• Projects may range from phase 1 to small phase 2 trials and should aim to de-risk and inform the design of more advanced trials.
• Must support a clinical trial and may not be used for preclinical research studies.
• Must include a Community Partner (e.g., patient/caregiver advisory panel) who will provide advice and consultation throughout trial planning and implementation.

Applications must address one focus area:

• Biomarker-Driven Interventions: Disease-modifying interventions of novel therapeutics or devices, incorporating mechanism-specific predictive, efficacy, and/or pharmacodynamic biomarkers.

• Clinical Care: Improving existing clinical care and/or symptom management strategies.

• Maximum funding of $2,000,000 for direct costs (plus indirect costs)


• Maximum period of performance is 3 years

• Supports the development and validation of clinical outcomes and/or biomarkers to improve clinical trials in ALS.
• Data sharing plans are an important and scored component.
• Interventional clinical trials are not allowed. However, non-interventional studies prospectively collecting biospecimens and/or data are permitted, including in parallel/add-on to existing trials.
• Projects that include prospective enrollment must include a Community Partner (e.g., patient/caregiver advisory panel) who will provide advice and consultation throughout study planning and implementation.
• Can be relevant to a specific therapeutic or a specific ALS subtype and does not have to broadly apply to all patients.

• Clinical Biomarkers: Identification, development, and/or validation of promising biomarkers for ALS. Biomarkers may include, but are not limited to susceptibility/risk, diagnostic, monitoring, prognostic, predictive, response, or safety biomarkers.
• Clinical Outcomes: Identification, development, and/or validation of clinician-, observer-, or patient-reported, and/or performance outcome measures for ALS.

• Maximum funding of $750,000 for direct costs (plus indirect costs)


• Maximum period of performance is 3 years

• Supports secondary preclinical validation and/or Investigational New Drug-enabling studies of therapeutics for ALS.
• Must begin with lead compound(s) in hand.
• Preliminary data are required including therapeutic efficacy in at least one relevant preclinical ALS model system.
• Mechanism-specific predictive/cohort-selective, target engagement, and pharmacodynamic biomarker development, in parallel to the main therapeutic effort, is a critical component. If mechanism-specific biomarkers are already available or currently in development, how the existing biomarkers will improve trial design, patient selection, and efficiency or interpretation of the proposed ALS therapeutic approach must be described.

• Maximum funding of $1,500,000 for direct costs (plus indirect costs)


• Maximum period of performance is 3 years

• Supports new, innovative, high-risk, high-gain hypothesis-driven ideas aimed at ALS drug or therapy discovery.
• Preliminary data are not required, though strong scientific rationale and experimental feasibility are important.
• Projects focusing primarily on ALS pathophysiology without development of a therapy are not allowed.
• All applications must include an aspect of mechanism-specific biomarker development in parallel to the main therapeutic effort.
• Proposals can be for a specific ALS subtype and do not have to broadly apply to all patients.

• Maximum funding of $600,000 for direct costs (plus indirect costs)


• Maximum period of performance is 2 years