Reblog - Recordings of people with the deadly muscle disease ALS. A campaign that’s sure to get attention due to the fact it won’t be released until the participant in the recording has died. Powerful, powerful stuff.
On 12/8/2010 when the neurologist at George Washington University Hospital confirmed my ALS diagnosis I really wasn’t sure at all about how many Holiday Seasons were still in the cards for me.
Well - this is my third one since that day … and I am grateful that I am still able to fully enjoy all of the things that come with this time of year - travel, family gatherings, great food, drink, giving and getting hugs & kisses, a loving family that is very dear to me and special friends.
Anyway - Just a quick note from my family and I to all of you out there who have made this journey of Transitioning from the Paved Roadway much easier …
Great news about my friend Ted Harada — Now if only we could all find some way to get this miracle in the hands of a lot more of us folks with ALS.
I’d give anything to have someone write this about me - His strength is back up, there’s a spring in his step, he’s got a strong grip back in his hands, and the symptoms of his ALS once again are in retreat …
Once again, Harada is easily going up the stairs to tuck his kids in at night and give them a kiss, instead of struggling up a step at a time, having to hold onto the handrail for support. Once again, he knows — or is as close to knowing as you can with such a disease — that he is part of something that will eventually change the death-sentence prognosis that until now has been a certainty as soon as there is a diagnosis with the dreaded words no one wants to hear: amyotrophic lateral sclerosis — Lou Gehrig’s disease.
“The first time, it’s easy to say it was an outlier. Luck. But I’ve been helped twice. Twice, and you can throw luck out the window. They’ve got to figure out, now, what’s going on with me,” he says. “We’ve got to turn Lou Gehrig’s disease into Lou Gehrig’s chronic illness.”
Some background: I interviewed Harada by phone in early October for a package of stories Crain’s ran Oct. 29 about successful Phase 1 human trials that University of Michigan and Emory University physicians and researchers had recently concluded in Atlanta, injecting stem cells into the spinal column of ALS patients.
Because Phase 1 trials are designed to test safety before any approval from the Food and Drug Administration to move on to Phase 2 trials, which test efficacy, researchers are cautious. They generally decline much comment for fear about running afoul of the bureaucrats.
But patients themselves are free to talk to anyone they want, and Harada was eager to tell his tale.
Harada, 40, is a former manager at FedEx who first noticed symptoms of ALS in 2009 while playing Marco Polo with his kids in the family swimming pool.
On March 9, 2011, he got an injection of 500,000 stem cells — the cells were derived by Rockville, Md.-based Neuralstem Inc. after a patient donated spinal-cord tissue in 2002 — as part of an 18-operation, 15-patient trial that last 2½ years.
The operations were conducted by Emory University Hospital physician Dr. Nicholas Boulis. The trial was designed, in part, by Dr. Eva Feldman, director of the A. Alfred Taubman Medical Research Institute at UM and director of the ALS clinic at the University of Michigan Health System. Boulis is a former colleague of hers at UM.
Harada was one of three patients who got two rounds of injections, the second this past Aug. 22. Researchers monitored all patients for side effects, of course, and the trials proved to be remarkably safe. The results were presented by Feldman in October at the annual meeting of the American Neurological Association in Boston.
Researchers also do a variety of tests on patients to look for signs of efficacy, too, to give them an idea of what they might expect should they get to Phase 2. Some patients showed little or no improvement. Others had modest gains.
Harada was off the charts.
When I interviewed Harada, he was feeling punk from fighting off a lingering staph infection and thought he was starting to see an improvement in symptoms as a result of the injection of cells Aug. 22. Because of the infection, it was hard to tell, and researchers at Emory hadn’t begun doing follow-up tests with him.
But there was no equivocation about the miracle that had happened after Harada’s first injection.
Two weeks after the operation, Harada thought he was feeling stronger, that there had been an improvement in his overall health. But he was afraid he was imagining things. That it was wishful thinking. Or a placebo effect.
Before the operation, Harada could barely limp with the help of canes or handrails up the steps to say goodnight to his kids at his home in McDonough, Ga. If he sat in a chair and his wife put the least bit of resistance on the top of his knee, he couldn’t budge his leg off the ground.
Harada didn’t wait for the doctors to test him.
“I asked my wife to come over and give me a test,” he told me in October.
She braced her hand against the top of his knee, as she had done many times. This time, though, his foot didn’t stay planted on the ground. It went up in the air.
They tried it, again. She pushed harder. He lifted his leg. A third time, his wife really pressing down her hand.
He lifted his leg.
