Parkinson’s disease is an increasingly common nervous system disorder, with a growing list of high profile patients who are helping to lead the fight for better treatment options and creating hope for a cure.Focused ultrasound is an early-stage, noninvasive therapy that is now in clinical trials to treat the motor symptoms of Parkinson’s. The Focused Ultrasound Foundation has funded and been involved with many clinical trials over the past 12 years, including a successful effort to find a treatment for essential tremor (ET), which is a movement disorder with symptoms similar to Parkinson’s disease. Our experience with ET has provided a base of knowledge that may be helpful as patients consider participating in a new clinical trial for Parkinson’s disease.
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Today at the largest and most influential international meeting dedicated to advancing dementia science – the Alzheimer’s Association International Conference (AAIC) in Chicago – thousands of attendees from around the world were among the first to hear the results of a landmark clinical trial of focused ultrasound to open the blood-brain barrier (BBB) to facilitate drug therapy in patients with Alzheimer’s disease. These pilot trial results were also published today in the prestigious, high-impact journal, Nature Communications.
Nir Lipsman, MD, PhD, of Sunnybrook Health Sciences Centre in Toronto, presented the findings of this first-of-its-kind study – Blood-Brain Barrier Opening in Alzheimer’s Disease Using MR-guided Focused Ultrasound – that demonstrated the feasibility and preliminary safety of focally, reversibly and repetitively opening the BBB. This is the first small, but critically important, step in a process that could potentially lead to a novel approach to delivering drugs to the brain to treat Alzheimer’s disease.
Recently, founder and chairman of the Foundation Neal F. Kassell, MD, spoke with Melanie Crandall, head of content at tech communications firm Red Lorry Yellow Lorry, to discuss why CES 2018 was such a great venue to showcase focused ultrasound technology and where he thinks the field is headed, particularly in regards to the treatment of essential tremor.
You were recently at CES with John Grisham. Was this your first time at the show and how was the technology received?Yes,it was our first time. We were somewhat anxious going into this, as we are definitely not consumer electronics. But our expectations of a positive reception were wildly exceeded. The response from show attendees was nothing short of astonishing. People came by the booth eager for more information and expressing enthusiasm about the potential of the technology. Attention from the senior leadership of CES was amazing. The panels we were part of and participating in Gary’s Book Club with John Grisham were incredible experiences. On a scale of 0 to 10, I would rate it a 16.
Consumer Technology Association Discusses Emerging Technology and John Grisham's The Tumor with Neal Kassell
[Adapted from Consumer Technology Association's Blog, Dec. 18, 2017]
Technology truly has the power to change the world and in the medical field, this is being proven time and time again. Dr. Neal F. Kassell is a neurosurgeon who is championing a new medical technology called "focused ultrasound," which is just one example of how science and technology is revolutionizing healthcare.
The “science of patient input” and “patient engagement” have been hot topics recently in discussions about the future of our biomedical innovation ecosystem. Patient organizations have long promoted the idea that patients should be partners with industry as they develop new therapies, that their perspectives and insights are valuable throughout the pathway from bench to bedside. We are also seeing new legislation, including the 21st Century Cures Actand the Prescription Drug and Medical Device User Fee Acts (PDUFA, MDUFA), capture these sentiments by encouraging an increased patient role in the regulatory process. The concept is also starting to catch on in industry with many device and drug companies creating new positions dedicated to patient engagement efforts. But there is still a lot of work ahead to develop practical methodologies for utilizing patient input and to show the return on investment.
At the Focused Ultrasound Foundation (FUSF), an important part of our mission includes identifying, educating, and mentoring future focused ultrasound clinicians and scientists. With this goal in mind, several years ago we launched our International Research Fellowship Program. Today this program includes internship and fellowship opportunities for the best minds in the field, both in the US and abroad.
The Foundation’s International Research Fellowship Program began in the summer of 2012 with our first visiting scholar, CNRS-funded research professor Jean-François (Jeff) Aubry, PhD, who took a year-long sabbatical from the Institut Langevin, Espci Paris to join FUSF. In his fellowship role, Jeff collaborated on research projects with both Foundation staff and researchers at the University of Virginia (UVA). His projects ranged from thermal dose predictive modeling to head phantom development for acoustic testing. Upon his return to Paris, Jeff was elected as president of the International Society for Therapeutic Ultrasound for a three-year term, and he continues to visit Charlottesville to advance ongoing collaborative research projects. It was clear to us after our very productive experience with Jeff that we had identified a crucial unmet need by offering this fellowship as a unique opportunity for inter-institutional collaboration in the field of focused ultrasound.
Innovation is the driving force for biomedical research as we strive for health care that is safer, more effective and of greater value. Continued innovation requires collaboration, particularly in this time of limited resources, global competition and long timelines from bench to bedside. The biomedical community is recognizing the need to break down the silos that impede progress, and foundations – including the Focused Ultrasound Foundation – are realizing a need for quick and widespread dissemination of research results that will forge new partnerships among multiple stakeholders including researchers, clinicians, industry, government and patients.
Like many of you, all of us at the Focused Ultrasound Foundation were surprised and saddened to learn of Senator John McCain’s recent glioblastoma (GBM) diagnosis. GBM and other brain tumors are a major focus of our research here at the Foundation, and unfortunately we are all too familiar with the devastating effects of this disease on patients and their families. Our thoughts and well wishes are with the McCains and we sincerely hope he will be one of the lucky ones to survive for many years to come.
When news of the Senator’s diagnosis became public, we were flooded with inquiries about whether focused ultrasound (FUS) could treat his tumor. Unfortunately the short answer, at the present time, is no. Focused ultrasound is still in the early stages of development for brain tumors. We hope and expect, however, that the technology will be available in the not too distant future as an effective, noninvasive alternative or complement to traditional GBM treatments to improve quality and longevity of life in these patients.
Focused ultrasound is a powerful technology poised to play a unique role towards achieving the vision of the Cancer Moonshot. This early stage, noninvasive, therapeutic technology has the potential to improve the quality and longevity of life and decrease the cost of care for certain patients with cancer by serving as an alternative or complement to each of the four pillars of cancer treatment: surgery, radiation therapy, chemotherapy, and immunotherapy. The Focused Ultrasound Foundation is dedicated to bringing this new therapy to patients as quickly as possible.
The Moonshot initiative has emphasized the need to break down silos that are impediments to progress in cancer research in order to promote collaboration and data sharing among multiple stakeholders including researchers, clinicians, industry, government and patients. The Foundation works hard to do just that with the growing community active in focused ultrasound research.