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.

Thursday, August 21, 2014

National Discussions Only Work If We Talk About the Answers to the (Impolite) Questions

On Monday in the Los Angeles Times, Michael Hiltzik wrote "A Few (Impolite) Questions About The Ice Bucket Challenge."

With all of the marvelous visibility given to ALS, I hope that our organizations will embrace all  questions and be part of a very public and continuing conversation about ALS.

One part of the article jumped out at me --
But ALS is also, by any definition, a rare disease; the threshold specified in the federal Rare Disease Act of 2002 is a nationwide incidence of 200,000 patients. The CDC estimates the prevalence of ALS in the U.S. at about 12,000 persons. The ALS Association says 30,000, but hasn't responded to my inquiry about the discrepancy.
I hope that the ALS Association has responded by now, and if they have, we would all benefit from their insights.  If they've not, I'll take a stab at it.  Forgive me if I sound like a middle-school math teacher, but it's my comfort zone.

Yes, legally, rare diseases are those "affecting fewer than 200,000 people in the U.S." "Affecting" is usually interpreted as "having it at any given moment."  That's prevalence.

Let's consider a factory.  The assembly line produces things and the warehouse holds things.  Prevalence is like the warehouse.  Incidence (the number of people diagnosed with a disease in a time period) describes the speed of the assembly line.

ALS is a high-incidence (you're very likely to get ALS -- as likely to get ALS as MS), low-prevalence (you're not likely to be alive with ALS) disease.  ALS has a fast assembly line and a very small warehouse.  What happens to everyone who doesn't fit in that small prevalence warehouse?  It's not good.

Now for the discrepancy.  All ALS numbers in the United States are extrapolated, based on smaller studies.  We don't have mandatory reporting of ALS by physicians.  Nobody is really counting every nose.  And you can sense that with the fast-moving numbers in and out of that prevalence warehouse that the numbers don't stand still very long.

The 30,000 number is a swag.  It has been used by organizations for decades.  In perhaps the only area where ALS organizations have cooperated and shared data, they have been complicit in propagating a 30,000 number that has been convenient but not well-rooted in reality.  ALSA has actually been quietly distancing itself from the 30,000.  Why?  As I said, nobody is counting noses and some people have a fixation on prevalence as being the important number.  It's not.  It's a one-legged data stool.  If you talk about a small warehouse without paying any attention to the speed of the assembly line, you miss the boat on the impact of a disease (or as I sometimes call ALS, a "diesease").

We're pretty sure that around 5500-6000 Americans are diagnosed with ALS every year.  Arithmetic tells us that if they live an average of 3 years after diagnosis, then we're talking around 16,000-18,000  Americans alive with ALS at any moment.  If they live an average of 5 years post dx, then maybe the number could approach 30,000, but that's a stretch.

Now for that 12,000 number that was published in the recent CDC ATSDR ALS Registry report.  This gets interesting.  We have paid over $40 million taxpayer dollars for a national registry and this was the first published report.  They gathered data from Medicare (people with ALS usually go on to Medicare quickly), Medicaid, and VA files along with a self-enrollment web portal.  They identified just over 12,000 people alive with ALS in the U.S. in a one-year period. Ugh.  How many Americans were living with ALS in that period who were not in those government files and who did not self-enroll?  We simply don't know.  How complete was their counting technique?  We just don't know.

And the clunker in all this is that the Registry did not address incidence (that pesky assembly line speed) at all in that period.  It gave us a one-legged data stool of a very low prevalence with no other supporting incidence or mortality data.

ALSA is obviously proud of getting the ALS Registry project passed.  I so hope that they have answered Mr. Hiltzik's question by now.  If not, you have my answer.

And with ALS in a wonderful national spotlight right now, what better time is there to talk of the correct numbers correctly?  ALS is a high-incidence, low-prevalence disease, and that's a troubling combination that should make every American squirm.

#ALSicebucketchallenge  #icebucketchallenge  #strikeoutals

Wednesday, August 20, 2014

We Have Rare Publicity for a Not-So-Rare Disease Experience

This morning I unwrapped the morning paper.  On the front page was a huge picture of the head of the IU Health ALS Clinic as the ice water hit.  That was the second time in two weeks that a nice picture mentioning ALS has been on the front page of the Indianapolis Star.  That was the second time in two decades, for that matter.  It as been an amazing few weeks for ALS awareness.

I will admit a bit of envy that I couldn't be there to help dump the ice.  My 18-year-old memories of the neurologist are complicated.  We had the confidence that we were getting the best care possible for Mom.  At the same time, I would have given anything to be able to dump ice water on a healthcare delivery system that was inefficient and slow and that lost the beloved patient.  This morning I wondered if the doctor would remember Mom.  She was special.  There can't have been that many ALS patients in Indiana, right?  Wrong.  Since he saw Mom, he has probably lost well over 2,000 ALS clinic patients.  He never has all that many alive and in his care at any moment, but the turnover is brutal.  Most are in his care for a very short time. Would he even remember Mom amidst the 2,000 others?  The numbers lost to ALS defy the concept of the word "rare."

The television is on in the family room right now.  They just told of a big #ALSicebucketchallenge that will be on the noon news today.  Every day this week the local stations have mentioned ALS.  

And yesterday one of our local radio sportscasters did one of the best  #ALSicebucketchallenge videos ever.  I share that with you here and ask that you might pass this along to people who don't know much about ALS.

