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

Look What A Few Million Eyes Saw In USA Today Today

Thanks to ALSA Florida Chapter for the pic and the heads-up.

We hope that this is a portent of a new and effective way of raising ALS awareness during the U.S. ALS Awareness Month!

It's Important To Be Able To Say, "I'm in!"

Physicians don't report cases of ALS to a health agency or to the CDC.  Until a few years ago we had no census of people with ALS.  Nobody was counting noses. 

The CDC's National ALS Registry changed that.  It's important that every person with ALS self-enroll in the National ALS Registry.  This message is simple and important, yet it somehow fails to find every person with ALS.

Please help spread the word.  It's critical.  Every person with ALS should be able to say, "I'm in!"

Time To End The "No Harm No Foul" Mentality

People with ALS often report a long and wasteful road to getting a correct diagnosis.  They are subjected to countless irrelevant therapies and surgeries before a neurologist says, "You have ALS."

Perhaps physicians operate under a kind of no-harm-no-foul attitude.  There isn't much that they can do for a person with ALS, so maybe the delay in diagnosis isn't as troubling to them as a delay in a cancer or a diabetes diagnosis.

Wrong, Doctor.

ALS clinical trials are often limited to patients within 24 months of onset.  That's not diagnosis, that's onset of the disease.  A patient often wastes over half of that window not knowing that the fasciculations and weakness and choking are ALS.  That late diagnosis is followed by the period of shock and then the period of searching for something (like a clinical trial) that might hold some promise.  Good luck finding the right trial and being accepted with the remaining window.

It's time to make sure that physicians realize that missing a prompt and accurate diagnosis of someone with ALS does a great deal of harm. 

No more no-harm-no-foul attitudes, please.

Ten Travel Tips For ALS Advocacy Day

If you're not registered for the ALSA Advocacy Conference, you might be interested in the conference materials -- https://docs.google.com/open?id=0B0yxxVCJLk5lS1FMZTFEOWdRMTA

If you are registered, here are some tips for your travel planning.

1. Take and wear things that let the public know that your cause is ALS.  It starts at the airport or the parking lot and continues to Starbucks and the hotel staff.  This can be a period of constant ALS awareness.
2. Ask lots of questions at the conference.  There is never enough time for Q and A at the conference sessions; however, that shouldn't discourage people from trying. 
3. Read up on the advocacy priorities.  Sure, they tell you that you don't need to be a policy wonk, but you'll be a lot more credible if you understand what you're asking for.
4. Google "Dee Chiplock ALS Advocacy," and read about a terrific advocacy role model.  She spoke up.  She made some presenters at the advocacy conference uncomfortable.  She acted like someone who needed to accomplish much in a short period of time.  She rocked boats and she rocked. She died before her second advocacy conference, but she left her mark.
5. Use your facebook page or a blog site or an organization's facebook wall or a message board or twitter and share your experiences.  Let those young legislative staff members you talk to that you'll be sharing online.  You'll be amazed at how many visits you'll get from .gov people when you put your impressions online for the world to see.
6. If you are taking the kids, get them engaged.  It's a great civics lesson.  Legislators are pushovers for kids, too, and even the most difficult ones will find it hard to dismiss a child asking for research for Mom or Dad's terminal disease.  Kids are more than little ALS fundraising machines.  We need their brains and their passion. One of those smart kids or a classmate may be the one to figure out ALS.  What better writing exercise than describing a day on Capitol Hill for their classmates?
7. Rest up if you can.  The day on Capitol Hill is exhausting.
8. A man with ALS told me at the end of the day on the Hill that he got some things off his chest.  Get some things off your chest.
9. Speak up. Speak up to the people who are supposed to be representing you in government. You pay their salaries. Speak up the the organizations that are supposed to be representing you.  Speak up to the government-sponsored researchers who are supposed to be delivering for you.  Speak up.  I realize that's a poor choice of words for people whose speech has been stolen, but please find a way to be heard or seen!
10. Find a way to make the message stick.  People go back every year and talk to legislative staff members about ALS and ALS in the military and ALS research.  Every year those staff members seem clueless. We need to make the messages stick!

Have safe travels and please raise some Cain.

We Hope Someone Will Record The Keynote

Welcome to New Orleans and ALS, AAN.

http://www.nola.com/business/index.ssf/2012/04/american_academy_of_neurology.html

ALS -- A Disease Like No Other

Thank God.

One of the subjects of the recent national media coverage of patients making their own drug trials told me, "The FDA needs to reassess its policy with regards to ALS. It is a unique disease and requires unique FDA policies."

Are we designing and pursuing change that will make a difference for those with the disease like no other?

Good Morning, America and the World

ABC News has picked up the story...

http://abcnews.go.com/Health/Wellness/lou-gehrigs-disease-patients-research-hands/story?id=16150103#.T46mgdWiZki

This is definitely worth reading and then rereading. Eric Valor is a rock star and a wise young man.

Are There Two Doctors Jonathan Glass?

April 13, 2012
http://www.mddionline.com/article/tedmed-stem-cells

Glass had one clear message for the audience: “We must reconsider, we
must redefine, and we must recalculate what is acceptable risk. And that risk
must fit the emerging technologies and emerging therapies that hold the enormous
potential to transform the treatment of disease in the practice of
medicine.”

April, 16, 2012
http://professional.wsj.com/article/SB10001424052702304818404577345953943484054.html?mg=reno-secaucus-wsj

Jonathan D. Glass, professor of neurology and pathology at Emory
University School of Medicine and director of Emory ALS Center, is one of the
NP001 site investigators. He said he is concerned that "these people could hurt
themselves. Who knows what they are actually making in their
kitchen."

It just gets curiouser and curiouser.

The Governor And The General Are Now Aware

...and helping to pass the word forward...




Thank you, Governor Daniels and General Umbarger.

Sometimes You Take Matters Into Your Own Hands

There is an excellent article in this morning's Wall Street Journal -- "Frustrated ALS Patients Concoct Their Own Drug," by Amy Dockser Marcus.

These are not whack-job patients who are grasping at theories that make no sense. These are young, bright patients who have done their homework and have a disease with no effective treatment options. The only options that the medical establishment offers them involve a quick downhill slide to death. Who can blame them for daring an intelligent roll of the dice?

Shame on us who are healthy and have provided them with a medical establishment that has not delivered for them and that cannot deliver in a time-frame that will help them. Shame on us for painting them into a corner. Bravo to them for making the world pay attention as they try to buy some time. Ben Harris and Eric Valor are heroes.

WSJ