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Targeting Tumors More Precisely, With Fewer Lasting Side Effects for Kids

First-in-pediatric trial to destroy relapsed or refractory tumors

Chicago, Ill. – Pediatric patients with cancer are often treated with a cocktail of therapies to attack the disease through a variety of mechanisms. While this approach has been instrumental in saving children’s lives, the life-saving therapies can be accompanied by acute side effects, and the treatments may have lingering impacts as cancer survivors enter adulthood.

Magnetic resonance-guided high-intensity focused ultrasound (MR-HIFU) holds the promise of surgically removing large tumors without exacting the same array of harsh side effects. Ultrasound relies on high-frequency sound waves to make diagnostic images. Those same sound waves can be used therapeutically to destroy tumors. Layering on MR imaging gives clinicians the ability to precisely guide the ultrasound therapy in real time.

A study led by Children’s National Health System researchers and clinicians is using MR-HIFU for the first time in children to examine its safety and feasibility. The project will treat patients younger than 30 whose solid tumors have stopped responding to other treatments or have begun to regrow. Lead author AeRang Kim, MD, PhD, an oncologist affiliated with Children’s National Center for Cancer and Blood Disorders, will discuss the study protocol during the American Society of Clinical Oncology annual meeting, held June 3 to 7 in Chicago and attended by an estimated 30,000 oncology professionals from around the globe.

“Advantages over conventional local tumor control - such as surgery, radiation therapy, or radiofrequency ablation - are that MR-HIFU is non-invasive, non-ionizing, and enables ablation of large tumor volumes,” Dr. Kim and colleagues write. “A transducer focuses ultrasound beams into a selected target site resulting in thermal ablation at the focus (temperature ≥55°C).”

The focused ultrasound kills just the solid tumor, while sparing neighboring healthy tissue.

The clinical trial, “MR-guided high-intensity focused ultrasound (HIFU) on pediatric solid tumors,” will use focused ultrasound to destroy refractory or relapsed solid tumors located in the bone or in soft tissue near bones while the children are sedated. The tolerability of treatment will be checked for two weeks after the ablation, and additional MR-HIFU treatment is possible at that time. Study participants will be followed for up to one year to gauge tumor response. Secondary outcomes, such as impact on immune markers, will be studied as focused ultrasound ablation has been shown in pre-clinical models to activate the immune system to mount its own anti-tumor response.

“Our hope is this will provide another way to precisely target just the area where there is a solid tumor,” Dr. Kim says. “With MR-HIFU, clinicians see how and where temperature rises in real time, moving from the blue toward the red end of the light spectrum. You can see exactly where that focused heat is being applied – and where it is not – ensuring that healthy tissue remains untouched. Our work will build a strong foundation for future pediatric trials using ablation, providing additional precision in treating children’s solid tumors,” she continues.

Related resources: Enroll in this clinical trial

Media Contact: Diedtra Henderson, Office:  202-476-4500, Mobile: 443-610-9826

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About Children’s National Health System

Children’s National Health System, based in Washington, DC, has been serving the nation’s children since 1870. Children’s National is a Leapfrog Group Top Hospital, Magnet® designated, and was ranked among the top 10 pediatric hospitals by U.S. News & World Report 2015-16. Home to the Children’s Research Institute and the Sheikh Zayed Institute for Pediatric Surgical Innovation, Children’s National is one of the nation’s top NIH-funded pediatric institutions. With a community-based pediatric network, seven regional outpatient centers, an ambulatory surgery center, two emergency rooms, an acute care hospital, and collaborations throughout the region, Children’s National is recognized for its expertise and innovation in pediatric care and as an advocate for all children. For more information, visit, or follow us on Facebook and Twitter.

About the American Society of Clinical Oncology

Founded in 1964, the American Society of Clinical Oncology (ASCO) is the world’s leading professional organization representing physicians who care for people with cancer. With nearly 40,000 members, ASCO is committed to improving cancer care through scientific meetings, educational programs and peer-reviewed journals.

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