10-month-old NARA ARISKA who suffers from a rare condition called hydrocephalus - known as water on the brain - sleeps at a hut in Toboali village on the South Bangka, of Bangka Belitung Islands in Indonesia on October 8, 2016. (Photo by Donal Husni
Researchers at the University of Cincinnati College of Medicine say an experimental gene therapy could eventually lead to a less invasive treatment for hydrocephalus, a condition in which excess fluid builds up in the brain.
The early-stage study was led by Agnes (Yu) Luo, Ph.D., and published in the journal Fluids and Barriers of the CNS.
Dig deeper:
Cerebrospinal fluid, or CSF, is a clear fluid that surrounds the brain and spinal cord. It helps protect the brain and carries nutrients and other important substances.
Hydrocephalus develops when too much of this fluid collects in the brain. The buildup can increase pressure, damage brain tissue and cause serious neurological problems.
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The main treatment is surgery to place a shunt, a small tube that drains extra fluid from the brain. However, shunts can stop working or become infected, sometimes requiring additional surgeries.
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Doctors may also use an endoscopic procedure to burn away some of the tissue that makes cerebrospinal fluid. That treatment can help some patients, but it does not significantly lower the amount of fluid produced and is not safe or possible for everyone.
Researchers say better treatment options are needed.
The backstory:
Luo and her team worked with researchers at the University of California, Davis, to test an experimental gene therapy in animal models.
The treatment uses an adeno-associated virus, or AAV. These modified viruses can deliver genetic material into specific cells without causing the illness associated with the original virus.
The therapy is designed to target the cells that produce cerebrospinal fluid. By reducing those cells, the treatment may reduce the amount of fluid made in the brain.
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The treatment may eventually be delivered through a less invasive procedure, possibly using an existing shunt.
Why you should care:
Researchers at UC Davis created hydrocephalus in adult animals and then treated them with the experimental gene therapy.
The treatment helped prevent edema, or excess fluid, from entering brain tissue outside the brain’s fluid-filled chambers.
Researchers later reviewed MRI scans without knowing which animals had received the treatment. That blinded review confirmed the findings.
Researchers are also studying whether lowering cerebrospinal fluid could affect the brain in other ways.
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The fluid may play a role in neurogenesis, the process through which the brain creates new nerve cells after birth. Early animal testing suggests that reducing cerebrospinal fluid production may moderately reduce the brain’s ability to make new neurons.
That could potentially affect the brain’s memory-related networks, researchers said. However, people with hydrocephalus already face the possibility of repeated surgeries throughout their lives, and those procedures may also affect cognitive function over time.
What's next:
Luo and Taranov said more research is needed to understand the treatment’s possible risks and benefits.
The researchers plan to test the gene therapy in larger animals and in human tissue. If those studies are successful, the treatment could eventually be tested in human clinical trials.
Luo and Taranov are also listed as inventors on a pending patent related to the therapy.
The Source: The information came from a preclinical study by researchers at the University of Cincinnati College of Medicine and the University of California, Davis, published in the journal Fluids and Barriers of the CNS. This story was reported from Los Angeles.