It has been some rare publicity for a disease that people need to understand.  It's about time.  

Tuesday, August 19, 2014

As We Ride A Marvelous Wave of ALS Awareness...

We simply don't want it to ever end.  What a few weeks it has been.  People are finally saying, "ALS."  Soon we need to get them to talk about ALS.  In the meantime, we'll enjoy this marvelous wave of the #ALSicebucketchallenge or #icebucketchallenge and hear the rich and famous and the rest of us mention ALS in every form of media.

It also proves the point that ALS leaves a broad wake of people who have lost loved ones, friends, neighbors, co-workers, acquaintences, or friends-of-friends to the disease.  Those people are all being called by the #ALSicebucketchallenge .  ALS organizations have not engaged them well in the past, but this is proof that we need to provide ways for them to help make noise about an outrageous disease.

This wake was built by the fact that every day over 350 people die from ALS globally.  Every day.  Yesterday, today and, yes, tomorrow.  This goes on relentlessly, seven days a week.  No weekends off for ALS.  The tragic destruction persists as we bask in the ice glory.  We must not forget.  We must not break our arms patting ourselves on the back for a social media phenomenon.   Part of the explanation is the huge wake of destruction that ALS has left over the decades.

We must make a substantial change in the fight against ALS so that this disease is finally stopped.

There is much to do, and it's a blessing to know that so many powerful and influential people care.

Thanks to all.


p.s.  There are two important action items that unfortunately have been buried by the ice fun.  If you can help via your personal action and social media reach, it would be appreciated.

1.  There is a Medicare policy issue that has a terrible impact on people with ALS who need devices to help them communicate.  Please read this link and consider and act, and please spread the word to your social media reach.

2. There is a study enrolling that needs volunteers with ALS who are able to get to Boston.  Information is at this link.  Once again, please help spread the information.

Thank you.

Saturday, August 16, 2014

We Now Have Our Celebrity Spokespeople, Thank You!

ALS has been a forgotten disease for decades.  There has been an abysmal lack of awareness and understanding.

So many diseases have had charismatic A-list celebrities raising visibility.  Our cause had lesser celebrities who read scripts and had no inner fire, and they came and went.  And when they went, they really went.

Three weeks ago when I first read of the ice-bucket dumping, I rolled my eyes.  Fine fun, but we have serious work to do.  I've grown weary of trying to deal with the serious problem of ALS with bake sales and cute fundraisers in a world that can't even remember the name of the disease.

Then in a few hours you could see the #ALSicebucketchallenge traction start.  It was people connected to Pete Frates.  I thanked God that people who played ball or went to school with Pete Frates obviously loved him.  They would walk through walls or dump ice water on their heads for him.  If you've ever met Pete, you would understand why.  And there were more connections to Pete and the love for him spread with a special energy.  And the city of Boston and the state of Massachusetts showed us a special kind of loyalty to a local hero.

And then even more connections started via another young man with ALS, Steve Gleason.  He, too, is beloved and has a special kind of charisma that you see in a smile that ALS hasn't stolen.  And the #ALSicebucketchallenge kept growing.  And the city of New Orleans and the Pacific Northwest showed us special loyalty to their hero.

In the past three weeks, we have seen the best celebrity spokespeople ever in the fight against ALS.  And they brought along their friends Ethel and Satya and Martha and Bill and Oprah and so many others. 

The irony of this is that Pete and Steve can't "speak" the same way you and I do.  It's not so easy for them, but thanks to technology, they are shining stars who can lead the discussion about ALS and show the world how important it is that we all pitch in and fix the problem.  

Thanks to today's technology and social media, we have the best celebrity spokespeople ever, and they have delivered a lot of friends to the fight, and we thank them.  

Friday, August 15, 2014

We Interrupt A Week Of Magnificent Ice-Dumping For A Few Thoughts

And what a week it has been.  Who knew that the letters A-L-S would be said by top celebrities, heads of corporations, politicians, elite athletes, (and tens of thousands of us who have lost loved ones to ALS) as we watched entertaining videos of ice water fall over heads?

And when Ethel Kennedy took the ice, we just knew that good things would follow.

It has been a good week for ALS awareness.

But we have much more to do.

  • What happens to the windfall of public generosity?  We hope that it will enable an infusion of radical new thinking in the fight against ALS.  Perhaps the two young men with ALS who got it all started, Pat Quinn and Pete Frates, would be good advisors for some "think different" uses for the generosity of so many.
  • Not everyone who has basked in a video has conveyed ALS awareness.  We hope that a communications person from one of our national organizations has had a chat with Brooke Shields and Wanda Sykes by now.  Obviously the ice doesn't magically impart an understanding of ALS.
  • Unfortunately the ice-dumpers have much higher odds of getting ALS than most of them would ever imagine.
  • And no media interview should ever forget to mention Pete Frates and Pat Quinn.  Ever.

Some friends have asked me about charities that might be like the small, high-impact charities that Bill and Melinda Gates often mention.  I'm about as far as it gets from Mr. and Mrs. Gates, and there are many good charities in the ALS space.  Many are woefully underfunded.  My new favorite question is, "How will you put my $100 donation to work in the next 30 days?"  Sometimes the answers aren't glamorous, but every charity should have a direct answer for you to measure next to your goals.  And certainly, Google and 990s are your friends.  Here are some thoughts.

