New US public-private partnership aims to accelerate ALS research

About $60M in funding committed to 5-year initiative for data platform

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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The Foundation for the National Institutes of Health (FNIH) is launching a public-private partnership to generate the largest openly available data platform in amyotrophic lateral sclerosis (ALS) research, with the goal of accelerating the diagnosis and treatment of the progressive neurodegenerative disease.

This five-year initiative will be conducted through the Accelerating Medicines Partnership (AMP) in ALS — known as AMP ALS. It brings together resources and expertise from government-funded entities, as well as from academic, industry, nonprofit, and patient advocacy sectors, which have collectively committed around $60 million to the project.

Public organizations involved in the effort include the National Institute of Neurological Disorders and Stroke (NINDS) and the U.S. Food and Drug Administration (FDA). The Critical Path Institute, a nonprofit created by the FDA in 2005 to facilitate such public-private partnerships, is also supporting the 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,” Walter Koroshetz, MD, director of NINDS, said in a foundation press release.

ALS patients and their caregivers, as well as individuals with a greater genetic risk of developing the disease, also will have a seat at the table, where they’ll offer insights to help shape the project.

“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,” Koroshetz added.

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Collaboration for research called for by ACT for ALS law

ALS is characterized by the progressive loss of nerve cells that control voluntary movement, leading to muscle weakness and problems with speech, breathing, and swallowing.

As of right now, patients wait an average of one year after their symptoms first emerge for a diagnosis to be reached. Closing that diagnostic gap — and possibly diagnosing patients even before they have symptoms — could allow for an earlier start on ALS treatments that markedly slow disease progression.

“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, 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,” Gerberding added.

The AMP ALS collaboration was called for in the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS; Public Law 117-79), which was signed into law by U.S. President Joe Biden in 2021. The law earmarks $100 million annually through fiscal year 2026 for efforts to improve ALS prevention, diagnosis, and treatment.

Now, AMP ALS intends to make it easier for researchers to access large amounts of data for ALS research by collecting and collating current and future ALS research datasets in one centralized, cloud-based knowledge platform that’s openly accessible.

Researchers in the field can then leverage these large amounts of data in their quest to find new insights related to disease biomarkers and novel therapeutic targets.

The data set that we are creating with AMP ALS will be the most comprehensive ever in ALS research. I am optimistic that I will live to see the discovery of treatments and biomarkers as the result of this major initiative.

Information contained in the platform also could be used to evaluate clinical outcome measures and design new ones that will make both clinical trials and routine patient monitoring more effective.

“The data set that we are creating with AMP ALS will be the most comprehensive ever in ALS research,” said Daniel Doctoroff, an ALS patient and founder of Target ALS, a nonprofit research foundation that’s supporting the AMP ALS initiative.

“I am optimistic that I will live to see the discovery of treatments and biomarkers as the result of this major initiative,” added Doctoroff, who will serve as an AMP ALS co-chair.

A full list of AMP ALS partners can be found on the AMP ALS webpage, where additional comments from collaborators also are available.

Another effort being conducted under the public-private partnership recommendations in the ACT for ALS law is a National Institutes of Health-funded consortium that is recruiting ALS patients and at-risk individuals for collection of clinical data and biological samples. Multiple clinical sites across the country have joined that initiative.