ALS Advocacy

ALS Advocacy
Lou Gehrig's Disease - Motor Neuron Disease - Amyotrophic Lateral Sclerosis
Thought it had been cured by now? Still no known cause. Still no cure. Still quickly fatal. Still outrageous.

Thursday, October 23, 2014

Let's Talk About Ebola, Placebos, and ALS

Ebola is dreadful and quickly fatal.  The only "promising" treatments are experimental and yet unproven.

Sound familiar?

There have been a number of articles on the experimental drugs that have been provided for Ebola patients in hopes that something might help.  It bothers the human conscience to hand placebos to these dying people in urgent need of help.  It bothers the human conscience  a lot.

"Experts Question Ethics of Placebo Drug Trials in Case of Ebola"
http://www.reuters.com/article/2014/10/13/us-health-ebola-drugtrials-idUSKCN0I20W520141013 

"Use of Placebos in Ebola Drug Trials Unethical: Experts"
http://medicalxpress.com/news/2014-10-placebos-ebola-drug-trials-unethical.html 

"Issues Continue To Dog the Testing of Ebola Drugs and Vaccines"
http://news.sciencemag.org/health/2014/10/issues-continue-dog-testing-ebola-drugs-and-vaccines

Why aren't our human consciences bothered by the idea of handing a person with ALS, another  quickly fatal disease with no effective treatment, a placebo?

It's interesting.  What's the difference?  Why is a placebo so clearly an unacceptable tactic to Ebola clinicians and at the same time the "gold standard" of good research to ALS clinicians?

Let's talk.  Seriously, let's talk.  What's the diff?

Tuesday, October 14, 2014

You Can Get Better Information On Where To Park Your Car In Chicago Than Where To Find Good Care For ALS

This is an idea whose time is way overdue.  I write today because last week I heard a story that should be the last straw.  A man with ALS who must rely on a professional care facility somehow received a painful injury attributed to a simple piece of equipment.   That is terrible.   As someone trying to help him from afar said, "The bedsores should have been a clue."  And I wondered what will keep the next person with ALS from ending up in this same place.

It's 2014.

Who doesn't look up a hotel on TripAdvisor before making a reservation?

Who doesn't look up a restaurant on Yelp or UrbanSpoon to see what people think of the food and service?

Who doesn't read the reviews of items for sale on Amazon?

Who doesn't look for online ratings of roofers or painters or butchers or bakers or candlestick makers?

There are even online tools that help us find the safest and most economical parking lots when we drive to a strange city.

(And it's always helpful to see how restaurants and hotels and merchants address bad reviews.  Yes, they pay attention.)
Yet there are no good online tools for people dealing with ALS to help share experiences to rate a nursing home or equipment dealer or pharmacy or brace fitter or an online nutrition vendor or a VA clinic or even a clinical trial site.

Almost two decades ago we needed to find a new neck brace for Mom that would work with difficult ALS neck weakness.  I found that the ALS clinic staff had no ideas.  They referred  me to an occupational therapy department that was ALS-clueless.  I spent an entire day on the phone and driving around trying to find someone who could help.  Finally we found him.  There was an excellent fitter at a large brace company who had the right brace and got the job done.  At Mom's next clinic visit I told the neurologist about the hunt and the good result and the name of the young man and the company that was so helpful.  A month later I asked the doctor if he remembered where Mom got her neck brace and the name of the brace fitter.  He didn't.  Would the next caregiver spend a day off work looking for a neck brace and not be so fortunate as we were?  ALS was and still is the disease of reinventing wheels.

We need a Yelp-like tool where people can share their experiences with those who provide goods and services to those with ALS. If someone has a good experience, others should know about it.  If the experience is bad, why should the next person blindly stumble into a disaster?

Decisions that you have to make quickly when dealing with ALS are a matter of life and death.  They are also expensive, and it's hard to undo a bad one.  They are a lot more important than my travel or dining choices where I am so well-armed with information today.

Capturing and sharing experiences could be a tangible game-changer for people with ALS and their caregivers.  Finally.






Tuesday, October 7, 2014

Who Is Leading Us To Our Promontory Summit?

And what will we find when we get there?

