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Cardiomyopathy

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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 research for this condition is in the laboratory phase and is not yet available for patients.

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 various types of cardiomyopathy. The initial work was done on hypertrophic cardiomyopathy, but as this approach, if successful could help with other forms, this is an exciting possibility for many types of cardiomyopathy. Another approach is using Low Intensity Pulsed Ultrasound on an animal model, to impact the ischemic cardiomyopathy. 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 an expanded importation of CRISPR-Cas9 adenovirus ribonucleoprotein into human pluripotent stem cells. To say this in a simpler format, focused ultrasound enables the ability to genetically edit the target cells to be better able to accomplish their tasks. The LIPUS treatment was preventative and alleviated ventricular remodeling, improved electrophysiological function and decreased the release of inflammatory cytokines.  

The primary options for treatment of cardiomyopathy are limited, and often include medications which can have adverse side effects.  

While significant work has been accomplished, there is still much to be done before this technology will be widely available. 

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 present time, there are no clinical trials recruiting patients for focused ultrasound treatment of cardiomyopathy.  

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/. 

Regulatory Approval and Reimbursement  

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

Notable Papers 

Wang C, Cheng H, Dong X, Zhan Y, Liu Y, Wu N, Tang R, He H, Cao Y, Yang L, Ren J, Li X, Li P. Early assessment and treatment of ventricular remodeling in vivo via a targeted ultrasonic molecular probe loaded with oxygen and cholecystokinin. J Nanobiotechnology. 2025 Feb 12;23(1):104. doi: 10.1186/s12951-025-03183-7. PMID: 39939853 

Owusu-Yaw BS. Focused-ultrasound-mediated CRISPR-Cas9 gene editing in human induced pluripotent stem cells. Mol Ther. 2025 Feb 20:S1525-0016(25)00096-6. doi: 10.1016/j.ymthe.2025.02.006. PMID: 39983712 

Lin ZH, Yu QL, Yi BH, Xu WC, He HL, Huang KY, Zheng C, Wu SJ, Lin JF Protective Effects of Low-Intensity Pulsed Ultrasound on Cardiac Electrophysiological Function in a Rat Model of Ischemic Cardiomyopathy. J Am Heart Assoc. 2025 Feb 14:e037402. doi: 10.1161/JAHA.124.037402. Online ahead of print. PMID: 39950540  

Hazel K, Singh D, He S, Guertin Z, Husser MC, Helfield B. Focused ultrasound and microbubble-mediated delivery of CRISPR-Cas9 ribonucleoprotein to human induced pluripotent stem cells. Mol Ther. 2025 Jan 10:S1525-0016(25)00017-6. doi: 10.1016/j.ymthe.2025.01.013. Online ahead of print. PMID: 39797397 

Gumusgoz E, Kasiri S, Youssef I, Verma M, Chopra R, Villarreal Acha D, Wu J, Marriam U, Alao E, Chen X, Guisso DR, Gray SJ, Shah BR, Minassian BA. Focused ultrasound widely broadens AAV-delivered Cas9 distribution and activity. Gene Ther. 2025 Feb 1. doi: 10.1038/s41434-025-00517-w. Online ahead of print. PMID: 39893321 

Ge N, Liu M, Li R, Allen NM, Galvin J, Shen S, O’Brien T, Prendiville TW. Using Ribonucleoprotein-based CRISPR/Cas9 to Edit Single Nucleotide on Human Induced Pluripotent Stem Cells to Model Type 3 Long QT Syndrome (SCN5A±). Stem Cell Rev Rep. 2023 Nov;19(8):2774-2789. doi: 10.1007/s12015-023-10602-5. Epub 2023 Aug 31. PMID: 37653182  

Click here for additional references from PubMed.