn't buy a great advocacy experience.
  • A chance encounter with your other gracious Senator on Pennsylvania Avenue can cap off a good day of advocacy.  I'll always remember talking about 21st Century Cures with him directly across the street from where I held a sign at the Rally for Faster Drug Approvals a day earlier.
  • My favorite hashtag for the fight against ALS is #notquietly . 

Monday, May 11, 2015

We Need To Put The Sign Back Where It Belongs


A year ago we talked to legislators about some Medicare rules being implemented that were a crazy trip back to 1980.  They were denying people with ALS access to modern, economical technology related to speech generating devices.  When we talked to legislators and staff members, they were 100 percent in agreement that the rules were antiquated and wrong.

That was a full year ago.  There has been lots of talk and there have been band-aids, but we learned that we need a law.  The buck can't stop where it belongs for Medicare recipients unless we make a law.  Today people with ALS are denied or lose the very devices that let them be productive and engaged parents, citizens, and patients.

Enter a small charity that couldn't let those people with ALS lose the purpose, productivity, and joy
that the simple ability to communicate provides.  They let the buck stop with them because the need is vital.  In fact, they have spent over $1.4 million bucks in the last few months supplying enabled devices to people with ALS that Medicare should have been providing.  Perhaps the fact that this organization is led by a young man with ALS who uses this very technology is the reason he decided to help in a substantive way... rather than pass the buck.

Charitable contributions should not be expected to cover for a broken government rule.  Government should not be allowed to pass the buck.

But we need a law. Now.

Please write your Member of Congress. Today.  Demand quick support of the Steve Gleason Act.  Here is information and a convenient tool to submit your thoughts --

In an ultimate twist of irony, President Johnson signed Medicare and CMS into existence at the Truman Library in 1965.  They should have paid more attention to the sign while they were there.

Sunday, May 10, 2015

Before We Ask For Another $10,000,000.00 For The ALS Registry - Question 2

Which is it, $1.1 million or $10 million?

In the 2013 OMB filing for this project, we see the following:
(And a hat tip to the ALS advocate who sent that document to me yesterday.)

14.  Annualized Cost to the Government
Data analysis by ATSDR may result in action taken by the Division of Toxicology and Human Health Studies in response to the required CDC mandate in maintaining preventive health activities and surveillance systems.  The action taken will vary, depending on the analysis.
 The total cost to the federal government for the collection of this information for the three year ongoing project is $3,300,000 as itemized below. Annual ATSDR personnel costs $420,000 Additional expenses will be incurred by ATSDR in order to operate a successful surveillance program/registry.  Four staff will contribute to this program: a Senior Scientist (25% contribution=$75,000), and a program analyst (100% contribution = $100,000)  A contractor will be used to maintain the web portal for case registration and participation in surveys in addition to providing public user support 40 hours per week ($500,000).  Lesser expenses may include computer resources, telephone calls, and recruitment materials (approximately $5,000).
 The estimated annual cost to the government is $1,100,000.

So we're confused.  We ask for another $10 million every year.  Why?

The ALS Registry budgeting process has always been a bit of a mystery.  Last year we learned at the ALSA Advocacy Conference that the original $10 million number was a number that the legislative sponsor thought could be raised. It was not built up from a businesslike budget.  We also got our first glimpse into annual expenditures with some very broad percentage breakouts at that conference --

It would be respectful of those with ALS and caregivers who are going to legislators to ask for another $10 million to know more clearly how that much money would be spent by project component.:

And yes, it continues to be a concern that the advocacy organization that sets the public policy priority that champions $10 million for the Registry has itself been a large, paid contractor on the project.

Why are we asking for $10 million for a $1.1 million project?  More does not mean better.

Thursday, May 7, 2015

Before We Ask For Another $10,000,000.00 For The ALS Registry - Question 1

Next week ALS advocates will be sent to Capitol Hill to ask for another $10 million for another very generous ALS Registry budget.

We have questions.

A PALS described a problem with the first Registry report issued last July --

Q1:  Have any of the local studies that were funded as part of the Registry project shed any light on this significant concern on how large the big "unknown" patient population is?  Are we able to extrapolate how many PALS are likely not found via the Registry's database mining and the online enrollment portal?

Friday, May 1, 2015

These Seats Have An Obstructed View

It's just wrong.  You can't follow a game when you can't see who has the ball.  You can't figure out when to cheer or boo.  It's incredible that in this age of the taxpayer-financed sports palace that fans can be stuck with expensive seats with obstructed views.

Such is also the case with our view of the back-and-forth between Genervon and the FDA.  Who has the ball?

Are we waiting for the FDA to give Genervon something?

Are we waiting for Genervon to give the FDA something?

All we know is that the most important people in the stadium, those with ALS and their caregivers, can see time ticking off on the clock, but they can't even figure out who has the ball.  They have been assigned seats with a conveniently obstructed view.

It's just wrong.

