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.

Sunday, December 18, 2011

Wish We Used Stats To Scare Up Support

In yesterday's Wall Street Journal, The Numbers Guy, Carl Bialik, has another interesting column. This time he writes of health not-for-profits who find ways to present scary statistics to state their cases for support.



Article (subscriber content)






Here is a comment posted below the article --



Unfortunately disease prevalence has traditionally been used to establish relative priorities for much research funding, and prevalence simply doesn't tell the story for some diseases. I've been involved in the fight against ALS (Lou Gehrig's Disease) for a number of years. It has a relatively low prevalence and a high incidence, and therein lies the problem. It's like a cruel game of musical chairs where we keep pouring people into the room and when the music stops, there are very few chairs. The only exit is a door labeled "death." Many of our ALS organizations are resigned to the fact that disease prevalence has been the gold standard for government and private research funding priorities. That attitude will never bring ALS the attention it needs to be handled as an urgent, fatal, and mysterious disease with a horrific throughput. I wish the ALS cause had some of those numbers people who can express the odds in a way that would make every healthy person squirm!

Saturday, December 17, 2011

Here Are Some Realistic Goals

Occasionally ALSadvocacy is chided by some people who work for ALS organizations. Some ideas such as inter-organizational coordination, awareness programs with global punch, a case study of the cost of the disease, educational programs at many levels, normalized reporting on annual investments in research, or a new standard for measuring a disease's impact are perceived as not being realistic.

Perhaps we can agree on a few goals for 2012 that are completely "realistic" from any perspective. Here are two.

Insure that every newly diagnosed ALS patient in the United States
1. Is immediately advised of the importance of self-enrolling in the CDC's national ALS Registry.
2. Is immediately advised of all clinical trial options available.

Those are simple. They are deliverable. They are measurable. They are important. They are not happening today. If they're not "realistic" then perhaps our organizations should find an easier disease to fight.

Thursday, December 15, 2011

Remember The Over 5,000 American Lives Lost to ALS in 2011

Thanks to ALSA Philadelphia Chapter for pointing this opportunity out to remember loved ones and to raise some huge ALS awareness in The New York Times --

http://submit.nytimes.com/lives-they-lived

Please spread the word to all who have lost loved ones to ALS in the last year. The holidays are rough, and this is a good way to remember and teach others about the toll that this beast of a disease takes.

Wednesday, December 14, 2011

Who Represents Patients In This Puzzle?

Here is an interesting perspective on clinical trials --

http://bioworld.blogs.bioworld.com/2011/12/13/it%E2%80%99s-about-the-patient/

ALS certainly poses a completely different set of challenges for the drug companies trying to move a therapy forward, yet the question about remembering the patient has a special sting with ALS.

Thursday, December 8, 2011

Wow!

Nothing Is Easy With ALS

And sometimes the obstacles are just wrong.

http://www.alsa.org/news/archive/fda-approves-neurx-diaphragm.html

There has been an interesting saga described on the forum at patientslikeme.

A man with ALS recently made arrangements to go hundreds of miles to Cleveland to have the DPS described in the article at the above link "installed."

At the 11th hour in Cleveland his insurance carrier denied this treatment. He packed up his things and went back home.

Note that this man has ALS and travel is difficult and the breathing that he is trying to preserve with the DPS diminishes every day in the relentless ALS downhill slide.

Now he has finally found out what the difficulty was with the insurance coverage. The insurance company was not aware of the FDA approval of the DPS for ALS. The insurance company hasn't learned how to Google? Everything is fine now and he can go back to Cleveland for the procedure.

No, everything isn't fine now. The patient was jerked around by a healthcare delivery system that included an insurance company that made a big mistake at his expense. Precious time was lost. Precious resources were spent by the patient because an insurance company made a big mistake.

Nothing is easy with ALS, and we have a healthcare delivery system that continues to show its weaknesses at the expense of people trying to deal with this disease. Just as they introduced stress tests for banks during the banking crisis, perhaps ALS could be the stress test for healthcare delivery. Few organizations would pass.

Wednesday, December 7, 2011

And Walks Are More Than Fundraisers

They are reasons to talk about ALS in communities and in the media. They are a means to generate the awareness that will finally impress the urgency of the problem upon the healthy. They are a way to put ALS in the spotlight it needs so that we can break out of our rut of still having no known cause and no cure.

Saturday, December 3, 2011

Are We Literally Protecting People With ALS To Death?

Here is an important perspective from a man with ALS. It was posted on the ALS TDI forum where patients with scientific backgrounds are collaborating and experimenting and sharing data.
I have a slightly different perspective than most coming from the radiation oncology field. The things we put our patients through would seem downright medieval to most. We expose people to enormous amounts of radiation that often makes them so sick they almost die form it. We have perfected the dose to bring them to the edge of death but not beyond. We all know what chemotherapy patients go through. They lose all the cells in their stomach and can barely eat for weeks. Again, we bring these people to the brink of death because that is what it takes to kill the cancer cells.

People with ALS are in no less dire straits than people with cancer. Getting sick from a potentially life saving cure should not deter us from trying. No doubt there is a risk with this sort of thing, perhaps even death, so only those willing to risk it all should attempt it, but we risk it all by doing nothing.

Thursday, December 1, 2011

Now THIS Is Awareness


Since the top leadership from ALS and MND organizations worldwide are in Sydney at this very moment for their annual summit, perhaps they'll get some ideas about how to raise awareness. It is important. The AIDS cause knows that. When will the ALS cause figure it out?

If any of the ALS delegates or researchers went to see Bono light up Sydney, lets hope that they went to be inspired and not simply entertained.

Monday, November 28, 2011

There's More To Chicago Than The Travelogue







We hope that the scientists will come to Chicago for more than the restaurants and the skyline and the shopping.



This is Chicago, too... a great American city with ALS on its mind!




























































There Is Redeeming Social Value in Twitter

We are getting an interesting window in to what is happening at the International ALS MND Symposium in Australia via twitter. Watch for hashtag #ALSSYMP ... and thanks to @alstdi and @mndaustralia for their interesting tweets from the Ask the Experts session.

