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 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

So we'll have both Albert and Alice and still have to constantly go back to the main hotel desk at (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 --

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 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.

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.

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.

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.

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.

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

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:

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.
" 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."
"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 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 .  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 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.

Wednesday, January 14, 2015

We Have Reached A Fork In The Road

People with ALS face choices every day.  Most of their options are poor, but they make the best
decisions they can.

The FDA faces choices every day.  They strive for excellence as we consumers and taxpayers trust and expect.

Drug developers face choices every day.  They answer to their investors who expect financial viability while they try to answer to the needs of people with ALS and the FDA.

It's difficult.  Today we have a choice that involves all three groups.  It's especially difficult, but that's good.

Two years ago many of us made trips to the FDA to offer testimonies to those who are trusted to regulate ALS drug approvals in the United States.  Over and over, the people with ALS (who have to pick from lousy choices every day) asked to be allowed to assume more risk in the drug development process.

Genervon is going to ask the FDA for "conditional approval" of their GM6 drug candidate.  Truly, that's a shortcut from the perfect path, but can we let the perfect get in the way of something that might be good?  There are people with ALS more than willing to roll the dice with eyes wide open.  There have been enough disappointing clinical trials for them to know that "promising" does not mean "will work" and "perfectly safe."  There is a drug developer willing to make product with a good safety profile more accessible to people with ALS in a closely monitored situation.  Data will be reported to the FDA.  Nobody is trying to sell a secret magic potion.  It's an option within the FDA regulatory environment that could give people living with ALS today some access to GM6.  If it works, awesome.  If it doesn't work, valuable lessons will be learned.

Perfect?  No.  Good?  Perhaps.

If we don't seriously discuss the options and design a responsible, creative solution, we will have let down all of those people who trekked (many with great difficulty) to Washington to testify to the FDA two years ago.

Your thoughts today to the FDA will be appreciated.  Click here for contact information (contact form or email address are at the top of that page).

In addition here is an online petition provided by a gentleman with ALS for your consideration.

Thank you.

Sunday, January 11, 2015

"The FDA Will Listen To Your Voice If You Choose To Express It"

The letter below was received by an ALS caregiver/widower/advocate. He had inquired for information from Genervon about their GM6 drug candidate.  GM6 was also in the news again this week.  There is an interesting conversation on the forum at that includes the gentleman with ALS treated in the recent news item.

Please read and consider and act.  It has been two years since the FDA ALS hearing.  We have a chance to speak again on something immediately actionable. If there is one thing I hope we can especially appreciate this week, it is our freedom of expression. Thank you.


Dear All,

Thank you for writing to Genervon about your interest in GM6. Attached are our two latest press release of our trials results. Sorry there is no giddy up option to Tijuana clinic or several variation of it as suggested by a few of you.

Genervon is meeting FDA next month for accelerated approval of GM6 for all ALS patients. Our plea is that ALS patients cannot wait and GM6 is very safe with no significant adverse events in any treatment group patients, plus incidentally GM6 is efficacious including Genervon trial programs for other neurodegenerative diseases. Genervon argued that GM6 should be advanced to conditional approval in a post-marketing surveillance program based on the following:
1. Demonstrated safety

2. Certain death for thousands of ALS patients during the time necessary for a Phase 3

3. Statistically-significant efficacy between treated and placebo groups in biomarker data

4. Statistically significant efficacy between treated and historical control groups in clinical data

5. Demonstrated ability to regulate homeostasis

As the FDA undoubtedly appreciates, an entire generation of newly diagnosed ALS patients likely would die in the time it would take Genervon to conduct another round of clinical trials before GM604 could be approved and made available to treat ALS patients. Although Genervon acknowledges the paramount importance of public safety in the FDA’s drug approval process, it also believes that ALS presents a compelling case for an exception to the usual process. This view is of course shared by many sufferers of ALS.

However, the chance of a quick approval by FDA is very small. FDA has the mandate and mindset to know as much as possible about the disease and the new drug no matter how long it would take. But all of you being an important constituent FDA will liTsten to your voice if you choose to express it. Your expression should be appropriate and appreciative. The best contact would be CDER

You can also express your support through social media, ALS non-profit associations or other channels. 

