A Picture Is Worth A Thousand Words

A thousand pictures are worth a million words.

Here's an idea for an ALS organization.

1. Buy a bunch of inexpensive 3x5 frames at Michael's or Hobby Lobby or some emporium with lots of inexpensive frames.  Buy a model that is pretty standard because you'll probably need to buy more soon.

2.  Every time you meet with a new person with ALS, get a picture.  Print it and frame it and hang it on an office wall.

The wall will fill.  You will be amazed at the the people you will have worked with over the years. You will not forget them.  They will remind you and everyone who enters your office why you're there.

Keep going down the hallway and around the supply closet if need be.  If you fill up ever inch of wall space, perhaps it's time to reevaluate the fight.

Let's Have The Ultimate Hat Tip Competition

This probably won't happen, but Sleepy can still dream.

Dear ALS Organizations,


We're having a contest for you.  


A mystery donor has recently won $1,000,000 to invest in the fight against ALS.  Your help is needed in designing a diversified portfolio for that donor.  Here are the rules:

• Design a portfolio of recipients of the entire $1 million that will best serve the battle against ALS.
• Your organization may receive up to half of the funds.
• You must pick four other IRS-recognized charities to be recipients of the remaining funds.
• If yours is an umbrella organization, the four others may not be under your umbrella.

The spirit of this contest is to recognize that there are a lot of good organizations that can complement one another in the fight against ALS.  There are different research organizations.  There are social service organizations.  There are advocacy organizations.  There is so much to be done in the fight against ALS that one organization can't do it all.  There is so much to be done in research that a single approach surely isn't a wise investment.


Please design and enlighten us.

Think of it as the ultimate hat tip.

Unfortunately People Won't Pay To Sit To Defeat ALS

Many years ago I was at a meeting where an ALS fundraiser was being discussed.  It was to be a lovely gala in a beautiful location.  There would be delightful hors d'oeuvres, drinks, and desserts in an evening of dancing.  Something bothered me about raising funds that way for a disease that steals your ability to eat and drink and dance and dress yourself in party clothes.  I understand the realities of fundraising, but the ironies of celebrating a cause that way still bother me. 

Perhaps sometime we should have an new kind of fundraiser where healthy people would pay to sit for three hours in a wheelchair without moving.  There could be dinner fundraisers where hungry guests would dine on puree and assorted vintages of chilled Ensure.  Or maybe some olympic-style events where individuals lift others off the floor and back into a chair.  Ah, the gravity games. The possibilities are limitless.

The realities of fundraising are that certain kinds of events like walks and galas work.  It still bothers me.  ALS is a disease like no other.  Perhaps it needs some new ways to raise funds and attention like no other.

Perhaps.

This Is It

May 31, 2012, the last day of ALS Awareness Month in the U.S.

We watch and listen for the noise and flash and splash of a grand finale.

I'm Leaving It All Up To You

Dear ALS Charitable Organizations,

Are you diversified or simply fragmented?

You decide.

Please grow up.

We Have A Simple Pizza Problem

We need more pizza to feed the team.  All the players are hungry.

Which team is more likely to win?

A. The one whose players plot and scheme to grab a bigger or extra piece of the pie?

B. The one whose players figure out a way to order a larger pizza?

Q and Plop

Sleepy asks a lot of questions.  It's genetic.  They are not meant to be irritating.  Sleepy likes to understand.

Lately we have seen hints of what may well be the largest expanded access program for an ALS drug candidate and we have seen many suggestions for FDA legislation and government funding.  We continue to have a large U.S. ALS Registry project with a dearth of status information.  Talk about a mother lode of question material!

We have opportunities to enlighten one another and engage in discussion that will improve the fight, and there surely is a large amount of room for improvement in that fight.

As long as ALS organizations ask for people to follow, they should be willing to engage in some public conversations that will help people understand and contribute to the journey with their ideas.  Organizations don't have to offer perfect solutions, but they should be willing to discuss what may be imperfections so that they have informed constituents.

Questions aren't all bad, and Sleepy does not mean to seem dopey.

Can Sheep Really Inspire Change?

Sheep are so comfortable staying tight with the flock. When one strays a little, it doesn't take much to modify that behavior right back to the flock. You can even train them with a clicker or hand motions. They frighten easily and can be discouraged from the slightest independent streak. Sheep are easy prey and they stick close together. A border collie can keep even the largest flock focused and headed in the right direction. You shear the wool regularly and sheep are the renewable gift that keeps on giving.

But a bunch of sheep never cured a disease. They have never modified a paradigm. Sheep are not change agents. Sheep don't challenge and inspire. They're sheep. They give you wool and they are ultimately someone's dinner.

Are ALS advocacy programs designed so that only the good little sheep are embraced?

Take The Pledge

Dear "Preeminent ALS Organization,"

Please take the pledge to respond to all emails within 24 hours. Simply respond.

Why would you not respond?

I'm Ticked

To the FDA, time is a necessary resource needed to do its job to protect Americans from harm.

To a pharmaceutical company, time is money.

To a person with ALS, time is a thief, quickly stealing life.

