One of the most devastating things about stroke and head trauma is that the damage to the brain often doesn’t end when the initial injury does. Though time really does heal many wounds, brain injuries can get progressively worse in the hours and days after a trauma, as changes in blood flow and other processes cause brain cells that escaped the initial assault to die.
Astrocyte Pharmaceuticals—a Cambridge, MA-based company built around research from the University of Texas Health Science Center at San Antonio, TX—is developing a new drug that it believes can help protect brain cells left at risk after a stroke or other blunt trauma. The treatment targets the most abundant type of cell in the brain, astrocytes, which help keep neurons—in essence, the electrical cabling of the brain—up and running.
Traumas like strokes can limit blood flow to parts of the brain, starving astrocytes of the chemical food they need to produce enough energy to perform a host of the functions that normally aid and protect neurons. When astrocytes’ batteries run low, neurons can suffer from chemical imbalances that cause them to malfunction, swell, and even burst, spreading the damage of the trauma further throughout the brain. Astrocyte Pharmaceuticals is hoping that its drug, which targets a receptor in astrocytes, can effectively recharge these important helper cells.
“We don’t let the damage get out of hand,” says James Lechleiter, the co-founder of Astrocyte and the scientist who discovered the drug’s potential in the lab a decade ago. “The sooner you can stop it, the sooner the astrocyte can get a hold of these recurrent waves of damage that are occurring. You can get it under control.”
Astrocyte announced in September it had received a $500,000 grant from the National Institute of Health to evaluate its drug for use in traumatic brain injuries and concussions. The constant beating some American football players take, and the subsequent concussions and other brain injuries they sometimes suffer, have drawn attention and headlines internationally. The grant was a validation of the research that Astrocyte has already done, Lechleiter says, including studies in mice and pigs.
The funding will help the company study the medicinal chemistry of its drug to help optimize the dosing and other factors, he says. If Astrocyte meets its milestones, it could seek a $3 million Phase 2 round from the NIH, says Lechleiter, where he is a professor at the University of Texas Health Science Center.
Some existing drugs have proven useful at limiting the damage of strokes, including aspirin and a clot-busting drug called tissue plasminogen activator, also known as tPA. TPA is the only treatment specifically approved by the FDA for stroke, and is only safe to use within a few hours have an incident, Lechleiter says. A peptide mixture marketed as Cerebrolysin by Ever Pharma is available in certain Asian and European countries as a treatment for stroke or traumatic brain injury, but there are no approved drugs for traumatic brain injury in the United States.
“There’s a huge interest in treating traumatic brain injury and stroke. There’s been this dearth or complete absence of compounds that are successful,” Lechleiter says, a claim that’s backed by other research. “A huge part of that is that they have focused so long on the neuron target, not the astrocyte.”
Astrocytes are a type of glial cell, which provide support to the body’s nervous systems and have been a major focus of Lechleiter’s career. The research that led to the development of this drug was, as is often the case in scientific discovery, serendipitous.
Lechleiter was actually trying to design a drug that killed cancer cells. But when he targeted astrocytes via a specific target called a G protein-coupled receptor it actually increased the cells’ ability to survive—an effect that Lechleiter quickly realized could be very helpful in the case of brain injury.
Though Lechleiter made his discovery almost a decade ago, he didn’t begin building a company until 2014, when he met William Korinek, who was then the vice president of Worldwide R&D Business Operations at Pfizer. Korinek left Pfizer to co-found Astrocyte and act as its CEO.
Astrocyte is now in the process of trying to raise a large seed round, which it hopes will be several million dollars, Korinek wrote in an e-mail. “Many people have suggested that there are likely former athletes or military personnel that would be eager to contribute to a promising potential concussion therapeutic, and could be interested in making an investment in small biotech company,” he wrote.
In May, the university gave Astrocyte the worldwide licensing rights to the research and patents for the class of small molecules. Terms were not disclosed.
Lechleiter also believes the drug might eventually prove useful beyond stroke and traumatic brain injury, in treating aliments as simple as headaches and as complex as Parkinson’s disease and Alzheimer’s. For now, the company is seeking to perfect its dosing and related pharmaceutical analysis so it can move forward with clinical studies.
“We’ve always been interested in how the brain works,” Lechleiter says. “As I get older, I’m interested in how to keep it working when it gets injured.”
