Posted 1 week ago

the NFL and ALS … 

Kevin Turner, a former N.F.L. player who has A.L.S., receiving help with shaving at his Florida home. CreditJosh Ritchie for The New York Times

PORT ST. LUCIE, Fla. — Kevin Turner and Sean Morey played a combined 17 years in the N.F.L.They were never teammates, but they became friends in 2010 when they worked with the Mackey-White Traumatic Brain Injury Committee for disabled retirees. They consulted with doctors studying the effects of concussions on football players. Morey raised money for Turner’s foundation after Turner received a diagnosis of amyotrophic lateral sclerosis, or A.L.S., four years ago.

So in January, before Morey joined six other former N.F.L. players to file an objection to the proposed settlement in a lawsuit that includes a promise by the league to compensate retirees with severe neurological problems, he called Turner to explain why he was taking a step that might delay much-needed money for Turner.

“My heart bleeds for Kevin,” said Morey, who said the settlement was flawed primarily because it covered too few conditions. “We both want what’s fair, adequate and reasonable, but unfortunately his condition is much more urgent.”


Turner (34) playing for the Eagles. He believes his condition was caused by hits to the head.CreditEzra O. Shaw/Allsport

Turner, 45, and Morey, 38, represent opposite views on whether players should accept the settlement; opt out and retain their right to sue the N.F.L.; or object and perhaps appeal to a higher court.

The debate is real now that 20,000 retired players and their beneficiaries are being sent an outline of the settlement, which is full of legal and medical jargon, descriptions of an assessment program and tables showing who is eligible for an award.

The decision about whether to accept or reject the deal, though, will present a moral quandary: Should the players accept a limited settlement so that people like Turner can get help quickly, or should they fight for something better and perhaps slow the distribution of payments to those in need?

Turner, who could receive as much as $5 million, said that he understood that the deal was not perfect, but that he wanted the retirees to accept it so he and others could get help sooner.

“I can empathize with players” who want a better settlement, Turner said as he sat in his living room in Port St. Lucie, his arms and hands largely inert, his neck stooped and his speech slurred. “But for me and people like me, time is a luxury we don’t have.”

At some level, all class-action settlements require plaintiffs to weigh their personal interests against those of a larger group, along with the odds of getting a better deal by fighting an uncertain court battle.

In cases involving, for example, faulty washing machines or billing errors by a bank, the calculus is often clear. But the N.F.L. concussion settlement involves emotional nuance because the retirees were teammates, friends and union members who are bonded by a sport that in many ways has shaped them as adults.

“This is not like a group of people randomly thrown together in an accident,” said Alan Morrison, who teaches at the George Washington University Law School and has worked on class-action settlements. “Many of them played together, and they’ve seen what’s happening to their friends, but nobody knows if they’ll have the same problems.”

Turner, the lead plaintiff in the suit filed by about 5,000 former players who accused the league of hiding the dangers of concussions from them, has deteriorated to the point where he needs a full-time nurse. He cannot help but see the settlement as a salve for players in dire need.

“It’s about helping people who had their brains affected in a very drastic way, and to make their lives so much more livable, not just them but their families, and to supplement their health care,” said Turner, who added that he wanted to use his award to cover his medical expenses and leave something for his three children.


Turner using an iPad in his Florida home. He said he viewed the settlement in terms of “how it would help me live longer.” CreditJosh Ritchie for The New York Times

Like many football players trained to follow orders, Turner wrestled with whether to sue the league that had helped him realize his lifelong dream. He began playing at 5 years old, starred for the Alabama Crimson Tide and played fullback for eight seasons for the New England Patriots and the Philadelphia Eagles.

He also sustained more concussions than he can count. In 2009 he started having trouble playing guitar. A few months later, he had vertebrae in his neck fused to repair a football injury, and his ability to control his hands declined to the point where he no longer could write. The next year, doctors said that he had A.L.S.

Turner, who declared bankruptcy in 2009 when his home-building business failed, was living with friends. He began to study his disease. He went to Boston University, where doctors were doing cutting-edge research on the effects of repeated brain trauma, and spoke to the Mackey-White Traumatic Brain Injury Committee, which Morey was part of.

Turner said he believed his A.L.S. had been caused in part by the head hits he absorbed. As he began to see the links between his injuries and his condition, he was convinced that the N.F.L. had played a role, too.

