Focus On Biomarkers Research

Photo of a neurological response to a sensory stimulus.

A biomarker is a defined characteristic that is used as an indicator of normal biological processes, pathogenic processes or responses to a therapeutic intervention.  Biomarkers are recognized as essential tools for the development of therapeutics, allowing improved and better informed clinical trial design through indicators of target engagement and those that enable patient stratification.  In addition, biomarkers can facilitate the evaluation of therapeutic intervention on disease progression or recurrence. Although biomarker discovery is a very active research area, few biomarkers have been sufficiently validated for use in clinical practice or clinical trials.

The Biomarker Development Process diagram

NINDS Biomarker Funding Opportunities

The NINDS Biomarker Program is focused on improving the quality and efficiency of neurotherapeutic clinical research by supporting rigorous biomarker development and validation.  In order to achieve this goal, the program: 1) promotes rigorous biomarker identification and validation through milestone-driven funding opportunities, and 2) facilitates the validation of biomarkers and biomarker signatures as fit for purpose tools for use in clinical trials to accelerate therapeutic development and aid in decision making for patient care.

Biomarker Development

PAR-22-089  R61/R33 Research Project – Exploratory/Developmental Grant

Analytical Validation

PAR-24-095  U01 Research Project – Cooperative Agreement
PAR-24-098  U44 SBIR Fast Track – Cooperative Agreement

Clinical Validation

PAR-24-097  U01 Research Project – Cooperative Agreement
PAR-24-096  U44 SBIR Fast Track – Cooperative Agreement


Biomarker Program FAQs

NINDS Funded Biomarker Projects and Publications
List of all current (active) NINDS supported projects available in the NIH RePORTER database, with associated publications. 

HEAL Funded Biomarker Projects and Publications
List of all current (active) HEAL supported projects available in the NIH RePORTER database, with associated publications. 


Resources and Tools


Funding Opportunities 

News & Events

NINDS Advancing Digital Endpoints and Digital Health Technologies for Neurological and Neuromuscular Disorders Workshop (September 18th, 2023, September 29th,2023 and October 10th,2023)

Researchers examine how Parkinson's disease alters brain activity over time

NINDS Biomarker Webinar (January 11th, 2024)

OPEN Stage webinar - Biomarker development and validation: Translating discoveries into fit-for-purpose clinical tools (January 30th, 2023)

NIH Workshop: Discovery and Validation of Biomarkers to Develop Non-Addictive Therapeutics for Pain (November 14-15, 2018)


Related Topics 

Provides draft guidance to industry on enrichment strategies that can be used in clinical trials intended to support effectiveness and safety claims in new drug applications (NDAs) and biologics license applications (BLAs).

Clarifies terminology and uses of biomarkers and endpoints as they pertain to the progression from basic biomedical research to medical product development to clinical care. Developed by FDA and NIH to promote consistent use of biomarker terms and concepts, and thereby advance biomarker science.

Banks and distributes biological samples that can be used to identify biomarkers of disease susceptibility, onset and progression for neurological and neuropsychiatric diseases. BioSEND currently banks biospecimens, including DNA, plasma, serum, RNA, CSF, and saliva.

Banks and distributes human post-mortem brain tissue and related biospecimens that span neurological, neuropsychiatric, and neurodevelopmental diseases and disorders. The NBB serves as a central point of access to the world-class collections of six biorepositories. In addition, the NBB provides researchers with a wealth of resources to facilitate their research, including medical records and clinical data sets (when available) as well as access to quality metrics and best practices used by each site.

An iPSC-based biorepository with cell sources that include fibroblasts and/or induced pluripotent stem cells for Alzheimer’s Disease, Amyotrophic Lateral Sclerosis (ALS), Ataxia-telangiectasia, Frontotemporal Lobar Degeneration (FTD), Huntington’s Disease, Parkinson’s Disease, and healthy controls.  Cell sources, including isogenic cell lines for current and new diseases covered by the NINDS are continuing to be added.

The Accelerating Medicines Partnership (AMP) is a public-private partnership between the National Institutes of Health (NIH), multiple biopharmaceutical and life sciences companies, and non-profit organizations. The research plan proposed for AMP PD encompasses a deep molecular characterization and longitudinal clinical profiling of PD patient data and biosamples with the goal of identifying and validating diagnostic, prognostic and/or disease progression biomarkers for Parkinson’s disease (PD). AMP PD utilizes well characterized cohorts with existing biosamples and clinical data that were collected under comparable protocols and using common data elements.

Extensible, scalable informatics platform for traumatic brain injury (TBI) relevant data (medical imaging, clinical assessment, environmental and behavioral history, etc.) and for all data types (text, numeric, image, time series, etc.). FITBIR was developed to share data across the entire TBI research field and to facilitate collaboration between laboratories, as well as interconnectivity with other informatics platforms.