Home Diseases and Conditions Neurofibromatosis

Neurofibromatosis

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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 neurofibromatosis. There are three major types of neurofibromatosis.

  • Type 1 are the most common and typically has areas of altered skin color, café au lait spots, bone abnormalities or learning abnormalities.  
  • Type 2 are vestibular schwannomas, that commonly involve the nerves to the brain and commonly have hearing loss and balance problems.  
  • The most rare form is schwannomatosis, which run near of peripheral nerves, which often create chronic pain.  

This novel technology focuses beams of ultrasound energy precisely and accurately on targets deep in the body without damaging surrounding normal tissue.

How it Works
Where the beams converge, focused ultrasound produces thermal ablation (burning of the target) that has been studies and has been recently approved by the European regulatory agency (CE Mark).    

The recent work by Toosonix involves the use of a transducer device that is specifically designed to treat close to the skin, as it can be done with very low risk of having skin burns. This device has resulted in the recent CE Mark approval in Europe, so the Toosonix device is now covered for this and a variety of other medical and cosmetic indications.   

Advantages
The primary options for treatment of neurofibromatosis, vary based on the location of the tumors, but can include medication and invasive surgery.

For certain patients, focused ultrasound could provide a noninvasive alternative to surgery with less risk of complications – such as surgical wound healing or infection – at a lower cost. It can reach the desired target without damaging surrounding tissue and is repeatable, if necessary.

Clinical Trials

clinical trial for Neurofibromatosis type 1 that impacts peripheral nerves in adults has been completed in Sweden.

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/

Find a Treatment Site

Search for a treatment center or clinical trial near you.

Regulatory Approval and Reimbursement

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

Notable Papers

Spiegel JL, De Maio A, Patel MV, Ungar OJ, Lai C, Truong T, Situ Y, Clapp DW, Conway SJ, Chen JM, Lin VYW, Hynynen K, O’Reilly MA, Le TN. Focused ultrasound-mediated drug delivery of bevacizumab in treating NF2-related schwannomatosis in an animal model. Sci Rep. 2025 Dec 24. doi: 10.1038/s41598-025-32031-9. PMID: 41444363 

Wozniak B, Bove T, Zawada T, Calik J. Treatment of Cutaneous Neurofibromas in Patients with Neurofibromatosis Type 1. Case Rep Dermatol. 2023 Oct 20;15(1):194-201. doi: 10.1159/000534270. eCollection 2023. PMID: 37899948 

Tydings C, Yarmolenko P, Bornhorst M, Dombi E, Myseros J, Keating R, Bost J, Sharma K, Kim A. Feasibility of magnetic resonance-guided high-intensity focused ultrasound treatment targeting distinct nodular lesions in neurofibromatosis type 1. Neurooncol Adv. 2021 Aug 18;3(1):vdab116. doi: 10.1093/noajnl/vdab116. eCollection 2021 Jan-Dec. PMID: 34604751

Click here for additional references from PubMed.