Children's Health

New study to investigate focused ultrasound’s role in addressing neurofibromatosis type 2

The Focused Ultrasound Foundation and the Children’s Tumor Foundation (CTF) have established a partnership to advance innovative, noninvasive treatments in pediatrics. For more than 15 years, the Focused Ultrasound Foundation has been dedicated to advancing the development and adoption of focused ultrasound. Likewise, for over 40 years, CTF has been a leader in driving research, expanding knowledge, and advancing care for the neurofibromatosis community.

To launch the partnership, the two organizations are co-funding an early-stage laboratory study to investigate focused ultrasound’s role in addressing neurofibromatosis type 2, or NF2 (now known as NF2-related schwannomatosis). NF2 is a rare genetic condition that causes tumors to grow along the nerves. While the tumors are benign, they can lead to serious health conditions, including hearing loss, tinnitus, hydrocephalus, and compression of the brainstem.

“Treatment options for NF2 patients are limited to high-risk surgery, chemotherapy, or radiation therapy, which carry high risks of morbidity. The development of more nonsurgical treatments, like focused ultrasound, offer much promise in more effectively and safely helping patients,” said Tracy Galloway, Children’s Tumor Foundation Board of Directors Chair. “On a personal level, it’s devastating to see what NF2-related tumors can do to a person, like my daughter, who just underwent her third brain surgery, this time to remove a vestibular schwannoma located at the juncture where the hearing, balance, and facial nerves come together. Her recovery is ongoing, but she lost her hearing and other side effects, and it would be our greatest hope to not have to subject patients to severe surgeries and their devastating after effects. Though this journey has been tough for us personally, we know new options are on the horizon and we are proud to partner with the Focused Ultrasound Foundation and Dr. Tyrone Porter in order to help as many patients as quickly as possible. We believe this could be a real game-changer in improving the quality of life of those living with NF2.”

Tyrone Porter, PhD, the Donald J. Douglass Centennial Professor in Engineering and an expert in image-guided drug delivery in the Department of Biomedical Engineering at the University of Texas at Austin, is the principal investigator for the study. He and his team will use focused ultrasound to deliver a drug commonly used for lung cancer to a preclinical model of NF2. The drug will be wrapped in a special thermosensitive liposome packaging, and researchers hope to determine whether this packaging will lessen the drug’s systemic side effects. If this is the case, it may be possible to increase the local concentration of the drug and thus enhance its effectiveness. This liposome encapsulation approach has helped with drug delivery to other types of brain tumors, including glioblastoma.

Dr. Porter is excited to partner with CTF and the Focused Ultrasound Foundation to develop a new treatment option for NF2.

Thermosensitive liposomes have proven to be an effective tool for localized delivery of anticancer agents to solid tumors, predominantly in adult patients. Focused ultrasound allows for triggered drug release specifically in the tumor, thus minimizing systemic exposure and the associated toxic effects. We believe this treatment strategy can be equally effective against pediatric brain tumors.”

Dr. Tyrone Porter, PhD, the Donald J. Douglass Centennial Professor in Engineering

“This study is a first step toward addressing some of the unmet needs in the pediatric field,” said Focused Ultrasound Foundation Chairman Neal F. Kassell, MD. “The Foundation recognizes the tremendous impact that focused ultrasound could have on young patients because it avoids surgery and radiation therapy and has fewer side effects than more traditional treatments. We are honored to partner with CTF and Dr. Porter on this project.”

In August 2020, the Focused Ultrasound Foundation and CTF first collaborated to host a webinar and virtual panel discussion that introduced focused ultrasound to the neurofibromatosis (NF) research community.

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