She pushed down with two hands. He lifted his leg. “It was shock. ‘Is this real? This isn’t supposed to happen,’ ” Harada recounted to me.
He called the folks at Emory to tell them the news. He doesn’t blame them for what happened next. They tried to temper his enthusiasm. They explained the power of placebo effects.
“I know what a placebo effect is. I’m not crazy. This isn’t a placebo effect,” Harada responded.
“If anyone was more surprised than me, it might have been my doctors,” he told me.
Subsequent tests showed emphatically that what was going on — the mechanism of which is still not understood — was clearly not a placebo. Across a range of tests, there was demonstrative, clear, seemingly miraculous improvement.
“Every night I went to bed worried I’d wake up and it would be gone, that I’d have made the whole thing up,” he said. And every day for two or three months, not only did he wake up and hadn’t made the whole thing up, he woke up stronger than when he went to bed.
“I continued to improve in quantum leaps,” he said.
About a year after the operation, Harada began to notice a gradual decline, a decline that continued until his second operation — though he was still stronger when he went into the second operation than he had been going into the first.
When I talked to him in October, Harada was pretty sure he was feeling a little better but was tempering his expectations. “It would have been greedy to expect such good results, again,” he said.
Today, though, his staph infection has been cleared up, and there’s empirical evidence another miracle is taking place.
“I’m definitely getting stronger, there’s no doubt. Tests are showing beyond a doubt I’ve gained strength again,” Harada said. “I have more energy. My legs don’t get tired as quickly as they did. My hands have gotten stronger, again.”
By Oct. 20, Harada was feeling strong enough that he took part in a 2.5-mile fundraising ALS walk in Atlanta.
“If the walk had been in July, I wouldn’t have attempted it,” he said. “After a third of a mile, I would have been done. I would have sat down and said, ‘Someone come pick me up in a car.’ “
Harada did the 2.5 miles, no problem, still going strong when he hit the finish line.
Harada said one researcher told him after putting him through his tests on a visit earlier this month that, in Harada’s words: ” ‘If I hadn’t seen it with my own eyes, I wouldn’t believe it. If I was at another hospital and reading reports about you, I’d say it had to be B.S.’
“I’ve been blessed beyond belief,” he said.
Harada still has ALS. He still knows the likely prognosis is death. For him. But based on what has happened to him, there’s hope the prognosis of death won’t always accompany the diagnosis. Not now, not that there’s clearly some possible mechanism for improvement, something researchers need to understand and refine.
Feldman is awaiting approval from the FDA for a Phase 1B trial that she hopes will begin soon in Ann Arbor. It involves injecting three patients with 1 million stem cells, double the dose of the first trials.
If there are no ill effects from doubling the amount of stem cells, a Phase 2 study of 32 patients could begin next summer.
It’s worth repeating Harada’s words: “We’ve got to turn Lou Gehrig’s disease into Lou Gehrig’s chronic illness.”
Based on what’s happened, and what is happening, with Harada, that no longer seems like wishful thinking.
From: Thomas J. Murphy
To: FDA CDER
1. I feel that we in the ALS community have an unprecedented and historic opportunity (not Pilot!) with this initiative. For the first time in over 100+ years we can actually SEE some real progress with respect to SEVERAL treatments that are currently in FDA sanctioned Clinical Trials that have a realistic chance to slow disease progression, improve quality of life and/or extend our lives. Just to name a few - Biogen IDEC Dexpramipexole (Phase 3 complete and Phase 3 extension started), Cytokinetics CK-357/Tirasemtiv (Phase 2 complete and embarking on Phase 2b), Neuraltus NP001 (Phase 2 complete - results being tabulated), Neuralstem NSI-189/MDD stem cell treatment (Phase 1 complete and Phase Ib underway) and Brainstorm Therapeutics NurOwn stem cell treatment (have applied to the FDA for a Phase 2 trial and awaiting decision).
2. None of these treatments I mentioned have been formally approved or conditionally approved for marketing as yet by the FDA and there has also been no movement in the area of Expanded Access/Compassionate Use in these areas … but if there was EVER a critical point in time for all ALS Patients to have a say in the risk/benefit decision process it is RIGHT NOW when several treatments should be being considered for approval, conditional approval and/or Expanded Access.
3. I feel (and so do a significant percentage of ALS Patients) that ALS could greatly benefit from a more systemic and expansive approach to obtaining the patient perspective on disease severity or unmet medical need —- and most importantly could become a terrific EXAMPLE for the success/non-success of this PDUFA V initiative given the confluence of several decision events that have the potential of being in front of us all within the next 6-18 months.