This is a not-for-profit biotech lab focused solely on ALS.  They have a specific, new research project that could use your $100 donation.  It's immediate and important for advancing the science.

The Gleason Initiative Foundation
Most of us know about "No White Flags," but few know that behind the scenes, Team Gleason works hard on issues of enabling technology for people with ALS. Your $100 can help supply technology to people with ALS in need.

And certainly there are more.

Not-for-profits are risk-averse, and it's time for us donors to start investing in and demanding some smart risk.  The fact is that what we have done in the past has failed to deliver a treatment for ALS.

And never forget Pete Frates and Pat Quinn

Holy moley, it's time for GMA and more ice buckets.  Will David Muir's hair go next?

#ALSicebucketchallenge  #StrikeOutALS

Wednesday, August 13, 2014

If You Can Read This, Thank An Internet Connection

Finally we have an action item regarding the Medicare rules changes for Speech Generating Devices.

The following is from the Center for Medicare Advocacy:

Recent changes to Medicare are drastically reducing the ability of the most vulnerable people with disabilities to communicate.
As of April 1, 2014, Medicare began denying payment for many of the medically necessary speech generating devices used by people with ALS (Lou Gehrig’s Disease), Cerebral Palsy, Spinal Cord Injuries, and other impairments, when they enter a health care facility, such as a skilled nursing home or hospice. Taking these highly specialized devices away leaves them no way to communicate at a vulnerable and terrifying time.
On September 1, 2014, many severely disabled individuals will have all contact with the outside world cut off. For many years, Medicare allowed individuals using Medicare-provided speech generating devices to use their own funds to "upgrade" the devices. This allowed them to communicate beyond the confines of their room through email, internet, and text messages. After September, Medicare will no longer pay for any device that has the potential to be upgraded to allow communication outside the room 
Currently, Medicare routinely denies coverage of the critical eye-gaze technology necessary by some people to operate these speech devices, even when its medical necessity is well documented.  They have no way to communicate as a result. After years of waiting for an appeal to Medicare, the eye-gaze coverage is routinely allowed, but individuals should not be forced to wait years without a voice.  
We need your "voice!"
Please click on the link above and take action.  There is a simple tool to write your Congressperson and your two Senators and Health and Human Services Secretary Burwell.
Please act (and be thankful you can).  The fact that you can read this means you understand the value of technology and the internet for communications in the 21st Century.  Please don't let basic communications be taken away from people with ALS.
Thanks to the Center for Medicare Advocacy and to Team Gleason for taking leadership!

Monday, August 11, 2014

Let's Challenge. Let's Think. Let's Get Lean.

The annual CDC ATSDR ALS Registry annual meeting will be webstreamed starting on Wednesday.

That's a big deal.  In the past it has been a secret until the report is published many months after the meeting.  This year we get to watch.  That's a baby step forward to some transparency.

There is a lot of serious business to discuss with this project.  We hope that those invited to be at the table and interact will be prepared and willing to ask some important questions.

Let's get the business arrangements out in the open up front.  Based on past meetings, the participants are largely employed on the project, either by ATSDR or large contractors on the project.  There have been few, if any, voices without such ties in the room.  Any patient representatives have been chosen by ALSA and MDA, both large, paid contractors on the project.  How about some disclosures?

Let's have some serious discussions about "the report," and what it says and what it didn't say.  We trust that participants will have read Stephen Finger's op-ed by now.  Let's talk about his observations on the quantum leap that was implied to the public about the completeness of the data in that report.

And will we ever have incidence data from this project?

And was there a communications plan beyond "the report" to talk about the correct numbers correctly?

And was the passive surveillance as complete as we thought it would be?

Let's get businesslike about the budget.  Last May we learned from the ALSA presentation at their Advocacy Conference that the amount requested for the Registry was simply the amount that the sponsoring legislators thought they could raise.  It was not built from the ground up based on a business case.  The core mission of the project has simply been overfunded.  Some may think that's great.  Others think that because a government agency has a lot of money to spend doesn't mean that it is the best place to do things.  Please have some serious challenges of the scope creep on this project.  And before the next dime is spent outside of the core mission of ALS surveillance, let's have a plan to evaluate effectiveness of every one of these side ventures via meaningful success metrics.

Let's challenge every added bell or whistle by asking, "Is this the best place to do that?"  Clinical trial matching?  Storing biosamples?  Continuing education?  Clinic finder?  Additional research project funding?  Microsites?  If it's not, let's not!  We're throwing government money at problems rather than designing and executing solutions strategically and effectively.

Let's stop being blinded by the wrong project metrics.  The number of emails sent regarding a small subset of enrolling clinical trials says nothing of the effectiveness of that tool.  The increase in uptake on risk-factor surveys means nothing if the overall self-enrollment is down.  Let's look at candid and pertinent project metrics.  And when something goes plop, let's be honest.

This project could use an annual meeting with more of a spirit of Kaizen than the love-fest that it has been in the past.  Let's challenge.  Let's think.  Let's get lean.

And that's hard to do in an overfunded government project.

Wednesday, August 6, 2014

Make That Six

Yesterday @phlu tweeted a link to an interesting article on "Five Diseases That Are Scarier Than Ebola."

It points out the real and much more likely threats of

  1. Influenza
  2. HPV
  3. Measles
  4. Antibiotic-Resistant Infections
  5. Foodborne Illness
There's a sixth that belongs on the list - ALS.

