Delivered by Andrea Pauls Backman, CEO of the Les Turner ALS Foundation. Slides can be seen here (PDF).
Open Public Hearing
FDA Advisory Committee on AMX0035
September 7, 2022
Good afternoon. My name is Andrea Pauls Backman, CEO of the Les Turner ALS Foundation. My only disclosure is that the Les Turner ALS Foundation receives less than 2% of all annual revenues from pharmaceutical companies, including Amylyx Pharmaceuticals.
Since 1977, it has been our mission to advance scientific research for the prevention, treatment and cure of ALS and to provide the most comprehensive care and support to people living with ALS and their families so they can confidently navigate the disease. I want to add our perspective on what access to AMX0035 would mean to the people we serve.
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This is our Support Services Team. We take an individualized approach to ensure each person living with the disease receives the very best quality of care. We visit them in their homes, we join them at the clinic, and we check in by phone and video. We treat each person we serve like family. And every year, we lose about one-third of them to this terrible disease.
These are a few of the people we’ve lost since the last advisory committee in March.
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Mary Ann Batterman–Daeschler was a mother of four, stepmother of five, and grandmother of nineteen. When we talk about a treatment that can add 10 to 18 months to a person’s life, we’re also talking about more time that nineteen children could have with the grandmother they loved. Some will go through life with clear memories of their nana, and some will only know the pain of absence. All of them will feel that loss for decades to come.
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Michael Snedden loved cooking for big crowds because it was a way to bring friends and family together and show his love for them. His epic tailgates became a successful catering business because he enjoyed providing delicious food for the important events in people’s lives. ALS robbed him of the ability to cook for others, and it eventually took away his own ability to eat. A treatment that could have slowed his decline of function by as much as 25% would have meant the world to him and to his loved ones.
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This is Kathleen Friend. Like Mary Ann and Michael, she is survived by a large and loving family. She loved Christmas, puzzles, and music. In every way, Kathy was larger than life. But ALS confined her within her body. Before she died, she told her family that when she dreamed, she was running free.
Today should have been her 73rd birthday.
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There is an urgent and unmet need for safe and effective therapies for ALS. On average, people live 2 to 5 years following an ALS diagnosis. Imagine what 10 to 18 more months would mean to them. Imagine what it would mean to slow the loss of their dignity and independence by as much as 25%, and spend that much more time sharing the memories and experiences that make life worth living.
On behalf of the people we serve, on behalf of the people we’ve lost to this disease and the loved ones they’ve left behind, we urge the FDA advisory committee to recommend full approval of AMX0035. We have no time to waste.
Thank you.
