Going to the Pool...

...or is it really the pools.

When we were kids we loved to go to the pool.  But when we got there, it really wasn't a pool.  There was a baby pool, a junior pool, a lap pool, a shallow pool, a deep pool, a diving tank.  They were separated.  Some of the water sloshed from one to the other, but they were built separately and cabbaged into a complex as the Boomer population grew.

Today we have scientists claiming to share data, but they're not building a pool.  Much like our swimming pool, they are adding different pools to serve different purposes.  Sure, they all contain data and some slosh around, but it's not a pool.  They people using the junior pool are not able to swim into the deep pool.

From the recent "Ask the Experts" session at the ALS Symposium in Orlando, there is a question at around 1:35 about two of the databases in our growing complex of pools...
http://asktheexpert.eventstreaming.tv

The answers weren't very good.  The projects are "different."  "Registry" is a bad word (no kidding). They can work together.  Right.  Show us exactly how the expensive CDC ALS Registry can work with the NIH CReATe Registry.

We need a pool for smart ALS intelligence.  We don't need a complex of segregated and redundant and expensive pools that serve independent needs.

Calling the place we went in the summer "the pool" didn't mean that it was really a pool.

The Golden Loofa

If only Senator Proxmire and his Golden Fleece Award were still around! Perhaps he would call this the Golden Loofa -- A disappointing, shady version of "you scratch my back, I'll scratch yours."

The Background

Well over a decade ago, those involved in the fight against ALS have wanted a good, central census of people with ALS -- a complete, statistically meaningful nose count of the who, where, when of the incidence and prevalence of ALS.

The ALS Association proposed that the CDC create such a central "registry."  That seemed like a good idea since individual state and private registries were counterproductive to getting a good national census.

ALSA (The ALS Association) has an annual advocacy legislative agenda.  Funding was to be requested for "building block" projects.  Seeds were sewn to have an ALS Registry enacted into law. The ALSA approach is for advocates not to worry about the technical details -- just tell your stories.  We had little information about the project design or anticipated budget details.  Many of us worked hard at our own expense to get the Registry passed.  Certainly it was important for us to collect the dots if we ever were to connect the dots.   The ALS Registry Act was finally signed into law in 2008.  https://www.govtrack.us/congress/bills/110/s1382

The annual dollars that we were to request to actually develop and deliver the Registry increased.  Development of the Registry would be expensive.  We received no businesslike itemization of the dollars we requested annually.  ALSA told us what to ask for.  We told our stories.  We worked hard.

http://web.alsa.org/site/PageServer?pagename=ALS_Registry_Background#Congress

The project was "generously funded" (the words of the CDC project leader in 2010).

The design of the new Registry turned out to be based on passive data mining of government files -- Medicare, Medicaid, VA.  People with ALS are eligible to get into those systems fairly quickly, so that looked like fertile ground for finding them (and avoiding any attempt at mandatory reporting of ALS as is done for cancers).  The concept of a web portal for voluntary reporting was added to the base design.  And we waited.  The most recent government files are already several years old by the time the CDC could get them.  Even though the web portal finally launched late in 2010, we couldn't get any data out until the passively-mined government files caught up.  We were curious and asked about progress and patient uptake.  In addition to the time lag excuse, ALSA staff told us that no information based on portal data entry could be released because of OMB rules.  The OMB excuse came up repeatedly from ALSA as those of us anxious to know how things were going asked questions.  The portal was clunky by 2010 standards.  We wondered how many people with ALS were finding it and entering their data. Were there barriers that needed to be addressed?  Our questions weren't answered.

Just tell your stories and continue to ask for unsubstantiated millions of dollars annually.  We were good soldiers.  We trusted.  We believed.  We didn't ask enough questions (not that they would have been answered anyway).

We weren't to get any until 2014.  Keep being good advocates and ask for the annual appropriations you're told to request.

The Accidental Discovery

In June, 2013, I ran across something at usaspending.gov quite by accident.  There were some large contracts handed (no-bid as the sole source for the services) to The ALS Association for "education and outreach" for the Registry. Surely this wasn't what it appeared to be!  It was.  The organization that works with the CDC and tells us the appropriation needed annually was actually a large contractor on this very project.  And we had never been told.

https://docs.google.com/file/d/0B0yxxVCJLk5lOElWR2g0S2VvVzg/edit

The Concern

We were dumbfounded that ALSA was being paid to do what we thought our donations were already paying them to do -- to raise awareness of the Registry and to promote patient self-enrollment.  And substantial taxpayer funds were being directed to ALSA without transparency or any competitive bidding.  They failed to tell us!  That's a pretty important tidbit since they are the ones who tell us advocates the grand sum to request (without any businesslike accounting of details) on Capitol Hill every year.

Add the fact that ALSA and MDA are the only ones allowed to nominate patient participants for  the Registry annual meeting.  It looked bad.  It smelled bad.  It was way too cozy for good oversight.

With a little more digging, we found that the MDA was about to be handed a no-bid contract of its own.  https://govtribe.com/project/als-outreach-and-education-activities-to-support-the-national-als-registry-2   Two sole sources?  What was going on here?  The phrase "turn at the trough" came to mind.

