Fighting to Get Effective Treatments to People with ALS
This year we continued to lead the global search for treatments and a cure as the largest philanthropic funder of ALS research around the world. Since January 1, 2020, The ALS Association has funded 46 research projects around the world. Here are a few examples of advancements in our research program.
Research funded by the Association led to the development of a promising new drug from Amylyx Pharmaceuticals. Clinical testing of AMX0035 showed it to be safe and effective at slowing the progression of ALS and helping people live longer. This research was one of the first projects to be funded with monies raised through the ALS Ice Bucket Challenge.
Due to the safety and effectiveness of AMX0035, The ALS Association, in collaboration with I AM ALS, collected more than 50,000 signatures on a petition we delivered to the Food and Drug Administration calling on the FDA and Amylyx to expeditiously work together to make sure AMX0035 is made available for people with ALS as quickly as possible.
Our $3 million commitment to help set up the first-ever ALS platform trial at the Sean M. Healey & AMG Center for ALS is accelerating the rate at which we can test and evaluate treatments. Even in the face of the pandemic, the Healey platform trial is already enrolling clinical trial participants. The project is on track to have 50 sites up and running by the end of December, with 100 people fully enrolled in the study by July.
Our partner Biogen published promising results from its phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS and began enrolling participants for their Phase 3 Valor study. Biogen also announced that there is an open-label extension available in the study, more promising news and hope for people living with ALS.
Fighting for Change for People Living with ALS
The ALS community earned a hard-fought victory this year with passage of the ALS Disability Insurance Access Act, ending the five-month waiting period for people with ALS to start receiving their Social Security Disability Insurance benefits. The ALS Association has been tirelessly engaged in building support for this policy in Congress since it was first introduced in 2016, working with congressional champions and ALS advocates over the years laying the groundwork for this incredible win.
Congress also authorized $40 million for research at the Department of Defense ALS Research Program – doubling the level of funding from the previous year. This is the second year in a row we were able to double funding for this critical program.
We also worked with lawmakers to strengthen a bill that would be an important step in bringing promising new treatments to people with ALS as quickly as possible. The ACT for ALS Act accelerates the fight against ALS by authorizing $100 million for ALS research and creating the first federal entity explicitly charged with developing treatments for neurodegenerative diseases.
While COVID-19 has impacted every part of our daily lives, it was felt most acutely in our access to health care. The ALS Association joined the American Academy of Neurology and other patient advocacy organizations to speak up for telehealth. Historic changes in telehealth were made in response to the pandemic, marking a big step forward in the use of digital technology to deliver health care. We will continue to press Congress to pass legislation to ensure expanded use of this vital health care tool is permanent.
Fighting to Make Life Better for People Living with ALS Today
In August we released the findings from the first ever ALS Focus survey , illustrating the extent of the financial burden people with ALS and their caregivers face during their ALS journey and the lack of understanding of insurance coverage paying for medical treatments and services. We have also launched a second survey centered around “what matters most” to people with ALS and caregivers and hope to share those findings in early 2021. The ALS Association is committed to taking these results and working with our partners in the pharmaceutical and insurance industries to turn them into action items, informing crucial changes in our system.
In 2020, we also awarded $470,000 in scholarships to 94 students for the 2020-2021 school year through The Jane Calmes ALS Scholarship Fund ensuring that kids impacted by ALS are able to continue their education. Established in 2019, the fund was created to support post-high school education for students whose lives have been financially impacted by ALS. Scholarship recipients receive $5,000 per year to help cover the cost of education.
While much attention is paid to the person living with ALS and their adult family members and caregivers, children are often voiceless, despite experiencing much of the same shock, sadness, caregiving, and grief as adults. With the help of Melinda S. Kavanaugh, PhD, LCS, The ALS Association released a new guide in our library of youth education materials, “At School: A Guide to Supporting Students Who Have Been Affected by ALS.” The purpose of this new publication is to help address the needs of school staff and personnel working with children impacted by ALS.
Communicating the most up-to-date information about ALS is critical for people living with the disease, their families, and their caregivers. This year the Association joined forces with the Minnesota/North Dakota/South Dakota Chapter to produce Connecting ALS, a weekly podcast aiming to discuss the latest research and technology developments, highlight advocacy efforts, and share the personal stories woven through the community. We also launched our new website, als.org , a mobile friendly environment to more effectively engage with our community, empowering them to make the critical decisions necessary in their fight against ALS. We plan to have our nationwide network of chapters migrated onto this new platform in 2021.
Vaccine approvals and distribution initiatives are generating streams of news and information that are relevant to our ALS community; however, given the volume and frequency of data and resources, it may be hard for some families in the ALS community to stay current. The ALS Association has created a COVID-19 Vaccine Information hub to capture breaking news and resources our community may find helpful to inform their conversations with physicians about the vaccines, their safety and efficacy, and availability. We’ve compiled information from critical sources such as CDC and FDA , which will be updated continuously through the COVID-19 crisis. We will also be sharing informed opinions and analysis about the vaccines and nationwide distribution plans from leading physicians and opinion leaders in the ALS space.
Fighting Every Day in Our Communities for People with ALS
In response to Covid-19, our nationwide network of chapters swiftly met the challenge of delivering care and resources to people with ALS despite the limitations imposed by social distancing recommendations. Across the country, chapter staff pivoted quickly to maintain local support and care, modifying programs and services to virtual platforms free of exposure to the coronavirus wherever possible.
Virtual check-ins and home visits by ALS Association care services staff, and online support groups for people living with ALS and caregivers, ensured that those living with ALS received access to care and remained connected with their community. Chapters also maintained delivery of critical programs – durable medical loan equipment, respite care, and caregiver support – and creatively found ways to expand their programs safely.
Some examples include:
- The St. Louis Regional Chapter and other chapters hosted online educational events focused on the latest developments in ALS care, research and advocacy.
- The ALS Association Iowa Chapter partnered with the University of Iowa Master of Counseling Program to develop a telemental health program intern partnership. The program provides free counseling via the Zoom platform or by phone.
- Ionis Pharmaceuticals established a $25,0000 scholarship to help people with ALS in and around San Diego access mental and physical wellness programs.
- The ALS Association Northern New England Chapter moved its day-long ALS Family Caregiver Lab, which teaches caregivers how to use specialized equipment, to a virtual learning lab.
- The ALS Association Rhode Island Chapter is supporting cutting edge research developing technologies to help people living with ALS communicate using brain signals.
The Walk to Defeat ALS® remained virtual throughout the year as the spread of the coronavirus and critical need for social distancing became the year’s “new normal”. Walk leaders around the country transformed their annual community celebrations into fun, friendly experiences online using social media, encouraging participants to create their own event by walking or riding in their own neighborhoods to protect everyone’s health and safety. All in all, the ALS community celebrated 170 “Walk Your Way” type virtual events nationwide in 2020 raising more than $17 million for care, support, and research.
As the year comes to a close, we recognize that none of this would be possible without the generosity and support of our donors and partners and want to thank everyone who continues to join us in the fight. And we’re hopeful 2021 will be a new year filled with new discoveries in ALS research, new treatments made available to people living with the disease, and new technologies to enhance the lives of those in need.
To learn more about the work of The ALS Association or find out how you can get involved, visit www.als.org.