A Roundtable That Omits Patients Isn't Really Round

A new report on a critical topic, "Opportunities for Improving Therapy Development in ALS: Roundtable Discussion Summary," was published this week.

http://www.alsa.org/news/archive/opportunities-for-improving.html

The lead authors were two prominent ALS scientists and  "The ALS Clinical Trials Working Group."

Digging into the paper itself, we found the membership of "The ALS Clinical Trials Working Group."

The ALS Clinical Trials Working Group

1. Lucie Bruijn, ALS Association;
2. Merit Cudkowicz, Massachusetts General Hospital;
3.  Barbara Bierer, Brigham and Women’s Hospital;
4. Jesse Cedarbaum, Bristol-Myers Squibb;
5. Chris Coffey, University of Iowa;
6. Ronald Farkas, Food and Drug Administration;
7. Daniela Grasso, Massachusetts General Hospital;
8. Petra Kauffman, National Institute of Neurological Disorders and Stroke;
9. Marianne Kearney, Massachusetts General Hospital;
10. Doug Kerr, Biogen Idec;
11. Karl Kieburtz, University of Rochester Medical Center;
12. Stephen Kolb, ALS advocate;
13. Eric Macklin, Massachusetts General Hospital;
14. Timothy Miller, Washington University, St.Louis;
15. Thomas Murray, The Hastings Center;
16. John O’Gorman, Biogen Idec;
17. Patricia O’Rouke, Partners Healthcare;
18. John Ravits, University of California, San Diego;
19. Jeffrey Rothstein, Johns Hopkins University;
20. Jeremy Shefner, SUNY Upstate Medical University;
21. Ira Shoulson, Georgetown University

Unless I'm missing something, it appears that they forgot to include anyone who actually has ALS.

The table wasn't really round, was it?

Let The Sunshine, Let The Sunshine In, The Sunshine In

Now that you're humming a hit from the 1960s...

Yesterday I read a journal article related to ALS clinical trials.  In the department of unintended consequences, the end of the article caught me totally off guard.

One of the authors, a long-time head scientist at an ALS charity, had disclosures to share under "Declarations of Interest," showing consulting (I'm assuming paid consulting) for three pharmaceutical companies.  Those declarations certainly don't include any dates, and they don't tell you whether it was a $25 lunch meeting a decade ago or a $2,500 speaking fee or a $25,000 consulting fee last year.

In recent years there has been sunshine (both voluntary and regulatory) shed on pharmaceutical industry payments to physicians.  Is there any light shining on such payments to the many non-physician medical researchers whose work is funded by our charitable donations?

I think there should be.  That would be easy enough for not-for-profits to do voluntarily and would certainly be pertinent information for donors and people with ALS to know. Everyone would win.

There Was Life Before ALS

A friend once told me that their family seemed to gauge all time relative to her husband's heart attack.  They moved after the heart attack.  A child graduated from high school before the heart attack.  A significant life event can become a "time fulcrum."  An ALS diagnosis certainly becomes such a fulcrum.  Unfortunately, to doctors and researchers, it is more of a "time chasm."

A person diagnosed with ALS starts seeing new doctors, often in a new clinic setting, with fresh new charts and lots of new tests.  Even old primary care physicians are often left for new ones who are more familiar with ALS or are in the new clinic network.  As those old ties are broken, so are the ties to a rich medical history which could well hold clues about ALS.

Worse yet, if a person with ALS is lucky enough to be accepted into a clinical trial, more chasms appear.  There are clues that already exist on both sides of the big chasm that are ignored.  The clinical trial gathers its own information and doesn't look back.

These time-and-information chasms can and should be bridged.  There was life before ALS.  There was a medical history before ALS.  There is information.  ALS is a mysterious disease. Why ignore the clues?

Could We Just Put A Mother In Charge Of Clinical Trials, Please?

At the beginning of every summer vacation, we kids liked to get new stuff for our summer play needs.  A nice fresh baseball, a basketball, ...

If I asked for a new baseball and my brother had already been granted the request and bought one, do you think I got one, too?  Of course not.  We could share.

If he asked for a new basketball and I already had bought one, do you think he got one for himself?  Of course not.  We could share.

Sure, we had our own gloves and bicycles, but some things just made sense to share.

Today on a webinar, I heard of the promise of MRIs in possibly diagnosing ALS.  It's very preliminary.  I searched clinicaltrials.gov for "amyotrophic and MRI."  There were a few recruiting trials involving MRIs, but all were different in what they were doing or why they were using MRIs.

And I thought wouldn't it be smart of all of those MRIs were available for the other researchers to see.

And then had another thought. Wouldn't it be smart if the thousands of people with ALS who have MRIs in their medical records could simply say, "Share this with researchers." 

