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, October 18, 2016

Actions >> Words

Yesterday I was involved in an interesting twitter exchange on the difference between the words we hear in the fights against diseases and the actions actually delivered.

And an apt example had played out on twitter just a few minutes earlier yesterday.

We got word from an ALS not-for-profit that they are trying to gather 1000 voice samples quickly for a research project.  The voices both of people with ALS and of healthy volunteers are needed. I did my own recording and found the process to be very simple, fast, and unobtrusive.  This seemed like a good job for social media.  1000 recordings should be a piece of cake, especially since some organizations have a wonderful social-media reach.   I posted the link and some requests for people and organizations to retweet.

Not one ALS organization retweeted.  Not one.  Individuals did, but not one ALS organization.  No action.

Here is one of the words that bothers me when I see this kind of pointless apathy and inaction -- "collaboration."  The use of that word has been pretty constant since the windfall of the ice of 2014.  There have been tens of thousands of dollars spent on talking about "collaboration."  Meetings and talk and hollow words don't solve problems.  Simple actions do.

When it came to actually doing something simple yesterday to fill one organizations's ALS study with quick voice samples, "collaborate" was just a word.  It didn't generate any action.  It meant nothing.

Talk's cheap.  Show us what you do.

Monday, October 10, 2016

A Study in Customer Experience in Clinical Trial Inquiry in the United States

Following is a link to the full paper written as a part of my epatient participation in the Precision Medicine track at
Stanford's Medicine X 2016:

"A Study in Customer Experience in Clinical Trial Inquiry in the United States"

A brief summary was also given in an Ignite! presentation at Medicine X, and results will be presented in a poster session at the 27th International Symposium on ALS/MND in December.

Many thanks to all with ALS and their loved ones who have ever sought to participate in clinical trials.  I hope that the information in this study will make an easier path in the future.

Monday, October 3, 2016

If You Can Only Go To One Conference, Make it Medicine X

That's advice that a respected friend gave me three years ago.  Boy, was he ever right.

This was my third experience at Stanford University's Medicine X  .  It was my second as an e-patient.  I thought after last year's experience that this year might be a letdown.  Boy, was I ever wrong.

Pre-Conference 1 -- "Providers are from Mars, Patients are from Venus: A workshop to improve patient-provider communication."
Faculty and staff of the Stanford Medical School facilitated a workshop that opened eyes to challenges and pitfalls of difficult provider-patient communications today.  Actors who work in the medical school gave excellent portrayals of the challenges.  I left with hope that healthcare delivery will improve, and I appreciate the insights into the training and challenges of the physician.  Thanks to Boehringer Ingleheim for this workshop.

Pre-Conference 2 -- "Shared Decision Making"
Eli Lilly and Company decision science specialists facilitated a workshop centered around a painfully difficult decision that a young couple faced regarding genetic testing and Alzheimer's disease.  The process of sorting out the elements of decisions was enlightening.  Thanks, Lilly.

And I was certainly glad to learn the California Duck Protocol.

E-patient Dinner
The night before the official start of Medicine X, the e-patient delegates are invited to a group dinner where we met a talented and diverse group of movers and shakers, many of whom are dealing with huge health challenges.  It's a special group and I'm grateful to have been included.

Medicine X Day 1

The speakers who work in our government were such refreshing highlights -- truly people who are able and willing to break the bureaucratic ruts that hold us back.  Claudia Williams, LaVerne Council, Susannah Fox -- the sharpest tacks in the healthcare toolkit as far as I'm concerned.

And then Eric and Terri Amador came on stage to teach all of healthcare through their ALS experience.  And Day 1 was good, so good.

Medicine X Day 2

As distracted as I was over my afternoon Ignite! presentation, it was again a productive day.  The
people you meet, the conversations you have, the presentations you didn't expect to attend are all some of the best things about Medicine X.

My Ignite! talk went fine, and I had questions about the clinical trial secret shopper project afterward, and I can't thank Louise Schaper enough for making a challenging 5 minutes fun and bringing out the best in every presenter.

It was a football Saturday at Stanford, and as I walked back to my hotel after a very long day, the campus quiet was almost eerie as tens of thousands of people were consumed with football in a far corner of the campus.

Medicine X Day 3

Just when I thought it couldn't get better, it did.  I attended the Million Veteran Project presentation in
the PMI tent.  They have half a million veterans' genetic typing completed and histories completed already.  They are looking for things to do with this rich set of data.  

They are looking at a long list of diseases that are often connected with those who have served in the military -- heart disease, PTSD,... Have they looked at ALS, I asked?  They did not know that there is double the incidence of ALS in US military veterans than in the general population.  They do now, and it is on their radar.  Think of the clues that all of the genomic data may hold related to the ALS-military mystery!