A sample of a patient's voice: 
"It is horrifying to be told that you will die very quickly and along the way be reduced from a vibrant adult to less than an infantile state, all while retaining your cognition but losing the ability to express it. I live this way every day. Every waking moment is a fierce battle between iron determination and utter despair. For the first time in history we have a treatment with major effect and an excellent safety profile. PALS need treatment options RIGHT NOW. The risk of GM6 at this point is extremely acceptable to us, especially given that without treatment the risk is 100% fatal. We cannot wait another few more years while tens of thousands of us die. Please allow GM6 preliminary approval and market surveillance for final determination. Doing so would very likely mean rescue from ALS for many tens of thousands and certain hope for all."
Genervon Biopharmaceuticals LLC
1055 E. Colorado Blvd. Suite 500
Pasadena, CA 91106
Tel: (323)-721-5500
Fax: (323)-721-9131

This email is sent on behalf of Genervon Biopharmaceuticals, LLC. It may be privileged and contains confidential information intended only for the use of the recipient(s) named above. Use by anyone else is strictly prohibited. If you have received this communication in error, please notify us immediately. 


Friday, January 9, 2015

Follow The Buckets

It's not easy.

Already, only a few months after so many people dumped ice and donated so generously, we're thoroughly confused by press releases and announcements, especially those related to new research grants.

We have an information problem.

It should be easy.  It's 2015.

Individuals investors with even the most modest brokerage accounts can look up their portfolio components and see totals and allocations and results very easily.  It's 2015.

Individual donors with investments in ALS research should be able to do the same with every research grant made with their donations.  That's their portfolio.

  • What ever happened with the 2013 grant made the XYZ University Stem Cell Lab?
  • What's the total year-to-date new money granted?
  • How much in total has been invested in ABC Labs in the last three years?
  • What was the outcome of the ZZZ clinical trial that we helped fund?
  • What percentage of 2014 grants was related to DEF technologies?
  • How much is budgeted for research in 2015 and not yet committed?
It should be easy.  After all, it's 2015.  Today for some inexplicable reason, it's impossible.

Monday, January 5, 2015

It's 2015. We Fight Smart. Usually.

Everything we fight these days benefits from data.  And our access to data gets better every year.

When the Colts battle the Broncos on Sunday, I'll have read a lot about playoff statistics that may affect the outcome.  Sports statisticians thrive these days and make us smarter fans.

When I get my electric bill, I'll have data to help me figure out how to fight the high cost of the low temperatures here.  Smarter meters and their data let customers be a lot smarter.

When I do my laundry and fight stains, I have an appliance that helps smarten up the water usage and cycles to match the load, and it's all based on data that washing machine gathers.

And then there's ALS.  It's a life-and-death fight for thousands of people, yet we fail to capture and share data.  And the ALS data situation seems to be getting less smart every day.

People with ALS try things.  They all do.  Some things are pretty simple while others are risky and expensive.  In 1996, my 78-year-old mother read that vitamin E might help with ALS. She tried it.  Little did she know that Lou Gehrig had tried it in 1939 with the same results that she was about to have. It made no difference.  Neither Lou nor Mom left the breadcrumbs to help the next person make a smarter decision about vitamin E.  The tools just weren't there.  But the tools are there today.

ALS has a cruel way of generating a new crop of rookies every year, and today they are stuck with anecdotes and trusted advisors (some more worthy than others) to help them make decisions.  They get more real data about their football teams or electric usage than they do about what they might try to fight ALS. offers a nice framework for people with ALS to track their individual case trajectories and interventions.  It can supply others with some reference points when they try to make smarter decisions.  If patients would maintain data profiles there, it would provide those bread crumbs for the next person.

Some neurologists and ALS organizations have discouraged people with ALS from participating there.  That puzzles me.  Patients will try things with or without data.  Please, all, let the data speak.  We're not talking about rigorous clinical trial data, but we're talking about clues and bread crumbs that will help every person with ALS and caregiver be smarter.

