We have made a promise to the ALS community – we will make ALS livable for everyone, everywhere, until we can cure it. But it will take all of us—working together—to accomplish that goal.
It will take researchers and clinicians collaborating to find new treatments and cures. That means more clinical trials and patient participation in research. That includes finding ways to optimize treatments and care, increasing access to that care, and improving assistive technologies to help people with ALS live the way they want, with a better quality of life.
It also means we will need to prevent or delay the harms of ALS. We need to diagnose the disease sooner and find ways to identify ALS risk factors so we can prevent it altogether. And as new treatments are available, we need to fight for policies and access to allow people with ALS the chance to get the help they need sooner.
As 2023 comes to a close, we wanted to share some of the impact we have made this past year, thanks to you and all those who have joined us in the fight to end ALS.
Finding New Treatments and Cures
The community experienced a huge win this past April when 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 part to ALS advocates across the country who urged the FDA to approve the drug as soon as possible.
This marks the first time the FDA has approved a treatment for ALS using the accelerated approval pathway 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 the agency approved a new drug to treat ALS, showing how we are accelerating progress in the search for treatments and a cure.
We are currently supporting 149 active research projects in 13 countries, including the United States. Some of the new projects initiated this year include:
- Sixteen (16) innovative Seed Grants that have the potential to significantly impact the experience of ALS.
- Our first six Prevention Grants—worth close to $2.4 million over the next two years—which are focused on finding ways to predict when ALS symptoms will develop as well as viable strategies to intervene before symptoms emerge.
- Four promising preclinical research projects assessing emerging ALS therapies in preparation for clinical testing, supported by our Lawrence and Isabel Barnett Drug Development Program.
- Efforts at 13 established and emerging ALS clinical trial sites to increase the number and diversity of people living with the disease who have the opportunity to participate in clinical trials and to improve the efficiency and pace at which these studies are conducted.
- A phase 2 trial of a new treatment approach for C9orf72-linked familial ALS through our Clinical Trial Awards Program.
- Five promising early career scientists selected for the Milton Safenowitz Postdoctoral Fellowship Program.
Our focus on the genetics of ALS is another key to making ALS livable and also preventable. In September, a nationwide group of 40 experts published the first evidence-based consensus guidelines for ALS genetic testing and counseling. Developed with our support, these guidelines are intended to improve and standardize practices among neurologists, genetic counselors and other providers caring for people living with ALS.
We also continue to build the future of ALS research through additional funding opportunities and partnerships:
- We launched two new grant funding opportunities designed to support research focused on better management of ALS and reduction of its complications. These grants will fund the development of new assistive technologies and strengthen the quality of ALS care.
- We partnered with ALS Finding a Cure to issue an international call for proposals designed to encourage the identification of early diagnostic markers for ALS. Seven grants worth a total of $2 million over the next three years were awarded to researchers at preeminent institutions around the world.
- In partnership with The Association for Frontotemporal Degeneration, we launched a research funding opportunity supporting the collaborative development of digital assessment tools for both ALS and FTD.
- We partnered with the Focused Ultrasound Foundation to advance research into diagnosing and treating ALS using focused ultrasound technology.
- We awarded a $500,000 Partnership Grant to Target ALS to support its In Vivo Target Validation Program, enabling investigators worldwide to test and generate proof-of-concept validation data for their therapeutic targets.
To continue to follow the latest news and updates on ALS research around the world, sign up to receive our monthly online newsletter, Research Matters.
Optimizing Current Treatments and Care
To help make ALS livable for everyone, everywhere, we are working to ensure people living with ALS and their families have access to the care they need regardless of where they live. Our local care teams and teams of healthcare professionals across the country work tirelessly each day with one thing in mind—enhancing quality of life for people with ALS and supporting families impacted by the disease.
Recognizing access to both care and support services should not be dependent on your zip code, we are continuing to strengthen our program offerings nationwide.
- We know multidisciplinary care for people living with ALS and their families is proven to extend survival and improve quality of life. Our work to expand our clinic network is very intentional, focusing on increasing access and utilization of this type of care. To-date, our nationwide network has grown to include 84 Certified Treatment Centers of Excellence™, 17 Recognized Treatment Centers, and 125 affiliated clinics, with many others in the process of joining.
- We launched Nationwide Connect, monthly online support groups for both male and female ALS caregivers designed with one thing in mind—enhancing quality of life and supporting families impacted by ALS regardless of where they live. They follow support group best practices and join people together who are dealing with similar experiences.
- Our Virtual Home Modification & Safety Assessment Program offers virtual home assessments, provided by a home modification specialist that helps people with ALS identify home accessibility, mobility, and safety challenges. Potential options and recommendations allow families to make informed decisions about the best modifications for accessibility and safety in their environment.
- Reducing the financial burdens of ALS is yet another part of our commitment to making ALS a livable disease until we can cure it. Our ALS Insurance and Benefits Resource Line is a free resource for people impacted by ALS and their families to help deal with complex financial or insurance issues. In partnership with Patient Advocacy Foundation, we have served over 325 families since February.