We need to be talking about the real and likely threat that ALS poses to healthy people.  We need to discuss the recent ALS Registry report and what it says and what it doesn't say publicly and intelligently.

And the American public needs to squirm.  ALS is not an unlikely threat.  And you can't avoid it by washing your hands.  

Thursday, July 31, 2014

Dear Smart People At Microsoft,

We've been reading about your work with Steve Gleason at your Hackathon this week.

You inspire us.

We know now that you've met Steve that he inspires you.

Let me introduce you to thousands of other people living with ALS today.  They will inspire you, too.  Some are even younger than Steve, and others are much older.  Some have great support systems and others have none.  Some have means, others nothing.  All are smart. Some have had the disease for a few weeks, others have been at it for months or years.  The turnover is brutal. All lose the ability to do something every day, and those abilities never come back.  All need the help of enabling technologies.

You'll follow Steve's difficult path now that you've met him.  As you think of him and read of his remarkable work, please think about some of those others, too.

ALS runs a difficult, out-of-control path that steals abilities.  Many of us who dealt with ALS in our families found that we were constantly throwing the ball five yards behind the receiver when it came to assistive devices.  The disease runs a fast downhill course.  There is no time to learn and adjust.  You just have to do it.

I remember the days (before you were born) when the first person in the office to get a PC had to go through weeks of training to learn MS-DOS and then the applications.  Thank heavens those days are over.  Those days need to be over for enabling technologies for people with ALS, too.

Thanks so much for what you are doing.  Steve is leading a cast of thousands of diverse people with this evil disease.  You can empower and enable them all to be productive contributors and lead their lives in dignity.  You get it now.

Tuesday, July 29, 2014

We Don't Know What We Don't Know

Many thanks to Dr. Kevin Horton of ATSDR for his assistance with my questions in my post from July 26, "Who are the 15 Percent?"

The bottom line is that "the 15%" in the recent Registry report does not represent those people with ALS who are not located via the administrative databases.  It only speaks to the quality of what is found within those Medicare, Medicaid, VA files.

The recent Registry report does not address the completeness of the ALS Registry in finding all cases of ALS in the United States.

So how did I (and some others) get a completeness expectation?  I felt foolish yesterday that I completely missed the boat by thinking that the Registry was designed to find around 85% of people with ALS via the passive mining of administrative databases.

But, yes, there had been assertions early on in the Registry approval process that the administrative files would contain all but 15-20 percent of cases of ALS in the Unites States.  Yes, per Dr. Horton, some original pilot sites indicated that they could find more than 80 percent of PALS via administrative files. We weren't hallucinating.

Take a look --
... the CDC has created an algorithm that will enable the Agency to accurately identify approximately 80-85% of ALS cases in the United States by examining large national administrative databases ...
...In order to identify the remaining 15-20% of cases, the CDC has launched an on-line web portal that allows people with ALS to self-enroll in the Registry via a secure website...

Now I understand that today, the data in the Registry report did not swing at this completeness nail at all (that I certainly had my eye on).   They did not address how many people with ALS would potentially be outside of the administrative data sets.  Per Dr. Horton, they will do future analysis on this.

Stephen Finger has a graphic in his op-ed that speaks volumes --

His point is important, especially since the Registry report is making headlines without any important context of completeness.

We have a Registry of people with ALS who are in "the system."  They were either in administrative databases or heard and responded to the call to self-enroll.

We have no idea of how many other people with ALS are living in the United States today.

We simply don't know what we don't know... and that's important to understand.

Saturday, July 26, 2014

Who Are The 15 Percent?

Note:  I goofed in my original numbers published on Saturday, July 26.  I apologize. Administrative databases contained 8472 PLUS 1789 = 10261 people alive with ALS.  The questions are still there, though.  Corrections made on Monday, July 28.  


One premise of the CDC ATSDR National ALS Registry was that by passively mining data from Medicare, Medicaid, and VA files, they could identify around 85 percent of ALS cases in the United States.

People with ALS go onto Medicare much more quickly than people with other diseases.  The premise seemed reasonable.

These administrative databases cover approximately 90 million Americans, and the algorithm identifies 80 to 85 percent of all true ALS cases when applied to these databases. 
We thought the files would only be missing around 15 percent of Americans with ALS.  Those were the people who could be counted only if they self-enrolled in the online portal.

Question 1 --

When the first Registry report came out this week, I was anxious to see how many of the 15 percent of people with ALS who are missing from these administrative databases took the initiative to self-enroll at the Registry web portal.

They found 8472 10261 cases of ALS by mining the administrative databases.  Fine.  If that's around 85 percent of ALS cases in the United States, that means that they missed (10261/.85)*.15 or 1810 cases.  There were a theoretical 1810 people living with ALS who were not in the administrative databases.

And how many of that theoretical 1810 self-enrolled at the web portal?  A remarkable 1926.  Yes, every last one of the 15 percent and then some took the time to self-enroll at the web portal.  Self-enrollments far exceeded what we expected in this "15 percent" group.

To add to the amazingness, only 17 percent of the people who were in the group that was in the administrative databases self-enrolled at the portal.  What a difference.

Something is really odd here.

Question(s) 2 --

The expectation based on small pilot studies that the Registry data mining algorithm could identify 85 percent of US ALS cases from files was clear to all of us who worked hard to get the Registry approved and well-funded.