Privacy need not be an issue.  You can use a profile name like "Lefty" even if you're a right-handed ballerina.  You can be as vague about your identity as you please.  You don't have to participate in the Forum (or any other forum) if you don't want to.  Today, there is a senseless waste of information among people who participate on message boards or social media with anecdotes and don't provide orderly data about their disease trajectories before and after intervention.

And anyone promoting an experimental protocol or other intervention owes it to everyone to ask all people trying it to post their data publicly and completely.  Yes, let the data speak.

Data waste is not smart.  We need to help every person with ALS fight smarter.  That requires data.  It's 2015.

Wednesday, December 31, 2014

The "Me" in SoMe isn't Me

It's the last day of of an incredible year for ALS in the social media.

So, let's think about what we have learned about the power of social media and the fight against ALS.  What's the first thing that comes to your mind?  Really, what's the first thing that comes to your mind?


Naw, you were thinking Fundraising, weren't you?

Has the idea of buckets of money made us myopic about the higher usefulness of social media to defeat ALS?

Has brand management overshadowed the power of ideas and interaction and accessibility that social media provide to a diverse population with physical challenges?

It shouldn't be all about the brand and fundraising.  It's not all about the organizational "Me."  It's about a very important "You" -- people with ALS and their caregivers.

Yesterday an apparently helpful tweet from an organization offered people newly diagnosed with ALS a link to information.  Upon clicking they were greeted with the year-end popup to donate.  Whoops.

Paul Wicks of PatientsLikeMe recently wrote an interesting piece on the Ice Bucket Challenge and ALS --

There's a lot to consider there.  The idea of organizational curation of Wikipedia information about ALS jumped out at me.  What a way to step forward and help maintain some quality in information that people find every day!  Some organizations don't have time?  Have we completely lost our vision?  You can be darned sure they would have time to take care of a Wikipedia entry on the organization itself.

The fight against ALS needs to turn itself inside-out.  It's simply going to take a major attitude change about the "Me" in SoMe.

Tuesday, December 16, 2014

Should This Disease Be Forgot?

A few weeks ago on a national NPR program, a caller mentioned last summer's dumping of buckets of ice and said, "Uh, what was that disease?"  Time passes on.  So do memories.  The disease that was on the world's mind and lips in August is fast fading back to its shadows.

There are year-end memories of the entertaining ice and the videos.  There are year-end memories of the buckets of money that were donated.  Who is talking about the outrageous disease that motivated it all -- ALS?

Picture a football stadium full of people.  Imagine that many people being killed by ALS.  Yes, that many people have been killed by ALS in the few months since we dumped ice on our heads.

During the heat of the ice bucket challenge, I made a series of very modest online gifts to a number of ALS charities (the kind at add up to hundreds of millions of dollars when people everywhere give them).  I got the autoresponders.  I got a few nice letters.  I got my tax receipts.

I've not gotten any insights into ALS.  I've not gotten any year-end "thanks and here's what we did with your contribution" messaging.  Were I not previously involved with ALS, I've not been given any reasons to stay committed to the disease.

ALS charities have email addresses from all of those who gave spontaneously and generously online in August.  Today's donors don't want constant requests for more money, but perhaps they would like some reasons to continue an interest in ALS.  Perhaps they need to be thanked again and again so that they know that the disease persists and must not be forgotten.

Awareness is critical.  It's a long-term proposition.  The success isn't in hundreds of millions of dollars. The success isn't in the entertaining viral videos. The success isn't in the media coverage that talks about dollars but not the disease.  True success will be in the awareness and relationships that will finally give the world the resolve to deal with this outrageous disease.  That awareness needs to be cultivated.

And today we already have people saying, "Uh, what was that disease?"

Thursday, December 11, 2014

The Ears Can Be Deceiving

If you've ever been owned by a Basset Hound, you know that the big ears can be deceiving.  Bassets aren't always the greatest listeners.  Why the big ears?  They're more about stirring up the scent for their noses to track things than actually to listen (especially to listen to their human beings who sometimes have things to say that are inconvenient to hear).  I love my hound but am realistic that's she's smart when she wants to be.  And listening isn't her forte.

