Thanks For Your Leadership, ALS Therapy Alliance

For the last 48 hours there has been internet buzz regarding Seth MacFarlane's use of ALS as a point of attempted humor in his new movie, "Ted."

Finally an ALS organization has stepped up with a statement.

http://www.marketwatch.com/story/seth-macfarlanes-movie-ted-causes-punch-line-about-lou-gehrigs-disease-to-become-pop-culture-chatter-on-twitter-facebook-2012-07-03

Now if only a few others would show that they have the backs of those with ALS by speaking up in the media.

Organizational backlash works.  This is an especially important week for the world to understand that there is still no known cause, there is no cure, and ALS isn't a disease to joke around about.

We All Know Poor Signage When We See It

And when the signs aren't clear, do we pay attention to any of them?

Please take a moment to read this report which was presented by a gentleman with ALS to some of the leaders of the U.S. National ALS Registry this week.  It contains important data and perspectives related to the awareness of the ALS Registry.

Every person with ALS needs to know the "what, when, why" of the National ALS Registry.  There are three important action items for each person diagnosed with ALS --

1. Self-enroll at http://www.cdc.gov/als
2. Complete the six additional Risk-factor Surveys
3. Complete the Ouality of Life updates at regular intervals

That's it. 1,2,3.  Repeat 2,3 as needed.

The message has been lost in the muddle of stuff that families hear in the first weeks after diagnosis.  Or has the message been delivered at all?

Taxpayers have spent millions and millions and millions of dollars on this project.  It's an important improvement over the decades when clues from people with ALS were buried with them.  The least we can do is get the message to people diagnosed with ALS clearly so that their data will be retained.

It's a matter of getting the right message to the right people at the right time.

And you don't fix poor signage by buying more signs to add to the message clutter.

What Comes To Mind When You Hear "Duke Blue Devils?"

Coach K?
Nah. As great as Coack K is, the definitive response is Dr. B.



This is well worth sitting back and listening for a few minutes.

Thank You, American Academy of Neurology

It was a wonderful surprise to see a tweet come through from @AANmember to urge neurologists to encourage their patients with ALS to self-enroll in the National ALS Registry.  Here's a new article on the AAN website --
http://www.aan.com/news/?event=read&article_id=10607

Immediately after diagnosis, the neurologist has a big impact on the actions that a person with ALS and the family take (or don't take).  Many patients have reported not hearing anything about the National ALS Registry from their physicians, therefore, they had been hesitant to self-enroll.  As of today, we see that changing.

Thank you, AAN!

July 4 Needs To Be Special At Every Baseball Game!

On July 4, how about having every baseball game remember Lou Gehrig's farewell?  Major League Baseball all the way to backyard Whiffle Ball games played while waiting for the burgers on the grill... just take a moment to remember an historic day in baseball.

This isn't complicated.  It's not a fundraiser.  It need not disrupt the fireworks and parties.  It's a very simple and fitting way to remember a great sportsman, a great sport, and a disease.  It would teach children who Lou Gehrig was.  It would remind us all of what is important in life.

Let's add this tradition to a sport that is rich with traditions.  Those who control the displays at professional ballparks, please simply play the speech.  Those who are playing on a sandlot or backyard, please take a moment to talk about what Lou had to say.

Some Of Us Need To Put More Fight In The Fight Against ALS

Chapter I


Doctor's Message - Day of Diagnosis

Doctor, you would be amazed at how much your patients and caregivers hear and retain that day.  You set the stage for a complicated trip that they face.  They have some fight in them.  Do you realize that when you tell them about an opponent that they can't beat?

Do you consider the importance of giving them some constructive action items?  They are important. They need to be clear.

Here are two that come to mind --

1. The patient should self-enroll in the National ALS Registry and continue to participate in the risk-factor surveys at www.cdc.gov/als . The patient and family need to know that you doctors do not report ALS cases to any health agencies.  The only way to guarantee patients' data and life factors will be retained is for the patient to take action to self-enroll 

2. The patient should become familiar with clinical trial opportunities immediately.  The window to qualify for a clinical trial is short, and patients need to be informed about the options.  Doctor, if you can't cure the patient, then please give the patient some ways to contribute so that the next person may not face the same unfair fight.

Those are two constructive action items that let the patient fight back a little.  They are important.  If patients and caregivers only get messages and materials that involve acceptance of the ALS beast, then we healthies have taken some of the fight out of the fight.  The action message needs to come from the neurologist. It needs to be clear.  It then needs to be reinforced.

We need to put more fight in the fight.

How About Some ALS Awareness On Flag Day?

