I’m done playing nice.
I’m supposed to be dead.
This is an urgent call to action. Keep reading.
I need your help!
With an ALS diagnosis in 2013, I was given 2-5 years. I’m alive but being protected to death by the FDA, and I’m not alone. Every 90 minutes someone is diagnosed with ALS and every 90 minutes someone dies from ALS.
The 2014 Ice Bucket Challenge raised over 100 million dollars for research. Today, promising treatments are sitting in the FDA pipeline. It takes decades and billions of dollars to get an effective treatment approved.
We’ve played nice and we’ve been ignored.
Two years ago, the National ALS Association (ALSA) wrote an FDA Guidance Document that is being lauded as the gold standard of industry guidance. Half of the 14 patients on the guidance committee died waiting for public comments. The FDA just released Its version of ALS Industry Guidance. I, the remaining committee members, and thousands of others will die waiting because the guidance does very little to advance treatments through the lengthy and costly approval process.
The voice, concerns, and suggestions of patients and our allies were largely ignored in the original guidance and our Call to Action was buried in the very last pages of a 139 page document. We had high hopes that the efforts of so many would dramatically impact drug trial design and approval process. Our lives depend on it. Nope. The new and exciting guidance from the FDA Includes: patients with ALS are willing to take greater risks because the condition is terminal. Thank you, Captain Obvious.
I’m waiting to test the California Right to Try law or use the FDA’s Expanded Access Program when a new drug I’ve been following passes Phase 1 safety and tolerability studies. There is no requirement for the company to give me the drug, only a moral imperative. I gladly spent a total of six weeks in the hospital and dozens of appointments in San Francisco. I have great hope that this new molecule will dramatically slow my progression or stop it all together. The trial is still in Phase 1 two years later. Waiting those two years cost me the functional use of my hands and arms, it cost other people their lives.
We are not asking for a safe and effective treatment for erectile dysfunction. We are demanding access to potential life-saving treatments that are stuck in a 15 year, billion dollar approval process. Answering FAQs and minor tweaking don’t come close to the paradigm shift required to save lives today. Stem cell therapies and promising treatments exist but are not accessible.
ACT UP seized the FDA 30 years ago with the same obstacles facing people with HIV/AIDS. It’s time for us to do the same. Most with ALS can’t march or yell because many are paralyzed and can’t speak. But we have assistive technology and 400 pound wheelchairs. Get on board or get out of our way.
Urgent Call to Action
- Immediate access to promising treatments that have passed safety studies.
- No placebos in clinical trials, use historical controls instead.
Stop protecting us to death. The cost of a military parade could provide life saving treatments to every American living with ALS. US veterans like me are twice as likely to have ALS. It’s time for the US Government to cut through the regulatory red tape of the FDA and provide life-saving treatments now.
What’s Next?
The patient community has the right and responsibility to get loud and demand our voices be heard. We need 1,000 public comments to get the FDA’s attention. You can help by leaving a public comment of your own. Click here.
Tell your story and demand access to treatments and radical changes in how ALS drug trials are designed now! Status Quo = Death.
It’s easy to comment. Here’s how:
- Click here to go to the public comment website.
- Type your comment. Note: It’s best if you write your own comment, but here are some suggestions on what to include:
1. Your relationship to ALS (patient, loved one, friend, etc)
2. A statement about how this guidance document is unacceptable. For example: The proposed guidance document doesn’t adequately address the urgent needs of patients who have a terminal illness – especially an illness without an effective treatment. It is cruel to withhold access to treatments that have been proven safe and show promise, but are stuck in a 15 year, billion dollar approval process. It is time for the FDA to stop protecting ALS patients to death. ALS patients need immediate access to promising treatments that have passed safety studies. ALS clinical trials should NOT require placebos and use historical controls instead. - DO NOT put your name and contact information in the comment as it will become part of the public record. There will be a place later in the process to put this information. It is optional and will not become part of the public record.
