ALS Advocacy

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

Tuesday, December 31, 2013

The Rolodex - Part I

A few weeks ago I was invited to a meeting of patient/caregiver/advocates held by the Clinical Open Innovation Team at Eli Lilly and Company. Their goal was to get direct patient perspective on how clinical trials can be more patient-centric. I hope the day was as productive for them as it was energizing for me.

There were some nationally prominent e-patients there. It was easy for me to feel a little lacking and very star-struck. There was also a large contingent of Lilly staff members included and they stayed with us the whole day. I realize it’s not an easy thing to block a whole day to focus on a single topic in corporate America. It happened.

As we talked and listened, I mentioned that I have a pretty big Rolodex of ALS patients and caregivers. The day before the Lilly meeting as I tried to prepare some thoughts, I found myself wanting to reach for the Rolodex to get their opinions that I value so. Each time I wanted to pick up the phone or send an email, I got that awful feeling that you get when you realize that a trusted advisor or friend is no longer with us. ALS is a disease that provides a big Rolodex and lots of those terrible losses.

Regina Holliday was one of the participants at the meeting. I had known of her Walking Gallery project http://reginaholliday.blogspot.com/ and thought it was a wonderful idea. When I saw some of the Walking Gallery jackets in person at the Lilly meeting, I thought it was more than wonderful. It was "wow." It has an impact.

Here are more links to give you some insights into Regina and her work --
http://en.wikipedia.org/wiki/Regina_Holliday
https://www.youtube.com/watch?v=-kMy1OvDJcw

After the meeting Regina offered to paint my Rolodex. Her offer still makes me grin from ear to ear. Now it’s time to gather some information for her about a few of the ALS rockstars in my Rolodex whose wisdom I miss so.



Mom
She is truly #1 in my Rolodex and in my life. Mom was a good mother and a wonderful homemaker and a wise human being. I have a feeling that instead of a portrait that she would prefer that I include a picture of how she raised ALS awareness a few years ago on Pennsylvania Avenue in Washington – as part of the stunning Piece by Piece display. She so wanted to get the word out about ALS – a disease that outrageously is without a treatment. She left us with some timeless quotes for the fight against ALS, including "If you can’t cure me, then at least learn from me." Amen. We lost Mom to ALS in 1997.

Persevering (the online name for Rob Tison)

 
Persevering was a young father and a really smart statistician. He was smart about a lot of things. His abilities in statistics showed us that patients can sometimes find the weaknesses in peer reviewed articles and in clinical trial design. Rob was a relentless recruiter and advocate for clinical trials, and he managed to start conversations among scientists and physicians and patients and advocates who usually don’t talk to each other directly. Rob was a game-changer for the fight against ALS. We lost Persevering to ALS in 2012.

 
Happy Physicist (the online name for Ben Harris)

HP as I called him was another really smart scientist and young father who was slapped with an ALS diagnosis. He worked up until the end as a physicist on proton accelerators used in cancer therapies. He once told me that he designed equipment that took people within inches of their lives so that their cancers might be treated. He didn’t see why someone with ALS didn’t have the same right. His mantra was, "If it is done in secret, then it is done in vain." He wanted people with ALS who were trying things (and they all do) to be sure to document their data so that others might learn and the science might move forward. One morning I opened up my Wall Street Journal there was HP on the front page, written up as a citizen scientist trying to find something to treat ALS and to push the clinical trial process aggressively. He was smarter at science than most citizens and most scientists. We lost Ben to ALS a few months ago.

And in one of life’s coincidences (or perhaps a bit of kismet?), Rob, Ben, and I all had dogs named Otis.


bbzinger (the online name for Barbara Brenner)

I first e-met bbzinger during a tweet-chat led by USAToday. She was the leader of Breast Cancer Action and that was the topic of the chat. She also had recently been diagnosed with ALS. I learned so much from her about the differences between advocacy and activism and about the ways organizations need to be challenged. She was an ALS clinical trial volunteer and kept her commitment to that trial even though it didn’t seem to have fair or transparent rules for her situation. She was a very smart attorney who was driven to make a difference with the FDA and organizations and the way diseases are fought. Her blog at barbarabrenner.net also taught us much about dying. We lost bbzinger to ALS earlier this year.

Pdwyer (the online name for Pat Dwyer)

Pat Dwyer was simply a great guy. Perhaps that’s not really so simple. Pat was a husband, father, and had a commercial fishing business in Seattle. He and his wife searched and found a clinical trial for a Diaphragm Pacer with the help of Dr. Google. They became a wonderful resource for people online seeking advice on the DPS and ALS in general. Their attitude was simply that ALS picked the wrong family when it picked on theirs (yes, another one of those "simple" things that’s not so simple in real life). His wife and young-adult children and relatives and in-laws and friends carry on the fight, having lost Pat to ALS in June. At his funeral the church was literally jammed with all kinds of different people with one thing in common – they loved a great guy named Pat.


The Rolodex - Part II to follow tomorrow, New Year's Day.

Monday, December 30, 2013

Good Financial Advisors Love Clients Who Ask Questions

And good financial advisors give good accounting about what went well and what didn't go so well in the past year.

You expect a few duds in a diverse portfolio. You expect to know about them and to learn from them.

You expect complete numbers that tell you what the returns on your investments have actually been.  You have a right to know how much money went into and is now in each of your portfolio folders.

If you knew someone naive with a financial advisor who didn't like questions or who didn't give a good, detailed accounting, would you not advise him or her to run away?   Something is terribly wrong. 

We trust our ALS organizations that fund research to be very much like good financial advisors. They help us place our investments in the most promising science. We know that some things might work and many won't work, and we expect to know about them and to learn from them.

Instead of good, businesslike accounting of projects, investments, and outcomes, we get cherry-picked  public statements on "promising" results that don't give us a clear picture of the portfolio. We see selective cumulative summaries instead of specifics.  Instead of answers to our questions, we often get evasive responses, or worse yet, no responses.

