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, 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.

Tuesday, May 27, 2014

Is "Right To Try" Right Right Now?

This is an extremely important discussion that affects everyone with ALS.

There are some new state laws intended to give people with untreatable, terminal conditions with a right to try experimental treatments.  The "right to try" name is awesome.  The concept makes sense to people who have a disease worse than any side-effect of any experiment.  The intentions are wonderful.

But I keep remembering my grandmother's words that the road to hell is paved with good intentions.  And if we provide experimental treatments to people with ALS, can we also advance science from their experiences?

I hope that the discussion will continue and grow in depth.  Already we have seen polarizing arguments based on emotion and lung power rather than on thoughtful consideration.

We clearly have a status quo that does not work for people with ALS.  We have failed.  We need to do better.

Here is the text of the legislation that recently passed in Arizona --

It's short and sweet.

“Right to Try” is a great name, but the devil is in the details.  Some of the details concern me a lot are --

  • What constitutes an “investigational drug?”
  • People with ALS are very vulnerable to hucksters, and unfortunately, some of those hucksters are even physicians.  Would this legislation permit anyone with a physician’s license (even a plastic surgeon or dermatologist) to make RTT therapy recommendations for a terminal patient with a complex neurological condition like ALS?
  • Do I understand correctly that the Colorado legislation allows the drug developer to charge patients for the investigational product? How do we prevent a system that excludes dying people who happen to be of limited means.  Will it be “Right to Try” or “Right to Buy?”
  • Attracting people to clinical trials is constant challenge in the fight against ALS.  How in the world would you ever get clinical trial volunteers (who have to agree to the coin-flip of a placebo)  if they had slam-dunk access to the real drug in a Right-to-Try program?
  • There are Expanded Access Programs that provide investigational drugs to people with ALS, and data are not tracked or reported.  We lose valuable clues both to ALS and to these experimental treatments when programs permit data to be lost.  My impression is that there is no requirement or incentive for drug developers to retain or report data on RTT participants.  If that is correct, it seems to me that we lose yet another opportunity to advance the science.  Patients not only have a right to try, they also have a right to have their data used constructively to help the next person.

Let the conversations continue. Please!  And let's come up with something that has both short-term and long-term benefits.

Friday, May 23, 2014

Time To Put Away The Theatrical Fog Machine

Yesterday an alert advocate for people with ALS sent me yet another one of those things that we stumble upon online.

May 7 was the day when we sat in a large meeting room in the JW Marriott for ALSA's advocacy briefing before hundreds of us were dispatched to Capitol Hill to ask for funds for the CDC's ALS Registry.

On that very day the CDC committed almost a million more dollars to the coffers of ALSA and the MDA.  When I saw the screen shot, I could not believe the date.

Was that not worth mentioning to us?  Was that not pertinent?  Why was that not included in the explanation of the project funding and status?  Did they forget about it?  The timing is interesting.

Instead of transparency on this project it seems like we get a continuous stream from a theatrical fog machine.  We were sitting there on that very day, for Pete's sake.  Those are big contracts.  That's a lot of revenue for those charities.  Not a peep.  

Time to stop the fog and speak to advocates and taxpayers clearly.  Transparency is a serious obligation.  Fog is a bad sign.

Thursday, May 22, 2014

Which Is It -- A Clueless Politician Or An Insensitive Clod?

From the Wall Street Journal Washington Wire blog --

So a politician thought it would be fun to fulfill some kind of baseball fantasy using the words of a great man dying from a disease that cut him off in his prime and for which there was no treatment.  Mr. Wolf obviously missed the beauty of the speech and the courage it embodied.

We see organizations celebrating the 75th anniversary of Lou Gehrig's farewell this year.  We also need to educate America that the outlook for people with ALS today is the same that Lou Gehrig faced in 1939.  ALS was and still is an outrageous, cruel disease.

If we ever heard the words, "How dare you?" from my mother, it was over a very serious breach of good behavior and judgement.  Mr. Wolf, how dare you?

Tuesday, May 13, 2014

Thanks For Asking

Last week I spent two days wearing Regina Holliday's Walking Gallery Jacket #300 in our nation's capital.  I walked on busy sidewalks, spent a half day at a conference of ALS advocates inside an upscale hotel, and called on legislators for a day on Capitol Hill with a curious painting covering the back of my yellow wool jacket.

