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.

Sunday, September 21, 2014

Was Basketball A Better Game Before The Shot Clock?

Were plays executed better?  Hardly.  Were more clever strategies employed?  No. Playcalling was quite the opposite.

Was football a better game before fans were allowed to see the play clock?  Not really.  Do play clocks spoil games?  No, in fact they have really added some ingenuity to modern football. Ask Peyton Manning.

Are golfers better when the PGA ignores speed-of-play rules?  Golfers never think they're playing slowly and they never seem have enough time to read a shot and rethink and rethink and change their minds and reread the shot.  Are slow players' shots better?  No.

Time taken for work and decisions has a bad habit of expanding to fit the time allowed, and the longer they take, the more "comfortable" the decisions become.

Sometimes an acknowledgement of extraordinary urgency can push someone to be creative and fresh and come up with decisions and execution that open new possibilities.

ALS is a disease of extraordinary urgency.  Time for a two-minute offense!

Friday, September 5, 2014

Easy Peasy Can Work

Yesterday I spent a day discussing patient engagement in clinical research with some impressive e-patients, healthcare professionals, and pharmaceutical company experts.  There is a critical area of mutual benefit, especially for patients with dire, untreatable conditions, that needs to be seized better.  It's a complicated problem.

People with ALS have a very short window of eligibility for clinical trials.  Trial sponsors have a problem akin to trying to catch fish in a very fast-moving river.  It's a constantly changing population and they're not in front of them for long.

After a good night's sleep, my mind is saying that maybe some of the solution isn't so complicated.

Here are two extremely easy ideas that could make a positive difference for both people with ALS and clinical trial sponsors --

  1. Start every ALS support group meeting with an announcement of all enrolling clinical trials within 500 miles.  Make it an institution.  That's the way every support meeting starts, everywhere.
  2. Put a 3-ring binder in every ALS clinic with a page for every enrolling clinical trial.  In addition to the old issues of People and Readers Digest, people with ALS and families can flip through clinical trials. PALS and CALS are actually at the perfect spot to talk to the doctors about anything interesting that they may see in that binder, even trials that are being conducted elsewhere.  It would be easy enough for a national organization to provide a fresh file to print and put into the binders weekly.

That's it -- two simple, easy ideas, and they don't take buckets of money.

Every day wasted filling a clinical trial is as important as a day of lab work or a day of data analysis or a day in the FDA approval process.  Every day is precious.

Thursday, September 4, 2014

This Is Not A Wish List

Yesterday a man with ALS asked some important questions.  We have so much to do.
"What topics are most urgent!? What needs to be discussed?"
Today's blog has my two-item response, but first, I had to find the right picture.  I didn't want a picture of a wish list.  These are not wispy dreams.  These are important. They can and must be delivered.  I rejected pictures of to-do lists. Those make us feel good when we cross off the trivial and recycle the important things.

This is a must-do list.

1.  The Medicare CMS handling of Speech Generating Devices is urgent and must be fixed immediately.   Current policies and execution hurt people with ALS and the American taxpayer.  We must fix this.  Now. It should never have been recycled on to-do lists for the last year.  It belongs at the top of a must-do list. Today.

2.  Now more than ever we need a good accounting of all ALS research grants made by our grantmaking organizations.  Nowhere is there a good, searchable list for donors (or for taxpayers or for scientists, for that matter) to see grant dates, amounts, subject matter, recipients, and outcomes.  We don't need cherry-picked project announcements and accounting by press release.  We need an orderly portfolio of the buckets of investments and outcomes. Yes, now.

That's it.

That's a two-item must-do list.

No wishing.  No putting things off.  No prioritizing until we get nothing done.

We must do these two things.  Now.

Wednesday, September 3, 2014

We Need To Press Medicare CMS Regarding Speech Generating Devices

Yesterday's article in the Washington Post and the related, delicately worded statement by CMS on their
policy on speech generating devices drew a lot of insightful comments online.

In addition, a relentless advocate for those with ALS who also knows a lot about these devices submitted a very informative letter to the Washington Post.

With her permission, I include it below.  I hope that the press will press for more information.  This is far too important to be glossed over with spin from a government agency.

