We Need A Simple Standard

Lest we lose our way in a fog of "partnerships," let this be the standard by which all ALS organizations provide transparency, clarity, and a celebration of generosity --

ALS organizations will publicly and promptly reveal exact donation amounts from any benefactor with an interest in selling goods or services to people with ALS.

It's simple.  Why not?  Really, why not?

Please Join Some New ALS Advocacy Grass Roots

Do you have ideas for better government policies related to ALS?

Would you find it energizing to engage in substantive discussions with other advocates?

Do you want to speak to your legislators from YOUR heart about priorities that YOU think are most important?

Here is a new gathering for you.

www.MoreThanOurStories.org

This is truly grassroots. It's about YOU and YOUR ideas and YOUR personal calls to action.

Please join the conversations on February 13-14.

Everyone is welcome.  Everyone is included.

My Random Walk Through #ALSSYMP

This was my fourth International Symposium on ALS MND.

After a couple of weeks, a few things have stood out to me to distinguish this one... and a few other things that remind me of the same old same old that needs to change.

The Symposium itself has some preliminary undercards to get us warmed up for the three days of hard-core science.

Ask the _______

They call it "Ask the Experts.  That name needs to change.  Actually, the event format itself could use some change.  Last year in Dublin the three neuroscientists on the panel gave crisp, timely, short presentations followed by time for Q and A.  This year the format reverted to that of my first two experiences where the neuroscientists repurposed longer presentations and talked too long, questions that were pre-submitted via email were never addressed, and the session finished with unasked questions still in the room.  It happens on a day that is convenient for the meeting schedule, but most inconvenient for people with ALS and caregivers who want to attend the three days of scientific symposium, too.

One of my questions made it to the panel -- What would it take to get fairly inexpensive potential biomarkers like serum creatinine or retina thickness (that have been suggested for years) to a definitive "yes it is or no it isn't" decision?  The answer was very vague.  Again, later in the scientific symposium, creatinine came up, Vitamin D came up, TSH came up.  These are not expensive things to test, but nobody is stepping up to come up with a definitive answer.

ENCALS Meeting

I appreciate that this meeting of European researchers the afternoon before the start of the scientific symposium was open for anyone to register and there was no charge.

It was enlightening.  One presentation from Dr. Tang took old Rilultek data and mapped it to the Kings ALS Staging.  Contrary to my intuition, the responders were in the Stage IV group (most advanced).  That seems to me to be a big deal as people with ALS choose among a very sparse set of treatment options.

ALS Patient Fellows Dinner

This year a program was started thanks to the help of ALSTDI and PatientsLikeMe and some motivated individuals to offer fellowships to the scientific symposium to people with ALS and caregivers.  They submitted applications showing their willingness and ability to participate in the science and move some patient voice closer to the scientists actually working in labs and clinics.  The patient fellows were fabulous -- smart, informed, insightful, challenging.  Our dinner was a highlight as we got acquainted in person and talked about the symposium and ALS in general.  Special thanks to Rob Goldstein and Paul Wicks.  I think that their later interactions and questions at the symposium proved the value of reducing barriers (such as the substantial fee for patients and caregivers) to have them be part of the fabric of the symposium.

28th International Symposium on ALS MND

The abstracts are all available online for those who want to dig in.  https://www.mndassociation.org/symposium/programme/abstracts-online/

The keynote from Dr. Rosenfeld was a refreshing dose of cold water in our faces.  He suggested some significant paradigm changes at defining ALS.  He even suggested that we have failed in the past, so it's time to change.   Unfortunately the whole paradigm shifting idea seemed to be lost after the next coffee break.  We fell back into our same old same old ruts.

We started seeing the use of the word "subjects" for people participating in ALS research in the first prize-winning presentation.  Oops.  It was better than in the past, but we still see that word used by far too many neuroscientists who should know to have more respect for the most important people on their clinical research teams.

A few of the typical acknowledgement slides at the ends of presentations included people with ALS.  Even fewer had them at the top where they belong.  Something so simple, so respectful, so appropriate is so difficult with this group.

During a panel on telemedicine and precision medicine, we heard again of the silos in precision medicine programs.  If this were a corporate shareholders' meeting, the crowd would have risen to say, "Fix the silos. Now."  This crowd nods and moves on.  I had my hand up for the "Fix the silos now" suggestion but time for questions ran out right before my moment.

A neurologist asked about how they would get paid for telemedicine.  Surely a practical question, but certainly if more people with ALS were present, it may have not been on the front burner.

At a breakout session (I was not present), one of the patient fellows evidently asked a mic-drop question to a panel about the cost/value proposition in comparing supportive care for vented people and expensive meh drugs for ALS.  You can consider his thoughts on this and another important topic, placebo groups  here.

And speaking of placebo trials, we saw to instances where placebo groups seemed to do relatively well.  Overachieving placebo groups spoil the spotlight for the drug.  So... live by the placebo sword, die by the placebo sword?  I wonder if randomization put the real drug in the hands of those who were in the overachieving placebo group if a meh drug would have been found to be wildly effective.

I learned the term "enrichment strategy."  It's a way of picking trial participants to make the drug more likely to show that (or look like) it works.

