Accelerating Progress Together: Target ALS’s Role in AMP® ALS
March 30, 2026
When progress against a disease as complex as amyotrophic lateral sclerosis (ALS) feels slow, the instinct can be to work harder in isolation. But some of the most meaningful advances happen when institutions choose collaboration over ownership, and shared infrastructure over individual credit.
That philosophy is at the heart of the Accelerating Medicines Partnership® ALS (AMP® ALS), a large-scale public-private effort designed to accelerate the development of biomarkers and effective treatments for ALS. Target ALS has been involved since the earliest conversations shaping the program and continues to play a central leadership and execution role as AMP ALS moves from design to delivery.
From early conversations to the launch of AMP ALS
AMP ALS emerged following years of advocacy that culminated in the passing of the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS), which called for the establishment of a public-private partnership for ALS, among other provisions. To turn this into action, the National Institutes of Health (NIH) engaged the Foundation for the National Institutes of Health (FNIH) to help build what would become AMP ALS, a partnership spanning government, industry, academia, nonprofits, and people with lived experience of ALS. NIH’s initial investment in AMP ALS was around $40 million to support a new, US-wide natural history study, the Access for ALL in ALS Clinical Research Consortium (ALL ALS), designed to operationalize the research goals of AMP ALS.
From the outset, Target ALS founder Daniel L. Doctoroff and CEO Manish Raisinghani worked together with Walter Koroshetz, former Director of the National Institute of Neurological Disorders and Stroke (NINDS), and Alessio Travaglia, Director, Translational Science, Neuroscience and Rare Diseases, FNIH, to help shape how those resources could be used most effectively. Early conversations focused on accelerating areas of research that would address high unmet needs for people with ALS and laying a foundation to enable real change over the life of the program.
In the design phase of the program, it was important to ensure that the generosity of people living with ALS translated into meaningful scientific progress. Establishing the right infrastructure to facilitate research was critical. “Collecting samples alone doesn’t do justice to the people who donate them,” noted Raisinghani. “Maximizing the impact of those samples by providing access to them for use through a transparent and equitable process as well as the data generated from them, is essential. Without that, you don’t have a strong study.”
Leadership grounded in action
From the outset, Doctoroff was a unanimous choice to serve as Co-Chair of the AMP ALS Steering Committee, alongside leaders from across the partnership. He has a long track record of turning vision into action, most notably through his role in reshaping New York City’s urban landscape as Deputy Mayor of Economic Development and Rebuilding. That same drive powered the rapid expansion of Target ALS’s scientific programs in 2022, shortly after his ALS diagnosis. Even as he has gradually lost speech and mobility, Doctoroff’s energy and resolve continue to propel AMP ALS forward as a bold, collaborative effort aimed at delivering transformational change.
Target ALS’s Vice President of Scientific Programs, Amy Easton, has been deeply involved across multiple working groups, from the earliest planning stages through execution. Taking over the direction of scientific programs for Target ALS in 2022, Amy brought 20 years of drug discovery experience in neurodegenerative disease. She shares her time and expertise on AMP ALS, working closely with Amelie Gubitz and Amy Tsou (both Program Directors at NINDS), David Shulman (person living with ALS), and Shubhangi Lal (Senior Project Manager, Neuroscience, FNIH) to co-lead the ALL ALS natural history study Working Group, which provides critical guidance for ALL ALS.
AMP ALS technical working groups span four critical areas: data infrastructure, biomarkers, clinical outcome assessments, and the ALL ALS natural history study. Funds for these initiatives were released starting in 2023, and the program is now actively delivering on its commitments. The first year of ALL ALS was enormously successful with more than 50% of the 2000 participants enrolled. The recent merging of the Natural History Study and the Biomarkers Working Group, led by Frank Shewmaker (Program Director, NINDS) and Aparna Vasanthakumar (Senior Principal Research Scientist, AbbVie), follows the first release of biomarker data and signifies an inflection point for the program, fueled by the entire team.
Why data generation matters
Open sharing of high-quality data removes unnecessary siloes between life science companies and academia and dramatically accelerates research, more so today than ever before. Target ALS has invested in state-of-the-art techniques to generate comprehensive multi-omic datasets from every patient and volunteer for the past 10 years. Target ALS’s role in AMP ALS is no exception. The organization has committed up to $5 million in in-kind support to enable multi-omic data generation. This pledge ensures that samples from the ALL ALS study are impactful and not left idle due to funding gaps.
Data from ALL ALS, Target ALS, and many other highly regarded studies will be housed in the newly built ALS Knowledge Portal, a cloud-based platform designed to centralize and enable analysis across datasets from many independent clinical and biologic studies. Importantly, this effort enables access to instructive ALS datasets not previously accessible to the broader research community.
In addition, through a collaborative initiative with other funders of big neurodegenerative datasets, Target ALS is working to break down technical and operational barriers to allow for improved data interoperability between its Data Engine and other excellent data resources that house big, curated ALS datasets complementary to those in the Knowledge Portal.
Anchored to clear, ALS community-informed goals
Guided by input from people with lived experience of ALS and the ALS advocacy community, AMP ALS was established with a clear set of goals: to accelerate the development of effective treatments by improving how ALS data is generated, shared, and used.
Those goals include:
- Establishing a central ALS knowledge platform for cloud-based data sharing, enabling AI/ML analyses
- Developing validated biomarkers for earlier diagnosis and treatment assessment
- Enhancing the sensitivity, responsiveness, and validity of clinical outcome measures
- Discovering promising therapeutic targets and risk factors
Target ALS’s contributions align directly with each of these aims. From shaping the design of the ALS Knowledge Portal, to funding large-scale genomic and proteomic analyses, to enabling rapid access to curated human datasets, the organization has focused on filling critical gaps between sample collection and scientific insight.
Only the beginning
While the launch of data portals and the generation of large-scale omic datasets mark important milestones, both Easton and Raisinghani emphasize that these are just the first steps. The true measure of success will be how widely the data is used and how effectively it informs biomarker development and accelerates the discovery of new therapeutic targets by project end in 2029.
For people living with ALS who donate biosamples, that outcome is the ultimate goal. AMP ALS, supported by Target ALS and its partners, is designed to honor that contribution by ensuring data is accessible, usable, and leveraged to its fullest potential. For Target ALS, AMP ALS is a unique forum of stakeholders where participation has been an opportunity for sustained engagement, open dialogue, and sharing of resources, all to move the needle for the ALS community at large.
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