ALS Advocacy

ALS Advocacy
Lou Gehrig's Disease - Motor Neuron Disease - Amyotrophic Lateral Sclerosis
Thought it had been cured by now? Still no known cause. Still no cure. Still quickly fatal. Still outrageous.

Thursday, January 31, 2013

Here Is A Request For Anyone Who Takes ALS Personally

Please help spread this link to friends, relatives, acquaintances, strangers, journalists, ...

http://www.youtube.com/watch?v=oxoBl5zZgNM&feature=youtu.be

Shelf life is short, and this is a compelling message on ALS.

This is raw ALS awareness.

We can all be pretty clever about multi-level marketing when it comes to fundraising.  This is easier and will have more impact if we can make ALS a gnawing concern on our nation's conscience.

Thanks, all.  Please, just do it.


This Is Leadership

http://www.nola.com/superbowl/index.ssf/2013/01/steve_gleason_gives_voice_to_a.html

“I believe that because ALS is under-funded and under-resourced many ALS patients end up fading away quietly and dying,” Gleason said. “For me, this was not okay. I did not want to fade away quietly. “

Thank heavens that Steve Gleason has the leadership abilities, the charisma, a great city, and a great team to bring ALS out of those out-of-sight, out-of-mind shadows. Super!

Monday, January 21, 2013

About To Be Served To Zillions Of Super Bowl Viewers

Here is the recipe for the greatest Super Bowl party dish ever --

ALS Awareness = O.J. Brigance + Steve Gleason

!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!!
(That's XLVII of them!)

Friday, January 18, 2013

Upon Further Review...

These days we take for granted that a football game will include numerous replays. Plays can be reviewed from every angle imaginable in order to get the calls right.

Would more or different eyes looking at clinical trial data in a different place at a different pace ever uncover some new or different insights?

Please treat people with ALS with the same respect that we treat a sport.  Please open their clinical trial data for use by scientists who might see something.

Wednesday, January 16, 2013

We Should Have The Mother Of All Pyramids Here

@team_gleason just tweeted, "Previous generations of pALS quietly faded into the shadows. This generation are not fading, quietly or otherwise. -SG  "
Every year in the United States alone there are around 6000 people diagnosed with ALS.  Suppose each has three loved ones.  Suppose each of those three carries on the fight against ALS after their beloved dies from ALS.  6000 Americans diagnosed per year * 3 loved ones * 73 years since Lou Gehrig's diagnosis = 1,314,000 potential Americans with ALS rage since the days of Lou Gehrig.

The United States only has 4.5% of the world's population.  We could have around 30 million people raising Cain about ALS globally.  Think of the attention they could get.

So where are all of these people who have been left in the wake of ALS?

A few need to "move on" after the difficulties of dealing with ALS.

Many more would be willing to stay involved in the fight if there were welcoming opportunities that fit their situations.  If not, they move on, too. 

Some become vested in ALS organizations and continue their work there.  That is valuable, but their focus often is on keeping organizations afloat rather than raising ALS awareness or rocking any boats.

In the last decade we have seen walks become major ways of drawing loved ones into the fundraising fight against ALS.  That has been impressive multi-level-marketing scheme; however, the retention of loved ones at walks after their people with ALS die is poor. Why?  As one once said, when the person with ALS is alive, others want to help, and fundraising is an easy way for them to do that.  That changes after the funeral.  Some walks aren't particularly welcoming to those left behind by ALS who may not be in a fundraising frame of mind, and that's unfortunate.

There are so many ways that have lost the powerful pyramid.  We need to embrace those who want to keep up the fight.  We need to recruit more voices of people who have lived the ALS nightmare.  They need to be welcomed to use their brains and offer their opinions.  We need our 30 million advocates and they need to know that it's time to make some waves.  We must not forget the staggering cumulative loss.

Thanks, @team_gleason for all you are doing to let our country and the world know of the outrageousness of ALS. Steve Gleason, you are an ALS rock star.  There are a few of us sticking around who have lost loved ones who would like to help you raise the noise level.  I was fortunate that Mom gave me some marching orders to get the word out about ALS.  Sixteen years later I won't let her or other PALS fade into shadows.

Tuesday, January 15, 2013

Which Out-of-Control Train Will He Board?


