Dr. Elias presenting the pivotal study design at the Focused Ultrasound Symposium
InSightec has completed enrollment in the pivotal study of their ExAblate Neuro system for the treatment of essential tremor. Physicians at eight sites worldwide have now treated 76 patients in the trial. The first randomized, controlled trial of unilateral MR-guided focused ultrasound thalamotomy with InSightec’s system began in August 2013 and completed enrollment in September 2014. Results will be submitted to the US Food and Drug Administration (FDA) for regulatory approval of the device. The pivotal study was designed based on the clinical outcome of the initial 15-patient pilot study that was conducted from 2011 to 2012 and published in an August 2013 issue of the New England Journal of Medicine.
"We all know someone with essential tremor; it's a common problem. Hopefully this study will lead to a treatment that might be applicable to many patients around the world." – Jeff Elias, MD, associate professor of neurological surgery at the University of Virginia and principal investigator of the study
Patients with severe, medication-refractory essential tremor were enrolled after being assessed for tremor severity and disability and then randomized in a one-to-three fashion to receive either sham (pretend) procedure or the focused ultrasound treatment. Participating patients are now being followed for one year.
Treatment safety and efficacy are the study’s primary endpoints, with secondary endpoints including durability at 12 months, quality of life, functional improvement, and comparison between the sham and treatment groups. During the follow-up period, an independent, blinded corelab group of neurologists specializing in the management of essential tremor rate the clinical outcomes from videotaped assessments, and the patients will complete disease-specific quality of life questionnaires. Final results are expected to be submitted to the FDA by the end of 2015.
"As long as you capitalize on change and are ahead of the curve, you can succeed." – Tomas Puusepp
Elekta’s Executive Director Shares Insight, Praises the Role of the Focused Ultrasound Foundation
Tomas Puusepp, Executive Director of the Board of Elekta and its former CEO, recently visited the Foundation and shared his experience in providing an emerging technology (the Gamma Knife) to the patients who need it. His thoughts on taking risks, competition, and overcoming barriers provide many valuable insights that apply to focused ultrasound.
When Puusepp joined Elekta in 1988, it was a $5 million company with a staff of 22—today it has $1.5 billion in revenue and 4,000 employees. With a background in electrical engineering and physics, he advanced through the management ranks within Elekta to serve as President and CEO from 2005 through April 2014.
Puusepp recognizes the benefit of a foundation in supporting technology. "It took decades for the Gamma Knife to become a standard of care. However, after we established the Leksell Gamma Knife Society, we gained traction much faster. If an organization similar to the Focused Ultrasound Foundation existed for advancing stereotactic radiosurgery from the start, this time would have been much shorter."
Learn more about the parallels between the path of the Gamma Knife and the development of focused ultrasound, his top three tips for device companies, and his praise for the role of the Foundation.
Council Co-Chair Jane Batten Hosts Virginia Beach Awareness Event
On November 3rd, Foundation Council co-chair Jane Batten and Vince Mastracco hosted a luncheon in Virginia Beach for 25 community leaders who were interested in learning about focused ultrasound and its potential to revolutionize medicine, save lives, and reduce suffering. During the program, Chairman Neal F. Kassell, MD, presented the Foundation’s history and a brief overview of the technology. A focused ultrasound patient then shared his story with the group.
The Winter School explores the rapidly emerging field of therapeutic ultrasound. Participants will learn the physics behind the technology, the biophysical effects in the body, and the clinical applications of the therapy.
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FUS Experts Believe Brain and Cancer Applications Have Most Potential to Improve QOL
The Foundation conducted a survey of 150 members of the focused ultrasound community on the state of the field prior to the October 2014 Symposium.
Respondents were asked to identify which clinical indications were most likely to improve patients’ quality of life. Neurological and oncological indications are tied for the most votes, with brain tumors, essential tremor, and pancreatic cancer being rated as the top three individual indications. Similarly, respondents were asked to rank the top three bioeffects that are most likely to have long-term therapeutic value. Thermal ablation is ranked first, followed by drug delivery and neuromodulation.
Read more to find out about the community’s perspective on:
Which indications are most likely to be commercially successful
What the obstacles are to widespread use
Whether there are any differences between scientist and clinician views on the potential of FUS
Focused Ultrasound Research Broadly Recognized at the 10th Interventional MRI Symposium in Germany
A research group based at Kobe University in Japan received the magna cum laude poster award at the 10th Interventional MRI Symposium held last month in Leipzig, Germany. Their winning project featured work in analyzing liver deformation to obtain MR images prior to focused ultrasound (FUS) treatment. Of the 63 posters accepted at this technically oriented meeting, seven included FUS.
CarThera Opens the Blood-Brain Barrier with Ultrasound
Although they do not use focused ultrasound, one of the developmental companies that presented at last month’s Symposium is using low intensity ultrasound waves to temporarily open the blood-brain barrier (BBB) to allow drug delivery to brain tumors. CarThera researchers gained attention after reporting that they initiated a Phase 1/2a clinical trial to open the BBB in recurrent glioblastoma patients last July.
At the time of the presentation, four patients had been enrolled in the study. A tiny ultrasound transducer device is inserted into a standard burr hole, fixed to the surrounding skull bone, and covered with skin. The energy is delivered once a month when the device is connected to an external generator system via transdermal needle. The patient receives up to two minutes of pulsed ultrasound. At the same time, an ultrasound contrast agent is given and BBB disruption is monitored via MRI. As the BBB is opening with ultrasound, the chemotherapy is administered.
Three interesting papers have recently been published about the use of focused ultrasound (FUS) in the brain. Could 3D MR thermometry play a key role in understanding and expanding the FUS treatment envelope? Does skull thickness matter? What parameters are needed to begin using low-frequency FUS in the brain?
Read these manuscripts, and one about liver disease clinical trials. READ MORE ►
This work from Switzerland supports the potential use of transcranial focused ultrasound for the noninvasive treatment of patients suffering from malignant brain tumors, especially in areas not amenable to conventional neurosurgical interventions.
Further treatments in the context of this ongoing pilot study will be needed to assess the feasibility and safety of the procedure. If the results are positive, then a larger study in the future will be needed to demonstrate safety and efficacy.
EDAP-TMS received a letter from the US Food and Drug Administration (FDA) indicating that, although their premarket application for the Ablatherm device to treat localized prostate cancer was not approvable in its current form, a path forward is possible.
The letter provides specific guidance and recommendations for modifying the patient population to those prostate cancer patients with a greater risk of morbidity and/or mortality from their disease and for using the company's European registry along with the already existing safety data to support approval for the modified indication. EDAP’s press release provides additional details.
"This response from the FDA reflects the extensive and ongoing discussions between our team and agency staff…We appreciate their constructive and comprehensive recommendations." – EDAP CEO Marc Oczachowski
Mark Carol, MD
Mark Carol Named CEO and President of SonaCare Medical
SonaCare Medical has named Mark Carol, MD, CEO and President. According to the company’s press release, Carol plans to focus on further developing SonaCare’s HIFU technology while overseeing commercialization, manufacturing, and finance.
Dr. Carol, who joined SonaCare in 2011 as Chief Development Officer, has led the company’s clinical study program and the ongoing effort for US regulatory approval of the Sonablate 450.