NORTH BETHESDA, Md.– The Foundation for the National Institutes of Health (FNIH) announces the launch of a public-private partnership to build the largest data source for amyotrophic lateral sclerosis (ALS) and expedite the identification of biomarkers and clinical outcome assessments that would allow earlier diagnosis and accelerated drug development for people living with the disease. ALS is a progressive and ultimately fatal neurological disease with few treatment options and no cure yet.
Managed by the FNIH, the Accelerating Medicines Partnership® in Amyotrophic Lateral Sclerosis (AMP® ALS) brings together the resources and expertise of the National Institute of Neurological Disorders and Stroke (NINDS) at the National Institutes of Health (NIH), the Food and Drug Administration (FDA), Critical Path Institute (C-Path), and other stakeholders from academia, life science companies, foundations, and patient-focused groups to tackle the tremendous challenges of diagnosing, understanding, and treating the disease. AMP ALS is supported by a combined commitment of approximately $60 million.
“ALS gradually robs people of their ability to walk, talk, eat—and eventually breathe. Patients can’t wait, given the typical survival time of two to five years after diagnosis,” said Julie Gerberding, MD, MPH, President and CEO of the FNIH. “AMP ALS will help define sorely needed diagnostic and treatment targets and bring hope to patients and their families who are faced with this challenging disease.” No available treatment halts or reverses the progression of ALS.
People with lived experience of ALS, including individuals and families affected by the disease, caregivers, and those at high genetic risk for developing ALS in the future, are providing their perspectives throughout this five-year project. Their insights into the most urgent needs of people with ALS, or at risk for developing ALS, are helping to shape the project’s research focus accordingly, and they will continue to be involved in this collaborative initiative.
“Working collaboratively, through AMP ALS, the partners can advance efforts to understand what triggers ALS and discover new targets for effective treatments, as well as identify biomarkers that can be used to predict whether promising interventions have the intended effects in people,” said Walter Koroshetz, MD, Director of NINDS. “Most of all, we are grateful for the significant contributions of people with lived experience who provided so much time and effort into the development of this program, and we greatly appreciate their continued participation.”
The AMP ALS research team will collect and centralize both current and future ALS datasets in a cloud-based, openly accessible knowledge portal. With access to these collective resources and by capitalizing on existing biosamples as well as clinical data, including that from digital health technology, researchers will focus on identifying and validating biomarkers and therapeutic targets specific to ALS. They will also address the need for new or modified clinical outcome assessments, including patient-reported outcomes.
Currently, the average time to diagnosis is 12 months. Biomarkers are crucial to earlier diagnosis of disease, potentially before symptoms appear, allowing for possible interventions and improved quality of life. In addition, validated biomarkers will help monitor disease progression and patients’ response to treatment, improve clinical trial design, and potentially lead to more effective, personalized drug treatments.
ALS, also known as Lou Gehrig’s disease, causes degeneration of the nerve cells in the brain and spinal cord that control voluntary muscle movement. As the nerve cells die, the brain can no longer send signals to muscles, progressively taking away an individual’s ability to move. Cognitively, most individuals with ALS usually remain able to reason, remember, and understand their loss of function, often leading to anxiety, depression, and tremendous stress on the entire family. It is estimated that ALS affects about 30,000 people in the United States, with 4,000 to 6,000 new cases diagnosed each year.
“I got involved with AMP ALS as co-chair because I believe in the importance of data and collaboration. The data set that we are creating with AMP ALS will be the most comprehensive ever in ALS research. And the FNIH is bringing industry together with NIH, FDA, and nonprofit organizations to make it happen,” said Daniel Doctoroff, who is living with ALS and whose father and uncle passed away from the disease. “I am optimistic that I will live to see the discovery of treatments and biomarkers as the result of this major initiative.” Doctoroff is the founder of the nonprofit research foundation Target ALS.
The initiative is a component of the public-private partnership called for in the Accelerating Access to Critical Therapies for ALS Act, signed into law by President Biden in December 2021. Multiple clinical sites across the country have already joined the NIH-funded Access for All in ALS (ALL ALS) consortium, which will recruit people with ALS and those at risk for the disease to collect important clinical data and biosamples.
