From the March 2018 issue of HealthCare Business News magazine

Even at her young age, the noncancerous tumor was beginning to impede her developmental growth and threatened to cause muscle atrophy and permanent damage to her blood vessels and nerves. Typically, these tumors are treated with chemotherapy, surgery and/or radiation. But, the efficacy is variable and the side effects can be great. In this case, first line chemotherapy failed to control the desmoid, and the second medical therapy resulted in her hospitalization to manage an allergic reaction.

After her physician told the family about focused ultrasound and referred them to Stanford’s Lucile Packard Children’s Hospital, she became the youngest child in the world to receive focused ultrasound treatment, for any disease. The treatment reduced the size of her tumor dramatically without side effects. A quick recovery meant that this child was able to return to enjoying gymnastics and swimming without having to worry about her tumor getting in the way. Although the tumor is still there, it can be retreated quickly and noninvasively if it grows.


Tackling other tumors
Desmoid tumors are relatively rare, with only 900 cases diagnosed annually in the U.S. But the potential applications, beyond desmoid tumors, for focused ultrasound technology are virtually limitless.

There is an ongoing clinical trial at Nicklaus Children’s Hospital in Miami to treat tumors within the brain. These benign brain tumors – called subependymal giant cell astrocytomas, or SEGAs – can grow and cause harm, resulting in cognitive delays, memory loss and/or seizures. While focusing beams of sound through the pediatric skull to kill the tumor is no easy task, SEGAs grow deep in the brain and have sharp margins, making them optimal targets for early focused ultrasound trials.

Other major children’s hospitals, including Children’s National, Cincinnati Children’s, University of Texas Southwestern, UCSF Benioff and Toronto SickKids, are conducting research to test this technology. This includes clinical trials to treat a painful benign bone tumor, called an osteoid osteoma, and other metastatic tumors that occur within the body of children and young adults.

Obstacles to overcome
As any pioneer knows, the key to adoption is widespread use. Focused ultrasound is getting there, but it will take time, especially for pediatric patients. Support is needed for continuing clinical trials to rigorously assess the safety and efficacy of focused ultrasound for these and other new pediatric applications. But enrollment in these clinical trials is a hurdle. Aside from simply finding patients that meet the treatment criteria, the parents also have to be on board. It is scary to be among the first to receive a new treatment, and even scarier when parents have to make the decision for their child. But, it is important to remember that with every treatment and every trial, focused ultrasound gets closer to being accepted as a safe and effective option.

March 2018 Magazine

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