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.

Saturday, December 3, 2016

For the "Ask the Experts" Session Prior To the 27th International ALS MND Symposium

1. At my last count, there are at least a half dozen completely separate "precision medicine" projects for people with ALS.
     a. Has anyone actually established protocols and shared data between any of these silos?
     b. Is anyone concerned about the duplication of resources and infrastructure?

2. Are there any specific novel clinical trial design features that you expect to see implemented in the next 12 months as a result of the Airlie House guidelines or the ALSA FDA draft guidance work?

Thank you.

__________________________

Information about "Ask the Experts" can be found at
http://www.mndassociation.org/research/international-symposium/symposium-live-2016/ask-the-experts-session/

We are grateful for the live stream this year and hope that more of the symposium activities will be live streamed in the future for those who are interested in the newest and best science but cannot travel.



Thursday, December 1, 2016

Stop, Look, Listen!


After a lively day of sausage-making, a 21st Century Cures Act passed the House yesterday and now it's the Senate's turn to make some things happen. I think that this can provide some good opportunities to advance the fight against ALS.  Increased NIH funding, FDA resources, and the Precision Medicine Initiative are all very important in my opinion.

But one item gave me pause when I saw the list of ingredients left in the House version:


Sure, a neurological disease surveillance system makes ultimate sense when there are so many devastating and unsolved neurological diseases.  But wait... "a neurological disease surveillance system coordinated by the CDC..." Been there, done that, and it has not gone well.

Please, stop, look, and listen to us.  We have an opportunity to do something great or to be part of a train wreck.

Today we have a tremendously expensive ALS Registry -- a neurological disease surveillance system coordinated by the CDC.  It has failed to live up to its potential, and it has cost us taxpayers over $70 million to date.  It has let us down on many levels.  It has generated scant reports whose numbers nobody wants to  use.  It has been fraught with scope creep and gingerbread of questionable value and unrelated to delivering accurate and timely disease surveillance.  And perhaps the most revealing problem of all has been its lack of transparency.  This year the invitation-only annual meeting was blacked out and we have not see a  transcript yet.  The CDC doesn't not seem to want to talk about it except to the voluntary health agencies that lobby annually for its funding (and in a cozy arrangement receive hundreds of thousands of dollars in no-bid contracts).

So please, stop, look, and listen to us.  Let's fix the CDC ALS Registry problem and move forward with something well-designed, nimble, transparent, and focused to provide valuable information on all neurological diseases.  Please learn from our journey and don't be part of a 21st Century train wreck.

Wednesday, November 23, 2016

We The People Get The Message

We simply don't accept it.

This year's CDC ALS Registry annual meeting was August 3-4, 2016.  It was blacked out.  No webstream was provided this year.

Here is some background:
http://als-advocacy.blogspot.com/2016/07/transparency-is-friend-of-good-public.html

Over three months later, we still have no public transcript, no minutes, no recording, no report from this meeting.

The project has been expensive.  We think that the cumulative total of our investment as taxpayers is close to $80 million.

The project results have been sparse and questionable.

Participants at the annual meeting are handpicked by those being compensated for work on the project.

The chief lobbying agency for the project, the ALS Association, is also a well-paid contractor on the project.  In a convenient cycle of silence, they are approaching time to have another $10 million etched into next year's appropriations without any discussion with the hundreds of advocates they like to send to Capitol Hill with their requests.

Our questions go answered.  We are kept in the dark.  We're tired of this.  The project and money are too important to be on a $10 million annual autopay without accountability and transparency and substantial results.

We the people do not accept this.


Thursday, November 10, 2016

The Principal Sets The Tone For The School

I learned a big lesson as a child.  It has served me well in life.

I'm a Boomer. Our grade school was in an area that experienced astronomical growth of families and homes.  Our classes were large and the students and families were serious about education in spite of diverse challenges.

We were blessed with excellent principals who were all business about providing quality life and
academic educations... except for a couple of years.  She was a well-intentioned but not very good principal.  The new policies were nutty.  Changes were ill-conceived and short-sighted.  We could have unraveled as a school.  We didn't.