- Uncheck the checkbox that says: I am submitting on behalf of a third party.
- From the drop-down category menu, choose: Individual Consumer
- Your comments won’t post until they have been reviewed by the FDA, so don’t be surprised if you see zero comments posted.
Make your public comment and share this far and wide. Thank you.
If you’re having trouble submitting a comment on the above link, try here and click the green button. Note that the 270 comments listed refer to a previous document from 2013.
If our voices are dismissed again, we will show up at their door demand action. Imagine 250 power wheelchairs at the FDA headquarters. Here’s how ACT UP did it 30 years ago.
***
Shelly Hoover 
February 19, 2018 at 5:58 pm
Shelly, Here is what I just submitted
Dear FDA:
My childhood friend and former classmate has ALS. I am writing to plead with you to enable people with ALS to have
immediate access to promising treatments that have passed safety studies and to conduct clinical trials with historical controls, not placebos.
Two years ago, the National ALS Association (ALSA) wrote an FDA Guidance Document that has been lauded as the gold standard of industry guidance. The FDA hasn’t adopted this guidance, issuing, instead, its own ALS Guidance for Industry which doesn’t come close to provide the paradigm shift necessary to save lives. Your document does acknowledge that “patients with ALS are willing to take greater risks because the condition is terminal.” If you can so casually mention such a painful and obvious aspect of this horrid disease can’t you also take the steps that will allow ALS patients to cut through the regulatory red tape of the FDA and provide life-saving treatments now?
It is cruel to withhold access to treatments that have been proven safe and show promise, but are stuck in a 15 year, billion dollar approval process. It is time for the FDA to stop protecting ALS patients to death. ALS patients need immediate access to promising treatments that have passed safety studies.
Please, do the right thing and work to make the necessary changes so ALS patients can access treatment.
Thank you for sharing this information and giving me, and others, the opportunity to do something to comment on this atrocity.
I have read and, I believe, commented, on every post you have made, until the last 2. I apologize. I read your Life or Death post and was so struck with the horror of the “choices” (using quotes because I can hardly consider them choices) that I didn’t know what to say. Cowardly.
I read your Let Me Know if There is Anything You Need post and wished I lived nearby so I could do any, or all, of those things. I also realized that I need help with some struggles, trivial in light of what you are facing. You gave me guidance how to better offer assistance to others and how to try to ask for it for myself. Your ability to share your self with others so that we learn from you is remakable. Your generosity in sharing yourself is brave and your insights are wise.
Again and again I say how sorry I am you are affected by ALS. It is a horrible disease which you and Steve are fighting with faith, courage, humor and grace. Thank you for sharing your journey with us. Thank you for letting us raise our voices to the FDA to tell them that they have a moral obligation to make treatments available.
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February 19, 2018 at 6:14 pm
Thank you Eileen! Your thoughtful words and encouragement mean the world to me!
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February 19, 2018 at 7:22 pm
Mrs. Hoover, I sent this message for Right to Try legislation in memory of my dad. I hope I did it right and I hope it helps:
Dear FDA
I strongly suggest you support Right to Try legislation. Because My father, Steve Mayfield was a highly respected high school coach in North Alabama, but roughly four years ago he was force to retire due to his diagnosis of ALS. Not only did he fought ALS but the FDA’s bureaucracy and on March 13th, 2017 he passed away from ALS. My dad was a wonderful man who, like other ALS patients, deserved a Right to Try experimental treatments that could potentially save their lives. But tragically my father never had the chance to even try. The FDA owes it to the families of those who have lost their lives to ALS, and those who are currently fighting ALS because denying their rights is merely cruel, destroying freedom itself.