Thank YOU, MVP.

Then I went to a session on the project in Portugal.  For years we have been wanting a resource where the McGyver hacks that people invent to outsmart ALS can be archived and referenced.  Is this it?  I think it may be.

Finally I attended a timely session presented by Joe Gulfo on the FDA we need in the 21st Century, certainly a timely topic for me.  We do need a strong regulatory body and Joe's insights into its history and weaknesses today were interesting.   Ironically this was the day before the FDA Sarepta announcement.  And I am better armed today to march forward with ideas to shorten the path to approval for experimental ALS treatments.

Thanks very much to Dr. Larry Chu and Sarah Kucharski and Zoe and all who made this a valuable and energizing conference for me again.

Monday, September 26, 2016

Feed the Monster

I've been going to (yes, actually going to) Walks to Defeat (nee D'Feet) ALS for 15 years.

Something has bothered me for 12 years.

ALS leaves a large, terrible wake of loved ones behind every time someone dies from the disease, and the deaths are constant.  Why doesn't the attendance at walks grow at a constant pace every year to reflect that wake?  Why isn't the crowd that raises awareness and makes noise in communities huge by now?  It seems like the size of the crowd remains pretty flat year after year.  It's large, and that's wonderful, but where is everyone with loved ones left behind who used to come to the walk three or four or five or more years ago?

Every not-for-profit needs to figure out a revenue machine that will keep it sustainable.  I get that.  The ALS revenue machine is almost too easy.  There are thousands of newly diagnosed people with ALS every year and their families and friends and coworkers and neighbors want to help.  Enter the multi-level marketing of the walk revenue machine, and those who want to help can donate, and they do.  The constant stream of new diagnoses supplies many new donors and old ones fade away a few years after the funerals.

And getting people to actually show up for the walk itself isn't even a priority for some chapters.  It's all about the revenue machine, and the relentless disease is the fuel that feeds it.

Some people think I'm crazy for going back year after year after year.  I'm not.  If more people did that, we would have more people who see the problem of feeding the monster without changing the fight.  You see, the talk of achievements is recycled year after year.  It doesn't take Einstein to see what has happened.

Saturday, September 17, 2016

Meet Elizabeth Cosby

Today I have the privilege of delivering an Ignite! talk at Stanford University's MedicineX.  I appreciate the opportunity to present a message about ALS and clinical research to so many outstanding people whose mission is to rebel and improve medicine.  Thank you, #medx .

The full paper on this project will be published on this blog before October 1.

The project results will also be presented in a poster presentation at the ALS MND Symposium in December.


The day Mom was diagnosed with ALS, it hit us from out of the blue. There is one thing I remember vividly about that day.
The neurologist pulled me aside and said, ”Get her to someone doing clinical trials because the only thing the least bit “promising” would be experimental.
We were fortunate.
We had an action plan on day 1.

You see, the ice didn’t cure ALS.
There is no effective treatment.
It’s a difficult disease.

That’s EXACTLY why clinical trials are so important to those with ALS.
1. They are a legit Hail Mary
2. They advance the science for others.
You who do clinical research RECRUIT for trials. 

But wait.  You are RECRUITING.  They are SHOPPING.
People looking for a clinical trial are doing what we all do every day when we look for a new car or a great, affordable restaurant in Palo Alto.
They do not seek to be recruited.
They seek the best trials for them.
Businesses use Customer Journey Maps to show the complexity their customers experience to do business with them.
Complexity is never good in customer service. They journey should be easy for the customer.
There are some basic elements of most Customer Journey Maps –
Awareness, Research, Purchase, OOBE (WOW) Disney Apple.
Lets back up to Awareness.  A lot of money is spent on making people aware of clinical trials. Great.
Based on personal experience, I suspect that we are losing people who are already aware of clinical trials in the Research part of their journey, and the problem is poor customer service.
Am I all wet? How could I put some data behind my suspicion?  Meet our secret shopper, Elizabeth Cosby.
Elizabeth is a fictitious woman who came to life a few months ago in a new gmail account.  Elizabeth has a fictitious aunt who was recently diagnosed with ALS. 
Elizabeth tried to help her find a clinical trial.
The first part of the journey was to find all recruiting, interventional ALS trials.
Elizabeth wanted to organize the information for comparison shopping.
That was terribly time-consuming.
Finally on the evening of April 4, Elizabeth emailed inquiries about 39 trials.
There were 5 simple questions in each email – things like is it still enrolling, time commitment, reimbursement…
The first response came back that very evening.  It answered all the questions and was encouraging. Was I all wet about poor customer service?
Unfortunately time told the tale.