It took strong, engaged parents who picked their battles carefully and taught their kids that the principal isn't always right, but she's the principal.

It took strong, competent teachers who cared more about teaching than administrivia.

It took students who could see what was going on but not let a principal stand between them and a good education.

Our parents addressed concerns quietly and directly and effectively.

It only lasted a couple of years. We survived.  It was a good life lesson for me.

I have since seen schools and organizations and businesses where the tone set at the top has not been good.  It takes a lot of work in the trenches to overcome that and not unravel completely.  We can't keep our eyes off of what's important... not for a second.


Thursday, November 3, 2016

Phone a Friend for a Lifeline



If you are not familiar with edaravone (Radicut or Radicava), here is some important background:

http://www.alzforum.org/news/conference-coverage/does-free-radical-scavenger-edavarone-slow-als

MT Pharma recently filed a New Drug Application (NDA) with the US FDA, asking for a "priority review."  That priority review would have been the FDA's commitment to review the application in six months rather than ten.  For some reason, the FDA declined the priority request and now we can't expect its decision until June, 2017.  That's four extra months for people with a disease that kills 500 Americans every month.

https://alsadotorg.wordpress.com/2016/09/13/fda-accepts-new-drug-application-for-potential-als-treatment-edaravone/

So now we wait while people die.  Or maybe we don't.

Normally I'm not a fan of Expanded Access Programs (EAPs) that the FDA allows for drugs that are not yet approved.  They can be terribly unattractive for drug developers, especially if their entire market for a brand new drug is an unmet-need disease like ALS.  They can be secretive programs where data are lost.  The right solution is to get drugs to market approval faster (where everybody has access and payers are paying),

Maybe in this case we shouldn't let the perfect accelerated approval concept get in the way of some possible benefits for the 4000 Americans with ALS who will die before the FDA review is completed and for the 4000 Americans who are about to be diagnosed with ALS in that same period.

Why is this case different?

  • We believe that MT Pharma America may be amenable to doing some kind of Expanded Access Program in the United States.
  • The product has already been approved and marketed for ALS in Japan since 2015.
  • The product had been approved and marketed for stroke in Japan since 2001.
  • The product has a good safety profile so far.
  • If people with ALS who might participate in an EAP would voluntarily self-report data, perhaps at a place like patientslikeme.com, all might learn more about the drug and potentially which subgroups of people with ALS it may help.
There has already been talk of medical tourism for people with ALS who have the substantial means to go to Japan.  There have already been shadowy internet schemes suggested to buy the product in other countries.  Neither of these paths of desperation is the right answer for Americans with ALS.  A A transparent and legitimate EAP would make so much more sense.

An EAP wouldn't be easy. This is an infusion product and the process won't be cheap.  But ALS isn't easy, and it is anything but cheap.

If you have any interest at all in seeing MT Pharma America provide an Expanded Access Program for edaravone, they need to hear from you.  If nobody asks, they will have no reason to go down that path.  Here is the contact information: 1-888-292-0058 (typo in phone number corrected Nov. 3, 2:30pm EDT... I apologize for the error) or email information@mt-pharma-us.com 



Some grass-roots ALS advocates started calling yesterday.  MT Pharma America has contractors fielding phone calls.  They will ask your name, phone number, email address for followup, and relationship to one with ALS.  Please take a few minutes and call or email them to let them know the importance that providing early access through an EAP could mean to the thousands of people with ALS who have no great options today.  And thank them for their consideration.

It's worth asking.  We certainly can't demand it.  This case may be different enough that an EAP might be possible for the drug developer to offer, could give people with ALS an option, and might even advance some scientific understanding more quickly.

Please consider and call today. This could be a viable lifeline for people with ALS.







Tuesday, October 18, 2016

Actions >> Words

Yesterday I was involved in an interesting twitter exchange on the difference between the words we hear in the fights against diseases and the actions actually delivered.

And an apt example had played out on twitter just a few minutes earlier yesterday.