“I remember times when I went back into games when I shouldn’t have,” said Turner, who has stopped his two sons from playing football. “I wasn’t given all the information to make that decision for myself.”

After turning down invitations to join a lawsuit, he sued the league in 2011. Given his diagnosis and the difficulty of winning a case against the league, he did not expect to see its conclusion. At least, he thought, his children might get some money.

Turner receives money from two N.F.L. plans for retirees with disabilities, yet the bills still pile up. He had to remodel his bathroom, buy new furniture and pay for nurses and trips to see doctors. He receives experimental therapies designed to slow the deterioration of his muscles, and he hopes to receive stem-cell treatments that could cost about $75,000.

Turner can still walk, but his upper body does not respond well. He uses an iPhone and can send text messages, but his voice is so garbled that the Siri program no longer understands his voice commands. But Turner, who was the subject of the documentary “American Man,” takes his role as lead plaintiff seriously. Last month, he went to Washington to appear at a Senate hearing on concussions in sports.

“I never imagined myself being part of something of this proportion,” Turner said. “Everyone is focusing on the number and how much am I going to get. But I am looking at it as how it would help me live longer.”

Morey, who experiences chronic headaches and irritability from the concussions he sustained, wants Turner to get what he deserves. But the deal, which may still be altered, has too many flaws to ignore, said Morey, who did not sue but is covered by the settlement, as all retirees are.

Many retirees will receive no awards because so few illnesses are covered in the deal, he said, and those in the worst shape, including some who are mentally impaired, will have to jump through many hoops to get awards. The plaintiffs’ lawyers did not disclose how they had reached the settlement, which makes it hard to know whether they got as much as they could have, and they rushed to settle so they could get paid, Morey said.

“At the end of the day, players just want a fair shake,” said Morey, who played nine seasons with four teams before retiring after the 2009 season. “In my mind, this is everything the N.F.L. wanted.”

Posted 3 weeks ago

A Much Welcomed Collaborative Initiative for the ALS Community …

Team Gleason Announces Collaborative Initiative: Answer ALS - ‎Jun 27, 2014‎
Given the successful collaboration among those impacted by ALS and researchers at the Summit, Team Gleason is launching Answer ALS to activate the findings from the Summit. Answer ALS is not another organization, but rather an initiative, led by a …

Steve Gleason is ready to tackle ‘the impossible’ and end ALS

The Times-Picayune - ‎Jun 26, 2014‎
Answer ALS is the by-product of another Gleason initiative, the 2013 ALS Summit, an unprecedented event which attracted leading researchers, patients, caregivers and advocates to New Orleans a year ago. Answer ALS wlll focus on two key elements:.

Steve Gleason seeks $500 million to find ALS cure

WWL - ‎Jun 26, 2014‎
After a summit on the disease last year, Team Gleason is now starting ‘Answer ALS,’ setting up a council of patients, care givers, doctors, researchers, and pharmaceutical companies to find new therapies for treatment, like using stem cells, and coordinate the …

Gleason launches effort to cure ALS - ‎Jun 27, 2014‎
On Thursday, Gleason’s foundation announced Answer ALS, calling it “the single largest effort to end ALS in the history of the disease.” It’s a major undertaking, and Gleason doesn’t flinch in the face of it. He embraces it. “[W]e are appealing to large investors, …

Steve Gleason Announces New Fund Raising Initiative to Fight ALS

WGNO - ‎Jun 26, 2014‎
Thursday afternoon at a hotel conference room in the CBD, Saints fan favorite Steve Gleason announced a new initiative in coordination with his Team Gleason group. It’s called Answer ALSand is designed to raise big money for medical research over the …

Steve Gleason Continues to Inspire New Orleans Saints

Rant Sports - ‎Jun 27, 2014‎
The program, called Answer ALS, looks to raise over $500 million for research and to unite people with ALS. According to Gleason, ALS isn’t incurable but severely underfunded. Odds are Gleason isn’t standing alone. The Saints franchise and its players will …

Gleason announces bold plan to end ALS

ESPN (blog) - ‎Jun 27, 2014‎
Former New Orleans Saints player Steve Gleason announced an ambitious new initiative Thursday called Answer ALS, which he described as “the single largest effort to end ALS in the history of the disease.” [+] Enlarge Steve Gleason. Cindy Ord/SIS/Getty …
Posted 3 weeks ago

75 years after Gehrig speech, slow progress treating ALS …

75 years after Gehrig speech, slow progress treating ALS

Karen Weintraub, Special for USA TODAY5:40 p.m. EDT July 3, 2014

The world was stunned 75 years ago Friday when baseball great Lou Gehrig announced that he had been diagnosed with a lethal disease but was still “the luckiest man on the face of the Earth.”