Surely they would be smarter advisors and we would all be better (and bigger) investors if we had businesslike accounting of their (ergo our) research investments in the last year. Surely it would advance the science and the funding available from investors.

Tuesday, December 17, 2013

We Got Plenty Of Nothing



When will there be a candid, honest discussion of the CDC's ALS Registry?  Was there one at the annual meeting in July?  Is anyone looking seriously at the scope, deliverables, and effectiveness of the very expensive project?  Is there a valid reason for the cloak of secrecy?  Really?

Following is the item in a recent advocacy "listening" survey (click here) (thanks to the chapter that shared) related to the ALS Registry:


6. National ALS Registry Issue- (may include- National Office to continue support Chapter outreach and enrollment activities by providing materials, equipment, education and training; Continued funding for the National ALS Registry; Expand the number of surveys in the registry. Encourage research use of the registry.)
   
 1st /2nd/3rd/Not a top priority this year



A single tick (or not) doesn't address the considerable concerns that many of us have --

  • What are the deliverables that ALSA and now MDA deliver to earn the millions that they have been granted in contracts? 
  • What are the performance standards for those deliverables and have they been met? 
  • Is anybody bothered by the lack of transparency over these contracts that advocates stumbled upon quite by accident? 
  • What is the uptake on self-enrollment? 
  • Who is questioning the scope-creep of this project? 
  • Are the risk-factor surveys eliciting sufficient response to be statistically meaningful? 
  • Who attended the annual meeting in July? What was discussed? Was anything challenged?
  • How do we proceed and best deploy resources to make this project effective?
So far we have plenty of nothing for tens of millions of dollars.  No answers.  No meaningful status information.  No sign of responsible project management.  No transparency from organizations that were given substantial, non-competitive contracts.

Nothing is not plenty for us.

Monday, December 9, 2013

Patients, Caregivers, and Families Have Questions. You Have Experts.

My, how they have questions.  My, how you have experts.

A reported 950 people participated in the big ALS MND symposium in Milan last week.  A vast majority of them were experts in some area of ALS research or care.  Almost of them were there as part of their jobs.

The "Ask the Experts" session that was held on Wednesday was similar to that held in past years.  We're not sure who was in the room.  Three experts talked and answered a few questions.  It was interesting, but it was hardly an inviting, inclusive way to get a lot of patient and caregiver questions to the experts.

How about crowdsourcing some questions and answers next year?  Six weeks before the symposium, reach far and wide to invite PALS, CALS, and families to ask their questions.  Encourage organizations to let their PALS, CALS, and families know about an amazing opportunity.  Gather their questions.  Some will be easy, some will be difficult, some may be uncomfortable, but all will be valuable.

Next invite every one of your expert presenters to have a crack at answering the questions in writing before the symposium begins.  Multiple answers to a question would be grand.  If experts differ on answers, that would actually an interesting dimension that has been been missing from the fight against ALS.  Let's swarm on those questions, experts.  If some experts don't want to answer questions, that would be a bit revealing, too, wouldn't it?

Finally, at the big conference, a panel of experts could discuss the themes and the responses.  And please retain the written content of the questions and responses in a public place.  That will help all of the families who feel like they are reinventing wheels every day in their fights against ALS.

This format would permit every question to be asked and answered.  It would draw in more PALS and CALS and experts into a direct conversation that they are not having today.

It would be a small step toward patient-centricity at an important symposium that seems to be tone-deaf to the concept today.  It might also enlighten some of the so-called experts that the real experts in ALS are those who are living the nightmare every day.


Saturday, December 7, 2013

That's My Horse!


There is a really troubling phenomenon in reports that we get from scientific meetings and publications these days.

If the presentation or report is positive, we get "we funded that research" messages from some ALS MND organizations.

In real life, we don't know if it was a $2 bet or a $2 million bet that it  placed.  A bunch of $2 bets can turn out to provide some pretty impressive advertising.

And we don't know if a not-for-profit shines kinder lighting on projects that it "funded."

Just an observation.  It's just another reason why we need better, centralized information on our not-for-profits' ALS research investment portfolios.

Friday, December 6, 2013

A Speeding Ticker Might Add An Interesting Dimension Next Year

There will be around 24 hours of scientific presentations over the next three days in the big global ALS MND symposium in Milan.

What if there were a ticker running under every Powerpoint presentation showing the names of the people who have died from ALS MND since last year's symposium presentations.

Would that be distracting?  Darned right it would be.  It should be.

So how fast would that ticker have to run?

Around 133,333 global deaths in a year / 24 hours = around 5,556 names per hour...

Around 5,556 names per hour / 60 minutes per hour / 60 seconds per minute = around 1.5 names per second.

Around 1.5 names per second moving relentlessly through all the scientific presentations and promise... That speeding ticker will be a measure that won't be pleasant to watch.  It's time we watched.


Thursday, December 5, 2013

Dove Sono I Pazienti?

The years pass and the problems persist.

http://als-advocacy.blogspot.com/2012/12/look-whos-not-listening.html

http://als-advocacy.blogspot.com/2012/12/watch-out-shes-gonna-blow.html

Almost a thousand ALS MND organization employees and scientists and clinicians are meeting in Milan for the big annual symposium this week.  The format, accessibility, and fees continue to insulate them from the very patients and caregivers whose lives are at stake.

So far this week we saw the rollout of a twitter campaign to #shoutloud about ALS.  My hopes were up that it was about raising awareness of the global problem with those who are unaware.  It was disappointing that the twitter campaign wasn't designed to engage journalists and drug developers and activists and scientists and celebrities.  It was yet another ALS social media fundraiser.  http://www.mndfundraising.asn.au/event/shoutloudforalsandmnd/

Yesterday they had the "Ask the Experts" session, and I'm grateful for the livestream (although the time and link were a bit fluid until the last minute).  The experts were interesting.  It was more of a "Listen to the Experts" session, but it was informative.  I have a feeling that if the room had been full of people with ALS that the doc who ended his presentation with what was supposed to be a cute "it could always be worse" ending might have rethought that one.