We got word from an ALS not-for-profit that they are trying to gather 1000 voice samples quickly for a research project.  The voices both of people with ALS and of healthy volunteers are needed. I did my own recording and found the process to be very simple, fast, and unobtrusive.  This seemed like a good job for social media.  1000 recordings should be a piece of cake, especially since some organizations have a wonderful social-media reach.   I posted the link and some requests for people and organizations to retweet.

Not one ALS organization retweeted.  Not one.  Individuals did, but not one ALS organization.  No action.

Here is one of the words that bothers me when I see this kind of pointless apathy and inaction -- "collaboration."  The use of that word has been pretty constant since the windfall of the ice of 2014.  There have been tens of thousands of dollars spent on talking about "collaboration."  Meetings and talk and hollow words don't solve problems.  Simple actions do.


When it came to actually doing something simple yesterday to fill one organizations's ALS study with quick voice samples, "collaborate" was just a word.  It didn't generate any action.  It meant nothing.

Talk's cheap.  Show us what you do.

Monday, October 10, 2016

A Study in Customer Experience in Clinical Trial Inquiry in the United States

Following is a link to the full paper written as a part of my epatient participation in the Precision Medicine track at
Stanford's Medicine X 2016:

"A Study in Customer Experience in Clinical Trial Inquiry in the United States"

A brief summary was also given in an Ignite! presentation at Medicine X, and results will be presented in a poster session at the 27th International Symposium on ALS/MND in December.

Many thanks to all with ALS and their loved ones who have ever sought to participate in clinical trials.  I hope that the information in this study will make an easier path in the future.

Monday, October 3, 2016

If You Can Only Go To One Conference, Make it Medicine X

That's advice that a respected friend gave me three years ago.  Boy, was he ever right.

This was my third experience at Stanford University's Medicine X  http://medicinex.stanford.edu  .  It was my second as an e-patient.  I thought after last year's experience that this year might be a letdown.  Boy, was I ever wrong.

Pre-Conference 1 -- "Providers are from Mars, Patients are from Venus: A workshop to improve patient-provider communication."
Faculty and staff of the Stanford Medical School facilitated a workshop that opened eyes to challenges and pitfalls of difficult provider-patient communications today.  Actors who work in the medical school gave excellent portrayals of the challenges.  I left with hope that healthcare delivery will improve, and I appreciate the insights into the training and challenges of the physician.  Thanks to Boehringer Ingleheim for this workshop.

Pre-Conference 2 -- "Shared Decision Making"
Eli Lilly and Company decision science specialists facilitated a workshop centered around a painfully difficult decision that a young couple faced regarding genetic testing and Alzheimer's disease.  The process of sorting out the elements of decisions was enlightening.  Thanks, Lilly.

And I was certainly glad to learn the California Duck Protocol.

E-patient Dinner
The night before the official start of Medicine X, the e-patient delegates are invited to a group dinner where we met a talented and diverse group of movers and shakers, many of whom are dealing with huge health challenges.  It's a special group and I'm grateful to have been included.

Medicine X Day 1

The speakers who work in our government were such refreshing highlights -- truly people who are able and willing to break the bureaucratic ruts that hold us back.  Claudia Williams, LaVerne Council, Susannah Fox -- the sharpest tacks in the healthcare toolkit as far as I'm concerned.

And then Eric and Terri Amador came on stage to teach all of healthcare through their ALS experience.  And Day 1 was good, so good.

Medicine X Day 2

As distracted as I was over my afternoon Ignite! presentation, it was again a productive day.  The
people you meet, the conversations you have, the presentations you didn't expect to attend are all some of the best things about Medicine X.

My Ignite! talk went fine, and I had questions about the clinical trial secret shopper project afterward, and I can't thank Louise Schaper enough for making a challenging 5 minutes fun and bringing out the best in every presenter.

It was a football Saturday at Stanford, and as I walked back to my hotel after a very long day, the campus quiet was almost eerie as tens of thousands of people were consumed with football in a far corner of the campus.

Medicine X Day 3

Just when I thought it couldn't get better, it did.  I attended the Million Veteran Project presentation in
the PMI tent.  They have half a million veterans' genetic typing completed and histories completed already.  They are looking for things to do with this rich set of data.  


