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UNC researcher works on innovative drug to treat Sickle Cell patients

Posted December 8, 2016
Updated December 15, 2016

A new drug shows dramatic promise to help Sickle Cell patients reduce painful episodes.

UNC's Dr. