To: My Niece Maggie …

When everyone else did “normal” themed 8th grade technology projects … you took the road less traveled and produced a very educational and moving video about ALS (Lou Gehrig’s disease) and your Uncle Tom.  As you are well aware, taking the road less traveled seems to be a trait we see a lot in our family.

I’m so glad (and very proud) that you took the time and energy to learn about this very rare, terminal disease and are using this project to educate others about something that has been with us for 140+ years, has no life extending treatments and is fatal for 15-18 people per day in the US.

I’m looking forward very much to visiting you all in FL on June 1st - see you soon!

Love,

Uncle Tom 

Click the link below to watch the video …

http://www.youtube.com/watch?v=0Cv8Q5CgiaE

Three very moving and educational videos …

It is videos like these (and hundreds just like them) that begin to explain why I’ve been wearing a blue bracelet with very bold white print for over 2 years that says:  ”Never Ever Give Up!”

It’s been 140 years since ALS was discovered and there are no life extending treatments … we really have to start moving the ball down the field.

1.  http://www.youtube.com/watch?v=He6HSwgcK7w&feature=share

2.  http://www.youtube.com/watch?v=JNZcC_3YLuo

3.  http://www.youtube.com/watch?v=qTlFWw3k6Iw

I made it in to another Clinical Trial …

After failing to qualify on my first attempt to be accepted into a second ALS Clinical Trial …

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The Cytokinetics Tirasemtiv Clinical Trial http://clinicaltrials.gov/ct2/show/NCT01709149?term=tirasemtiv&rank=1  

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The good folks at UVA gave me another shot today at passing the Slow Vital Capacity (SVC) lung/breathing test. 

I was given 3 more attempts to achieve a score of 60 or better.  The first time my scores were 48, 51 and 53 and I failed.  

Today my first try was measured at 51 … my second try was measured at 53 and it seemed once again that I would fail and not be allowed to participate in this very promising trial.  We began to discuss not taking a third try and just asking the Trial Sponsor for a possible exception but decided to make one last attempt.

Not sure what I did that was different but the third attempt was a 61 and I’ve qualified!

Murphy’s Law in reverse - What a Deal!

FEB 25th FDA ALS Specific Public Meeting …

Details about the meeting and Draft Agenda:

IMPORTANT: If you are unable to attend the meeting in person on Monday, February 25, 2013 you may view the live webcast at https://collaboration.fda.gov/als/ . There is no opportunity to ask questions during the webcast and we encourage you to submit your comments to [Docket No. FDA-2013-N-0035].

This hearing has generated an extraordinary amount of interest within the patient and health professional community.  As a result over sixty (60) speakers registered to offer comments.  In consultation with the ALS community, we were advised to limit the duration of each speaker’s testimony.  Each registered speaker has a maximum time limit of six (6) minutes.  Please adhere to this time frame out of respect for your fellow speakers. 

==============================

DRAFT AGENDA

Morning Session

9:00 a.m. Welcome & Overview and Rules of Part 15 Public Meeting TBD

9:10 a.m. Testimony from ALS community

• Jane Gilbert
• Lucie Bruijn, PhD
• Ken McGunagle
• Annie Kennedy
• Sally Okun, RN, MMHS
• Tom Murphy
• Wendy Abrams
• Rob Goldstein or Dr. Steve Perrin
• Stephen and Barbara Byer

10:10 a.m. Break

10:20 a.m. Testimony from ALS community

• Benjamin Rix Brooks  MD
• Stanley H. Appel MD
• Clive Svendsen, PhD
• Dr. Dominic D’Agostino
• Donna Bozyczko-Coyne, Ph.D.
• Sara M. Feldman, PT, MA, DPT, ATP
• Mary Paolone, MSRN
• Latoya Weaver, PSC
• Keith Segalman MD and Paula Kovarick
• Jonathan D. Glass, MD

11:20 a.m. Break

11:30 a.m. Testimony from ALS community

• Debra Quinn
• Miss Kristin M. Quinn
• Michelle Farr
• Christopher Hanes
• Karen Bessel DiGiacomo 
• Robert Anderson
• Pete Johnson
• Hollister J. Lindley 
• Wes Rose, Ph.D.