The people who went out of their way to ask me about the jacket were interesting -- the staff at my less upscale hotel, many strangers on the Metro, people waiting to cross streets, security guards at a House office building, workers at the snack bar in the basement of a Senate office building, a lady who saw me in line at Starbuck's, several legislators who really wanted to learn about the backstory, and on and on.

Perhaps the people who didn't ask reflect something important about the fight against ALS.

The Walking Gallery has an everyman quality.  It draws interest from real people relating to other people.

How do we move clinicians and other healthcare professionals and scientists and organizational executives to have that same personal curiosity that everyman displayed last week?

The Walking Gallery makes some powerful statements in many ways.

Thursday, May 8, 2014

Today There Is An Action Item for You and for You, Too

Your words and thoughts matter.  They are needed before Sunday.  Please act.  Yes, you.

We saw Medicare rules go into effect on April 1 regarding Speech Generating Devices (SGDs) that just have stupid written all over them.  They do not make sense in the 21st Century.

We have already written about how a well-intentioned move of the devices to rental status has all kinds of unintended consequences for people with ALS.  As our grandmothers taught us, the road to Hell is paved with good intentions.

You perhaps think my use of the word "stupid" is a little much?  I think not.

  • The rental status means than if a person with ALS is transferred to a hospital or hospice, that rental device will not go with the person.  We are moving people into situations where communication is more important than ever, and we take away their right to communicate.  That'll keep them quiet.

  • Devices are locked from internet access.  The bureaucrats say that's an old rule that they should have been enforcing all along.  Rental just makes enforcement all the easier.  Peachy.  Let's unhook PALS from the best communication network known to mankind.  That'll keep those patients quiet.

  • But wait, there's more!  We hear that on September 1, a government bean-counter somewhere will celebrate.  There are indications that devices that include word processing, email, or remote control will not be covered at all.  That will really keep those patients quiet.

  • There have been recent denials of eye-gaze devices.  Evidently some Medicare reviewers say that this is not needed and people use them to play games and surf the internet. Is your blood boiling yet?
Please consider a quick action item if you have ALS, know someone with ALS, or simply care about government getting in the way of common sense.

Please write a quick message telling the importance of Speech Generating Devices to those with ALS. Your letters will be delivered to the Medicare powers that be on Monday by executives from Tobii, the company that has developed much of this enabling technology. The following is from Tobii:

 Here are the kinds of things you should write about. Remember this is Medicare. Use terms like "medically necessary."

How has a communication device changed your life?
What impact has eye tracking had?  How would you function without eye-tracking?
Has eye tracking been instrumental to have them work longer? Stop abuse? Communicate an emergency? Able to communicate with their children? (any other examples?)
What should Capitol Hill be aware of if patients going forward will be denied eye gaze across the board ? What if all claims would have to be appealed on all eye tracking? (CMS appeals right now at three year wait, yes that is not a typo)
How would losing the ability to use skype , email, text, phone , teleconference with their doctors, and internet impact you?
Have you been denied access to a communication device with eye tracking what has been the trail or timeline for process and if possible how have your symptoms have changed.

Please sign your name, town and state and if willing, connection to pALS, age and disability. 

These are just quick thoughts/ideas. You need to think like the government. Why should they care? We need the patients & families to remind them who they were before the disease, who they have been able to be after and why communication is so so critical. 
We are asking for the letters by Sunday and from people throughout the country. They will be hand delivered to CMS. Please email your letters to:

Please submit something.  It doesn't have to be elegant.  Direct is always better. Please act.  It's up to you and you and you and me.  Let's send them to Washington with a flood of messages, many of which will not be possible to write after September 1.

Thanks, all.  Please help spread the word.  Friends and their friends can speak to how important it is to be able to interact with those with ALS.  Any American taxpayer should speak up. Yes, you.

Wednesday, May 7, 2014

Today Is Brought To You By The Letter "Q"

Click here for the ALSA legislative priorities.

