DoD ALS Therapeutic Idea Grant: Advancing Early-Stage Treatments for Amyotrophic Lateral Sclerosis

The FY21 ALSRP Therapeutic Idea Award supports hypothesis-driven therapeutic development and is designed to promote new ideas aimed at drug or treatment discovery that are still in the early stages of development. Development and/or modification of preclinical model systems or the application of high-throughput screens to define or assess lead compounds for ALS treatment may also be proposed. All research projects should include a well-formulated, testable hypothesis based on strong scientific rationale that holds translational potential to improve ALS treatment and/or advance a novel treatment modality. Preliminary data are not required. While the inclusion of preliminary data is not prohibited, the strength of the application should not rely on preliminary data, but on the innovative approach. Projects that focus primarily on investigating the pathophysiology of ALS, without consideration of therapeutic development, are not within the scope of this Funding Opportunity.

Innovation and impact are important aspects of the Therapeutic Idea Award. Research deemed innovative may introduce a new paradigm, challenge current paradigms, introduce novel concepts or technologies, or exhibit other uniquely creative qualities that may lead to potential therapeutics for ALS.

The Therapeutic Idea Award encourages applications submitted by early-career investigators as well as applications that include meaningful and productive collaborations between investigators. Applicants from outside the ALS research field are encouraged to include collaborators with the necessary relevant expertise, such as experience with ALS model systems, endpoints, and pathogenic findings.

Biomarker Option: The FY21 ALSRP Therapeutic Idea Award Biomarker Option encourages applicants to include the development of biomarker(s) in parallel with the main proposed Therapeutic Idea Award research effort. Additional funding, as described in Section II.D.5, Funding Restrictions, is being offered for the co-development of biomarkers that will enhance the drug development process. Efforts may include development of target engagement biomarkers, objective pharmacodynamic biomarkers to measure the biological effect of an investigational therapeutic, or predictive/cohort-selective biomarkers that indicate whether a specific therapy will be effective in an individual patient or patient subgroup. Development of markers for the purposes of diagnosis, prognosis, or measurement of disease progression apart from consideration of the therapeutic development process will not be supported.

To apply for this option, applicants must clearly describe the marker and its potential to improve the effectiveness of the therapeutic development process. For further description, see Section II.D.2.b.ii, Full Application Submission Components.