They are looking at a long list of diseases that are often connected with those who have served in the military -- heart disease, PTSD,... Have they looked at ALS, I asked?  They did not know that there is double the incidence of ALS in US military veterans than in the general population.  They do now, and it is on their radar.  Think of the clues that all of the genomic data may hold related to the ALS-military mystery!

Thank YOU, MVP.

Then I went to a session on the patient-innovation.com project in Portugal.  For years we have been wanting a resource where the McGyver hacks that people invent to outsmart ALS can be archived and referenced.  Is this it?  I think it may be.



Finally I attended a timely session presented by Joe Gulfo on the FDA we need in the 21st Century, certainly a timely topic for me.  We do need a strong regulatory body and Joe's insights into its history and weaknesses today were interesting.   Ironically this was the day before the FDA Sarepta announcement.  And I am better armed today to march forward with ideas to shorten the path to approval for experimental ALS treatments.

Thanks very much to Dr. Larry Chu and Sarah Kucharski and Zoe and all who made this a valuable and energizing conference for me again.

Monday, September 26, 2016

Feed the Monster

I've been going to (yes, actually going to) Walks to Defeat (nee D'Feet) ALS for 15 years.

Something has bothered me for 12 years.

ALS leaves a large, terrible wake of loved ones behind every time someone dies from the disease, and the deaths are constant.  Why doesn't the attendance at walks grow at a constant pace every year to reflect that wake?  Why isn't the crowd that raises awareness and makes noise in communities huge by now?  It seems like the size of the crowd remains pretty flat year after year.  It's large, and that's wonderful, but where is everyone with loved ones left behind who used to come to the walk three or four or five or more years ago?

Every not-for-profit needs to figure out a revenue machine that will keep it sustainable.  I get that.  The ALS revenue machine is almost too easy.  There are thousands of newly diagnosed people with ALS every year and their families and friends and coworkers and neighbors want to help.  Enter the multi-level marketing of the walk revenue machine, and those who want to help can donate, and they do.  The constant stream of new diagnoses supplies many new donors and old ones fade away a few years after the funerals.

And getting people to actually show up for the walk itself isn't even a priority for some chapters.  It's all about the revenue machine, and the relentless disease is the fuel that feeds it.

Some people think I'm crazy for going back year after year after year.  I'm not.  If more people did that, we would have more people who see the problem of feeding the monster without changing the fight.  You see, the talk of achievements is recycled year after year.  It doesn't take Einstein to see what has happened.








Saturday, September 17, 2016

Meet Elizabeth Cosby




Today I have the privilege of delivering an Ignite! talk at Stanford University's MedicineX.  I appreciate the opportunity to present a message about ALS and clinical research to so many outstanding people whose mission is to rebel and improve medicine.  Thank you, #medx .

The full paper on this project will be published on this blog before October 1.

The project results will also be presented in a poster presentation at the ALS MND Symposium in December.

---------------------------------------------------------

The day Mom was diagnosed with ALS, it hit us from out of the blue. There is one thing I remember vividly about that day.
The neurologist pulled me aside and said, ”Get her to someone doing clinical trials because the only thing the least bit “promising” would be experimental.
We were fortunate.
We had an action plan on day 1.

You see, the ice didn’t cure ALS.
There is no effective treatment.
It’s a difficult disease.

That’s EXACTLY why clinical trials are so important to those with ALS.
1. They are a legit Hail Mary
2. They advance the science for others.
You who do clinical research RECRUIT for trials. 