In the session on recent clinical trial results, it was a lot of "more study needed."  To my eye, either the drugs aren't working or the trial designs aren't working.

There was a chippy moment when a well-known neurologist suggested that reproducibility of trial results was needed for edaravone.  The drug company doc didn't like the suggestion.  Chippy moments are a lot more constructive when we have good discourse and not indignation.

There was a panel on Right to Try, Expanded Access, etc. that included an ethicist, an entrepreneur,

and a neurologist.  Does anyone notice who is missing in this discussion?  Dr. Bateman-House from NYU was quite good, and I appreciate her tweeting answers back to me while she sat on the panel on the big stage.

I saw one presentation that I didn't think was very solid science, and it was an area where I feel I have some background.  A neurologist who is vested in the project got the mic at the end and said, "Very nice work..." before his question.  That spoke volumes to me.

An ALS advocate at the symposium mentioned more than once that she got the best information from symposium participants at the hotel bar.  That's true sometimes.  I learned of a study on lithium that showed actual efficacy in a specific subgroup.  I saw a communications technology expert help one of the patient fellows who had been led down the wrong technology path.  Much goes on outside the sessions here.  I even learned on an elevator that my tweets about the word "subjects" are useful in educating students to avoid that term.

There was another mic-drop moment in a large session when a man with ALS asked a presenter if people with ALS were included in the study design process.  Nope.  Awkward.

There was much, much more.  Poster sessions were not as accessible as they had been at past symposia, and I felt like I missed a lot of material and good conversation there.

I realize that it takes far too much time and effort to implement even the simplest changes in the fight against ALS. I feel like as much as we rely on placebo trials, they aren't so stellar that we should stop looking for alternatives.  I feel now more than ever we need to embrace people with ALS and caregivers who are interested in the science at these symposia.  And I am especially grateful for the inaugural Patient Fellows who made the trip and added to some important conversations.


Late addition:  Oh, and take your Vitamin D.  One study it as a possible biomarker to disease progression.  More study needed.  A neurologist asked a refreshing, common-sense question -- "Why not test people with ALS for Vitamin D deficiency?  It's not expensive.  Vitamin D supplement is low risk.  Just do it."  At times like this, if all neurologists exercised such common sense and reported findings, we could figure out if it's a helpful biomarker or not (and perhaps help some people with ALS along the way).

Meet The ALS Clinical Research Swear Jar

Last week on a webinar, I heard a clinical investigator puzzled at the low enrollment in a particular trial.  How about dropping the word "subjects" for the most important people on your clinical research team, Doc?

Nobody aspires to be called "subject."  It's not respectful.  It's objectifying.  It conjures up images of royal underlings.  People who put their lives on the line to advance science deserve respect.  Words matter.

Reference to "subjects" has long been banished in many disease areas and industries.  ALS researchers don't seem to have gotten the message.  Maybe this will help.  Let's have a jar where ALS scientists have to drop a Benjamin every time they slip with the "s" word for a valued clinical research participant?

ALS MND Symposium presenters, use a more respectful word or have your wallets ready.

We Can Multitask. We Must!

You never know when a new, hot-button ALS advocacy issue will pop up.  ALS policy issues don't happen in an orderly set of three things that can be addressed every May.

Things can happen quickly, especially the bad things, and we need to jump on them and speak up to our legislators.

For the last three months, the Orphan Drug Tax Credit has been on the chopping block as part of the tax-reform proposals.  If you're not familiar with the Orphan Drug Tax Credit, Google is your friend.  It has been effective in stimulating development for low-prevalence diseases like ALS and dozens of others.  It's an odd tax credit to eliminate since the research is valuable and the orphan diseases are expensive in many ways.  There have been tax-credit abuses, but you don't throw the baby out with the bathwater.  We all need to make sure our legislators know that this tax credit must not be eliminated in the name of tax reform.

We need to jump on this.  We need to email our legislators now.  This is urgent.

Other things pop up constantly -- inconsistencies in Medicare coverage, loss of Medicaid, changes in technology access, drug approval processes, SSDI rules, CDC Registry dysfunction, etc. -- that we need to speak out about.  Our elected officials and government employees need to know our concerns and expectations.

Where are our calls to action?  It's frustrating (and wasteful) when our large advocacy organizations are slow to mobilize their large bases of advocates on issues of immense and urgent importance.  If only their passion for SoMe fundraising also applied to public policy issues.

ALS develops the world's greatest multitaskers.  Once you've been a hands-on ALS caregiver, you can juggle and complete dozens of tasks simultaneously.  You must.  We advocates can multitask.  We can handle more than a neat list of three priorities in May.

A concerned, capable base of advocates that wants to be effective and engaged is a terrible thing to waste.

Which People With ALS Count?

Dear CDC,

You published your first prevalence report from your ALS Registry in 2014 and asserted that ALS was white guys' disease.

The next day you were warned of publishing assumptions based on incomplete data in a thoughtful paper written by a man with ALS.  You obviously didn't pay attention.

You kept misinforming in the next annual report published in 2016.

We persisted to try to point out that we don't know what we don't know.  Which dots was the CDC collecting?