Perhaps this will plant a seed that people with a disease that steals their ability to speak and travel and be mobile deserve more opportunities to be a part of the conversation with those who are searching for a treatment. Surely there would be some synergies to these parties knowing each other better.

Yes, twitter is for more than self-serving organizational announcements.

Saturday, November 26, 2011

Ask The Experts, But Don't Count On Asking Too Much

Tomorrow night (U.S. time) the big international symposium on ALS kicks off, and the traditional "Ask the Experts" session is being hosted by MND Australia, the host organization. This year we are pleased to see that people will be able to participate online.


Take a look at that agenda. An expert moderator and four subject-area experts, each of whom will speak for 20 minutes. They have left a whole 60 minutes at the end of the agenda for Q and A.

Ok, experts, let's think about this. The title of the session is "Ask the Experts," yet you're doing most of the talking. The asking part isn't until the very end, and if it's anything like last year's session, there won't be time for over a couple of questions. Perhaps "Listen To The Experts And Ask A Question If You're Incredibly Lucky" wasn't a catchy enough title?

Let's hope that the experts keep things on schedule and don't have another commitment that will keep them from answering all the questions that may be sent their way.

People with ALS are constantly fighting the ticking clock and a healthcare delivery system that doesn't listen. Hmmm.

For those in the U.S. who are interested, timing is 10pm start EST... Access information is can be found with the agenda at the above link.

Monday, November 21, 2011

And We Wonder Why ALS Clinical Trials Aren't All Full

Pretend you were just diagnosed with ALS and are googling to find out what you can do to fight it. Your family members are probably doing the same thing. Have them join us in some experiments.

You go to the trusted sites of the big organizations to get information.

Go to www.alsa.org ... www.mdausa.org ... www.als.net ...

Experiment 1:
Look around at their information for new patients. Are clinical trials brought to the forefront as something that you should be considering now, before your short window of eligibility closes?

Experiment 2:
This time forget about them suggesting clinical trials to you proactively. This time pretend that you're on a mission to get some information about clinical trials. Look directly for clinical trial information and try to find a resource that you can email or call to discuss the clinical trial process. NEALS and ALSA have a dedicated clinical trial expert available. It's not easy to find the contact information, is it?

Experiment 3:
Find the most promising clinical trial for your situation.

And we wonder why ALS clinical trials aren't all full.

In recent months we have seen patient advocates take on the mission of filling clinical trials themselves by e-word-of-mouth. They realize that every day that a trial is unfilled is another day's delay in having clinical trial results. Why can't our trusted ALS organizations make clinical trial information a clear part of every new patient's action kit?

_____________________________________________________

If you were stumped by Experiment 2, the information is --
An ALS clinical trial expert can be reached at (877) 458-0631 Monday through Friday between the hours of 9 a.m. and 5 p.m. eastern time and by e-mail atalstrials@partners.org.


Friday, November 11, 2011

Every American Needs To Hear This Today

Here is a 30-second message that is direct, clear, and important. Please listen, then pass it along.


Tuesday, November 8, 2011

This Is Outrageous

Shame on us.






Perhaps this Veterans' Day we need to stop patting ourselves on the back for the past work done for veterans with ALS and start fixing a few things!

Sunday, November 6, 2011

Millions of People Looking for Bargains Are Made Aware of ALS



Thanks, Walgreens and NOVA, for helping good business intersect with vital ALS awareness in today's ad.


Wednesday, October 26, 2011

Wish We Could Go Back To GI-Bill Education And A Great Home Loan

... rather than having our vets get the awful fringe benefit known as ALS.



Reverse Engineering To Defeat ALS

Consider a disease that seems to find very smart people. The disease strips them of their motor functions, but it lets them remain very smart. The disease has no known cause. The disease has no cure.

Consider a disease with a history of disappointing clinical trials... trial after trial after trial... until this summer when one product (Neuraltus NP-001) seemed to have some positive effects on some individuals. It wasn't a miracle of biblical proportions, but it was like a parting of the Red Sea by ALS standards.

Consider a disease whose clinical trials involve a placebo group and have a timeline that is longer than the life expectancy of many patients. Consider a disease that leaves thousands of patients on the bench because they have had the condition too long to qualify for a clinical trial.

Should it surprise anyone that the same smart people whose lives are at stake have turned out to be a pretty impressive online band of reverse engineers? Give them a slight glimmer of hope and they don't sing and dance. They put their minds to work figuring it out. Smart people from every walk of life get ALS, and it has been pretty impressive how they found each other and decided not to be spectators in a life that dealt them a really bad hand.

Take a look --
http://www.als.net/forum/Default.aspx (Oral Sodium Chlorite thread)


Certainly it's risky. There is no doubt that these are courageous people who are cornered by a relentless disease and a drug development system that has failed them in the past. They're not going without a really good fight and an attitude to do something so that the next person will have a fairer fight.

Friday, October 21, 2011

This Is Why The Great Nolan Ryan Is Still Great

Thanks, Nolan Ryan. Thanks, Texas Rangers. Thanks, MLB.



I'm still for the Cards but feeling awfully guilty about that now ;-) This is huge visibility for ALS.

Thursday, October 20, 2011

Here Is A Perspective On A Birthday

Oh, that some creative minds might find effective ways to increase self-enrollment and patient participation in the supplemental surveys for the CDC's ATSDR National ALS Registry!

Here is some perspective on a "birthday" and on keeping our eyes on the prize --

http://alsspreadtheword.blogspot.com/2011/10/its-project-for-petes-sake.html

Let's make this registry work to provide the epidemiological clues that scientists need. Let's not sing until the data speak.

Tuesday, October 18, 2011

Is This A Sign Of New Things To Come?






On the main page of http://www.thehill.com/ ... there was a scrolling ad about a veteran with ALS. It had an ALSA logo and was promoting PDUFA.



We can't wait to see if this is a sign that ALS is about to have some in-your-face awareness!



Veterans - Time To Be Seen And Heard!