Thanks to all who have mentioned the use and interest in a Global Unique (or Unifying depending on what you read) Identifier, GUID, for people in research studies. We now know that the NeuroBANK project that received a big bucket of funding from the ALSA windfall will be using GUIDs to key its data.

http://www.neurology.org/content/82/10_Supplement/S46.007


I found the information from the NIH National Database for Autism Research to be a most helpful  backgrounder on the concept --


The words that we are getting from ALS organizations and researchers are reassuring.  Since a wise parent once said, "The best predictor of future behavior is past behavior," we have questions about how this it will really happen.  How will will ALS researchers actually deliver new repositories of ALS data containing this common thread that can potentially relate data from multiple data silos?

Here are some questions --

  • Will the GUID generator be available to all researchers?
  • It appears that it's uncommon but possible for a GUID generator to come up with a previously assigned ID, and someone needs to referee and fix that problem.  Who is doing this small but important function for ALS studies? 
  • There won't be any proprietary pieces to getting and using GUIDs, will there?  The last thing in the world we need is for anyone to "own" or charge others for this simple collaborative piece of information.
  • When the personal information that is fed into the GUID generator is not available for a patient, it looks like a researcher can assign a temporary fake GUID.  Will there be a way to insure that fake GUIDs are consistent among studies or replaced quickly?  These pseudo-GUIDs could defeat the whole purpose.
  • Will people with ALS be told what their GUIDs are?  That would make a nice safety net to make sure that their subsequent studies are using the correct GUID, and maybe some of them will wear their GUIDs proudly as study participants.
  • Who is leading to make sure GUIDs in ALS research happen now?  We are at a critical juncture for ALS research and it's important that things move quickly and smartly, but somebody has to lead!   
If we don't get this right now, we'll pay the price forever. Studies are starting and expanding at this very moment. We're not asking organizations to share data today.  We're simply asking that they don't eliminate that possibility in the future by talking about GUIDs but not delivering now in a way that works for all researchers.

I'm sure that we'll have more questions.  Thanks to all who have enlightened us so far.


Saturday, October 4, 2014

Be Prepared


So far we have some "don't worry" and "we have it covered" and a "we'll encourage" reactions from ALS organizations regarding using a common, universal patient identifier among their newly funded information repositories.

I do worry, and I think that all involved can do more than encourage.

Will ALSA, each of it's IBC grantees, ALSTDI, AnswerALS, Project ALS, and others pledge to use a common, universal patient identifier in their IBC-funded information repositories?

We're not asking that they share data.  We're simply asking that they establish the groundwork so that if anyone would ever decide to share data, we would have a prayer that they would be meaningful. It's collaborate 101. Be prepared.



We've devalued so many words in the fight against ALS over the years -- "promising," "breakthrough," "exciting."  Please don't let "collaborate" be the next.

Thursday, October 2, 2014

NOW Is The Time To Get This Right! Collaborate.

The ALS Ice Bucket Challenge has primed the pump and we're starting to see some projects leave the starting gates.

At least three of the new ALS research initiatives involve people with ALS having their genomes mapped and a wealth of information assembled on each participant for researchers to mine.

Will it be business as usual?

Picture this --

Joe Doe has ALS.  He volunteers for Project Purple and is assigned a de-identified code 13579.  Joe leaves tissue and his genome is mapped. He leaves all kinds of health data that will be mined by researchers.  Joe gets a device that will measure his movements and the disease trajectory at periodic intervals.  Joe's tissue can be used as a lab model of ALS.  Project Purple even promises that other researchers will have access to its data.  Cool. 
Joe hears about Project Black and is assigned a code of 24680 when he is accepted into Project Black.  Joe leaves tissue and his genome is mapped.  He leaves all kinds of health data that will be mined by researchers, with many of the types of information different from what he gave Project Purple.  Joe will be contacted by Project Black researchers from time to time for more data and perhaps to be approached as a candidate for trials needing his variety of ALS, and again his tissue can become a lab model. Great. 
Wanting to give back as much as possible to the fight against ALS, Joe asks to be a part of Project Red that just got started in the U.S. as a result of some new funding.  Joe is given research patient  code ABCD12 and gives some tissue and his genome is mapped.  Joe becomes part of a huge data global bank of genetic mapping of people with ALS.  Awesome.