Question 1 now has me wondering if that expectation was simply wrong.

From the recent Registry report --

The algorithm was developed initially during the pilot
projects and categorized persons as either “definite ALS,”
“possible ALS,” or “not ALS,” with a sensitivity of 87% and specificity of 85%

Now I'm wondering if the premise should have been that if a case was identified in the passive data mining that  87 percent of the time it was really ALS (and that if somebody looked like a non-ALS person in the administrative data that 85 percent of the time that person did not have ALS) ?

To add to my head-scratching, if they found MND codes (rather than the more specific ALS codes) in the administrative files, that could well account for around 15 percent of the mined records being a MND other than ALS.

Does the 15 percent refer to something strictly within those in the administrative files (and not the Americans who aren't in the files)?

Was the idea that the Registry could identify 85 percent of ALS cases in the US by passive data mining wrong?

Do we really know anything about how many people with ALS are expected to be outside of these administrative databases?

It seems like the answer to Question(s) 2 will tell me if Question 1 is a silly question asked by an advocate who didn't read the fine print closely enough.

Friday, July 25, 2014

We Need To Talk!

Yesterday we got the long-awaited first data from the CDC ATDSR ALS Registry project.

Now we need to talk.  We need to talk a lot.

This is not a time for those with the bully pulpit on ALS to be shy.  This is not a time to distract us with other projects that the Registry's budget can swoop in and do.  The Registry was always about the incidence and prevalence of ALS in the United States.  We need to speak to the numbers now.

ALSA and MDA and Communicatehealth Inc. have all received hundreds of thousands of dollars for education and outreach and communication related to the Registry project.  This is a good time for them to step up and educate and reach and communicate.

Here's the report.  It's a good starting point for a much-needed national conversation on ALS.

There's a lot to talk about.

Prevalence -- A One-Legged Data Stool

We learned how many living people with ALS that the Registry could find in that 13-month period.  It said nothing of how many people were actually newly diagnosed with ALS in that period.  It said nothing of how many people died from ALS in that period.

There are three facts that together should make every American squirm --
1. You are quite likely to GET ALS. (incidence)
2. You are quite unlikely to be ALIVE with ALS. (prevalence)
3. You are therefore quite likely to DIE from ALS. (mortality)

If they only look at the second fact, healthy Americans get quite a peachy, safe feeling.  It's the feeling that ABC News propagated last night.  Yes, "rare" makes healthy people feel safe from the threat.  Nothing could be farther from the truth.

High incidence and low prevalence are a highly toxic combination.

The Registry did not address incidence, and the report admits a huge blind spot in that regard.

Say What?

Organizations have played fast and loose with estimates for years -- a combination of a crutch and telling people what they want to hear.  We need to learn to speak to the correct numbers correctly.  ALSA and MDA have been insiders and paid contractors on this project.  They have had time to prepare for this day.  We need to rid the world of past inflated estimates of numbers of people ALIVE with ALS.  The "up to 30,000" myth needs to stop.  Once diagnosed, people die fast and there is nothing to slow ALS down.  As much as we like to think that they are living longer because of compassionate care, they don't live long.  As much as organizations like to tell us that they each support around 14,000 people with ALS in their clinic systems, that looks pretty impossible now.

Demographics -- Who Gets ALS or Who Makes It to the System?

This is a wonderful opportunity for us to step back and evaluate whether the demographic data cuts we saw in the report are really reflective of ALS or are reflective of those ethnic and socio-economic groups that make it to ALSA and MDA and Medicare and Medicaid and the VA with an ALS diagnosis.  That's an important conversation.  Do we lose people to ALS who don't look like Lou Gehrig and therefore die before they are ever diagnosed?

Don't Clam Up

After the unfortunate ABC News item last night, I was hoping that ATSDR or MDA or ALSA or someone with a bully ALS pulpit would have been on GMA this morning to talk about the numbers and the threat of a disease that you are likely to GET and unlikely to be ALIVE with.

It's also unfortunate that for the first time in years, ALSA issued a press release and did not post in on its facebook wall were it could be discussed openly.

We have spent over $40 million and have waited far too long to find just over 12,000 living people in the USA with ALS in a 13-month period.  That's worth a lot of conversation.  If you avoid that conversation, you'll miss the value in knowing the real threat of a high-incidence, low prevalence disease.

Tuesday, July 22, 2014

This Senator Is Different

Every year hundreds of people interested in advancing the fight against ALS call on U.S. legislators. Some officials are polite and others, not so much.  Most listen.  The message only seems to stick with those who have some past connection with ALS.  Some try to help.  Most file the ALS message in their minds along with dozens of other diseases that need more attention.

But there is one United States Senator who is different.

ALS caregiving changes a person.  "Caregiving" isn't just running errands or delivering an occasional casserole or visiting on a Sunday afternoon.  ALS caregiving involves an intense mix of physical and mental and emotional challenges.  You work a feeding tube and clean the suction machine and invent ways to communicate and deal with body functions and learn how to defy gravity and skip sleep and hold the hand of a dying loved one and watch that loved one slip away regardless of all you try to do.

And people who have been ALS caregivers have an unspoken bond.

There is one member of the Senate who shares that bond.  She is a one of our gang of ALS caregivers.

