Researchers at Virginia Tech’s Fralin Biomedical Research Institute are developing a method to deliver creatine directly to the brain using focused ultrasound. The technique is being investigated as a potential intervention for children with creatine transporter deficiency, a condition that can impair speech, memory, and learning.
Creatine plays a role in cellular energy production and neurotransmitter regulation, making it critical not only for muscle but also brain function. While oral supplements often improve muscle mass in patients with creatine deficiency, they do not consistently address neurological symptoms, in part because creatine has difficulty crossing the blood-brain barrier.
“Creatine is very crucial for energy-consuming cells in skeletal muscle throughout the body, but also in the brain and in the heart,” says Chin-Yi Chen, a research scientist working on the project.
Ultrasound delivery to bypass the blood-brain barrier
The research is led by assistant professor Cheng-Chia “Fred” Wu, who studies therapeutic focused ultrasound, a noninvasive technique that uses sound waves to temporarily open the blood-brain barrier. The goal is to allow beneficial compounds, like creatine, to reach brain tissue without damaging surrounding cells.
The project, supported by a $30,000 grant from the Association for Creatine Deficiencies, builds on Wu’s broader work using ultrasound to improve drug delivery for pediatric brain cancer. His collaboration with Dr. Seth Berger of Children’s National Hospital led to the idea of applying the same method to treat creatine deficiency.
“Through the partnership between Virginia Tech and Children’s National Hospital, I was able to present our work in focused ultrasound at the Children’s National Research & Innovation Campus,” says Wu. “There, I met Dr. Seth Berger, a medical geneticist, who introduced me to creatine transporter deficiency. Together, we saw the promise that focused ultrasound had to offer.”
Collaboration supports early-stage translational research
Both Virginia Tech and Children’s National have been named Centers of Excellence by the Focused Ultrasound Foundation, a designation that supports collaborative research bridging lab studies and clinical applications. Chen’s initial work will focus on whether creatine can be successfully delivered across the blood-brain barrier and restore normal brain mass in lab models.
“It was a moment that made me really excited — that I had found a lab where I could move from basic research to something that could help patients,” says Chin-Yi Chen. “When Fred asked me, ‘Are you interested in this project?’ I said, ‘Yes, of course.’”