DOD’s Amyotrophic Lateral Sclerosis Therapeutic Development Grant Supports Preclinical and IND-Enabling Research

The FY24 ALSRP Therapeutic Development Award supports research ranging from preclinical validation of therapeutic leads through Food and Drug Administration (FDA) Investigational New Drug (IND)-enabling studies. The proposed studies are expected to be empirical in nature and product-driven. Applicants with limited ALS experience are strongly encouraged to include collaborators with substantial experience in the relevant ALS model systems, endpoints, and pathophysiology.

Applications supported by this award must begin with lead compounds in hand and must include proof-of-concept efficacy data in at least one preclinical model system of ALS, including whole animal and cellular model systems.

Examples of activities that will be supported by this award include:

• Confirmation of candidate therapeutics obtained from screening or by other means, including optimization of potency and pharmacological properties and testing of derivatives and sister compounds.
• Validation of pilot efficacy studies (such as from an ALSRP Therapeutic Idea Award [TIA]), including the use of additional ALS model systems and/or replicating preliminary data with more time points or additional doses.
• ND-enabling studies to include: compound characterization; absorption, distribution, metabolism, and excretion (ADME) studies; studies on formulation and stability leading to Good Manufacturing Practice production methods; dose/response and toxicology studies in relevant model systems.

Applicants seeking support for basic research focused on ALS drug discovery are encouraged to apply for the FY24 ALSRP TIA (Funding Opportunity Number HT942524 ALSRP TIA), which does not require preliminary data (https://cdmrp.health.mil/funding/alsrp).

Mechanism-specific, predictive/cohort-selective, target engagement, and pharmacodynamic biomarker development, in parallel to the main therapeutic effort, is a critical component of the FY24 ALSRP Therapeutic Development Award. If 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 apparent in the application. Development of biomarkers for the purposes of diagnosis, prognosis, or measurement of general disease progression without consideration of the therapeutic development process will not be supported. Applicants seeking support for biomarker development independent of therapeutic development are encouraged to apply for the FY24 ALSRP Clinical Outcomes and Biomarkers Award (Funding Opportunity Number HT942524ALSRPCOBA).

The FY24 ALSRP Therapeutic Development Award supports research ranging from preclinical validation of therapeutic leads through Food and Drug Administration (FDA) Investigational New Drug (IND)-enabling studies. The proposed studies are expected to be empirical in nature and product-driven. Applicants with limited ALS experience are strongly encouraged to include collaborators with substantial experience in the relevant ALS model systems, endpoints, and pathophysiology.

Applications supported by this award must begin with lead compounds in hand and must include proof-of-concept efficacy data in at least one preclinical model system of ALS, including whole animal and cellular model systems.

Examples of activities that will be supported by this award include:

• Confirmation of candidate therapeutics obtained from screening or by other means, including optimization of potency and pharmacological properties and testing of derivatives and sister compounds.
• Validation of pilot efficacy studies (such as from an ALSRP Therapeutic Idea Award [TIA]), including the use of additional ALS model systems and/or replicating preliminary data with more time points or additional doses.
• ND-enabling studies to include: compound characterization; absorption, distribution, metabolism, and excretion (ADME) studies; studies on formulation and stability leading to Good Manufacturing Practice production methods; dose/response and toxicology studies in relevant model systems.

Applicants seeking support for basic research focused on ALS drug discovery are encouraged to apply for the FY24 ALSRP TIA (Funding Opportunity Number HT942524 ALSRP TIA), which does not require preliminary data (https://cdmrp.health.mil/funding/alsrp).

Mechanism-specific, predictive/cohort-selective, target engagement, and pharmacodynamic biomarker development, in parallel to the main therapeutic effort, is a critical component of the FY24 ALSRP Therapeutic Development Award. If 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 apparent in the application. Development of biomarkers for the purposes of diagnosis, prognosis, or measurement of general disease progression without consideration of the therapeutic development process will not be supported. Applicants seeking support for biomarker development independent of therapeutic development are encouraged to apply for the FY24 ALSRP Clinical Outcomes and Biomarkers Award (Funding Opportunity Number HT942524ALSRPCOBA).