Huntington's Disease

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Focused Ultrasound Therapy

Focused ultrasound is a noninvasive, therapeutic technology with the potential to improve the quality of life and decrease the cost of care for patients with Huntington’s disease. This novel technology focuses beams of ultrasound energy precisely and accurately on targets deep in the brain without damaging surrounding normal tissue.

How it Works
Where the beams converge, focused ultrasound produces several types of actions which may be helpful in the treatment of Huntington’s disease. One option is precise ablation (thermal destruction of tissue). A second is temporary opening of the blood-brain barrier, which may allow therapeutic agents to access the brain.

For certain patients, focused ultrasound could provide a noninvasive alternative to medications that would enable symptom relief without the medication-induced side effects.

  • 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.

Clinical Trials

At the present time, there are no clinical trials recruiting patients for focused ultrasound treatment of Huntington’s disease.

Regulatory Approval and Reimbursement

Focused ultrasound treatment for Huntington’s disease is not yet approved by regulatory bodies or covered by medical insurance companies.

Preclinical Laboratory Studies

Preclinical studies are investigating focused ultrasound’s potential to treat the underlying genetic cause of Huntington’s. Focused ultrasound can temporarily open the blood-brain barrier (BBB) to improve the delivery of genes, stem cells, or other neuroprotective and/or neurorestorative drugs that could potentially be important in treating Huntington’s.

Notable Papers

Suggested Reading: Focused Ultrasound: Transforming treatment of Neurodegenerative Diseases August 2022

Lin CY, Tsai CH, Feng LY, Chai WY, Lin CJ, Huang CY, Wei KC, Yeh CK, Chen CM, Liu HL. Focused ultrasound-induced blood brain-barrier opening enhanced vascular permeability for GDNF delivery in Huntington’s disease mouse model. Brain Stimul. 2019 Apr 27. pii: S1935-861X(19)30203-7. doi: 10.1016/j.brs.2019.04.011. [Epub ahead of print] PMID: 31079989

Gutierrez MI, Penilla EH, Leija L, Vera A, Garay JE, Aguilar G. Novel Cranial Implants of Yttria-Stabilized Zirconia as Acoustic Windows for Ultrasonic Brain Therapy. Adv Healthc Mater. 2017 Aug 2. doi: 10.1002/adhm.201700214.

Burgess A, Huang Y, Querbes W, Sah DW, Hynynen K. Focused ultrasound for targeted delivery of siRNA and efficient knockdown of Htt expression. J Control Release. 2012 Oct 28;163(2):125-9. doi: 10.1016/j.jconrel.2012.08.012. Epub 2012 Aug 19.

Click here for additional references from PubMed.

Clinical Trials