And here we are today with your new report that should cause outrage among all who have been paying for this Registry.  You finally admitted that you don't know what you don't know.

We repeat, the CDC ALS Registry found only 2720 people with ALS in areas where other studies identified 4767 people in a corresponding time period.  Of the 2047 who were missed, they were more likely to be people of color and younger than the group that the CDC ALS Registry found.

This is more than an "oops" moment.  This is serious.

Our data represent us.  With a poorly understood and poorly diagnosed disease, the Registry's failure to count some demographic groups has real consequences.

1. We don't know how many cases of ALS are not diagnosed before people die.  Saying that ALS is white guys' disease can be a self-fulfilling assertion.  How many doctors miss the ALS diagnosis in people of color?  After all, the CDC said it was mostly white guys' disease. 
2. Good epidemiological data should inform science and priorities.  Your misleading data are perhaps steering precious research funds down some wrong paths.
3. Finally, you brag of your Registry's old-fashioned emailing system to inform some people with ALS of some clinical trials.  Now we know it actually contributes to the problem of lack of diversity in trial participation.

Every person with ALS counts.  We must stop the madness of publishing data that suggest that all people with ALS look like the incomplete subset of people that the CDC's ALS Registry found.

This should be the end of the CDC's ALS Registry.  An algorithm, process, and reporting that systematically discriminate are just wrong.

Sincerely,

A Disappointed ALS Advocate and Citizen and Taxpayer

Are You Sure You Want To Drive Down This Foggy Road?

Transparency.  It's a word that every not-for-profit knows is important.  We must not take it for granted.

The fight against ALS is fortunate to have multiple industries with business interests in selling services and products to those dealing with ALS.  Many of the companies in these industries have generous philanthropy programs.  We are grateful for those.

Over the past few years we have seen the generosity lost in the fog of the word partnerships.

The corporate logos on websites and banners are not accompanied by any indication of the sizes of or restrictions on the donations.

Not-for-profit communications that shed light on services and products to benefit those dealing with ALS do not share that there have been charitable donations from those fine "partners."

Transparency.  We need to take it seriously with policies and disclosures that lift a potentially dangerous fog.

Organizational blind spots are bad enough. Why would we drive into a dense fog?

Ahoy! Tweet Us Some Doubloons of Valuable Content Thar

We're not mere sprogs.  We're interested in the science.  We care about clinical research.

This week NEALS is holding a big annual conference in Florida.  The agenda is extremely interesting for those interested in ALS clinical research and changes that may be in the wind.

https://www.neals.org/uploads/blog/doc/2017_Annual_NEALS_Meeting_Agenda.reformat_v.1.0.pdf

The meeting is not webstreamed to our knowledge.  A few (including @NEALSconsortium ) have tweeted or posted on Facebook.  Unfortunately they have not given us any insights into valuable content.

There was the news of the Pirate Cruise --

We get lots of title slides and pictures of presenters, but we get no content.

There was a special kind of irony when we saw this slide.  Were they actually talking about "nothing about us without us" without us?

And we learned about the Luau.

The dozens of people with ALS who have been trained at the post-conference CRLI sessions in past years may actually be interested in the science and clinical research processes you are discussing this year.  Hundreds of us who donate and advocate for better clinical trial participation care deeply about the valuable discussions that take place after all those title slides.

Avast, ye.  We're here. #PatientsIncluded #EveryoneIncluded , please.

Aloha.

What's A "Partner"

https://www.merriam-webster.com/dictionary/partner

Organizations like to refer to their partners in the fight against ALS.  It's a feel-good concept.  Everybody loves supportive partners.  Partner is also word that is vague. It clouds the true nature of the relationship. It flies in the face of transparency that not-for-profits say they strive for.

Huge donor?  Major donor?  Minor donor?  In-kind donor?  Volunteer?  Vendor?  Client?  Party to a contract?  Party to a handshake?  One with implicit or explicit access?  Endorser?  Endorsee?

To live up to the spirit and letter of transparency, how about simple crystal clarity on specific monetary arrangements and promises and expectations between partners?

It's simple.  It's good for everyone involved because transparency is good for everyone involved.

MIngle, If Not To Help, At Least To Learn

Dear ALS Organizations, Drug Developers, DME or Technology Suppliers, and Anyone Else Who Is Paid To Help People With ALS,


There are places online where people with ALS go to get help.  Some are your own message boards.
Some are on Facebook.  They are not hard to find.  These places are extremely useful and efficient for people with ALS and caregivers to get support.

As a man with ALS once said about paid ALS professionals avoiding participation in online support, "It's like they don't want to mingle with the unclean masses."

You need to mingle.

It is absurd when people who participate in online support groups constantly have to tip off organizations and companies that they have issues that need attention.

It is unconscionable for an ALS organization to host an online support group (with a big red logo at the top of the page) where people seek help only to find out later that the ALS organization itself doesn't take the time to look regularly, let alone participate.

You all need to mingle.  If you refuse to supply help in a medium that is efficient for the people who seek assistance daily, then at least look and learn.

The unclean masses await your respect and interest.

Ask The (Please Be Specific)

Over the years I've experienced many "Ask the Experts" sessions related to ALS.  It's always a panel of neuroscientists who talk a lot about ALS research and then take a few questions.

