Hydrocephalus pediatric research and clinical trials

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Contact the Hydrocephalus Program

At Boston Children’s Hospital, our care is informed by our research, and our discoveries in the laboratory strengthen the care we provide at each child's bedside. Children’s scientific research program is one of the largest and most active of any pediatric hospital in the world.

In particular, our neurosurgery and neurology researchers are:

  • making new inroads in understanding the causes and development of nervous system diseases and disorders
  • paving ground for promising new treatments and interventions
  • offering ideas and discoveries that could lead to better treatments and cures

Here are some of our current research projects with promise for treating hydrocephalus.

Avoiding shunts in hydrocephalus

During his years as a medical missionary in Africa, Boston Children’s neurosurgeon Benjamin Warf, MD, realized that many children who received shunts for hydrocephalus could not make return visits to have their shunts checked and maintained, and had nowhere to turn if their shunts failed. So he developed a one-time, minimally invasive procedure called endoscopic third ventriculostomy/choroid plexus cauterization (ETV/CPC) that can be used instead of shunting. He brought the operation back to Boston Children’s, and clinical trials are showing good results. Dr. Warf’s pioneering work earned him a 2012 genius grant” from the John D. and Catherine T. MacArthur Foundation.

Read more in our our health blog, Thriving.

Changing views on role of cerebrospinal fluid in hydrocephalus may lead to new treatments
Traditionally, physicians have believed that hydrocephalus is caused by an imbalance in the production and absorption of cerebrospinal fluid—but Children's neurosurgeon Joseph Madsen, MD, and his team have discovered that the central problem in hydrocephalus might not be the amount of fluid in the brain: Instead, it may be the fluid's pulsing motion. These natural pulses, linked to the heartbeat, are believed to ensure adequate blood flow throughout the brain. However, at the same time, the smallest blood vessels need to be protected from the pulses to prevent mechanical stress.

Madsen and his colleagues have identified the system that provides such protection in the normal brain, and are speculating that a malfunction of this system may contribute to the development of hydrocephalus.

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 Children's Vascular Biology Program, and Leonard Zon, MD, PhD, director of 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.

Learn more about scientific research at Children’s.

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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