But wait.  You are RECRUITING.  They are SHOPPING.
People looking for a clinical trial are doing what we all do every day when we look for a new car or a great, affordable restaurant in Palo Alto.
They do not seek to be recruited.
They seek the best trials for them.
Businesses use Customer Journey Maps to show the complexity their customers experience to do business with them.
Complexity is never good in customer service. They journey should be easy for the customer.
There are some basic elements of most Customer Journey Maps –
Awareness, Research, Purchase, OOBE (WOW) Disney Apple.
Lets back up to Awareness.  A lot of money is spent on making people aware of clinical trials. Great.
Based on personal experience, I suspect that we are losing people who are already aware of clinical trials in the Research part of their journey, and the problem is poor customer service.
Am I all wet? How could I put some data behind my suspicion?  Meet our secret shopper, Elizabeth Cosby.
Elizabeth is a fictitious woman who came to life a few months ago in a new gmail account.  Elizabeth has a fictitious aunt who was recently diagnosed with ALS. 
Elizabeth tried to help her find a clinical trial.
The first part of the journey was to find all recruiting, interventional ALS trials.
Elizabeth wanted to organize the information for comparison shopping.
That was terribly time-consuming.
Finally on the evening of April 4, Elizabeth emailed inquiries about 39 trials.
There were 5 simple questions in each email – things like is it still enrolling, time commitment, reimbursement…
The first response came back that very evening.  It answered all the questions and was encouraging. Was I all wet about poor customer service?
Unfortunately time told the tale.

Of the 39 trials, Elizabeth got absolutely no response (not even an autoresponder) regarding 11 of them.  Crickets.

39 clinical trials.
28 responses.
11 crickets.
From the 28 responses we a variety of data on the five questions.
Please read my paper at www.alsadvocacy.com for the full scoop.

This project was as much about the journey as the five questions.
Elizabeth assigned a completeness score to each response.
If you answered all 5 questions, you got a 5.
If you punted or simply referred Elizabeth to a consent document, you got 0.
Average completeness score was 3.  On average, Elizabeth had 3 of her 5 questions answered.
Elizabeth assigned a tone score to the responses (respectful, collaborative, encouraging).  Sure, it’s subjective, but that doesn’t mean it’s not important.
Average 4
It really got interesting when we added the scores together for each response.
13 of the 28 responses got perfect scores of 10.
What we saw was that
when they were good, they were very, very good, and when they were bad, they were horrid.

Good customer service. 
It’s not rocket science.  It’s not neuroscience.  It’s common sense. 
If we fix some easy customer service problems that Elizabeth uncovered, the clinical trial path will be faster and easier.  Everybody will win!

Wednesday, August 10, 2016

The Scales Tell More Than Your Weight

They tell a lot about poor healthcare delivery.

Story I.

Mom was around 5'6" tall and was always fit.  She lost a lot of weight because of bulbar-onset ALS.  We called it her Gandhi weight.  She was so very thin.  At a neurology appointment the helper took her to the scales on the way to the exam room.  She wrote something secretively on the chart.  I asked her how much Mom weighed.  She said 168.  My reaction -- "Oh, come on."  She said, "What's wrong with that?"  I said, "Look at her."  The helper was not happy, but we got another weigh-in at about 50 pounds less.  Had I not asked, Mom's medical record would have been dangerously wrong.

Story II.

I had a weigh-in last winter at a busy doctor's office.  I had my backpack and coat with me, neither of which I wanted to include with my body weight.  The helper said, "Step on the scales."  I looked around for a place to put my stuff.  She offered no option except the floor.  I said, "You really need a hook next to the scales."  Silence.  I said, "You know they sell them at Home Depot."  She snapped back, "I  can't do that."  Next year I think I'll take one that sticks on the wall because I didn't detect any initiative on the healthcare worker's part to fix a problem.

Story III.

This isn't my story, but it's a must-read.  And the story isn't really about the scales, but in a way it is.

http://www.speed4sarah.com/clinic-visit/

When I read Sarah's 65-pound story, I thought of Mom's Gandhi weight.  Then I thought of our vet's office.  It's pretty basic.  It's not a fancy place.  They have a scale at floor level with a huge metal plate that the big dog walks onto.  It's simple.  I'm thinking that kind of scale would work for a wheelchair.  Drive it on, read the weight, subtract the weight of the wheelchair.  Voila.  Why would an ALS clinic not have a scale like West 56th Street Veterinary Hospital's?

I know it's hard to fix healthcare, but please, it's not that hard to get the weigh-in right.