The "Expert" crown defaults to them.  They speak from the dais.  We get to listen a lot and ask.  They are by default "The Experts" in the fight against ALS.

But wait, there are all kinds of people with superb and valuable knowledge related to ALS --

• There are people who know a lot about insurance.
• There are people who know a lot about communication technology.
• There are people who know a lot about low-tech hacks.
• There are people who know a lot about suction machines.
• There are people who know a lot about breathing.
• There are people who know a lot about the FDA.
• There are people who know a lot about moving a person on and off a commode.
• There are people who know a lot about taxes.
• There are people who know a lot about drugs.
• There are people who know a lot about data.
• There are people who know a lot about choking.
• There are people who know a lot about financing research.
• There are people who know a lot about stem cells.
• There are people who know a lot about feeding tubes and nutrition.
• There are people who know a lot about what's important in living with the beast ALS.
• There are people who know a lot about primary care in ALS.
• There are people who know a lot about constipation.

Our default implication for the word experts being neuroscientists is revealing. There are many people in the fight against ALS, including those living with it, who know a lot.  We still live in a hierarchy where people with ALS and caregivers are at the bottom.

Words matter.  "Expert" is not a royal title to be owned by anyone by default.

It's time for simple changes to some traditions.  "Ask the Neuroscientists," anyone?

That Looked A Lot Like The Basset 500

Anyone whose life is blessed with a basset hound knows about the Basset 500.  Occasionally hounds
will break into running gleeful circles.  Sometimes they do big circles outside in the yard.  Sometimes they zoom around a circular floor plan inside.  It's exercise  They have fun.  They get nowhere except right back where they started.

Last week my hound was in the office with me when we watched the CDC ALS Registry annual meeting.  She could relate to what happened.

Every year the paid contractors (ALSA, MDA, and now Les Turner ALS Foundation) come to the meeting without any data to measure their effectiveness.

Every year the group ponders how to increase Registry self-enrollment.  There is lots of discussion of things to do that might increase people with ALS contributing their data.

Every year one of the people with ALS invited to the meeting gives the common-sense advice that you have to measure and you have to set goals.  There are nods, but nobody discusses specific metric goals because nobody establishes the pertinent metrics and nobody comes prepared with a measure of the actual performance.

Every year the meeting concludes with no goals, no targets.

My prediction for 2018:

• The contractors will come to the meeting without any data that measure their effectiveness. 
• The government project leaders will insist on calling these paid contractors "partners" again (clouding the true relationship and a need for accountability for paid work).  
• There will be discussion about all the stuff you can do to increase self-enrollment.  
• A common-sense person (not paid to work on the project) will suggest that they need to set goals and measure.
• They'll talk more about stuff.
• The paid facilitator will never bring them back to the idea of measurable goals.   
• They will leave for another year.

Every year they get lots of exercise and get nowhere except right back where they started.

The dog wants to know how she can get paid half a mil a year to run in circles.

Expressing Gratitude

It's not just good manners.  It's important.

Yesterday afternoon I listened to a NEALS webinar about a clinical trial.  A gentleman with ALS spoke about his personal experience in this and other trials.  Listeners received wonderful insights.  At one point he had advice for those in charge of the trials.  It's important to say, "Thank you."  Boy, is he ever right about that.

Every year I sit through a lot of ALS research presentations.  There is traditionally a slide at the end of the deck that thanks the funding agencies.  Seldom is there a slide that thanks the people who volunteered for the research.   It should really be the first one in the deck.

Earlier in the week I listened to two days of presentations that included ALS epidemiology studies funded by the CDC ALS Registry project.  Never did I see all those anonymous people who supplied data (often having to supply the same data multiple times in two or three different studies) specifically thanked for their contributions to a study.

There is a lot of angst over getting people with ALS to participate in clinical studies.  Perhaps researchers could simply stop calling their project volunteers "subjects" and start genuinely thanking them as the most important members of their project teams.

Last evening I was invited to a gathering that really drove this home.

There is a project, Hero's Journey Art, that Eli Lilly and Company has been developing.  People who have participated in clinical trials have been invited to submit wooden bricks decorated with whatever their thoughts are about participating in clinical research.  No rules.  No censorship. No coloring in the lines.  Just express yourself. I did my brick many months ago.  It wasn't hard for me.  Mine wasn't a graphic masterpiece, but it contained the words that made a clinical trial so important to Mom.

Last night one of the three sculptures was (literally) unveiled in a cancer support center in my city. There was a larger crowd than I expected.  There were lots of people who had participated in cancer trials.  It was a good reminder to me that there are a many evil diseases out there that still need so much research so that people, especially young people, can live.

When the sculpture was unveiled, the artist, John Magnan, told us a little about the journey and invited us all to look closely and even touch the sculpture.  And that's when this art touched me in a way I never expected.

People approached it, and as I did, looked for "their" bricks.  But they also looked for their friends' bricks.  And they looked at the messages on bricks of people they didn't know but who shared an unspoken bond of having participated clinical research.  The brick themes were all so different, yet they formed something cohesive about facing disease. It was captivating to meet over 300 important and diverse contributors to research via their bricks.