Today the scientific presentations begin and we continue to have a virtual moat around the conference hotel.  Thanks to the few tweeters who move past pep-rally tweets and send us their insights into interesting content.

Every day people are diagnosed with ALS and they and their loved ones scramble to find out the most promising (and practical) ways to proceed.  It's hard to sort out.  This week we have the most renowned scientists discussing the most "promising" science, and those people with ALS and their loved ones are on on the outside, waiting for information through the lens of someone else.  It's nuts.  And it's nuts that people working on ALS science aren't hearing from directly from patients and seeing patients and learning from patients.

We live in a world that talks about the importance of patient-centricity.  Talk's cheap.


Saturday, November 16, 2013

My Fifteen Minutes Are Up

Lately there has been an abundance of platitudes about and for caregivers as November is National Caregivers’ Month.  There are days when it seems to have that aura of a Hallmark holiday.   I can envision ALS caregivers wanting to scream.  Telling ALS caregivers to be good to themselves and to make time for themselves usually comes from people who have little ALS caregiving experience.  It’s a nasty, demanding disease, and to add to the physical exhaustion, caregivers are sucked into a uncontrollable catastrophe where a loved one is slipping away.

So instead of suggestions of support groups and massages and special time to oneself, we would all do better to drop off a dinner (everything… not just a main course) or rake the leaves or run some errands or clean the gutters for a caregiver.

And I will pass along  one thing that was so helpful to our family in dealing with ALS.  We learned it from another family that had been dealt some pretty crummy cards in life.  They had a 15-minute rule.  You get 15 minutes of self-pity every day.  When your 15 minutes are up, you have to move on and deal with life. 

I can’t tell you how many times we had to remind one another that our 15 minutes were up.  We smiled and moved on.  The 15-minute rule was a caregiver’s best friend.

May God be with all who are caregiving and dealing with ALS, both this month, and every month.

Wednesday, November 6, 2013

Travel To Defeat ALS?

In the last year the whole concept of travel in the fight against ALS keeps popping up in different ways.

  • One 501c3's IRS Form 990 shows that a huge proportion if its investment in the fight against ALS is spent on travel... to some pretty nice spots.
  • At big ALS meetings, a large percentage of the attendees are often employees of our not-for-profits or our government.  They are on the meter with their travel expenses paid.
  • At ALS conference during the government shutdown, a government-employee panelist did not show.  Did the sense of commitment stop when the expense account was turned off?
  • The July Team Gleason Summit in New Orleans had a large number of ALS scientists and not-for-profit employees whose expenses were paid by Team Gleason.  Would they have engaged if they had to buy their own tickets and hotel rooms?  Would they have been willing to go to Fargo?
  • I often wonder if some people who make good livings and blather about their commitments to the fight against ALS would really show up on their own dimes.  It's business to them.

As businesses get more savvy about confining reimbursed travel to trips that will make a tangible difference, have our not-for-profits become more sloppy?

And at the end of every ALS meeting, before adjournment, let's start to require a commitment to some action item that will make a tangible improvement in the fight against ALS within 30 days.  If you can't do that, maybe you should get a bill for your travel expenses.


Tuesday, November 5, 2013

Let's Go For Some First Downs

In the big meetings of 2013 when we heard talk of winning the game with transformative game plans.  We heard of lots of hope for scoring against ALS.  We heard a lot, yet little was delivered.

Let's play some small-ball and go for some first downs before the end of the year.  We can advance the ball in some very realistic ways.

1. Let's put heads together and come up with a searchable inventory of research projects.  Let's treat ALS research like an investment portfolio that everyone should want to understand and, therefore, support.

2. Let's put heads together and share a calendar of activities pertinent to ALS.  One calendar would help everyone be aware of events and conferences.  One calendar would help organizations in scheduling.

Those are pretty simple plays.

We'll never win the game if we're not able to move the ball just 10 yards.




Saturday, November 2, 2013

Some Of The Most Powerful Ideas Are So Simple


http://www.gocomics.com/peanuts/2013/11/02#.UnTZ5xzxZ9R


Peanuts by Charles Schulz
Gocomics.com
November 2, 2013

Every family dealing with ALS today is hanging hopes on something.  Every organization funding ALS research is promoting its hopeful work.  All place their bets the best way they can today and believe and hope.

For decades ALS has outsmarted hopes and dreams.  ALS is a cunning enemy.  When things families and organizations hope for don't happen, people die and families and organizations move on.

Whoops!  What's to keep the next "generation" of people dealing with ALS from pinning their hopes on a concept that already failed?  What's to help people investing in organizations' hopes from placing some very bad bets?

It's difficult for people and organizations to admit that they have failed, especially when optimism drives fundraising success.  Everybody loves a winner.  

We need to be honest that we have all failed, both individually and collectively, to find an effective treatment for ALS.  It's that simple.

And to make some good come out of those failed hopes, let's start keeping some records of what didn't work.  Please!  Let's build on the information of the past rather than continuing to recycle hope. 

It's time for a good, searchable database of ALS research investments and outcomes.  We need to learn to admit when we were wrong and to build on that knowledge.  We need to expect some refreshing honesty and accountability from organizations, and we donors need to support organizations that are big enough to document what has failed.

Roy hit the nail on the head in this morning's newspaper. 

Friday, October 18, 2013

Paging Dr. Google, Paging Dr. Google

If you are new to ALS and searching for a treatment or a cure or simply hope, here's a tip.