We hope that the following questions will be addressed before we are asked to advocate for more funds on Thursday:

·         How have the Registry funds been spent in the past few years and what are the plans for the $10 million being requested?  Approximately how much is attributable to the cost of data files, basic data file mining, website, risk-factor surveys, patient support, analysis and reporting, travel and meetings, clinical trial matching, promotional materials, special studies, local studies, biorepository feasibility, etc. ?  

 ·         Who have been the government contractors on the project and what are (or were) their roles and deliverables?

 ·         Has anyone re-measured the Rob Tison metrics?  If so, what were the results?

 ·         Do you know what percent of the identified cases of ALS were found in data files only, in both data files and self-enrollment, and in self-enrollment only?

 ·         Do you know how many newly diagnosed cases of ALS enter the ALSA clinic system annually?  Do you know how many of those have self-enrolled in the Registry?

 ·         How many clinical trial enrollees has the Registry notification produced?

 ·         How are the participants for the Registry annual meeting, for the DOD ALSRP Integration Panel, and for the DOD ALSRP Peer Review group chosen?

Today is brought to you by the letter "Q."

We hope that tomorrow will be presented by the letter "A."

Tuesday, May 6, 2014

It's Time For Some Three-Point Shots

For over a year I've been scratching my head about the MODDERN Cures Act.

Recently an outstanding advocate/tweeter/patient/person  put me in touch with the folks at the Campaign for Modern Medicines who have gone above and beyond the call of duty to help me understand MODDERN Cures and exactly what's in it for people with ALS.  I am grateful.

As we perceived years ago, the dormant therapy features of MODDERN Cures are the most interesting for the fight against ALS.

Here is a blog from the Campaign for Modern Medicines that answers most our questions --

And here is a recap of some salient points for those interested in the fight against ALS --

Are these "dormant therapies" only things that never made it to market for another indication?
That is correct.  Think about things that didn't get through FDA approval for their original intention.  Those things that are sitting on shelves in labs somewhere could have their patent "clocks" reset so that there would be incentives for drug developers to try them on ALS.  MODDERN Cures would not apply to a lithium or a ceftriaxone, but it would apply to something that never made it through FDA approval before. 

How does it work when something is already off patent?  

There are two things that are really important in this case -- patent protection and data protection. 
The MODDERN Cures Act aligns patent protection and data protection at the date of FDA approval. 
Even if there is no patent protection left, the drug receives data protection.  A generic or biosimilar manufacturer cannot generally receive FDA approval to launch a competing product during that protection period.  If a competing manufacturer started clinical development of an off-patent molecule before the first manufacturer received FDA approval, then competing products could also be approved.

Can anybody pick up a potential drug and run with it, or does it have to be the company that owned the rights when it was on patent?

If it still has any patent life, an interested scientist would have to work with the patent holder on a license agreement; otherwise, scientists, please go for it!

OK, what happens when something has just a little patent time left?

The MODDERN Cures Act allows the patent clock to be reset during the development process and run from the date of FDA approval. This is a big incentive.

We know that it's going to take a lot of shots on goal to finally find some therapies for ALS.

We need for drug developers to make smart (and long) shots.  If something is sitting on a shelf with little or no patent protection, we owe it to people with ALS to make it viable for drug developers to pick up one of those oldies and shoot.

Many thanks to the folks at for their time and help.  If I got anything wrong, I'm all ears.  

Finally I think we have some clarity on what's in it for people with ALS.

Monday, May 5, 2014


That's the best title I can come up with for a post I never expected to have to write.

Last year some of us stumbled across some large no-bid government contracts given to ALSA and then to MDA from the CDC ATSDR ALS Registry funding.  The MDA's contract appeared in September and the amount, $2,695,642, floored some of us.

On October 18 I emailed a question directly to the head of the project at ATSDR who is a frequent speaker to groups on the Registry project.

Dr. Horton, Are you able to tell me what MDA is doing for this sum, please?  
I have a bad case of sticker shock.  
Thanks very much for any insights.

On October 21, I received a reply from Abby Martin of the CDC.
Neither Mr. Horton or I have the authority to respond to requests for public information not in the public domain.  (This includes any request for information via social media.)  All such requests should be directed to the CDC/ATSDR Freedom of Information Officer for a response 
Okeydokey.  I learned how to submit a Freedom of Information Act request.  The following two requests were submitted and accepted on October 22.