Of the 39 trials, Elizabeth got absolutely no response (not even an autoresponder) regarding 11 of them.  Crickets.

39 clinical trials.
28 responses.
11 crickets.
From the 28 responses we a variety of data on the five questions.
Please read my paper at for the full scoop.

This project was as much about the journey as the five questions.
Elizabeth assigned a completeness score to each response.
If you answered all 5 questions, you got a 5.
If you punted or simply referred Elizabeth to a consent document, you got 0.
Average completeness score was 3.  On average, Elizabeth had 3 of her 5 questions answered.
Elizabeth assigned a tone score to the responses (respectful, collaborative, encouraging).  Sure, it’s subjective, but that doesn’t mean it’s not important.
Average 4
It really got interesting when we added the scores together for each response.
13 of the 28 responses got perfect scores of 10.
What we saw was that
when they were good, they were very, very good, and when they were bad, they were horrid.

Good customer service. 
It’s not rocket science.  It’s not neuroscience.  It’s common sense. 
If we fix some easy customer service problems that Elizabeth uncovered, the clinical trial path will be faster and easier.  Everybody will win!

Wednesday, August 10, 2016

The Scales Tell More Than Your Weight

They tell a lot about poor healthcare delivery.

Story I.

Mom was around 5'6" tall and was always fit.  She lost a lot of weight because of bulbar-onset ALS.  We called it her Gandhi weight.  She was so very thin.  At a neurology appointment the helper took her to the scales on the way to the exam room.  She wrote something secretively on the chart.  I asked her how much Mom weighed.  She said 168.  My reaction -- "Oh, come on."  She said, "What's wrong with that?"  I said, "Look at her."  The helper was not happy, but we got another weigh-in at about 50 pounds less.  Had I not asked, Mom's medical record would have been dangerously wrong.

Story II.

I had a weigh-in last winter at a busy doctor's office.  I had my backpack and coat with me, neither of which I wanted to include with my body weight.  The helper said, "Step on the scales."  I looked around for a place to put my stuff.  She offered no option except the floor.  I said, "You really need a hook next to the scales."  Silence.  I said, "You know they sell them at Home Depot."  She snapped back, "I  can't do that."  Next year I think I'll take one that sticks on the wall because I didn't detect any initiative on the healthcare worker's part to fix a problem.

Story III.

This isn't my story, but it's a must-read.  And the story isn't really about the scales, but in a way it is.

When I read Sarah's 65-pound story, I thought of Mom's Gandhi weight.  Then I thought of our vet's office.  It's pretty basic.  It's not a fancy place.  They have a scale at floor level with a huge metal plate that the big dog walks onto.  It's simple.  I'm thinking that kind of scale would work for a wheelchair.  Drive it on, read the weight, subtract the weight of the wheelchair.  Voila.  Why would an ALS clinic not have a scale like West 56th Street Veterinary Hospital's?

I know it's hard to fix healthcare, but please, it's not that hard to get the weigh-in right.

Thursday, July 28, 2016

It's A Gene

Google may have brought you here.  You may have been reading about an exciting or promising or breakthrough new discovery that you funded with your ice bucket challenge donations.  Here are some fast facts:

It's a gene.

It's important.

It's not a treatment.

It's one small piece in a gigantic puzzle that is literally missing too many pieces.

Ice bucket money and money from many other sources helped find this NEK1 gene that affects a small percentage of people with ALS..

There are lots of other identified genes and more are discovered all the time.

New knowledge is good.

It's not a treatment for anyone's grandparent or child or niece or neighbor.  It's not even close.

It's cruel when organizations with an eye on fundraising pitch a feel-good story to major news outlets that inevitably put the magic words "breakthrough" or "exciting" or "promising" in the text. Grandchildren or parents or aunts or uncles or neighbors read those and unrealistic hope erupts.  It's cruel.

Saturday, July 23, 2016

The Drips Don't Add Up

Yesterday we received a promotional infographic -- for "Every Drop Adds Up" (a registered trademark of the ALS Association).

One drip caught my eye --

Whoa. The CDC's ALS Registry had nothing to do with the ice bucket challenge.  It had been going on for years and is completely funded by taxpayers.  We advocates are sent by the ALS Association to Capitol Hill every May to request those increased and generous funds.  And in 2014 and 2015, the ALS Association itself actually got $659,451 from the CDC to promote the Registry per  That would have happened regardless of the ice.  And the total price tag on this "largest" project is well over $70,000,000 in taxpayer funds.

