New Method Developed to Identify Potential Stroke Therapies

Researchers identified uric acid as a potential therapy to enhance recovery from acute ischemic stroke. The study was published in Science Translational Medicine.

In the study, researchers from NIH’s Stroke Preclinical Assessment Network (SPAN) rigorously tested the effectiveness of six novel therapies in reducing ischemic brain injury in rodents, including four FDA-approved drugs to treat other conditions. Uric acid was the only candidate that passed the efficacy boundary through all phases of analysis.

Ischemic stroke, a leading cause of disability and death in the U.S., occurs when a blood clot or other blockage in an artery cuts off blood supply to the brain. Current treatments are aimed at removing the clot by dissolving it with blood-thinners, surgically removing it from the blood vessel or a combination of both.

Although these treatments help patients recover, scientists are seeking a therapy that could protect the brain from damage, known as a cerebroprotectant, that occurs before or during the restoration of blood flow. 

“We were faced with a critical need to redesign the entire preclinical approach,” said Dr. Francesca Bosetti, NINDS program director. “SPAN successfully applied well-known clinical research practices to a preclinical trial—randomization, pre-determined sample sizes, treatment masking, blinded analysis, and efforts to make results reproducible in other laboratories.”

In the current study, the efficacy of each of six treatments was assessed by giving animals a series of behavioral tests. After collecting the data, researchers used a new statistical method to evaluate the therapies at four points in the testing process. They also measured MRI brain scans of lesion volumes. 

Uric acid has previously been tested in stroke patients, but not in combination with the clot-removal treatment modeled by the study, suggesting that the drug could do well in future trials. However, investigators recommend further testing in animal models before clinical trials in humans.