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, 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.