NIH – Disease Mechanisms of Prenatal and Pediatric Hydrocephalus (R01, R21 Clinical Trial Not Allowed)

February 2, 2018 by School of Medicine Webmaster

The following description was taken from the R01 version of this FOA.

Hydrocephalus is a condition in which the primary characteristic is excessive accumulation of cerebrospinal fluid (CSF) in the brain resulting in enlargement of the ventricles.  Prenatal and/or pediatric hydrocephalus may be caused by either intrinsic factors including genetics or extrinsic factors including hemorrhage or infections. It occurs in 1 in 1000 newborns.  The most common treatment for hydrocephalus is surgical drainage by the placement of a shunt or third ventriculostomy.  Little is known about the pathogenesis (biological mechanisms leading to the disease state) of prenatal and/or pediatric hydrocephalus.

The concept for this initiative originated from the Midbrain/Hindbrain Malformations and Hydrocephalus: Understanding the Causes, Consequences and Gaps in Understanding Workshop held in 2014 and hosted by NINDS.  Over 35 scientists, clinicians and advocacy members came together to address the main objectives of the workshop.  The objectives were designed to facilitate both the basic and clinical research in midbrain/hindbrain malformations and hydrocephalus with the long-term goal to enhance the detection, diagnosis and classification of these disorders as well as to identify potential biological pathways to lead to new therapeutics.  One of the major workshop recommendations for field of hydrocephalus was to address the need for a clearer understanding of disease mechanism.

There have been recent advances in understanding the etiology of prenatal and pediatric hydrocephalus including investigations into the role of neural progenitor cells and blood phospholipids.  Despite these advances, there are still significant gaps in our understanding the molecular, cellular and developmental mechanisms involved in the pathogenesis of hydrocephalus.


This FOA invites hypothesis-driven research projects that focus on deciphering the molecular, cellular and developmental mechanisms involved in the pathogenesis of prenatal and/or pediatric hydrocephalus.  A greater understanding of the disease mechanisms of prenatal and/or pediatric hydrocephalus is critical for the establishment of preventative measures and new therapeutics.  Topics of interest include 1) understanding the pathogenesis of prenatal and/or pediatric hydrocephalus due to intrinsic factors including genetic factors; and 2) understanding the pathogenesis of prenatal and/or pediatric hydrocephalus due to extrinsic factors including hemorrhage and infection.

Studies that focus on the following will be considered out of scope: research related to acquired hydrocephalus in adults (e.g., due to brain tumor, traumatic brain injury and hemorrhage); research related to normal pressure hydrocephalus; pediatric research not focused on hydrocephalus pathogenesis but on perinatal traumatic brain injury, brain hemorrhage and hypoxia; shunt development; natural history studies and clinical trials.

Research that proposes to develop or substantially modify existing cutting-edge technologies that will advance prenatal and/or pediatric hydrocephalus research is being supported by PA-18-623.

Deadlines:  Standard dates and standard AIDS dates apply


Filed Under: Funding Opportunities