Almost simultaneously with an op-ed piece by Steve Gleason on the importance of technology and communication for those diagnosed with ALS and other physical disabilities, the Health & Science section of the Washington Post, published a statement from Medicare regarding speech generating devices. 
As a volunteer advocate for people with ALS, I can tell you unequivocally that the statement they made is not true. 
I realize this is a bold statement to make, but since April 1, 2014, CMS has been denying as well as requiring manufacturers to remove all software and internet capabilities from the devices or they will not be covered for payment. 
One such distributor is Eyegaze, Llc. Feel free to contact them and inquire as to what they are now permitted to ship. 
I have been trying to help people with ALS obtain these communication devices for many years and the sudden change as of April, has truly affected the lives of people with ALS in a detrimental way. Many are losing their will to live as the speech generating device they now have is a very expensive alphabet box. No access to even save their sentences or manage their finances via internet.

I'd just like to note, there is no cost savings to Medicare by implementing these new rules. As a matter of fact, it is more costly due to the administrative costs they now must carry. The device costs exactly the same as it used to, except it simply has been stripped of its capabilities to add software or to access the internet.
Since 2001, SGD's have always been shipped "locked" from the internet and a user could call the manufacturer of their device and pay a small fee to have it unlocked. Usually about $50.
That capability has been removed with no explanation and causing months of letter writing campaigns and lobbying.
To read this statement from CMS is actually a slap in the face to all who have been suffering because of its implementation as well as all who have been trying to reverse the coverage rules. I urge you to dig a little but deeper and help make a change for those who are already locked inside their bodies. They shouldn't also be locked away from the world due to Medicare.

Tuesday, September 2, 2014

I Have Options. People With ALS Do Not.

It was pouring rain when I awoke this morning.

My early morning routine starts with a big pot of tea and my morning newspapers, read at the kitchen table as they were meant to be read.  It's a quiet time to read and learn and think that I've enjoyed since I was a child.  Real newspapers give me a good start to my day.

The papers had not been at the foot of the driveway for long when I went out to retrieve them in the dark this morning, and they were in a plastic bag.  When I picked the bag up, I knew today was not getting off to a great start.  The bag was heavy.  Two hours later the papers are still hanging on a clothes rack in front of a fan as I try to get them dry enough to read.  So much for my morning routine.

I am fortunate to have the digital option for my morning papers.  I have a good internet connection and can read the news that is drying in the laundry room.  If need be, I can pay to get behind the paywall for an interesting story, and I can even forward news to friends or write a blog post on something.  I have really good options.

Online I read a Washington Post story of Steve Gleason that was about much more than Steve Gleason.  It was about living a full and meaningful life with the assistance of technology, including the same internet technology and text editor that I use to write this blog.

Then I read a statement in the same newspaper from Medicare CMS about the devices that they are supplying to people with ALS.

A spokesman for the Centers for Medicare and Medicaid Services said last week that coverage rules relating to speech-generating devices are being reviewed but that no change or reinterpretation will reduce access for ALS patients who need them.
“We are committed to ensure that beneficiaries have access to needed technologies to improve their quality of life, including the coverage of speech-generating devices for individuals with a severe speech impairment,” the spokesman, Aaron Albright, said via e-mail. “Medicare has covered speech-generating devices since 2001, which greatly improve the day-to-day lives of many beneficiaries. This policy has not changed, nor have we proposed this coverage to change. With the rapidly evolving technology in this area, our Medicare payment contractors are currently undergoing a product review of available speech-generating devices to ensure that they comply with our coverage rules and the Medicare law. CMS extended this review until Dec. 1, 2014 to ensure that we [are] able to review and address issues that stakeholders have ... raised, including the ALS community’s desire for beneficiaries to have access to devices with expanded functionality.”

Say what?  Is this good news?  Nope.  Read again.  Read again.  Read again. Dad would say that this was written by a Philadelphia lawyer.  Read again.

CMS is supplying speech-generating devices.  They will generate speech.  They won't let you do anything else that requires "expanded functionality."  No internet.  No word processor.  No email.  And they'll make darned sure to uphold the rules so that speech-generating devices are as functional as the ones people got in 2001.

People with ALS don't have options like the rest of us.  They can't even turn the pages of a newspaper, and those who have received their Medicare-supplied devices lately can't even read the Washington Post online or see this blog.

The internet is their lifeline.  It's not "expanded functionality."