When using Google, be sure to try

  • Search Tools
  • Any Time
  • And change the time frame to some years in the 1980s or 1990s or 2000s.
That won't be a pleasant experience, but it will help you sort out what's new versus what has been around and tried for decades.  It will help you sort out the revisionist history that you may hear from people or organizations.

We all wish you well in your search.  There are certainly things worth trying, but please go in with eyes wide open that some concepts have been around since Lou and Eleanor and that some so-called experts have been tossing those "exciting" and "promising" and "breakthrough" words out for decades.

And if you try something, even something simple, be sure to leave a trail of your experiment at a public place like www.patientslikeme.com so that others may benefit.  Let the data speak.  As you have probably found by now, people with ALS hear far too many anecdotal reports and not enough data.

Prayers for all fighting this beast.  


Tuesday, October 15, 2013

Travel Teaches Lessons That Last A Lifetime

The year: 1989
The place:  Moscow, in the olde U.S.S.R.

Every time our government-guide/chaperone pointed out something of interest on our left, we soon learned that there was something on our right that she didn't want us to notice.

Who knew that this lesson would come in so handy in life?

Saturday, October 12, 2013

Thank Heavens These People Didn't Design Our Interstate Highway System

Imagine a highway system where the people who wanted a highway from New York to Chicago built one.  And the people who wanted a highway from Cleveland to Saint Louis built themselves a road.   And the people who wanted a highway from Kansas City to Denver poured their own pavement.  And the people who needed to get from Dallas to Los Angeles constructed their own road.  And the people in Seattle who wanted to get to Miami blazed a long and impressive trail.

A mess?  Indeed.
An expensive mess?  Yep.
An unbelievable premise?  Sure.

Today we have a very expensive ALS "Registry" project that was implemented by the CDC.  We know little of its status because of the cone of silence that has enveloped those who are invited to participate in its annual meetings.  We know that it has passive surveillance of government records of ALS cases at its backbone, and we know that there is some self-enrollment that complements the mined data.

We also have a PRO-ACT database provided by NEALS that houses clinical trial data on many of the same people who are in the CDC's registry.

And the MDA has recently announced the launch of its own patient "registry" that will capture data related to patient care and natural histories.  We assume that this will house information on some people with ALS who have fragments of information in the CDC's "Registry" and the PRO-Act database, too.

And at the recent Team Gleason Summit, leading ALS researchers spoke of the need for some "big data" concepts of holistic views of patients so that every patient can be a research patient.  Oh, and nobody present involved with the CDC project or the MDA project mentioned those within earshot of the webcast.

A mess?  Indeed.
An expensive mess?  Yep.
An unbelievable premise?  We're living it.  Oh, and we're paying for it.

Friday, October 11, 2013

Lou Knows



Ability is what you're capable of doing

Motivation determines what you do 

Attitude determines how well you do it 



The fight against ALS has failed to deliver a treatment. We must face that we collectively and individually have failed. Feel-good pats on the back for effort don't save lives. We have not won the fight.

Is it our ability?
Is it our motivation?
Is it our attitude?

We must fix all three.  Lou knows.

Wednesday, October 9, 2013

This Is Putting Heads Together?

  1. A few weeks ago Team Gleason assembled an unprecedented group of scientists, clinicians, and organizations (at Team Gleason expense) to put their heads together and fix the fight against ALS.  The MDA was among the organizations represented.
  2. Throughout the Team Gleason Summit, conversations touched on information needs and the data concepts that would be central to speeding the development of therapies.  It was a persistent thread.  
  3. As I listened on the webcast, I didn't hear a peep from the MDA about information or data.  Not a word.  Nothing.
  4. Today we see this big announcement from the MDA --  http://mda.org/media/press-releases/quintiles-selected-muscular-dystrophy-association-develop-us-disease-registry


So much for putting heads together.  I'm not sure that Team Gleason got its money's worth.

Thursday, October 3, 2013

The Difference Between Hope and Hype Is A Single Letter

We learned a lot from the discussions at ALSTDI's conference today.

One of the afternoon panelists made the statement that stuck with many of us -- The difference between hope and hype is just one letter.

I've longed for some kind of Pinocchio-meter for all of the "promise" touted in the fight against ALS.  Perhaps we can use a Hope-Hype image that fades to the correct vowel when time reveals the truth.

Please tame the hype.

Wednesday, September 25, 2013

It's Time To Dump The "Pick A Leader And Get In Line" Fight Against ALS

Occasionally a big light bulb turns on in my dim mind.  This week I experienced a lot of lumens as I worked in an online group trying to plant some new seeds in the fight against ALS.

People waste a lot of time and energy vying for so-called "leadership" in this fight.

When people experience ALS, it is overwhelming.  You have to pick your battles and you have to learn to trust people who can help you.   You also have the urge to make a difference.  It's a delicate equation -- trying to find your leader who has blazed trails before and trying to help the next person.

Add the whole instinct to survive which drives you to those leaders with "hope" written on their backs.

And there is a constant stream of rookie recruits.

And the disease awareness is so poor and the disease demands are so high that resources are scarce.


For years we've operated under a pick-your-leader-and-march mentality.  It hasn't exactly worked well.  Organizations and individuals vie for followers, and people in lines aren't exactly using their creative juices well.

We need to smarten up.  We need to recognize strengths and weaknesses in organizations and people.  We need to break up the lines and learn how gang up on problems and not each other.  We need to stop meandering behind leaders.

Oh, and those who think they were born to lead us need to stop spinning stories to attract followers.  Let the data speak.  We'll figure out when and where and whether to follow today.

And sometimes the one who appears to be the least among us at the back of that line has the idea or the common sense that we really need to follow today.

Tuesday, September 24, 2013

Let's Have The Most Painful Meeting Ever

Since this seems to be the year of meetings to defeat ALS, maybe this concept of two serial meetings would actually move us forward together.

