Home Diseases and Conditions Neuroblastoma

Neuroblastoma

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Development Stage

Early Stage

Focused ultrasound research is in the laboratory phase and is not yet available for patients.

Clinical Trials

Focused ultrasound for this condition is being researched in clinical trials.

International Approval

Focused ultrasound is approved to treat this condition outside the US. Patients can seek commercial treatment at participating international sites.

FDA Approved

The US Food and Drug Administration has approved focus ultrasound for this condition. Patients can seek commercial treatment at participating sites.

Early Stage

Clinical Trials

International

FDA Approved

Focused ultrasound for this condition is being researched in clinical trials.

Focused Ultrasound Therapy

Focused ultrasound is a noninvasive, 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.

How it Works
Where the beams converge, focused ultrasound produces several therapeutic effects that are being evaluated.

One mechanism is the precise ablation (thermal destruction of tissue), which can be done to completely destroy the target or to partially treat it. Partial treatment is believed to stimulate the patient’s immune response, which may have a broader impact.

Another mechanism is to produce local hyperthermia of the targeted tissue, which can have a variety of beneficial effects including the release of chemotherapy drugs that are contained in temperature sensitive molecules. This allows delivery of a higher level of chemotherapy precisely to the targeted tissue, while limiting the effects for the rest of the body.

Advantages
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 could provide a noninvasive alternative to surgery with less risk of complications at a lower cost. 

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 using thermosenstitive doxorubicin in a variety of tumors, including neuroblastoma has been completed.  

The Foundation updates these pages regularly, but with the increasing number of clinical trials, we want to be sure that our audience has the latest information available. Therefore, we also added the website search information for the above trials. If you click here, it will take you to the latest information available from https://www.clinicaltrials.gov/.   

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

Sundland RM, Ballan D, Callier KM, Ayemoba J, Bellary A, Iwanicki IJ, Wu LL, Larkins T, Flores-Guzman F, Gomez-Villa J, Wyles G, Feshitan J, Kandel JJ, Sirsi SR, Hernandez SL. Sonopermeation With Size-sorted Microbubbles Synergistically Increases Survival and Enhances Tumor Apoptosis With L-DOX by Increasing Vascular Permeability and Perfusion in Neuroblastoma Xenografts. Ultrasound Med Biol. 2024 Nov 23:S0301-5629(24)00410-1. doi: 10.1016/j.ultrasmedbio.2024.10.014. PMID: 39581819 

Bellary A, Nowak C, Iwanicki I, Flores-Guzman F, Wu L, Kandel JJ, Laetsch TW, Bleris L, Hernandez SL, Sirsi SR. Non-viral nitric oxide-based gene therapy improves perfusion and liposomal doxorubicin sonopermeation in neuroblastoma models. Theranostics. 2023 Jun 4;13(10):3402-3418. doi: 10.7150/thno.81700. eCollection 2023. PMID: 37351172 

Bellary A, Villarreal A, Eslami R, Undseth QJ, Lec B, Defnet AM, Bagrodia N, Kandel JJ, Borden MA, Shaikh S, Chopra R, Laetsch TW, Delaney LJ, Shaw CM, Eisenbrey JR, Hernandez SL, Sirsi SR. Perfusion-guided sonopermeation of neuroblastoma: a novel strategy for monitoring and predicting liposomal doxorubicin uptake in vivo. Theranostics. 2020 Jul 9;10(18):8143-8161. doi: 10.7150/thno.45903. eCollection 2020.

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.

Click here for additional references from PubMed.