4. I’d like to recommend that these sessions and processes designed to create a more systemic and expansive approach to obtaining the patient perspective on disease severity or unmet medical need absolutely include not only the patient, caregivers and the FDA but very importantly – the pharmaceutical companies and the disease medical doctors and experts – each of which are critical players and stakeholders in this process. I feel that the dialog will be much more productive and effective if we have all of the critical influencers in the room TOGETHER.
5. Lastly, I’d like to say that this unprecedented opportunity for the FDA and the ALS Community also brings with it the chance to take advantage of Broad Patient Inputs via Patients Like Me (PLM) and the 5,500+ ALS patients that are members of PLM.
The EMPOWER (Dexpramipexole) Phase 3 Clinical Trial ended for me on 10/5/2012 and I started on the ENVISION Extension Trial on the same day —- http://clinicaltrials.gov/ct2/show/NCT01622088?cond=%22Amyotrophic+Lateral+Sclerosis%22&lup_s=09%2F24%2F2012&lup_d=14&show_rss=Y&sel_rss=mod14
The following information depicts my progression since January 2011 when I started keeping metrics:
Hoping that my involvement in this Expansion Study does not preclude me from participating in the Neuralstem and Brainstorm Stem Cell Trials or the Cytokinetics CK-357/Tirasemtiv and Neuraltus NP001 follow-on Trials that all seem to show tremendous possibilities for the ALS community.
Also hoping that when the “24 month since diagnosis” line in the sand hits in December that I won’t be kept from participating in ALS Trials as has been tradition …
Can’t beat it — she’s just about 1 year old and a very special little girl …
I know I’ve posted this before but it is one of my favorite pictures of my little brother Patrick and our times together fishing …
Pat - you’d be very proud of Sean and Maggie today - they are beautiful and loving human beings …
We all miss you very much and I’ll see you soon - save a rod for me buddy …
P.S. … and I know you are smiling a lot more knowing that bin Laden has been ejected from the world since 5/2/11!
If you haven’t seen this moving video - I highly recommend it …
Also can be found at http://isaiah35-6.tumblr.com/
Among the most common neuromuscular diseases worldwide, amyotrophic lateral sclerosis (ALS) attacks motor nerve cells that control voluntary muscles. It was first described by French neurologist Jean-Martin Charcot in 1869.
WHO GETS IT?
An estimated 5,000 people in the U.S. are diagnosed with the disease each year; 30,000 have it at any given time. ALS has been diagnosed in people of all races and ages and is most common in people ages 40-60. Men are affected more often. Five percent to 10% of cases are inherited.
WHAT HAPPENS TO PEOPLE WITH ALS? Patients lose strength and the ability to move their arms, legs and body. When muscles in the diaphragm and chest wall fail, patients can no longer breathe without ventilator support.
WHAT’S THE LIFE EXPECTANCY? Most people with ALS die from respiratory failure, usually within three to five years of the onset of symptoms; about 10% of ALS patients survive 10 years or more. HOW IS IT TREATED? Rilutek is the first and only medication approved by the U.S Food and Drug Administration for ALS. It appears to extend life by a few months, but it cannot restore the physical strength.
How the stem cell trial works
The current trial led by University of Michigan researchers involves injecting 500,000-1 million stem cells into a patient’s spine. Dr. Nicholas Boulis, a former U-M resident of neurosurgery and now chief surgeon for the ALS clinical trial in Atlanta, invented the device used in the surgery. Maryland-based Neuralstem, which is funding the first phase of the trial, holds the license.
From Michigan and Diagnosed 2 years ago - ALS has reduced Regis Kleiss, 28, a formerly thick-bodied shot and discus thrower and captain of the high school track team, to a bony echo of himself.
Paralyzed except for some minor movement he can make with his head, he will spend his final days on a feeding tube.
He is hoping to get involved in the Neuralstem trial that has been based in Atlanta since 2010, but Michigan has requested approval from the FDA to expand it and move it to Ann Arbor.
We really need the FDA to begin to encourage Expanded Access to and Early Approval of the potentially effective and medically appropriate investigational drugs/treatments that exist right now for ALS sufferers who die at the rate of 15 per day.
Most ALS Patients believe that there are several treatments worth exploring RIGHT NOW for Expanded Access and Early Approval such as NP001, Dexpramipexole, CK-357, Nerualstem Phase I stem cell trial and the Brainstorm Phase I/II clinical trial with NurOwn™.