If there were ever an issue of respecting life in the United States, this is it.  We have vibrant, bright people who want to live their difficult lives to the fullest.  Boat-anchor speech-generating-devices that are "dumbed down" to prevent communication beyond speech in a room are simply morally wrong and a bad business decision on the part of CMS.  Why is the government purchasing technology with locks and blocks that is more expensive in the long run?

I'm grateful that I had the option this morning to jump online and read and give my two cents.  Doesn't everyone with ALS deserve as much?

Sunday, August 31, 2014

Do What's RIght - ALS Ice Bucket Challenge Edition

The thoughts below are from a response I made to a prominent ALS clinician who had given his opinion on how the ALS Ice Bucket Challenge windfall should be spent by the ALS Association. I couldn't have disagreed more with his priorities.  I share my thoughts here because I think that the ALS Association has both an obligation and an opportunity to do the right thing.  It's also the smart thing.


Donor Intent Cannot Be Ignored

Donor intent simply cannot be ignored.  Millions of dollars were donated
spontaneously after celebrities and corporate leaders and friends urged people to join with donations for ALS research or to find a cure, followed by a mention or flash on the screen to donate at  Jimmy Fallon's or GMA's cries were not to support a three-pronged organizational infrastructure. The call was to support ALS research.  Whether or not ALSA authorized the enthusiastic research pleas, the fact is that those millions flowed in from generous people who intended to fund ALS research.  That cannot be ignored.

Well over $60 million in ALSA national's donations were made before there was even an option on its donation form to clearly restrict donations to research.  Every organization loves unrestricted gifts, but to ignore the intentions of donors who thought they were giving to research is just wrong.

ALSA has since told people that they can contact them with details of their gifts and they will backtrack and restrict them.  That will eat those donations up in an administrative circus.  ALSA should just do the right thing, earmarking for research everything donated under the IBC that did not have an option to restrict.  

Will (Or Should) The Mississippi River Suddenly Flow North?

ALSA currently has a sustainable revenue model that flows from chapters to the national office.  Contrary to some people's perceptions, funds donated to ALSA chapters (including the substantial walk donations) are shared with the national office.  Chapters have to sustain themselves and most do a good job of that.  They supply a portion of their donations to the national office, some of which comes back to the local clinics in the form of grants.  Money does not rain down on chapters (and their clinics) from the national office.  And even if buckets of IBC (Ice Bucket Challenge) dollars would suddenly start pouring down from the national ALSA heavens onto chapter patient services, would it end up breaking the sustainable revenue engine they already have?  

Catsup Is Not A Vegetable

Some at ALSA have asserted that clinics are really research, so money to clinics is really research money.   Nice try, but until ALSA can deliver on the concept that every clinic patient is a research patient, clinics are patient care.  The spotty uptake on ALS Registry self-enrollment is testament to the fact that not all clinic patients are getting consistent messages of their important role in ALS research.  The fact that a lot of clinic patients are not aware of clinical trial opportunities makes it clear that clinics today are not intrinsic gears in an ALS research machine.  I truly wish that would change, but lack of funds isn't the root cause.

Don't Waste 93 Octane Fuel In A Car That Runs Well On 90

The ALSA public policy organization boasts of being a stellar success.  It attracts its own sponsors and donations directly, has revenues from its conference,  and in addition has a substantial revenue stream from being paid by the CDC for the ALS Registry project.  Even ALSA has not suggested that it is a prong in need of any help from the IBC windfall.  Can they do more regarding public policy?  Sure.  Is it important?  Absolutely.  Does it require IBC money?  No.

Research Isn't Like Mindlessly Buying Lottery Tickets

I understand that the odds of a research success that will work for people with ALS are long.  A high school math teacher (who made a very good living investing) once told his students only to gamble on things where you can increase your odds.  Walk away from lottery tickets.  Walk away from slot machines.  Study and make smart investments.  We know that ALS research is going to produce a lot more failures than successes, yet we can do things that increase the odds of success.  It's worth the investment if we make smart choices that give us the best odds possible, and then we can build on the information that failures provide to make the next bets even smarter.  If we don't invest more in ALS research, we are raising white flags in the war on ALS.  That's just wrong.  

A 21st Century version of the parable of the talents keeps running through my head with the reluctance to move forward now with even a small IBC injection of funds into ALS research.  We don't want ready-fire-aim, but right now it's feeling a lot more like ready-aim-aim-aim-aim with the IBC money. 