Today it seems that we buy a lot of baseballs and basketballs that stay in individuals' closets.  They need to be shared.

We need a mother.

Does The "Rare" Word Hold Us Back?

The Perception

Most Americans don't associate themselves with rare things, except perhaps a steak dinner.  We don't have rare coins in the jar of change on the bookcase.  We don't have rare stamps in our desk drawers.  Our dogs aren't rare species.  The vase in the living room is hardly rare.  We dream that the autograph that we collected in fourth grade might someday be rare and valuable, but that never happens.  If you told me that I might have something rare in my home, I would roll my eyes and say, "Dream on."

That "rare" word doesn't project the belief that most of us have that something is possible.  It seems
incredibly unlikely.  It's pretty much impossible to us.

We have better words for those unlikely things that we really believe happen to us -- exceptional, unusual, notable, exclusive, ...  We do believe that unlikely things can and might happen to us.  It's just that we never hear anyone use the word "rare" when they speak of a winning lottery ticket or a particularly talented child.

"Rare" just doesn't impart a sense of possibility to most people.  We need to change that.

The Reality

"Rare" is an important legal designation for diseases, and I understand that. I so wish that they had chosen a different word... and a better definition.  A rare disease is defined as one that "affects fewer than 200,000 Americans."   That definition is normally interpreted as a disease with which there are fewer than 200,000 Americans living at this moment.  It's not a definition that speaks to the likelihood of an American getting a disease.  It does not speak to the possibility of it turning your life upside down.  It speaks to the likelihood of your living with the disease at this moment.

The prevalence-based definition bolsters the perception of rare disease not really being a possibility for most of us.

Think again.  Your odds of getting ALS are high.  Your odds of living with ALS for long are low.  It's a small peer group.  Think about that!

The numbers may fit the definition of a rare disease , but we need make it clear to people that it's not an unlikely disease.  It's not impossible.  It's very possible to affect you and your family.

The upcoming Rare Disease Month should make every American squirm with the real possibilities.

We Are Grateful for the As That Go With Our Qs Regarding Project MinE

During the big ALS symposium in Milano last month, we were engaged in some tweets regarding Project MinE --
 www.projectmine.com

Robbert Jan Stuit @rjstuit of Project MinE was kind enough to respond to some of my tweets and subsequent questions.  His answers are with my questions below.

Thanks for tweeting back this week.

I'm not at all well versed in genetics and have questions (and frankly, concerns based on the uncoordinated information silos we have in the US regarding ALS patient data).

If you do a complete genome sequence on a patient, does that automatically give you everything you need to know when a new ALS gene is discovered? Yes, it does, all information needed is there.

Do you retain tissue for further testing? Yes

Does you work fit together at all with Steve Kolb's C9 project in the US? We do not know about this project.

Will your genetic data stand alone, or will it be possible to add patient background or progression information to each patient's genetic profile? The patient background and progression information is key to have along with the genetic profile.

Do I ask a lot of questions?

I was surprised at the cost.  Something is rattling around in my head that one of the Miami FALS researchers once said that complete genome sequencing could potentially be done on PALS for < 1000 US$.  Perhaps I misunderstood. They price we communicate is currently one of the lowest offered by whole genome sequencing companies as far as we're aware. You might have misunderstood and the other researchers may have referred to 'exome sequencing'...

I feel strongly that we need to have complete, accessible data on each ALS patient including genetic information.  Today we have what amount to scraps of notepaper here and there and everywhere, and nobody can tape Mrs. Smith's information into a cohesive and meaningful resource. We do not collect DNA without all the other information about a patient. We also have several studies ongoing into environmental factors that may cause ALS, see www.als-onderzoek.nl (a Dutch site unfortunately, but Google translate might work...)

Years ago a PALS in the US told me that he felt like he had left tissue all over the country.  Nobody is keeping track in a central place of where the tissue is and what information it has yielded. In Europe we are coordinator of an EU-funded project that focusses on solving exactly this. Many European ALS centers are involved, a website on this project will go live early 2014. The project is called 'SOPHIA'. 

Thanks for any insights.

The Rolodex - Part IV

The patient story is so important to making ALS real to people.

Get ready, world, because there is a lot more to these ALS patient stories than you may have realized.

These are not passive glimpses into their diaries with tidy "The End" pages.

These are not "Queen For A Day" stories of misery designed to raise compassion for a cause or funding.

These are vital narratives of people's pasts that can set us on a course for change.  These are people who even after their deaths put us at the corner of the past and the future with some pretty clear directions for a better path for tomorrow.