He was grateful for his career, his coaches and, most of all, his family, he said in a Yankee Stadium speech that brought to public consciousness a disease called amyotrophic lateralsclerosis, or ALS — still often called Lou Gehrig’s disease.

"I might have been given a bad break, but I’ve got an awful lot to live for," the "Pride of the Yankees" said slowly and calmly to 62,000 fans.

The course of Gehrig’s disease was typical. Diagnosed a few weeks after ending his 2,130-game playing streak, he would live only two more years.

Treatments for ALS, which damages nerve cells in the brain and spinal cord leading to progressive weakness, didn’t change much for seven decades after Gehrig’s death.

But over the past five years, scientific research has finally begun to pay off for patients.

"People are living longer with ALS today than they were a couple of years ago, because the care is better," says Merit Cudkowicz, chairwoman of neurology at Massachusetts General Hospital in Boston and a professor at Harvard Medical School.

By paying more attention to patients’ quality of life, researchers have learned new ways to control pain, encourage exercise and support breathing, she says. One research trial showed that patients could live longer simply by maintaining their normal weight.

"Patients used to be told there’s nothing to do and go home," says Cudkowicz, who treats about 200 ALS patients a year. "It’s completely different now."

She also encourages her patients to participate in one of the dozens of clinical trials underway to better understand and treat ALS. More than 30 genes connected to ALS have been identified, and several drug companies have targeted and tested possible therapies.

Kevin Eggan, a stem cell researcher at Harvard, discovered a promising treatment by using stem cells from patients who died of ALS to re-create the disease in a dish. He then tested thousands of compounds on those cells to find one that could reverse electrical signaling problems characteristic of the ALS cells. That drug is scheduled for testing in patients later this year.

All of this research is encouraging to people in the field.

"It’s a very difficult disease, but I’m very hopeful," says Lucie Bruijn, a pharmacologist and chief scientist with the ALS Association. "There is such talent thinking about this challenge."

So far, though, more than a dozen drug studies have been failures, and nothing has been found to truly change the course of ALS. The disease has proved far more complex than researchers originally realized.

Doug Williams, head of research and development at the Cambridge, Mass., biotechnology company Biogen Idec, says that after repeated failures, his company decided to go back to the lab to see if they could better understand ALS and what it does to the nerve cells that control muscles.

"What we’re trying to do is really understand what are the critical defects that lead to the death of motor neurons,” he says. “If you think about ALS as a movie with a bad ending, we’re starting at the end of the movie and running it backward, trying to understand what’s happening in each frame.”

Christine Wickmark hopes it doesn’t take too long. At 56, the Idaho mother of three and former Air Force drill sergeant says she’s “too damn young to get out of here anytime fast.”

She’ll sign up for any clinical trial that wants her, she says, in hopes of changing the course of her ALS. So far, her legs are holding up fine — she can still volunteer at the senior center, dancing disco with people a generation older, and enjoy her 75 pigs, including 600-pound Charlie, who she swears danced with her recently to Stayin’ Alive.

But her hand control is limited, her voice is harder to understand, and she’s often congested. She’s tried acupuncture, vitamins and peppermint oil for the congestion.

"I’ll try anything," she says. "I just want to stop this thing."


In amyotrophic lateral sclerosis, or ALS, nerve cells in the brain and body progressively die, disrupting signals from the brain to the muscles.

Usually, the first symptoms of ALS are signs of weakness, such as falls, or sudden loss of strength or speed. Slurred speech is also common.

The disease generally progresses very quickly, with most people dying two to five years after diagnosis. If someone is diagnosed late, death can come much sooner.

ALS manifests itself differently in different people, starting in different parts of the body and progressing at different paces. A few people, such as world-renowned scientist Stephen Hawking, can have the disease for decades. Such different paths make it difficult to tell whether a drug is actually helping slow a patient’s course.

