A novel pivotal trial validating the use of focused ultrasound to treat the cardinal features of Parkinson’s disease has been published in the New England Journal of Medicine.
In “Randomized Trial of Focused Ultrasound Subthalamotomy for Parkinson’s Disease,” Professor José Obeso and his team at HM CINAC, a part of Hospital Universitario Puerta Del Sur in Madrid, Spain, collaborated with W. Jeffrey Elias, MD, at the University of Virginia School of Medicine. Both groups used Insightec’s Exablate Neuro focused ultrasound device to noninvasively ablate a portion of the brain called the subthalamic nucleus (STN). The STN is a proven neurosurgical target for treating the main movement disorder features of Parkinson’s disease: tremor, bradykinesia, rigor, and postural instability.
“Prof. Obeso and his team are leaders in focused ultrasound research and Parkinson’s disease,” says Foundation Chairman Neal F. Kassell, MD. “This study is an important milestone in the evolution of focused ultrasound as it becomes a standard of care for treating a wide variety of serious medical disorders.”
Previously, the Madrid team completed a pilot study treating the STN and the results were published in Lancet Neurology (Martinez-Fernandez et al., Lancet Neurology, January 2018).
The current pivotal study began in March 2018, and the investigators enrolled 40 patients with markedly asymmetric Parkinson’s symptoms through May 2019. The double-blind study investigated the safety and efficacy of focused ultrasound to treat the principle motor features of Parkinson’s disease. Dr. Martinez-Fernandez et al. concluded that focused ultrasound subthalamotomy in one hemisphere improved the asymmetric motor features of Parkinson's disease. The adverse events, which were mostly minor and temporary, were similar to other techniques and included speech and gait disturbances, weakness on the treated side, and dyskinesia.
“The role of the STN in the pathophysiology of Parkinson´s disease has been well known for three decades now, but the need for surgery (i.e., mainly deep brain stimulation) limited treatments to patients with severe complications,” said Professor José Obeso, MD, PhD, a neurologist and the trial’s principal investigator. “Our study shows that using focused ultrasound, a far less invasive technique, allows us to target the STN and restore motor function. Adverse events are always a limitation and worrisome aspect of newer therapies. There is a learning curve and an optimization process alike any other new treatment. In the future, subthalamotomy may well be applicable earlier in the evolution of Parkinson's disease, thus more definitively impacting disease evolution.”
Importantly, this study confirms that focused ultrasound can successfully target the STN and offers patients a new treatment choice. Besides providing a noninvasive way to treat the cardinal signs and improve the quality of life in patients with Parkinson’s disease without surgery or radiation, focused ultrasound has the advantages of being available to treat older patients that are not good candidates for more invasive treatment options and will also likely allow patients to be treated earlier in the evolution of the disease.
The pivotal trial was funded by Insightec, the Focused Ultrasound Foundation, Fundación MAPFRE, Fundación Hospitales de Madrid, and the University of Virginia Focused Ultrasound Center of Excellence.
An associated editorial about this study by Joel S. Perlmutter, MD, and Mwiza Ushe, MD, describes the current standard of care for Parkinson’s Disease and how focused ultrasound could add a new dimension to improve options for patients. See Parkinson’s Disease – What’s the FUS? in the New England Journal of Medicine.