2023 Mid-Year Report

People living with ALS

The ALS Association is committed to making ALS a livable disease for everyone, everywhere, until we find a cure. That means continuing to support research in the search for new treatments, while advocating with our legislators on behalf of the community and improving access to the critical care people with ALS and their families need regardless of where they live.

Progress is being made. Never before in the history of this disease have we seen so many talented scientists and researchers, ALS advocates, and health care professionals, working together, providing even more hope for the families we serve.

And we will not stop until we create a world without ALS. Read more:

 

research

Finding New Treatments

In March, the U.S. Food and Drug Administration approved tofersen (Qalsody) for the treatment of people living with ALS connected to mutations in the SOD1 gene, thanks in no small part to ALS advocates across the country who urged the FDA to approve the drug.

This marks the first time the FDA has approved a treatment for ALS under accelerated approval and serves as a promising endorsement of the antisense technology that underlies tofersen. It was the second time in less than a year and the third time in six years that the agency approved a new drug to treat ALS, showing that we are making incredible progress in the search for treatments and a cure.

In addition, QurAlis began testing QRL-201 in a phase 1 study – the first clinical trial to evaluate a therapy that rescues STATHMIN-2 expression in ALS patients. The primary objective of the study, which received support through our Lawrence and Isabel Barnett Drug Development program, is to determine the safety and tolerability of multiple doses of QRL-201 in people living with ALS.

We are currently supporting 165 active research projects in 14 countries, including the United States.

We are also building the future of ALS research through:

 

ALS care

Optimizing Current Treatments & Care for Everyone Everywhere

Ensuring people living with ALS and their families have access to the critical care they need regardless of where they live continues to be a priority for us. Our local care teams and teams of healthcare professionals across the country work each day with one thing in mind—enhancing quality of life for people with ALS and supporting families impacted by the disease.

So far this year we have welcomed three new Certified Treatment Centers of Excellence™, and several current affiliated clinics are moving toward formal designation.

These three clinics bring a variety of expertise, innovation and research opportunities to the ALS community while increasing access to multidisciplinary care.  

Last month we launched a new web-based platform, My ALS JourneyTM, which connects people with ALS to personalized, valuable resources and assistance. Developed in consultation with the ALS community, My ALS Journey is designed to put individuals living with the disease in the driver’s seat of their own health care journey. This new interactive tool provides a personalized experience, relevant information, articles, and actionable checklists that help people living with ALS plan as they experience the stages and progression of the disease. With a personalized profile, individuals can learn about the appropriate – and time sensitive - interventions available to truly live stronger and better with ALS.

We recognize language can be a huge barrier to understanding and receiving critical care for people living with ALS. Our Remote Language Interpretation Program is available to the families we serve at no cost across our care services network and continues to benefit ALS families across the country. Since January 1, 47 conversations have been interpretated in four different languages.

Our Living with ALS Resource Guides were created because of the rapidly expanding information and research in the clinical management of ALS. These cornerstone educational materials were designed to inform and educate people about ALS in a comprehensive and easily understood format. Available on our website free of charge, these guides have recently been updated and are provided in both English and Spanish.

Our global research program has invested in ways to enhance the quality of care available and improve the quality of life for people living with ALS through:

  • Assistive Technology Grants, which is a new program that will support efforts to develop or adapt technologies that can help maintain or improve the health, independence, and/or quality of life of people living with ALS.
  • New Quality of Care Research Grants, which support studies focused on improving the quality of ALS care or increasing the implementation of evidence-based care. 
  • Early Diagnostic Grants in partnership with ALS Finding a Cure®, which support the identification and development of new methods that can be used to reduce the time it takes to diagnose ALS.
  • A new partnership with the Association for Frontotemporal Degeneration to help advance digital tool development focused on developing tools that use smartphones, wearable trackers or other devices to remotely gather data on a person’s symptoms or functional ability.

 

ALS Advocates

Fighting for Better Public Policies

ALS advocates have spent countless hours informing their senators and representatives about the critical needs of people living with ALS and their families and caregivers. The relationships they have built helped secure the signatures of 80 representatives and 32 senators on 2023 “Dear Colleague” letters requesting increased funding for ALS in support of our effort to find new treatments and cures, optimize current treatments and care, and prevent or delay the harms of ALS.

We led efforts to secure Medicare coverage for power seat elevation systems as a medically necessary safety feature. In addition to our own comments supporting the policy change, we also submitted comments on behalf of 540 advocates from 47 states – people living with ALS and caregivers – explaining why seat elevation systems are important in their daily life.

We established a nationwide state policy and advocacy department committed to enhancing our advocacy efforts on the state and local level and empowering people living with ALS and their families to fight for better public policies in their community. In 2023, we have taken a position on 349 state bills, of which 87 have been enacted, and 8 have been sent to the Governor for signature.

ALS volunteers and leading advocates held more than 200 meetings with members of Congress and their staff during our virtual fly-in event on April 19, focused largely on securing increased federal funding for ALS research. Advocates from across the nation made over 250 calls to their members of Congress to support our appropriations requests.

Maryland lawmakers are considering a bill we spearheaded that would prohibit life insurance, long-term care insurance, and disability insurance policies from discriminating against people based on the results of a genetic test. ALS Association leaders and advocates testified on the importance of genetic testing for people with ALS in support of the bill before both the Maryland House of Delegates and the Maryland State Senate in what we hope will be a model to move similar legislation in other states.

We also recently submitted a series of regulatory filings to reduce administrative burdens placed on people living with ALS. The filings are in support of administrative rule changes at the Centers for Medicare and Medicaid Services that would reduce some administrative delays by improving prior authorization claims processes, instituting how and when Medicare Advantage plans develop and use coverage criteria and utilization management policies, and ensure Medicare Advantage coverage policies are equivalent to traditional Medicare coverage.

 

Walk to Defeat ALS

Raising Awareness and Support

Each year, people of all ages and all walks of life gather together in communities across the country to show their support for people living with ALS and their families, while celebrating and honoring the lives of those who have passed from the disease. These Walk to Defeat ALS® events are so important to the ALS community, raising critical funds for research and ALS care while raising awareness of the disease and showing the impact it has on the families it touches.

This year we developed a new PSA highlighting these celebrations and encouraging others to join the fight against ALS by raising their voices and raising hope that one day, we will see a world without this disease.

Receiving more than 5,200+ airings in the first six weeks, the PSA continues to air in markets across the country in both English and Spanish.

None of these advancements would be possible without the generous support of our donors, partners and the families we serve. To learn more about the work of The ALS Association and how you can get involved today, visit als.org.

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