We got the first report on national ALS prevalence from this "largest" project in 2014, the year of the ice, and it identified just 12,187 people living with ALS in the entire country.  It asserted prevalence based on that number.  The ALS Association concurred with the report and its assumptions and has called the CDC's Registry a resounding success.

Hold that thought.

Our information-seeking eyes moved to another drip --

The ALS Association (which serves only a subset of the total people with ALS in the United States) has personal contact with 15,000 people with ALS annually?

Whoops.  These drips certainly don't add up.

This makes the CDC's 12,187 publication look absurd.

This makes the CDC's well-paid contractor look ineffective at moving the 15,000 people it serves annually to self-enroll in the Registry.

This makes the ALS Association look complicit in promoting a project and its published data as successful and valid when it knows otherwise.

The parting drip --

It's past time to turn over some stones.

Sunday, July 10, 2016

I Dream

Sometimes when I want to write about something, I look through stock photos for the perfect picture for the blog.  Today I searched "dream" images.  There were hundreds about dreaming big, about reaching for the stars, about "if you can dream it, you can do it."

My dream is incredibly small.  It's not reaching for the stars.  It's about simple business 101.  It's about good project management.  It's what millions of workers do every day. It shouldn't even be a dream. It should be reality.  It's about a responsible, productive annual project oversight meeting.

There is no image for a dream this small.

I simply dream that --
  • Every person invited to this year's annual meeting for the CDC's ALS registry on August 3-4 will have read the meeting reports from the past decade's meetings in chronological order.  The annual meeting does not need to spend hours in basic orientation every year for those appointed to be the project's Advisory Committee.  Historically the meetings ramble and have led us to disappointing results despite all the back-patting.  This is serious business.  Be prepared.
  • Every person at the annual meeting will clearly state who invited him or her and what business relationships he or she has with other advisory meeting participants.
  • The CDC will present a detailed report of annual project expenses for the past three years and the budget status for the current year.  The cumulative expense also needs to be made public to the advisors.  Details, please (not broad category pie charts).
  • Project contractors will come prepared with meaningful metrics.  Good advisors make people sweat to present meaningful project metrics and not fluff at status meetings. Numbers of emails sent mean nothing.  Measure results.  I dream of sweat. 
  • Participants will speak candidly about registry completeness and design flaws.  Advisors will ask lots of questions and not let them go unanswered.
  • Advisors will challenge the status quo.  They will not be enablers.  Advisors make the same demands of this project that they would in their everyday business dealings.
  • All American citizens will be able to watch a new kind of oversight unfold live.
So I dream.  This should not be a dream.  This is business 101.  This is basic good governance.

Friday, July 8, 2016

Transparency Is The Friend of Good Public Stewardship

The ALS Registry Act, which many of us worked hard to have passed in 2008, allowed for the formation of an "Advisory Committee" which could exert a lot of influence on the direction of this huge government project.

In the past there has been an annual, invitation-only meeting of people who seem to serve the role of such an Advisory Committee. The selection of attendees is tightly controlled by the CDC and the ALS Association and the Muscular Dystrophy Association (the latter two are paid contractors on the project).

We receive the written reports from those meetings many months after they occur.

And to this day, a lot of very reasonable people find the deliverables from this project to be of questionable value and effectiveness relative to the price tag.  That makes the annual meetings very important.
  • In 2013 we really challenged the veil of secrecy around the annual meeting.  We could not find out attendee names in advance.  We could not view proceedings live.  
  • By 2014 the CDC decided to webcast the proceedings after a lot of prodding in social media.  This was a huge step in the direction of transparency where we could view the actual conversations and group dynamics and competencies live. There was no mechanism for us to submit questions, but we were happy with the positive baby step toward transparency.
  • One day of the 2015 meeting was also webcast and again, some sunshine was cast on the questions that were (and were not) asked and the answers that were (and were not) given.  The second day was blacked out for some reason.
After repeated inquiries about the 2016 meeting dates and webcast information, we got the following message yesterday from :
The dates for ATSDR’s Annual ALS Surveillance Meeting are August 3 – 4, 2016. The meeting will not be live streamed this year. The meeting will be recorded and posted on the National ALS Registry website at a later date following the meeting. The posting of the recorded meeting on the National ALS Registry website will be announced on the Registry website home page and through CDC social media channels.
This is troubling.  Now the CDC doesn't want us watching live.  Why?  Live proceedings can provide energy and conversation that can be quite valuable.  Why stifle that?

My response to the email was
The lack of a webstream of the annual meeting this year is very disappointing and a step backward in transparency.  I hope that those who made that decision will reconsider in the spirit of transparency and good public stewardship.
Thanks very much.
Please take a moment and send your thoughts to today. It's important. Thank you.