Thursday, July 28, 2016

It's A Gene

Google may have brought you here.  You may have been reading about an exciting or promising or breakthrough new discovery that you funded with your ice bucket challenge donations.  Here are some fast facts:

It's a gene.

It's important.

It's not a treatment.

It's one small piece in a gigantic puzzle that is literally missing too many pieces.

Ice bucket money and money from many other sources helped find this NEK1 gene that affects a small percentage of people with ALS..

There are lots of other identified genes and more are discovered all the time.

New knowledge is good.

It's not a treatment for anyone's grandparent or child or niece or neighbor.  It's not even close.

It's cruel when organizations with an eye on fundraising pitch a feel-good story to major news outlets that inevitably put the magic words "breakthrough" or "exciting" or "promising" in the text. Grandchildren or parents or aunts or uncles or neighbors read those and unrealistic hope erupts.  It's cruel.











Saturday, July 23, 2016

The Drips Don't Add Up

Yesterday we received a promotional infographic -- http://www.alsa.org/fight-als/edau/ibc-progress-infographic.html for "Every Drop Adds Up" (a registered trademark of the ALS Association).


One drip caught my eye --



Whoa. The CDC's ALS Registry had nothing to do with the ice bucket challenge.  It had been going on for years and is completely funded by taxpayers.  We advocates are sent by the ALS Association to Capitol Hill every May to request those increased and generous funds.  And in 2014 and 2015, the ALS Association itself actually got $659,451 from the CDC to promote the Registry per usaspending.gov.  That would have happened regardless of the ice.  And the total price tag on this "largest" project is well over $70,000,000 in taxpayer funds.

We got the first report on national ALS prevalence from this "largest" project in 2014, the year of the ice, and it identified just 12,187 people living with ALS in the entire country.  It asserted prevalence based on that number.  The ALS Association concurred with the report and its assumptions and has called the CDC's Registry a resounding success.  http://www.alsa.org/news/media/press-releases/atsdr-reports-first-data-set.html

Hold that thought.

Our information-seeking eyes moved to another drip --



The ALS Association (which serves only a subset of the total people with ALS in the United States) has personal contact with 15,000 people with ALS annually?

Whoops.  These drips certainly don't add up.

This makes the CDC's 12,187 publication look absurd.

This makes the CDC's well-paid contractor look ineffective at moving the 15,000 people it serves annually to self-enroll in the Registry.

This makes the ALS Association look complicit in promoting a project and its published data as successful and valid when it knows otherwise.

The parting drip --


It's past time to turn over some stones.



Sunday, July 10, 2016

I Dream

Sometimes when I want to write about something, I look through stock photos for the perfect picture for the blog.  Today I searched "dream" images.  There were hundreds about dreaming big, about reaching for the stars, about "if you can dream it, you can do it."

My dream is incredibly small.  It's not reaching for the stars.  It's about simple business 101.  It's about good project management.  It's what millions of workers do every day. It shouldn't even be a dream. It should be reality.  It's about a responsible, productive annual project oversight meeting.

There is no image for a dream this small.

I simply dream that --
  • Every person invited to this year's annual meeting for the CDC's ALS registry on August 3-4 will have read the meeting reports from the past decade's meetings in chronological order.  The annual meeting does not need to spend hours in basic orientation every year for those appointed to be the project's Advisory Committee.  Historically the meetings ramble and have led us to disappointing results despite all the back-patting.  This is serious business.  Be prepared.
  • Every person at the annual meeting will clearly state who invited him or her and what business relationships he or she has with other advisory meeting participants.
  • The CDC will present a detailed report of annual project expenses for the past three years and the budget status for the current year.  The cumulative expense also needs to be made public to the advisors.  Details, please (not broad category pie charts).
  • Project contractors will come prepared with meaningful metrics.  Good advisors make people sweat to present meaningful project metrics and not fluff at status meetings. Numbers of emails sent mean nothing.  Measure results.  I dream of sweat. 
  • Participants will speak candidly about registry completeness and design flaws.  Advisors will ask lots of questions and not let them go unanswered.
  • Advisors will challenge the status quo.  They will not be enablers.  Advisors make the same demands of this project that they would in their everyday business dealings.
  • All American citizens will be able to watch a new kind of oversight unfold live.
So I dream.  This should not be a dream.  This is business 101.  This is basic good governance.