There are no treatments that profoundly change the course of the disease, but recent research has shown that patients who can maintain their pre-diagnosis weight live longer than those who lose weight. Exercise and breathing support can also be helpful.

Posted 3 months ago

Now THIS would be ALS/MND Patient-Centric behavior …

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.

Wednesday, April 16, 2014

Now THIS Would Be Patient-Centric

"I feel like I’ve left tissue all over the country." — Words of a man with ALS

Today there are countless opportunities for people with ALS and their healthy relatives to give blood and skin and fingernails and all kinds of tissue samples for ALS research.  Unfortunately the donors are treated like renewable sources of tissue rather than valued people.  Researchers who often brag that they work together all gather their own samples for their own needs.  It’s not about the patients. It’s about their immediate research needs. 

Picture this.

A person diagnosed with ALS is given a unique identifier on the day of diagnosis.  Maybe even a nice card with a bar code is included.  After that, every medical record, every blood sample, every MRI, every test result includes that identifier.  A person’s information and samples are suddenly connected with the person rather than with a scientist.  And shortly after diagnosis, people with ALS are offered the opportunity to opt-in to a master bio-bank.  The tissue isn’t stored in one place, but the information about where the tissue samples reside is stored centrally.  It’s called patient-centric. 

Think about the way your tire store can find a set of tires that fit your car overnight, even if two tires come from a warehouse in Saint Louis and two from a warehouse in Cleveland.

When a researcher or a pharmaceutical company needs a random set of blood samples from people with C9 genes, they can get them.  When a researcher needs a completely random set of skin sample from people with ALS, they are accessible.  When a research needs some blood from young SOD1 male PALS with slow limb-onset, they can get them.

Fix the information problem  Fix the center of attention.  It’s the patient.  Donate once, use many times.

This would be patient-centric.

Next time you hit a chuckhole and need a new tire, think about the days when Lou had to wait for the factory to make more 4.50x21s to fix the Model A.
Posted 3 months ago

The origin of Lou Gehrig’s disease may have just been discovered …

Two very hopeful news items in a single week.

 1.    The origin of Lou Gehrig’s disease may have just been discovered

 2.    Patient stem cells help identify common problem in ALS

Discovery will lead directly to clinical trials

Posted 3 months ago

2 More ALS R&D “Breakthroughs” in March 2014 … hope it doesn’t take another 7-10 years to introduce these to HUMANS

 I don’t think I have that long …


Experimental stroke drug also shows promise for people with Lou Gehrig’s disease

Date:  March 3, 2014

Source:  University of Southern California - Health Sciences


Neuroscientists have found that early muscle impairment related to Lou Gehrig’s disease, also called amyotrophic lateral sclerosis, or ALS, in mice is proportional to the degree of damage to the blood-spinal cord barrier, which protects the central nervous system from toxins. Repairing damage to and restoring the blood-spinal cord barrier’s integrity with an experimental neurovascular medicine being studied in human stroke patients appears to delay disease progression.

Credit: Photo courtesy of Ethan A. Winkler and Berislav V. Zlokovic/University of Southern California

[Click to enlarge image]


A fluorescent image shows cells of the neurovascular unit in the mouse spinal cord, which consists of motor neurons (green) and blood vessels containing pericytes (red) and endothelial cells (blue). Winkler et al. show that disruption of blood vessels accelerates injury of motor neurons in amyotrophic lateral sclerosis.

Keck School of Medicine of USC neuroscientists have unlocked a piece of the puzzle in the fight against Lou Gehrig’s disease, a debilitating neurological disorder that robs people of their motor skills. Their findings appear in the March 3, 2014, online edition of the Proceedings of the National Academy of Sciences of the United States of America, the official scientific journal of the U.S. National Academy of Sciences.

"We know that both people and transgenic rodents afflicted with this disease develop spontaneous breakdown of the blood-spinal cord barrier, but how these microscopic lesions affect the development of the disease has been unclear," said Berislav V. Zlokovic, M.D., Ph.D., the study’s principal investigator and director of the Zilkha Neurogenetic Institute at USC. "In this study, we show that early motor neuron dysfunction related to the disease in mice is proportional to the degree of damage to the blood-spinal cord barrier and that restoring the integrity of the barrier delays motor neuron degeneration. We are hopeful that we can apply these findings to the corresponding disease mechanism in people. "

In this study, Zlokovic and colleagues found that an experimental drug now being studied in human stroke patients appears to protect the blood-spinal cord barrier’s integrity in mice and delay motor neuron impairment and degeneration. The drug, an activated protein C analog called 3K3A-APC, was developed by Zlokovic’s start-up biotechnology company, ZZ Biotech.