We're at that time of year when the ALS Association is conducting its "listening tour" for public policy priorities for next year.  It consists of an email sent to chapters that has not changed a lot over the years. Chapters have a short due date to reply to the email with their three priority picks.  That's it.  Some chapters reach out to people with ALS and caregivers and their chapter benefactors.  Others don't.  They pick three and reply.  It's traditional.

Below is some text from this year's email.  In 2014 we have some wonderful social media tools that would cast the nets more broadly and generate some energy and ideas beyond pick-three.  They would make it much easier for everyone truly to listen.  Ah, the same tools that were so effective for engaging people to dump ice and be interested in ALS in August would also work for them to hear and be heard in December.


The ALS Association Public Policy Department is currently in the process of working with local chapters to identify the issues most important to the ALS community nationwide.  Feedback received will help to inform our 2015 public policy priorities.  Please take a few minutes to review the following public policy issues.  I’ve included links for ease of finding information on the topic. 
Afterwards, we ask that you IDENTIFY and RANK your top THREE public policy issues and submit your responses to us by email.  You may reply directly to this email.  We need all responses by Monday, December 15.  Thank you for your participation!
Public Policy Issues
  • MODDERN Cures Act
  • PDUFA implementation
  • Compassionate use programs
  • Drug development and approval process
  • “Right to Try”
2.      Health Care Reform Issue may include:
  • Ensure key provisions of health reform enacted in 2010 remain law, including:
    • Closing the Medicare prescription drug benefit coverage gap
    • Eliminating preexisting condition exclusions
    • Eliminating lifetime and annual caps on health insurance coverage
  • Promote respite care and respite care funding
  • Implement the CLASS Act, legislation included in health care reform enacted into law in 2010 that establishes a national program to enable people to purchase long term care services
  • Provide Medicare and private coverage of in-home custodial care services and home health aides
  • Change Medicare’s homebound requirements
  • Reimburse/health benefits to family caregivers
  • Establish caregiving tax credits separate from the deduction for medical expenses
4.      General Medicare Issue may include:
  • Eliminate therapy caps and/or extend the process to receive an exception to caps on coverage
  • Streamline and speed processing of claims
  • Improve coverage of Durable Medical Equipment
  • Provide Medicare coverage for iPads, iPhones and other similar devices
  • Oppose implementation of/repeal Competitive Bidding for DME in Medicare
5.      Medicare Prescription Drug Benefit (Medicare Part D) Issue may include:
  • Implement provisions of health reform enacted in 2010 that eliminate the coverage gap
  • Ensure Medicare coverage of specific prescription drugs (Rilutek is currently covered)
  • Ensure Medicare coverage of drugs prescribed off label
  • Limit the use of utilization management tools such cost sharing, tiering, therapeutic/generic substitutions, quantity limits and other policies of Medicare prescription drug plans
6.      National ALS Registry Issue may include:
  • National Office to continue support Chapter outreach and enrollment activities by providing materials, equipment, education and training
  • Continued funding for the National ALS Registry
  • Expand the number of surveys in the registry
  • Encourage research use of the registry
  • ­Continued funding for ALS research or policies and programs that advance ALS research at:
    • the ALS Research Program at the Department of Defense
    • the National Institutes of Health
    • the Food and Drug Administration
    • the Department of Veterans Affairs
    • the Centers for Disease Control and Prevention
  • Stem cell research including expediting human trials
8.      Speech Generating Devices Issue may include:
  • Ensure access to non-speech functions such as email, internet access and environmental controls
  • Capped rental
  • Eye-gaze technology
  • Eliminate the 5-month waiting period
  • Eliminate the “20-40 rule” under which a person with ALS must have worked five of the previous 10 years in order to qualify for SSDI
10.    Veterans Issue may include:
  • Assistance navigating/accessing VA benefits
  • Change functional criteria and other elements of the adaptive housing grant process
  • Establish a formal process to expedite claims
  • Develop partnerships with VA for clinical care
  • Increase grants for adaptive housing and accessible vehicles

Monday, December 8, 2014

#alssymp Days +2,+3 -- Research Symposium

Here are more stream-of-consciousness notes from the second and third days of the big research symposium.  