Sunday, June 26, 2016

ALS Does Affect Memories

And we don't do enough to fix that very fixable problem.  Last week we saw an exquisite example.

People who are perfectly healthy today can be slapped with an ALS diagnosis at any time.  Welcome to the rookie camp you never wanted to join. Wham. It's a fast-moving nightmare and families scramble to become knowledgeable and find some ways to fight and cope.  There is so much to learn and the downhill train is going fast.  And it's often over in a few months.

The ALS Association had been promoting its June 21 "Virtual Listening Tour" for weeks.  That's a nice concept -- for people in the ALS trenches to be heard.  Many of us preregistered as instructed.  A few hours before it was to start, I received an email that it was only for people with ALS and "current" caregivers.  I did not join as a result.  Later I got the link to the recording that I requested.  And as I listened to the listening, the memory problem became so apparent.

There was a chippy exchange between ALSA officials and a young woman with ALS over the investment of the ALS Ice Bucket Challenge millions.  The dying young woman spoke to urgency and boldness.  ALSA representatives rigidly defended a deliberate, long-term approach to its windfall.  The gap in perspective was huge (and I hope that there actually was "listening" amidst the defensive dialogue).

So what does this have to do with memories?

It has everything to do with institutional memories and public access to pertinent information.  You see, people in the throes of ALS don't have the benefit of knowing a lot of ALS organizational and scientific history.  Most everyone is a relative rookie and extremely busy.

Pertinent history and memories are continually lost in haystacks that are impossible for those rookies to search.  There is no orderly place to go look for past scientific investments and outcomes.  You constantly reach for information and get press releases and announcements.  You peruse the obvious from the nearest haystack and simply don't have time to dig deeper.  That's a terrible problem.

People dealing with ALS need orderly access to research investment amounts, dates, recipients, project descriptions, and outcomes.  Come to think of it, scientists and investors and donors and the organizations themselves could benefit from that history, too.

How are we to understand exactly where ALS Ice Bucket Challenge dollars have been spent or committed and projects' progress?  We have uplifting press releases and tax returns and financial statements and PubMed searches, none of which tell the story clearly.  If you have a rock-solid story to tell, please tell it transparently and continuously!  

Neuroscience research is risky, but we should learn from every dime that was spent on ALS research 20 or 10 or 5 or 2 years ago or 2 weeks ago.  We need searchable, accessible documentation to leverage the disappointments and the duds.  We get nothing.  I can't tell you how many times those of us with longer memories who hang around the fight wonder what ever happened to various "promising" or "exciting" research from the past.  Information is a precious resource whether or not it is uplifting to review.

It's quite possible that no matter how slowly or quickly ALSA takes to spend the ice fortune that we won't get a successful therapy, but we had darned well better learn more than we get today from a very staged haystack.

And, oh my, dates and outcomes will shine light on those urgency gaps, too.

Every year since I can remember, I've heard how this is the most exciting time for ALS research.  When I was a "current" caregiver 20 years ago, that made me feel good.  Now that I have more memory, it makes me realize what a poor job we do at learning from the past in order to work smarter and faster for the future.

Is anyone willing to fix a very fixable memory problem?

Wednesday, June 22, 2016

Listening 1,2,3

Chapter 1

Nice.  It's always helpful to feel that you are being heard, and I always learn a lot from hearing the perspectives of others.  I registered.  I blocked time on my calendar.  I prepared some thoughts.

Chapter 2
A couple of hours before the call was to begin...

Well, bummer.  Caregivers who have lost loved ones to ALS have a lot of interesting perspective and experience to offer.

Chapter 3
I asked if I could submit comments for ALSA executives and trustees "listening" and was given this email address.

I submitted the following:

Below are two comments that I would like to have made on yesterday afternoon's Listening Tour call.  I hope that this perspective will be helpful to the executives and trustees who are listening. Thank you.

1. ALS Registry

The CDC’s ALS Registry has been a disappointment on many levels – from process to oversight to deliverables.  ALSA controls most of the oversight by being lobbyist-in-chief for the project funding and by influencing so many of the appointments to the annual meeting group. 
Please make a serious, tough, businesslike review of this terribly expensive project. 
It has few of the characteristics of a smart, effective disease registry as cited by a recent report from FasterCures.  Completeness is certainly an issue that clouds any ATSDR reporting from the registry.  The project has a classic case of scope creep and distracting gingerbread.  Delays are rampant (we’re still waiting for the second annual report due last summer) and they are sloughed off with “it’s the government” excuses.  Appropriations are never substantiated to advocates with budget details before we're sent to Capitol Hill to ask for yet another $10 mil.  Oversight is hardly independent, especially given the financial relationships among those at the annual meeting.