12:30 Lunch 

Afternoon Session

1:15 p.m. Testimony from ALS community

• Stephen Kolb
• Debbie Gattoni
• Mary Catherine Collet
• Anthony Peter Conway
• Don Farrell
• Peter Frates
• Nancy Frates
• Fred Carlson
• Carey and John McNeeley 
• Clare Durrett

2:15 p.m. Testimony from ALS community

• Hollister Lindley 
• Dick Jacobson
• Donald Farrell and Joan Farrell
• Amanda Makki
• Alan G. Croasdale
• Diane Blantz
• Ms. Kerry Luck-Torry
• Anthony Topazi
• Michael Gollin
• John L Johnson Sr

3:15 p.m Break

3:30 p.m. Testimony from ALS community

• John H Stadler
• Suzette Malveaux
• Alison Greenspan
• Kathleen Thompson
• Tony Gegor 
• Ted Harada
• Mark Eugene Sawyer
• Andrea Kline, RN-BC, MAG
• Charles Scott Guenard
• Scott Johnson
• Kathy Demarino
• Prof. Trevor G Marshall, PhD
• RJ Tesi, MD 
• Jason R. Williams, MD

5:00 p.m. Closing Remarks

TBD

ALS Specific FDA Hearing 2/25/2013 - need your voices heard …

Please read this entire post … I think we all have a tremendous opportunity to give the FDA a very good idea related to a very critical decision point in the treatment development arena for ALS — the BENEFIT-RISK DECISION POINT.

We’ve all seen the “risks” this community seems to be rightly willing to take when the alternative is certain agony and death — Sodium Chlorite and this 2012 ABC News article is a good example — as is ALS Patients Conduct DIY Drug Trials  and of course it is the risk tolerance of this community that spurred the creation of ALS Untangled.

I ask that all the PALS, Caregivers and other stakeholders in this community (thousands of us) provide the FDA with your personal tolerance for risk related to ALS Treatments.  Instructions for how to submit your inputs are at the very end of this post.

Here are mine (very simple):

• I believe in Early Approval and/or Expanded Access for existing treatments that have already proven safe in a Phase II trial and might work at extending the lives of those who cannot wait the years it takes for those treatments to become fully FDA approved. 

• Bring an ALS Patient Perspective and reinforce the fact that dying patients are not well served when regulatory conditions deny them the opportunity to pursue proven-safe treatments that may help them survive. 

• Work together closely with the pharmaceutical companies, Advocacy Organizations and Regulators to make these drugs/treatments available without further delay, to as many willing/informed ALS patients as possible.

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The FDA is holding this hearing to seek input from ALS patients, caregivers, advocates, academia, health care providers, the pharmaceutical industry, and other interested parties on their experience with, concerns about, and suggestions for, the way FDA regulates the scientific evaluation of, marketing authorization for, and post-marketing surveillance of, drugs for treatment of ALS. The scope of the presentations may include, but are not limited to, nonclinical testing, clinical trials, and decisions regarding marketing authorization and post-marketing surveillance of products for the diagnosis or treatment of this disease.  The input from this public hearing will help inform the work of FDA offices that review applications for drugs for the treatment of ALS.

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If you submit a “Risk Tolerance in ALS” comment - please send me a quick note at tmurphy58@gmail.com — I’d like to get a feel for the magnitude of responses prior to 2/25 … many thanks!

HOW TO SUBMIT ELECTRONIC COMMENTS 

go to http://www.regulations.gov/#!docketDetail;D=FDA-2013-N-0035

CLICK THE BLUE BUTTON Comment Now 
FILL IN INFORMATION. Note the session will time out in twenty minutes, so you might want to copy/paste. 
YOU CAN SIGN UP FOR EMAIL ALERTS WHEN COMMENTS ARE POSTED.

Passing of Michael Schwartz …

Michael was the Chief of Staff for Senator Tom Coburn for 15 years. Although we first met last March and spent just two days together on Capitol Hill helping to raise awareness about ALS and perhaps move some folks in the House and Senate to legislative action — Michael Schwartz made a very big impression on me and I thank him very much for that. 