The FOIA Request

I asked the CDC ATSDR ALS Registry administrator what work product, deliverables, and work standards were on those no-bid contracts. What were ALSA and MDA actually doing for those large sums?  The response was that I would have to file a Freedom of Information Act request.  What was a simple question took us on a long journey.

The FOIA requests I submitted in 2013 were simply for the work product, deliverables, and work standards for two of the contracts -- one for ALSA and one for the MDA.

And we waited.


The Wait

Fortunately I'm persistent and healthy (knock on wood).  For over two years I followed up on the status  of the requests.  The CDC FOIA office form response was that these things can take years.  Two of my friends with ALS who helped with the FOIA request died during the wait.

During the course of the wait, the first report from the ALS Registry was finally published in 2014, and it was certainly disappointing in substance.  It raised a lot of questions about the completeness of the Registry.  It raised a lot of concerns about self-enrollment.  It yielded numbers that the very organizations that got big contracts on the projects don't seem to want to use, yet they continue to pat themselves on the back.

Any oversight on this project seems to be done by people with substantial financial interests in calling the project a success.  Just tell your stories, advocates.  There are always new, naive ALS advocates to take the places of those who start to ask questions like my buddies who died waiting for the FOIA requests.  The annual meeting patient representatives are hand-picked by the contractors ALSA and MDA.  And we've never seen the budget discussed in the minutes of those annual meetings.

And we continued to wait.

Finally, Two Contracts

In November 2015, after another status inquiry, a large envelope appeared in my mailbox.  It contained a letter explaining some of information withheld along with a CD with two files.  A CD.  Who still has a computer with a CD drive?  Oh, well, I'm persistent.   Here they are: an ALSA contract and an MDA contract.  Both have information redacted, but there's plenty there to substantiate concerns about these sweet deals.


ALSA's Deal
.
The first contract was signed on September 19, 2011  Keep in mind that the Registry had been promoted by ALSA for years before that and that the online portal for people to self-enroll had been opened in 2010.

The contract purpose is pretty a pretty vague "to promote the National ALS Registry through an exptensive nationawide network of ALS Clinics, Chapters, and Certified Centers of Excellence."

The "technical requirements" provide us with some deliverables that are things and tasks (and not  Registry results).  ALSA will fulfill their responsibilities by doing the things many of us thought they should do as part of their mission that we already support with donations.  They are being paid to do tasks but there is no linkage to any results in increasing or even maintaining self-enrollment in the Registry.  It's a pretty sweet deal, especially when you don't have to bid. And it amounted at that time to over $1.5 million over five years.  But wait, there's more!

Less than two weeks after both parties signed the original contract, it was modified to an even bigger, sweeter deal.  The amount was increased to a redacted amount.  ALSA got the leeway to expand Registry education and outreach work to MLB, the NFL, MLS. And that has increased self-enrollment how?   Again, there was still no tie or performance expectation to maintain or increase self-enrollment in the Registry.

And the seeds of scope creep were planted.

And in this bigger, better version of the contract, the monthly compliance-reporting requirement in the first contract was suddenly changed to, "To be discussed at Kick-off Meeting."  OK, who was at that rather important Kick-off Meeting?

In both editions of the contract, there is a clear obligation that a contractor may not lobby for funding:

And we who go to the ALSA Advocacy Conference or who are online advocates hear about how great the Registry is from ALSA.  ALSA employees tell us to ask our legislators for another $10 million.  I suspect that through an intricate tapdance of who is on whose meter while talking to advocates, ALSA managed to fulfill the letter of that clause while defying its spirit.

There is also a requirement: "4.  Educate and train Contractor's local chapters' staff to identify ALS patients, increase awareness about the National ALS Registry, and if necessary, provide assistance in registry enrollment.  Contractor's chapter staff will not recruit ALS patients for the registry and will only assist with registration for those patients who request such assistance."  Read that paragraph a few times and scratch your head.

There are pages and pages in that pdf that was on the CD, and it's not easy reading for a lot of reasons.  By the time you plow to the end, you see a contract that somehow grew to $2.2 million.

A copy of some pages of the contract with my comments is here.  I will call them lowlights rather than highlights.

MDA's Deal

The MDA no-bid contract was dated September 12, 2013, two years after ALSA had received its no-bid contract.  The amount is a whopping $2,695,642 over five years.  It has another interesting clause:  "The contractor chosen must have direct and proprietary access to a high percentage of ALS patients in the United States."  Chosen?  Proprietary access?

In the base year MDA was to produce a Registry toolkit whose requirements are remarkably like we just paid ALSA to do.  Why not just use ALSA's, folks?

MDA got the same missive as ALSA to supply "relevant equipment" to district offices, clinics, and support groups help promote the registry.  There is no standard for "relevant equipment" nor is there any accountability.  That's kind of hard to swallow when we see how hard people with ALS have had to fight government rules to supply them basic communication technology.

