Hydrocephalus Program | Research and Innovation

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Ben Warf speaks about ETV, an innovative new treatment for infants with hydrocephalus

Clinical Innovation: New treatment for infants with hydocephalus

Hydrocephalus DiagramThe problem
Endoscopic third ventriculostomy (ETV) —in which a small perforation is made in the third ventricle of the brain, allowing cerebrospinal fluid (CSF) to circulate—can often be a permanent treatment for hydrocephalus that avoids the complications of shunt dependence.

But this procedure has been less successful in infants, most likely because it requires competent CSF circulation and absorption pathways that may not have had time to develop.

Innovative solution
A treatment pioneered by Boston Children's Hospital Benjamin C. Warf, MD, combines ETV with choroid plexus cauterization (ETV/CPC), decreasing the amount of CSF produced and thereby reducing the demands the ETV places on the structure of the developing brain.

Hydrocephalus treatment results
Research has shown that ETV/CPC can obviate the need for a shunt in infants with hydrocephalus, in association with spina bifida or encephalocele more than 75 percent of the time, in association with neonatal infection more than 60 percent of the time and in association with other causes of congenital hydrocephalus around 70 percent of the time. Preliminary data in Dr. Warf's current investigation indicates that ETV/CPC could allow half of infants with post-hemorrhagic hydrocephalus of prematurity to forego shunt dependence.

Our innovative approach to treating hydrocephalus in kids

Hydrocephalus Brain Boston Childrens

Boston Children's Hospital's Hydrocephalus Program is dedicated to basic science and translational research that informs medical and surgical approaches to disorders of the brain, spine and nervous system.

Our research program is the most prolific at any pediatric hospital in the world, and our clinicians spend each day seeking new insights.

Our hydrocephalus research includes looking at:

  • how and why diseases like hydrocephalus occur
  • what role genetics play in the onset of hydrocephalus and related conditions
  • how a child's brain develops and where it is most vulnerable to defects and injury

Our Hydrocephalus Program physicians—who are also members of the Harvard Medical School faculty—conduct ongoing research here in our hospital laboratories. They also collaborate routinely with scientists at Boston's world-renowned universities, other biomedical facilities and fellow leading medical centers. 


Is the motion of cerebrospinal fluid responsible for hydrocephalus?

Traditionally, hydrocephalus was thought to be caused by an imbalance of production and absorption of cerebrospinal fluid. Studying the problem, Boston Children's neurosurgeon Joseph Madsen, MD, and colleagues realized that the key to hydrocephalus may not be the amount of fluid in the brain, but rather the fluid's pulsing motion.

These natural pulses, linked to the heartbeat, may help ensure adequate blood flow in the brain. However, the smallest blood vessels need to be buffered from the pulses to protect them from mechanical stress.

Madsen and colleagues have discovered a system that does this in the normal brain, and speculate that malfunction of this system contributes to hydrocephalus. "One reason people with hydrocephalus get headaches may be that their shunts don't solve the problem of buffering the pulsations," Madsen says.

Making strides in developing drug therapies for hydrocephalus

New research is suggesting several new ways to look at hydrocephalus—and even the possibility of treating the condition with drug therapies. Michael Klagsbrun, PhD, of Boston Children's Vascular Biology Program, and Leonard Zon, MD, PhD, director of Boston Children's Stem Cell Research Program, have created animal models of hydrocephalus—in mice and zebrafish, respectively—that could be used to test new theories and, potentially, new drug treatments.

Creating noninvasive ways to measure intracranial pressure

Thanks to a grant from the Boston-based nonprofit Center for Integration of Medicine and Innovative Technology (CIMIT), Children's neurosurgeon Joseph Madsen, MD, is creating a system to noninvasively measure pressure inside the skulls of patients with hydrocephalus, head injuries, subarachnoid hemorrhage and other related conditions. His goal is to create a portable device that can be used by emergency technicians, as well as by medics in battlefield situations.

We are grateful to have been ranked #1 on U.S. News & World Report's list of the best children's hospitals in the nation for the third year in a row, an honor we could not have achieved without the patients and families who inspire us to do our very best for them. Thanks to you, Boston Children's is a place where we can write the greatest children's stories ever told.”
- Sandra L. Fenwick, President and CEO

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