Tuesday, April 28, 2015

The Tone-Deaf Won't Defeat ALS

For decades some people with ALS and caregivers have worked hard to nudge and push the fight in new directions.  Organizations and the FDA often appear to be listening, but in reality, they have been tone-deaf to ideas that will require big changes.  They are confident to stay their course, and besides, the annoying people demanding change eventually fade away with this quickly fatal disease.

Yesterday I went through transcripts of the February, 25, 2013, FDA hearing on ALS.  What a complete disappointment that none of the stakeholders in that room delivered on any of the ideas that people with ALS and their caregivers presented there.  Nothing.  That's tone-deaf.  That's a waste of time, money, energy, and, yes, lives.

"Stay the course" is nuts.  The failed status quo must go.  The tone-deafness must end.

A group of thoughtful, unfunded, grassroots people with ALS and those who care about them have found their voice thanks to social media.  Below is an announcement that they issued for your consideration.  They deserve to be heard by people who can actually grasp what they are saying and who are capable of change.

Members of the  ALS patient community are pleased to announce the creation of a new organization, Hope NOW for ALS, ( )  The  mission of this grassroots organization, comprised of people with ALS (PALS) and their families and friends,  is “to expedite access to promising investigational drugs for the current generation of PALS through the FDA’s Accelerated Approval Program (AAP) and other pathways, and to speed up ALS drug development by promoting improved alternative and humane designs for clinical trials.”   Hope NOW for ALS is hosting the “ALS Rally for Faster Drug Approval” in Washington D.C. on May 11 and hundreds are expected to attend from across the country. 
ALS, also known as Lou Gehrig’s Disease, is a fatal disease for which there is currently no cure and no effective treatment to stop the progression.  The average life expectancy after diagnosis is two to five years.  Some live longer but about half will pass away in under three years.  The genesis for Hope NOW for ALS was a petition,, started in January calling for “accelerated approval” of a promising new ALS drug called GM604.  Through the campaign, many in the patient community became aware of the outdated and broken drug approval system based on the 1962   “gold standard” or double blind randomized controlled trial (RCT) protocol required for new drugs to be approved.  This 53-year-old protocol requires Phase I, II, III trials that take 10 to 15 years and require tens of millions of dollars.  Many promising drugs never make it to Phase III due to the prohibitive cost, or because they don’t meet stringent FDA criteria (P=.05) that often dismisses data even when they demonstrate a drug is effective on a subset of the population.  The current generation of people with ALS has almost no hope for survival unless treatments become available much faster through the Accelerated Approval Program (AAP) adopted in 1992 as a result of AIDS-HIV activism.  AAP was designed to speed up drug approvals for serious conditions that have an unmet medical need.  Still, not a single drug for ALS has moved through the AAP process.  Hope NOW for ALS intends to change that. 
Nick Grillo, Jehad Majed, Eric Valor, and Linda Clark

Saturday, April 25, 2015

Did Anybody Ask Dr. Brantly

Are you worried about side effects from this experimental drug?

Are you worried that it might make you worse?

Do you want a chance at a placebo?

Are you concerned that another scientist might have an option that would be much smarter for your disease... after you are dead?

Tuesday, April 21, 2015

Time Out! It's Time To Listen To The Adults In The Room

OK, everyone, this is about much more than GM604.

Genervon and the FDA have been engaging in an interesting p.r. war, lobbing statements overhead while people with ALS and their caregivers are stuck in the trenches.

Genervon has been obvious (and, therefore, not very effective) in trying to make a very public appeal for FDA Accelerated Approval of GM604 for ALS.

The FDA has been very nuanced in making statements to cast aspersions on Genervon.  I'm pretty sure this recent public statement cost the American taxpayer a small fortune

And the media know an interesting story when they see one.

So who is using whom here?  I don't know.  I don't really care.

We need to take a time-out from this p.r. circus and listen to the adults in the room -- the people with ALS and their caregivers.

This is about much more than Genervon.  This is about a failed process for testing and approving therapies for ALS.

Fact:  A number of years ago an FDA representative explained to some ALS advocates that the FDA can't just do things.  They need to react to things that are brought to them, and, frankly, the FDA doesn't get much for ALS beyond a few traditional Phase 1,2,3 trials.

Fact:  Genervon took them something different -- a request to use the Accelerated Approval path that was established for the FDA some years ago.

Fact:  People with ALS and their caregivers have asked repeatedly for the chance to assume more risk in order to try investigational therapies and to advance the science more quickly.  The FDA and our ALS organizations have nodded but delivered nothing to let that happen.

Fact:  We don't know if GM604 will work, but its safety profile to-date does not put the fear-of-God in most people with ALS.

Fact:  Traditional Phase 2 trials are not big enough to be definitive.  They are not designed to look for efficacy, yet for a disease like ALS, every trial is being scrutinized for some sign of efficacy.

Fact:  Traditional Phase 3 trials are dreadfully expensive; therefore, "promising" drug candidates sit on shelves while people with ALS die.

We have a failed process.  It takes too damned long.