Each of these are drugs/treatments that have performed favorably in human safety studies/trials and have already shown positive signs of efficacy at modifying the disease — we need treatments like this RIGHT NOW because most of us don’t qualify for the clinical trials and we don’t have time to wait the traditional 8 - 14 years for FDA approval of a treatment.
Just to let everyone know that with your very generous help - Murphy’s Might, ALS Treat Us Now and ALSTDI had a very successful ride and hopefully have made a difference in the acceleration of safe and effective treatments into the ALS Community world-wide.
As of this morning, Murphy’s Might has raised a total of $20,755.00 with $15,735.50 going to ALSTDI (#7 on the list of top fundraising teams) and $5,019.50 to ALS Treat Us Now.
Many thanks to my son Tom, my brother-in-law Tony Scarfo and Scott Corson for the riding they did - it was phenomenal!
I only rode about the last 7 miles to the finish line with Tom Jr. - but next year I think maybe I’ll try the last 12 miles (I think I could have done that this year but didn’t want to push it too much).
A few pictures below - it was a great experience! Once again, I thank you all for your generous donations for this very important cause.
Around the horn from top left: Tom Jr. - Tom Sr. - Scott and Tony
Tom Jr. and Tom Sr.
What took place this past month at Hadassah Hospital’s Department of Neurology was not expected even by the most senior neurologists.
An ALS patient- a chronic disease with no known cure that causes a slow and painful process of muscular degeneration - who was wheelchair-bound and had difficulty speaking and breathing, underwent a complete turnaround. From being handicapped and dependent on others for his most basic daily needs, suddenly Rabbi Rafoel Shmuelevitz stood on his feet, and returned to teaching at the flagship Mir Yeshiva, which he heads.
Rabbi Rafoel Shmuelevitz recalls, “I couldn’t talk. It was difficult for me to breathe, and my lack of balance made it impossible to get up from my wheelchair. Even when they supported me I was able to walk only with difficulty. My students couldn’t understand me when I spoke.”…
The treatment that is responsible, it appears, for the dramatic improvement in his condition was developed by the Israeli biotechnology company Brainstorm, and is based on stem cells. This is the first time that someone who has been treated with stem cells has regained abilities which were previously lost. Despite the great caution that is guiding the doctors as they discuss the rabbi’s condition, it is difficult to ignore the excitement which surrounds even the most stern of staff members when they talk about the turnaround that the rabbi has experienced…
=== WATCH THIS FOX NEWS VIDEO INTERVIEW ===
SOURCE: Brainstorm Cell Therapeutics Inc.
June 25, 2012 00:21 ET
An End to Amyothrophic Lateral Sclerosis (ALS)?
Although the primary purpose of the Phase I/II trial being conducted at the prestigious Hadassah Medical Center in Jerusalem is to ensure safety and tolerability, initial data has shown efficacy. Professor Eldad Melamed, the lead investigator of the trial, said, “There have been no significant side effects in the initial patients we have treated with BrainStorm’s NurOwn™ technology. In addition, even though we are conducting a safety trial, the early clinical follow up of the patients treated with the stem cells shows indications of beneficial clinical effects, such as an improvement in breathing and swallowing ability as well as in muscular power. I am very excited about the safety results, as well as these indications of efficacy, we are seeing. This may represent the biggest hope in this field of degenerative diseases, like ALS.” Dr. Adrian Harel, CEO of Brainstorm, indicated trial data from the first cohort of 12 patients with ALS will be made available to the public sometime in July 2012.
It is expected that a Phase II/III ALS trial application will be filed with the FDA in the United States in the second half of 2012. Brainstorm recently received FDA Orphan Drug Designation for its NurOwn™ cell therapy for ALS in the U.S. The trial site in the U.S. will be conducted at Massachusetts General Hospital (MGH) and is expected to begin late 2012.
Brainstorm Cell Therapeutics is working with the Northeast ALS (NEALS) consortium to design a Phase II clinical trial in the United States.
A protocol is currently under development for ALS human clinical trials at Massachusetts General Hospital (MGH) and the University of Massachusetts Medical School.
The University of Massachusetts Medical School team will be led by Professor Robert H. Brown, MD, DPhil., Chair of the Neurological Department at University of Massachusetts Medical School. Professor Brown is a leading expert in neuromuscular genetics and is world renowned for his expertise in ALS.
The MGH team will be led by Prof. Merit Cudkowicz, an expert in clinical trial design and therapy development for neurodegenerative diseases. She co-chairs the Northeast ALS consortium and directs the MGH Neurology Clinical Trial Unit.
