Home Diseases and Conditions Neurofibromatosis, Type 1 (NF1)

Neurofibromatosis, Type 1 (NF1)

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

Focused Ultrasound Therapy 

Focused ultrasound is a rapidly evolving, therapeutic technology that could transform the quality of life and decrease the cost of care for patients with neurofibromatosis, type 1 (NF1). This novel technology focuses beams of ultrasound energy precisely and accurately on targets 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 (https://toosonix.com/) 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.  

The primary options for treatment of NF1 include medication and invasive surgery. For certain patients, focused ultrasound could provide a non-invasive alternative to surgery with less risk of complications and lower cost.  

Advantages 

  • Focused ultrasound is non-invasive, so it does not carry added concerns like surgical wound healing or infection.  
  • Focused ultrasound can reach the desired target without damaging surrounding tissue. 
  • It can be repeated, if necessary. 

Clinical Trials 

At the current time, there are no clinical trials recruiting patients for research into NF1.  

Regulatory Approval and Reimbursement  

Focused ultrasound treatment for NF1 is covered under the European CE Mark, so it may be covered by medical insurance companies. 

Notable Papers 

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 Jan-Dec. 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.