We are hardly in the league of special interest groups or fancy lobbying organizations.  We simply care and respect one another.  We don't have to talk much.  We know each other well regardless of how our lives differ in other ways. We have lived first-hand the ugly ALS menace.  We have a rage.

We are grateful that she shares the caregiver bond and that she is willing to address the nuts-and-bolts of making a difference for the next person.  Please take a look at this op-ed on the leadership of Senator Lisa Murkowski of Alaska --

This Senator is different.  Thank God.

Thursday, July 17, 2014

And There Are So Many Books That We Would Love To Finish

A few weeks ago there was a list published of the books we start to read and never finish.  Thanks to e-readers, we are caught in our tracks.

In 1996 on the day that Mom was diagnosed with ALS, I went home and started the research that every family member does.  I got out the old Merck Manual and the Dorland's and didn't like what I saw.  I used the then state-of-the-art dialup modem and got online at Prodigy and Compuserve and searched. Blogs weren't invented yet, but I found a couple of message boards that served a similar purpose.  There was a woman on one who gave a continuing stream of some very helpful nuts-and-bolts information about dealing with ALS.  I knew the outlook wasn't good and that we would need all the help we could get.  I read her posts in chronological order for nearly an hour. She had a gift for being helpful.  They suddenly, there was a post that she died, surrounded by family.  No!  Out of the blue the stark reality of ALS hit me.  Bright, helpful, productive people who are able to write one day are stopped in their tracks by a disease with no mercy.  I went back and reread looking for a better ending.  There was none.  Her story was ended long before its time by ALS.

Over the years blogs have arisen and it's not unusual for people with ALS to blog their journeys. Some are warm and hopeful, others are edgy and dark. Some are pleasant and cooperative, others are outraged and demanding change. Some are all about ALS, others speak little of the disease itself.  They all are important for understanding some courageous people who are facing an outrageous disease.

The big problem today is the same I experienced that night in 1996.  The blogs all end before the author is finished.  ALS stops it all.  Tomorrow the next person starts a new blog.

Fortunately the internet preserves some of these valuable writings long after the blogger has died.  The endings are often abrupt.  The work is not finished.  That's ALS.

There are many blogs from living people with ALS and you can find those with a Google search.

Here are some oldies that are part of a valuable archive of how much really hasn't changed in the world of ALS.  They will give you painful insight into the people and the disease, and we should all look for the ideas that will finally fix the fight against ALS. Some started before the writers even heard of ALS. All were ended by the writers' deaths.  You'll get the best understanding if you read them from oldest to newest entries.  I've tried to point you to the older posts.

Wednesday, July 16, 2014

People With ALS And Their Caregivers Need A Place At THE Table, Not Just A Table

It's too easy to break out another table and say that people with ALS have a place at the table.  A separate table is not THE table.

People with ALS and their caregivers need to be at the same tables with researchers and clinicians.  They need to talk directly.  

I was fortunate to go to a college where faculty and administrators sat with students in the same cafeteria at the same tables.  That was a good thing for all.  A few years later I worked at a large corporation where the man whose name was on the company skipped the management dining room and joined employees at the tables in the regular cafeteria.  That was a good thing for all.

ALS meetings, be they scientific or policy or clinical, need to include people with ALS and their caregivers.  And "include" doesn't mean a separate session or a separate table.  When meetings exclude the very people with the disease, it speaks volumes about one big blind spot in the fight against ALS.

We can and must do better.  People with ALS and their caregivers must be welcome at every ALS meeting, both in person and virtually.  They must be at the table, both in person and virtually,  whenever direction is given to the fight.  They are the ultimate experts regardless of what scientific and organizational egos think. 

Saturday, July 12, 2014

Some Words Blind Us

The fight against ALS has never been a well-oiled machine.  Everyone knows that it would be more effective if people worked together better.

We love the word "partnership."  That gives us visions of people and organizations finally working together.

The word "collaboration" makes us so happy.  At last people are sharing to help everyone advance the science.

Then we find out, often much later or by accident,  that a "partnership" came with a big money contract.  And next we discover that people in a "collaboration" were paid for the arrangement.

Those magic words that give us visions of people doing the right things for the right reasons have blinded us to pertinent business arrangements.

There's nothing wrong with a mutually beneficial business deal, but people need to know who is being paid by whom to do what in the fight against ALS.  We donors and advocates hear the magic words and assume things that may well not be true.  Scientists and not-for-profit employees participate in meetings where there is not a clear picture of who is being paid by whom to be there and to "partner" and to "collaborate."

Obligations for transparency go far beyond annual reports and tax forms.  People need to understand who is being paid by whom to do what.  Every time that the word "partnership" is used in an arrangement where there is an exchange of money, we have a right to know that.  Every time a "collaboration" is a business arrangement, we need to know that.  Whenever people are paid to participate in a meeting, that should be on the table.  It's material information to understand an individual's or an organization's perspective.

We need to cut the blindfolds on "partnerships" and "collaborations."

Friday, July 4, 2014

What Does Every American Need To Learn Today?

Yesterday @alsadvocacy tweeted a question --

"What's the most important fact that every Jane or Joe Doe needs to know about ALS?"

That's the kind question that public relations and communications firms ask before they design campaigns.

Almost immediately @MrChuckMorris, a gentleman with ALS, tweeted back --

"Incurable, terminal ALS could affect them, or someone they love, any day."

That's it.  Nail hit on the head.  Clarity. No expensive p.r. firm needed.