Friday, July 8, 2016

Transparency Is The Friend of Good Public Stewardship


The ALS Registry Act, which many of us worked hard to have passed in 2008, allowed for the formation of an "Advisory Committee" which could exert a lot of influence on the direction of this huge government project.

In the past there has been an annual, invitation-only meeting of people who seem to serve the role of such an Advisory Committee. The selection of attendees is tightly controlled by the CDC and the ALS Association and the Muscular Dystrophy Association (the latter two are paid contractors on the project).

We receive the written reports from those meetings many months after they occur.

And to this day, a lot of very reasonable people find the deliverables from this project to be of questionable value and effectiveness relative to the price tag.  That makes the annual meetings very important.
  • In 2013 we really challenged the veil of secrecy around the annual meeting.  We could not find out attendee names in advance.  We could not view proceedings live.  
  • By 2014 the CDC decided to webcast the proceedings after a lot of prodding in social media.  This was a huge step in the direction of transparency where we could view the actual conversations and group dynamics and competencies live. There was no mechanism for us to submit questions, but we were happy with the positive baby step toward transparency.
  • One day of the 2015 meeting was also webcast and again, some sunshine was cast on the questions that were (and were not) asked and the answers that were (and were not) given.  The second day was blacked out for some reason.
After repeated inquiries about the 2016 meeting dates and webcast information, we got the following message yesterday from ALSsystemadmin@cdc.gov :
The dates for ATSDR’s Annual ALS Surveillance Meeting are August 3 – 4, 2016. The meeting will not be live streamed this year. The meeting will be recorded and posted on the National ALS Registry website at a later date following the meeting. The posting of the recorded meeting on the National ALS Registry website will be announced on the Registry website home page and through CDC social media channels.
This is troubling.  Now the CDC doesn't want us watching live.  Why?  Live proceedings can provide energy and conversation that can be quite valuable.  Why stifle that?

My response to the email was
The lack of a webstream of the annual meeting this year is very disappointing and a step backward in transparency.  I hope that those who made that decision will reconsider in the spirit of transparency and good public stewardship.
Thanks very much.
Please take a moment and send your thoughts to ALSsystemadmin@cdc.gov today. It's important. Thank you.



Sunday, June 26, 2016

ALS Does Affect Memories

And we don't do enough to fix that very fixable problem.  Last week we saw an exquisite example.

People who are perfectly healthy today can be slapped with an ALS diagnosis at any time.  Welcome to the rookie camp you never wanted to join. Wham. It's a fast-moving nightmare and families scramble to become knowledgeable and find some ways to fight and cope.  There is so much to learn and the downhill train is going fast.  And it's often over in a few months.

The ALS Association had been promoting its June 21 "Virtual Listening Tour" for weeks.  That's a nice concept -- for people in the ALS trenches to be heard.  Many of us preregistered as instructed.  A few hours before it was to start, I received an email that it was only for people with ALS and "current" caregivers.  I did not join as a result.  Later I got the link to the recording that I requested.  And as I listened to the listening, the memory problem became so apparent.

There was a chippy exchange between ALSA officials and a young woman with ALS over the investment of the ALS Ice Bucket Challenge millions.  The dying young woman spoke to urgency and boldness.  ALSA representatives rigidly defended a deliberate, long-term approach to its windfall.  The gap in perspective was huge (and I hope that there actually was "listening" amidst the defensive dialogue).

So what does this have to do with memories?

It has everything to do with institutional memories and public access to pertinent information.  You see, people in the throes of ALS don't have the benefit of knowing a lot of ALS organizational and scientific history.  Most everyone is a relative rookie and extremely busy.

Pertinent history and memories are continually lost in haystacks that are impossible for those rookies to search.  There is no orderly place to go look for past scientific investments and outcomes.  You constantly reach for information and get press releases and announcements.  You peruse the obvious from the nearest haystack and simply don't have time to dig deeper.  That's a terrible problem.