Here is a nice website from the ALS Association...

http://www.alsa.org/advocacy/veterans/

If you are a veteran who has ALS or you have lost a loved one to ALS who served in the military, please add a picture. This will give the mysterious connection between military service and ALS some much-needed visibility!

Please pass this word along. Thank you.

Let's Hope

Let's hope that Jason Alexander noticed the new shoes on the man with ALS. Those shoes will never be worn out. Let's hope that Jason Alexander noticed that the man with ALS didn't sip on coffee like the others in the room. Let's hope that Jason Alexander takes Steve and his disease personally. Lets hope that Jason Alexander will talk off-script about this outrageous disease. Let's hope.



From the statement from the ALS Association :
"Over the coming weeks the ALS community will begin seeing these materials launched as part of The Association’s more aggressive public awareness efforts. 'We are determined to bring ALS out of the shadows and into the light of public awareness' said Gilbert."

Let's hope that it will be aggressive and part of everything ALSA does. Let's hope.

Let's deliver.

YOYO (You're On Your Own)



For the recently diagnosed ALS patient, it's not quite as easy as it sounds. Here are some things that may help.

  1. Some neurologists aren't current on the status of clinical trials. Some neurologists discourage clinical trial participation lest the trial may do harm to the patient (like ALS isn't the ultimate harm). Some neurologists advise patients only of clinical trials in which they are investigators. Some neurologists advise patients only of clinical trials in which they are not investigators. Some physicians have the human "not invented here" frailty. Patients shouldn't stop with the neurologist. Ask questions and ask more questions and contact the Clinical Trials Expert (who is a physician sponsored by several ALS organizations and should be able to provide objective information) alstrials@partners.org or (877) 458-0631. That contact information isn't easy to find on any organization's website, yet it's probably one of the single most important resources for a patient who might qualify for a clinical trial. Go figure. Nothing is easy with ALS.
  2. Going to the ALS Association website isn't going to provide you with a current or comprehensive list of clinical trials. Try www.clinicaltrials.gov and search for "amyotrophic." There you will find the most current list of trials and their locations and current status. It's a more up-to-date database of trials than any other organization's website provides.
  3. Look at forums at www.patientslikeme.com (also includes patient data reported by patients in formal clinical trials and in diy treatments) and www.als.net and www.alsforums.com ... and realize that not everybody there is an expert (and it's always easy to fall into the trap of listening to what you want to hear)... but you may find some information that will help you make a more informed decision about a clinical trial.
  4. Time is of the essence. Many trials limit participation to those within 24 months of onset. Many patients have lost much of that eligibility window just trying to get a correct diagnosis.
ALS is unfortunately the ultimate YOYO disease. You're on your own to dig and find answers. Don't stop with one neurologist and one website.

p.s. To ALS organizations lurking here... Please try an experiment. Pretend you were just diagnosed with ALS and want to find out about clinical trials. Do it when you're exhausted and after you've whirled like a dervish for three minutes since that's the feeling that a lot of people with ALS and caregivers have all the time. Google. Look around at websites. Confused? Try to find the phone number or email address for the Clinical Trials Expert. Confused? Look at the CDC's website. Should you expect a phone call telling you that you are a candidate for a trial? Confused?

Monday, October 17, 2011

Now We're Cooking

Video from a recent "Ask The Experts" ALS research conference in California is available here .

Thanks to the organizers and the organizations for finding a practical and considerate way to deliver information for patients, families, and other interested people.

Friday, October 7, 2011

This Red Sox's Fan Is A Champion



Thanks, Clear Channel Outdoor!



http://www.boston.com/Boston/businessupdates/2011/10/clear-channel-billboards-help-umass-and-cellucci-fight-als/ctFFUD67WW6i78aAPSiKlK/index.html

What We Have Here Is A Failure To Communicate

Last week the Diaphragm Pacing System (DPS) was approved by the FDA for people with ALS.

For years patients who were in the clinical trial of this pacemaker to keep the diaphragm working attributed it to enhancing their quality of life. Finally all patients have access to this option which could be a valuable tool as an early intervention. It's a simple concept.

This week patients are asking their physicians about the DPS and some of the docs don't seem to know about the DPS. "I'll have to look into that," translates into wasted weeks for a person dealing with the ticking ALS clock.

If there were a new prescription drug for ALS, you can be sure that docs would be ready, able, and willing to get patients started. Why the DPS, only the second FDA approved product for ALS, is such a secret among healthcare professionals is just incredible.

So, in case this is of help for those reading this, here are some links with information --
Synapse Biomedical Information (site includes Medicare information and locations with expertise)
Important Patient Q&A Document (Dr. Onders at The Cleveland Clinic is the developer of the DPS for people with ALS)

C'mon healthcare delivery system. It's past time to start delivering for people with ALS! This is an early intervention that is a much simpler decision than those later interventions. When dealing with ALS, "first do no harm" involves some added responsibilities.

Tuesday, October 4, 2011

Now, Can The Organizations Execute?

That was "organizations" (plural). Please. It's all in the execution now.


Wednesday, September 28, 2011

Tuesday, September 27, 2011

Are The Right Agencies Doing The Right Things?

A perspective on some of the proposed "enhancements" to the CDC's ATSDR ALS Registry...

http://alsspreadtheword.blogspot.com/2011/09/whoa-government-is-moving-too-fast.html

It's troubling to see such expensive "feature-itis" growing from a project that needs to be focused on a successful core mission.

Sunday, September 25, 2011

NFL ALS Roll Call Grows - September, 2011 Edition

Steve Gleason
Eric Scroggins
O.J. Brigance
Glenn Montgomery
Pete Duranko
Peter Demmerle
Steve Smith
Tony Proudfoot (CFL)
Bob Waters
Matt Hazeltine
Gary Lewis
Orlando Thomas
Wally Hilgenberg
Kevin Turner
Bob Hohn

There are thousands more who have played college football.

This mystery needs to be solved!

p.s. If anyone knows of other NFL players who have been stricken with ALS, please let me know. Thanks.