Lou Gehrig’s disease, also called amyotrophic lateral sclerosis, or ALS, attacks motor neurons, which are cells that control the muscles. The progressive degeneration of the motor neurons in ALS eventually leads to paralysis and difficulty breathing, eating and swallowing.

According to The ALS Association, approximately 15 people in the United States are diagnosed with ALS every day. It is estimated that as many as 30,000 Americans live with the disease. Most people who develop ALS are between the ages of 40 and 70, with an average age of 55 upon diagnosis. Life expectancy of an ALS patient averages about two to five years from the onset of symptoms.

ALS’s causes are not completely understood, and no cure has yet been found. Only one Food and Drug Administration-approved drug called riluzole has been shown to prolong life by two to three months. There are, however, devices and therapies that can manage the symptoms of the disease to help people maintain as much independence as possible and prolong survival.

Story Source:

The above story is based on materials provided by University of Southern California - Health SciencesNote: Materials may be edited for content and length.

Journal Reference:

  1. E. A. Winkler, J. D. Sengillo, A. P. Sagare, Z. Zhao, Q. Ma, E. Zuniga, Y. Wang, Z. Zhong, J. S. Sullivan, J. H. Griffin, D. W. Cleveland, B. V. Zlokovic. Blood-spinal cord barrier disruption contributes to early motor-neuron degeneration in ALS-model miceProceedings of the National Academy of Sciences, 2014; DOI:10.1073/pnas.1401595111


  1. II.            Key player in motor neuron death in Lou Gehrig’s disease identified


Key player in motor neuron death in Lou Gehrig’s disease identified

Date:  March 26, 2014

Source:  Nationwide Children’s Hospital


Amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, is marked by a cascade of cellular and inflammatory events that weakens and kills vital motor neurons in the brain and spinal cord. The process is complex, involving cells that ordinarily protect the neurons from harm. Now, a new study points to a potential culprit in this good-cell-gone-bad scenario, a key step toward the ultimate goal of developing a treatment.

Amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, is marked by a cascade of cellular and inflammatory events that weakens and kills vital motor neurons in the brain and spinal cord. The process is complex, involving cells that ordinarily protect the neurons from harm. Now, a new study by scientists in The Research Institute at Nationwide Children’s Hospital points to a potential culprit in this good-cell-gone-bad scenario, a key step toward the ultimate goal of developing a treatment.

Motor neurons, or nerve cells, in the brain and spinal cord control the function of muscles throughout the body. In amyotrophic lateral sclerosis (ALS), motor neurons die and muscles weaken. Patients gradually lose the ability to move and as the disease progresses, are unable to breathe on their own. Most people with ALS die from respiratory failure within 3 to 5 years from the onset of symptoms.

For the study, published online this month in Neuron, researchers examined a protein involved in transcriptional regulation, called nuclear factor-kappa B (NF-κB), known to play a role in the neuroinflammatory response common in ALS. NF-ƘB has also been linked to cancer and a number of other inflammatory and autoimmune diseases.

Using animal models, the researchers studied disease progression in mice in which NF-ƘB had been inhibited in two different cell types — astrocytes, the most abundant cell type in the human brain and supporters of neuronal function; and microglia, macrophages in the brain and spinal cord that act as the first and main form of defense against invading pathogens in the central nervous system. Inhibiting NF-ƘB in microglia in mice slowed disease progression by 47 percent, says Brian Kaspar, MD, a principal investigator in the Center for Gene Therapy at Nationwide Children’s and senior author of the new study.

"The field has identified different cell types in addition to motor neurons involved in this disease, so one of our approaches was to find out what weapons these cells might be using to kill motor neurons," Dr. Kaspar says. "And our findings suggest that the microglia utilize an NF-κB-mediated inflammatory response as one of its weapons."