ALS has been compared to a ride on an out-of-control downhill train.  You can't stop it.  You are a passenger.  You do your best to cope.  The destination is never good.  No two people with ALS are on exactly the same train.

Some people's ALS speeding ride lasts only a few months.  Others are on a different itinerary and their downhill slide lasts for years.

You can't tell the timetable or itinerary of a case of ALS by taking a snapshot of a person with the disease, especially when the passenger has just stepped on the train.  Snapshots of passengers don't tell anything about the downhill slope ahead that the train will take.


When people are chosen for ALS clinical trials, the selection often focuses on people who have not been on their trains for long, without regard for the vastly different trains.

Do we somehow stack the clinical-trial deck by the way patients are chosen for ALS trials by time stamps on their tickets?



Every day scientists learn more about those trains -- the genetic variations, the potential biomarkers, the itineraries themselves.  Do ALS trials contain and follow sufficient diversity of trains?







Friday, January 4, 2013

Yesterday Was A Lemon Of A Day

Early yesterday morning financial-market tweets started mentioning Biogen Idec, and the news wasn't good.  Their most prominent of the "promising" ALS treatment candidates, Dexpramipexole, had failed to show efficacy in its large, Phase III clinical trial.  As an ALS advocate soon posted on facebook, "Dex is done."

Nobody believed that this would be a miracle treatment, but most of us believed that for a drug candidate to come this far over the last decade, surely it would help slow the disease in some patients.

No. Yesterday people with ALS felt a devastating blow.

Today we need to figure out how to make some lemonade out of a lemon of a day.  Over 900 clinical trial volunteers were involved in the clinical trial for this drug which was dubbed the "Empower" trial.  Today we need to empower their efforts by learning lessons from the trial process and making positive changes.

If a drug company has a dud in the pipeline, it's good both for the company and for the patients to eliminate the dud.  Good, but how could we have identified this dud sooner?  Time is money for a pharmaceutical company.  Time is lives for those dealing with ALS.

The traditional clinical trial sequence is to have two safety phases followed by efficacy phases.  That makes a lot of sense for most diseases, but ALS isn't most diseases.  We need to face the fact that even a Phase I or II safety trial is an efficacy trial in the eyes of volunteers with ALS.  They're looking for improvements (or more typically, a sense of getting worse less quickly) from the very first dose.  Last year a scientist with ALS who had participated in a Phase II trial asked a very interesting question.  Should we not have larger, more definitive Phase II ALS trials designed to discern safety and some efficacy more clearly?  Risk is tolerable.  You could ask the man who made this suggestion, but his funeral was four months ago.

Yesterday we also saw a quote from the ALS Association's Chief Scientist in the Wall Street Journal --
"The important thing we're learning about ALS is it's a spectrum of diseases," Lucie Bruijn, chief scientist at The ALS Association, said recently. "There are some who respond to treatment and some who don't; at the moment, we lump all the people together and that might be one of the reasons why the trials are failing."
What's the action plan to fix the next clinical trials so that they recognize the spectrum of diseases?  Months ago another man with ALS who had participated in a Phase II trial asked why clinical trials did not insure genetic diversity to prevent the exact kinds of failures described in the quote.  The "spectrum of diseases" concept isn't new.  It has been around for years.  The problem needs an action plan to fix the problem for the next clinical trials.

While were thinking about lemonade recipes, how about giving some other researchers access to that large set of Empower clinical trial patient data?  Biogen Idec has made a huge investment in ALS.  Yesterday they emphasized that they're still committed.  Can they lead by opening up access to the clinical trial data in hopes that something there will add insight to scientific research?  Can they be bold and start an open data standard for ALS clinical trials?  The volunteers surely deserve to have their data be useful since most of them will not survive for any product to make it to market.

Let's prove that the "Empower" word was more than a catchy name to promote clinical trial enrollment. Let's make some lemonade out of yesterday's lemon of a day.  Let's learn from what just happened.  Let's act.

Thursday, January 3, 2013

OK, Which Are The Stones That Have Already Been Turned?


Every day people with ALS and their families and friends search for hope.  They search for things to try.  They search for medical research that merits their donations.  They search.  They try. They invest their lives and their money in formal and informal research. They don't live long.  They die.  
For every person lost in the relentless stream of funerals, ALS recruits someone new who searches anew with family and friends to find the most promising things to try and invest in.