I highly support the Senate bill S.204 Right to try act of 2017 sponsored by Senator Ron Johnson, and has passed the Senate through unanimous consent, but is to this day, waiting in the US House. The reason I still fight for ALS patients and other terminally ill Americans is because I do not ever want any family to go through the pain and suffering my dad and my family had to endure when he suffered and lost his life to ALS, especially when there was a simple way of preventing his death. Please allow S.204 Right to Try Act of 2017 be put to a Floor Vote in the US House of Representatives and have it passed as soon as possible.
Also, the FDA must listen to the people and support Right to Try. I remember when I sent an email on behalf of my dad, they said that if it was their father who had ALS they would do the same thing I did for my dad. So, here’s the question, what makes the People of America different? There is no difference because all human life is sacred and should be taken care of with the utmost decency. Let the terminally ill people of America choose for themselves. Because they have every right to decided what to do with their own lives. Right now, in some states, there is a Right to Die Law, which allows terminally ill patients to be legally euthanized to end their suffering, but yet we do not have a Right to Try law that could potentially save their lives when they have nothing else to lose? That is just too cruel, horrible, no better than tyranny. That is why I stand firmly for America and ask you please allow the Right to Try Act of 2017 (S.204) to be put on a Floor Vote and be passed by the US House so the President who supports Right to Try, can sign it. This is a human rights issue, not a political one. So please, let us choose for ourselves so we can have a chance to live. Because my dad would want this as well.
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February 19, 2018 at 8:31 pm
going to reblog
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February 19, 2018 at 9:22 pm
This is a critical issue. This does not just affect ALS , this type of legislation will affect Epilepsy, cancer, and other diagnoses that debilitate or even lethal.
The “Right to Try” could possibly save lives. As long as the patient is informed and educated of the possible risks, it is their body to try and find relief or a cure.
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February 19, 2018 at 11:19 pm
i commented – thanks for being a leader Shelley
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February 20, 2018 at 5:18 pm
I have two friends currently towards the end of their journey with ALS. If there is a possible treatment available in the FDA pipeline, PLEASE release it without the usual placebo protocols . These people dont have time left in their lives for that. DO IT NOW and give them a chance.
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February 20, 2018 at 8:18 pm
We need hundreds of comments at regulations.gov !
Here is some guidance that the FDA issued for drug development for a specific bladder cancer https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM529600.pdf
“5. Single-Arm vs. Randomized, Controlled Trial Design
Single-arm trials are appropriate in clinical settings where a randomized, controlled trial is either
unethical or not feasible. Randomizing patients with BCG-unresponsive NMIBC to a placebo or
minimally effective drug as a concurrent control raises ethical concerns. Currently, single-arm
trials are appropriate for assessment of therapies for patients with BCG-unresponsive disease
(CIS with or without resected papillary disease) because, currently, no effective medical
therapies are available and the only alternative is radical cystectomy. Sponsors should use
randomized trials in clinical settings in which an active or placebo control or a time to event
endpoint is appropriate. If effective therapies become available in BCG-unresponsive NMIBC, a
randomized trial may be appropriate.”
It’s outrageous that they are being rigid on double-blind, placebo trials for people dying from ALS.
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February 22, 2018 at 1:19 am
Here’s a letter I wrote them about NurOwn:
Paul S. Rowe
February 11th, 2018
Dr. Peter Marks
Center for Biologics Evaluation and Research, FDA
10903 New Hampshire Avenue
Building 71 Room 3103
Silver Spring, MD 20993-0002
Dear Dr. Peter Marks,
My name is Paul S. Rowe. I am an educator at Endicott College in Beverly, Massachusetts, a town that has been struck time and again by the devastating neurological disorder known as A.L.S. Many of those affected by this have been young. Peter Frates was diagnosed at the age of 27 in March of 2012 and given 2-5 years to live, while another Beverly resident named Bobby Forster was diagnosed at the age of 25. We stand at the threshold of a turning point for these patients. As you are aware, the company Brainstorm Cell Therapeutics has begun a pivotal phase three trial of its stem cell therapy NurOwn for the treatment of A.L.S. This technology converts mesenchymal stem cells into biological factories secreting neurotrophic growth factors. These growth factors have been proven to support the survival of neurons in humans in over 60 patients through clinical trials conducted in both the United States and Israel, establishing an excellent safety and tolerability profile while demonstrating clear signs of efficacy. Bobby Forster was a participant in the U.S. phase two trial of NurOwn conducted at Mass General Hospital. After two weeks of treatment he went from barely able to stand for more than ten seconds, to being able to walk with a walker, to being able to walk unassisted. He also saw significant improvements in his forced vital capacity (FVC) and speech. His petition to the FDA and Brainstorm Cell Therapeutics to grant A.L.S. patient access to NurOwn has reached 136,000 supporters online. Now, Bobby lives in Newport. He is completely incapacitated, waiting for access to this groundbreaking treatment.