Attendance was inversely proportional to the time lapsed from the start of the conference.  The dropoff in attendance was pretty stunning to me, especially for a meeting with a such a substantial registration fee.  The benefit of the smaller crowd on day 2 (and the much, much smaller crowd on day 3) was that there was that people became much more congenial and interactive.

There is a definite distinction between the clinical track and the research track and I’m glad we were free to jump back and forth. 


On the morning of day 2 I decided to give my brain a much-needed stretch by attending the sessions on in-vitro modelling rather than the care-practice sessions (which included a lead speaker on e-health, a topic that I am very interested in).  It’s the old dilemma of trying to be in two places at once, although I’m glad I did the brain-stretching.  I was able to listen and soak in a lot of the principles without getting lost in the weeds. 

Speaking of weeds, I learned a lot about iPS models and can really relate them to agriculture.  We heard from Dr. Eggan in much more intricate scientific detail that sometimes you get what you planted, sometimes you get some weeds, and sometimes you grow something a little different from what you intended.  All those possibilities have to be evaluated for the model to make sure you have what you thought you were making.

And in the iPS models, you can try things, but you don’t have a patient who can say, “That helped,” or “That makes me feel terrible.”  These models force the biomarker issue.  So the process is complicated.

He showed some work that they have done with lights that cause TMS excitability in the model, giving new significance to the flashing blue light. The activity has led them to want to test an epilepsy drug, Retigabine, in humans with ALS.  They will not only look for efficacy but also will be able to compare the activity in humans to help validate the model itself.  

I followed another talk by Dr. Bakkar on RBM45 and KEAP1 remarkably well, but I kept wondering how long all of this intricate modeling took and how much did it cost.  I believe that it’s essential that we have this kind of basic science funded to keep the pipeline full for later clinical trials, but it has to be a tremendously expensive endeavor, and nobody really talked about the prices.

And speaking of the expenses, every presenter had a wonderful slide of acknowledgements at the end of his or her Powerpoint deck.  Colleagues and funders were appreciated and thanked.  A big step in a better direction would be if every presenter had a big thank you at the very top of each acknowledgement slide thanking the people with ALS and their families who made their work possible – by volunteering for studies, by being the rainmakers who raise large amounts of funds, or by being the advocates who make sure that ALS and MND are on national agendas.  The few times I heard the people with ALS thanked at this symposium, it was almost like an afterthought. 

The next sessions I chose were on epidemiology. During the afternoon, I heard valuable human volunteers for studies called “subjects” and “controls” repeatedly. Kind of like some royal underlings and television remotes.  Those terms need to go.  They reflect an attitude that hardly reflects respect for those who are the most valuable intrinsic contributors to projects.

The presentation by Dr. Liying on the first epidemiological study on ALS MND in China was fascinating. One of the lead slides showed the vast range of prevalence numbers from studies in other countries. I think we really need to question whether those differences are real, are a result of our inabilities to count noses well, or are perhaps from diagnostic differences.  The Chinese study showed much less C9 ALS than in the US, and a younger onset.  As the genetics get more complicated, I think that the geography of the genetics gets very interesting.

Dr. Weisskopf’s study looked at military data, this time from death certificate data from veterans of various eras.   The data indicated that WW II veterans were most affected by ALS, yet something in the back of my mind says that WW II vets have also generated more death certificate than later veterans.  I’m sure more is to follow.  This study was one funded by ALS Registry funds but is separate from the Registry and did not use the Registry or the VA Registry for any of its data. I was able to ask a question that I have asked over the years, and I think I got the right answer this time – Have there been studies of military of other nations besides the US?  Yes, there was a French study, but it had some significant flaws.  Weisskopf is working on a study of Denmark’s military as we speak.  I look forward to those results.

The presentation that got the blockbuster tweets was the Fournier study on people with ALS who had experienced head trauma.  It showed no correlation between head trauma and the rate of decline in those people with ALS.  All of the people in the study already had ALS, so it was simply about decline among those already with ALS and not about head trauma causing ALS.