When we walked the Halls of Congress together it was amazing the number of times Mike was stopped by others just to say Hi and ask how he was doing and how many times he would stop others to ask about them and their families … at some points, I wasn’t sure that we would ever make it to our meetings on time. :)

Michael passed away on Sunday February 3rd — http://www.nationalreview.com/corner/339766/when-we-have-our-eyes-fixed-unseen-inwardly-we-are-being-renewed-kathryn-jean-lopez — take some time to listen to the tribute to Michael by Senator Tom Coburn.

RIP Michael Schwartz … You were a very inspirational person … 

Steve Gleason makes plea for $5 million in donations to fight ALS before Super Bowl

This is a tremendous plea by Steve Gleason - but this ALS Community (individuals, Non-Profits, Federal Agencies, Research Organizations, Advocacy Organizations, etc.) have been appropriating, collecting and donating huge amounts of funds for over 100 years and I wonder if this is just going to be another heroic “individual” effort or an effort that will be executed collaboratively with other organizations that have existing initiatives underway that are all UNDERFUNDED. I was diagnosed with ALS on 12/8/2010 and I am very passionate about this issue of “Teams” working together in lock step AND TAKING ADVANTAGE OF EXISTING INFORMATION, ACCOMPLISHMENTS AND FUNDING to truly bring about “a research revolution to put new medical treatments on the market” as soon as possible.

I think that it would be great if Team Gleason would make their plan PUBLIC for what will actually be done with the $5M that will be collected/donated and what collaborative activities have been planned and committed to.

Will the $5M be used to work collaboratively/in partnership with any of these 15 folks below or will it just be another “Silo of ALS Excellence”? …

ALS Treat Us Now - http://www.facebook.com/pages/ALS-Treat-Us-Now/220968231317148

Or the ALS Emergency Treatment Fund - http://alsetf.org/

Or the ALS Therapy Development Institute (ALS TDI) - http://www.facebook.com/ALSTherapyDevelopmentInstitute

Or the Northeast ALS Consortium (NEALS) - http://www.facebook.com/NortheastALSConsortium

Or the National Organization for Rare Disorders (NORD) -http://www.facebook.com/NationalOrganizationforRareDisorders

Or the ALS Association - http://www.facebook.com/alsassociation

Or the MDA ALS Center of Hope - http://www.facebook.com/pages/MDA-ALS-Center-of-Hope/142645842465243

Or the Prize4life - http://www.facebook.com/Prize4Life

Or the Abigail Alliance - http://www.abigail-alliance.org/

Or Team R.A.R.E - http://globalgenes.org/who-we-are-2/

Or the Les Turner ALS Foundation - http://www.facebook.com/LesTurnerALS

Or the Brigance Brigade - http://www.facebook.com/BriganceBrigade

Or the Pooled Resource Open-Access ALS Clinical Trials Database (PRO-ACT) - https://nctu.partners.org/ProACT

Or the International Alliance of ALS/MND Associations - http://www.alsmndalliance.org/

Or Patients Like Me (PLM) - http://www.patientslikeme.com/

Steve Gleason makes plea for $5 million in donations to fight ALS before Super Bowl

shar.es

SportsNOLA.com provides news on all New Orleans sports including the Saints, Hornets, LSU Tigers, Tulane Green Wave, High School Sports and everything in between.

ALS “Cure” or Life-Extending Treatments/Therapies?

Click on the URL links below to see the recent ALS stories and video’s …

RE:  ‘No White Flags’: Gleason builds movement against ALS

David Meeks, USA TODAY Sports12:19a.m. EST January 29, 2013

http://www.usatoday.com/story/sports/nfl/2013/01/28/steve-gleason-super-bowl-als/1872379/

NFL players’ dramatic video highlights ravages of ALS

David Meeks, USA TODAY Sports4:41p.m. EST January 28, 2013

What we need is more focus on accelerating life-extending TREATMENTS for folks with ALS!  

