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Key Points Patients with essential tremor can now have their second side treated with focused ultrasound. ET commonly affects both sides of the body, and to date, focused ultrasound was only approved for unilateral treatments. The ruling was based on data that showed a highly significant reduction in tremor following treatment of the second side. The US Food and Drug Administration (FDA) will now allow appropriate patients with essential tremor (ET) to have focused ultrasound treatment on the second side of their brain. ET is the most common movement disorder, affecting an estimated 3% of the population, or approximately 10 million individuals in the US. It is often viewed as a relatively benign disease, but it can have substantial effects on quality of life for many patients. The disorder commonly affects both sides of the body. In July 2016, the FDA cleared Insightec’s Exablate Neuro focused ultrasound device to treat one side of the brain, generally the side that alleviates tremors on the patient’s dominant side. Now, patients who have undergone focused ultrasound treatment on one side can have the second side treated at least nine months after the initial procedure. This “second side” procedure may also be referred to as bilateral treatment. According to an Insightec press release, the ruling was based on data from a study that showed a highly significant reduction in tremor following treatment of the second side. Learn more about that trial > Focused ultrasound is also being investigated in clinical trials for the bilateral treatment of Parkinson’s disease. Read the Insightec Press Release > Meet Tom Tom was thrilled when he was invited to participate in a clinical trial to receive a second focused ultrasound procedure to address the tremor in his non-dominant hand. Tom and his brother Phil talk about their experience with focused ultrasound and their hope that the technology can become more readily available for others, including many in their extended family who have also been affected by ET.

Part 5: Physicians – This blog addresses the challenges in asking physicians to adopt a new medical technology, including hurdles such as payment discrepancies, turf battles, and education.

Key Points EANO held its 17th Annual Meeting from September 15–18 in Vienna.Carthera had a prominent presence at the meeting, hosting a lunch symposium and sharing data from its glioblastoma clinical trial.The liquid biopsy consortium meeting included an overview of the ongoing work with focused ultrasound. The European Association of Neuro-Oncology (EANO) held its 17th Annual Meeting from September 15–18, 2022, in Vienna. In the setting of the Hofburg Imperial Palace, approximately 850 neuro-oncologists, neurologists, neurosurgeons, nurses, and scientists shared and discussed recent findings in basic, translational, and clinical science from all fields of neuro-oncology (brain tumors). French ultrasound manufacturer Carthera sponsored the meeting lanyards and presented a well-attended (350 attendees) corporate lunch symposium with presentations and a panel discussion featuring: Michael Canney, PhDAlexandre Carpentier, MD, PhDAhmed Idbaih, MD, PhDRiccardo Soffietti, MDAdam Sonabend, MDRoger Stupp, MD In the highlights from clinical abstracts session, Dr. Idbaih shared new data from Carthera’s Phase 1/2 clinical trial of blood-brain barrier (BBB) opening with the SonoCloud-9 implantable ultrasound device in participants with recurrent glioblastoma (GBM) receiving IV carboplatin. He summarized the results from the trial, which enrolled 33 participants who received 90 sonications. The procedure was easy to perform in less than 10 minutes, well tolerated, and repeatedly feasible over a large volume. Sonication was shown to improve drug delivery (i.e., a 5.9x increase in carboplatin concentration in a select group of patients) and improve radiological and clinical outcomes when performed at peak concentration of carboplatin. A pivotal randomized trial for recurrent GBM is planned. “Beyond the Carthera presentation, focused ultrasound was mentioned in several of the talks, especially for drug delivery,” said Suzanne LeBlang, MD, the Foundation’s Director of Clinical Relationships, who attended the meeting. “Even researchers who do not currently use focused ultrasound were describing how it could impact the field by delivering different substances to the brain.” Hideho Okada, MD, PhD, and Nino Chiocca, MD, PhD, presented information on immunotherapy approaches and suggested that innovative strategies, such as convection–enhanced delivery and ultrasound with microbubbles, can help deliver high molecular weight agents that cannot cross the BBB alone. A dedicated session on “Liquid Biomarkers for Diagnosis/Disease Tracking in CNS Tumors” presented many new advances for the field. “I was appreciative for the invitation to also participate in a separate liquid biopsy (LB) consortium meeting led by Susan Short, MD, PhD, where I presented an overview of focused ultrasound–enhanced LB,” Dr. LeBlang added. The LB consortium will reconvene for another progress update during the Society of Neuro-Oncology meeting in November. In another session, Sabine Mueller, MD, PhD, MAS, delivered the presentation “Clinical Trials – A Transatlantic Perspective,” in which she discussed the Focused DMG Consortium to assess the use of focused ultrasound in diffuse intrinsic pontine gliomas. Participating institutions include Children’s National Hospital in Washington DC, Prinses Maxima Centrum, University Kinderspital Zurch, Virginia Tech, and UCSF. EANO showcased more than 370 oral and poster presentations. The following abstracts, which address the treatment of GBM, are of interest to the focused ultrasound community: KS05.6.A Oral DNA vaccination targeting VEGFR2 combined with the anti-PD-L1 antibody avelumab in patients with progressive glioblastoma – final results. NCT03750071 Dr. LeBlang reports: In this presentation, Professor Michael Platten, MD, described how focused ultrasound could be used to activate T cells and make the tumor microenvironment “hot.” The resulting discussion focused on the tumor microenvironment and the role of cytokines. OS07.3.A Phase 1/2 clinical trial of blood-brain barrier opening with the SonoCloud-9 implantable ultrasound device in recurrent glioblastoma patients receiving IV carboplatin. Dr. LeBlang reports: In this clinical trial, 33 participants were implanted with the SonoCloud-9 device and received sonications with IV carboplatin for a total of 90 sonications. Adverse events included two wound infections and two cerebrospinal fluid leaks. The study showed that chemotherapy uptake was 5.9 times higher in the treated area of BBB opening. In a cohort that received IV carboplatin just prior to sonication (n=12 patients) a 1-yr OS of 58% was reported. P14.05.B Plasma-EV based liquid biopsy for precision medicine in the treatment of glioblastoma Dr. LeBlang reports: In this study, the group isolated EV from 2 mL of blood plasma and found the RNA and DNA to mirror the genomic profile of the parental tumor. The group concluded that “Plasma-EV based liquid biopsy could implement the personalization of GBM care for every timepoint of the disease course.” The Foundation sponsored a booth at the meeting where many attendees stopped by to learn more about the technology, manufacturers, our organization, and the larger research community. Dr. LeBlang encourages more focused ultrasound manufacturers to attend future EANO meetings! See the Meeting Abstracts >

Part 4: Reimbursement – Reimbursement includes all the steps necessary to get paid by insurance payors for using a technology. All parties in a technology company – management, board, investors, employees – should understand the true requirements for a successful reimbursement strategy. While there are exceptions to every rule, the process generally flows in a consistent direction from regulatory through coding to assignment of payment to determining coverage, with the final step being payors paying (or not) the physician and/or facility.