On June 27, 2012, I had the following short dialog with Dr. Merit Cudkowics related to my possible candidacy in the U.S. Trial to be sponsored by Massachusetts General Hospital:
From: Cudkowicz, Merit E.,M.D.
Sent: Wednesday, June 27, 2012 9:14 PM
To: Thomas J. Murphy
Subject: RE: Possible candidate for planned U.S. Phase II/III trial involving NurOwnT cell therapy for ALS ?
Dear Mr Murphy,
Thank you for your email. The criteria for the study won’t be final until we have FDA and IRB approval. Lets stay in touch (by email if helpful) - and once we know more about status of study, we could talk more about how to enroll.
If helpful, we could have an email by end of summer? Merit
From: Thomas J. Murphy
Sent: Wed 6/27/2012 10:21 AM
To: BrownRHJr; Cudkowicz, Merit E.,M.D.; firstname.lastname@example.org
Cc: MGH ALS Trials
Subject: Possible candidate for planned U.S. Phase II/III trial involving NurOwnT cell therapy for ALS ?
Professor Brown, Professor Cudkowics and Dr. Harel …
As a person with ALS (diagnosed 12/8/2010), I am a strong advocate of anything that will speed access to new treatments for people with ALS —- improving and strengthening the FDA’s Fast Track and Accelerated Approval processes and incentivizing the FDA to approve new treatments quicker.
Myself and others who are members of ALS Treat Us Now (http://www.treatalsnow.org) are very focused on treatments that have shown early indications of safety and efficacy in Phase I and II trials and getting those into the ALS community as soon as humanly possible for those of us who have no other options but death and don’t have time to wait for the traditional process to run its course.
I am very interested in possibly participating in the planned U.S. Phase II/III trial involving NurOwn™ cell therapy for ALS should the FDA approve the application that will be filed in the second half of this year and might get started in late 2012. I have been a participant in the EMPOWER Dexpramipexole Trial since May 2011 and have had a non-declining FRS for 12 months – and am still able to walk, talk, eat, swallow, work, etc. without assistance of any kind. In fact, I plan to attempt to ride at least 10 miles per day in the upcoming ALSTDI Tri-State Trek.
Maybe you can get back to me and let me know if I would be a viable candidate for this planned trial via MGH and Brainstorm Cell Therapeutics and if you would strongly consider my candidacy for participation.
Did an Israeli Company Develop a Cure for ALS?
Monday, July 02, 2012 - Iddo Genuth
Unprecedented Medical Miracle: 1st ALS Patient to be Cured is Mir Rosh Yeshiva
This summer - I - along with the Murphy’s Might Team - my son Tom Murphy, Jr. and brother-in-law Tony Scarfo - will attempt to ride as many of the 270 miles from Newton, MA to Greenwich, CT as possible to help the ALS Treat Us Now and ALSTDI organizations to identify and quickly bring safe and effective treatments to ALS patients - there are NO effective treatments that exist today for ALS patients.
Transitioning from the Paved Roadway …
Transitioning from the Paved Roadway …
On 8 December 2010 I was formally diagnosed with ALS and began the steady transition from the safe ”Paved Roadway” that I have been so familiar with in my life to a more unstable and less friendly path where there are unpleasant surprises awaiting me as I undoubtedly face what is the toughest journey and challenge of my life.
This summer I am riding some number of the 270 miles with the Murphy’s Might Team from Newton, MA to Greenwich, CT to help the ALS Treat Us Now and ALSTDI organizations to identify and quickly bring safe and effective treatments to ALS patients- there are NO effective treatments that exist today for ALS patients.
I truly hope that you can support the efforts of ALS Treat Us Now, ALSTDI and the Murphy’s Might Team as we do our part to make a difference in the acceleration of safe and effective treatments into the ALS Community.
Given the cooperation between ALS Treat Us Now and ALSTDI – the Murphy’s Might Team would like to have your generous donations split with 50% going to ALS Treat Us Now and 50% to the ALSTDI Tri-State Trek. Here are the links that provide the donation instructions for both organizations:
***ALS Treat Us Now Donations *** (Credit Card or Check)
Check - all checks should be written to “PCUT / ALSTUN” and sent to:
PCUT - Attn: ALSTUN - PO Box 4845 - San Jose, CA 95124. If you send a check … please send us an email at email@example.com let us know.
***ALSTDI Tri-State Trek Donations*** (Credit Card or Check) https://tst.als.net/Donate/murphysmight
Please also take some time to learn about and support the mission and objectives of ALS Treat Us Now at: