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Focused Ultrasound for Pediatric Brain Tumors: Clinical Trial Begins in Toronto

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Key Points Researchers at Sunnybrook Health Sciences Centre and The Hospital for Sick Children are collaborating on a clinical trial using focused ultrasound in patients with diffuse intrinsic pontine glioma (DIPG). DIPGs are highly aggressive and devastating brain tumors that affect young children. In this trial, physicians are exploring focused ultrasound-induced blood-brain barrier opening to enhance delivery of chemotherapy to the tumor. Researchers at Sunnybrook Health Sciences Centre and The Hospital for Sick Children (SickKids) have begun a collaborative clinical trial using focused ultrasound to open the blood-brain barrier (BBB) and deliver chemotherapy in patients with diffuse intrinsic pontine glioma (DIPG). DIPGs are highly aggressive and devastating brain tumors that affect young children, typically aged 5–9. They affect a region of the brainstem that regulates the body’s involuntary activities such as breathing, heart rate, and swallowing. Only 10 percent of children survive past the two-year mark after diagnosis because current therapies are highly ineffective. One reason that brain tumors – including DIPG – are challenging to treat is the BBB. This protective layer of tightly joined cells that lines the blood vessels in the brain prevents harmful substances, such as toxins and infectious agents, from diffusing into the surrounding brain tissue. However, it can also prevent therapeutic agents – like chemotherapy – from getting into the brain. Focused ultrasound has been shown to temporarily disrupt the BBB in a noninvasive, safe, and targeted manner. The ultrasound waves interact with microbubbles in the vessels, causing them to vibrate rapidly and create openings that can enable therapies to pass through. In this trial, researchers are investigating the safety and feasibility of using Insightec’s Exablate Neuro device to open the BBB in 10 participants between the ages of 5 and 18. Participants will undergo focused ultrasound therapy in conjunction with chemotherapy during three treatment cycles, about four to six weeks apart. The first patient underwent the initial procedure earlier this month. The trial is being led by Nir Lipsman, MD, PhD, neurosurgeon and director of Sunnybrook’s Harquail Centre for Neuromodulation, and James Rutka, MD, PhD, director of the Arthur and Sonia Labatt Brain Tumour Research Centre at SickKids.  “DIPG is a devastating pediatric brain tumor which is inoperable due to its location in the brainstem,” says Dr. Lipsman. “Focused ultrasound is an innovative and noninvasive approach to more effectively delivering chemotherapy directly to the tumor. Our hope is that this continued research will bring us closer to enhancing treatments to help change the course of the disease.” Sunnybrook Health Sciences Centre is a Focused Ultrasound Foundation Center of Excellence, and the Foundation is funding a portion of this clinical trial. For PatientsIf you are interested in learning more about this clinical trial, please contact James Rutka, MD, (416-813-6425, james.rutka@sickkids.ca) or Maheleth Llinas (416-480-6100 ext 2476, maheleth.llinas@sunnybrook.ca). Read Sunnybrook’s Announcement >See Coverage by CTV > Additional DIPG Trials in the USFocused ultrasound is being investigated to address DIPG at other sites in the US. At Children’s National Hospital (CNH) in Washington, DC, a team of researchers is conducting two clinical trials. One approach is similar to the Sunnybrook trial, where focused ultrasound will be used to open the BBB in an effort to deliver chemotherapy to the tumor. The other trial at CNH is testing sonodynamic therapy as a way to destroy the tumor. Both clinical trials are currently recruiting patients. CNH became the Foundation’s first pediatric Center of Excellence in 2020. Another DIPG clinical trial investigating focused ultrasound-induced BBB opening to deliver chemotherapy began in July 2021 at Columbia University.
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Focused Ultrasound Now FDA Approved to Treat Essential Tremor Patients’ Second Side

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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.
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Focused Ultrasound for Pancreatic Cancer: Trial Results Prove Safety, Initial Efficacy

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Key Points The data from an initial clinical trial in Korea were critical for establishing safety, preliminary efficacy, and the protocol parameters for a larger study. Jae Young Lee, MD, PhD, used the ultrasound-guided Alpinion focused ultrasound device plus standard-of-care chemotherapy to decrease tumor size in patients with pancreatic cancer. A phase II clinical trial is now underway in Korea and nearing its enrollment goal. Early, proof-of-concept safety data and the protocol parameters from a pancreatic cancer focused ultrasound plus chemotherapy clinical trial have now been published. This small, 9-participant clinical trial, which completed enrollment in 2019, was critical for establishing safety, preliminary efficacy, and the protocol parameters for the phase II clinical trial that is now underway. All participants were pathologically diagnosed with unresectable pancreatic cancer. Jae Young Lee, MD, PhD, professor of Radiology and president at Seoul National University Medical Research Center in Korea, is the principal investigator for both (phase I and II) studies. He and his team are using the ultrasound-guided Alpinion focused ultrasound device and standard-of-care chemotherapy regimens to address pancreatic cancer that is unresectable (which is the case in about 80% of patients with pancreatic cancer). “Pancreatic cancer typically presents with a dense fibrous stroma and low vascularity, which limits drug delivery and efficacy,” said Dr. Lee in the publication. “Therefore, there is an urgent need to develop new treatment options to enhance drug delivery.” Dr. Lee hypothesizes that focused ultrasound can weaken the tumor’s dense stroma to allow chemotherapy to penetrate the cancer cells. As published in European Radiology, the nine participants in the phase I study were assigned to one of three predefined treatment intensity groups (low, intermediate, or high) for six combined treatments of focused ultrasound plus nab-paclitaxel plus gemcitabine chemotherapy. Although it was primarily a safety study, after treatment, the team also measured changes in tumor size, tumor response, tumor marker levels, patient-reported outcomes, and survival. The treatments were safe, and participants experienced no adverse effects. Tumor size decreased more than 15% in seven of the nine participants at both the immediate and 3-month follow-up computed tomography scans. Tumor marker levels decreased in all participants. The intermediate intensity treatment was the most effective, with this group showing the largest decreases in tumor size (more than 30%) and tumor markers plus a significant improvement in survival (p < 0.05). “A key point of the proof-of-concept study is that the Seoul National team showed that they were able to access the patients’ tumors and treat them without complications,” said Tim Meakem, MD, the Foundation’s Chief Medical Officer. “This was not a small issue to solve, because accessing the pancreas with focused ultrasound has been challenging. Some efforts to treat similar patients using MRI guidance have been difficult, simply due to the size and access limitations of the MRI. Dr. Lee used ultrasound guidance for this procedure, which avoided these issues. The safety and initial efficacy of this project were well received by his colleagues, and almost all of the participants for the phase II study have already been enrolled.” The phase II, 60-participant study is being funded by the Focused Ultrasound Foundation. For phase II, the team is using a FOLFIRINOX regimen of chemotherapy. Dr. Lee presented interim phase II results at the 8th International Symposium on Focused Ultrasound. See the Video > See European Radiology > Related StoriesPancreatic Cancer Clinical Trial Begins in Korea October 2021 Research Awards Update: 15 Projects Initiated in the First Six Months of 2021 September 2021
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Parkinson’s Patient Story: Focused Ultrasound Clinical Trial Turns Skeptic into Advocate

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Chuck’s Parkinson’s disease symptoms – including tremors and rigidity – disrupted his personal and professional life. Although Chuck was excited to take part in a focused ultrasound clinical trial, his wife Maureen, a physician, was hesitant. Now, three months after the procedure, Chuck has resumed his work and hobbies and is preparing for his second focused ultrasound treatment.
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