AMP Amyotrophic Lateral Sclerosis (ALS) Design Phase

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AMP Amyotrophic Lateral Sclerosis (ALS) Design Phase

Overview

The AMP Amyotrophic Lateral Sclerosis (ALS) Program concept proposes to accelerate therapeutic and biomarker development for familial and sporadic ALS by addressing the challenges of disease heterogeneity, segmentation of research efforts, decentralized data storage, and a lack of standardized clinical tools.

ALS is a relentless disorder characterized by the degeneration of both upper and lower motor neurons, leading to progressive weakness and paralysis. In approximately 30% of cases, non-motor neurons in the frontal and temporal regions of the brain are also affected, causing impairments in various cognitive domains. The disease is uniformly fatal, with an average survival of two to five years after diagnosis, although some individuals succumb even faster, and others survive for more than a decade.

To date, treatment options for ALS remain severely limited, have modest benefits, and no known treatment halts or reverses the progression of ALS. Hence, there is an urgent unmet need to develop more effective treatments for ALS

The AMP ALS design phase started March, 2023

Goals

Guided by input from people with lived experience of ALS and the ALS advocacy community, AMP ALS has a common goal of establishing a comprehensive strategy to expedite the development of effective new treatments for people living with ALS, or at risk for developing ALS. This will be achieved through:
1) the establishment of a central ALS Knowledge Platform for data sharing and analysis through a cloud-based infrastructure;
2) the development of validated biomarkers for early diagnosis and treatment assessment;
3) improved clinical outcome assessments; and
4) discovery of new therapeutic targets and risk factors.