Inhibiting the protein in astrocytes had no impact on disease progression, so the search for the weapons that cell type uses against motor neurons continues. These preliminary findings also don’t tell scientists how or why NF-κB turns the ordinarily protective microglia into neuron-killing molecules. But despite the mysteries that remain, the study moves scientists closer to finding a treatment for ALS.

The search for an ALS therapy has been focused in two directions: identifying the trigger that leads to disease onset and understanding the process that leads to disease progression. Changes in motor neurons are involved in disease onset, but disease progression seems to be dictated by changes to astrocytes, microglia and oligodendrocytes. Some cases of ALS are hereditary but the vast majority of patients have no family ties to the disease. The complexity of the disease and the lack of a clear familiar tie make screening before disease onset nearly impossible, highlighting the importance of slowing the disease, Dr. Kaspar says.

"Focusing on stopping the changes that occur in astrocytes and microglia has clinical relevance because most people don’t know they’re getting ALS, says Dr. Kaspar, who also is an associate professor of pediatrics and neurosciences at The Ohio State University College of Medicine. "We have identified a pathway in microglia that may be targeted to ultimately slow disease progression in ALS and are exploring potential therapeutic strategies and may have broader implications for diseases such as Alzheimer’s and Parkinson’s Disease amongst others."

Story Source:

The above story is based on materials provided by Nationwide Children’s Hospital.Note: Materials may be edited for content and length.

Journal Reference:

  1. Ashley E. Frakes, Laura Ferraiuolo, Amanda M. Haidet-Phillips, Leah Schmelzer, Lyndsey Braun, Carlos J. Miranda, Katherine J. Ladner, Adam K. Bevan, Kevin D. Foust, Jonathan P. Godbout, Phillip G. Popovich, Denis C. Guttridge, Brian K. Kaspar. Microglia Induce Motor Neuron Death via the Classical NF-κB Pathway in Amyotrophic Lateral SclerosisNeuron, 2014; 81 (5): 1009 DOI:10.1016/j.neuron.2014.01.013
Posted 5 months ago

Another Great TED Talk …

Siddharthan Chandran: Can the damaged brain repair itself? 

After a traumatic brain injury, it sometimes happens that the brain can repair itself, building new brain cells to replace damaged ones. But the repair doesn’t happen quickly enough to allow recovery from degenerative conditions like motor neuron disease (also known as Lou Gehrig’s disease or ALS). Siddharthan Chandran walks through some new techniques using special stem cells that could allow the damaged brain to rebuild faster.

Siddharthan Chandran explores how to heal damage from degenerative disorders such as MS and motor neuron disease (ALS).

Posted 5 months ago

Superbowl and ALS …

Watching the ‪#‎SuperBowl‬ later today? Keep an eye out for this minute-long Microsoft commercial that will feature former New Orleans Saint Steve Gleason, who is living with ‪#‎ALS‬. …

Posted 6 months ago

Another “First” in Life Experiences …

I’ve slept on the floor, on the ground, in a tent, in a sleeping bag, on a couch and in a bean bag chair … but last night was my first time sleeping the whole night in a recliner connected to a BIPAP machine (BIPAP tutorial below).  Now I know why so many folks with ALS sleep in recliners and not beds.

It was the best, uninterrupted, nights sleep I’ve had in a few weeks - was not worrying about my compromised breathing — and woke up this morning without a headache (due to low blood oxygen) for the first time in a while … What a Deal!

Posted 7 months ago

Maybe we can make a difference in the ALS arena …

ALS - Veterans Support Foundation Inc.

We must never forget all that veterans have given, every day protecting the privileges, comforts and freedoms that WE enjoy.  Service men and women know they were fighting honorably for something larger than themselves — the same holds true for this fight versus ALS …

Posted 7 months ago

3 Years and Still Transitioning :) …

Well it has now been 3 full years since the neurologist at GW University told me I had 2-4 years to liive.  

I am thankful to be a slow progressor and for the many wonderful things I have had the good fortune to experience in these past 3 years.

Wanted to share the history of my ALS journey so far …




Listened to Carrie Underwood sing this the other day — from The Sound of Music — I think this what I do:

When the dog bites, when the bee stings
When I’m feeling sad
I simply remember my favorite things
And then I don’t feel so bad

Posted 8 months ago

More progress in the possible treatment of ALS …

But how long will it take for the US to “adopt” this area of possibilities and begin human testing?