Request 1 I would like to know what the work product, deliverables, and performance standards are for the work in the MDA contract for “ALS Outreach and Education Activities to Support the National ALS Registry.”

Contract Award Number:
Contract Award Dollar Amount:

Request 2 I would like to know what the work product, deliverables, and performance standards are for the work in the ALS Association contract for “Education and Outreach for the National ALS Registry.”

Dollars Obligated
Current Contract Value
Identifying Agency ID
Procurement Instrument Number
Modification Number
Transaction Number
Fiscal Year
IDV Agency ID
IDV Procurement Instrument ID
IDV Modification Number
Solicitation ID

There is a nice online status tool that they provide to track the status of your request.  The status of both requests went from and initial  "Pending Program Search" to "Pending Final Review" within a few weeks.  Great!  I honestly expected to have answers by the end of the year.  Silly me.

By January 23, I felt that surely something had gotten lost somewhere in the "Final Review."  I emailed the CDC FOIA officer who had helped me with the requests.

The online status for both of these has been “pending final review” for over two months. Is there a way to check to make sure that something didn’t get lost in the process? Thanks very much for your help.  
I immediately got a reply that floored me again.

This is in response to the email below concerning your Centers for Disease Control and Prevention and Agency for Toxic Substances and Disease Registry (CDC/ATSDR) Freedom of Information Act (FOIA) requests #14-00039 and 14-00043.
 As we advised in our acknowledgement letters dated October 22, 2013, CDC processes all FOIA requests on a first-in, first-out basis, which is the court-approved method for agencies operating under a backlog. Program staff have completed their search for the records you requested, and your case is currently in this office awaiting final review. A thorough document review will be conducted by the CDC FOIA staff, and all releasable records will be provided. Processing time is contingent upon the number of requests ahead of yours and their complexity and volume. Therefore, we are unable to give you an exact timeframe for completion of your request but estimate that it may take up to 36 months for your request to rise to the top of the complex queue based on current workloads. Please be assured, however, that a response will be sent to you as quickly as possible.
 You may continue to check on the status of your case by going to our FOIA webpage at and entering your request number. The fiscal year is the first two numbers and the request ID is the second set of numbers.

Hmmmmm.  Why is this so hard?  Thirty-six months?  Does someone not want to talk about this?

My response, still on January 23, was --
I appreciate the insights.With matters ALS, the clock ticks quickly.  One of the advocates who was interested in the information has died already.I’ll keep watching the status and hope that it finds the top soon.Thanks.  Have a good afternoon.

The status is still "Pending Final Review."  That must be some fancy review.

This started as a very simple question.  The answer should be pretty simple.  It just gets curiouser and curiouser when people make everything difficult.  This is information that should be very transparent, especially since the contracts required no bidding and since we advocates go to our legislators to ask for the project's funding every year.

It should be simple, but it's not.  Such is the way with matters ALS.


Friday, May 2, 2014

Help! Someone, Please Help!

Eight years of advocating for a national ALS Registry.

I felt and still feel strongly that we need a census of cases of ALS.

$40 million taxpayer dollars spent.

This is the spring when we finally expect to see some numbers.

And so far we see a flock of poster sessions from this week's AAN meeting.  Here are a couple.

I feel like I've been spun around a few times and the blindfold has been partially removed.  Now I certainly need some help with some questions.  My comments and questions are in hot pink below.  These are things that we need to understand.  I think we've earned that right.

Using the National Amyotrophic Lateral Sclerosis Registry as a Subject Recruitment Tool for Research (P5.077)

