We Can't Wait Action Meeting with FDA: What We Heard and What's to Follow

We Can't Wait Action meeting

We are  deeply grateful to the eight speakers who shared their wisdom and personal experience with the FDA,  as well as the  more than 150 others from the community  who have done so  online. We continue to hear that people with ALS want effective treatments now, even if they offer modest benefit, and are willing to face considerable uncertainty and risk to do so.

We know that 17 FDA staff in attendance heard the same message. It was clear from her remarks that Dr. Patrizia Cavazzoni heard our community.  She spoke about the need for new treatments, that treatments might have modest benefit, and that they might not work for everyone with an ALS diagnosis. She talked about the need for regulatory flexibility and speed, and expanded access to experimental drugs. We are heartened by her message, and believe it was a genuine response, we need to make sure this is followed up with action. With words must come concrete actions.  

In the immediate future, we will be asking the FDA to recommit to its 2019 ALS clinical trial guidance, to explain how it has implemented it, and how it plans to get effective treatments to people with ALS faster. We will be asking for a detailed report on these issues with a deadline.  

This is part of a larger strategy to ensure the FDA is as fast and flexible as possible. This strategy entails several additional actions. In our appropriations request to Congress, we are asking for $50 million for the FDA to fund research on treatment and regulatory issues to benefit people with ALS. Second, we are supporting the Promising Pathways act to give the FDA additional tools to work faster. In addition, not only does the newly released ACT for ALS expand access for ALS treatments, it would give the FDA additional research capacity and public private partnerships to speed the development and approval of new ALS treatments. And, we are asking Congress to fund a study by the National Academies of Science and Medicine to help coordinate the entire ALS research agenda.   

This is a complex set of interrelated proposals that should help keep the FDA on task.  We will discuss them further at our annual Advocacy  Conference,  which begins on June 8. (If you have not done so, you can register for the virtual conference here.) The recording of today’s meeting and the portal to submit additional testimony from people living with ALS are accessible at www.als.org/fda.

This morning  was very inspiring, we want to genuinely thank the brave individuals with ALS who spoke their truths. Our urgent work continues. We will keep you posted and look forward to discussing these issues further at our Advocacy Conference.   

We Can't Wait Action Meeting


Submitted by: David D. on Wed, 05/26/2021

Dr. Cavazzoni clearly accepted no resposability for the lack of approved ALS drugs. However she told us to take a lesson from the Cancer Community and learn more about our disease and develop targeted therapies... This is trash, first give us cancer money and we will develop targeted therapies. Also accept the responsibility thay you and the FDA have failed to use flexibility and speed when dealing with ALS. I watched, I listened, now prove to the community you are not one more mouthpiece in the line of many before you!

Submitted by: Mary T. on Thu, 06/03/2021

My sister lived with ALS for ten years; it was painful to watch her deterioration yet we were blessed with her strength and persistence. Trials that can enhance the ALS patient's life would ease so many individuals. I support the continued effort to expand research and development of medications and therapies.

Submitted by: Larry G. on Fri, 06/04/2021

I was diagnosed with ALS 4 1/2 years ago. I had symptoms a year before being diagnosed. I LIVE ALONE. I am in a wheelchair but can get around the house in a rollator. I still drive. I work for a major hospital in SW MO. I cannot wait for 2030,,probably not even until 2022 or 2023. I am disappointed that we have no one willing to go out non the limb for us. Keep watching us die. How can you sleep at night?? How many people have to die an agonizing death...and life.

Submitted by: Amy L. on Tue, 06/08/2021

Hi Larry. So sorry to hear you are living with ALS. We are working every day to discover treatments and a cure for ALS, and to serve, advocate for, and empower people affected by ALS to live their lives to the fullest. We want ALS to be a livable disease as soon as possible.

Submitted by: Donna I. on Mon, 06/07/2021

My husband has suffered from ALS for the last four years. It has progressed to the point where he cannot move a muscle in his body and yet his mind is perfect and he is able to speak well. However, I am very disappointed that even at the beginning his doctors were Not forthcoming with clinical trials and availability for clinical trials for him. I had to ask the questions and force answers from them. Very disappointing and very frustrating.

Submitted by: William R. on Mon, 06/07/2021

I am a ALS patient..i lost my voice literally in this battle with ALS..
We need new ALS drugs to be released by FDA...please help us!!

Submitted by: Virginia D. on Tue, 06/08/2021

My husband was a healthy, active, athletic, incredible human being that was diagnosed with this horrible disease and succumbed within 3 years at 45 years old. He knew he was going to lose his battle, but he would have tried anything to try and help others diagnosed with this nightmare. Give them the opportunity, FDA, what do they have to lose?

Submitted by: Jasson R. on Fri, 06/11/2021

Iam 42 years old I have ALS iam progressing alot more these days with more bad days than good.i have found it is hard to get clinical trials to help live a better life or to find a cure.i have a 3 year old daughter and have come to the relisation that I will not be here for alot of her life.i would do anything and I mean anything to have one more second of one more minute of another hour of one more day with my family my wife and kids.it seems iam dieing a slow agonizing death of Wich there is no help.and the access of help is being ignored by the FDA if anything I wish they would open there eyes and help one more little girl and one more wife or one more family not go through what ours are.help us help ourselves open your eyes to the heart break that this disease does to everyone connected to it not just those of us living with the disease.

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