We moved on to a session that demonstrated an ALS “survival score” that included creatinine in its variables.  Later Dr. Mitsumoto did a biomarker presentation that involved good old serum creatinine.  I’m sure that all people with ALS have lots of creatinine data in their old medical records.  If only we had ourselves organized so that every person with ALS can opt in to having medical data used for research.  Also, I noticed that Dr. Mitsumoto always referred to sporadic ALS as “so-called sporadic ALS.”  Hmmm.  The plot thickens?  And Dr. Bowser talked of the huge gap between the discovery of suspected biomarkers and the validation.  And I learned about microfilaments.  There were lots of biomarker candidates that were framed as "promising."  There's that word again.


I started the day in some nutrition sessions (although the concurrent twitter buzz told me that there was significant action in the other track with the Beckman presentation on copper chaperones). The feeding tube discussion in the nutrition track gave data but really didn’t break new ground for me.  I did appreciate the dysphagia presentation by Dr. Plowman.  She made it personal, and I think that was a refreshing entrance to her data.  I know that the clinical studies are important in proving hypotheses with data, but sometimes I think that was a lot of time and money to tell us something that we already know.

Later I moved back to the research science track and was fascinated by the non-neuronal cell presentations.  Now I know that those scientists have produced some imaging that shows stunning differences (even to my untrained eye) between those with ALS and those who do not have ALS.  There were also a very interesting so-what challenge from the audience regarding one of the imaging studies.  The questioner asked why it was important if it only showed what the clinician was able to observe with the patient anyway.  That’s when I realized that for every presentation, there should be a “what difference does it make tomorrow” closing.

I am very grateful for the recap of significant presentations that was given to us at the very end of the conference.  That helped me know about what happened in the tracks that I couldn’t attend, and it also helped me see which presentations stood out to the scientists.  We also got wind of a new ALS gene and I’m sworn to social media secrecy until it is published soon.

Some Reflections

Social media can add a lot to the conference experience for those who choose to do so.  There were some tweeters of all backgrounds who added a lot to the understanding and tone of the meeting.  Click here for the people who made the official #alssymp tweeter list.  I was also immensely grateful for the good wifi in the facility.  It let me google at will when I heard a foreign term or wanted to look up a name.

So What
The symposium represents a huge investment of time and money.  At the end of every presentation, I think we should have the “so what” discussion.  What will we do differently tomorrow because of these findings.  If that question isn’t easy to answer, then I think it's fair to question some priorities.

The word came up repeatedly when I chatted with the daughter of a man with ALS who took it upon herself to attend.  Her life is built around urgency.

Back when we were dealing with ALS, I found it frustrating that people we depended on didn’t move quickly or with purpose.  I got the same sense of frustration with the feeling of pace, progress, and urgency at the symposium.

Patches, And No Way To Make A Quilt
I think that the single most significant improvement that we could make in the fight against ALS would be for the ALS MND Alliance of organizations to have a shared information policy to insure that any granted dollars come with an agreement that study data adhere to some standards.  That’s the only way there will ever be potential to put 2+2 together from these discrete study successes.

It would also be an improvement to insure that all granting organizations have data on grants, amounts, recipients, outcomes, etc. that are consistent and transparent and searchable by the public.

At the symposium we saw three days’ of scientific study accomplishments with no common threads for information synergy.  It’s the ultimate silo system.

We have an ALS information crisis and it needs to be fixed, and great neuroscientists are not necessarily great information scientists.

“We Speak A Different Language”
I had a conversation with a lovely woman who had presented at a prior symposium.  I spoke of my feeling of needing to have PALS, CALS a part of the fabric of such a symposium.  She disagreed strongly.  She said there was a person with ALS present when she spoke and she was not comfortable with that.  Besides, she said, “We speak a different language.”

Perhaps that is precisely the reason why people with ALS and caregivers need to be there!

Can you imagine the perspective and clarity that interested people with the ultimate skin in the game would have for these scientists?  Can you imagine the light that those living with the ticking clock could shed on vying priorities?  Can you imagine how much the scientists could learn from the questions and comments of people with ALS and their families?  Can you imagine how much more powerful advocates people with ALS and their caregivers could be for clinical science with more knowledge? Can you imagine? 

I can.