During a few days of marvelous celebration of the 75th anniversary of Lou Gehrig's historic, beautiful farewell, we've learned much about Lou Gehrig -- the man, the athlete, the celebrity, the American.

As baseball fans leave ballparks this week after all of the celebrations and festivities, will they have any idea that incurable, terminal ALS could affect them, or someone they love, any day?

As Americans hear the messages of hope and celebration will they grasp that incurable, terminal ALS could affect them, or someone they love, any day?

As we are told to contribute to all of the promising work and research that done by ALS organizations, will Jane and Joe make the leap that ALS is still terminal and incurable and likely to strike after all these 75 years?

There are some history lessons that America is missing today.  Lou Gehrig died less than two years after that speech.  Hundreds of thousands of people have been killed by it since.  They still die today.  The prognosis is the same that Lou and Eleanor Gehrig faced in 1939.  That's outrageous. It's a difficult disease. There is no known cause. There is no effective treatment.

Incurable, terminal ALS could affect YOU, or someone you love, any day.

Tuesday, July 1, 2014

We Delivered, On Time, Every Time

Yes, we American taxpayers deliver the funds for the CDC to do its work.  We deliver every April 15.  Every year.  On time.

Yes, we ALS advocates delivered the requests for the CDC to produce an ALS Registry.  We've delivered over $40 million dollars.  Every year we answer the ALSA call.  On time.

As recently as May we were promised at the ALSA Advocacy Conference (yes, we showed up on time and went to Capitol Hill to request more funds on time) that the first data sets from the Registry would finally be published in MMWR in June.  We assumed that was June, 2014.

Every week in June, we looked at to find the data they promised.  Nothing.

We paid $40 million.  They didn't deliver anything... even an excuse.

Wednesday, June 25, 2014

Dear New York Yankees, Major League Baseball, AT&T, The ALS Association,

I beg you, please stop!

On July 4, 1939, the image of a courageous, eloquent man bidding us farewell was frozen in time.

Because of the sensibilities of the era and a respect for celebrity privacy that we don't have today, we saw little of Lou Gehrig after that.  It's hard to visualize his out-of-control, downhilll journey to his death less than two years later.

Any baseball fan knows that he took himself out of the game.  We know that something was happening to his remarkable athletic ability.  We can stretch our imaginations to see him eventually not being able to use those powerful arms and legs, but unless you've seen ALS in person, you may not realize that it's more than arm and leg strength that are stolen by ALS.

Neck weakness is one of the most insidious parts of ALS.  It's hard to understand until you've seen it and tried to deal with it.  Your head is heavy.  If its center of gravity strays from directly over your shoulders, without neck strength, you are toast.  Your head flops painfully.  You can't pick it back up.  You can't see.  You can't breathe.

That handsome Yankee who still looked fit and strong in 1939 faced a difficult ALS path that hundreds of thousands have taken since.  It includes that evil neck weakness that few realize.

This is why the Lou Gehrig bobblehead promotions are offensive and disrespectful.

This is why we need to make the 75th anniversary of Lou Gehrig's farewell a moment when we all respect and honor a great Yankee and American, and at the same time respect all of those who have dealt with the disease that bears his name.

I cringe to think that 18,000 baseball fans could listen to Lou Gehrig's farewell while holding little statues with his head wobbling.  If that happens, we will have lost our way.

I beg of some of the most trusted brands in the world.  You know how to lead.  You know that doing the right thing is important.  Please do the right thing.

Thank you.


Saturday, June 21, 2014

What We Can Learn From A Tale of Two Airport Shuttles

A few years ago I had a business trip to a major American city that has an airport far from downtown.  The meeting I was attending suggested a specific airport shuttle service to downtown hotels.  I followed the suggestion and to this day have bad memories of that shuttle ride.  We had long waits, an inexplicable change to a different van before we even left the airport, and uncommunicative driver who seemed to be driving in some odd directions.  Each passenger was glad to exit an unpleasant and long shuttle experience.

Last week I returned to the same city.  A few things were different this time around.

I was able to do my homework.  I knew to check online reviews of shuttle services.  Sure, price is important, but so are my time and safety.  I made a more informed decision than before.

I booked the trip online and that started a reassuring information connection with the shuttle company.

The minute I turned on my phone as my flight taxied to the gate, I had a message giving me directions to baggage claim and the shuttle.  After I picked up my bag, I received another message with a van number and an estimated number of minutes until I would be picked up.  Everything seemed to move faster since I knew what was going on.  And I didn't feel forgotten or misplaced.  I boarded my van, it made a couple more pick-ups at the airport, and we were on our way.

I looked at my phone's map app and saw a 28-minute eta to the downtown area.  Good.  And I could
see that the driver was taking the freeway route.  About halfway through the trip, there was a large traffic backup ahead.  The driver bailed onto an open exit.  Ut, oh.  Here we go.  My map was able to show me where we were going, I learned a few things about local geography and landmarks, and we eventually met back up with the main route.  And as passengers were dropped off downtown, I could see the locations of the hotels and was not puzzled over the driver's sense of direction.

There was such a difference in shuttle experiences, but why?  The trips themselves were not all that different, and there were snags in each.  The difference was information.  I felt that I made a more informed decision, and using the shuttle's technology and my own tools, I knew what was going on every step of the way.

When will we equip people with ALS with the information and the tools that they need to make informed clinical trial decisions and to know what is going on every step of the way?

