Focused Ultrasound Therapy
Focused ultrasound is an early-stage, non-invasive, 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. Where the beams converge, the ultrasound produces precise ablation (thermal destruction of tissue) enabling non-invasive treatment of the movement disorders associated with Huntington’s disease. It is important to note that this does not treat the underlying genetic cause of Huntington’s disease. However, it can lessen the severity of the motor symptoms which can be very debilitating.
For certain patients, focused ultrasound could provide a non-invasive alternative to medications that would enable symptom relief without the medication-induced side effects.
A clinical trial is assessing the use of focused ultrasound to treat the movement disorders in patients with Huntington’s disease. Please note that this trial is only open to residents of Canada.
For a full list of Huntington’s disease clinical trials with focused ultrasound, please see here.
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.
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.