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."