Pivotal Study Results Published in New England Journal of Medicine (NEJM) Confirm Safety and Efficacy of INSIGHTEC's Exablate Neuro System for the Treatment of Essential Tremor
HAIFA, Israel, Aug. 25, 2016 /PRNewswire/ -- INSIGHTEC, the leader in MR-guided Focused Ultrasound (MRgFUS) therapy, today announced that the New England Journal of Medicine (NEJM) has published the results of a pivotal study of their Exablate Neuro system for the non-invasive treatment of essential tremor (ET). The study met its primary endpoint with patients treated with Exablate Neuro demonstrating a clinically significant 47% improvement in a composite tremor score at three months as compared to no change in the sham cohort.
The NEJM article presents findings from an FDA study designed to evaluate the safety and efficacy of Exablate Neuro for the treatment of essential tremor in subjects for whom medications were not effective. Results of this randomized, double-blind, multi-center clinical study were submitted as the key data resulting in FDA approval of INSIGHTEC's Exablate Neuro as the first focused ultrasound device to treat essential tremor.
"MRI imaging and thermometry provide a high level of control during focused ultrasound thalamotomy. Importantly, before treatment, adjustments can be made based on intraoperative imaging and clinical feedback from the patient, contributing to a safe procedure," said Dr. W. Jeffrey Elias, Director of Stereotactic and Functional Neurosurgery at the University of Virginia and primary author of the NEJM paper.
Seventy-six patients were enrolled in the study and randomly assigned to receive the Exablate treatment (56 patients) or the sham procedure (20 patients), the exact same procedure but without transmission of ultrasound energy. Patients in the placebo treatment arm were later allowed to undergo an Exablate Neuro treatment.
"The robust study findings showed that focused ultrasound safely improved hand tremor in patients with refractory tremor by precisely targeting and ablating deep brain tissue without surgical incisions, thus minimizing risk of infection, bleeding or other complications sometimes seen in surgical operations," said Dr. William G. Ondo, Tremor Research Group President.
The primary endpoint combined the Clinical Rating Scale for Tremor (CRST) Part-A and Part-B, an 8-element measure of tremor and hand function. For the Exablate Neuro treatment group, a mean score of 18.1±4.8 at baseline was reduced to 9.6±5.1 at 3 months (47% change), while the sham group showed no change, demonstrating a highly statistically significant (p<0.001) difference between groups.
Patients in the treatment group showed a 63.4% reduction in tremor amplitude alone (CRST A) which was maintained at one year follow up. Additionally, the total disability score (CRST part C) improved by 68% and patient assessment of their quality of life (QUEST) improved by 51% overall at 12 months for treated patients.
Most adverse events were transient, minimal or unrelated to the Exablate Neuro procedure.
"We are pleased with the results of this pivotal study that addresses a major issue for the five million Americans and millions more worldwide that suffer from this debilitating disease," said Maurice R. Ferré MD, INSIGHTEC Chief Executive Officer and Chairman of the Board. "With this positive data and the subsequent FDA approval of the Exablate Neuro system, patients can be treated and return to performing everyday tasks with greater ease and enjoy a better quality of life."
The sites participating in the clinical study included: University of Virginia, Stanford University Medical Center, University of Maryland, Brigham and Women's Hospital, Swedish Medical Center, Yonsei University Medical Center (South Korea), Sunnybrook Health Sciences Centre (Canada) and Tokyo Women's Medical University (Japan).
Funding for the study was provided by the Focused Ultrasound Foundation and BIRD (US-Israel Binational Industry R&D) and INSIGHTEC.
Exablate Neuro has now received FDA, Health Canada and Korean approval for essential tremor and CE mark in Europe for essential tremor, tremor dominant Parkinson's disease and neuropathic pain.
About Exablate Neuro
Exablate Neuro uses high intensity focused ultrasound waves to ablate targeted tissue through an intact skull with no incisions. During planning and treatment, the patient is fully conscious lying on the treatment bed in an MRI scanner. MRI provides high resolution visualization, patient-specific treatment planning and continuous real-time monitoring of the procedure. Focused ultrasound technology has been successfully used to treat other clinical indications including uterine fibroids and painful bone metastases.
