On Wednesday, August 12, the Focused Ultrasound Foundation and the Children’s Tumor Foundation held a webinar and virtual panel discussion that introduced focused ultrasound to the neurofibromatosis (NF) research community.

A series of brief presentations by experts in NF or focused ultrasound aimed to determine if and how focused ultrasound could be investigated as a therapy for neurofibromatosis type 2 (NF2) patients. 

See below for an overview of each presentation:

headshot KALAMARIDES Michel sqMichel Kalamarides, MD, PhD
Professor of Neurosurgery, Pitié-Salpetrière Hôpital, Paris, France

Dr. Michel Kalamarides presented an overview of the benign tumors that occur in NF2, including vestibular schwannomas, meningiomas, and spinal cord tumors (schwannomas, meningiomas and ependymomas). Currently, surgical resection of these tumors is the primary treatment option if an intervention is necessary. The challenge with surgical resection of these tumors, especially vestibular schwannomas, is preservation of the normal brain, nerve, and spinal cord tissue. Within the past decade, a medication called Bevacizumab (Avastin) has also been approved for the treatment of vestibular schwannomas and has shown to control tumor growth and preserve patient’s hearing.  However, there are negative side effects associated with Avastin, including kidney toxicity with prolonged usage of this medication. Thus, alternative therapies for these benign tumors are needed.

headshot McDannold Nathan sqNathan McDannold, PhD
Research Associate, Brigham and Women’s Hospital, Boston, MA
Professor of Radiology, Harvard Medical School, Boston, MA

Dr. Nathan McDannold introduced focused ultrasound for brain applications. In particular, he highlighted focused ultrasound-induced blood-brain barrier (BBB) opening and non-thermal ablation of brain tissue using microbubbles.

headshot Tyrone Porter sqTyrone Porter, PhD
Professor of Biomedical Engineering, University of Texas at Austin, Austin, TX

Dr. Tyrone Porter highlighted his work in focused ultrasound combined with nanoparticle injection to achieve both non-thermal tumor ablation and enhanced drug delivery for the treatment of cancer.

headshot Kim AeRang sqAeRang Kim, MD, PhD
Associate Professor of Pediatrics, Solid tumors faculty member, Children’s National Hospital, Washington, DC

Dr. AeRang Kim shared her experience in the treatment of pediatric solid tumors using MR-guided focused ultrasound alone and in combination with ThermoDox.


Discussion
The subsequent discussion and Q&A period allowed the panelists to begin to brainstorm how focused ultrasound may benefit NF2 patients. It was determined that the best preclinical pathway forward is to study focused ultrasound effects on meningiomas and vestibular schwannomas using both non-thermal ablative and enhanced drug delivery techniques. The Foundation looks forward to continued collaboration with the Children’s Tumor Foundation and NF scientists to move research forward and eventually improve the lives of NF patients using focused ultrasound.



About the Participants

Jessica Foley, PhD
Dr. Jessica Foley joined the Focused Ultrasound Foundation in 2012 after a more than 11-year career in the field of focused ultrasound that spanned academic, industrial and government worlds. As Chief Scientific Officer, Foley guides the strategy, development, and implementation of the scientific and research programs and activities within the Foundation, and aligns these with communications and development efforts. She also leads alliance-building efforts with external stakeholders including (but not limited to) governmental policymakers, regulatory agencies, and disease-specific foundations whose interests are complementary to those of the Foundation. She is a representative and advocate for the Foundation and the focused ultrasound community among a wide variety of stakeholders. Her experience prior to the Foundation included several years with InSightec as the Neuro Projects Manager and Clinical Marketing Manager; Senior Scientist at Medtronic; and serving as a 2011-2012 AAAS Science and Technology.

Michel Kalamarides, MD, PhD
Prof. Michel Kalamarides is a Professor of Neurosurgery at Pitié-Salpetrière Hôpital (Faculté de Medécine, Sorbonne- Université) in Paris France. He is specialized in vestibular schwannoma and meningioma surgery. Within a large INSERM unit-U 1127 at Brain and Spine Institute (Pitié-Salpetrière), he is leading a research group on meningioma, particularly using mouse models to dissect the mechanisms of meningioma initiation and progression. Prof. Kalamarides is leading the French NF2 reference clinical center and the French NF2 network. With Prof. Olivier Sterkers, he organized the first NF2 state of the Art meeting in 2006 in Paris. He was the co-chair of the 2018 Joint Global Neurofibromatosis conference held in Paris.

AeRang Kim, MD, PhD
Dr. AeRang Kim is an associate professor of pediatrics and a solid tumors attending in the Oncology Division at Children's National Hospital in Washington, DC. Her research has focused on the development of novel therapeutics for pediatric cancer, sarcomas and NF1 associated tumors. She oversees the development of the novel device of high-intensity focused ultrasound for pediatric cancer applications and leads multiple phase I/II trials in pediatric oncology, sarcomas, and NF1.

Nathan McDannold, PhD
Dr. Nathan McDannold is a Research Associate at Brigham and Women’s Hospital and a Professor of Radiology at Harvard Medical School. His research has focused on brain applications, drug delivery and neurodegenerative diseases. A medical physicist, he has been a participant in the care of every focused ultrasound patient treated at BWH since 1996.

Tyrone Porter, PhD
Dr. Tyrone Porter is a Professor of Biomedical Engineering at the University of Texas at Austin. He completed his graduate work in the Department of Bioengineering at the University of Washington in 2003. He was awarded the Frederick V. Hunt Postdoctoral Fellowship and the R. Bruce Lindsay award from the Acoustical Society of America (ASA) in 2003 and 2008, respectively, and in 2017 he was inducted as a Fellow of the ASA. Dr. Porter's research interests lie at the intersection between biomedical ultrasound, materials science, biophysics, and nanomedicine. Under his leadership, the Nanomedicine and Medical Acoustics Laboratory (NanoMedAL) has made significant contributions to the use of ultrasound for noninvasive ablation of solid tumors, permeabilizing biological interfaces for drug delivery including the blood-brain barrier, and triggering drug release from stimuli-responsive nanocarriers.

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