Let's start with a virtual room full of people with ALS, caregivers, some retired pharmaceutical executives, and some retired FDA officials.

Why the retirees?  They know their businesses, yet they have no horses in this race.  They don't have to worry about walking on eggshells around people who are dying.  They can be brutally honest.  They can be informative and candid.

Stakeholders need to stop trying to assume that they know what the others want or need.  They need to talk.

Meeting 1:

OK, let the first meeting begin.  Let's talk about investment in ALS drug development.  Let's talk about the business of bringing ALS drugs to market.  Let's talk about the short-term and long-term financial incentives.  Let's talk about the approval process as it really is.  Skip the Ph I,I,III basics. We hear those over and over but seldom get past them.  Let's talk about breakthrough status and expanded access programs and markets for drugs. Let's talk about how drugs are paid for. Let's talk about how a program that is good for a rare cancer drug may be not so good for an ALS drug.  Let's hear the heartburn that each special program brings to each person in that room.  Let's learn from one another.  Let's tell it like it is.

That's a pretty painful meeting for all parties, but it would be tremendously constructive.  Today patients dream dreams about programs that they think could bring drugs to them.  Are they even viable?  Are they conducive to industry investment in ALS?  Are they products of an FDA that today doesn't have good "special" programs designed for an ALS?  Are there paths we've not considered.  What do patients really want?  What do drug developers really want?  What do investors want, today and tomorrow?  What does the FDA need? What does everyone need?  

All the cards and reality need to be on the table.  We need to be candid with one another.  That's a constructive, uncomfortable, painful meeting that has never happened before.

Meeting 2:

Ah, now we're ready to design some solutions.  Now that we've stopped assuming and mind-reading, we can start the creative process.  Let's break through the maze and find a path to get therapy candidates to patients more quickly.  Let's not try to bend existing programs to fit ALS if they don't really serve all the stakeholders well.  Let's move the fight forward creatively on solid ground.


We've had far too many variations of Meeting 2 already this year.  We need a Meeting 1.


Saturday, September 21, 2013

Just The Facts, Please

History is an essential asset, yet in the fight against ALS, we do a very poor job of retaining it.

The nature of ALS is that the people dealing with it don't have memories of what happened five or ten or twenty years ago.  They are slapped with a diagnosis and are largely on their own to find information and to figure out whom to trust.

We need to do a better job of retaining what really happened two or five or ten or twenty years ago.  We need to make the fact-finding easier for those with ALS who seek a ray of hope.

Occasionally those of us who have been paying attention to matters ALS for more than a decade see or hear someone spouting revisionist history.  It may be innocent, or it may be manipulative.  In either case, it's wrong and it does not help the fight move forward.

Today's technology could give us an excellent electronic archive of historical facts.  It's time for organizations and individuals to contribute facts lest we forget or are led astray or repeat the mistakes of the past.

Saturday, September 14, 2013

Pick Your Game Of Musical Chairs

The same number of people will be added to each game continuously.  There are a lot more chairs in the blue game than in the red.

The undertaker removes people who don't have chairs when the music stops.

Which game would you rather join?


This shows the flaw in using prevalence (the number of chairs) as the measure of a disease's impact.

The small number of ALS chairs doesn't mean that it's a small problem.  It means that it's a huge problem!


Saturday, September 7, 2013

Aren't There Ever Any Scouts At The Table During Trial Design?

Envision a person with ALS who has volunteered for a clinical trial -- a trial designed in much the same way that researchers have traditionally designed trials for rashes or cholesterol control or male pattern baldness.  There is trial period with the drug or placebo, and when that's over, everyone waits while results will be evaluated and the drug company will decide if and how to proceed to another trial phase.

For ALS trials, it seems like the trial designers never seriously consider what happens to their trial volunteers if this stuff really works.  It's not like they'll just have to scratch an itch or watch their diets or comb over while they wait to see what's next.  They decline quickly. They die.

People with ALS who have volunteered for trials and who perceive improvements during the trials are left hanging out to dry.  This all could be avoided with some creative trial design that gives them options to continue on the drug (or the placebo... if it works, they really don't care) before they sign on the dotted line.

Instead we have traditional trial designs and the subsequent cry for Expanded Access Programs after the fact.  Perhaps we should get in front of the problem and have trial designers, including patients, talk about what happens to trial volunteers if this stuff really works.  Be prepared.




Wednesday, September 4, 2013

Stuck In The Middle WIth You

The organizational fight against ALS loves the middle.

At the recent Team Gleason Summit or February's FDA Hearing, who was dreaming big, daring to change the basic ways that business is done to fight ALS?

Who was dreaming small, willing to step up and deliver some simple basic changes that would tear down silos that make the armies fighting ALS so inefficient?

The battlefield against ALS is so comfy in that middle where "promising" projects become expensive and big and not game-changing... and the deliverables are poorly measured and nobody is really held accountable.

Pffft.


Friday, August 30, 2013

Pick Your Bus

Here are the ground rules.

  • There is one empty seat on each bus.  Take your pick.
  • Each bus will pick up five people today.  Five riders will have to give up their seats to make room for the new riders and will be taken off the bus by the Grim Reaper.


One bus has 15 seats.

The other bus has 65 seats.

Which bus would you pick?

The smaller bus is a much bigger threat, eh?  Who would ever pick it unless you were anxious to get kicked off to meet the Grim Reaper?

This demonstrates the myth that smaller disease prevalence (the size of the bus) means smaller disease impact.

ALS is the Grim Reaper Express Microbus.  It's a lot worse than a big bus.

Tuesday, August 27, 2013

I Was Hungry...


They say that the bitterest words at final judgment will be, "I was hungry, and you formed a committee."

Perhaps a fitting corollary to that will be, "I was dying, and you had a meeting."

Food for thought in this, a big year for big ALS meetings.