MDA is also being paid to educate its own "stakeholders" about the differences between the CDC's ALS Registry and other ALS-related registries.  You see, MDA launched its own registry after the CDC's launched.  Now we pay the launcher to clear that up among its own "stakeholders."  Perhaps they can enlighten us on comparative data at the annual CDC Registry meeting sometime.

And maybe at the next CDC Registry meeting each invitee will introduce herself or himself as a project contractor, if appropriate.  And perhaps the patient and caregiver representatives will tell us which ALS organization nominated them for participation.

Our Deal

We got the dirty deal.  We got a national Registry with significant design flaws.  We got poor execution.  We got insider oversight and no accountability.  We got a project whose scope was allowed to creep to fit a very generous, unsubstantiated budget.  We got a project that continues to grow in search of things it might do well.  We got a cozy, secretive relationship that defies the spirit of government rules that prohibit agencies and contractors lobbying for funds.  We got a dirty deal.  People with ALS were fleeced.  We taxpayer-advocates were fleeced.  Or loofaed.

Driving Ahead, Leadership, Risk, and the Importance of the Rear View Mirror

Last week I attended some of the ALS TDI Leadership Summit events.  It is clearly the most
welcoming conference for people with ALS and caregivers who are interested in science. Access to a variety of scientists, especially during break time and lunch, was outstanding.  Many of the conversations were candid and unfiltered, and that's a refreshing concept in the fight against ALS. Some miscellaneous impressions follow.

• In the last decade it seems like we've gone through a full philosophical cycle -- From trying to understand a complicated disease to trying to throw things at it regardless of the rationale (just put out the fire) back to trying to understand the many things going on with ALS (figure out if it's a grease fire or an electrical fire).
• The people with the most at stake in the fight against ALS don't have the benefit of a lot of institutional history.  Those with ALS and their loved ones are largely new at it.  Few with ALS live long enough to remember all that had been tried in the past, and organizations are not good at keeping the loved ones engaged for the long-term.  This provides some convenient blind spots when some of the science is explained.  
• Many promising drugs have been screened and tested over the last decade, and there is an ever-growing dud pool.  Knowing about the volume and variety of duds is helpful to put the difficult road ahead into perspective.  And we have to be realistic that today's "promising" may well end up in the dud pool.  That doesn't mean stop.  It means that we need to smarten up and shorten the route.  
• Fundraising is essential, and it's a lot easier to raise funds based on "promising" than it is looking at a rear-view mirror full of expensive duds.  Are we distorting the difficult road ahead by doing what's best for fundraising?  
• People with ALS want to assume more risk, yet the scientists and clinicians aren't likely to step forward and lead that fight (or even lock arms with them as one man with ALS suggested).  I'm more convinced than ever that patient-led movements will cause the change in this area.  Organizations simply won't take the professional, public risks to be that bold in a heavily regulated field.
• Having a gentleman with professional and academic chops on the afternoon panel was priceless.  The lithium story is one that has been subject to a lot of revisionist history.  Sometimes the establishment tries to tell that story in a way that makes the patients look gullible.  We had a person with ALS remind the panelists that the patients were the ones who showed that lithium didn't work.  People with ALS were the ones who begged everyone to stop wasting money on trials.  They should be respected for what they did.
• We heard that small ALS trials are largely destined to fail. It sounds more credible when panelists say it.  Some people with ALS have been saying it for years.  We even heard that at one time some clinicians wanted a very small lithium trial destined to fail to make that problem go away.  There was some refreshing candor from Dr. Katz about the hype of some clinical trials.  At the end of that discussion on trial design, some of us in the audience had a serious question as to whether there is anything currently worth the investment in a clinical trial?  And I wondered what would happen to fundraising today if an organization would honestly say that there is nothing currently worth that investment?  

Depressing?  Not at all if you acknowledge the rear-view mirror and decide to take some new roads forward.   We need to learn not only from the duds but also from slow, inefficient processes.  

The father of a remarkable young woman with ALS asked if we are letting the perfect get in the way of the good.  That sums it up.  We need to forge ahead with admission of what's in the mirror and a willingness to take risk ourselves in many new ways so that people with ALS can take the risks that they accept (with eyes wide open) in order to move the science ahead faster.

You Wouldn't Think Of Letting That Elevator Door Close

Especially when the waiting person is in a wheelchair.

Yet we close the door in the faces of those with ALS all the time.

Here's an example from twitter today.

It was an interesting article on the virus theory that has been in the news lately.  A woman with ALS responds below.  She's interested in the trial cited in the article.  The article failed to contain trial contact information for those who actually have ALS.

On a long shot, I searched clinicaltrials.gov.  Odds were that I wouldn't have the right keyword for the products involved and wouldn't find it.  I got lucky and found it.

Everything is hard for those with ALS.  Life would be so much better for them (and quite frankly, trials would be filled much sooner) if people would simply make sure that they're holding the door open for those who don't need more barriers in their lives.

After all, they are why you're doing the science that is published in the first place.

It's Much More Than A Game

I live in an NFL city that has a team that set high expectations and has performed terribly this year.

Everybody has an opinion about the problem --

The GM is the problem.
The coach is the problem.
The offensive coordinator is the problem.
The QB is the problem.

