The Therapeutic Idea Award is designed to promote new ideas aimed at drug or treatment discovery that are still in the early stages of development.
Development, modification, and/or validation of preclinical model systems or the application of high-throughput screens to define or assess lead compounds for ALS treatment are of interest. Parallel development of methods to measure either target binding or proximal downstream effects (target engagement) and the potential for undesirable activities at related but unintended targets (selectivity) are encouraged.
All proposed 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. Projects that focus primarily on investigating the pathophysiology of ALS are outside the scope of this Program Announcement/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 must also have the potential for a major impact and accelerate progress toward treatment for ALS. The impact may be near-term or long-term, but must be significant and move beyond an incremental advancement. Applications must articulate the pathway to making a clinical impact for individuals with, or at risk for, ALS, even if clinical impact is not an immediate outcome. Presentation of preliminary data is not consistent with the intent of the Therapeutic Idea Award mechanism. While the inclusion of preliminary data is not prohibited, the strength of the application should not rely on preliminary data, but on the innovative idea and the potential impact it will provide. The Therapeutic Idea Award encourages applications that include meaningful and productive collaborations between investigators. As appropriate, applicants are encouraged to collaborate with industry partners and/or established ALS researchers.