We have people with ALS and caregivers who are demanding that the word urgency finally be given some teeth in the fight against ALS.  They are the ones who need to be heard in this conversation.  They are smart.  They are thoughtful.  They are facing a fast and difficult path to death.  They understand good science.  They have eyes wide open about risk.  They know p.r. volleys when they see them, too.

Genervon has pushed the boundaries of the same old same old with their request for Accelerated Approval.  They have upset those who seem to believe that the existing Phase 1,2,3 trial model is the only way to do good science.

Listen to the adults in the room -- those with ALS and their caregivers.  They are smarter than any of the p.r. wonks realize.

You will have a chance to listen to some of them in person .

They are the ones acting like adults here.  They need to be heard.  And this is about much more than GM604.

Sunday, April 19, 2015

And The Problem Is... The Big "Therefore"

A public comments period is underway for another new component of the U.S. ALS Registry --

Please read the proposal on increasing self-enrollment and comment before May 25.

Following are my comments:

"Therefore, efforts to increase Registry awareness among non-referral center neurology practices/neurologists is needed to increase self-enrollment of persons with ALS."

That's a pretty big "therefore."  They are jumping to a conclusion without data, and that's very wrong on a project that needs to be data-driven.

There is nothing in the MMWR report that addresses the completeness of the administrative databases or the self-enrollment portal.  We simply don't know now many noses were missed in their counting techniques.  We have no idea how incomplete their census of people with ALS was.

1. What basis in fact is there that the root cause of low self-enrollment is neurologists at non-referral centers?

ATSDR decided to give large, non-competitive contracts to the ALS Association (and subsequently to the MDA) for "education and outreach" for the ALS Registry.  I believe that the rationale for such no-bid contracts was that they had access to the vast majority of people with ALS in the US via their networks of ALS clinics and neurologists.  They had the captive audience to promote self-enrollment and they were being paid handsomely to deliver them to the self-enrollment portal.

How complete are the data in the MMWR report?  How many noses were not counted by either method used?  We must answer those questions before we fly off and try to solve a problem that we can't quantify.

It is clear from the MMWR report that ALSA and MDA were not able to deliver as many people with ALS through the self-enrollment portal as the administrative databases found.  Is that a problem with the size of the population of people with ALS in their clinic networks, or is it a problem with their education and outreach to motivate their own clinic neurologists and people with ALS to self-enroll?  It's time to do some measuring and to speak to data rather than to assume that the problem must be outside these contracts.

How many unique people with ALS were seen annually in ALSA and MDA clinics, starting with the MMWR report period?  That should be a first clue as to how incomplete the self-enrollment portal data were.  That is also a big clue as to the effectiveness of these no-bid contracts.  Without that introspection and some data, why would we assume that neurologists who are not involved in ALS clinics are "the problem."  

2. Even if the root cause of the low self-enrollment problem turned out to be non-clinic neurologists, what basis is there for the assumption that cold phone calls to neurologists' staffs will actually affect physician behavior to encourage people with ALS to self-enroll?  That seems to me to be an expensive, time-consuming project not based on any kind of behaviorial studies, and it certainly lacks measurable goals for outcomes. 

People with ALS, their caregivers, and American taxpayers need a more accountable, businesslike approach to a project that is important to the fight against ALS.  We need data-driven decisions and not a convenient "therefore" regarding the completeness and root problems of the ALS Registry.  

Sunday, April 5, 2015

We're Ticked

The ALS ticking clock goes tickticktickticktick.  And then they're gone.

The clocks of those who are to deliver on behalf of those with ALS often go tick........tick................tick................................tick...............................................................tick.                                                          

The difference is palpable.

On February 25, 2013 (that's over two years or a quarter million ALS funerals ago), there was an FDA hearing on ALS.  The big ALS organizations were in the front row with bells on.  They had a lot to say.


MDA Press Blurb

Joint ALSA MDA Statement

When you look at the joint ALSA MDA statement, an FDA guidance document for ALS is mentioned repeatedly.  Back at the time of the hearing, the first such document had just been issued for Alzheimer's Disease and it was felt to be a huge step forward in helping researchers design trials that would hit the marks that the FDA needs to do its job.  Having a similar document for ALS seemed to be a no-brainer for a disease needing faster trials and approvals.  The big ALS organizations talked of driving that effort.

Where is our document? It was not on the list of guidance documents that the FDA is scheduled to release anytime in 2015.  Tick.

DMD advocates submitted their draft last year.
DMD Draft FDA Guidance Document

Is it true that wagons are just starting to be circled for ALS organizational stakeholders to begin to talk about an ALS document?   Over two years later?  Tick.

At least the DMD folks blazed a nice trail to follow.

Need anyone wonder why people with ALS and caregivers are rallying in Washington again on May 11 to demand faster FDA paths to approval for possible ALS therapies?  They're ticked, and they live at a far different pace than bureaucracies.  I encourage you to stop by the rally and listen.  They are thoughtful and bright people, and, yes, they're ticked.  All of the talk of over two years ago has delivered nothing. And it seems like organizations are now convening to talk so they can talk about talking at ALS Advocacy Day in May.  We should all be alarmed.

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.