OK, we know that organizations and researchers love to say that they collaborate but they seldom really do.  Business as usual gives us three silos that don't even know whether Joe is in one, two, or three projects' gold mines of data.  And did they duplicate expensive screening processes that they could have shared?  Who knows?

Now's the time to put a very easy collaboration concept to good use.  Let's get this right. Now.

If all three projects would simply agree to share a research identifier for a person with ALS, think of the potential that we would have for the future.  If Joe were patient 265371 in all three projects, that would be one small step that would turn into a giant leap in the fight against ALS.  It would finally be possible to get inter-silo insights.

This is about as easy as collaboration gets.  This is about as valuable as collaboration gets.

We must get this right.  We must do it now.



Wednesday, October 1, 2014

We Learn From Questions AND Answers

Recently there have been some public online events on ALS organizations, their work, and the
#ALSicebucketchallenge windfall.  All have welcomed questions.

All have left my questions submitted online unasked publicly and unanswered.

While there is never enough time to get to every question during these events, there is surely time afterward to answer the unanswered questions in a public milieu.

We learn from questions and answers.

Burying questions isn't a healthy sign.

Friday, September 26, 2014

There Are Better Ways To Keep Track Of A Portfolio

For years we've asked for an orderly, searchable portfolio of ALS research grants.  Trying to keep track of press releases with no accounting of outcomes is a poor way for donors and people with ALS to understand and evaluate the research portfolio.

We recently got a Word document with new research grants.

There are better ways, and they're not rocket science.  Try this.

Search and find.  Leave fields empty to find all.

And wouldn't it be nice if the projects were soon to be filled in with grant amounts and outcomes?


Online Database by Caspio

Click here to load this Caspio Online Database.

Thursday, September 25, 2014

Gone In Sixty Seconds

On Sunday, August 31, I posted the following in the comments area of the ALS Association facebook page:

Some folks who participate supportively on an ALS message board started a petition a few days ago to ask that the ALSA IBC windfall be substantially directed to research.  They asked me to post the petition here.  It now has over 1000 signatures that include PALS, CALS, families, friends, donors. The message is simple. Thank you for listening.
https://www.change.org/p/urge-als-association-to-dedicate-at-least-75-of-its-ice-bucket-challenge-donations-to-research


Within minutes (literally) it was deleted and I was blocked from commenting or liking anything there again.

Also deleted in the Sunday-night purge was one other posting that I had made days earlier  suggesting that an infographic showing what happens to a hundred dollar walk donation and what happens to a hundred dollar national donation would be most helpful.

ALSA certainly has every right and responsibility to manage its facebook area as it thinks best.

I still believe that both comments are worth some thought and archive them here.


Wednesday, September 24, 2014

Let's Rewind The #ALSIceBucketChallenge Tapes And Look Again

It's remarkable as you look up old ALS ice bucket challenge videos on YouTube or national media websites.  Most of the big ones mentioned donating to "ALS research" or "to find a cure" before flashing the link to donate at www.alsa.org.  The frenzy was built around that message blurted out by so many national celebrities and leaders.

Clearly, donor intent was ALS research.  One can't assume otherwise, especially since the online #IceBucketChallenge donation form had no gift-restriction options for weeks.

As the long-awaited plan for the funds is being designed and the fight against ALS moves into high gear thanks to the buckets of generous donations, we trust that donor intent for ALS research is being honored.

It's simply the right thing to do for many reasons.

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. 
http://www.asha.org/News/2014/Action-Alert--Help-Protect-SGD-Access-for-Medicare-Patients/ 
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 www.alsa.org.  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 --

http://www.pbs.org/newshour/bb/will-als-ice-bucket-challenge-money-spent/
"... 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.

ALS TDI www.als.net
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. http://www.als.net/ips/prescreen/?f=hr  It's immediate and important for advancing the science.

The Gleason Initiative Foundation www.teamgleason.org
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 www.petefrates.com and Pat Quinn www.quinnforthewin.com

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:

http://org.salsalabs.com/o/777/p/dia/action3/common/public/?action_KEY=16250

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.

https://wwwn.cdc.gov/als/Default.aspx

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.