Hemophilia

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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. Similar work is being done with Hemophilia A. This novel technology focuses beams of ultrasound energy precisely and accurately on targets in the liver.

How it Works
Where the beams converge, focused ultrasound enables the release of gene-expressing vectors (in Hemophilia B) and non-viral genes (Hemophilia A), without damaging surrounding normal tissue. This preclinical work will be important in the development of future human trials.

Advantages
For certain patients, focused ultrasound could provide a noninvasive alternative treatment 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

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.

Notable Papers

Lo WS, Sheen JM, Chen YC, Wu KT, Wang LY, Lau YC, Hsiao CC, Ko JY. The Application of Focused Medium-Energy Extracorporeal Shockwave Therapy in Hemophilic A Arthropathy. Healthcare (Basel). 2022 Feb 11;10(2):352. doi: 10.3390/healthcare10020352.

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

Click here for additional references from PubMed.

Early Stage