The National Academies of Sciences, Engineering, and Medicine (NASEM) has released a report drafted by a committee of distinguished medical scientists, including Eva Feldman, M.D., Ph.D., that identified“vital actions” that would end the death sentence for those with ALS within a decade.”
Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurologic disease in which motor neurons in the brain and spinal cord degenerate, leading to loss of muscle function and, eventually, death. With no effective treatments currently available, the prognosis is for patients to live only two to four years after diagnosis.
In response to the devastating nature of this disease, Congress enacted a directive (Consolidated Appropriations Act of 2022) for the National Institutes of Health to commission a study by the National Academies of Sciences, Engineering and Medicine (NASEM) (NASEM) “to identify and recommend actions for the public, private, and non-profit sectors to undertake that would transform ALS from a terminal to a chronic disease.” The NASEM established a committee of 18 volunteer experts from various medical fields, including Dr. Feldman, to accomplish just that.
“This report, nearly two years in the making, presents a roadmap to improve the lives of persons with ALS and their families,” explained Dr. Feldman. “Critical aspects of the report include restructuring of clinical centers, access to early interventions, durable equipment, and clinical trials emphasizing equal care for all persons with ALS.
“If this report’s recommendations are put into practice, ALS will definitely become a more livable disease.”
The committee defined ALS as being “livable when an individual diagnosed with the disease can survive, thrive, and live a long, meaningful life while meeting the medical, psychosocial, and economic challenges of the disease.”
Short-term recommendations with immediate impact:
The report recommends concrete, immediate actions that would have a significant impact on livability for people with ALS and their caregivers. Here are two examples:
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Centers for Medicare and Medicaid Services (CMS) and private insurers should act quickly to provide coverage for and ensure access to home-based and outpatient and support services for persons with ALS as necessary and to do so in an expedited fashion.
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Congress should provide a tax credit to unpaid caregivers of individuals living with ALS. CMS should test approaches for paying stipends directly to caregivers on at least a monthly basis and providing access to high-quality respite services.
Building the ideal ALS care delivery system:
The report also made long-term recommendations to make ALS a livable disease, noting that to do so would require diagnosing individuals earlier and initiating evidence-based, multidisciplinary care by ALS specialists immediately and continuously. It urges the development of an integrated, multidisciplinary system that would provide high-quality care and access to research opportunities for all people with ALS. Here are some details:
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The system — modeled after “hub and spoke” systems of care and research for cancer and stroke — would build on what already exists in the ALS clinic system and comprise three care settings: community-based ALS centers, regional ALS centers, and comprehensive ALS care and research centers.
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CMS and the National Institute of Neurological Disorders and Stroke should develop the system in partnership with current ALS multidisciplinary care leaders.
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Reimbursement practices should be realigned to support this multidisciplinary care model.
Advancing ALS research and accelerating the development of therapies:
The report noted that research advances have identified a wide range of potential therapeutic pathways, drug targets, and genes associated with ALS. However, a lack of biomarkers, an overall limited understanding of the disease, and the small pool of available research participants are factors contributing to the lack of significant success in ALS drug development.
The report recommends the following:
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Creation of a centralized, dedicated ALS clinical trials network that builds on existing ALS clinical trial consortia. This network would provide a coherent approach to clinical trials and natural history studies, simultaneously allowing faster answers to multiple questions. The National Institute of Neurological Disorders and Stroke should ensure such a network is distributed across diverse geographic regions in the U.S. and coordinated and funded by NIH.
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The expansion of translational research and identifying additional research priorities.
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More funding for neglected areas of research that would yield near-term gains in quality of life for people with ALS, such as research on physical therapy and speech and language support
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The creation of a comprehensive ALS registry capable of collecting detailed longitudinal data on all individuals living with ALS, as well as people at increased genetic risk of developing ALS.
The report also noted that while the Department of Veterans Affairs has demonstrated an exemplary system of ALS care, veterans receiving ALS care within VA clinics have limited access to participation in clinical trials. Given the known higher prevalence of ALS in veterans, it is critical to include the VA in the new integrated ALS network of care and research proposed in the report.
Preventing ALS:
The report suggests that additional research is also needed on populations at risk of developing ALS. It notes the following:
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Research that identifies new targets for therapeutic intervention, biomarkers of ALS, and risk factors and environmental exposures contributing to the development of ALS raises the possibility of developing agents or interventions that can delay or even prevent the development or progression of ALS.
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Research funders should partner with drug developers and the ALS community to advance research focused on people at risk of developing ALS, including at-risk genetic carriers.
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CMS and private insurers should increase access to genetic testing and counseling for people with ALS and their families.
“Making ALS a livable disease within a decade will require commitment, resources, and leadership from many relevant parties, but the impact will be great for the more than 30,000 individuals living with ALS and the thousands more who are at risk of developing this terrible disease,” said Victor J. Dzau, president of the National Academy of Medicine.
“Carrying out the report’s recommendations would provide greater and more equitable access to state-of-the-art multidisciplinary care, accelerate the development of more effective treatments, improve the quality of life and health of those individuals suffering from ALS both now and in the future, and provide essential support for families and caregivers.”
The medical experts producing the study is officially known as the Committee on Amyotrophic Lateral Sclerosis: Accelerating Treatments and Improving Quality of Life.