Saturday, August 24, 2013

What Are The Barriers?

A few months ago I was discussing ALS with some people far smarter than I.  A gentleman asked me a thoughtful question -- "What are the barriers between you and a treatment for ALS?"

Wow.  I had to think about that.  I had a diverse list of answers.

What if various stakeholders in the fight against ALS put heads together and were candid with each other about the barriers they perceive?

We all might learn a few things.  We all might be able to move a few of those heavy sandbags.

Friday, August 16, 2013

Happy Physicist Taught Us All A Few Things

A gentleman with ALS who went by the online name "Happy Physicist" died yesterday. 

HP taught us all much. 

He was a scientist and is best known for his constant study and experimentation on himself to see if he could unlock a clue to treating ALS.  He understood science and he knew what ALS would do to his future if he sat back and watched.  And HP encouraged all people with ALS who tried things (and they all do) to share their data.  "If it is done in secret, then it is done in vain."

There is another mark that he left that some may not remember, so I take this sad day to remind us all.

HP studied available clinical trials and signed up for one he thought had promise -- Neuraltus NP001.  HP also knew that a day waiting to fill a clinical trial was as precious as a day waiting for the FDA or for scientists.  He took matters into his own hands to get that clinical trial filled quickly.   He used every social media device known to man to let people with ALS know about the trial.  He was a gazelle.

HP was from Bloomington, Indiana, and he traveled to Lexington, Kentucky, for his clinical trial infusions.  That was a long drive but worth it to HP. On one trip, HP got online and found the ALS Association Lexington support group meeting was happening while he was there.  He showed up at the support group meeting to try to recruit his peers to give the NP001 trial a whirl.  Here's the shocker -- they had no idea of this clinical trial opportunity in their own back yard.  Several signed up right away.

So the next time clinical trial sponsors or organizations or research scientists complain about patients not willing to enroll in clinical trials, look upward and remember HP, and tell them that perhaps someone should simply tell the patients about the trials.

Thank you, God, for HP.

Saturday, August 10, 2013

Prevalence Is A Red Herring

For years we have been told by our largest ALS organization that disease prevalence is the "gold standard" by which the public sector measures the relative impact of diseases.  That's just the way it is.

Questions from Congresspeople or Senators about constituents with ALS are typically answered with a logical guess based on a number that has been couched and hedged for years -- "As many as 30,000 Americans have the disease at any moment in time."  Carefully worded, eh?

Everybody knows somebodies who lost somebodies to ALS.  Oh, it's so easy to want that prevalence number to be higher so that ALS will rank higher on America's priority list.

That's a red herring -- a big stinky red herring.

We believe that as many people are diagnosed with ALS as MS every year.  That's incidence.  

The number of people living with ALS at any moment (prevalence) is a tiny fraction of those living with MS.  What happened to all the people who were diagnosed with ALS?  Hint:  It's not good.

Does smaller prevalence make ALS a smaller problem than MS?  Heavens, no!  It makes it a much deadlier problem.

High incidence and smaller prevalence form a toxic combination.  If you only look at prevalence, you are missing the boat.  Low prevalence does not make ALS a small problem. It makes ALS a huge problem.

So as the ALS Registry numbers unfold, we'll probably find in the data that there are far fewer than 30,000 people living with diagnosed ALS at any moment in the US.  Should we avoid that low-prevalence fact?  We need to stop leaning on a shaky prevalence guess that feels good for fundraising and speak to the data.  High incidence paired with even smaller prevalence mean that we have a large, urgent problem.

It takes a smart hound not to be thrown off by the scent of a stinky fish.  We need to be smart in the fight against ALS.  Leaning on an assumed "gold standard" with a shaky number isn't going to defeat ALS.

This troubling ramble that worships the red herring is from the report of the CDC's 2012 ALS Registry Annual Meeting at http://wwwn.cdc.gov/als/Download/2012%20ALS%20Annual%20Meeting%20Summary%20Report.pdf
While he did not want to hide information, it could possibly come out that there are not 30,000 people 
with ALS and that it is much lower.  There would be a lot of negative connotation about what this 
all means if there is a map with people in each state.  He could hear appropriators and 
Congressman saying, “You only have 2 people in the whole State of Texas who have this 
disease?”  He thought it was very important that consideration be given to everything in terms of 
research projects, because the intention was never to figure out the number of people who have 
the disease.  If incidence is higher in one state, that is important.  While they should not hide 
information, they should also not be distributing information that could be damaging to the 
research tool they wanted to create, particularly in a budget-conscious environment.  That could 
hurt the overall goal of finding a treatment and cure. 
[Hat tip to The ALS Battlefield for all his work.]

Friday, August 9, 2013

Let The Data Speak

What are the barriers between people with ALS and a treatment?

That's an important question.  There are some answers that may be surprising.

Organizational secrecy causes some big barriers, and it serves nobody well except organizations.

Think about it.

Let the data speak.

Thursday, August 1, 2013

Please Love Us (And Our Friends) For Our Minds, Too


Dear _____,

It's that Walk time of year.  As a participant in past Walks, we want you to be among the first to sign up and help us raise valuable dollars in this year's Walk.

Please sign in online and donate what dollars you can and tell your friends and ask them to donate as many dollars as they can.  Spread the word.  It makes a difference.  Your contributions advance the valuable work of our organization that is focused on beating ALS.

Thank you for your past contributions and we look forward to your help and the help of your friends and theirs and theirs as you all contribute valuable dollars to help in this Walk to fight ALS.

Sincerely,

________________

Ok, now simply substitute the word "ideas" for "dollars" and substitute "Advocacy Effort" for "Walk."

Get my drift?



Wednesday, July 31, 2013

Dear People Invited To The CDC ATSDR ALS Registry Surveillance Annual Meeting Today

If the veil of secrecy is required, please explain that requirement to us.