I’d raise my hand and volunteer … the potential side effects couldn’t be much worse than what’s already happening to me.


A major step towards the cure of sporadic ALS

Experimental gene therapy for a sporadic ALS mouse model

Amyotrophic lateral sclerosis (ALS) develops mainly in the middle-aged and elderly and is characterized by progressive muscular weakness and muscular atrophy. There is no known cure for this incurable neurological disease, which leads to death due to paralysis of the respiratory muscles within a few years of onset. The research group of Project Professor Shin Kwak (Visiting Researcher, Clinical Biotechnology Laboratory, Center for Disease Biology and Integrative Medicine, Graduate School of Medicine, the University of Tokyo; Project Professor, Clinical Research Center for Medicine, International University of Health and Welfare) had discovered in earlier research that the enzyme ADAR2 was involved in neuron cell death in non-familial ALS, which accounts for the great majority of cases of the disease.

© teamkwak, A single intravenous injection of AAV9 vector successfully delivered the human ADAR2 (RNA editing enzyme) gene to motor neurons of AR2 mice, in which the ADAR2 gene was ablated selectively in the motor neurons, therefore comprising a mechanistic mouse model of sporadic ALS. The resulting expression of ADAR2 effectively rescued progressive motor dysfunction and death of motor neurons in the AR2 mice.

In this study, Project Professor Kwak and Project Researcher Takenari Yamashita (Clinical Biotechnology Laboratory, Center for Disease Biology and Integrative Medicine, Graduate School of Medicine, the University of Tokyo), in collaboration with Special Professor Shinichi Muramatsu’s research group (Jichi Medical University), developed an adeno-associated virus serotype 9 (AAV9) vector that would enable gene delivery only to the neurons of mouse brain and spinal cord. When this vector was administered by intravenous injection into the sporadic ALS model mice (AR2), the researchers succeeded for the first time in stopping the degeneration and loss of motor neurons and the progression of symptoms of the disease.

Further, even when administered after the emergence of symptoms, expression of the ADAR2 gene in motor neurons stopped the process leading to cell death and symptoms due to cell death were prevented without any apparent side-effects. It is conventionally thought that it is difficult to introduce genetic material into the brain and spinal cord by intravenous injection, but one intravenous injection alone was sufficient to bring about long-lasting expression of an effective quantity of the ADAR2 gene through the use of the AAV9 vector to trigger gene expression only in neurons.

While this result was achieved with a model mouse, it is thought that a similar molecular mechanism underlies sporadic ALS in human patients, and as human ADAR2 gene had a therapeutic effect in the model mouse, it is anticipated that a similar form of gene therapy will be effective in treating human ALS as well. Further, the AAV9 vector is known to be safe, and after confirming the safety of the improved AAV9 vector and determining optimal dosage, it is hoped that this research will open a new route to the treatment of ALS. Currently gene therapy has a strong image as a replacement therapy for rare genetic disorders, but this research is unique in that it shows that gene therapy is possible even in sporadic cases if the molecular pathology of the disease is understood.

This research was published in EMBO Molecular Medicine (24 September 2013 online edition). The present study was conducted with the support of the Japan Science and Technology Agency Strategic Basic Research Programs (CREST) and the Ministry of Health, Labour and Welfare Illness-related Disabilities Measures Research Program.


Takenari Yamashita, Hui Lin Chai, Sayaka Teramoto, Shoji Tsuji, Kuniko Shimazaki, Shin-ichi Muramatsu and Shin Kwak,
“Rescue of amyotrophic lateral sclerosis phenotype in a mouse model by intravenous AAV9-ADAR2delivery to motor neurons,”
EMBO Molecular Medicine Online Edition: 2013/9/24 (Japan time), doi: 10.1002/emmm.201302935.

Posted 8 months ago

A MILE IN ALS SHOES … A dose of reality

Saw this today and wanted to share it with others …


People ask me often what it’s like to live with ALS.   It’s a brave question because the answers are not very pleasant.   But it’s also such a worthy question because understanding how this disease impacts those who suffer from it creates empathy which is so valuable; it carries us into another person’s world and allows us to understand what they’re feeling and how they’re hurting.  As I watch my strong husband struggle with things that used to be easy and automatic, I sometimes wish that everyone could see life from his perspective.