There are around 6000 annual deaths from ALS in the United States every year. Approximately 12.1 percent of U.S. citizens are veterans. If veterans experienced ALS at the same rate as the general population, there would therefore be around 726 U.S. military veterans' deaths per year from ALS; however we know that the military experience ALS at twice the "normal" rate. So we're looking at around 1452 U.S. veterans dying from ALS every year. Please check my arithmetic because that seems so very high. Perhaps we should have 1462 American flags displayed in every metropolitan area on Flag Day. That would start some conversations.

Sic Transit

Curing ALS Is Difficult. This Would Not Be Difficult.

This morning my Google news alert popped up with an ALS obit (which it does often). The name was unusual and the same name as a minister in my city who died from ALS around 10 years ago.


"Familial?" certainly popped into my mind. Or maybe simply coincidence.

For many years I've thought that it would be a valuable project for an ALS organization to archive ALS obits and establish a very simple online database of last names and dates of death. It surely wouldn't be difficult to capture the public information online. It seems like a simple project for an intern or a dedicated volunteer.
It could be useful not only for identifying possible family ties but also to retain more about the remarkable people who are stolen by this stinking disease.  The endless stream of obituaries supplies us with information that is well worth keeping for many reasons.
 

The Point Can Be Boiled Down To 140 Characters

As many ppl get ALS as MS. Wow. Not as many ppl w ALS in our midst, tho. So what happens to all the people w ALS? Clue: They don't get well.

It's Time We Addressed the "R" Word With Some Numbers

Tonight Canada Is Shedding Light On ALS

Tonight the CN Tower will feature purple lights in honor of ALS MND Awareness.

http://www.prlog.org/11889867-commemorative-lighting-of-cn-tower-candle-lightings-on-parliament-hill-halifax-waterfront-in-june.html

http://www.cntower.ca/en-CA/Plan-Your-Visit/Attractions/Night-Lighting.html

And there is a  moving event on facebook where people are joining by lighting candles tonight to remember those fighting ALS and those loved ones whose lives have been taken.

https://www.facebook.com/#!/events/329018427174261/331159963626774/?notif_t=plan_mall_activity

Today is a day we can be thankful for the power of the internet to spread an idea. Please spread the word and make tonight's a global observance.

Thanks, ESPN

Here's a nice piece to start the finale of ALS Awareness Month...

http://espn.go.com/college-sports/recruiting/football/story/_/id/7982519/kelly-hilinski-takes-nana-randolph-last-words-heart

We need more grit like his in the fight!

Meanwhile Back At The VA Hospital

On Sunday ALSadvocacy joined a few hundred thousand Americans in honoring our veterans at the Indy 500.  There was a parade where every grandstand stood and clapped for the military marching around the track.  There was the playing of taps that reminds us of the sacrifice that so many have made.  There was the National Guard car that carried everyone's favorite sponsor.  This past weekend our nation swelled with pride and honor for those who served and continue to serve.

Meanwhile back at the VA hospital, the following happened (as posted by a veteran on his facebook wall):

Maybe it was the consult, but this clinic really didn’t do its homework on me before I arrived. My experience below…

My PEG experience at the Augusta, VA has been interesting. I needed a PEG feeding tube to help ward off respiratory infections when my ALS starts to cause problems swallowing. I was scheduled to receive a PEG when my lung capacity dropped below 40 percent in March 2012. My VA neurologist recommended I get one, and submitted it as an internal VA consult.

I received one call before receiving my appointment date and instructions in the mail. The call from the GI clinic asked how urgent the PEG requirement was. I told them I had ALS, explained my dropping lung capacity, and requested an early date. They cut me off before I could explain I was wheel chair bound or discuss the procedure. No pre-op appointment either.

On the day of, we arrived promptly at 9 AM. By 9:15 the staff had its first issue. How to move a 200 lbs invalid from power chair to stretcher? No plan and no equipment. It’s rectified by 9:45 after a 5-floor move, and I’m on the stretcher with an IV. Now I wait, wait and wait.

No pain meds since midnight. I wait, I wait, I wait. Hips, lower back and butt are throbbing, burning, and aching, and I wait. Hey, why not just leave me in my comfortable fully articulating power chair until the last minute? Why are you surprised my blood pressure is high? Did you read my file?

At 12:30 PM I tell the nurse, something’s got to change or they can yank the IV. Ten minutes later I’m swishing down some pain stuff in preparation for the insertion of a scope down my throat. Next thing I remember is waking up and being told they couldn’t do it because I was too restless under the local anesthesia. They want me to come back on Friday so they can do the surgery under general anesthesia.