He Continues To Wow Us

Five years ago Steve Gleason wowed his city and a world of football fans with a blocked punt. Today he wows everyone involved in the fight against ALS with his attitude and courage.

Dan Dierdorf broke the news on the CBS pregame show, and he was visibly moved.


The troubling list of NFL players with ALS grows. In the meantime, we watch Steve Gleason make some remarkable good come from the rotten cards he was dealt.

Friday, September 16, 2011

When Dealing With ALS, It Pays To Read Things Twice

When I read Tuesdays With Morrie, I found warm messages on values and life. When I read it a second time, I found wisdom that I didn't see the first time. I saw a wise man's path through the ALS journey.

Yesterday there was a beautiful, short biographical piece on Lou Gehrig written by Reinhardt Krause in Investor's Business Daily --


The first reading reminded me of much that I knew about the remarkable Lou Gehrig. The second reading turned on some lights in my mind on how much Lou Gehrig's life teaches us personally on winning and achieving greatness. The fight against ALS has not yet been won, and "greatness" is not a concept would be associated with our teams fighting ALS today.

Please read the article. If you are involved in the fight against ALS, please read the article twice. The self-help is right under our noses.

Sunday, September 11, 2011

We Remember

As we remember the all of the heroes lost ten years ago today, here is a piece that speaks volumes about the American spirit --

HETRICK: Ten years after 9/11, keep doing what you do | 2011-09-10 | Indianapolis Business Journal | IBJ.com

May we achieve the national greatness that we saw in so many individuals ten years ago today. May that greatness include finding a treatment for ALS. We have lost over 60,000 Americans to ALS in the last decade, many of whom served bravely defending our country (and were never counted in our casualties of war) or served us all in public safety roles.

Saturday, September 10, 2011

Will People With ALS Get The FDA They Need From This?

Here is some reading about the FDA's latest PDUFA reauthorization proposal. If you're not familiar with PDUFA, google and enjoy the trip on how we have chosen to fund and improve the FDA over the past decades. Here is some overview information on the PDUFA V Technical Agreement.

Below are some organizational perspectives.

National Health Council Announcement

The National Health Council applauds the FDA for agreeing to include in its recommendations three proposals championed by patients: the development of an objective, qualitative benefit-risk framework that includes robust patient input; the advancement of new therapies through the expanded use of biomarkers and patient-reported outcomes in clinical trials; and the creation of regulatory policy, procedures, and guidance to encourage the development of treatments for rare diseases.

All three of these components are necessary for advancing access to desperately needed treatments and cures. To exclude any one of these elements would be to offer a reauthorization package that would not meet the needs of patients – particularly those with few or no treatment options.

ALSA Announcement

Since July 2010, The ALS Association participated in monthly meetings with FDA officials to bring the perspective of people with ALS to the discussion on the reauthorization of PDUFA. Three key provisions advocated by The Association were included in the proposed agreement. They include:

•Developing Treatments for Rare Diseases: Directs FDA to issue regulatory policies and guidance that encourage the development of drugs to treat rare diseases and to include patients as active participants in this process.
•Assessing Risk-Benefit: Instructs the agency to develop an objective and qualitative framework for assessing risk and benefit during the drug review process and to ensure patient involvement. The provision takes into account the fact that people with ALS who have no treatment options and who will die in just two to five years have a much different view of risk and benefit than does someone with a treatable or chronic condition.
•Utilizing Biomarkers in Clinical Trials: The proposal encourages greater use of biomarkers and other markers, or “sign-posts,” to help determine risk and benefit when reviewing new drugs. Biomarkers have the potential to significantly accelerate access to new drugs by helping to determine much sooner whether a treatment works and in which patients.

Comments From A Pharmaceutical Industry Representative

PDUFA is only part of larger FDA reforms needed to improve public health.


____________________

It's not a done deal. There are aspects that are a matter of life and death for people with ALS who are currently without an effective treatment and without a biomarker. It's important that we all pay attention as this proposal moves forward. Mission has not been accomplished.

Also, for ALS, a disease without biomarkers, are we going to slow down trials that might be mixing biomarker theories with efficacy theories? Sure, it would be great to use proven biomarkers in efficacy trials, but ...

Thursday, September 8, 2011

Here Are Some Words On Health Activism

...from a health activist.





It's terrible that ALS has drafted another, but we're glad for Barbara Brenner's perspective. The fight is perhaps changing for the better very soon.

The plot definitely thickens.

...and Ron Leuty continues to pay attention to how the course of a disease may be changed thanks to enabling technologies...

Saturday, September 3, 2011

Patients Are Changing The Same Old Same Old

Organizations are joining the conversation.

Many thanks to Ron Leuty of the San Francisco Business Times for an interesting piece of journalism on patients taking the lead in clinical trial enrollment. Very seldom have we seen so many important perspectives reflected in one article related to ALS.


It's definitely worth your time to read!

ALS often steals a person's ability to speak. These patients will not let it silence their voices.

Tuesday, August 30, 2011

People With ALS Deserve A Little Good News!

...and they got some really good news tonight.


http://www.bizjournals.com/sanfrancisco/blog/biotech/2011/08/neuraltus-als-lou-gehrig.html?s=print

By Ron Leuty in the San Francisco Business Times

Neuraltus ALS drug wins FDA orphan and fast-track status!

Now to fill the few remaining seats in the trial so that we can see how fast the fast-track is.

Hot Off The Press - ALS Registry Annual Meeting Summary


The meeting notes from the 2010 annual ALS Registry meeting were put on the CDC's website last week, over nine months after the meeting.


We believe that the 2011 meeting may be happening this week. We hope that it doesn't again take almost a year to see the summary of the proceedings. People with ALS don't have that kind of time to wait!

Monday, August 29, 2011

Lights, Camera, Action To Defeat ALS

It's that time of year again for entries in the American Academy of Neurology 2012 Neuro Film Festival

http://www.aan.com/press/index.cfm?fuseaction=release.view&release=978

http://patients.aan.com/go/about/neurofilmfestival

Please spread the word. This is a great opportunity to raise some critical ALS awareness and have a little fun, too.