And a major pharmaceutical company was simply saying, "Thank you," in a remarkable way.

In the next year when I need a quiet moment to boost my faith in the fights against diseases, I'm lucky that I can stop by and soak in that sculpture.

Thank you to everyone who contributed to a clinical trial and told a story in a brick, and thanks, Lilly and John Magnan, for an important expression of gratitude.

Buy Your Own Lunch

Over the last few days we watched the webstream of the CDC's annual ALS Registry meeting.

I'll never forget one disappointing moment that had little to do with the Registry itself.

In the last session, people chimed in with suggestions for the coming year. Fine.

One world-famous ALS clinical research neurologist suggested that there be a meeting of ALSA and MDA clinic doctors the afternoon before the big ALS MND Symposium in December to speak to the value of the Registry.  Fine.

He turned to the CEO of the ALS Association to ask for coordination of such a meeting.  Fine.

He then matter-of-factly turned to a representative of a pharmaceutical company actively involved in ALS research and suggested they "sponsor" the event and even perhaps provide lunch.  Now wait a minute.

These are employed neurologists.  Buy your own lunch. 

This is a Registry project that burns through a budget of $10,000,000 every year.  It can pop for an urn of coffee and some sodas in a hotel meeting room.

Let the pharmaceutical company invest all it can in ALS research.  Expect the pharmaceutical company to invest all it can in ALS research. Treat that relationship in a businesslike and respectful and ethical manner.  Don't treat it like your personal piggy bank or a money tree to be shaken down.

There's no such thing as a free lunch.  Think about it.

Someone, Please Help Ask Some ALS Registry Questions

Tuesday (August 1) afternoon and Wednesday morning, we hope to hear answers.

We also hope that many will listen in to the CDC's ALS Registry Annual Meeting --

https://www.alsregistrymtg.com/

Attendance at the meeting is invitation-only.

For some background, here is the recently published report from last year's meeting --

https://wwwn.cdc.gov/als/Download/2016%20ALS%20Annual%20Meeting%20Summary%20Report.pdf

Here are some questions that I've written in the margins of my dog-eared copy of last year's meeting report:

1. Why was last year's meeting not webcast?
2. Why did we only get a report for the first day of last year's meeting and not the second?
3. Last year's meeting report told us --  "Another important thing is capture/recapture methodology. Lorene Nelson will be giving you a presentation tomorrow on that, and that will allow us to estimate the number of possible missing cases in the Registry."  This seems like a critical project, especially since you are publishing reports that are asserting the demographics of ALS. In reality, we don't know what you don't know.  Could we get some information on what Dr. Nelson presented last year on that second day regarding the recapture and completeness, please?
4. In its organizational marketing materials, ALSA continues to assert that they help 15,000 people with ALS annually.  In the past, MDA has published similar numbers.  How do we reconcile those totals to the 15,908 people who were found in the entire nation in the last published report from the Registry?
5. In last year's meeting report, Dr. Weisskopf made a comment -- "So just thinking about it now, it strikes me that another way to sort of look at this is kind of potentially missing cases, is actually to take the people who respond to the portal that are captured in your other databases and determine who in your other databases those web people 22 are missing; right, and then you can sort of use that to figure out for that 20% that don't show up in your databases how many people they're missing sure. And you can up at that point."  That suggestion ended without discussion in the written report.  Plop.  Did anyone discuss that further?
6. In this climate of likely CDC budget cuts, is anyone making any contingency plans to save the tissue and data in case this project is not funded in the future?
7. The 21st Century Cures Act included an integrated neuro-surveillance system for a number of neurological diseases. Scientists and taxpayers don't need redundant systems, and to have more neurological diseases represented in a well-designed database could provide valuable insights.  The Cures neuro-surveillance also included authorizations of appropriations of just $5,000,000 per year.  How can they do so much more on so much less budget?
8. Last year this comment was made in a discussion --  "But after appropriations and CDC overhead, we're more down toward the 6-7-million-dollar mark."  We advocates were told to ask for and we got you a $10,000,000 appropriation for the Registry for one year.  I'm surprised nobody asked this at the meeting, so I will now:  What in the world is CDC overhead that eats up 40% of a project's $10,000,000 that we thought was necessary for the Registry?
9. Why have you never invited Stephen Finger, PhD to an annual meeting?  http://www.huffingtonpost.com/stephen-finger/national-als-registry_b_5620631.html
10. In last year's meeting report, a lot was made of difficulties since ALS is not notifiable. In all the years we were told to advocate for ALS Registry legislation, did anyone designing the legislation ever ask that ALS be made notifiable?
11. Your past meeting reports often mention that Registry reports are consistent with "the literature" or other published reports and registries.  Are there any results from the Registry that are not consistent with other findings?
12. Has anyone gathered data on what percentage of people with ALS are in Medicare Advantage plans by state or by clinic (in hopes of better defining the related data problem)?
13. How many researchers have actually requested data from the Registry?  Tissue?
14. All the paid contractors on this project should be able to provide some data on the effectiveness of their work and not just all the stuff they do or plan to do.  For ALSA, MDA, and Les Turner:
1. How many people living with ALS were in your clinic system at the end of 2016?
2. How many new people with ALS entered your clinic system during 2016?
3. How many of those new people with ALS in your clinic system also self-enrolled in the CDC's ALS Registry in 2016?

