Focused ultrasound may offer a way to bypass the brain’s natural defences, delivering creatine directly into the brain. This could help patients with creatine transporter deficiency who struggle with speech, memory, and cognitive delays.
For most people, creatine is associated with bodybuilders, bulging biceps, and gym supplements. But beyond the world of fitness, it may hold the key to something far more critical: healthy brain function.
At Virginia Tech, researchers are testing a new way to sneak creatine past the brain’s natural defences. Backed by a grant from the Association for Creatine Deficiencies, the team is exploring the use of focused ultrasound to briefly open the blood-brain barrier – giving creatine a chance to do its job inside the brain.
Creatine Is Not Just About Muscle
Creatine helps the body make adenosine triphosphate (ATP), the energy molecule that powers nearly every cell. Most people know about its effects on muscle, but brain cells need it too. Without enough creatine, the brain’s ability to support memory, learning, and speech may be compromised.
“Creatine is very crucial for energy-consuming cells in skeletal muscle throughout the body, but also in the brain and in the heart,” explained Chin-Yi Chen, a research scientist at Virginia Tech’s Fralin Biomedical Research Institute at VTC.
Creatine in the Brain
Inside the brain, creatine does more than support energy production. It interacts with GABA, a neurotransmitter that calms brain activity and helps regulate learning, memory, and development.
Some researchers even speculate that creatine may act as a neurotransmitter itself, as it is transported from glial cells to neurons and appears to influence how brain cells communicate.
When brain creatine levels are too low, the effects can be serious. In children with creatine deficiency, brain development may be impaired, even if muscle growth improves with supplements.
Unfortunately, the brain’s own security system – the blood-brain barrier – makes treating deficiencies complicated. This barrier is designed to block toxins and pathogens, but it also stops many helpful substances like creatine from entering when levels are low.
While creatine supplements often help patients gain weight and muscle mass, many do not experience the same benefits on the cognition front.
A Sonic Shortcut
Enter Dr. Cheng-Chia “Fred” Wu and his team. They are developing a technique called focused ultrasound, using sound waves to open up small, targeted areas of the blood-brain barrier. The idea is to create a temporary gateway that lets creatine pass through, without damaging nearby tissue.
Wu, who initially studied this technique for paediatric brain cancer, believes it could have major implications for treating creatine transporter deficiency, a genetic condition where creatine struggles to reach brain cells.
But a meeting with Dr. Seth Berger, a medical geneticist, introduced him to creatine transporter deficiency. That conversation sparked a new line of research: using ultrasound to get creatine into the brain.
From Bench to Bedside
With funding in place and a research partnership between Virginia Tech and Children’s National Hospital, the team is now testing the method in preclinical 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,” Chen said.
Their first step is to confirm whether creatine can actually cross into the brain with the help of ultrasound. If it works, the team will evaluate whether this approach can improve brain function or development in models of deficiency.
Both Virginia Tech and Children’s National are recognised Centres of Excellence by the Focused Ultrasound Foundation. This gives the team access to clinical trial expertise and the infrastructure needed to bring this therapy closer to patients.
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