InSightec Ltd, the global leader in MR guided focused ultrasound, announced that the first patient has been treated as part of an ExAblate Neuro Phase III pivotal trial. The trial is a multicenter, double-blinded, randomized study to evaluate the efficacy and safety of treatment using the ExAblate Neuro in medication-refractory essential tremor patients. The study builds on promising pilot studies demonstrating the preliminary safety and effectiveness of MR guided focused ultrasound technology in treating target areas deep inside the brain, and published in the New England Journal of Medicine and The Lancet Neurology.
ExAblate Neuro combines focused ultrasound and magnetic-resonance imaging (MRI) to ablate a tiny portion of the brain circuit responsible for the tremor. Over one thousand ultrasound waves are focused simultaneously on a single site in the thalamus for the treatment -- much like a magnifying glass can generate heat by focusing light. Because sound waves can pass through the skull, there's no need for scalpels or anesthesia. The real-time MR images allow the surgeon to target the sound waves very precisely and also provide continuous guidance and thermal feedback throughout the procedure.
In this study 72 patients will be enrolled in up to eight centers around the world and randomized to either an ExAblate Neuro or sham (no) treatment. All patients will be assessed at 6 and 12 months, and followed, as directed by their doctor, for up to 5 years. The results of this trial are expected to support a submission of the ExAblate Neuro to the FDA for Pre-Market Approval (PMA).
"This study is an important step toward providing a unique, non-invasive alternative treatment for patients who are severely disabled by essential tremor, a very common movement disorder," said Eyal Zadicario, Vice President of R&D and Director of InSightec's Neuro program. "The Phase I studies showed that patients experienced immediate and durable symptom improvement with a high safety profile. We expect that the results of this Phase III trial will demonstrate similar long term results and open the door of this technology to other applications in the brain."
The BIRD (US-Israel Binational Industry R&D) Foundation, the Focused Ultrasound Foundation and InSightec are partnering in a unique public-philanthropy-industry collaboration to support this clinical trial.
BIRD promotes collaboration between Israeli and American entities in various technological fields for the purpose of joint product development and will provide partial funding. The Focused Ultrasound Foundation, which supported the initial clinical studies for essential tremor, will organize and fund investigator meetings, as well as the trial's Data and Safety Monitoring Board, maintenance of the video core lab and certain patient costs. InSightec will provide regulatory, clinical and technical support for the trial.
"Focused ultrasound is an alternative to surgery with the potential for less risk of hemorrhage, infection and brain damage," said Dr. Neal Kassell, chairman of The Focused Ultrasound Foundation. "We hope this pivotal trial will demonstrate safety and long-term efficacy, leading to the regulatory approval of this non-invasive technology to help improve quality of life for people suffering from essential tremor. The Foundation is dedicated to accelerating the development of focused ultrasound for the brain and to building innovative collaborations to rapidly advance more effective and less costly treatments for patients with unmet medical needs."
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