Friday, August 26, 2011

If You Were Recently Diagnosed With ALS, Your Best Friend May Be alstrials@partners.org



You need to get to a neurologist doing clinical trials because anything the
least bit "promising" would be experimental.


Jose Biller, M.D. upon diagnosing a patient with ALS in 1996


That was guidance that unfortunately not every patient hears on the day of diagnosis. It is important because every patient deserves the opportunity to make informed decisions regarding options to fight the incurable disease and to contribute to the knowledge of that disease for future patients. Those informed decisions cannot be made after the window of clinical-trial eligibility has slammed shut, and in the case of ALS, many patients are not even diagnosed until much of their eligibility window has passed. After 24 to 36 months after first symptoms, even if you're lucky enough to still be alive and kicking, most clinical trials won't accept you.

People with ALS get spotty information from their neurologists regarding clinical trial opportunities. Some are actually discouraged because a trial might have bad side effects. Some docs don't mention trials outside of their own institutions. Others don't mention clinical trials at their institutions. This is nuts. People with ALS are perfectly capable of making their own medical decisions. Having information is a critical part of making medical decisions. Withholding information does not help.

There is a well-kept secret that can be of immense help to patients and families who would like to make their own informed decisions. There is a clinical trial expert available via email at alstrials@partners.org or via phone at (877) 458-0631 Monday through Friday, 9 a.m. - 5 p.m. eastern time. This person is a physician and is knowledgeable on all ALS clinical trial possibilities. The position is funded by ALSA and NEALS (an ALS clinical trial incubator) and is a source for objective information to assist patients in their decisions.


Last week a message board told of a patient who was pursuing a clinical trial that was filling fast. She could not get a response from the investigator's email contact at clinicaltrials.gov. She contacted the clinical trial expert and got some action. That's progress!


Thursday, August 25, 2011

Show Some Compassion


If You Can Cure ALS, Then You Can Cure _____________ (Fill In The Blank)

The headline on the following article may seem ironic:

"Cause of ALS is Found, Brings Hope for a Cure for Alzheimer's Disease"


My first reaction was, "Well, what about hope for a cure for ALS?" ... then I read the article.

Moving Pictures, Inc. (MPI) has doubled the percentage of their profits that they donate to Alzheimer's and dementia research, excited by the recent scientific breakthrough from Northwestern University. A new University study published in Nature identifying evidence of a common cause in all forms of amyotrophic lateral sclerosis (ALS, better known as Lou Gehrig's disease), a neurodegenerative disease causing fatal paralysis, opens a door to a cure for a disease that has long stumped scientists.

True scientific breakthroughs that address ALS have been few and far between. It is acknowledged as a nettlesome, complex neurological challenge. Many feel that if you can solve the ALS problem, then you can solve a lot of neurological problems. This article was a testament to that theory.

It's wonderful that the wave of scientific ALS interest in the last week is leading to related scientific excitement for Alzheimer's and Parkinson's and other neurological conditions. Perhaps if we can unravel ALS to its common denominators then we can unravel a lot of other diseases that will make new drug development for ALS an attractive proposition.

Let's hope that companies like Neuraltus and Biogen Idec can bring their current "promising" ALS clinical trials to successful conclusions quickly so that possible therapies for ALS can be evaluated and delivered... then perhaps, they can leverage ALS success into success in other much larger patient groups. After all, if you can cure (or even treat) ALS, then you can cure ______.


Here Is A Nice Honor And Awareness-Raiser

Paul Wicks is indeed an innovator, and it's nice for people who read an MIT publication to see his work in the context of ALS.


In a time when ALS clinical trials finally may actually be doing something, the role of PatientsLikeMe will be interesting.

Tuesday, August 23, 2011

Let's Hope This Discovery Lives Up To The Headlines

Over the weekend we saw a lot of news coverage of the article in Nature regarding Dr. Siddique's work on a biomechanical commonality among the variations of ALS.



The headlines imply that "the cause" has been found. It's a biomechanical cause. It's not so actionable as when we learned that smoking caused cancer; however, it's an important element of understanding a disease that has been a medical mystery for far too long.

Saturday, August 13, 2011

THIS Is Why DOD Medical Research Makes Sense

Earlier this year Senator John McCain said,

“The aspects of the Defense Appropriations bill that need to be taken away, eliminated, are $300 million for medical research. I am sure the medical research is important, but it has nothing to do with national defense.”

Now take a look at this Marine's obituary --

http://www.tampabay.com/news/military/advocate-for-veterans-in-camp-lejeune-tainted-water-case-dies-at-age-72/1185584

Joe Moser is exactly why the DOD should be doing medical research.

If a defect in airplanes were causing those serving our country to be killed, you can bet the DOD would swarm over that problem, and rightly so. If a defect in a rifle were causing our soldiers to be killed, you can bet the DOD would be investigating and correcting that problem. If uniforms were causing horrible rashes, surely the DOD would be figuring that problem out. Why would we ignore ALS and breast cancer?

When service-related diseases are killing our Marines, medical research is a defense issue.

Friday, August 5, 2011

This Is Pretty Cool

...but we hope not too cold. We love people who make waves!




They Had No Idea That This Was Service-Related

And there is no excuse for our legislators to have no idea. This is an unacceptable "fringe benefit" of serving our nation in our military.

Mike keeps fighting for our country in ways that he never expected.

Tuesday, July 26, 2011

After All, It's All About The Patients

In the last few days patients have continued to lead the way.



The Good News



  • http://www.clinicaltrials.gov/ has traditionally been the best database of clinical trials, yet it was not completely accurate on the eligibility window for one ALS trial. Since ALS patients are seldom diagnosed quickly (that's a whole other story), they often have very little time in their clinical-trial eligibility windows by the time they know they have ALS. It's a big deal if a trial will accept patients up to 36 months (rather than 24) from onset. Patient communication to the trial sponsor resulted in an important update to an important clinical trial's listing -- Neuraltus NP-001 -- http://www.clinicaltrials.gov/ct2/show/NCT01281631?term=neuraltus+amyotrophic&rank=1


  • A new location was announced for the same clinical trial and within hours, our patient mapmaker had updated his work and communicated it to dozens of ALS websites.