People dealing with ALS need orderly access to research investment amounts, dates, recipients, project descriptions, and outcomes.  Come to think of it, scientists and investors and donors and the organizations themselves could benefit from that history, too.

How are we to understand exactly where ALS Ice Bucket Challenge dollars have been spent or committed and projects' progress?  We have uplifting press releases and tax returns and financial statements and PubMed searches, none of which tell the story clearly.  If you have a rock-solid story to tell, please tell it transparently and continuously!  

Neuroscience research is risky, but we should learn from every dime that was spent on ALS research 20 or 10 or 5 or 2 years ago or 2 weeks ago.  We need searchable, accessible documentation to leverage the disappointments and the duds.  We get nothing.  I can't tell you how many times those of us with longer memories who hang around the fight wonder what ever happened to various "promising" or "exciting" research from the past.  Information is a precious resource whether or not it is uplifting to review.

It's quite possible that no matter how slowly or quickly ALSA takes to spend the ice fortune that we won't get a successful therapy, but we had darned well better learn more than we get today from a very staged haystack.

And, oh my, dates and outcomes will shine light on those urgency gaps, too.

Every year since I can remember, I've heard how this is the most exciting time for ALS research.  When I was a "current" caregiver 20 years ago, that made me feel good.  Now that I have more memory, it makes me realize what a poor job we do at learning from the past in order to work smarter and faster for the future.

Is anyone willing to fix a very fixable memory problem?






Wednesday, June 22, 2016

Listening 1,2,3

Chapter 1

Nice.  It's always helpful to feel that you are being heard, and I always learn a lot from hearing the perspectives of others.  I registered.  I blocked time on my calendar.  I prepared some thoughts.

Chapter 2
A couple of hours before the call was to begin...

Well, bummer.  Caregivers who have lost loved ones to ALS have a lot of interesting perspective and experience to offer.

Chapter 3
I asked if I could submit comments for ALSA executives and trustees "listening" and was given this email address.

I submitted the following:

Below are two comments that I would like to have made on yesterday afternoon's Listening Tour call.  I hope that this perspective will be helpful to the executives and trustees who are listening. Thank you.
_____________________

1. ALS Registry

The CDC’s ALS Registry has been a disappointment on many levels – from process to oversight to deliverables.  ALSA controls most of the oversight by being lobbyist-in-chief for the project funding and by influencing so many of the appointments to the annual meeting group. 
Please make a serious, tough, businesslike review of this terribly expensive project. 
It has few of the characteristics of a smart, effective disease registry as cited by a recent report from FasterCures.  Completeness is certainly an issue that clouds any ATSDR reporting from the registry.  The project has a classic case of scope creep and distracting gingerbread.  Delays are rampant (we’re still waiting for the second annual report due last summer) and they are sloughed off with “it’s the government” excuses.  Appropriations are never substantiated to advocates with budget details before we're sent to Capitol Hill to ask for yet another $10 mil.  Oversight is hardly independent, especially given the financial relationships among those at the annual meeting.

It’s easy for charities to develop blind spots.  Has ALSA simply become an enabler on a most expensive project that has failed to live up to what a registry needs to be in 2016?  Please take a look at this report as a comparison to what we get -- https://www.cff.org/2014-Annual-Data-Report.pdf

We need some accountability at all levels in this project.  $70 million+ is an obscene amount of money to have plowed into  something that has not delivered what we need in an ALS registry.  

2. ALSA Inspire "Community"

I don't understand why you have allowed the Inspire ALSA “support group and discussion community” (formerly known as the “virtual advocacy community”) to languish in the manner it has.
People needing support and answers stumble across that site, they see the ALSA logo, and they expect trustworthy help.  Instead they often get terrible or no advice and there is no active contribution from ALSA.  That’s misleading for people with ALS and caregivers, and they sure don’t need misleading. 
Putting up a “support group and discussion community” website and simply slapping your logo on it and stopping by every few weeks to censor inappropriate postings isn’t right.  Please just pull the plug on it.

 I hope that we receive the link to the transcript soon.  I would like to hear or read what others had to say.