We hope that someone who is invited to the meeting will ask and that we all will learn the answers.  Please.  Thank you.

Dear Contractors, Please Come Prepared

Dear MDA, ALS Association, Les Turner ALS Foundation,

It's nearing time for the CDC's Annual ALS Registry Meeting (at least we think it is).  You know the date for sure because you are the only organizations allowed to nominate participants for the meeting.

You are also well paid as education-and-outreach contractors on the project.

Please prepare some very simple data for the meeting so that we all can measure success.

1. How many people living with ALS were in your clinic system at the end of 2016?
2. How many new people with ALS entered your clinic system during 2016?
3. How many of those new people with ALS in your clinic system also self-enrolled in the CDC's ALS Registry in 2016?

That's it.  Three simple measures will make that meeting a lot more productive.  Please be prepared.

Thank you.

Sincerely,

@ALSadvocacy

p.s. For inquiring minds, the most recent annual payments to the three per usaspending.gov were:
Les Turner ALS Foundation $100,521
ALS Association $376,852
MDA $548,658

Watch You Don't Break Your Arm Patting Yourself On The Back

That was Dad's standard line whenever we got braggy at the dinner table or a sporting event or a school activity.  Hubris was never a problem at our dinner table.  Humility was valued by my parents' generation, and we learned lessons about the important distinction between arrogance and self-confidence.

Every time I go to an ALS fundraising event or a scientific meeting, Dad's words go through my mind.

"We have failed" does not make a good fundraising slogan or justification for the next big research grant.  ALS events are celebrations of accomplishments that will give the public the confidence to invest.  But, Dad's words ring true to me when I hear all the "accomplishments" and "promise."

We have failed.  Individually and collectively, we have failed.  Sure, progress has been made, but people are still dying from ALS, and the more we discover, the more complicated we realize the problem is.

The first time I went to an ALS walk many years ago, I followed a man who clearly had ALS.  He struggled walking, but he walked.  As I trailed him, I wondered what he had been doing a year before.  Playing golf?  Skiing?  Running marathons?  And I thought that it was unlikely that he would be at the walk the next year.  That's a stark, ugly reality of ALS.  That gentleman walking directly in front of me helped me understand that we really had failed him despite all the fundraising and balloons.

Some folks on the forum at als.net have been giving me grief about my encouraging people with ALS to apply for the fellowships for the International ALS MND Symposium in December.  What difference does the symposium really make?  What have all those scientific accomplishments that are celebrated every year really achieved?  I understand what my friends there are saying.  I understand 100%.

Sometimes it seems like scientists need my Dad to tell them to watch they don't break their arms patting themselves on the back.

People living the reality of ALS at the symposium could convey that, too, simply by their presence and participation.  Their reality in the midst of the celebrating scientists could add a much-needed dose of realism and humility and perspective.

I think Dad would encourage them to be there.

https://alsmndpatientfellows.wordpress.com

This Small Step Is A Giant Leap

Over the years I've been critical of the International ALS MND Symposium not embracing patient attendance and participation.  Today, we see a small step forward in a direction to fix that problem.

The Symposium is an old-school scientific meeting.  The ALS topics discussed are broad and very
deep.  https://www.mndassociation.org/research/international-symposium/

In the past, if anyone with ALS has chosen to attend, it has required payment of the substantial registration fee (plus a separate fee for an accompanying caregiver).  Add travel and hotel expenses, and there has been a large financial barrier between people with ALS and the Symposium.

This year there is a new program to help mitigate some of the financial barriers --

https://alsmndpatientfellows.wordpress.com

This is a giant leap toward having people with ALS in the midst of some important discussions.

Anyone with ALS who is interested in the science now has a chance to be in the center of the newest science in Boston in December.  This is a big meeting typically filled with big players in ALS research and with big announcements.

People with ALS are notoriously bright and high achievers.  Finally, a step has been taken that can make them part of the scientific discourse.

Please apply if you have ALS and are interested in the science.

The Race To Eureka Needs To Start Now

And people with ALS need to be in charge of the race.

20-20 Hindsight

• How helpful would it have been if we could have seen individual trajectories for people with ALS who have used riluzole over a number of years?
• How helpful would it have been if we could have observed data on Americans who have been using generic edaravone with or without riluzole for the past several years?
• How helpful would it have been to have access to data on ethnicities and genetics and experiences of people with ALS who try any number of things in their journeys?

That's water over the dam, but the massive information loss need not continue. There are a lot of good reasons to start the race to Eureka now!

To Get To Eureka, You Need Data

The promise of precision medicine is to know which potential treatments will work for which people.

And in recent days with the approval of a new ALS treatment, we have seen clinicians suggest that combinations of drugs may hold the most promise for effective treatment for people with ALS.

And there are more promising (yet not blockbusting) drug candidates that may be approved in the next several years.

We're in a world of promise without the data to take us to true Eureka moments that we need.