This is an example of what happens when healthcare professionals don't understand ALS. Patient and caregiver time and precious vitality were wasted, pain was inflicted, and taxpayer resources were wasted. Studies have shown that patients who receive care from specialized, interdisciplinary clinics have better outcomes than others, yet our veterans, who have twice the likelihood to need ALS care than the rest of us, have a VA system that clearly failed to deliver that skilled, interdisciplinary expertise.

So we cheered and honored our military on Sunday and Monday, and then we let this happen. That's just wrong.  Talk's cheap. We need to do better for our veterans with ALS.

This Is Exactly Why

Ron Leuty has an interesting article on the hundreds of clinical trials emanating from the Bay Area biopharm hotbed.  He even calls out ALS in his article.

http://www.bizjournals.com/sanfrancisco/blog/biotech/2012/05/bay-area-clinical-trials-database.html?ana=RSS&s=article_search&utm_source=twitterfeed&utm_medium=twitter

Now click on the link in the article that will take you to the long, online list of clinical trials.  Click on the Indication header to put them in alphabetical order.  Now look for ALS.  Does anybody see a problem?

This is exactly why Dr. Stanley Appel once said to Congress, "ALS is not an incurable disease. It is an underfunded disease."

It's So Good That Peter King Met Steve Gleason

From Peter King in SI -- http://sportsillustrated.cnn.com/2012/writers/peter_king/05/29/mail/index.html

Steve Gleason goes to the United Nations, and other tidbits from Monday's column and beyond, before I get to your mail: • Gleason, the 35-year-old former New Orleans Saint special-teamer now battling Lou Gehrig's Disease, is one of the speakers at the Social Innovation Summit at the United Nations in Manhattan Thursday. More than 200 executives and high-level philanthropists will gather to discuss strategies and causes like Gleason's teamgleason.org, which helps Gleason raise awareness and money to help ALS patients live more active and normal lives. In particular, the summit could help Gleason focus on new technologies to assist the victims of the neuro-muscular disease. Read more: http://sportsillustrated.cnn.com/2012/writers/peter_king/05/29/mail/index.html#ixzz1wHwhrPbg

Ahhh, finally... the last day of ALS Awareness Month. Squeak.

Never Underestimate A Person With ALS

Lize and Braveheart are a magnificent team.

And 12,000 Next Year and 18,000 The Next And On And On Until The Carnage Stops!

Here is a wonderful display that helps us visualize the annual American lives lost to ALS.  That's just one year. That's just the USA.

http://commcgi.cc.stonybrook.edu/am2/publish/Medical_Center_Health_Care_4/6_000_Pinwheel_Display_Symbolizes_Lives_Lost_to_ALS_at_Ride_for_Life_Event.shtml

Stay Tuned For The Greatest Spectacle in Racing

Thirty-five years ago this month, Sid Collins, the Voice of the Indianapolis 500, died shortly after having been diagnosed with ALS.  If you ever listen to old broadcasts of the Indy 500, you will hear the gift that Sid Collins had with words.  If you ever watch any old videos of Victory Banquets, you will see the gift that Sid had with a crowd.

ALS is still the cruelest diagnosis for anyone with Sid's gifts.

Here are Sid's parting words from his final 500 broadcast in 1976.

So now, the 60th running of the 500 here is now history. Since 1911, the hypnotic effect of speed upon driver and spectator alike is never dim. The run from the green flag to the checkered and on to Victory Lane here is a pursuit only one man in the world can accomplish once a year. Today, once again, Johnny Rutherford etched his name and his achievement upon the granite of time. He reigns supreme as the champion of the sport of auto racing this day and forever more. The massive crowd of more than 350,000 has threaded its way towards the exit gates as their eyes have taken a final sweep over the track before departing. For some, this has been a once-in-a-lifetime experience, others will come back, but in every case, it's always difficult to relinquish one's grasp on the pulsating emotion that is the 500. And at this microphone we share that reaction of having to say goodbye to you across the many miles that separate us. But, another icy Indiana winter will come and go, and before we know it, springtime returns, it will be May, and the roar of engines will once again breathe life into the lazy Hoosier sky and bring us back together. God willing, I'll be here to greet you for this annual reunion through our mutual love of auto racing and the Indianapolis 500...

...And now this final thought for our winner. Enthusiasm with wisdom will carry a man further than any amount of intellect without it. The men who have most-powerfully influenced the world have not been so much men of genius, as they have been men of strong conviction with an enduring capacity for work coupled with enthusiasm and determination. Johnny Rutherford showed these qualities today in becoming victorious over the Indianapolis 500...

...So until next May, this is Sid Collins, the Voice of the 500, wishing you good morning, good afternoon, or good evening, depending upon where in the world you are right now. We're here at the Indianapolis Motor Speedway, at the Crossroads of America. Goodbye.