The three clocks are not at hopelessly crossed odds. There are mutually beneficial solutions to the problem. The consequences are huge, but the problem itself isn't the biggest in the world.

Will Friedman's next op-ed be "Why America Can't Even Solve A Small Problem That Has Huge Consequences?"

It's Time To Wake Up!

Yesterday morning Sleepy received a mass emailing from an ALS organization that was a huge wake-up call. The organization is touting a two-year "study" that it is doing to locate ALS clusters worldwide using an online survey.

That's a very interesting and noble topic. Here's the claim --

Through the information obtained, we will create an extensive database and interactive online map that will identify all suspected or reported clusters, patterns and hotspots of the disease around the world.

Sleepy's no genius, but even Sleepy knows that it would take a remarkable and tremendously expensive effort to deliver on that claim. Today it's easy to write an online survey and map results. It's a lot harder to gather and interpret scientifically sound data that would be useful to scientists. That's one of the reasons why ALS advocates have been working for years to have an epidemiologically sound National ALS Registry at the CDC.

Sleepy's wake-up message for the day is for all patients with ALS in the United States to self-enroll in the U.S. National ALS Registry at
http://www.cdc.gov/als
...and after that if you choose to contribute personal information to any other surveys, be aware that "interesting" is not the same thing as "statistically significant."

There's Irony In Those Tweets

A tweet yesterday from an ALS organization...

Who does #ALS affect? How many people does it affect annually in the United States? Does it effect men more than women?http://ow.ly/8kMgq

I was anxious to click on the link to find the answers (which I don't believe we really have).

Now that's irony.

Perhaps 2012 Will Force Some New Dimensions to "ALS Advocacy"

There are some interesting things bubbling that may move ALS organizations to break out of their ruts.

1. There is an ethics issue that would be wrong to ignore.
2. We spend millions on an national U.S. ALS Registry, yet are patients really self-enrolling? Why not? Mission not accomplished.
3. Studies and organizations have touted ALS multidisciplinary clinics as providing clearly better outcomes for people with ALS, yet our veterans with service-related ALS are not provided with multidisciplinary clinics by the VA. What is wrong with this picture?
4. Newly diagnosed ALS patients report vastly different experiences in clinical trial recommendations from their neurologists. Some encourage, some discourage, some seem clueless.
5. ALS is still the best-kept-secret killer disease.

Sleepy won't expect miracles regarding number 5 (it's not new) but expects that the first four will boil to the point that our ALS organizations will need to step up and do some new things. Same old same old won't cut the mustard in 2012.

A Picture Is Worth A Thousand Disconnected Patient Files

Please take a look at this article (thanks to @paullikeme and @epatientdave for the tip) --

http://e-patients.net/archives/2011/11/kenneth-s-spriggs-do-it-yourself-electronic-health-record.html

Now imagine how constructive it would be for physicians to supply a similar template to all newly diagnosed ALS patients to fill in some history of symptoms, of health, and of environmental exposures.

It might even be therapeutic for people to write things down in a manner that could be shared and that could contribute to the set of clues that scientists and other patients need to deal with ALS.

Simple. Visual. Direct. Valuable. Four words that are seldom seen together when dealing with ALS.

Who Wants To Tell A Very Interesting Story With Numbers?

Dr. Google brings many people to this site.

So if Google brought you here and you happen to be a journalist interested in telling a very interesting story with numbers, please write yourself a note. A clinical trial by Neuraltus with the experimental drug NP001 has been providing some very interesting numbers reported by trial participants who post data at www.patientslikeme.com .

This morning Sleepy happened to watch this video with Lizzie O'Leary --

There is a story with numbers about ALS that will knock the socks off the subprime mortgage story if NP001 is something that might actually work to save hundreds of thousands of lives. There are data. There are some compelling faces to put on the story. There are patients in the trial noticing things happening... some good and some not so good. Anything good is a huge breakthrough with ALS. There are patients who didn't qualify for the trial who are trying to reverse-engineer the active ingredient so that they can try a do-it-yourself trial. There are business leaders who will have difficult decisions to make. There are regulators watching. This is a very interesting story with numbers and with people who can bring those numbers to life.

Who wants to tell an interesting story of numbers that are starting to speak?

It's A Project, For Pete's Sake!

Yesterday we saw many congratulatory messages on the first birthday of the launch of the CDC's ATSDR National ALS Registry. As Sleepy watched the electronic high fives and pats on the back, Sleepy also wondered in what other sector do you party one-year post-launch simply because it's one-year post-launch?

Launch+365 is normally a time for some hard looks at project milestones and accountability. It's one of the many spots on the project timelines to evaluate the project status with an eye toward continuous improvement. What is working? What isn't working? What adjustments are in order?

Yesterday stakeholders celebrated. There's no reason to break our arms patting ourselves on the back yet. So far there have been no deliverables of epidemiological data from the project. There is an apparent low patient uptake on the supplemental surveys. Sleepy hopes that while the celebratory messages were strewn yesterday that somebody was working on the project milestones and adjustments to get the right deliverables to the scientists who can use the information.