You Own Your Own Data

There has never been a better time in history to get ALS data out in the sunshine for all kinds of smart people to look for insights.  People with ALS own their own data, the most powerful factor in the race to Eureka.

The old public lithium spreadsheet proved that people with ALS can contribute data that will tell a story in a hurry.  We have better tools now, e.g. www.patientslikeme.com , but the tools only work if people contribute their precious data.

There are several individual patient tracking portals (e.g. ALSTDI PMP, AnswerALS) that could release selected data to a common public place like patientslikeme.com to keep people from ALS from entering data in several portals.

And there are patient organizations that could encourage information sharing but have traditionally avoided it like the plague for some reason.

Who really has the incentives and motivation to figure out what specific subgroups of people respond to an ALS treatment? Think about that. It's the people with ALS.

Do you want to go to Eureka?  The race can start now with people with ALS in command.

There Are Elephants In The Room

A little over a week ago we got the wonderful news that a drug had been approved for ALS in the US.  Since then we have had multiple news reports and webinars and communication from ALS organizations and MT Pharma America about the drug, Radicava (edaravone).

Don't get me wrong.  I ecstatic about having another tool in the toolkit to treat ALS.  There are just these two elephants bothering me.

Elephant 1 - The Awkward Age Of Precision Medicine

This is a drug that was developed in Japan in the late 1980s and was approved for use in strokes there in 2001.  Based on later ALS studies in Japan, it has been used there for ALS since 2015.

I sensed the first elephant right away.  We've been so conditioned of late to the heterogeneity of ALS and the genetic differences that I wondered how people with ALS in Japan differ from those in the United States.  What are the most common ALS genes in Japan compared to the the US? Is there anything in the Japanese data that will help us predict which people in the United States are most likely to respond?

The elephant sat quietly until I listened to Friday's webinar when Dr. Palumbo from MT Pharma America showed a slide modeling comparable blood levels between people of Japanese ethnicity and Caucasians.  I think that showed us that the drug gets to where it needs to be in both groups, but it really doesn't tell us anything about how the groups responded to the drug, does it?

The concepts of precision medicine today constantly drive us to know more about individual cases of ALS and what interventions are likely to work in specific individuals.  New concepts are to know everything possible that distinguish an individual case of ALS.  Can old clinical trial data shed that kind of light? The elephant emerges. How can we figure out which people in the United States are likely to respond (and as important, not respond) to Radicava?

Elephant 2 - The Price

I'm the person in the room who normally defends drug pricing.  New drugs are expensive to discover and develop into commercial products.  As expensive as pharmaceutical products are, they usually provide results that are both cost effective and beneficial to our lives.  I'm pretty immune to drug price sticker shock.

Right after the Radicava approval announcement when some pricing information was revealed, an ALS caregiver asked me what I thought about the price.  I said that I thought if they had a slam-dunk product and could say that people with specific flavors of ALS would receive significant therapeutic benefit, that the extremely high price was probably in the right ballpark.  Unfortunately, the data that we've seen don't make it likely that this is a slam-dunk product for anyone.  It will be something to consider and try, and it may help, and it may not help, and it may be hard to perceive the difference individually.  Elephant 2, meet Elephant 1.

People with ALS will try it and I'm glad they can.

It's a fact that people who are not going to be responders to this product are going to pay the astronomical price, and we don't have data to minimize the size of that group. It's not like Costco where you get your money back later if it doesn't work.

The webinars we've heard have been great about explaining the process of getting payers to pay for new indications.  If it's likely that Medicare will pay for Radicava and its administration for people with ALS, that will be good.

That doesn't mean that we shouldn't talk about the price elephant.  We absolutely should.  It's not harmless for the government to overpay for something, especially when we may be on the verge of more product approvals for ALS in the US.

Over the past couple of years I have gained huge respect for the way DMD parents advocate for approval of therapies for their kids and then for access to those therapies.  We could learn from them about not ignoring elephants.

We Need A Toolkit

Dad was an engineer.

He taught us that you always need a good basic set of Craftsman tools, and every tool has its own use.  Never use a screwdriver when you should use an awl.  Never mess up a bolt with the wrong sized wrench.  Never hammer with anything but a hammer.  And duct tape has no correct application in plumbing.

On Friday we got the wonderful news that MT Pharma America was granted FDA approval for
Radicava (edaravone) for ALS.  There is joy in hearing that there is finally a new tool in a barren toolbox.

We can't stop building a toolkit with one new wrench of unknown size.  We need to figure out for whom Radicava works, and we need to keep complementing the toolkit with new options.  This is one tool.  We need that Craftsman set to address a nasty, heterogeneous disease like ALS.

Edaravone has been used in Japan for a number of years for stroke, and it has been approved and in use there for ALS for almost two years.  I acknowledge that this is fast approval by FDA standards, but two years is a lifetime for many with ALS.  We are grateful for Friday's news.  We must forge ahead more quickly and more aggressively.

We have a toolbox to fill with the right tools for the right people.

It's An Expensive Trip To G12.21

Every person with ALS can tell about an expensive journey to get to the diagnosis.