MS is diagnosed in about 5,000 people per year (same as ALS) but there are 700,000+ folks living with MS each day compared to 25-30,000 with ALS.  This is because there are several life-extending treatments for MS patients that help them live long productive lives and ZERO for ALS patients after 100+ years (the HIV/AIDS scenario is very similar).  We need the FDA, pharmaceutical companies, Advocacy organizations and researchers to have life extension as a primary goal and not the “cure” or making people comfortable before they pass in 2-4 years on the average.

How much longer will we have to wait for FDA approval of the U.S. Trial …

For all of us folks with ALS — we can’t afford “waiting time” when we measure our remaining lives in days and months … do we all need to begin a trek to sit on the doorstep of Hadassah Medical Center in Jerusalem (“the Promised Land”) if we can’t count on an aggressive and expanded trial approval from the U.S.?

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ALS Stem Cell Trial in Israel Is ‘Accelerated’

http://alsn.mda.org//news/als-stem-cell-trial-israel-accelerated

Thanks to promising early safety results, BrainStorm’s phase 1-2 safety trial of adult stem cells has been advanced to a phase 2a dose-escalating trial. 

The Israeli Ministry of Health has given BrainStorm Cell Therapeutics approval to accelerate its current phase 1-2 safety trial of the company’s NurOwn stem cell therapy in amyotrophic lateral sclerosis (ALS).

A new phase 2a dose-escalating trial, designed to evaluate the safety and preliminary efficacy of the experimental therapy in ALS, will be launched immediately at the Hadassah Medical Center in Jerusalem. Last year, the company reported preliminary data that showed the experimental therapy was well-tolerated, and that some functional improvements were seen in participants.

According to a BrainStorm press release issued Jan. 7, 2013, approval for the acceleration was based on evaluation of the first 12 (of an expected total of 24) study participants in the company’s phase 1-2 trial at Hadassah.  

In the phase 2a trial, the second group of 12 participants will receive combined intramuscular and intrathecal (into the fluid around the spinal cord) administration of NurOwn cells, with increasing doses. The study participants, who already have been recruited, will be followed for three to six months after transplantation.

BrainStorm plans to add US trial sites

Pending FDA approval, BrainStorm plans to add U.S. trial sites to the ongoing NurOwn clinical trial in 2013. Prospective sites include Massachusetts General Hospital in Boston and the University of Massachusetts Medical School in Worcester, Mass.

Several steps are necessary before NurOwn stem cells can be tested in the United States. These include:

• development of a trial design;
• identification of treatment production facilities;
• completion of a formal collaborative agreement between BrainStorm and the participating institutions; and
• FDA approval of the new trials.  

If you missed this on ESPN this weekend … take the time to watch it

http://espn.go.com/video/clip?categoryid=2378529&id=8815080

Time is lives for those dealing with ALS … What Next?

I was diagnosed with ALS on 12/8/2010 (with initial symptoms in January-February 2010 — While on the EMPOWER Dexpramipexole clinical trial my ALSFRS-R stayed steady for 18 months without losing a point (I’m told that it is not unusual for an ALS patient to typically lose a point per month) - were there others like this that may have responded positively to the treatment? 

Perhaps folks saw this quote from the ALS Association’s Chief Scientist in the Wall Street Journal on 1/3/2013?

“The important thing we’re learning about ALS is it’s a spectrum of diseases,” Lucie Bruijn, chief scientist at The ALS Association, said recently. “There are some who respond to treatment and some who don’t; at the moment, we lump all the people together and that might be one of the reasons why the trials are failing.”

Given this lumping all the people together, what was the percentage of folks that may have responded positively?  Given an ALS patient death rate of 15/day, approximately 8,100 folks will have passed away during this 18 month period - perhaps I would have been one of those 8,100 had I not been in this Trial?

This has been the relevant issue and “problem” with ALS for well over 100 years and still NOT ONE treatment that really delays progression or improves quality of life for any of us that have been “lucky” enough to have this horrific disease.

It’s time for some OUT-OF-THE-BOX thinking, solutions and ACTIONS.

