First Focused Ultrasound Pediatric Brain Tumor Study Begins

Researchers at Nicklaus Children’s Hospital in Miami, Florida have performed the first procedure in a pediatric and young adult brain tumor study. The trial aims to demonstrate feasibility and safety of using focused ultrasound to ablate a variety of benign tumors located in the central part of the brain in ten patients, ages 8 to 22. Left untreated, these tumors often cause seizures, cognitive delays, or other complications as they grow.

NCH logoThe first patient enrolled in the study suffered from debilitating seizures as a result of a hypothalamic hamartoma. After a single focused ultrasound procedure on March 7 using Insightec’s Exablate Neuro system, MRI scans showed complete ablation of the tumor. The patient was able to go home the following day, returned to school days later, and remains seizure-free.

The medical and research team at Nicklaus Children’s consists of Dr. John Ragheb, Director Division of Neurosurgery; Dr. Travis Tierney, neurosurgeon and the trial’s Principal Investigator: Dr. Ian Miller, Director of Epilepsy Program; Dr. Sanjiv Bhatia, neurosurgeon; Dr. Nolan Altman, Director of Radiology; Dr. Prasanna Jayakar, Chairman of Brain Institute; and Dr. Jennifer McCafferty, Director of Research, Miami Children’s Research Institute.

“Any procedure that is the first of its kind is a bit like launching a space shuttle, but we were confident that focused ultrasound is a perfect match for these types of tumors.” says Dr. Travis Tierney. “Nicklaus Children’s is a leader in treating pediatric epilepsy, so to have a new, innovative tool to offer children and families suffering with these seizure-inducing brain tumors is an exciting achievement.”

Traditional treatment options available include anti-epileptic medications or conventional surgery to remove the mass. However, invasive techniques are associated with risks of bleeding, infections, and even developing new neurological problems. Anti-epileptic medications can also have undesirable side effects or may be ineffective for many patients.

”Focused ultrasound offers many benefits over traditional surgery that are particularly important to pediatric patients.” says Focused Ultrasound Foundation’s Chief Medical Officer, Tim Meakem, MD. “It is incisionless, avoids ionizing radiation, offers less risk of infection and disruption of adjacent normal tissue, can be repeated if necessary, and affords minimal downtime from daily activities.”

The study is jointly funded by the Foundation and Nicklaus Children’s Hospital. Insightec is the regulatory sponsor.

“Expanding focused ultrasound’s reach into pediatrics is one of our main goals in 2017,” says Foundation Chairman Neal F. Kassell, MD. “The technology has proven itself safe and effective in ablating tissue in the adult brain for movement disorders, and it is already being investigated for soft tissue tumors and painful bone tumors in younger patients. We look forward to continuing to work with Nicklaus Children’s and other sites worldwide to advance this innovative care for children.”

“Congratulations to the team at Nicklaus Children’s for another first with our MR-guided focused ultrasound technology. We are committed to supporting research efforts such as these that push the boundaries of our technology to treat more and more patients,” said Maurice R. Ferré MD, CEO and Chairman of Insightec.

Patients seeking further information about the study should contact .

A similar study for adults with epilepsy is underway at the University of Virginia.

Read Nicklaus Children’s Press Release >

About Benign Tumors in the Study
There are a number of types of benign brain tumors that can be treated in this trial. See the full list > 

One class, subependymal giant cell astrocytomas (SEGAs), often cause pediatric epilepsy and can obstruct ventricles, leading to hydrocephalus - a buildup of fluid in the brain. Another central tumor - a hypothalamic hamartoma - can cause gelastic (laughing) seizures and cognitive decline. Other low grade astrocytomas may also be screened.

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