There are millions of dollars worth of wrong diagnoses along the way.  There are tests.  There are surgeries.  There are physical therapies.  There is lab work. There are speech therapies. There are more wrong diagnoses.  There are MRIs.  There are drugs.  There are scans.  There are psychiatrists.  There are more doctors.  There are braces. There are more wrong diagnoses.  All the while, the person whose ALS is not yet diagnosed continues to decline.

Finally, one day, often a year or more after the start of the expensive journey, a physician says, "You have ALS."  G12.21 is encoded on a medical record.

Mom had one of those long, expensive journeys. She declined terribly along the way. She didn't get her ALS diagnosis until just four months before the ALS took her life.  But for the initiative of a sharp speech therapist, the diagnosis may never have happened.

Some cases of ALS are fast.  Most cases of healthcare delivery are slow.  They are even slower for people who don't have good insurance coverage.

How many people die from ALS without ever finding out that they had ALS?  I guarantee that problem will get worse if yesterday's AHCA legislation moves forward.

The Path From Gingerbread to Disrespect

Since the CDC launched its ALS Registry website in 2010, it has been fraught with gingerbread.

Early on, they installed a "Service Locator."  At the time I thought it was a poor decision.  They
already had links to ALSA and MDA sites where chapter and clinic locators are available and kept current.  Why would the CDC want to maintain with its own databases of chapters and clinics?

The Service Locator is on the lower left of the main page at www.cdc.gov/als 

Through the years it stopped working for me.  I've asked others to test in their browsers, and nobody I know has been able to make it work for many months.

We went from an unnecessary piece of gingerbread to a broken piece of gingerbread.

People seeking ALS services are typically in the throes of a disease that consumes all of their time and energy.  They don't need to type their zip codes into a trusted site only to find out that nothing happens.

We went from an unnecessary piece of gingerbread to a broken piece of gingerbread that is disrespectful of those who try to use it.

And it's not that hard to make broken gingerbread disappear from a website.

Why?

Obviously I'm interested in ALS advocacy issues.

I read a lot and try to study them.  Our voices are important as are our ideas, and I believe that we can make a positive difference.

Every year I go the the ALSA ALS Advocacy Conference and Day on Capitol Hill.  I pay the fee and pay my own expenses.

In an email yesterday, I received a nice invitation:

I requested to join on facebook and was accepted. Nice.  I liked the idea of this new dimension to Advocacy Day preparation and discussion.

This morning I submitted four items to post.  Evidently, everything is moderated, so all were pending moderation when I signed off of facebook this morning.

Thank you for providing this area.  It’s a nice addition to let us all discuss substance that has been difficult with the format of the conference over the past few years.  We are all more than just our stories and I feel strongly that policies and our voices and thoughts matter. 

I have three topics related to the Registry that I will post and I hope they will be part of the discussion.
Thank you.

__________

1/3  Last summer’s annual Registry meeting at the CDC was blacked out.  All we have is an “executive summary” report that is not at all substantive.  When I inquired months ago about getting a detailed report, Dr. Mehta said that there would be a recording of the proceedings posted.  When I inquired of ATSDR about an ETA for the detailed proceedings, I got the unhelpful response, “When it’s ready.”

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

We are being told to request another $10,000,000 for the project without any substance on who was invited to that meeting, what was discussed, what problems were (or were not) addressed, and what direction the leadership is taking the project.   How can we be responsible advocates without having that information?

__________

2/3  We have never seen a businesslike accounting of how ATSDR spends $10,000,000 per year on the Registry.  One year after we pressed the issue, we got a vague set of percentages in some Powerpoint pie charts at the Advocacy Conference.  It’s not unreasonable for us advocates to know how that budget was or will be spent, in dollar categories (like any good budget analyst would supply). 

As a friend once said in a motherly way, “I’m not giving you another $10,000,000 until you tell me what you did with the last $10,000,000.”  

__________

3/3  What is the cumulative total that has been appropriated for the Registry over the many years, starting with the so-called building-block appropriations that happened in the years before the 2008 passage of the ALS Registry Act. 

We advocates can’t make good cost/value assessments without having a good handle on the costs. 

__________

This evening I find myself locked out of the group.  I can only assume that my discussion points were never posted.

Perhaps the group is to stick to dinner plans and not substance?

Why, oh, why would you stifle polite, informed discussion on something this important?

Why?

ColLABorate, Please, Now!

A young father with ALS is in this clinical trial, on the drug protocol for more than 60 days --
https://clinicaltrials.gov/ct2/show/NCT02437110

He actually has experienced clinically measurable improvements.  That's huge.  Normally an ALS trial success consists of getting worse less quickly than before.  Here, we're talking improvements.

I asked him if he knew if anyone else on the drug was having similar results.  Unfortunately he was the only one he knew of on the drug protocol.  NIH had a very small sample set and found very few HERV K positive people with ALS.

Isn't this the promise of precision medicine?  To test the right therapy on the right patients?  Why aren't we finding the right patients for this clinical trial with some urgency?

There are numerous, separate precision medicine projects in the ALS space.  There are even more repositories of tissue of people with ALS.  Cmon, folks, let's screen for HERV K and find some candidates and get this trial filled and completed quickly. Now!

It's not that hard if you really mean it when you say you collaborate.