See the ALS Advocacy Blogspot from 1/4/2013:  Yesterday Was A Lemon Of A Day - http://als-advocacy.blogspot.com/2013/01/yesterday-was-lemon-of-day.html

The blog read as follows and I AM STRONGLY SUPPORTIVE …

Today we need to empower their efforts by learning lessons from the trial process and making positive changes.

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

The traditional clinical trial sequence is to have two safety phases followed by efficacy phases.  That makes a lot of sense for most diseases, but ALS isn’t most diseases. 

We need to face the fact that even a Phase I or II safety trial is an efficacy trial in the eyes of volunteers with ALS.  They’re looking for improvements (or more typically, a sense of getting worse less quickly) from the very first dose.

The large amounts of information collected each month by Biogen Idec during this trial needs to be made available to the entire “ALS Community” immediately and as well as other vast stores of ALS specific data/information that has been collected for years in “stovepipes” and “silos” by the National ALS Registry, Northwestern University ALS Biogistry, ALSTDI databases, Neuraltus NP001 trial data, Cytokinetics CK-357 trial data, the Veteran’s Administration, Patients Like Me (PLM) Public Registry, the Familial ALS Registry, the ALS Clinical Assessment, Research and Education (C.A.R.E.) Program, the ALSoD, the ALS Mutation Database, the ALS Gene Database, the Prize4Life PRO-ACT database, NEALS (6 databases),   the National Institute of Neurological Disorders and Stroke (NINDS) NCT00362362, Athena Diagnostics, Baylor University ALS DNA Banking Project, University of Miami ARC, DNA, Blood and Skin Cell Repository for Research on ALS and Related Neurodegenerative Disorders at Mayo Clinic Florida, Massachusetts General Hospital Trial Databases, University of Virginia Trial Databases, the Longitudinal Study of ALS Biomarkers at Mayo Clinic, Neuralstem, Brainstorm Therapeutics and Emory University collected data, the International Alliance of ALS/MND Associations data collections and many, many more too numerous to name here.

The more data dots that we can share together and provide for the entire ALS R&D community the higher the probability that patterns in the data will become easier for these scientists and researchers to isolate and more and more dots can be connected leading to more INFORMATION and KNOWLEDGE and much faster PROACTIVE DECISION MAKING.  

Over 100 years with no viable treatment progress for a disease that takes the lives of the afflicted within an average of 2-4 years —- IS THERE ANYONE WILLING TO UNITE, COLLABORATE AND OPENLY SHARE WITH OTHERS AND STEP UP TO FIND A WAY TO MOVE THIS BALL FORWARD AS A COMMUNITY AND NOT A BUNCH OF “INDIVIDUAL” AND “SEPARATE” ENTITIES AND/OR ORGANIZATIONS?

Geeeez … What a Deal - Key ALS Clinical Trial Considered a FAILURE

I’ve been in the Biogen Idec EMPOWER Phase 3 Dexpramipexole Trial since May 2011 and have been thinking that the fact that my Functional Rating Scale (FRS) disease progression hadn’t changed in 18 months meant that perhaps the ALS Community finally had a viable treatment option … Murphy’s Law is in play again …

I’m all in with ALSTDI regarding this news:

Statement from the ALS Therapy Development Institute:

http://www.als.net/Media/5426/News/

This morning, Biogen Idec announced that dexpramipexole failed to demonstrate sufficient effectiveness in slowing down the progression of ALS or extending survival in a phase III clinical trial.

The information put out to date is limited to only that which appeared in the press release. We need to see the actual data to understand the implications of these findings. We hope that Biogen will release more results soon.

If there is any “good news” here, it is that now many of us with ALS that were part of the 900+ in this failed trial may have the chance to participate in the 49 currently “Open” ALS Clinical Trials (see listing at http://clinicaltrials.gov/ct2/results?recr=Open&no_unk=Y&cond=%22Amyotrophic+Lateral+Sclerosis%22&pg=1  - if we can qualify.  

