Development of Biomarkers or Composite Biomarkers for Neurological and Neuromuscular Disorders (R61/R33 - Clinical Trial Optional)

The National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH), is offering a phased R61/R33 grant to advance biomarker development for neurological and neuromuscular disorders. The initiative aims to promote the identification, optimization, and initial clinical validation of candidate biomarkers or composite biomarkers that could improve the efficiency of clinical trials and assist in patient care. The project begins with the R61 phase, which focuses on developing a biomarker concept and validating its relevance to a biological process or clinical context. If milestones are met, projects then transition to the R33 phase, where the biomarker undergoes further clinical validation to confirm its utility for one or two specific clinical applications. Studies should be designed to meet an unmet need and should demonstrate how the biomarker improves upon current standards, providing value as a tool in therapeutic development or clinical care. This funding opportunity invites applications that rigorously test candidate biomarkers using human samples and data. Each project should clearly define its biological rationale, targeting neurological or neuromuscular disorders aligned with the NINDS mission. The biomarker's intended clinical use, or "Context of Use" (COU), must be specified, with applicants expected to outline how the biomarker will benefit clinical trials or health care decisions. Research should focus on one or more biomarker categories as defined by NIH, including diagnostic, prognostic, predictive, and safety biomarkers, among others. Each application must also include a Plan for Enhancing Diverse Perspectives (PEDP), reflecting NIH's commitment to fostering diverse research teams and broad scientific inclusivity. Failure to include a PEDP will result in the application’s administrative withdrawal. The project budget is not capped but should be appropriate to the project's needs. The R61 phase can last 1-3 years, with the R33 phase lasting up to 3 years, allowing a maximum total project period of 5 years. Eligibility is open to higher education institutions, nonprofits, government entities, small businesses, and other qualifying organizations, including those from underserved backgrounds. Applicants are encouraged to utilize existing NINDS resources, such as BioSEND for sample collection, and to work with diverse, multidisciplinary teams that possess expertise in biomarker development, clinical research, biostatistics, and neurological disease. To apply, a letter of intent is due 30 days before the application deadline. Required materials include a detailed research strategy that covers the biomarker’s clinical relevance, biological rationale, study design, statistical analysis plan, and a timeline of Go/No-Go milestones necessary for transitioning from the R61 to the R33 phase. Applicants are encouraged to consult with NINDS staff early in the planning phase, as this can help align project aims with NIH guidelines and program goals. Applications must also conform to NIH's updated forms (FORMS-I), which will be accessible 30 days before the earliest application due date. Evaluation criteria include scientific significance, innovation, rigor of the approach, feasibility, and the expertise of the research team. Reviewers will also assess the PEDP and intellectual property plans (if applicable). Projects that address a critical gap in biomarker validation for neurological or neuromuscular disorders will be prioritized. Applications are reviewed by NINDS’s Scientific Review Group, followed by an Advisory Council review. Selected projects will be notified by a Notice of Award, with start dates following the review timeline. Key dates include the first application deadline on February 21, 2025, followed by standard NIH review and award cycles. This funding opportunity expires on January 8, 2028. Pre-application webinars are available, typically held annually, to provide guidance and answer applicants’ questions. For further details, applicants may contact NINDS staff or refer to the NIH Grants Policy Statement and NIH website.