And with every assessment of "the problem," there is an expected action that someone will be let go and changes will be made.

I live in a world of ALS that has teams that have set high expectations and have yet to deliver a big win.

It's much more than a game.

Do we ever assess "the problem" and take expected actions?

Ever?

There Really Is Educational TV

Last night Charlie Rose featured a segment with Dr. Collins of the NIH and then another with PALS Jay Fishman and Dr. Rothstein.  It was late.  After a few hours of sleep, a few very random thoughts are stuck in my mind.

• NIH funding is precious.
• Why were NIH funds needed for this new report?  ATSDR has generous funding for the Registry.
• The delay to diagnosis for ALS is outrageous.  Charlie Rose grasped it.  Jay Fishman lived it.  Dr. Rothstein didn't want to cast aspersions on the fellow physicians who fail to diagnose it. That delay to diagnosis is costly in many ways, and the patient is the one who pays the price, yet we give physicians a professional free pass when they miss it, and they often miss it.
• The ALS Ice Bucket Challenge missed opportunities.
• We need better accounting and accountability in the fight against ALS.
• Money won't cure a disease that is short on new ideas.
• The cure isn't "around the corner."  I took that to mean that Dr. Rothstein has no impending  treatment for Mr. Fishman.  I appreciated the candor.
• Too bad the conversation didn't then jump on shortening "the corner" by getting experimental drugs to PALS much more quickly.
• Charlie Rose is on too late in my city. 
• Jay Fishman has an attitude that reminds me of my mother when she faced ALS.

We need to have more serious conversations and more action to change the status quo.  Thanks, Mr. Fishman.

What Would You Fight For? This!

I was more than pleased to receive the email below.

To have neuroscience students interested in this beast of a mystery disease is so important! And those of us fortunate enough to be graduates of the University of Notre Dame have a big added dose of joy to see them raising awareness, research funds, and their prayers to fight ALS.  It is a special place and so many alumni, family members, and friends of the school (and its rivals) have been lost to ALS.

Please participate.  The campus is gorgeous in the fall and is not far from a lot of major cities in the midwest.  And the t-shirts are awesome.  Please support these students.  Thank you.  Go Irish!  Beat ALS.

Hi!  My name is Chris Ferari and I am a junior at the University of Notre Dame as well as one of the officers of the new Neuroscience Club on campus.  I am very excited to announce that this year the Neuroscience Club will be hosting the 1st Annual Notre Dame ALS Walk, co-sponsored by the College of Science!  This scenic approx. 2.7 mile walk around campus will begin and end at Jordan Hall of Science and include a short group prayer in the grotto.  Here are the details of the event:
 When: October 31st, 9-11 AM
Where: Across campus, beginning and ending at Jordan Hall of Science
Who: Notre Dame students, faculty, and friends

• -Proceeds benefit ALS Research and Programs at Notre Dame
• -Bagels, muffins, and coffee will be served before the walk!
• -Costumes are encouraged!
• -Notre Dame ALS Walk t-shirt guaranteed for the first 300 people to sign-up
• -Sign-up is $10 per person and additional donations are encouraged!
• -Sign-up and donate by following the link below:
• https://shop.nd.edu/C21688_ustores/web/store_cat.jsp?STOREID=8&CATID=725&SINGLESTORE=true

 We are incredibly excited about starting this walk on campus, and we hope that it can become a yearly opportunity for the Notre Dame community to raise money for a damaging neurological disorder that has affected many members of the Notre Dame family.  Please consider donating to or attending this special event!  Thanks in advance for your support!
 Go Irish!!!
Chris Ferari

We Need To Flip The Orbits

Often I wonder how some ALS organization employees or board members can be so out of touch.  Are they not paying attention, especially to issues and concerns that are so apparent to people with ALS and caregivers and advocates on the internet?

We have a kind of solar system where organizations have placed themselves the shining stars of the internet, providing messaging and resources for those who orbit their sites.  They build.  People with ALS and caregivers come to orbit them.  They occasionally stave off rogue planets that appear to go astray.  The star shines at the center of its solar system and can burn off stray matter.

Who knows what those organizations and board members could be if they revolved online around people with ALS and caregivers for a change.  You see, it's about PALS and CALS. With the tools that today's internet provides, we should all be orbiting the thoughts and needs of people with ALS and their caregivers.  They really are the stars.  Everyone should get in touch with that concept.  Orbit them.  Get in touch.  

Please, Stop The Information Waste!

People with ALS try things.  It's a fact.  Some things are pretty innocuous and other things are more complicated.

Families rely a lot on Dr. Google to find things to try.  They search.  They try to sort thought it all.  They are fighting a terribly fast clock.  The information they seek is in stunning disarray.

Mom read that Vitamin E might help ALS.  I got her a bottle of liquid Centrum with Vitamin E at Walgreen.  Later that same day I read in My Luke and I by Eleanor Gehrig that they had tried Vitamin E.  We were doing the same #$%^&*  thing that Lou Gehrig tried in 1939.

The information gaps have us blindly trying the same things over and over.

