A San Antonio discovery is on the edge of becoming the first big leap in stroke treatment in decades, according to James Lechleiter, PhD, professor of cell systems and anatomy at UT Health San Antonio. “The only treatments are the removal of a clot. That's it. There have been no small therapeutics that have successfully reduced brain injury,” he said.
Lechleiter explained that much of stroke research focuses on neurons, but he’s interested in star-shaped cells called astrocytes.
“The neuron is not very good at taking care of itself,” he pointed out. “So the astrocyte has an enormous number of functions, right after an injury. It tries to restore that environment, to try and help even repair it.”
Astrocytes are “caretaker cells,” Lechleiter said, and in the early 2000s, his team noticed something interesting about a drug he was working on in brain cancer. When they used it, Lechleiter expected astrocytes to die, but they did the opposite.
Professor of Cell Systems and Anatomy
Director of the Optical Imaging Core
The University of Texas Health Science Center at San Antonio
“We noticed that when we applied this drug, that caretaking cell in the brain survived much longer in harsh conditions,” Lechleiter said.
The drug appeared to have the same effect on astrocytes following a stroke or other type of brain injury. “What our therapeutic does is boost their energy so they've got more resources to clean up the environment, to restore the normal function in the brain, and to heal it, if possible,” Lechleiter said.
The medication doesn’t have a catchy name yet. They call it AST-004. But it’s headed for human trials this year. One trial has been approved by the National Institutes of Health as part of its StrokeNet research. The other trial will test its effectiveness in improving outcomes after concussion. That trial will study Australian Football League players who’ve taken a hit to the head. There are even fewer treatment options for concussions than there are for strokes.
“The only treatment there is rest. Get off the field and don't get back out there. You've bruised your brain. Take it easy,” Lechleiter said.
The NIH trial has not yet been funded, and the current uncertainty around everything the federal government has committed to funding exists here, too. “We expect, if it is fully funded, we should be in a clinical trial with that probably by the end of the summer.”
The Australian Football trial is a go, regardless.
Lechleiter and his team are thrilled to have taken this drug from discovery to development to human trials over the last two decades. The ultimate goal, of course, is to have the medication evaluated and approved by the FDA.
“We’re close…and we think we’ve got a chance,” Lechleiter concluded.
Science & Medicine is a collaboration between TPR and The University of Texas Health Science Center at San Antonio that explores how scientific discovery in San Antonio advances the way medicine is practiced everywhere.