It’s easy for charities to develop blind spots.  Has ALSA simply become an enabler on a most expensive project that has failed to live up to what a registry needs to be in 2016?  Please take a look at this report as a comparison to what we get --

We need some accountability at all levels in this project.  $70 million+ is an obscene amount of money to have plowed into  something that has not delivered what we need in an ALS registry.  

2. ALSA Inspire "Community"

I don't understand why you have allowed the Inspire ALSA “support group and discussion community” (formerly known as the “virtual advocacy community”) to languish in the manner it has.
People needing support and answers stumble across that site, they see the ALSA logo, and they expect trustworthy help.  Instead they often get terrible or no advice and there is no active contribution from ALSA.  That’s misleading for people with ALS and caregivers, and they sure don’t need misleading. 
Putting up a “support group and discussion community” website and simply slapping your logo on it and stopping by every few weeks to censor inappropriate postings isn’t right.  Please just pull the plug on it.

 I hope that we receive the link to the transcript soon.  I would like to hear or read what others had to say.

Saturday, June 18, 2016

___(Your Name Here)___, Tear Down This Wall!

It's a "now" moment for each of us.  Yes, you.  The stars are aligning and we must seize this moment.

We finally have some considerable focus on the huge protective wall between people with quickly fatal, untreatable diseases and experimental drugs.  We need to punch some holes in that wall.  Now.  Today.  We can do it. We must.

For a little over a year, a grassroots group of people fighting ALS became an informed activist group pressing the FDA, legislators, and drug developers.  They are smart.  They are full of energy.  They are fearless and nimble. Many of them are dying.  They experience the pain and understand the flaws of the status quo.  They are and they are on the FDA"s radar.

A few months ago we saw some considerable activity from another small group, . They proposed three pieces of legislation to punch some holes in the big wall between people dying of ALS and investigational drugs.  Their rally on Capitol Hill drew national attention on Thursday.  We stood in visible solidarity with advocates representing other terminal, unmet-need diseases.  Finally.

And last night, we saw a Vice feature, "Die Trying," on HBO that said it all, thanks to Angelina Fanous, a talented Vice writer/reporter with ALS.  Follow her @notsovanilla on twitter.  You will learn a lot.  And watch the Vice piece.  

The people with ALS whom we saw on Vice last night are still alive.  It's critical that we punch some holes now in the big protective wall that the FDA has around us all.  Sure, relatively healthy people need lots of protections.  Sure, people with existing treatment options need protection from things that may not be any better (or may be worse).  But people dying from ALS do not need to be protected to death.  We must act now.

When people with ALS have died, we seem to engage in some kind of societal hand-washing.  They're gone and are no longer suffering.  They are remembered as courageous and have impressive obituaries.  We move on and keep raising funds and talk of "promising" research.  That stinking treadmill must stop.  Now.

So please act, now.  It's as simple as sending some "Tear Down This Wall!" emails.  Watch "Die Trying" first, and you'll know what to say. #viceonhbo

Please speak up to:
  1. Dr. Janet Woodcock at the FDA .  I'm asking her to use paths such as Accelerated Approval more aggressively with terminal, untreatable diseases.  She gets it.  She just needs to feel the pressure that only we citizens can exert.
  2. Your two U.S. Senators and your Member of Congress, asking them to  co-sponsor any or all of Federal Right to Try legislation, the REGROW Act, and the RESULT Act.  You can read up on those at and they have tools for submitting your request.  These are pieces of legislation that will literally punch through some of those 1960's concrete barriers.
  3. President Obama at telling him that it is urgent that the Executive Branch's FDA not build walls between dying people and experimental treatments but rather tear a few down.
This is our "now."  This is the moment.  Five emails.  Please act.  Thank you.

Sunday, June 12, 2016

But You Were Just Here

May 10, 2016, was ALS Advocacy Day on Capitol Hill.  Hundreds of us asked for legislation, including faster paths for investigational drugs to reach people dying from ALS.  May 10.  Just five weeks ago.

"But you were just here.  These things take time."
"Nothing can be accomplished before the election."
"It's politics."
"Legislation can take years."
"You don't understand the system."
"Recesses are coming up. Not much will happen."

Sorry.  We're not accepting any bureaucratic excuses.  We can do this. We must.  Here's why.