Neuroscience research is dreadfully expensive, but information is its sustainable feature.  If we gather and use the information well, even the failures will have been a good investment. 

Bringing Us Full Circle -- Donor Intent Aligns With A Good Change In The War Against ALS

If we beef up three prongs and do what we've always done, we'll just get more expensive versions of the past.  That's not good enough.  Let's keep doing what already works (and has already proven to be monetarily sustainable) and add some brains and resources to research efforts.  Donor intent matters, and in this case, it can indeed change the game with some new game plans and execution dedicated to smarter ALS research.

Thanks for listening.  If I'm wrong on anything, I'm all ears.

Wednesday, August 27, 2014

Do We Have An App For You!

It will send you a text message with the name of a person who just died from ALS.

You'll get a new message every four minutes.

That's every four minutes all day.  During breakfast. During conference calls.  Through lunch. During meetings.  Every four minutes your dinner will be interrupted with the news of another death.

Turn on your phone after a 3 hour flight, and you're looking at 45 names of people who just died.  You may have five more messages when you get out of the shower.  And 30 more people are gone in the time it takes you to mow the lawn.

ALS doesn't take weekends off.  During an hour at church you get another 15 new names.  And after a concert, there may be 50 more.

It won't stop while you sleep.  Every four minutes.  After a blissful night of sleep, you'll wake up to over a hundred new names of people who died while you snoozed.

Every four minutes.

And for those waiting until October for a meeting where you will work on a plan for the #ALSIceBucketChallenge windfall, you will have been informed of over 10,000 new deaths before you've invested a dime in new ALS research.

Sunday, August 24, 2014

Let's Have A National Science Lesson

The ALS Association recently announced that over the years they have provided some funding for 98 research projects that are still active.

They also have what must now be close to a million #ALSicebucketchallenge investors of all ages and stations in life.  Many are even students who were drawn in with buckets of ice and now know that ALS is an unsolved medical mystery -- and what student doesn't love an unsolved mystery?

How great would it be to turn this into a national online science lesson that would stimulate interest in ALS and help all of those young and old investors be excited about their new projects?

I'll bet you could hire some high-school science teachers to write summaries of the 98 projects in language that turns on those mental light bulbs that let us understand the basics of the science.  No doubt a social media location with information and conversation could be a lively place that ignites interest and insights into ALS science and scientists.

This could be a sustainable classroom for our nation on the science of ALS.

And there's no need to dip into the research windfall to start such a thing.  This could be a winning grant request. Time to strike while the ice-bucket-challenge iron is hot and STEM education is a national priority.

Saturday, August 23, 2014

Information -- It's What's Sustainable

Last night we heard an interesting interview by Judy Woodruff on the PBS Newshour about the #ALSIceBucketChallenge and the use of the windfall of donations --
"... whatever we do in research, care services, and even advocacy has to be sustainable, has to be sustainable."  Barbara Newhouse, President and CEO, ALS Association
Often not-for-profits think of sustainability as the fuel that feeds next year's revenue machine.

Medical research is expensive.  Medical research in neurosciences is mega-expensive.  There are many experiments that go plop.  We know that all too well from years of "promising" experiments and trials for ALS treatments that have proven to be duds.  ALS research is costly, risky business.

So how can you possibly look upon some big, immediate investments in this kind of research as being sustainable?  They could follow the trail of decades of failures.  They could be risky money pits, and not-for-profits often aren't good at risky anything.

It's all in the information that we retain and make accessible, both to scientists and to next year's donors.   It's going to take a lot of smart investments to beat the cunning ALS beast, and we know that many more battles will be lost before the war is won.  We need to document every investment and every outcome. We need to open up results.  That's how you sustain both the research environment and attract new, smart investments.  That's how you avoid reinventing wheels and duplicating efforts among organizations.

A simple start would be to have a transparent, organized, searchable, complete portfolio of research project investments and amounts and outcomes.  Today we get research grant announcements via press release and occasional reports of "promising" results via a few more press releases.  It's impossible to leverage the information when we just can't get to it.

We can build on what we learn from failures.  We must.  We can get better from the information that every research investment leaves behind.  If only we would treat the information as the precious sustainable resource that it is.

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."