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. Similar work is being done with Hemophilia A for non-viral delivery of genes to the porcine liver. This preclinical work will be important in the development of future human trials.
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.
Regulatory Approval and Reimbursement
Focused ultrasound treatment for enhancing the gene-expression treatment for Hemophilia is not yet approved by regulatory bodies or covered by medical insurance companies.
Tran DM, Zhang F, Morrison KP, Loeb KR, Harrang J, Kajimoto M, Chavez F, Wu L, Miao CH. Transcutaneous Ultrasound-Mediated Nonviral Gene Delivery to the Liver in a Porcine Model. Mol Ther Methods Clin Dev Jul 26 2019; 14:275-284. doi: 10.1016/j.omtm.2019.07.005.
Additional material about this use can be found at his link.
High Intensity Focused Ultrasound for Hepatic Gene Transfer. Presentation by Cynthia Anderson, PhD. 2018