If the veil of secrecy is desired, please explain that to us, too.

Such secrecy is not healthy for good project governance and is clearly not in HHS's spirit of patient-centricity.

Thank you.

Monday, July 29, 2013

Howdy, Partners

We think that the supporters of the CDC's ATSDR National Registry and our major ALS organizations deserve some candid and clear information about who has been paid to do what from the government funds that we fought hard to have approved.

From ALSA's 2012 Annual Report, the most recent on its website, under the heading of "Fighting for the Interests of the ALS Community," there was mention of a "partnership" with the CDC to help raise awareness of the National ALS Registry.  There was absolutely no mention of any kind of contractual or vendor relationship or compensation related to the $6 million which we taxpayer/advocates requested of all taxpayers for the Registry.


 
 
Then we stumbled across this cool million (quite by accident) a few weeks ago at usaspending.gov.



 
 
And what in the world is this about?


 

We understand that the ALS Registry powers that be are meeting in Atlanta this week.  We hope that they'll shine some sunshine on their proceedings and processes and "partnerships."

Wednesday, July 24, 2013

We Should Have A Large, Angry Crowd By Now

Around 10 million people have died from ALS/MND since Lou Gehrig's diagnosis in 1939.

That's like the entire population of Belgium or Sweden.

That's an outrageous loss of life.

Who can lead the many more loved ones of these 10 million people into a cohesive movement?  The base grows, yet the engaged base doesn't.

Porquoi? Varfor?

Saturday, July 6, 2013

We Have Three Very SImple Questions Regarding The CDC's ATSDR National ALS Registry

What percentage of the total patients in the Registry have been identified...

Q1: Both by your passive data mining and by self-enrollment?

Q2: By passive data mining only and not by self-enrollment?

Q3: By self-enrollment only and not by passive data mining?

How else are we to know the effectiveness of the self-enrollment efforts?  Whoops, I guess that's a fourth question (but only if we can't get answers to Q1, Q2, and Q3).

And if we can't get the answers because of some OMB policy, then yet another question is whether the OMB gag order is OMB's alone or whether there was an agreement presented to OMB by the CDC which prevented this disclosure.

Yes, it turned out to be five questions, but there are only three if we get the simple (and important) responses to Q1, Q2, and Q3.


Thursday, July 4, 2013

The Greatest Day Ever For ALS Awareness Was July 4, 2009

http://en.wikipedia.org/wiki/Michael_Goldsmith

Through the work of one wise and driven man with ALS, every major network and every major publication carried an ALS feature.

This website gets a pretty steady amount of traffic.  On that July 4 weekend four years ago the traffic was astronomical.  It came from thousands of people searching for words related to ALS.  Never before, never since has there been anything like it.

"4 ALS" wasn't about fundraising.  It wasn't about an organization.  It was about ALS awareness and it worked.

Michael Goldsmith, we miss you.

Monday, July 1, 2013

If You Could Propose One Idea To Improve Healthcare Delivery...


@phrma asked us on twitter, " If you could propose one idea to help improve health care delivery in the United States, what would it be? - See more at: http://www.phrma.org/phrmapedia/conversations/improve-us-health#sthash.KrzGflZr.dpuf "

If anyone knows where the rough spots are in healthcare delivery, its a person with ALS or a caregiver.
 
This idea is simple. It's incredibly simple. It's low-tech.  Its need reflects a healthcare delivery system that still doesn't get patient-centricity though.

Dear people in the entire healthcare delivery process, I, the patient or caregiver, am a valuable resource for fixing a few things that aren't hard to fix.  Please listen to me.  Really listen.

After waiting for an appointment under the racket of a waiting-room television, I mentioned to the receptionist that it would be nice if there were a quiet area for patients who choose to read or work.  Even my car dealer does that these days so that I can be productive while I wait.  Did that suggestion make it to a staff meeting or a process-improvement team at the doctor's office?  Probably not.  I think a lot of those patient comments are ignored by the front-line folks because the patient can always mention them on the customer satisfaction survey.  Besides, is the receptionist expected to be a waiting-room change-agent?

When I said to the physician, "Gee, the walls are thin in these exam rooms," that was a big clue that I heard things that were none of my business.  What happened to that comment?  Probably nothing.  Yet we spend gazillions on HIPAA forms.

When I as a caregiver mentioned to the doctor that it took me two trips to the BMV to get the handicapped hang tag and if the doctor's office simply kept those forms, it would only have taken one, did anything happen to save the next caregiver a trip?

Healthcare delivery stakeholders, your patients are a gold mine.  Are front-line healthcare deliverers motivated to be part of solutions?  Between it's-not-my-job syndrome and leaving the voice of the customer to the customer satisfaction surveys, we miss the gold mine.  Are patients who make observations and suggestions valued or are they wasting your valuable time?  It's up to you, healthcare delivery.

Saturday, June 29, 2013

We Need More Idea-Thons To Defeat ALS

We're so good at walk-a-thons.  Let's get really good at idea-thons.

The idea struck me during the consensus-building final portion of the Team Gleason ALS Research Summit yesterday. www.teamgleason.org/summit

It's harder to do online than they did at the tables, face-to-face, but I think some clever people could come up with a good online format that should include everyone -- rocket scientists down to low-i.q. bloggers.

This would be like speed-dating for ideas.  Get some sparks of creativity out there.  Capture them.  See if they generate other sparks or plop.  Move on.

In an online, chat-like format:

  • Pick a general topic
  • Have someone float an idea
  • People who want to discuss stay in the chat and start chatting
  • People who don't want to discuss or who don't like the idea stay on the sideline
  • Have at it for fifteen minutes
  • Float another idea...

Eventually the idea bag is full and it's time to deliver (thanks, Dreamfinder and Figment), but in the fight against ALS, we've not been very good at the new ideas. The more ideas you have today, the more ideas you'll have tomorrow.