We need to capture information.  We need to let the data speak.  PALS need to be encouraged to self-report their information in a public and organized and searchable manner.

PALS and CALS are vulnerable to hucksters, and often on the day of diagnosis they are warned of
predators who will want to replace their fillings or shoot some fat cells into them.  And new PALS and CALS are often frightened by their clinic staffs and ALS organizations on the perils of the internet.

This is so wrong.  It is so wasteful.  Instead why don't we encourage some productive use of the internet (beyond the miracle of fundraising)?

Let the data speak.  If everyone who had fillings popped out self-reported on results, it would give PALS information for more intelligent choices.  If everyone who tried Vitamin E self-reported on results, it would let PALS make more informed decisions.

Please, let the data speak.

Ironically, www.alsuntangled.com has been using self-reported data at www.patientslikeme.com where the data contributions from PALS seem to have dropped off severely in recent years.  The ALS establishment needs to encourage the self-reporting rather than frightening PALS away!

People with privacy concerns can be as obscure in their personal identities as they want.  A right-handed ballerina can call herself "Lefty" and post her data at patientslikeme.  I cite that website simply because it has a good data presentment dashboard for those with ALS.

The information waste we are experiencing today is insane.  It is wrong.  Self-reported data are not perfect, but with a fatal mystery disease with no good treatment options, the imperfect data are valuable.  To bury clues with PALS is just madness.

And perhaps it's time for all involved in the fight against ALS to ask PALS why they aren't self-reporting data today.

Let the data speak.  Let PALS make more informed decisions.  Give scientists a clue, too.  Please.  Information is a terrible thing to waste.

ALS, American Airlines, 6pm.com, La-Z-Boy, and YOU

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

	Information.  It’s the food we use to improve everything we do.  It’s the feast that drives innovation, yet we waste so much.
	Food deserts are a big problem in our urban areas.  I think that information deserts are a big problem in healthcare.  Do we find ourselves munching on what’s convenient rather than what’s really good for us?
	People dealing with ALS have a bumper crop of information that is often left to wilt on the vine.  That's an unacceptable waste, especially with a fatal mystery disease.
	You see, the ice didn’t cure ALS.  There is still no known cause.  There is no effective treatment.  It is a difficult, steep, downhill path to death.
	Yet everyone in this room, regardless of what your role is in medicine, can benefit from information from those dealing with ALS – you, physicians; you, drug developers; you, entrepreneurs; you, smart students.  Let’s talk a little about harvesting that information.
	Before Mom died from ALS, she liked to take a trip every now and then.  American Airlines retained more information about her travel history than medical science retained about her case of ALS.  That’s outrageous.  All the clues that her life and medical history harbored were buried with her.  We’ll never connect the dots if we don’t start collecting the dots in an orderly and complete manner.  Somebody, call American Airlines, please!
	Shopping for shoes is challenging for me, but not nearly as challenging as shopping for an ALS clinical trial.  Often when someone is diagnosed, a family member is put in charge of Dr. Google to go find something.
	You hope that “something” is a clinical trial.  It advances the science and is a legitimate Hail Mary for someone with ALS, but trials are not easy to find.  You have to sift through enrolling interventional trials to try to find the most “promising” science.  And you get emails out of the blue.  It’s confusing.  It’s time consuming.
	And speaking of time, most ALS trials have a 24-month eligibility window from onset of symptoms.  There is typically a 12-month delay-to-diagnosis with this disease.  See a problem with time, here?
	Last week I got an email from 6pm.com, Zappo’s outlet store – “The perfect shoes for you, and on sale!”  I took the bait.  I clicked on the message.  Up popped a cute pair of shoes that looked like they would work for me.  I’ve bought a total of three pairs of shoes in my entire life from 6pm.com, and they were able to make a pretty good match for me, yet clinical trial matching is stuck in the 1980s.  Somebody, call 6pm.com, please!
	Yesterday we talked a lot about precision medicine.  I think we need to talk about accurate medicine, too – the right intervention at the right place at the right time.  So often with ALS it looks more like this… We constantly throw the ball five yards behind the receiver.  That does not help the patient.  It’s wasteful.  It’s expensive.
	I have a wonderful old reading chair – a 50-year-old La-Z-Boy.  It is so comfortable.  Last winter it broke.  It was stuck in the upright and locked position.  I was heartbroken.  I called my local La-Z-Boy store and a fellow in the warehouse was able to diagnose the problem over the phone – a broken part.  Oh, no.  He said not to worry.  They would find one. I could pick it up after noon the next day.  Really?  Try breaking a part on a power wheel chair that is far more important than my reading chair.  You get a shrug and , “Four to six weeks, what can you do…”
	I’ll tell you what you can do.  You can and you must measure.  It’s about information.  You measure performance.  You set benchmarks.  You improve processes.  Somebody, call La-Z-Boy, please!
	I have a challenge for you all and it doesn’t involve ice.  Please consider ALS to be a stress test for whatever it is you do in medicine.
	You’ll never become a great skier if you stay on the bunny slopes, and trust me, ALS is no bunny slope.  It’s a difficult disease.  It’s a demanding disease.  It’s often a very fast disease.
	If you can learn from those dealing with ALS, you will improve.  You will innovate.  And I guarantee you will be better for every patient you ever serve.
Copyright 2015 @ALSadvocacy All rights reserved.