Friday, June 13, 2014

No, Everything Is Not Fine

The concerns about the Lou Gehrig bobbleheads have been around for years.  ALS neck weakness is one of the most difficult parts of this stinking disease.

In this, a special year to remember Lou and to educate people about ALS, it's time to address the problem seriously.

A few weeks ago on the ALS forum at, a woman with ALS posted her comments about a fundraising message she had just received promoting a Lou Gehrig bobblehead as a premium.  She was shocked.  Her final words in the posting were, "Does it come with a neck brace?"  More patients spoke up. No response.

Then on the ALS Association of Greater New York facebook wall where the fundraising promotion was posted, a number of people spoke up, too.  No response.

Then on Jonathan Eig's facebook wall and twitter feed, he asked a thoughtful question about these bobbleheads being offensive.  Good discourse.  Not 100 percent agreement, but the overwhelming sentiment among those who have dealt with ALS is that these are offensive.

And during this whole time this blog site has had an interesting array of daily traffic watching the postings regarding the bobbleheads.  Isn't anyone going to take some constructive action?

Everything is not fine.  Those who who hold the responsibility to stop the promotion of these little statues need to step up, but first they need to take off the sound-deadening earmuffs and listen.

Please bury the bobbleheads.  You know they are simply wrong.  You know that this is an opportunity to educate people about ALS and about what happens to people like Lou Gehrig after they say their courageous farewells.

I don't know what Eleanor Gehrig would say, but I had a loved one who struggled terribly with ALS neck weakness.  Imagine your heavy head resting down on your chest and your neck being completely powerless.  That's ALS.  That's why these trinkets are wrong.  Then again, perhaps I do have an idea of what Eleanor Gehrig would say.

No, everything is not fine right now. Thanks for listening.

Thursday, June 12, 2014

What We Have Here Is A Failure To Communicate

The April 1 CMS-Medicare rules for Speech Generating Devices are simply in a time warp relative to today's technologies and the needs of people with ALS.

Now we understand that on September 1 there will be another giant step back to the 1970s --

Effective September 1, 2014, if a person's SGD has the potential for any function other than speaking (for example, email), then Medicare will not pay for the person's ability to speak through an SGD.

The people who write these rules must save them on computers with floppies that really flop while they listen to John Denver's Greatest Hits on their 8-track players.

People live with ALS in the real world of 2014 are unable to communicate without the enabling (and relatively inexpensive) technology of 2014.  To deny them that is wrong.

Maybe we should have an "rap with your kids about technology" day for the rulemakers at CMS.  Way cool.

Tuesday, June 3, 2014

Please Bury The Bobbleheads

Please stop all the promotions now.

Please educate.

Please explain why a Lou Gehrig bobblehead is an offensive and disrespectful trinket.

Please (literally) bury the inventory.

Enough is enough.

Saturday, May 31, 2014

"ALS Awareness Month" in the US Is Officially Declared A Failure

We now have the most objectionable fundraising premium in the history of fundraising.

We understand that the people who run a factory in China that makes little dolls whose heads wobble when you tweak them have probably never seen the neck weakness associated with ALS.

We realize that baseball team gift shop managers who like to sell souvenirs have never had to shop for a neck brace that a person with ALS needs to prevent a heavy head from flopping painfully.

And we know that collectors on eBay have no idea of practical difficulties (from breathing to vision) of neck muscles that simply can't support a head.

These people don't know what happens to people like Lou Gehrig after they say their farewells and slip privately into a difficult journey with ALS.

But people who accept paychecks from an organization whose mission begins with "Leading..." and contains the word "compassionate" should be able to grasp that a little statue of a man with ALS whose head wobbles uncontrollably is simply offensive and disrespectful.

And they should educate.

We need an advocacy organization that stands up for the respect and civil rights of people with ALS.

It's not that hard.  You don't mess with PALS or disrespect them.  Period.

Thursday, May 29, 2014

More Qs For The Right-To-Try Discussions

The op-ed in USAToday leads us to believe that Neuralstem is full-speed-ahead with a Colorado Right-To-Try stem-cell program for people with ALS.

The discussion is making us all think.

There are some thoughts and questions that I would like to add to the discourse --

  • Is anyone willing to commit to conscientious and open data collection for any people with ALS who enter a Neuralstem RTT program?  Colorado law does not have any data requirements that I am aware of.  We need to raise that bar voluntarily lest these programs be no better than the stem cell programs in Mexico and China that thrive on anecdotes rather than trustworthy data on all patients.
  • It would be very enlightening for us to see a specific head-on comparison of what Neuralstem and PALS would have to do in an FDA Compassionate-Use program versus a Colorado Right-to-Try program.  Also interesting is the question of whether any people with ALS had applied for Neuralstem compassionate use prior to this Right-to-Try push.
  • The USAToday op-ed again cites the truly miraculous outcome of Ted Harada who participated in the Neuralstem clinical trial.  We know that ALS is a spectrum of diseases.  Is anyone willing and able to provide a profile of Ted's ALS, e.g. known genes, onset details, pre-trial trajectory, etc.?  I would think that people with ALS that most closely resembles Ted's ALS would have the best roll of the dice in a Neuralstem Right-To-Try program. 
Thanks to all who are helping the conversation continue. I hope that it helps us figure out ways to move ahead more quickly and still advance the science for all with ALS.