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 --

ALSA Announcement

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)

Information Letter

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.

Is There A Lesson For The Fight Against ALS Here?

Now, Can The Organizations Execute?

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

"The Facts Are Not Discouraging"

And that's a big deal with an ALS trial.

CNN covers news from the Neuralstem Phase I trial --

http://www.cnn.com/2011/09/28/health/early-als-trial-results-encouraging/index.html

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.

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.

http://www.nola.com/saints/index.ssf/2011/09/new_orleans_saints_cult_hero_s.html

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.

http://www.team-gleason.com/

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 --

What Put Gehrig First? Quiet Dignity, Iron Will - Investors.com

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.

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.

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 ...

Here Are Some Words On Health Activism

...from a health activist.

http://barbarabrenner.net/?p=299

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...

http://www.bizjournals.com/sanfrancisco/blog/biotech/2011/09/als-lou-gehrig-facebook-twitter-youtube.html

Don't you love it when the plot really thickens?

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.

http://www.bizjournals.com/sanfrancisco/print-edition/2011/09/02/patients-enlist-in-fight-against-als.html

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.

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.

http://wwwn.cdc.gov/als/Download/2010%20ALS%20Annual%20Meeting%20Summary%20Report.pdf

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!

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.

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!

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"

http://www.sfgate.com/cgi-bin/article.cgi?f=/g/a/2011/08/23/prweb8736207.DTL

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.

http://www.technologyreview.com/tr35/profile.aspx?trid=1080&p1=TR35

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

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.

Short Article in Time

Helpful Explanation from MNDA

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.

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.

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.

It's Still A Mysterious And Deadly Disease

http://www.wtae.com/r/28744335/detail.html

Interesting video from Pittsburgh's South Side.

Governor Cellucci's Public Service Continues