Yesterday we had sparks of creativity and the discussions were all-inclusive. That's good and that's different.


Wednesday, June 26, 2013

Are We Really Keeping Score?

Every team knows you have to keep score.  Every business knows you have to keep score.

Ask the head of any ALS not-for-profit how last year went.  The answer will typically be financial. "We had a record fundraising year."  "It was a tough economy but we did fine by cutting expenses."   "We are thrilled with $3 million in new government grants."



After the financials, some other statistics may or may not follow.  "We screened 40 therapy candidates in the lab.  Two have 'promise.'"  "Fifty research grants were made."  "We held 4,211 support group meetings helping 12,200 patients." "Twenty-two dead mice eliminated some duds from the research pipeline." "We had ten mentions in national media."  "We helped pass one piece of legislation."  "1,214 new people self-enrolled in the ALS Registry."

Nobody says, "Over 125,000 died again from ALS."

Are we keeping the right numbers on the scoreboard?

Are we demanding the right statistics during the game?

Tuesday, June 25, 2013

Dear Smart ALS Scientists Who Are Meeting In New Orleans This Week

We need a shared, searchable, online source of ALS-related grant information that includes grantors, $, start dates, end dates, receiving institutions, investigators, topics, and outcomes.

Something like this but with more of the pertinent details like amounts and outcomes.  

That would really jazz your donor base.  It would help people searching for hope to have a real place to search.

It's our portfolio, yet nobody tells us exactly what's in it and how it's doing.

p.s. The search tool needs to be shared because we're tired of funding expensive, redundant, incomplete  silos of data like we have bought for clinical trial information.

Saturday, June 22, 2013

Let's Have A Pact

Here is a press release from yesterday --

http://online.wsj.com/article/PR-CO-20130621-906271.html?mod=wsj_share_twitter

The ALS Association is working to change that and has committed more than $67 million to find effective treatments and a cure. 
Wow, is that $67 million last year or over the last 30 years?  Does "committed" mean your direct expenditures?  That's a huge chunk of money.

Here's a past statement from the MDA --
MDA (mda.org) is the world’s leading nongovernmental provider of ALS services and funder of ALS research. Over the years, MDA has led the fight against ALS, investing more than $307 million in its ALS research, services and information programs.
Double-wow, is that $307 million over the last 70 years or the last 7 years?  How much scientific research is buried in that number.

And this is from als.net, the website of ALSTDI, a smaller, all-research organization --
12 M spent on research a year
That's a good wow because that is totally research and an annual number.  But how much of that came from the MDA?  Are we double-counting dollars?

One's impression is that ALS research is tremendously well funded.  The fact is that it's not.  Our ALS organizations, in an attempt to impress potential donors with their commitments, give the world a distorted view of the low research funding levels that have held back the fight against ALS.

So, how about a pact, ALS organizations?  It would be good for all.  Really.

Here's the pact:

Every year use a common format and share with the world --

  • The dollars you spent directly on ALS research in the last calendar year.
  • Any dollars that you received from other ALS 501c3s that were also counted in their statements.


That's it.  Simple.  No glitz. No spin. It gives your donors and the world a normalized view of the good research work you do and the tremendous need for more resources in the fight.  Please enter a pact.  We need a strong handshake and no arm-wrestling.  C'mon.  You can do this.



Thursday, June 20, 2013

Time To Raise The Lid And Smell The Cooking?

[If you've not read the June 15 post, please give it a quick scan for some background first.]

The national ALS registry concept was an easy case to make.  Take a look at www.alscounts.com if you're not familiar with the need and the history.

We fight hard every year for taxpayer dollars that will let the CDC's Agency for Toxic Substances & Disease Registry (ATSDR) develop its ALS Registry well.  You'll not find ALS advocates getting stingy over such an important project.  They go to great personal expense to get ATSDR what ALSA says it needs.

Here's the recipe.

  • ALS advocates get ALSA's legislative priorities in the first quarter of every year.  Those legislative priorities include continuing funding for ATSDR for the ALS Registry.  ALSA presents us with the number.  This year and last, the requested number was $10 million per year.
  • ALSA has an advocacy fly-in during the first quarter of the year for invited advocates and ALSA employees to promote the priorities with legislators.
  • In May all advocates who are able to make the trip and pay the fees and travel expenses are invited to a bigger Advocacy Day to call on legislators.  Many ALSA employees also make the trip at the expense of their donors as part of their jobs.  Some people with ALS may be assisted by their chapters' benefactors or by other not-for-profits to help with travel expenses.  This year, under the guidance of ALSA, we advocates asked legislators for another $10 million for ATSDR to continue the ALS Registry project.  Here's a recent ALSA pitch.  
  • Annually there is an invitation-only meeting of a committee that gives input to ATSDR on the Registry project.  The public is not informed of the meeting in advance.  The minutes of the meeting come out long after the meeting, and the minutes reflect a group very happy with the project.  There is little critical discussion.  Nobody's feet are held to any fires.  Expenses are not reviewed.  You can see for yourself here.  The list of 2012 participants is at the end of the document.  You'll see that ALSA, MDA, and the CDC and ATSDR and its contractors are very well represented.  There is only one ALS patient on the list of whom I'm aware, and he died shortly after the meeting.

That's it.  We sing for ATSDR's supper every year and we work hard so that they have the generous funding that they and ALSA say they need.

I was flabbergasted when I found this the other day at usaspending.gov.

Then when I downloaded the data, I became even more concerned.

Has ALSA been the recipient of large, no-bid, noncompetitive contracts out of the generous funds they had us request for ATSDR?

Was this ever mentioned to us -- the advocates who pitched hard for them for their suggested funding level for the Registry?

Has the ALSA-ATSDR relationship become too cozy?

This at best smells bad.