Creatine is widely known as a supplement used to build muscle, but it also plays a crucial role in how our muscles, brain, and heart function.
In fact, it can be a matter of life or death for people who have creatine deficiencies.
“Creatine is very important for cells that use a lot of energy, especially in the muscles, brain, and heart,” said Chin-Yi Chen, a research scientist at Virginia Tech’s Fralin Biomedical Research Institute.
Chen is part of a team working on a new way to deliver creatine directly to the brain using focused ultrasound. This project, led by Assistant Professor Cheng-Chia “Fred” Wu, recently received a $30,000 grant from the Association for Creatine Deficiencies.
In the brain, creatine helps produce a key energy molecule called adenosine triphosphate, or ATP. ATP powers many processes in our cells.
Creatine also plays a role in brain signaling, especially in systems that use a chemical called GABA, which helps calm down the brain. These systems are involved in important functions like learning, memory, development, and even preventing seizures.
There is also growing evidence that creatine may act like a neurotransmitter itself. It is passed from supportive brain cells, called glial cells, to nerve cells and may help control how messages are sent between them.
People with creatine deficiency disorders often have weak muscles and heart problems, but their brains are also severely affected. While creatine supplements can help build muscle and increase body weight, many patients still struggle with learning and speaking.
One big reason is that the brain is protected by the blood-brain barrier. This barrier blocks harmful substances from getting into the brain, but it can also prevent helpful ones, like creatine, from getting in when they are needed most.
Dr. Wu studies how focused ultrasound—a treatment that uses sound waves—can help open up the blood-brain barrier for a short time. This allows medicine to get into the brain without damaging healthy cells. While he first studied this technique for treating childhood brain cancer, he now sees it as a possible way to treat creatine deficiency.
At a research event hosted by Children’s National Hospital, Wu met Dr. Seth Berger, a medical geneticist who introduced him to creatine transporter deficiency. This meeting sparked the idea of using focused ultrasound to help patients with this condition.
Virginia Tech and Children’s National Hospital are now recognized as Centers of Excellence by the Focused Ultrasound Foundation. This means they are leaders in using this technology for medical research. Wu said the partnership allows experts from different fields to work together and design studies that could lead to future clinical trials.
Chen said he was thrilled to join the project because it connects basic science with real ways to help patients. His early research will focus on using focused ultrasound to get creatine into the brain in animal models of creatine deficiency. The goal is to restore normal brain development and function.
If successful, this research could offer hope to people with creatine-related brain disorders, giving them a better chance at healthy brain development and a fuller life.
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