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 aftereffects. 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, Ph.D., 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.

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