Since these trials are currently the ONLY possible treatment avenue available to ALS patients they are the only hope we have.  The trials on my radar right now are:

• Cytokinetics CK-357/Tirasemtiv  http://clinicaltrials.gov/ct2/show/NCT01709149?term=tirasemtiv&rank=1
• Neuraltus NP001 http://www.neuraltus.com/pages/news_rel10_30_12.html
• Neuralstem  http://www.neuralstem.com/cell-therapy-for-als
• Brainstorm Cell Therapeutics   http://www.brainstorm-cell.com/index.php/clinical-trials/us-clinical-trials

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Top-line results from Biogen Idec’s dexpramipexole Phase 3 trial on amyotrophic lateral sclerosis

Published on January 3, 2013 at 11:55 PM · 

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Bloomberg news article: Biogen Sinks as ALS Drug Fails to Show Efficacy in Trial

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2013: Year of the Stem Cell for ALS?

Six Biotech Developments in 2012 and Predictions for Next Year

By Paul Diehl, About.com Guide

http://biotech.about.com/od/Biotech-and-society/a/Six-Biotech-Developments-In-2012-And-Predictions-For-Next-Year.htm

Here is the prediction for 2013 related to Stem Cell Treatments – now if we can just find some near-term treatments that slow the progression of ALS enough for a lot of us live long enough to make it to 2014.

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Stem Cells Continues Their Advance

In 2012, we saw Drs. Gurdon and Yamanaka receive the Nobel Prize in Medicine for their work on reprogramming normal body cells to make stem cells, known as induced pluripotent stem cells (iPSC). Promising stem cell research has been moving forward too, such as the study working out how to use stem cells to regenerate neurons, and the recently published work using iPSC to correct amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, in mice. Also, preliminary human trials, such as UCSF and StemCells, Inc. collaboration treating 4 children affected with a genetic neural degenerative disease, and another trial correcting spinal injury are showing real promise.

As a result of this progress, it might be reasonable to expect big advances in the stem cell field in 2013. The title of a recent article in the Atlantic, 2013: Year of the Stem Cell, seems to suggest this but actually concludes on a more realistic assessment, stating, “2013 probably won’t be the year that the big miracles anticipated from stem cell research will come to pass. But it does promise to be one full of more small, but significant advances toward that future goal.” This is my prediction also.

Stem cell research as been gaining some significant momentum and, in 2013, I expect to see promising new results that will likely lead to therapeutic trials in subsequent years. For a perspective on the potential, listen to UC San Diego’s Dr. Larry Goldstein comments on the California Institute of Regenerative Medicine’s website. As he notes, “If the public continues to adequately fund research with stem cells we will see breakthroughs that are absolutely unexpected and that will change the way that we deliver medicine.”

Also, you might take a look at The Range of Stem Cell Technology and Experts Assess the Current Status of Stem Cell Research for a general overview of Stem Cell technology.

It’s not just happening to ALS Patients …

I enthusiastically signed this petition yesterday and would encourage others to do the same … http://www.change.org/petitions/fda-please-approve-the-medicine-my-boys-need-to-survive-both-of-my-sons-deserve-to-live?utm_campaign=action_box&utm_medium=twitter&utm_source=share_petition

My two oldest children, Max and Austin, have a lethal genetic disease called Duchenne Muscular Dystrophy (DMD). Right now, only one of my sons has access to a new “miracle drug,” and it has changed our lives. I am watching one of my boys thrive, as my other gets tragically worse while he waits for access to the medication that could save him.



Every day, children with DMD get weaker, it causes loss of muscle, to the point where children with the disease cannot breathe on their own. It is a slow death sentence.



Miraculously, one of my sons has been saved. Max, just 10 years old, was lucky enough to take part in a medical trial. For the last 60 weeks, Max has been taking this new medication. This year, my son who was once dependent on a wheelchair most of the day, marched in a Halloween parade.



But our journey has been bittersweet. As we watch Max get better, we also watch his older brother, Austin, 13, get worse. He suffers, silently, as his disease progresses. Austin was unable to take part in the drug trial that helped his brother so much.



The note I wrote with my signature:

Does this really have to be a “struggle” when it is clearly the right thing to do?  

What could possibly be standing in the way of Expanded Access or Early Approval?