WASHINGTON — A new congressionally mandated report from the National Academies of Sciences, Engineering, and Medicine recommends actions Congress, federal agencies, insurers, and others should take to strengthen health care and support services for people with amyotrophic lateral sclerosis (ALS) and accelerate research on therapies for the disease — with the goal of turning ALS into a livable disease within a decade. 

ALS — also known as Lou Gehrig’s disease — is a rapidly progressive, fatal neurological disease whose hallmark is the degeneration of motor neurons, which causes a gradual loss of motor functions. People with ALS often find tasks such as walking, eating, and communicating increasingly difficult as their disease progresses, and death usually results from respiratory failure. Currently there are no treatments that stop or reverse disease progression, though services exist to help manage symptoms. At any given time, at least 30,000 individuals in the United States are estimated to have ALS.

“Receiving a diagnosis of ALS is devastating for the individual, their family, and their caregivers,” said Alan Leshner, chief executive officer emeritus, American Association for the Advancement of Science, and chair of the committee that wrote the report. “Dealing with this illness requires a complex array of medical and support service interventions, and the intensity of care required increases exponentially over time. Implementing the vision laid out in our report would go a long way toward achieving the goal set in the committee’s charge of making ALS a livable disease within a decade.”

The committee — which included scientific and clinical experts from a variety of fields, as well as people living with ALS or at clear risk for developing it — 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 actions that are feasible immediately

The report recommends concrete immediate actions that would have an important impact on livability for people with ALS and their caregivers. For example, the 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 physical and other support services for persons with ALS as necessary, and to commit to expedited (within 72 hours) responses to prior authorization requests for all therapies, durable medical equipment, assistive technologies, and services for persons with ALS.

In addition, Congress should provide a tax credit that could be used by unpaid caregivers of individuals living with ALS, the report says. 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

Making ALS a livable disease requires diagnosing individuals earlier and initiating evidence-based multidisciplinary care by ALS specialists immediately and continuously, the report says. 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, filling gaps in access across the U.S.

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 would comprise three care settings: community-based ALS centers; regional ALS centers; and comprehensive ALS care and research centers. The system should be developed by CMS and the National Institute of Neurological Disorders and Stroke, in partnership with leaders of current ALS multidisciplinary care clinic systems. Reimbursement practices should be realigned to support this multidisciplinary care model.

Advancing ALS research and accelerating development of therapies

The history of drug development for ALS is filled with many failures and too few successes. Over the past decade, research advances have identified a wide range of potential therapeutic pathways and potential drug targets and genes associated with ALS, the report says. However, a lack of biomarkers, overall limited understanding of the disease, and the small pool of available research participants — which is exacerbated by restrictive clinical trial eligibility criteria and delays from symptom onset to diagnosis and clinical trial entry — are factors contributing to the lack of significant success in ALS drug development.

The report recommends the 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 that permits faster answers to multiple questions at once. The National Institute of Neurological Disorders and Stroke should ensure the existence of such a network, distributed across diverse geographic regions in the U.S., and coordinated and funded by NIH.

The report also recommends the expansion of translational research and identifies additional research priorities; urges 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 supports; and proposes 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 notes that while the Department of Veterans Affairs has demonstrated an exemplary system of ALS care that is interdisciplinary, proactive, and patient-centric, 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 ALS system of care in the new integrated ALS network of care and research proposed in the report.

Preventing ALS

Additional research is also needed on populations at risk of developing ALS, the report says. 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. 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. In addition, 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 study — undertaken by the Committee on Amyotrophic Lateral Sclerosis: Accelerating Treatments and Improving Quality of Life — was sponsored by the National Institute of Neurological Diseases and Stroke of the National Institutes of Health. The National Academies of Sciences, Engineering, and Medicine are private, nonprofit institutions that provide independent, objective analysis and advice to the nation to solve complex problems and inform public policy decisions related to science, engineering, and medicine. They operate under an 1863 congressional charter to the National Academy of Sciences, signed by President Lincoln.

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