Scientists at the University of Missouri have found a new way to get medicine past the brain’s natural defenses, offering a bit of hope for those living with ALS. Also known as Lou Gehrig’s disease, ALS is notoriously difficult to treat because the brain has a “gatekeeper” called the blood-brain barrier. This layer of cells is great at keeping out toxins, but it’s also very good at blocking the medicine doctors want to get inside.

Smita Saxena, a professor at the Mizzou School of Medicine, found a clever workaround. Her team took a natural molecule called GM1 and tucked it inside a tiny bubble of fat. This microscopic package acts like a delivery truck, allowing the medicine to slip through the brain’s defenses.

For the Brain

To understand why this helps, you have to look at how ALS works. The disease causes a series of breakdowns in the brain that eventually lead to muscle weakness and fatigue.

“Our research team’s previous work showed that neurons in those with ALS are very sensitive to endoplasmic reticulum stress, which limits the mitochondria’s ability to produce energy,” Saxena said. “This, in turn, limits the ability of neurons to send messages across synapses when we want to move our muscles.”

Basically, the brain’s power plants stop working correctly, and the messages telling your muscles to move get lost. By delivering that extra GM1 molecule, the researchers were able to help those neurons stay healthy. In tests with mice, the therapy improved their motor neurons and helped them move more easily.

Moving toward human trials

This delivery system has already been tested in humans for Parkinson’s disease, where it proved to be safe. Since we already know it’s well-tolerated, the team is looking toward human clinical trials for ALS.

If it works, the treatment could be life-changing. Researchers think it could eventually be given to younger people who carry ALS mutations, potentially stopping the disease before they ever feel a symptom.

“The NextGen Precision Health building is the perfect place for this research,” Saxena said. “By having research and clinical space under the same roof, we can speed up the process for translating foundational research into human clinic trials to ultimately help improve quality of life for Missourians and people worldwide.”