Last spring we launched My ALS Journey™, a web-based platform connecting people with ALS to personalized resources and assistance. This free interactive tool provides a tailored 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 their unique profile, individuals can learn about the appropriate—and time sensitive—interventions available to truly live stronger and better with ALS. To date, more than 4,500 people from all 50 states and 76 countries have used this new tool.
We also understand the importance of education and resources to help people provide proper care for people living with the disease.
We added three new free professional education courses to our online library:
- Understanding Amyotrophic Lateral Sclerosis (ALS) provides a general overview of ALS, highlights some of the challenges in getting ALS diagnosed, understanding of ALS treatment and an overview of the roles of the ALS Association.
- Respiratory Strategies for Improved Quality of Life for ALS Patients was developed to support Allied Health Professionals working with people living with ALS, identifying ways each member of the multidisciplinary team can take an active role in optimizing respiratory health for ALS patients.
- Improve ALS Patient Outcomes Through Engaging Their Caregivers is intended for health care professionals, within all settings, who work with ALS patients and their informal caregivers. It highlights caregiver needs and helps professionals of varied disciplines incorporate caregiver wellness into their treatment of ALS patients, benefiting both the patient and their caregiver.
Our Caregiver Education Course launched last year for paid caregivers, family caregivers and others has now reached audiences in India, Chili, Kenya, Spain, South Africa, Canada, and Lebanon in addition to the U.S., and we have had more than 750 new enrollments so far this year.
This year we also developed new at-a-glance educational resources for people with ALS and their caregivers called “10 Things to Know,” a series of topic-specific handouts in an easy-to-read format, available in English and Spanish.
To continue to follow the latest information about ALS care, sign up to receive our quarterly online newsletter, Care Matters.
Fighting for Better Public Policies
Throughout this year, with the help of ALS advocates across the nation, we have spent countless hours informing and lobbying members of Congress and state officials in all 50 states, urging them to support the critical needs of people living with ALS and their families and caregivers.
Early this year we established a state policy and advocacy department committed to enhancing our advocacy efforts on the state and local level, empowering people living with ALS and their families to fight for better public policies in their own community. In 2023, we took positions on 403 bills across 49 states and the District of Columbia. Of those, 113 have been enacted, and seven have been sent to the Governor for signature.
We achieved unprecedented success in our fight for state appropriations, securing funding for ALS care services, clinics, and research. Thanks to the tireless efforts of our advocates, we secured more than $20 Million from 15 states including Alabama, California, Florida, Indiana, Maryland, Minnesota, Missouri, New Jersey, New Mexico, New York, Ohio, Pennsylvania, South Carolina, Tennessee, and Virginia.
Taking advantage of the opportunity to meet directly with legislators in their home districts, we launched ALS Advocacy Action month, a multi-faceted campaign for advocates to engage with Congress. The campaign provided “8 Ways to Take Action in August” encouraging advocates to ask for increased funding of ALS research.
Our national policy and advocacy department continues its robust campaigns to influence Congress. For example, advocates urged senators and representatives to provide more funding for ALS research at NIH, the Department of Defense, the National ALS Registry and Registry and the FDA. As a result of these efforts, 80 representatives and 32 senators signed on to support increased funding for ALS research through the House and Senate “Dear Colleague” letters.
We also engaged on more than 27 federal bills covering a wide range of issues impacting people with ALS and their families. The list includes urging Congress to continue telehealth coverage, expand access to ALS clinics, ensure veterans with ALS and their families are treated fairly, and require airlines to take better care of people living with ALS.
In addition to legislative advocacy, we have influenced the National Academies as it develops its report on “Amyotrophic Lateral Sclerosis: Accelerating Treatments and Improving Quality of Life” and pushed Medicare to eliminate onerous prior authorization techniques that allow some Medicare Advantage plans to delay, require fail first, or deny medical care or ALS drugs.
With 2024 right around the corner, we encourage advocates to take action and support our ongoing campaigns to benefit the ALS community including:
- The ALS Better Care Act (H.R. 5663), a bill that would increase Medicare reimbursements to ALS clinics, establishing a $800 additional payment to ALS clinics per Medicare beneficiary visit—critical legislation to help ensure people with ALS have access to the care they need, regardless of where they live.
- Justice for ALS Veterans Legislation (H.R. 3790/S.1590), making sure spouses of veterans who lose their battle with ALS are not denied access to survival benefits they are owed.
- The Elizabeth Dole Home Care Act (S. 141) would raise the current cap for veterans with ALS so the VA would pay for home care costs up to 100% of what a nursing home would cost—or more.
To learn more about how you can become an advocate for the ALS community, visit our Advocacy Action Center.
Raising Awareness and Support
More awareness means more funding, and our community events play a major role in helping people understand the devastating impact ALS has on the families we serve.
Each spring and fall, people of all ages and all walks of life gather together in communities across the country to show their support for people 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 invaluable to the ALS community, raising awareness of the disease and critical funds for research and care.
Last spring 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.
It continues to air in markets across the country in both English and Spanish and, to date, we have received almost 18,000 airings resulting in over 201 million impressions.
None of these advancements would be possible without the ongoing generosity and support of our partners, donors, and the many families we serve. To learn more about our work and how you can get involved today, visit als.org.