On Thursday, June 16, 2016, hundreds of us are showing up on Capitol Hill again.  So soon?  Yes.  You see, there were 600 Americans alive with ALS who saw what happened on May 10 who have since died.  600 families who heard about all the hoopla on May 10 have since buried their loved ones.  600 American lives in just a few weeks.  600 lives.  So many.  So soon.  So fast.

We need for legislators to do their jobs.  Now.  ALS moves quickly.  We need to step it up a notch to address the needs of every American family dealing with ALS.  Now.  We can. We must.

Please speak up.  Please contact legislators and let them know that these 600 lives matter as do the next 600.  This fast, continuous carnage is outrageous.  The FDA processes that work well to keep us healthy people safe literally protect dying people to death.  There are three pieces of legislation for your consideration and action at .

600 lives in just the few days since May 10.  600 funerals.  600 families grieving a mother, father, son, daughter, loved one.  Just since May 10. #600lives

It's urgent. We will not accept any bureaucratic excuses.  Period.

Thursday, June 2, 2016

He Took The Ego Out Of SoMe And Made A Huge Impact

Late in 2014 a few people with ALS were asking questions about an experimental drug in the forum at  After  few weeks of frustration over the time it takes for people with ALS to get their hands on investigational drugs, a gentleman with ALS quietly posted a petition requesting access.  These things happen all the time, but Nick Grillo struck a chord with hundreds of people who want those with ALS to have a better path to experimental therapies.  Nick worked online and a few hundred signatures became a few thousand.  Wow.  People were interested.  Nick kept working and updating the petition and it became ten thousand.  Holy cow.  People were paying attention.  Media caught wind of it and it grew.  Organizations were having to answer questions and were trying to figure out who in the world this Nick guy was.  He quietly brought together different petitions and became the thoughtful glue that brought individuals into a smart new organized group called Hope Now for ALS.

On a cold, dreary March day in 2015, individuals showed up in Washington to make some noise.  This was not a highly-orchestrated Advocacy Day.  This was individual people in the trenches with ALS who had studied and broadened their scope beyond one drug.  By this time there were hundreds of thousands of signatures on the growing petition.  Nick couldn't be  at the rally in person, but this was his in many ways.  This was a moment in the fight against ALS.

By May, 2015, outside the big orchestrated Advocacy Day hotel, an even bigger group of individuals rallied.  These were thoughtful, well-informed people involved with ALS.  They were getting the attention of the FDA.  Hope Now for ALS became stronger yet still true to its grass roots.  And the petition grew and the ALS establishment was still trying to figure out who Nick Grillo was.

And the petition had almost 800,000 signatures.

That's big.

Nick died this week.  His quiet impact was huge.  He helped individual advocates find their voices and power.   Nick wasn't about Nick.  His work was always about getting faster access to investigational drugs for all with ALS.

Nick Grillo changed the game, and we are grateful.  And we must carry on.

Monday, May 30, 2016

Comments On Draft FDA ALS Draft Guidance

Following are the comments that I submitted to regarding their draft of the FDA ALS Draft Guidance document:

I tried to look at this document with two standards in mind:
1. Would it be helpful to someone developing an ALS therapy?
2. Would it change anything from what the FDA is doing today?

I worry that it achieves neither.


The commentary on the ice bucket challenge isn't valuable to drug developers or the FDA (but since you brought it up as the enabler of this document, how much was spent on this document and what was it spent on?).

The reference to the ALS Registry adds no value.

The explanations of the difference between the ALS "guidance" and "guidelines" are just silly.  The guidelines meeting was fare more specific and focused than this document.  I would hope that you could have simply dropped some of the advice from the guidelines group directly into this document.

"The goal of clinical trial guidelines is to lead to more effective and efficient trials but they do not directly impact the FDA regulatory process."  What in this guidance draft will directly impact the FDA regulatory process?  I would love for that to be the case, but I just don't see it in what is presented in this draft.


I think that the references to statistics are not needed and the numbers presented are not helpful.  Any drug developer seeking guidance from the FDA probably already knows more about the ALS market than those statistics reflect.  The "1 of 800" citation is in a paper that uses it with citations to two other papers. Did the references to the demographics come from the CDC's Registry?  In that case there are questions as to the completeness and validity of the data.   On page 17 there is a reference to 25,000 Americans living with ALS with a citation to a paper that just says it without any further background.

At the bottom of page 14 it boasts of guidance that will produce a "clinical development strategy that will provide the best opportunity to demonstrate a treatment's effectiveness and safety..."  I think that isn't the main goal.  The main goal here is to save some lives.

