DOD Amyotrophic Lateral Sclerosis, Therapeutic Idea Award

The FY23 ALSRP Therapeutic Idea Award supports the initial exploration of innovative, high-risk, high-gain ideas aimed at Amyotrophic Lateral Sclerosis (ALS) drug or treatment discovery. The studies supported by this award mechanism are expected to be hypothesis-driven and generate preliminary data for future avenues of therapeutic investigation. 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. Applications may demonstrate the ability to achieve interpretable results in the absence of preliminary data supporting the hypothesis. 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. Research with high impact should pioneer transformative research that could lay the foundation for a new direction in the field of ALS therapeutic development. Projects should strive to produce the type and amount of data needed to apply for the next stage, i.e., ALSRP Therapeutic Development Award or other mechanisms for ALS therapeutic advancement.

The Therapeutic Idea Award encourages applications submitted by early-career investigators as well as applications that include meaningful and productive collaborations between/among 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 FY23 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, Attachment 9, Biomarker Statement.