  1. Paul Mehta1,2
  1. Neurologyvol. 82 no. 10 Supplement P5.077
Objective: To describe how the National ALS Registry links persons with ALS (PALS) to scientists who are conducting ALS research.Background: Subject recruitment for research is critical for understanding inherently fatal diseases like ALS, however, linking patients with researchers is not so straight-forward. Researchers often face challenges in timely recruitment, obtaining sufficient sample sizes, and/or in enlisting patients that meet certain eligibility requirements. Similarly, patients can face difficulties in finding specific research studies for which they are eligible.Design/Methods: The federal Agency for Toxic Substances and Disease Registry’s (ATSDR) National ALSRegistry now links Registry-enrolled PALS with external scientists who are conducting ALS research. TheRegistry’s new online Research Notification Mechanism allows ALS researchers to upload an application that briefly describes their research study and objectives, while PALS can elect to be notified about these opportunities.If the application is approved, ATSDR then queries the Registry for PALS meeting the study’s specific eligibility criteria (e.g., age, sex), Does the registry have onset date? and then distributes the researcher’s study material and contact information to PALS via email. PALS have to directly contact the researcher to take part in any research.Results: Since the Research Notification Mechanism’s May 2012 deployment, approximately 96% of Registry enrollees have elected to be notified about ALSresearch opportunities. Additionally, ATSDR has approved a number Do we know what the number is? of institutions to use the Registry for research recruitment, sending out thousands Do we know how many thousands? of e-mail notifications to PALS Do we know how many PALS have been targeted? on behalf of the recruiting And do we know what percentage of the open recruiting clinical trial seats were represented? institutions. And do we know the most important metric of all, how many clinical trial seats have been filled because of this mechanism? Conclusions: The National ALS Registry’s Research Notification Mechanism is an effective ??? tool for linking PALS with ALS researchers who are conducting epidemiologic studies or clinical trials. The Mechanism benefits PALS by conveniently delivering timely and tailored research opportunities via email. It also benefits researchers by helping to speed-up the recruitment process, increasing the study sample size, and efficiently identifying PALS meeting specific eligibility requirements.  Do we know it actually sped or increased sample size in any studies?
Disclosure: Dr. Horton has nothing to disclose. Dr. Antao has nothing to disclose. Dr. Mehta has nothing to disclose.
Wednesday, April 30 2014, 3:00 pm-6:30 pm

Amyotrophic Lateral Sclerosis (ALS) Estimates from National Databases in the United States - 2001-2010 (P2.072)

  1. D. Kevin Horton1,2
  1. Neurologyvol. 82 no. 10 Supplement P2.072
Objective - To present preliminary ALS estimates from national databases in the United States.Background - The uncertainty about the incidence and prevalence of amyotrophic lateral sclerosis (ALS) in the United States, as well as lack of knowledge about the role of environmental exposures in the etiology of ALS, have created a need for data collection through a national ALS registry. In 2008, the Agency for Toxic Substances and Disease Registry (ATSDR) conducted four pilot projects to determine the feasibility of creating a national registry. It showed that approximately 80% of ALS patients can be found through national databases. The first approach utilizes existing national administrative databases to identify prevalent cases. The second approach uses a secure web portal to identify cases not included in the national databases. This latter approach allows patients to self-identify and enroll in the ALSregistry and take risk factor surveys. Design/MethodsWe sought to identify U.S. residents with ALS in Medicare, Medicaid, Veterans Health Administration (VHA), and Veterans Benefits Administration (VBA) databases for the years 2001-2010. Records were searched and identified in these databases for ALS and Motor Neuron Disease (MND) codes.Results - A total of 147,889 individuals, with any MND code Are those distinct individuals? So over a period of nine years, 147,889 distinct MND individuals were identified?, were identified across the four national databases. Medicare contributed the largest number of individuals. The total number of individuals identified having ALS was 36,547, which is 24.7% of the total number of individuals identified with any MND. I always thought that ALS was by far the largest subset of Motor Neuroon Diseases.  Am I wrong?  Do they really think that over 75% of the MND cases are not ALS... or did they just fail to identify a lot of MND cases as ALS?  Conclusions - This is the first effort to identify ALS cases for a national registry. The number of individuals identified via the national administrative databases is not a prevalence estimate Why not?; however, it does indicate that a large portion Based on what assumption? of ALS individuals can be identified for the National ALS Registry by using the national databases selected. How do you arrive at that conclusion?  It sure seems like a jump to me!  
Disclosure: Dr. Sanchez has nothing to disclose. Dr. Mehta has nothing to disclose. Dr. Kayehas received personal compensation for activities with McKing Consulting Corporation as an employee. Dr. Antao has nothing to disclose. Dr. Horton has nothing to disclose.
Tuesday, April 29 2014, 7:30 am-11:00 am