DOD Amyotrophic Lateral Sclerosis, Therapeutic Development Award

The Department of Defense (DOD) Amyotrophic Lateral Sclerosis (ALS) Therapeutic Development Award (ALSRP) for the 2024 fiscal year supports both preclinical validation of therapeutic leads and Food and Drug Administration (FDA) Investigational New Drug (IND)-enabling studies focused on ALS treatment.

The grant encourages applications from researchers with varying levels of ALS experience. Those with limited ALS experience are urged to partner with collaborators who have in-depth experience in ALS model systems, endpoints, and pathophysiology.

Applicants must already have lead compounds and proof-of-concept efficacy data from at least one preclinical ALS model system, including whole animal and cellular model systems. This should serve as the starting point of research supported by this award.

Possible activities funded by this award include confirmation of therapeutic candidates acquired through screening or other methods (e.g. optimizing potency and pharmacological properties, testing derivatives and sister compounds), validation of initial efficacy studies, and IND-enabling studies that encompass compound characterization; absorption, distribution, metabolism, and excretion (ADME) studies; formulation and stability studies that lead to Good Manufacturing Practice production methods; and dose/response and toxicology studies in relevant model systems.

Applicants focusing on basic research for ALS drug discovery are encouraged to consider the ALSRP Therapeutic Idea Award (TIA), which doesn't require preliminary data.

The 2024 ALSRP Therapeutic Development Award also places significant emphasis on development of mechanism-specific, predictive/cohort-selective, target engagement, and pharmacodynamic biomarkers alongside the principal therapeutic effort. Existing biomarkers or those in development should be integral to improving the design, selection, and interpretation of the proposed ALS therapeutic approach. This grant does not support the development of biomarkers for diagnosis, prognosis, or general disease progression without considering the therapeutic development process. Those interested in biomarker development independent of therapeutic development should consider the ALSRP Clinical Outcomes and Biomarkers Award.