We need to follow them.  Yes, they lead if we pay attention to their whole stories including their ideas and the outrageous circumstances they stood up to.  It's up to us to listen to them -- their ideas, their wisdom -- and forge a better path.  To do less is to miss their whole stories.  Get ready, world.

The Rolodex - Part III

The Rolodex of people I have known with ALS has grown to be huge.

It can teach us some lessons about how we need to speak to the correct numbers correctly.

I don't clean out my Rolodex annually to throw away the cards of those who have died nor do I re-use the cards of those who have died.  If I did, it wouldn't really grow.  It wouldn't be representative of the cumulative loss that ALS has caused.  It would not be respectful.

But... when we permit prevalence to be "the gold standard" (a term used by a major ALS organization) for measuring disease impact, that's exactly what we are doing.  We are only counting people living with ALS at this moment.  We are discarding the deceased.  We are kidding everyone, including ourselves, about the numbers and the impact of ALS.

If we reuse those Rolodex cards so that there is just one card per person living with ALS (as the prevalence metric does), we somehow confuse Mom with the six people who have followed on her card or with the dozens who preceded her.  Ditto with Rob's, Barbara's, Pat's, Ben's, and now Scott's cards.  Those are all distinct, beloved human beings with their own families and friends and talents and contributions.  One card should not be shared.  

And cards should never be discarded.  Ever.

It's time that we learned to explain the impact of ALS and why prevalence (the number living with it at this moment) is a red herring.  ALS has a relatively small prevalence, yet a huge incidence (the number of people who get it).  That's a toxic combination.

Just look at a Rolodex of people with ALS that doesn't toss out or recycle the cards.  It grows painfully and quickly -- more quickly than most people perceive.

Let the conversations begin.

The Rolodex - Part II

On Monday Regina Holliday and I corresponded briefly about some logistical details for the Walking Gallery jacket that she was about to paint. In closing I mentioned that I had just received word of the death of yet another of our online ALS advocates.

 Every time I get one of these messages, it's like ALS has sucker-punched us again. It doesn't stop. Over and over they die. Regina graciously asked me if I wanted to include this gentleman in the Rolodex painting that she was still designing. Yes.

Scott Johnson was Grizzly15 on the ALS message boards and @scottcjohnson2 on twitter. Read his tweets. He pushed and prodded for constructive change for vets with ALS and for the ALS Registry. 

Very early on Tuesday morning I woke up to a message from Regina that included the painting.  Number 300 in the Walking Gallery, "The Rolodex,"  was finished.

Wow.  She got it!  There's a lot there.  There's a lot to talk about.

Then yesterday afternoon Regina posted a year-end message in her blog that shed even more light on this remarkable piece --

http://reginaholliday.blogspot.com/2013/12/2013-in-rearview-mirror.html?spref=tw

As I was designing this jacket, Colton (our family friend and 4^th grade pupil) asked what was the thing that I was painting.  I told him it was a Rolodex.  He asked, “What is a Rolodex?”  I smiled at this small digital native and explained before computers we kept our business contacts, friends and family’s information in these handy devices. 

As I explained the concept of a Rolodex, I thought of the many years that I would spend the quiet days between Christmas and New Year’ cleaning out old cards and making room for new ones.  I would pull card after card of sales reps who had moved away, friends whose Christmas cards came back “return to sender” and the cards of friends and family who had died. 

I looked at Colton and explained everyone in the Rolodex I was painting died as result of ALS.  He asked me what was ALS.  I explained it was a disease that made it hard to use your muscles.   First walking would be hard to do, then using your hands and then talking.   As the disease continued a patient would talk using their eyes, then finally they would not be able to breathe.  They would descend into the complete silence in the end.

Colton said, “That sounds like a hard way to die.”  I told him we could do something special for these people we lost.  We could tell their story.

I told Colton, “After Fred died I Googled him.  He had only two hits.  One was from the obituary in the paper and one was from American University where he worked.  Then I spent the last 4 years on medical advocacy and speaking about Fred.  Would you like to see how many hits Fred has now?”

Colton nodded yes and his eyes grew big as he looked at the result: Frederick Holliday II PhD had over 6 million hits. 

Online the names from a Rolodex live on.  We get to meet Cathy’s friends and family; Barbara Brenner, Betty Collet, Pat Dwyer, Ben Harris, Scott Curtis Johnson and Rob Tison.  We see them leave the dusty card and walk into an eternity of advocacy. 

That is my 2013 in the review mirror.  The things we did, the people we met will create ripples in the years to come.  We will never really finish this time in our lives nor truly say goodbye to those we love; this time continues in our hearts and within our digital lives.

Let the conversations begin.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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.

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. 

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.  

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?

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.

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.

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.