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Focused Ultrasound Therapy

Focused ultrasound is an early-stage, non-invasive, therapeutic technology with the potential to improve the quality of life and decrease the cost of care for patients with neuroblastoma. This novel technology focuses beams of ultrasonic energy precisely and accurately on targets deep in the brain without damaging surrounding normal tissue. Where the beams converge, the ultrasound produces a variety of therapeutic effects that could potentially enable neuroblastoma to be treated without surgery.

The mainstay of treatment for neuroblastoma is surgical resection coupled with chemotherapy. However, many patients do not respond adequately to chemotherapy and commonly relapse. Furthermore, most long-term survivors suffer adverse effects from their treatment. 

For certain patients, focused ultrasound ablation – to kill tumor cells – could provide a non-invasive alternative to surgery with less risk of complications and lower cost. Focused ultrasound-induced hyperthermia – moderate heating of tissue that is below the threshold for cell death – has the potential to increase blood flow to the tumor and enhance the local delivery of chemotherapy, thereby minimizing its adverse effects.

Potential advantages as compared to current treatments:

  • Focused ultrasound is non-invasive and radiation-free – no incisions, holes in the skull, electrodes in the brain – and therefore has reduced risk for infection and blood clots. 
  • Precise targeting minimizes damage to non-targeted healthy brain.
  • Treatment can be conformed to a wide variety of shapes and sizes.
  • Tissue temperature can be monitored in real-time using magnetic resonance imaging (MRI).
  • Treatment can be a complement to drug therapy, enabling effective treatment with lower doses of drugs and minimal toxicity.

Clinical Trials

A clinical trial is using thermosenstitive doxorubicin in a variety of tumors, including neuroblastoma. 

A second clinical trial is using focused ultrasound to ablate recurrent or refractory solid tumors, including neuroblastoma. 

Regulatory Approval and Reimbursement

Focused ultrasound treatment for neuroblastoma is not yet approved by regulatory bodies or covered by medical insurance companies.

Preclinical Laboratory Studies

Early-stage preclinical studies suggest that focused ultrasound may play a beneficial role in the treatment of unresectable neuroblastoma.  In one study, mice with neuroblastoma were treated with chemotherapy, focused ultrasound plus chemotherapy, or focused ultrasound alone. Following treatment, 53% of mice treated with focused ultrasound and 80% of mice treated with focused ultrasound plus chemotherapy were cured with no evidence of tumor at 200 days, versus 100% fatality at 45 days in the control group and in mice treated with just chemotherapy.

Additional Information

Children’s Neuroblastoma Cancer Foundation

Notable Papers

Eranki A, Srinivasan P, Ries M, Kim A, Lazarski CA, Rossi CT, Khokhlova TD, Wilson E, Knoblach S, Sharma KV, Wood BJ, Moonen C, Sandler AD, Kim PCW. High Intensity Focused Ultrasound (HIFU) Triggers Immune Sensitization of Refractory Murine Neuroblastoma to Checkpoint Inhibitor Therapy. Clin Cancer Res. 2019 Oct 15. pii: clincanres.1604.2019. doi: 10.1158/1078-0432.CCR-19-1604.

Tung S, Fahy AS, Lamberti-Pasculli M, Waspe AC, Pichardo S, Gerstle JT. Magnetic Resonance-guided High-intensity Focused Ultrasound(MRgHIFU) Virtual Treatment Planning for Abdominal Neuroblastoma Utilizing Retrospective Diagnostic 3D CT Images. J Pediatr Hematol Oncol. 2019 Jul 26. doi: 10.1097/MPH.0000000000001563.

Shim J, Staruch RM, Koral K, Xie XJ, Chopra R, Laetsch TW. Pediatric Sarcomas Are Targetable by MR-Guided High Intensity Focused Ultrasound (MR-HIFU): Anatomical Distribution and Radiological Characteristics. Pediatr Blood Cancer. 2016 Oct;63(10):1753-60. doi: 10.1002/pbc.26079. Epub 2016 May 19.

S. Wang, V. Frenkel, and V. Zderic, “Optimization of pulsed focused ultrasound exposures for hyperthermia applications.,” J. Acoust. Soc. Am., vol. 130, no. 1, pp. 599–609, Jul. 2011. 

H. Matsumoto, “Revisiting sensitization mechanisms in cancer thermochemotherapy.,” Fukuoka igaku zasshi = Hukuoka Acta medica, vol. 100, no. 4, pp. 95–103, Apr. 2009.

Yang R, Reilly CR, Rescorla FJ, Sanghvi NT, Fry FJ, Franklin TD Jr, Grosfeld JL. Effects of high-intensity focused ultrasound in the treatment of experimental neuroblastoma. J Pediatr Surg. 1992 Feb;27(2):246-50

Click here for additional references from PubMed.


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