With limited treatment options for stroke patients available, two UConn researchers are developing an experimental drug that is capable of protecting the brain and improving recovery after a cerebral vascular accident also known as a brain attack.
Rajkumar Verma, assistant professor at UConn Health who leads a research program for advancing innovative therapies for stroke, and Raman Bahal, associate professor in the Department of Pharmaceutical sciences at UConn, targeted a small regulatory biological molecule called microRNA, which becomes abnormally elevated after stroke and promotes inflammation, contributes to tissue loss and causes a decline in neurological function, the researchers told the Courant.
MicroRNAs (miRNAs) are a class of non-coding RNAs, which do not translate into proteins, that play important roles in regulating gene expression, according to the National Institutes of Health and the researchers.
“We developed a next-generation inhibitor of this MiRNA to block its harmful effects,” the researchers said. “Unlike traditional experimental drugs that target only a single protein or molecule, this approach simultaneously suppresses multiple damaging processes by targeting several proteins. This reduces brain injury, inflammation, and the damage of the tissue while enhancing protective factors that support repair.”
Current therapies for stroke are limited and include clot-busting drugs and surgical clot removal which are only available to 10 to 15% of patients, Verma said.
“No FDA-approved drugs exist to protect the brain or aid recovery once damage begins,” said Verma.
The Centers for Disease Control and Prevention reported that stroke remains one of the “leading causes of long-term disability and death.”
Further, CDC statistics show that about 800,000 people in the United States experience a stroke each year with approximately 160,000 losing their lives.
Leading causes of stroke include high blood pressure, high cholesterol, smoking, obesity and diabetes, according to the CDC.
Verma said the experimental drug will help to reduce “the secondary damage that occurs after the primary injury” that takes place during the first couple hours of a stroke.
Verma said the use of single proteins to target acute damage by stroke were not effective because stroke affects large number of proteins that may be contributing to brain damage.
Bahal said the research is critical because “we are exploring new chemistries and drug delivery technologies to counterattack this kind of devastating disease.
“It is very different from conventional treatments for stroke,” he said. “We are not using existing conventional small molecules. We want to make sure these treatments are effective but safe at the same time.”
Bahal said research has found that “a single dose of our next-generation miRNA inhibitor administered after a stroke not only decreased brain damage but also markedly enhanced movement, memory, and long-term recovery, with protective effects lasting up to 15 days in a mouse stroke model.”
“Compared to earlier drug candidates, our miRNA inhibitor is more potent, highly specific, longer lasting, and safer,” Bahal said.
The researchers have filed a patent for their MiRNA inhibitor and are in communication with pharmaceutical companies and other partners to develop it into an investigational new drug candidate for a future clinical trial. They have also received a $2.6 million grant for five years from the National Institutes of Health.
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