Scientists Awarded Grants to Expand Our Knowledge on Hydrocephalus
There is a lot we do not know about how hydrocephalus develops and how to best treat the condition across our many communities. The Hydrocephalus Association’s 2021 Innovator Award recipients are looking to change that by exploring new ideas about why hydrocephalus develops and testing new treatments to improve long-term outcomes.
These scientists are expanding our knowledge about the causes of hydrocephalus and working to develop new treatments that could impact our entire community, from infants with posthemorrhagic hydrocephalus to older adults with NPH.
Dr. Bonnie Blazer-Yost
Professor of Biology at Indiana University – Purdue University Indianapoliswill explore whether targeting inhibitors of SGK1, which is a protein that has been implicated in a variety of pathways related to hydrocephalus pathophysiology, is a feasible treatment for hydrocephalus. This study will hopefully lead to the production of a clinically relevant pharmaceutical treatment. Dr. Blazer-Yost’s research is supported by Team Hydro.
Dr. Justin Cetas
Department Chair of Neurosurgery at the University of Arizona, will examine a potential new mechanism for Post-Hemorrhagic Hydrocephalus (PHH), exploring the role of certain byproducts present after ischemic and hemorrhagic stroke to hopefully aid in the development of new therapeutic targets for hydrocephalus.
It is with great sadness that we announce the passing of Dr. Justin Cetas. Our heartfelt sympathy is with his family and friends.
Dr. Joanne Conover
Professor at the University of Connecticut, will investigate the role of the inflammatory response in Post-Infectious Hydrocephalus (PIH). This research will hopefully lead to the identification of PIH biomarkers, which can in turn help in the development of therapeutics. Dr. Conover’s research is funded through the Rudi Schulte Research Institute.
Dr. Sheng Chih (Peter) Jin
Assistant Professor at the Washington University School of Medicine, will use whole genome sequencing to identify potential novel genomic elements which may contribute to the development of congenital hydrocephalus (CH). This research could lend to more comprehensive diagnoses of CH as well as inform the development of therapeutic targets.
Dr. Mats Tullberg
Professor & Senior Consultant of Neurology at the University of Gothenburg, Sweden, will aim to better quantify gait disturbances in idiopathic Normal Pressure Hydrocephalus (iNPH) using 3D gait analysis. This study will hopefully lead to better diagnostic criteria for NPH as well as clearer outcome measures to determine treatment efficacy.
Every year, we award grants to brilliant scientists to fund their high-impact research projects. The Innovator Award provides seed funding for bold and innovative work with the potential to transform hydrocephalus research.
Funding for the 2021 Innovator Awards was made possible through the support of the Posthemorrhagic Hydrocephalus Campaign and individual donations.
AHCRN Awarded $14M NIH Grant to Study Shunt Treatment for NPH
Idiopathic Normal Pressure Hydrocephalus (iNPH) is a complex disorder of the elderly that affects as many as 700,000 persons in the U.S. The only treatment requires brain surgery, typically implanting a permanent internal drainage system called a shunt into the brain to drain the excess cerebrospinal fluid.” However, not everyone in the medical community agrees that shunt surgery is an effective treatment for iNPH, and, as a consequence, many patients who could benefit from shunt surgery do not receive it. Now, thanks to a $14 million National Institute of Neurological Disorders and Stroke (NINDS) grant, the largest grant ever awarded to study adult hydrocephalus, the 8 sites of the Adult Hydrocephalus Clinical Research Network (AHCRN), working with 13 other health centers, hope to change that perspective with a study designed to prove conclusively whether shunt surgery for iNPH is beneficial.
The NINDS funded grant, called “A Placebo-Controlled Efficacy in iNPH Shunting (PENS) Trial” will be the first large scale, multi-center, blinded, randomized controlled trial to evaluate the true response of shunting in patients with iNPH.
