Focused Ultrasound Therapy
Focused ultrasound is an early-stage, noninvasive, therapeutic technology with the potential to improve the quality of life and decrease the cost of care for patients with hemophilia B, also known as Christmas Disease or Factor IX Deficiency. This novel technology focuses beams of ultrasound energy precisely and accurately on targets in the liver, enabling enhanced deposition of microbubbles that contain gene-expression vectors, without damaging surrounding normal tissue. Preclinical work has begun in this area, and further work will be needed before human trials.
An option for treating Hemophilia B would be to use ultrasound-targeted microbubble destruction, and the use of focused ultrasound to enhance the vascular permeability in the liver would make the process more efficient.
For certain patients, focused ultrasound could provide a noninvasive and more effective form of treatment.
- Focused ultrasound is noninvasive, 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.
At the present time, there are no clinical trials recruiting patients for focused ultrasound enhancement of gene-expression vectors to treat Hemophilia B.
Regulatory Approval and Reimbursement
Focused ultrasound treatment for enhancing the gene-expression treatment for Hemophilia B is not yet approved by regulatory bodies or covered by medical insurance companies.
High Intensity Focused Ultrasound for Hepatic Gene Transfer. Presentation by Cynthia Anderson, PhD. 2018