  • Several patients chided ALSadvocacy for not being more specific when referring to an important phase III trial in a prior posting. Here you go. -- Biogen Idec Dexpramipexole http://www.clinicaltrials.gov/ct2/show/NCT01281189?term=dexpramipexole&rank=3


  • The facebook commentary regarding clinical trials has increased significantly. Patients are encouraging each other to "try out." Patients may or may not qualify, depending on how much damage the ALS has already done to them. The support and information being exchanged on facebook are impressive.

  • Patients are hearing from their own trial coordinators of an uptick in inquiries based on patient referrals.


  • Some trials provide unadvertised assistance with travel expenses. Patients are learning to ask.


  • Finally, there are rumblings that some ALS organizations may finally be working together to make a single best information source of clinical trial possibilities. Based on past performance, that's a refreshing new goal to prove that it really is all about the patients. If a reliable, up-to-date, easy-to-use source of ALS clinical trial information emerges, then there may still be hope that all organizations might cooperate on having one excellent source of information. Patients have been begging for inter-organizational cooperation for years. This is a chance to help patients and leverage resources.

The Bad News




  • There continue to be reports of large groups of patients at support groups and clinics who are not being informed of clinical trial options by their organizations and healthcare professionals.


  • The online information regarding clinical trials has been updated on some ALS organization sites but not on others, and obsolete information is worse than no information! ALSA chapter sites are not accurate at this time. There is an old saying that everything is harder with ALS, and this is an ironic source that reinforces the adage. ALSadvocacy hopes that organizations will remove the red-herring information and simply supply a link to http://www.clinicaltrials.gov/ct2/results?term=amyotrophic&recr=Open It's all about the patients, right? It seems unconscionable that a patient might miss a trial opportunity based on wrong information on a trusted organization's site.


  • Not all trial locations supply email addresses in addition to phone numbers for the contact people. A disease that steals the ability to speak would be served well with more appropriate communication options for patients to inquire about clinical trials.


  • Clinics that are holding trials may occasionally be forgetting to tell patients about other clinical trial options that are available elsewhere. In the same vein, are not-for-profit trial sponsors forgetting to tell patients about trials sponsored by for-profit pharmaceutical companies?

The Bottom Line

It should be all about the patients. There are some current clinical trials that actually may provide improvements to patients, but we'll never know until the trials are filled and completed. There are therapies that will be huge if they live up to their promise, but only the data will tell. Patients are leading the way in getting the information to their peers so that the seats will be filled. Our ALS and healthcare delivery and clinical trial organizations have a perfect opportunity to prove that they are all about the patients. Simply listen to your patients and act, quickly, please.


Thursday, July 21, 2011

Wow ... The Patients Are Taking Matters Into Their Own Hands

And they're making a big difference.

Right now there are some very interesting clinical trials being conducted for ALS. Over the years there have been hundreds of such "promising" trials, always with the quotation marks around "promising," and the disappointments and failures have been heartbreaking. This year it seems different. There are anecdotal reports of improvements in one trial. There is another that is a Phase III trial. Both are being led by some of the finest neurologists throughout the country. After all of the recent buzz and hope of finally removing the quotation marks from "promising," one fact remains -- we'll not know if these drugs will live up to the promise until the clinical trial seats are filled and the trials are completed.

One would think that patients with an untreatable, terminal disease would be knocking down the doors to get to these clinical trials. One would think. It seems that the process for getting information to ALS patients regarding clinical trials is horribly flawed. Many rely on their neurologists for information and guidance, yet often trials aren't introduced to patients and when they ask, the physician may not be familiar with the possibilities. Many rely on their trusted ALS organizations for information and guidance, yet we find a melange of outdated and incomplete information on their websites.

In the last few weeks, we have seen patients enrolled in trials taking matters into their own hands with some impressive results. Any patient who is enrolled in a clinical trial can be highly motivated to get the other seats in that trial filled so that it can move ahead. We see patients contacting others via social networking to encourage and inform regarding trials they are in. We see patients and caregivers asking trusted ALS organizations to speak up to patients regarding enrolling trials (and there have been some results). We see patients contributing to information online so that their peers can understand the relative advantages and disadvantages of trials. We even saw one patient who had traveled away from home for his monthly clinical trial infusion who dropped into a support group meeting in the clinical trial city. He found peers who were not aware of what was happening in their own back yard. One patient published a map online to show his peers all of the cities offering his trial. Facebook friends are posting information on walls wherever people with ALS and families might visit, and patients who do not qualify for trials are being a huge help to spread the word to others who might. Patients are posting their data at http://www.patientslikeme.com/ so that others may learn.

The system for spreading clinical trial information is broken. Some say there is no system. There is no excuse for a patient who qualifies for a clinical trial to be unaware of the possibility. Patients are leading the way right now. We hope that organizations and clinics and physicians will follow their lead and step it up a notch or ten!

Friday, July 15, 2011

Saturday, July 9, 2011

Diesel Pete, Rest In Peace

Pete Duranko has died from ALS.

http://www.denverpost.com/sports/ci_18446945

Diesel Pete made this college freshman think that national championships were the normal part of a college football experience. Many years later he showed this ALS advocate the meaning of courage when facing this beast of a disease.

Another of our best and brightest has been taken. When will we stop this madness?

Friday, July 8, 2011

We Hope And We Pray

Over the last two decades there have been hundreds of clinical trials for ALS drugs that have delivered nothing but disappointment. We continue to raise funds and awareness so that research may continue.

Over the last several weeks there have been early reports of some positive results. It's way to early to know if early reports mean anything, but it is a most encouraging buzz among some of the trial participants.

If you have ALS or if you know someone with ALS, this Neuraltus NP001 trial might be worth some serious consideration. One must be within 36 months of disease onset to be eligible. If travel is required to get to a trial site, we understand that there is some assistance available.