Perhaps it's the nature of government work to need to sing for next year's supper. Sleepy would rather it had been a song about project milestones and adjustments rather than "Happy Birthday."

Be Careful

Those were my words to a man with ALS as we parted, having discussed some patients' do-it-yourself trial of an experimental treatment. The look on his face made me add, "I realize that the 'careful' bar is a lot lower when you have ALS."

Bright patients have researched and with neither multi-million-dollar budgets nor a cadre of scientists and clinicians, they have decided to roll some dice. They know that the risks are there, but the risk of not doing anything is clear, too.

We watch and we pray that they do no harm and perhaps will find something that will help... or at least will provide some clues for scientists and other patients.

http://sites.google.com/site/alschlorite/

The speed of research and clinical trials and approvals and healthcare delivery can't come close to the speed of ALS. We must fix that!

ALS and Drug Shortages

With serious drug shortages in the news, we have a new tagline --

ALS has the ultimate drug shortage. There are none.

You Asked For My Phone Number, You Asked For My Email Address

Why was that, Dr. Clark?

Car salespeople touch base with even their most infrequent customers when there is a rebate that they would like to know about.

Bankers and brokers touch base with even their most impecunious customers when there is a policy change that they need to know about.

Shoot, our local ice cream stand reaches out to its customers via email so that they know when to stock up for the winter with their favorite flavors lest they miss their chance.

Why don't doctors reach out to their patients as soon as a clinical trial is recruiting that may make sense? Why don't doctors actively call or email patients who may qualify for a newly approved device such as the diaphram pacer that was approved for people with yesterday. With ALS speeding through their patients' vitality at an incredible clip, why do patients not hear about things that may help until their next regularly scheduled appointments. Three months can be the better part of a lifetime for a lot of people with ALS.

Those patient phone numbers work for things other than billing departments. Sleepy wishes that docs would take the same initiative to take care of patients as my car dealer or the ice cream stand.

Whoa, The Government Is Moving Too Fast?

Or perhaps the government is moving without thinking past its fiefdoms.

https://filemanager.capwiz.com/filemanager/file-mgr/alsa/Senate_LHHS_ALS_Registry_FY12.pdf

"The Committee is particularly supportive of the addition of a biorepository component and the development of strategies that can enhance clinical trials and clinical trial enrollment."

It appears that the CDC's ATSDR, the "owner" of the important ALS Registry project, is ready, able, and willing to take on scope creep that will enhance its budget and footprint, and could ultimately take its eyes off of the core mission of providing complete epidemiological data on ALS cases.

In the past few months we have seen how broken the process of getting good clinical trial information to patients is. Having the ALS Registry do an expensive push of information to potential patients won't fix the root problems. It will only add another problem.

Can someone in our government step back and look at the bigger picture? Can someone in our trusted ALS organizations step back and take on a more difficult problem than appropriations for a Registry project? Can someone in our clinical healthcare delivery system take on some responsibility for getting information to patients in a consistent, timely manner?

It doesn't take a Six Sigma Black Belt to figure out that we have a very broken process and taping expensive band-aids on it won't fix it. Who will take on the problem statement (that may leave a little egg on the faces of some in the current process to get information to patients)? Anyone?

Here are just a few of the process concerns that have jumped out to me as I've watched patients take clinical trial recruitment into their own hands in order to fill the Neuraltus NP-001 clinical trial.

• Are patients relying on their neurologists for guidance on whether a clinical trial is a good idea and on which clinical trials hold the most "promise?"
• Are patients getting that information from their neurologists?
• Are members of our ALS organizations' staffs who deal with patients knowledgeable at least on the phase 2 and 3 clinical trials within 500 miles of their locations?
• Are patients and families being led down wrong paths by the proliferation of websites that say they have clinical trial information (but carry information that is incomplete or outdated)?
• Do patients and families know that the most dependable source of clinical trial information is www.clinicaltrials.gov?
• Has anyone talked to HHS about putting a nicer, more friendly gui on www.clinicaltrials.gov so that patients could do things like a zip code search for nearby clinical trials?
• Would a better gui on www.clinicaltrials.gov eliminate the need for redundant websites that are provided for a number of medical conditions?
• Do some neurologists discourage patients from clinical trials? Why?
• Can patients in clinical trials have a formal feedback process that would provide better trial design for the next drug candidates?
• Can the 24-month-from-onset "rule" be revised based on the results of the recent 36-month-from-onset trial?
• Does the CDC's Registry even have onset date (which would seem to be required for any kind of matching of candidates to clinical trials)?
• What are the results of the recently introduced NEALS/ALSA Clinical Trial Expert?

Many of us complain about the length of time that it takes the FDA to evaluate and approve new drugs; however, every day wasted in filling a clinical trial is as critical in the timeline as an FDA day. We definitely have a problem in getting good information to eligible patients in a manner in which they can make informed decisions quickly. To add an expensive feature to a Registry that has its own problems getting people to self-enroll isn't going to solve the problem. We need to step back and look at all of the possible factors and fix some of the problems at the root. Throwing Registry money at this problem will not fix a broken process.

My plumber can offer to fix a structural defect in my home, but that's probably not a good way to really fix the problem.