Empathy isn’t the same as sympathy.  Sympathy means we feel sorry for people, empathy means we work to understand and identify with their pain.  Empathy isn’t always easy to squeeze into our busy and difficult lives, but it’s worth it.  It makes us more human in all the ways that matter and – in the search for a cure for ALS – empathy is our most effective motivator.

If you would like to experience just a tiny corner of an ALS life, I have a list of Empathetic Experiences for you.  These are things you can do to walk for just a mile in ALS shoes.  If you try one, take a little time at the end to consider that people actually living with the disease have a million miles more to go.

  1.  Pick up a 10-pound weight.  Now imagine it’s your fork and move it from your plate to your mouth repeatedly without shaking.
  2. Sit in a chair for just 15 minutes moving nothing but your eyes.  Nothing.  No speaking, no scratching your nose, no shifting your weight, no changing the channel on the television, no computer work.  Only your eyes.   As you sit, imagine: this is your life.  Your only life.
  3. Borrow a wheelchair or power scooter and try to maneuver quickly through the aisles at Walmart, without speaking.  Note the way people react to you.
  4. Strap 25 pounds to your forearm.  Now, adjust your rearview mirror.
  5. Using none of your own muscles, have your spouse or child or friend get you dressed and brush your teeth.  Write down some of the feelings you have being cared for in this way.
  6. Before you eat your next meal, take a good, long look at the food.  Inhale deeply and appreciate the aroma.  Now, imagine never being able to taste that – or any other food – for the rest of your life.
  7.  Put two large marshmallows in your mouth and have a conversation with your friends.   How many times must you repeat yourself?  How does this make you feel?
  8. Go to bed and stay in one position for as long as you possibly can, moving nothing.
  9. Strap weights to your ankles and climb a flight of stairs, taking two at a time.  That’s the kind of strength it takes for someone with ALS to tackle the stairs on a good day.
  10. Install a text-to-speech app on your phone or iPad and use it exclusively to communicate for one day.

And to my friends living with ALS:  please give us more ideas and help us move into your world for a bit.  We want to help make your lives rich and full and I’m not sure we can do that without at least a basic understanding of what you are facing. I think I speak for many when I say:  you are superheroes and we are in awe.

With unending hope for a million-mile cure,


Posted 9 months ago

10/12 - 10/13/2013 … A Weekend to Remember Always (A Birthday and a Wedding)

The weekend in Charlottesville, VA (yes … home of the Wahoos) started on 10/12 with Harper Kate’s Birthday - she’s 2 years old already!





To top off the day of 10/12 — a great wedding rehearsal dinner hosted by the Raedy Family …




The CRUSTIES were amazing …


Then the big day 10/13 — the marriage of our daughter Sarah Elizabeth Murphy to Brendan Daniel Raedy …


If someone had told me in December 2010 that I’d be walking my “baby” daughter Sarah down the aisle and be able to dance with her at her wedding this past weekend (or even be around for her wedding) — I wouldn’t have believed it.

Sarah and I just before the ceremony …


Our son Tom escorting the Mother of the Bride and Harper Kate …


The Bride and Dad walk down the aisle at the Old Metropolitan Hall -


"I pronounce you husband and wife — you may kiss the bride" …


Back up the aisle - but now as husband and wife …


From left to right the bridal party:  Tom (our son), Mike, Jeff, Jeff, Matt, Brendan, Sarah, Holly, Sora, Jessica, Kristin (our daughter and Maid of Honor) and Hannah.


My speech and toast …


First dance as husband and wife …


Dad and Bride danced to “Somewhere over the Rainbow” - Israel “IZ” Kamakawiwoʻole -


Pastaccio wedding cake - delicious!


The finale at the end of the evening …



At the end of the evening Keri looks tremendous and I look like I’m about to pass out from exhaustion …


A Thank You FB Post from Sarah …


And they are already in the Dominican Republic on their honeymoon on Tuesday afternoon 10/15 … What a Deal …


***** Big photo credits to Carly Romeo (Two Spoons Photography) for her pictures that I used for ths blog post *****

Posted 10 months ago

Can’t Remember the Last Time I Received a Participation “CERTIFICATE” …


FDA Patient Representatve Class of 2013 

(I’m the guy in the back row middle)