Saturday, December 6, 2014

#alssymp Day +1 -- Research Symposium

The first big day started in a lovely convention center.  After several introductions (including an appearance by the Prince... not the singer, but the real Prince) the presentations began.

The first general presentation was by Dr. Sandrock of Biogen IDEC.  He spoke of clinical trial design and some better principles for Phase II trials.  Some of what he said reminded me of what a gentleman with ALS suggested several years ago -- larger, more definitive Phase II trials.   He also alluded to a preliminary observation period.  I see the value in that, but why eat up more clinical trial time.  I still think that on day of diagnosis patients should have the option of opting in to having their data available for clinical trial use.  That would give any trial a trajectory of any patient's ALS before the start of any trial therapy or placebo.  And we learned at other sessions yesterday how important and variable patient trajectories are with ALS.  I really have trouble with the word "progression."  Sandrock gave a summary of the characteristics of an optimal Phase II trial.  (The slide is in my twitter stream and unfortunately I am not able to post pictures in Blogger from this connection.)  Unfortunately without reliable biomarkers, the characteristics that he described can't be delivered.  I'm pessimistic that we'll see another trial from Biogen Idec until that kind of trial can be designed and delivered.

The first breakout I attended included Dr. Benetar speaking on diagnosis.  There is an approximate 12-month diagnosis delay for people with ALS and that has been the case for more than a decade.  That is outrageous in my opinion.  He indicated that much of the delay is attributable to a period between the primary care physician and getting the patient to an ALS neurologist.  I wish they would crowdsource this sometime and find the real reasons.  I suspect that could reveal some of the weaknesses of American healthcare delivery, including inept scheduling.  He showed data on interventional delay, too. Again, I wonder if that's not as much an indictment of American healthcare delivery as physician behavior.  Have you ever tried to get a riluzole prescription and then get it filled?  Benetar showed some disease trajectories and showed what many of us have also observed -- ALS trajectories are often not linear as neurologists often describe.  Some patients have a linear decline followed by a sudden dropoff.  They hit a wall.  It was nice to see some data that admit that.

The next presenter was interesting, but so help me, I thought I would scream when I heard clinical trial volunteers called "subjects."  Time to move on from that term.  And while we're at it, "controls" is a pretty disrespectful word for people who volunteer their data and parts of their lives for medical research.  Let's use references that respect all who volunteer for clinical trials as team members who are as valuable as those paid to do the research.  And while we're at it, every acknowledgement slide at these meetings would do well to thank the patients first.  I'm glad that at this meeting the importance of the data from those people who volunteer for the control group has been emphasized.

Dr. Schoenfeld gave an interesting presentation on trial design when the treatment being tested poses some potential harm in its delivery mechanism and therefore puts placebo patients at risk.  That's a big deal in the world where stem cells and other things are being placed in bodies.  He suggested that an add-on trial design could be a solution to some of the placebo dilemma in these cases.  And again the concept of an observation period in a trial came up and I ask why we aren't tracking all with ALS from day of diagnosis.

Dr. McGrath from Neuraltus spoke on the biomarker that they were tracking in the NP001 trial.  I was interested in this because some friends participated in this trial and crashed during the washout period when the biomarker was being tracked.  He showed the LPS+ data they tracked and how responders' data did correlate.  I asked about whether they would design their Phase II trial differently if they knew then what they know now.  That washout period with no further therapy was cruel to patients who had responded well during the therapy period.  He indicated that the trial design was based on budget.  They had not funds to do anything more than the six-month trial regardless of whether the drug worked or not.  OK, lesson learned, folks.  Every patient who volunteers for a trial needs to ask, "What happens to me if this stuff really works?"

My day moved on to some presentations on communication devices (while I watched Steve Gleason's entertaining and effective twitter stream on communication device comments to CMS).  There was a brain-interface presentation that interested me, and I learned that this kind of brain interface requires some gazing.  I wonder if some of the problems that people have with today's eye-gaze devices will carry forward to that technology.

I hope that all of the poster presenters will upload their data to the sharable site where we can view in more detail and with more energy than we had at the end of a long day.

Your best tidbits from this symposium will come from following #alssymp on twitter.