Benefit Risk

Words matter.  On page 15 we see that people with ALS are "vulnerable" to investigational new treatments supported by only minimal evidence of tolerability, safety, and efficacy.  Vulnerable?  The whole idea of risk-tolerance is the ability and right to make informed choices.  Vulnerable?  And then we read about the "rigorous" standards that must remain in place... so that informed decisions can be made that do not unnecessarily "compromise the health and safety of people with ALS."  Oh, please.  These people are dying a difficult death and don't need such paternalistic attitudes.  If we put those words in the FDA's mouth, we're crazy.

On page 16 there are references to oncology patients versus ALS patients attitudes.  Does the fact that many cancers have available therapies make this an apples-to-oranges comparison

There is a reference to perhaps having an influence on lower percentages of people with ALS taking riluzole.  Perhaps a more basic reason is the same underlying reason that people stop using drugs in general -- the cost-benefit proposition.  Why blame social media?

At the bottom of page 16 there are comments on exclusion criteria that indicate that patients want them "less strict."  Again, words matter.  "Less strict" gives an impression that changing exclusion criteria would diminish the science.  I don't think that is the case.

The subsequent comments about the difficulty of some with ALS to get to clinical trial sites was a wonderful opportunity to introduce guidance that would permit more telemedicine and remote monitoring of participants in clinical trials.  Will the FDA guidance encourage that they will accept more remote data from participants?

The first guidance on page 18 left me scratching my head.  What difference does that guidance make from the status quo?

There is a reference to DiPALS vs DPS to question the use of historical controls.  My understanding is that there were procedural differences between DiPALS and the original DPS participants and that this isn't resolved science.  It's probably not a great example to cite.

On page 19 there is a passing reference to Type II errors.  This needs much more attention in my opinion.  This is the crux of much of angst over the eteplirsen situation for DMD.  If a drug has a reasonable safety profile and the disease is quickly fatal without an effective treatment, the risk of making a Type II error is a problem that needs to be addressed.  The FDA should be willing to accept some uncertainty regarding efficacy rather than dismiss drugs that may work on some patients.  If we don't discuss this in this guidance document, we will have missed a major opportunity to actually "impact the regulatory process."

Expanded Access and Accelerated Approval

Why in the world were those two things glumped together?  The are vastly different.  They need to be clarified.

Expanded Access programs have a mechanism that may or may not be attractive to a drug developer.

Accelerated Approval gets products to market faster, and that is very different from EAPS and is far superior from many aspects.  Drug developers need to know that the FDA is willing and able to use the Accelerated Approval path aggressively, and that should be spelled out very clearly in this guidance document.


There is a lot of description of standard interventions affecting ALS prognosis, but are these interventions ever considered to be standard-of-care issues for clinical trials?  Some trials exclude people on NIV or feeding tubes.  Should FDA guidance be standing up for these standard interventions to be a patient right as standard-of-care?

On page 71 you actually used the "gold standard" term.  Last year I got the message from a gentleman with ALS that the gold standard isn't even the "gold standard" any longer.  Words matter.  Let's drop the baggage that makes us think that there is only one way to do clinical trials well.

Things I Didn't See That I Wish Were Included

There was discussion at the guidelines meeting that perhaps biomarker trials should be separated from efficacy trials.  Are we slowing down efficacy trials by adding companion biomarker components?  Are we putting trial participants at risk by tracking biomarkers during washout periods as was done in the NP001 Ph 2 trial?

Is there any mention in this document on rejecting placebo procedures that pose risks to patients?  Many people find placebo surgeries draconian.  It would be refreshing for the FDA to say that they expect alternatives to placebo surgeries or other procedures that would put placebo participants at risk.

Is there anything in this document that would have changed the big dexpramipexole trial design?

Many ALS trials start measuring volunteers from the day they enter the trial -- as if their ALS started on that day.  Others have short lead-in periods to gather some "history," and that lead-in actually delays access to the investigational drug.   It seems to me that historical medical data from clinical trial participants could be very helpful in determining whether an investigational drug is actually doing something in certain patients.  It would be helpful if the FDA guidance could specify acceptance of some lead-in data from trial participants medical histories.

I think that trial data using ALSFRS-R should always include the components of the score.  Perhaps the more granular data might yield some insights that the summary score does not reflect.

The Last Word

There were 38 references to "subjects" in this document.

"Subjects" is a disrespectful word for the most important people in clinical research.  It objectifies them.  It conjures up images of royal underlings.

Many pharmaceutical companies eliminated the word from their vocabularies long ago.

We need to eliminate the word from ALS research, and it has no place in an FDA Guidance document.  If anything, the FDA should tell drug developers that it expects that they will not use the offensive term "subjects" for people who volunteer for clinical studies.

Thank you for the opportunity to submit comments.