Terms You Never Hear In The Same Sentence -- Nimble, Information, ALS

Several years ago, a local investigative reporter did a segment on lead in dishes and children's health. Our Board of Health offered free testing of dinnerware.  Lead is never good.

I took one of my everyday dishes to the testing site and brought along one of Mom's dishes that she and our family used every day for half a century.  Hers was Franciscanware, a high-quality, everyday china that millions of housewives of the 1950s treasured for their families.  The patterns each family had were expressions of mothers' favorite colors and themes.

When I had the china tested, I expected that my everyday china that I had bought on sale at Marshall's would test high and I would toss it.  Lead is never good.

I also expected that the Franciscanware would be deemed safe.  What a surprise I got.  Yes, my inexpensive everyday china tested high, but the Franciscanware tested even higher.  Yikes.  Lead is never good, and if you've ever seen ALS, you have no desire to take any potential chances with any substance that can possibly mess with your neurons.

On a recent webinar on possible causes of ALS, I asked if anyone had ever studied the lead content of   the dishes that people with ALS had used.  I didn't get a direct reply to my question.  The scientist just said, "Lead is never good."

ALS is a fatal mystery disease, yet we have no nimble systems for gathering and reporting on possible risk factors or causative factors.  The studies take years and the information-gathering nets are not cast widely (quite possible because the studies take years and the disease often kills people quickly).

We need nimble.  We need complete and organized information.  This is not rocket science.  We need ways to gather and harvest information quickly.  What if there are some simple preventive measures we can take in our everyday lives?  This is a matter of life and death.

Lead is never good.  In the meantime, I've tossed all the dishes.  I've seen ALS and it is never good, either.

Is It Wrong To Yell "Fire Extinguisher..."

...in a crowded, burning theatre where the doors are all locked?

Of course not if you have a fire extinguisher in your hands and are trying to clear a path to help.

It would be very wrong if you have a great blueprint for an awesome  extinguisher (that you consider to be a "breakthrough" in snuffing fires)  and a successful experiment and a patent application but have yet to go through rigorous testing, certification, approvals, pilot production, or production.  You would set a really cruel expectation amidst a lot of people facing death.

Last week was an interesting one in the fight against ALS.  We had already seen the news published August 7 about TDP-43 in Science https://www.sciencemag.org/content/349/6248/650.abstract

That was certainly another good scientific step forward.

In a tenuous attempt to link the scientific good news to last August's ALS Ice Bucket Challenge, we started getting crafty statements.   What better way to encourage donations this year than to associate it with a recent advance in understanding ALS?

Media coverage was being cultivated, and this makes a feel-good story about the marvelous, fun phenomenon of August, 2014.  It makes a wonderful feel-good story.

Then there was the Reddit AMA exchange with one of the paper's authors followed by some national interviews.  A tie of the #ALSicebucketchallenge to this project was emphasized.  Wow.  We did that with the ice, eh?  A young scientist almost seemed giddy with enthusiasm about the significance of his discovery.  Double-wow.  And this was emanating from one of the finest academic medical institutions in the nation, Johns Hopkins.

As Answer ALS aptly described in a statement on Facebook, "The headline seen this evening had the phrase 'A Cure for ALS Could Be Coming Soon.'  As the last few days have gone on, the headlines seemed to create themselves... "

And for families craving optimistic sound-bites as they deal with ALS, were they ever getting the optimistic sound-bites, right from the scientist's mouth.

And the ecstatic news went on and on.  Mission accomplished?

Perspective, anyone?

• When did this work start?
• How much did the project cost and exactly who supplied what funding? 
• What are the next steps?
• How much money is needed for the next steps?
• What is the most optimistic timeline for the next steps?
• What are the barriers (scientific or regulatory) to bringing this finding to a therapy?

That clarity and perspective will help us all to understand not only the significance of the findings but also what we all need to do to help bring those headlines to reality.  

Maybe we need to talk about patient-centric p.r. and fundraising.  A respected medical institution needs to celebrate its achievements and raise funds with respect for the patients and caregivers and loved ones who are trapped in a burning theatre.  They just heard "fire extinguisher" on the national news last week.

Say What?

It started on August 13 with a tweet from an ALSA executive, "#ALSIceBucketChallenge donations have led to a significant ALS research discovery! http://bit.ly/1DQByYZ  "

Wow.  Impressive.  Holy cow.  That was fast.  Say what?

The press release was very carefully couched and didn't quite mesh with the cause-and-effect excitement of the tweet, but the seed was planted.

Then it continued with organizational tweets with wording that matched exactly.

Amazing. Remarkable. Cool.  Say what?

Then yesterday there was the ALSA mass emailing -- Subject line: "This is amazing news!"

Yes, amazing.  Fabulous. Incredible.  Say what?