If Doctor Google were to be candid with us, we would find that the new recruits search over and over and over about many of the same things that have been around for decades.  The constant stream of ALS rookies asks the same questions year after year.  They experiment with the same concepts for decades.  It's terribly hard for them to know what's really new and what has been tried already.

Who can organize the pile of rocks and tell them which are the unturned stones and which have already been exposed as having no value for those with ALS?  Who can tell them which stones have scientific promise?  Where is a simple place that they can go to find the results of the medical research that families and friends have been funding for decades? 









Tuesday, January 1, 2013

It Is With Mixed Emotions That We Face 2013

The Good News

For the last couple of weeks we have received messages from our ALS not-for-profits celebrating the achievements of 2012.  As we all considered our year-end donations, those ALS organizations burst their buttons with the "promise" that they delivered in 2012.

The Bad News

Last year was also a horrible year for over 120,000 families globally (over 5500 families in the United States).  They buried loved ones who died because of ALS.

The horrible years have gone on year after year after year.  The numbers have not diminished in any way.  The cumulative loss is staggering.  The world learned of ALS when Lou Gehrig was diagnosed in 1939, and we have failed to deliver on any of the annual "promise."  

So...

There is a quote attributed to Einstein that it's insanity doing the same thing over and over and expecting different results.  Today is a good day to reflect on that.  

Resolving and Evolving and Revolving

On New Year's Eve in Italy, there is said to be a custom of tossing out old pots and pans and dishes and furniture through the windows as a kind of cathartic housecleaning ritual.  In the fight against ALS, how do we figure out which is the clutter that is holding us back?  How do we toss it out?  

In the difficult world of not-for-profits trying to establish sustainability, do we get stuck in some ruts of "promise" that never have to face the fact that we have collectively failed year after year after year to make a meaningful dent in the tragedy of ALS?  "We have failed" does not make a great fundraising slogan.

Today I ask myself how we can make some meaningful change in 2013 by letting go of some old pots and pans.
  1. As news has been all about the Fiscal Cliff, the U.S. National ALS Registry has come to mind. We have fought hard for the approval and continuing funding of the Registry.  It is critical to have the epidemiological clues that it will yield; however, have we been overfunding the project?  Did we lose focus by appropriating extra funds that let the CDC stray from the core project with unnecessary and confusing tools? Has the project grown to spend a budget because nobody gets a gold star for not spending a government budget?  Can we regain focus on the Registry itself in 2013?
  2. Travel agents and Realtors have had to redefine their roles in the last decades.  They can no longer be the owners and purveyors of information that made them indispensable.  The world of information has opened up access and they had to find different ways to add value to business propositions.  Our disease-related not-for-profits have long been used to being the voices of their patients and have owned much of the messaging and information.  Those days are quickly coming to a close, too.  Patients can and should be heard directly.  Information can and should be accessible.  Can we help our not-for-profits adjust their roles to add value without being the sole controllers and purveyors of information and messaging? 
  3. A scientist with ALS has a mantra about the experiments that people with ALS try -- "If it is done in secret, then it is done in vain." From the days when Eleanor Gehrig tried special greens laden with Vitamin E from Central Park in Lou's diet, people with ALS try things just in case they might help.  Some of these experiments are pretty innocuous.  Others are risky or expensive.  If patients would simply share their data (good, bad, or indifferent) it would be of immense help to those who follow.  It might even give some scientists some clues.  For decades we have lost valuable information. I'll bet that somebody today is looking for Vitamin E for a person with ALS because somebody thought it might help.  Think of how the fight against ALS might change for those dealing with it today if patients and families could look at the use of Vitamin E by people with over the years.  Many people with ALS look to our ALS organizations for advice.  They can't recommend experimental treatments, but they certainly can encourage people trying things to capture their data in a place where other patients can benefit from their experience.  The technology at a place like www.patientslikeme.com makes this very possible.  Is it time to encourage the patients and their data to speak?
Best wishes for a happy, healthy new year, and to all of those who have been dealing with some tough holidays without their loved ones lost to ALS, you can be assured of our prayers and our resolution to fix some things in the fight.