An Angry Bird Is Tweeting This Morn

We All Need To Pay Attention To The Rest Of The Story

Almost a year ago, people with ALS were anticipating several important clinical trial opportunities.  One was a phase III trial for Biogen Idec's Dexpramipexole  The other was a phase II trial for Neuraltus' NP001.  We had been hearing high promise for Dexpramipexole for years.  NP001 was new on the radar.

http://www.alsa.org/news/archive/neuraltus_7_19_11.html

Both trials filled enrollment quickly.

Patients talk online.  Normally with ALS clinical trials, patients try to figure out if they are less worse taking the test drug than they would have been without.  The bar is low.

NP001 (or an incredible placebo) actually made some clear positive differences for some patients.  That is a first in the 15+ years I've been paying attention to ALS.  Now what?

The trial design called for six months of test therapy followed by a three-month "monitoring period."  Patients were to get no drug during the monitoring period by trial design.

Because of all of the years of poor (or no) results from clinical trial drugs, the expectations were low and the monitoring period didn't seem harmful based on all of the dud drugs we've encountered.  But... until the whole cohort of patients in that phase II trial clears the six months of therapy plus the three months without drug, the data are not able to speak to scientists and regulators.

This isn't scientific, but I've met five patients in my small e-circle who had experienced improvements or plateaus while on NP001 (or the incredible placebo) who are now crashing during the monitoring period.  Really crashing.

I heard a leading ALS organization dismiss the situation because Neuraltus is not as well funded as Biogen Idec.  Sorry, but a money excuse is specious. The trial had a design.  Is there not room for some kind of adaptable trial design?  Will leading ALS organizations allow future trial designs to maintain a barrier between patients and the drug should it appear to perhaps be effective?  Does mixing companion diagnostic aspects with efficacy trials set patients up for a crash period (that we hadn't considered before since nothing had worked before)? Are our organizations really paying attention and willing to lead?

NP001 may not be the treatment we've dreamed of, but it (or the incredible placebo) was doing something. How do we sleep nights when patients who volunteered to test it are allowed to crash?

Yesterday we heard more coverage of patients who are trying to take the science into their own hands so that future patients may fare better.  Guess what drug the featured patient trialed?

http://hereandnow.wbur.org/2012/05/21/als-diy-trials

Your Opinions Are Valued!

ALS is not reported to health departments as cancers and many communicable diseases are.  Before 2008 when the ALS Registry Act was passed, patients' clues were buried with them.  The National ALS Registry changed that.

A bright and hard-working gentleman with ALS serves on an advisory panel for the National ALS Registry.  They made a wise choice in selecting him to represent the perspectives of people with ALS.  Below is a message from him for all people with ALS.  Please pass this request along to anyone you may know who has ALS. He will appreciate your help. Thank you.

Please help. Late next month, I will be serving as a panelist with the National ALS Registry team with the ATSDR / CDC for the 3rd time, as a PALS representative. I have been asked to give about a 15 minute presentation on PALS’ perception of the Registry and especially to discuss ways to reach out to all PALS.



I have prepared a simple 10-question survey aimed at PALS' awareness of the Registry, on my own, located at the following weblink:


www.surveymonkey.com/s/CYF8ZW2


I'd like to get as many responses as possible from PALS (or CALS on their behalf). The target respondents are American PALS (or CALS). Please only one response per PALS (no need for PALS and CALS pairs to both respond. Also, excluded are CALS of PALS who died before October 2010, since they could not register.


Responses are 100% anonymous. Please respond whether or not you have registered and whether or not you are satisfied with the Registry (to prevent selection bias).


Thanks in advance!!!

Way To Grease Some Skids!

Nice to see some important information in the top banner space on Politico.com this morning... just 24 hours before ALS advocates hit Capitol Hill.

Thx 4 Tweets

If you are at all interested in familial ALS, be sure to follow @smfarr on twitter today (or every day for that matter).  Very interesting updates are coming from the Familial ALS Summit being held in conjunction with the ALSA Advocacy Conference. It's the next best thing to being there. Thx, @smfarr.

The Barriers Are At Last Coming Down

Next week when people interested in advancing the fight against ALS meet in Washington, DC, those who can't travel are getting some windows into the conference.

For those interested in having questions addressed at the ALS Association Town Hall Meeting, you may submit online here.  All topics are fair game.

General sessions are being streamed at http://www.livestream.com/alsadvocacyday

Monday, May 14:

9:15 -- 10:45 am EDT: Government ALS Research

11:00 am -- 12:30 pm EDT: Government ALS Research (repeat, not live) 

2:00 -- 2:45 pm EDT: Advancing the Search for a Treatment: The MODDERN Cures Solution and PDUFA 

4:00 -- 5:30 pm EDT: ALS Clinical Trials