The cause-and-effect implication is clear.  IBC donations to ALSA led to this discovery?  That's fast science.  How refreshing.  Say what?

We didn't get a good list of  #ALSicebucketchallenge checks written to researchers last year. We saw numbers related to committed funds.  In these lists of funded research, I don't see this project listed in the 2014 file but Dr. Wong is listed as a $160,000 grant recipient in 2015. 

The full research paper on this "significant #ALS research discovery" sits behind a paywall.  My curiosity won last night.  I bought access. Let's see how this breakthrough was funded with IBC funds, was discovered, was written up, was reviewed, and was submitted to a journal between August. 2014, and March, 2015.  That's cool fast science.  Say what?

Acknowledgements at the end of the paper included that the project was funded in part by

• The Robert Packard Center for ALS Research
• Muscular Dystrophy Association
• The Amyotrophic Lateral Sclerosis Association
• Target ALS
• The Johns Hopkins University Neuropathology Pelda Fund
• The Johns Hopkins University Alzheimer's Research Center (NIH P50AG05146)
• The Samuel I. Newhouse Foundation

Unfortunately we don't know how many dollars were involved or how much each agency contributed or the dates of the grants.

The paper itself is complicated and involved experiments and evaluations on mice followed by more work on deceased PALS' brains.  There isn't a timeline but it would have taken a miraculous change in the ALS research paradigm for any substantial amount of this discovery work to have been carried out in Q4 of 2014.  I would be amazed if the illustrations alone could have been made and approved in six months, let alone all of the underlying work.

And there was even mention in the paper of a patent application filed by two of the authors. 

So the "Say what?" response keeps going off when we read the tweets and the emails.  How can this be?  Is it a deceitful way to make people feel good about their last year's IBC generosity so they'll do it again?  Or was there really a scientific funding-to-discovery-to-journal miracle in six months?

People with ALS and their caregivers have been pressing hard for the FDA to speed up approvals. They get pushback from organizations including ALSA that good science takes a long time.  Then we get self-serving fundraising messages that our donations led to a "breakthrough" in under six months in some remarkable fast science.  Say what?

People are dying.  They deserve some clarity.

If my "Say what?" reaction was out of line, I'm all ears.

"Do You Know What The Worst Part Is?"

#whatwouldyougive

That's a movement that originated with @speed4sarah.  You can read up on it on her blog http://www.speed4sarah.com/sarahs-blog/

I decided to give it a try.  I would try to give up something that ALS takes from people.  It would raise some awareness.  It would raise some funds.

One thing that really stuck with me when Mom had ALS is that we take so much for granted -- taking a sip of water without choking, working a door lock, talking to one another.  There was such a spectrum of things that I could "give up" for #whatwouldyougive.

Mom still had good grip strength during her ALS but it was terribly difficult for her to lose the dexterity in her fingers.  The inability to work a lamp switch or button a button was so frustrating for her.  More on that later.

I set my #whatwouldyougive bar very low.  I didn't want to fail.  My left hand in a ski mitten  for a day shouldn't be that tough.  I'm right-handed.  I picked a weekend day when I wouldn't have to do my day job. I didn't want to have to ask anyone for help, so I kept it simple.  One free hand would work.  I would be safe driving.  The bar was so low that I felt like a sissy.

Enter Sunday morning.  I put the mitten on first thing.  Immediately I realized I couldn't scratch the dog like she expects every morning.  Not a big deal, right?  On to fix my toast and tea.  It wasn't pretty when I realized that I could not slice my toast the way I've sliced my toast since I was six years old and learned to make my own toast.  Thirty minutes into my experiment, I said, "Boy, I'll be glad when this day is over."  Ninety minutes in, I was totally frustrated.  I wanted my old life back where I could slice my toast my way.

I've broken my right wrist twice in my life and I was never this frustrated (there's that word again).  I decided that immobilization feels good when something hurts and there is healing going on.  That's not how this was.  It was just exasperating.  I couldn't clean my eyeglasses.  I couldn't floss.  My right arm itched.  It was incredibly frustrating.

For my sanity, I decided to try some distraction and went to the grocery store.  It went well until I needed a produce bag.  Ah, plastic bags were my bane all day, at the store, in the wastebasket, on the dog walk.

By late morning I was really hungry and had no desire to frustrate myself more by cooking.  I stopped in a local restaurant that was still serving breakfast.  It was one of the best experiences of the day.  We talked about the mitten and the hashtag and they fixed me a good breakfast that required no knife dexterity.

I had been making a list of the day's challenges and finally stopped listing them.  We take so much for granted.

The first time Mom asked me to help her button her blouse, she asked, "Do you know what the worst part is?"

No, I didn't.

"When I've lost the ability to do something, I know I'll never do it again."

She had buttoned her last button.

At the end of my experiment I took off my mitten.  My life goes on.  My toast is right this morning.  And how many mothers with ALS buttoned their last buttons yesterday?