We hope and we pray that this portends a viable treatment for ALS that can be delivered quickly. Around 500 Americans with ALS die every month.

Wednesday, July 6, 2011

Swim The English Channel to Defeat ALS

This is pretty cool...

I'm writing to you because I hope will will help spread the word about a remarkable man taking on a remarkable effort for the benefit of ALS research .....

In August, Doug McConnell, 53, of Barrington, Ill, will attempt to become the 48th person over the age of 50 to successfully cross the temperamental English Channel. He will be swimming more than 21 grueling miles in memory of his father who passed away from ALS (Lou Gehrig's disease). Doug is using this epic effort to educate the public about ALS and raise funds for medical research at Northwestern University ’s Feinberg School of Medicine in Chicago.

McConnell truly loves swimming and appreciates physical challenges. As a child, during a family vacation to England, he saw the Channel and thought to himself that one day he'd swim across the busy waterway. He and friend Don Macdonald committed to this endurance challenge and have been training for this event for approximately two years.

In late 2009, Doug developed a medical setback – a severely herniated disc between two cervical vertebrae, resulting in the loss of all use of his left arm. After physical therapy and other unsuccessful treatments, Doug underwent a cervical disc replacement procedure. The surgery was successful. Today, Doug has regained strength in his arm and the ability to move his neck to breathe while swimming, and he swims without pain. This was not enough to stop Doug's pursuit of his dream.

Medtronic, the manufacturer of the disc that was implanted into Doug's body, has pledged up to $50,000 in support of Doug's efforts. He is well on his way to raising a significant amount of money.

We hope you will post the press release on your blog. Please feel free to contact me with any questions or if you need additional information.

Regards,

Beth Richman
Les Turner ALS Foundation
773-252-3069

Tuesday, July 5, 2011

Here Is More On The Theme, "Let Patients Help!"

Please read the article at this link!

http://fastercures.blogspot.com/2011/07/building-bridges-across-valley-of-death.html



...He talked about how CFF’s successes – more than 30 drugs in the development pipeline, four of them already FDA-approved – have been a result of holding its partners’ feet to the fire, and keeping patients at the table every step of the way to create the urgency Feigal talked about.

But it goes beyond just having a seat at the table. While patient presence is critical to humanizing research and speeding progress, patient voices can’t just be loud, they also have to be smart on the science and knowledgeable about the regulatory environment. The recently released paper Back to Basics: HIV/AIDS Advocacy as a Model for Catalyzing Change, co-authored by FasterCures and HCM Strategists, distills lessons learned from the HIV/AIDS movement that can be replicated to address today’s medical research advocacy challenges. HIV/AIDS activists were successful because they refused to accept that the system was unchangeable, and instead took the time to figure out what it should be and how it should work.



In case you didn't click on that Back to Basics: HIV/AIDS Advocacy as a Model for Catalyzing Change link, please do so. Perhaps there really is a roadmap after all.

Friday, July 1, 2011

We'll Never Hit A Home Run If We Don't Swing The Bat

Two years ago this weekend something happened in the United States that was wonderful. As we approached the Independence Day celebrations, traffic on this site was astronomical. People were searching for information on ALS. Each major network was doing news features on ALS, Lou Gehrig, or the 4 ALS celebrations being held on July 4 in all of our Major League Baseball parks. It was a good buzz that permeated our nation on a weekend when we feel good about being a nation that can do good things. It raised awareness of Lou Gehrig's Disease in a generation of baseball fans who weren't all that familiar with Lou Gehrig. It was a weekend when people talked to each other about ALS.

Michael Goldsmith was a remarkable man who made all of this happen with the wonderful help of Major League Baseball.



We need to rekindle the spirit delivery of Michael Goldsmith and make ALS an important part of every July 4 in our country. It's not about short-term fundraising. It's about raising ALS on America's radar on an important day when baseball and good works are as important as fireworks. It takes media savvy and interorganizational cooperation and hard work.

It's interesting. People like Michael Goldsmith with ALS understand the "how" and "why" of ALS awareness. Unfortunately they die all too quickly. Michael Goldsmith pitched a nice fastball right down the center for us to hit out of the park. Our ALS organizations need to get the bats off the shoulders and learn how to hit a home run like Michael Goldsmith.

Wednesday, June 29, 2011

With ALS, Patients Have To Take The Healthcare Bull By The Horns

Interesting perspectives from an empowered cancer patient...

"Let Patients Help!"








Thursday, June 16, 2011

It's Good To Remember Lou on His Birthday

And it will be great if we can rid the earth of this beast of a disease before his next birthday!

Wednesday, June 15, 2011

Eight Years... 50,000 American Funerals Later


Remember June 19, 2003? It was Lou Gehrig's 100th birthday. We passed out baseball cards and gathered donations for nice commemorative pins. We started to realize that there are a lot of young baseball fans who don't realize who Lou was. We raised awareness the way we always raised awareness -- with modest and diligent and sporadic efforts at small events.

We have lost around 16 Americans to Lou Gehrig's Diseae every day (seven days a week) in the last eight years. We still try to raise awareness in very modest ways, the way we always have.

ALS is a big problem. It's time for some big, effective, continuous efforts to bring the ALS problem into serious focus. If most people don't know ALS is still around, we certainly can't blame them for ignoring it.

Shame on us if we keep raising awareness the way we always raised awareness.

Tuesday, June 7, 2011

Advocacy Action Requested

Following is from an email received from ALSA:


Earlier this year, we requested that you contact your Members of Congress to urge them not to eliminate the ALS Research Program at the Department of Defense. Thanks to your outreach, Congress provided $8 million for the program this year! You made a difference. But we need your help again.

The House Appropriations Committee is scheduled to vote on the FY 2012 Department of Defense Appropriations Act on Tuesday, June 14 and we have learned that an amendment may be offered that would eliminate the ALSRP next year. We cannot let this amendment pass. If it passes, at least $8 million that is so urgently needed for research no longer would be available. It would mean that research specifically designed to find a treatment would not take place.

