Posted 8 months ago

I have a feeling …

that I was one of the 58 ALS patients involved in the “error”.  But I’ve also been told that I will never actually know …

Cytokinetics: Programming Error Increases Tirasemtiv Trial Costs by $5 Million

Wall Street Journal

Cytokinetics Inc. (CYTK) said a programming error tied to a trial meant to evaluate a treatment for amyotrophic lateral sclerosis will increase its direct clinical trial costs by about $5 million in 2013 and 2014, while also saying it has amended the

Posted 9 months ago

CNN Special on ALS - Part 1 of 3 …

I’ll post all 3 parts as they are released and shown on CNN.

Just click this url link below.

Posted 9 months ago

On the much brighter side of life …

If someone had told me in early December 2010 (when I was formally diagnosed with ALS) that my son Tom and his wife Anna Kate were going to have a daughter in October of 2011 and that I would still be around and functioning enough to enjoy my granddaughter when she was 20 months old in 2013 … I would never have believed it …

For Father’s Day this month I received a great coffee mug with pictures of Harper Kate all over it that said:  I Love Papa and she was also wearing her Papa Makes Me Smile shirt.  I wanted to share pictures of the mug as well as some additional recent pictures of Harper Kate who really brightens my life every time I see her and she calls out for her Papa.

Posted 9 months ago

Latest writings by my friend Stu Rakoff …

I responded to Stu’s latest inspring Blog post with the following …


Change We Can Live WIth

Posted on June 22, 2013by rakoff

We just experienced the longest day of the year – summer solstice. Ironic since my days are growing shorter. This week also marked one year since I first became aware of my ALS symptoms. And it was also Lou Gehrig’s 110th birthday.

I have tried hard to find some meaning in these overlapping occurrences, but have come up empty.

The once constant is change – where have I heard that before.  My whole life these days is about accepting and adjusting to changes I cannot control.

Now that’s a new theme!

Because the muscles in my mouth and face are growing weaker, and I cannot close my lips tight, sneezing has become a grand adventure  – especially if I have recently eaten.  No more tissues for me – I now require whole sheets of paper towel to catch the flying elements.

Most solid food has left my diet, replaced by thick soups, chilis, and smoothies. I am starting to eat pudding again. I now take a good portion of my nutrition through my feeding tube. I buy whole cases of instant breakfast and thickening jells.

I find myself saying goodbye to so many things these days.  To eating solid food.  To bike riding.  To wine and spirits that aggravate my throat.  To some toxic people who were simply making demands on me that went far beyond my capacity to absorb.

I spend part of every day sucking small pieces of paper through a straw and depositing them into a cup.  I went to graduate school for that?

So a new theme is simplicity.  My diminishing physical strength and coordination means I must really concentrate on what I am doing at any particular moment. Now that keeps me anchored in the present, with only brief excursions to how it used to be or how it will be.  I could choke or stumble while letting my attention drift from the next step.

All in all, it’s not such a bad way to live.

My family gave me a father’s day gift – a tandem parachute jump when we are at the beach in August.   Now that’s an activity that will really get me focused on the absolute present.  I have never felt tempted by these extreme physical challenges, but I just might do this one.  My friend Tom often reminds me that we PALS (Persons with ALS) have a different risk-reward calculus – and not just about drug trials.

I have a good friend dealing with cancer and another dealing with the impacts of cardiovascular disease and strokes.  Their lives are filled with treatments, each of which offers new “hope” for a “cure”.  My challenge is that there are no treatments and no cure  – there is only coping with my disease and learning new skills.  So I approach every day as an adventure, treasure my family and friends, and concentrate on the challenge of living well.

And that is “Change we can live with.”

Posted 10 months ago

Ever Heard of the Rare Disease Legislative Advocacy (RDLA)????

Holly Ladd (colleague with ALS) sent this to me yesterday … in the 30+ months since my diagnosis I’ve never heard about the Rare Disease Legislative Advocacy (RDLA) at or ever heard it mentioned … I thought I had a handle on most things like this but I learn something new every day it seems (once again thanks to Holly Ladd!)

 RDLA is a collaborative organization designed to support the advocacy of all rare disease groups. By growing the patient advocacy community & working collectively we can amplify our many voices to ensure rare disease patients are heard in State & Federal Government.

I’d strongly recommend that all ALS Community folks ask your Member of Congress to join the Rare Disease Caucus at — It’s easy – the Action Center has a draft letter …


 Many Thanks!


Posted 10 months ago

Another step in the right direction for sporadic ALS …

More hope for life-extending treatments for folks with ALS - another breakthrough that moves the ball down this very long field toward the goal line.  I’m beginning to think that my Blog title needs to possibly be changed to “Repaving the Once Paved Roadway” …

Also please note the Global Collaborative Contributors to this ball movement and some very familiar name in our ALS Community:  Alessandra Chesi, Brett T Staahl, Ana Jovičić, Julien Couthouis, Maria Fasolino, Alya R Raphael, Tomohiro Yamazaki, Laura Elias, Meraida Polak, Crystal Kelly, Kelly L Williams, Jennifer A Fifita, Nicholas J Maragakis, Garth A Nicholson, Oliver D King, Robin Reed, Gerald R Crabtree, Ian P Blair, Jonathan D Glass and Aaron D Gitler.