_____

Please set a challenge for yourself and try it.  Low bars are fine.  Trust me, it's not easy.  And then support https://www.razoo.com/story/Sarah-Coglianese-Fundraising-For-Whatwouldyougive

Or give directly to @speed4sarah 's research cause at www.als.net 
Or give to www.teamgleason.org that supplies technology to those with ALS to help compensate for those abilities that ALS steals.
Or do all of the above.  After all, we take so much for granted.
Thanks.

Let Me In, Whee-Ooh

"Let Me In"
The Sensations - 1962
Let me in whee-ooh (whee-ooh, whee-ooh, hoop-whee-ooh) 
(Whee-ooh, whee-ooh, hoo-ooh-oop-whee-ooh, whee-ooh) 

I can see the dancin' (let me in) 
The silhouettes on the shade 
I hear the music (music), all the lovers on parade 
Open up (let me in), I wanna come in again 
I thought you were my friend 




The big ALS symposium is coming up in December.  Already we're seeing news of abstracts accepted and discoveries that will be presented.

The registration fee of approximately $600 goes up even more after August 19.  http://www.mndassociation.org/research/international-symposium/registration/

There are people with ALS and their loved ones who are interested in good science.  They spend countless hours searching.  A gentleman I knew with ALS attended the symposium in 2010 and found an interesting drug candidate and enrolled in the clinical trial as soon as it was available.

Why would we not want people with ALS or their loved ones at such a symposium?

Why would there be so many barriers between PALS and this festival of information about good science?

The fee without any consideration for people with ALS and caregivers is one of the big barriers.

The lack of welcoming information for those who aren't insiders is another.

The spirit and letter of the Patients Included movement needs to be in the fabric of every ALS meeting, especially the premier scientific symposium.  It's past time for this meeting to be physically and virtually accessible to every PALS or loved one who is interested.

Of the PALS, By the PALS, For the PALS

Action.

Traction.

It's happening thanks to some formidable people with ALS and caregivers.  They aren't waiting for a system that doesn't understand urgency.

Here is a posting from a gentleman with ALS on the forum (it requires registration to view) at als.net:

Rich Casey, the CEO of Neuraltus, mentioned that ALSA is considering funding an Expanded Access (Compassionate Use) program for NP001, in addition to its $1.5M grant for a new clinical trial. As you know, Phase II clinical trial of NP001 yielded promising results in 2012, though falling short of its effectiveness goals. However, post hoc analysis identified "responder" biomarkers. Neuraltus has been trying to secure more clinical-trial funding ever since. Now ALSA expressed interest in underwriting a Compassionate Use program for NP001, along with the trial. Neuraltus believes there is an immense interest in the ALS community for this idea, but would like to have concrete evidence. Having a ready list of potential participants would help this cause, demonstrating the unmet need to ALSA as the sponsor. If you are interested, send a letter to Neuraltus requesting compassionate use access to NP001. This would strengthen Neuraltus's hand in dealing with the FDA as well as ALSA. We also should emphasize that the drug should not be withdrawn from those it helps, to prevent " crashes", like those experienced by Persevering and others during the wash-out period of the 2012 trial.

To: Rich Casey rcasey@neuraltus.com
Below is a  bare-bones sample letter to sign and send, but it would be good include your personal information and why you need NP001. 
Thank you. 

Dear Mr. Rich Casey,
As a person with ALS I want to join the Compassionate Use program for NP001. I am aware that the upcoming clinical trial would have only a limited enrollment. A large Expanded Access arm would benefit ALS research and help PALS. It is also important that long-term access to the drug should be ensured for those participants that benefit from it. Sincerely,
Xxxxxxxx Yyyyyyyyyyy

There are a number of threads on the forum at www.patientslikeme.com that discuss NP001.  I urge any PALS, CALS interested to read them.  It definitely did something in some PALS in the Phase 2 trial.  Some also crashed terribly when the drug was taken away.

Here is an article from the WSJ regarding one of the PALS who was in the last Phase 2 trial in 2012:
http://on.wsj.com/1w4lvRl

And finally, here is a powerful letter that one of the Phase 2 volunteers just wrote to Mr. Casey:

Dear Mr. Rich Casey,
As a person with ALS I want to join the Compassionate Use program for NP001. I was in the NP001 Phase II trial and received the high dose. NP001 stopped my ALS dead in it's tracks during the time I was on it. I know first hand that NP001 is a safe and effective treatment for ALS patients. I am aware that the upcoming clinical trial would have only a limited enrollment. A large Expanded Access arm would benefit ALS research and help PALS. It is also important that long-term access to the drug should be ensured for those participants that benefit from it. Thank you for the opportunity to provide NP001 to the broader ALS community, I know it will save many lives.
Kindest regards,

Certainly an expanded access program isn't ideal, but it is something.  This potential therapy for some PALS (elevated IL-18 was cited as being characteristic in responders) has sat on the shelf for almost three years while the drug developer has not been able to raise the capital needed for a Phase 3 trial.  The system is broken.  Why scientists have not been more curious about what was happening with this drug is beyond me.

People with ALS and their caregivers are taking matters into their own hands and trying to open up some options.  They are demanding and getting attention.

I encourage all PALS, CALS who are at all interested in trying to email Mr. Casey now at rcasey@neuraltus.com .