Please contact your Representative TODAY! Tell them to save the ALSRP and work with the Appropriations Committee to make sure our government continues to support the fight to find a treatment and cure as soon as possible!

You can email your Representative directly from our website, here. Do it today. And tell your friends, family and everyone else you know to do the same. Your outreach made a difference earlier this year and we are confident that it can again -- but only if you take action today.

Thank you!

Friday, June 3, 2011

Let's Stop Shooting Our Cause In The Foot

Years ago a really smart person came up with the tagline, "Every 90 minutes someone is diagnosed with ALS. Every 90 minutes someone else dies from ALS." That's a great, concrete, attention-grabber. Unfortunately it doesn't tell the whole story.

That "90 minutes" is based on U.S. statistics only. Around 6000 people in the U.S. are diagnosed with ALS every year and a like number die from ALS. That's around 16 U.S. diagnoses and deaths per day, or one every 90 minutes. Great. But it's U.S. only!

ALS is a global problem.

The United States has around 4.5% of the world's population according to the U.S. Census Bureau.

So... assuming that the incidence of ALS is similar globally, let's try some arithmetic...



6000 / .045 = 133,333 new cases and funerals annually worldwide. Whoa.

133,333 / 365 = 365 new cases and funerals daily worldwide. Wow.

365 / 24 = 15 new cases and funerals per hour worldwide. Holy moley.

60 / 15 means that every 4 minutes someone is diagnosed with ALS. Every 4 minutes someone else dies from ALS. Yikes.

So let's get it right. Either "Every 90 minutes someone in the U.S. is diagnosed with ALS. Every 90 minutes someone else in the U.S. dies from ALS," or "Every 4 minutes someone is diagnosed with ALS. Every 4 minutes someone else dies from ALS."

Tick, tick, tick.




Or maybe, "There are well over 100 thousand new cases of ALS annually. There are well over 100 thousand others who died from ALS last year."

p.s. If my arithmetic is off, I'm all ears.





p.p.s. Check out the first paragraph http://lillypad.lilly.com/corporate-responsibility/fighting-hunger-bold-goals-moral-obligation They made a problem come to life!




p.p.p.s. 1pm EDT Fixed the arith. Got carried away with zeroes. Sorry about that. Phew!

We Need More Of This Attitude!

Thursday, June 2, 2011

It Doesn't Take Long

Two years ago this coming July 4 we celebrated the 70th anniversary of Lou Gehrig's farewell speech.

Today is the 70th anniversary of Lou's death.

It didn't take long for a man to go from baseball mega-superstar to the grave. This disease persists. The outcome today is much the same that Lou Gehrig experienced.

Perhaps our tagline should be, "ALS -- I'll bet you thought someone had cured it by now."

In the time since 1941 think of the advances in science and medicine and life. Polio and smallpox were conquered. Cancers are often treatable. MS patients have a long life expectancy. We have tiny cellphones and make long distance calls at will. We have an internet. We have dishwashers and wonderful television pictures and trash bags. Why have we not figured out ALS?

These Are Not Sickly People

They are hale and hardy and playing golf and sailing one month, and suddenly ALS hits. We need more ways to show this. Here is one created by a gentleman with ALS.




Every healthy person needs to squirm a little. Nobody is immune.

Wednesday, June 1, 2011

We Hope Scott Pelley Remembers Steve and ALS


If you saw the 60 Minutes piece on the 21st Century Snake Oil , then you saw Steve Watters. He was an ALS patient who went undercover with Scott Pelley to expose the con man with a bogus stem cell cure for a price. Steve was always a thoughtful contributor on ALS message boards, and he turned out to be a huge contributor to the awareness of ALS in his role on that 60 Minutes segment.

Steve Watters has died.

Another nice person. Another achiever. Another contributor. Another funeral.

Friday, May 27, 2011

What Are The Odds?

ALS advocacy involves starting the simple conversations that cause so many connections to ALS to come to light.

Last week alsadvocacy was in seminars with 20 people. Wearing a www.stealingpieces.org shirt to breakfast one morning was a conversation starter. A couple said that they had known General Tom Mikolajcik, a hero in the fight against ALS who enabled the service-related benefits for veterans stricken with ALS.

Later the next day prayers were requested for a friend of a meeting leader who had just received a serious diagnosis. It was ALS. Unbelievable.

That caused even more conversations in the group about the disease. One gentleman shared that he had lost a college roommate to ALS. Another talked of his uncle who died from it.

Twenty people. So many ALS connections. This disease is not as rare as people like to think. It strikes and kills quickly. It then moves on to strike and kill more people. We must keep the conversations and connections alive so that the world might realize that it is a problem urgently in need of a solution. We must speak up for all of those who have been silenced by ALS.

The Leadership And Results Are Clearly Refreshing

This message was posted on the ALS Association's facebook wall... from the ALSA President and CEO --
I had the distinct pleasure this afternoon of receiving a phone call from Eddie Martiny, General Manager, and Marc Sherman, FM Operations Manager, of the Clear Channel stations in Houston. They were calling at the request of Mr. John Hogan, President of Clear Channel Radio, who received my letter asking for a public apology. They were interested, compassionate and want to help us with an awareness campaign through their sister stations. They also gave me permission to share with you the words of the public apology that was made on KKRW station on Tuesday:

“KKRW and Walton & Johnson would like to apologize for insensitive remarks about Stephen Hawking’s physical condition that have caused distress to people with disabilities, including those with ALS – which Professor Hawking suffers from – and their families. We recognize that there are many people in Houston who suffer from serious, debilitating illnesses and no offense to them was intended.”

They are working on producing public service announcements, and will run them on all of their stations during the next couple of weeks. I urge all of you to join me in thanking them for their concern and their response to the ALS community. I want to go on record with a huge thank you to the management of Clear Channel Communications, who recognizes the mistake that was made and are now doing everything they can to help us.



Clearly the use of online media to get Clear Channel's attention along with the refreshing ALSA leadership to meet the problem head-on made a difference.

This is ALS advocacy. This is ALS awareness. This is newsworthy. This is a huge step in a good direction.