Delivered by Lauren Webb, LCSW, Chief Advocacy and Outreach Officer of the Les Turner ALS Foundation. See also remarks by Senda Ajroud-Driss, MD, Director of the Lois Insolia ALS Clinic at the Les Turner ALS Center at Northwestern Medicine.
Open Public Hearing
FDA Advisory Committee Hearing on tofersen
March 22, 2023
Good afternoon. My name is Lauren Webb and I’m the Chief Advocacy and Outreach Officer for the Les Turner ALS Foundation. My only disclosure is that we receive less than five percent of our annual funding from pharmaceutical companies, including Biogen.
Thank you for the opportunity to speak today.
Our foundation has been closely connected to SOD1-ALS for many years. In fact, SOD1 was co-discovered at our Les Turner ALS Center at Northwestern Medicine, and the Center was a site for both the Phase 3 VALOR clinical trial of tofersen and the open label extension.
We understand the science behind SOD1-ALS and we are encouraged by the results that suggest an early start and extended use of tofersen may help stabilize muscle strength, respiratory function and quality of life.
And with more than 45 years of experience supporting people living with this and all other forms of ALS, we also understand the urgency of the need for this treatment.
Our support services coordinators work with families who have lost loved ones across multiple generations to SOD1-ALS. From firsthand experience, we can testify the emotional trauma for these families is overwhelming.
We have worked with people who have lost both a spouse and a child to this disease. We provided a wheelchair to one woman and a few years later provided the same to her brother.
Speaking personally for a moment – early in my career, I met with a 35-year-old woman who had an A5V variant in the SOD1 gene. This was a very aggressive form of ALS that also affected her mother. I remember taking her 10-year-old daughter to get a snack while the genetic counselor explained the results to her parents. We sat in the hospital cafeteria and I quizzed her on her state capitals. That little girl lost her mother nine months later.
People living with SOD1-ALS need more than support from us. They need the chance to share more of their lives with their families. They need cause to believe the pain of this terrible disease will be a different experience for their children. They need hope.
Nothing could mean as much to these families as the first therapy to slow the progression of a genetic form of ALS. And that is what tofersen does.
I think back on that cafeteria and I want that story to unfold differently. I want to take that 10-year-old girl back upstairs to find her mother holding a copy of her SOD1 lab report and a prescription for tofersen. I want that mother to have a chance to see her daughter graduate.
We believe that tofersen represents a significant advancement in the treatment of SOD1-ALS. The need is urgent and unmet, and the evidence is compelling.
We urge you to recommend approval. Thank you.