Osiel Mendoza is another A.L.S. patient and advocate for treatment who might benefit from NurOwn. During his senior year at the University of Oregon, Mendoza was diagnosed with A.L.S. at the early age of 21. Unfortunately, he isn’t alone. He is one of many young people facing this disease without an effective therapy in 2018. This disease strikes people at younger ages these days, and you are in a position to accelerate approval of NurOwn as Director of the Center for Biologics Evaluation and Research. I’ve attached a video of Osiel Mendoza’s graceful speech at the A.L.S. Therapy Development Institute in Cambridge, M.A. “I’m not the only one,” Mendoza says. “There are others and there are many of them. But I’m standing right here tonight representing all of them while I can still stand. I’m the newest and I’m the youngest generation of A.L.S., and I really need to be the last. So please help.” You can help, Dr. Marks. You can work closely with Brainstorm Cell Therapeutics to accelerate the approval of NurOwn. Furthermore, you have an opportunity to evaluate the midline data from the ongoing phase 3 trial of NurOwn and approve this lifesaving treatment based on those results. As you know, the fight against A.L.S. is a race against time, and the FDA’s current one-size-fits-all system doesn’t work for a swift neurodegenerative disease such as A.L.S. These are cold, hard facts of the matter. Financial economists such as M.I.T. economist Andrew Lo has aptly noted that the FDA’s current system of approval treats all medical conditions the same way, from A.L.S. down to acne. Until reform is achieved and patient risk is judged on a disease by disease basis, the threshold for approving A.L.S. therapies will not be lowered and pharmaceutical companies will not have the proper incentives to invest in treatment. Nevertheless, you can act now. Dr. Marks, I’m asking you to provide A.L.S. patients like the ones I’ve mentioned above with access to a treatment that will alter the trajectory of their disease, prolong their lives, and keep them around until a cocktail of treatments to stop progression or a cure is discovered. You can save lives if you commit to the swift approval of NurOwn. I trust you will make the right decision to create a brighter future
Thank you for considering this opportunity to change the outcome of the battle against A.L.S. I look forward to hearing back from you.
Respectfully, Paul S. Rowe
CC: F.D.A. Commissioner Dr. Scott Gottlieb and Director of the Center for Drug Evaluation Research Dr. Janet Woodcock
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February 22, 2018 at 1:52 am
Awesome! Did you get a response?
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February 22, 2018 at 9:02 pm
Please keep us posted on how we can stay engaged with you. I LOVE the idea of showing up at the FDA. Count me in!
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February 22, 2018 at 9:14 pm
Thanks Craig. It’s up to us to demand change!
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May 9, 2021 at 5:35 am
My first symptoms of ALS occurred in 2014, but was diagnosed in 2016. I had severe symptoms ranging from shortness of breath, balance problems, couldn’t walk without a walker or a power chair, i had difficulty swallowing and fatigue. I was gien medications which helped but only for a short burst of time, then I decided to try alternative measures and began on ALS Formula treatment from Tree of Life Health clinic. It has made a tremendous difference for me (Visit w w w. treeoflifeherbalclinic .com )
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