“This grant will allow the AHCRN to use its network of neurosurgeons, neurologists, and medical institutions to conduct a clinical trial that will finally give us the answers we’re looking for about whether shunting is an effective treatment for iNPH,” explained Dr. Mark Hamilton, Neurosurgeon, AHCRN Chair, and Vice Chair of the Hydrocephalus Association Medical Advisory Board.
The study is being conducted in partnership with the Johns Hopkins Cerebral Fluid Center. Johns Hopkins will also serve as the primary coordinating center.
“This study is important because right now some in the medical community are not convinced that shunts are an effective treatment for iNPH. Much of this uncertainty is due to the lack of a high-quality randomized controlled trial,” said Dr. Mark Luciano, Neurosurgeon, of the Cerebral Fluid Center at Johns Hopkins and Principal Investigator of the study.
During the PENS trial, 100 patients with iNPH who are undergoing shunt surgery will be randomly assigned to one of two groups, either with the shunt valve open (on) or closed (off) for the first 3 months after surgery, after which all shunts are in the open setting. The shunt valves can easily be adjusted to an open setting in the clinic with a simple tool that does not require additional surgery. Evaluations before and after surgery will compare the response of iNPH symptoms such as slowed walking speed, impaired cognition, mood, and bladder control at the end of 3 months in the two groups and will continue to follow the symptom response in all patients for the following year while all shunts are in the open setting.
The AHCRN, one of three research networks funded by the Hydrocephalus Association, is a network of eight hospitals that conducts clinical research to improve treatment for the adult forms of hydrocephalus.
Tessa van der Willigen
HA Research Co-Chair
“My husband and I are incredibly excited about the progress HA has made possible in research on hydrocephalus. In just the last decade, the number of scientists looking for better treatments has exploded, in large part because of HA's willingness to fund early-stage, innovative research. There are so many more ideas out there, and connections to be made to draw even more people into the field. We've already seen breakthrough discoveries. This is why my family chooses to support HA’s research.”
Nurturing Future Researchers
As part of our efforts to grow and nurture hydrocephalus researchers, in 2021 we launched the Ralph Kistler Research Internship. The Ralph Kistler Research Internship is for undergraduate college students who are interested in the sciences, public health, and non-profit operations.
Our first Ralph Kistler intern was Elliot Myong. Elliot recently graduated from the University of Southern California with a degree in Biomedical Engineering. He’ll be working as a medical scribe in Marina del Rey for the next year and is currently applying to medical schools.
“Coming into this internship, I knew that I wanted to pursue a career in medicine. I was always captivated by the field of neurology, as I found the complexities of the brain to be quite beautiful. Throughout the internship, my interactions with neurologists and neurosurgeons specializing in hydrocephalus treatment opened my eyes to the type of impact I could make in that field,” Elliot said.
While interning at HA, he was involved in the Community Research Priority Assessment, which solicits input from the hydrocephalus community to develop a top 10 list of research priorities. This list will then guide HA’s future research endeavors.
For Elliot, the experience he gained was invaluable in helping him decide what research to focus on as he moves ahead in his studies.
“I was not only able to gain experience with a research effort at a national scale but was also able to gain insight into the current gaps in hydrocephalus treatment. This is what actually drove me to join a lab at USC, which focuses on building micro sensors for cerebral spinal fluid flow through hydrocephalus shunts, as shunt failure was something that was highly ranked in the priority assessment,” he explained.
Elliot also had the opportunity to learn about how the condition impacts children and adults, and to witness the resiliency of the hydrocephalus community.
“Seeing the power of a community united by their illness has reshaped my view on medicine, and I now aspire to be a physician who not only tends to the needs of individual patients, but who understands and advocates for the needs of entire communities. Through the relationships I built with the staff at HA and community members, I left the Ralph Kistler Research Internship feeling forever connected to the hydrocephalus community,” he said.
The Ralph Kistler Research Internship was made possible thanks to contributions from our donors. Special thanks to Emily and Russel Fudge, and Dr. Jack Walker, for their incredible fundraising efforts. We hope to continue the internship for many years to come so that it can continue to serve as a launching pad for the new generation of hydrocephalus researchers and clinicians.