I AM ALS statement at NASEM ALS conference

I AM ALS joined other partners on May 18, 2023 for the National Academies of Sciences, Engineering and Medicine (NASEM) for their meeting titled “Amyotrophic Lateral Sclerosis: Accelerating Treatments and Improving Quality of Life.” Below are the comments from I AM ALS’ very own Sonya Elling.

You can watch the full discussion here, with Sonya’s remarks beginning at 1:13:32.

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Good morning. My name is Sonya Sotak Elling and I am the Chief Government Affairs Officer for I AM ALS. It is my distinct privilege to be here today on behalf of our organization, driven by an incredibly passionate community advocating tirelessly on behalf of over 30,000 people living with ALS and their caregivers and loved ones. 

Before answering your specific questions for this panel I want to recognize the importance of the work you are doing and thank each of you for the effort you are putting into gathering this important information to make ALS a livable disease within a decade.  

A decade.  

Sadly, in a decade, most, if not all of the people living with ALS will no longer be with us. I am by no means diminishing the important work being undertaken here but do want to stress the urgency that all sectors- public, private and nonprofit- must remember every day. 

The ALS clock is cruel. The Morris ALS Principles remind us that this disease will take 50% of the people currently living with ALS in 3 years & will kill 90% of the people with the disease in 5 years.

90% of the people watching, listening, and engaging in this committee’s efforts will most likely not be here to see its end results. I urge everyone to remember this urgency when developing the NASEM recommendations and actions. They must be swift. They must be efficient and they must not require more studies or evaluations. The ALS clock is cruel and requires revolutionary behavior.

The questions you have posed to the panelists today are, “What can make the nonprofit community more impactful? What is missing in the nonprofit community? What tools or capabilities should we work to integrate into the nonprofit community?”

I believe that this question is the perfect way to introduce I AM ALS and why we were created. I doubt this was meant as a lay up for me but, I am going to take it as one anyway!

I AM ALS was founded in 2019 to fill a distinct gap in the ALS environment: the clear and distinct voice of people living with ALS. I AM ALS is a patient-driven, community-based organization that collectively works to identify the shortcomings in the system from real-life experiences and bring real solutions to the table, quickly. This is what is needed to make the change necessary for so many living with ALS. I AM ALS is creating  a new playbook for how to do this work, to find a cure, and ensure every person with ALS has access to treatment. Since being founded I AM ALS advocacy has resulted in the authorization of more than $600M in new funding for ALS research.

One of our founders, Brian Wallach says that when diagnosed with ALS he had two options: Curl up into a ball or act.  He chose to act and that is precisely what so many in the ALS community are doing now that they are empowered through their work with I AM ALS. 

Improve collaboration and elevate patient voice. One of the community’s most notable successes was the passage of the ACT for ALS. The expanded access program under ACT for ALS is a revolutionary idea. People with this fatal disease are finally getting access to promising, investigational treatments, and critically, previously unfunded research is now funded. As this program moves forward, the patient community would like to see stronger collaboration between FDA, NINDS, and the ALS community. This means valuing constant patient voice and leadership. 

As an example, the FDA released a five-year action plan for ALS without any meaningful patient input. These examples should not happen and will not happen if FDA and NIH start to incorporate patients into the ACT for ALS implementation. We are better served when research and regulatory innovation are conducted in partnership with the ALS community rather than keeping it at arm’s length. 

An example of success is the NINDS Strategic Plan for ALS, which incorporated many members of the ALS community and is a clear example of how to work together to create a plan with the community.

If we work together on all aspects of ACT for ALS, it will help fund new clinical trials, discover new treatments, and drive regulatory advancements for everyone living with ALS and other rare neurodegenerative diseases today and those who will be diagnosed tomorrow.

Our recommendations include: 

1. Improved clinical knowledge, awareness, and access to quality care and trials.

As a rapidly progressing disease with frequently delayed diagnoses, coupled with existing barriers to care and social determinants of health, individuals with ALS often miss opportunities for trials and quality care options. Attention to these barriers along with the following solutions can improve and extend lives: 

• Awareness and familiarity of ALS among medical providers.
• Efforts to identify patients from underserved areas for clinical trials. Deserts of care classified as  200 miles or 90 minutes from the nearest ALS clinic mean unique barriers to getting specialized ALS care.
• Access to primary care physicians and specialists to reduce  wait times to see a doctor and quick access to resources, care, information and even a diagnosis
• Higher rates of un- or under-insured individuals – leading to people not receiving or being able to afford new drugs and technologies 
• Access issues to reliable transportation – leading to delayed or missed appointments

2. Increased focus on veterans living with ALS. 

I know my colleague and friend Juliet from Paralyzed Veterans of America is also speaking today but the issue of veterans with ALS is incredibly important to today’s discussion and to the I AM ALS community. As you look at ways that the nonprofits can engage this is an amazing case study.   

The higher incidence of ALS in veterans, as a 100% service-connected disease, received widespread awareness because the I AM ALS Community Veterans Team was launched by Veterans Living with ALS.  This awareness has led to significant efforts in Congress, DOD-funded CDMRP ALS research, and with the VA.

In closing, we have seen meaningful action taken by the medical community and policy makers over the last four years. We firmly believe this is because the ALS patient and advocate community was finally  heard, as they rallied for action that has created hope for this community. This type of collaboration and attention is the only way to scale progress so we can make ALS a chronic disease.

Thank you for allowing me the privilege to speak today for our community and thank you for all that each of you are doing for people living with ALS.