HAIFA, Israel, Aug. 25, 2016 /PRNewswire/ -- INSIGHTEC, the leader in MR-guided Focused Ultrasound (MRgFUS) therapy, today announced that the New England Journal of Medicine (NEJM) has published the results of a pivotal study of their Exablate Neuro system for the non-invasive treatment of essential tremor (ET). The study met its primary endpoint with patients treated with Exablate Neuro demonstrating a clinically significant 47% improvement in a composite tremor score at three months as compared to no change in the sham cohort.
The NEJM article presents findings from an FDA study designed to evaluate the safety and efficacy of Exablate Neuro for the treatment of essential tremor in subjects for whom medications were not effective. Results of this randomized, double-blind, multi-center clinical study were submitted as the key data resulting in FDA approval of INSIGHTEC's Exablate Neuro as the first focused ultrasound device to treat essential tremor.
"MRI imaging and thermometry provide a high level of control during focused ultrasound thalamotomy. Importantly, before treatment, adjustments can be made based on intraoperative imaging and clinical feedback from the patient, contributing to a safe procedure," said Dr. W. Jeffrey Elias, Director of Stereotactic and Functional Neurosurgery at the University of Virginia and primary author of the NEJM paper.
Seventy-six patients were enrolled in the study and randomly assigned to receive the Exablate treatment (56 patients) or the sham procedure (20 patients), the exact same procedure but without transmission of ultrasound energy. Patients in the placebo treatment arm were later allowed to undergo an Exablate Neuro treatment.
"The robust study findings showed that focused ultrasound safely improved hand tremor in patients with refractory tremor by precisely targeting and ablating deep brain tissue without surgical incisions, thus minimizing risk of infection, bleeding or other complications sometimes seen in surgical operations," said Dr. William G. Ondo, Tremor Research Group President.
The primary endpoint combined the Clinical Rating Scale for Tremor (CRST) Part-A and Part-B, an 8-element measure of tremor and hand function. For the Exablate Neuro treatment group, a mean score of 18.1±4.8 at baseline was reduced to 9.6±5.1 at 3 months (47% change), while the sham group showed no change, demonstrating a highly statistically significant (p<0.001) difference between groups.
Patients in the treatment group showed a 63.4% reduction in tremor amplitude alone (CRST A) which was maintained at one year follow up. Additionally, the total disability score (CRST part C) improved by 68% and patient assessment of their quality of life (QUEST) improved by 51% overall at 12 months for treated patients.
Most adverse events were transient, minimal or unrelated to the Exablate Neuro procedure.
"We are pleased with the results of this pivotal study that addresses a major issue for the five million Americans and millions more worldwide that suffer from this debilitating disease," said Maurice R. Ferré MD, INSIGHTEC Chief Executive Officer and Chairman of the Board. "With this positive data and the subsequent FDA approval of the Exablate Neuro system, patients can be treated and return to performing everyday tasks with greater ease and enjoy a better quality of life."
The sites participating in the clinical study included: University of Virginia, Stanford University Medical Center, University of Maryland, Brigham and Women's Hospital, Swedish Medical Center, Yonsei University Medical Center (South Korea), Sunnybrook Health Sciences Centre (Canada) and Tokyo Women's Medical University (Japan).
Funding for the study was provided by the Focused Ultrasound Foundation and BIRD (US-Israel Binational Industry R&D) and INSIGHTEC.
Exablate Neuro has now received FDA, Health Canada and Korean approval for essential tremor and CE mark in Europe for essential tremor, tremor dominant Parkinson's disease and neuropathic pain.
About Exablate Neuro
Exablate Neuro uses high intensity focused ultrasound waves to ablate targeted tissue through an intact skull with no incisions. During planning and treatment, the patient is fully conscious lying on the treatment bed in an MRI scanner. MRI provides high resolution visualization, patient-specific treatment planning and continuous real-time monitoring of the procedure. Focused ultrasound technology has been successfully used to treat other clinical indications including uterine fibroids and painful bone metastases.