Mutated gene tie to motor neuron disease


Monday, 27 May 2013

Dani Cooper  ABC


New insights into the cause of motor neurone disease have been uncovered with a study showing mutations in a specific gene lead to the death of the nerve cells responsible for powering our muscles.

The collaborative study led by Stanford University has been searching for the genetic causes of the disease, also known as Lou Gehrig’s disease after the famous US baseball player or amyotrophic lateral sclerosis (ALS).

Australian co-author Associate Professor Ian Blair, from the Australian School of Advanced Medicine at Macquarie University, says there are two forms of the disease.

About 10 per cent of cases have a family history of the disease, while the majority of cases are sporadic ALS. "In the past few years there have been a lot of breakthroughs in the familial form, but sporadic ALS has been a really hard nut to crack," says Blair.

He says it has long been suspected that gene mutations were behind sporadic ALS, but the technology has not been available to test this theory.  However recent dramatic advances in genome sequencing technology - known as next-generation sequencing - have now made testing this possible.

Progressive paralysis

ALS is a devastating neurodegenerative disease resulting in progressive paralysis and, eventually, death.  “It specifically targets the motor neurons that pass the signal to move limbs and also are critical to talking, swallowing, eating and breathing,” says Blair.  “In most cases the mind is spared so these people are completely aware of what is happening to them but progressively become unable to move and unable to communicate.”

Blair says in Australia about one in 5000 people aged over 50 will be affected with more deaths annually from ALS than HIV/AIDS.

MY PERSONAL INSERT: In the U.S., the approximate annual death rate from HIV/AIDS is 2.7 per 100,000 and for ALS it is approximately 2.17 per 100,000 (although this could be a much higher number since death by ALS is not required to be reported).

In the study published today in Nature Neuroscience at:

The team was searching for de novo mutations, which are defects in the gene that are not present in either parent and have occurred for the first time in the patient.  They found a mutation in the CREST gene, which is expressed more abundantly in neurons, in one of these patients.

Blair says the Australian element of the study was to screen for mutations in the CREST gene in a large collection of familial ALS samples.  “If this protein is so critical that when it is mutated it can lead to the death of motor neurones, it is reasonable to assume mutations in this gene might also occur in familial ALS,” says Blair.  “We found a mutation in a family, so that strongly supported the role for CREST in causing the disease.”


First step

These findings were also supported by laboratory tests that showed these mutations caused dysfunction in neuronal cells.  “This is the first step,” says Blair. “It highlights what is wrong, now the challenge is to uncover how that is causing the death of motor neurons.”

Blair says the finding adds to the bank of knowledge about ALS and can help to develop effective models of the disease on which to test new therapies.  “We are trying to find the smoking gun that causes this disease,” he says. “Each gene [we discover] might be that smoking gun that allows us to develop models to help us to develop therapies.”  Blair says the approach they have used to uncover these mutations could be applied to all neurodegenerative diseases such as Parkinson’s and Alzheimer’s disease.

Posted 11 months ago

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!


Uncle Tom 

Click the link below to watch the video …

Posted 11 months ago

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.




Posted 1 year ago

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 …


The Cytokinetics Tirasemtiv Clinical Trial  


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!

Posted 1 year ago

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 . 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. 



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


Posted 1 year ago

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.


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.


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



go to!docketDetail;D=FDA-2013-N-0035

FILL IN INFORMATION. Note the session will time out in twenty minutes, so you might want to copy/paste. 

Posted 1 year ago

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 — — take some time to listen to the tribute to Michael by Senator Tom Coburn.

RIP Michael Schwartz … You were a very inspirational person … 

Posted 1 year ago
Posted 1 year ago

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 -

Or the ALS Emergency Treatment Fund -

Or the ALS Therapy Development Institute (ALS TDI) -

Or the Northeast ALS Consortium (NEALS) -

Or the National Organization for Rare Disorders (NORD) -

Or the ALS Association -

Or the MDA ALS Center of Hope -

Or the Prize4life -

Or the Abigail Alliance -

Or Team R.A.R.E -

Or the Les Turner ALS Foundation -

Or the Brigance Brigade -

Or the Pooled Resource Open-Access ALS Clinical Trials Database (PRO-ACT) -

Or the International Alliance of ALS/MND Associations -

Or Patients Like Me (PLM) -

Steve Gleason makes plea for $5 million in donations to fight ALS before Super Bowl provides news on all New Orleans sports including the Saints, Hornets, LSU Tigers, Tulane Green Wave, High School Sports and everything in between.
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Posted 1 year ago

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

Lots being written, tweeted, blogged and publicized about ALS over the last week or so by NFL players and Steve Gleason and hopefully that will cause a much needed light to shine on this rare disease with NO TREATMENTS.


I was diagnosed on 12/8/2010 with ALS but I can still walk, talk, swallow, etc. - although I have lots of arm and hand troubles and breathing issues.  

As all of us folks with ALS know very well, an ALS “cure” is MANY years away and it is highly likely that Steve Gleason and I will no longer be on the playing field of life when this occurs. 


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

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.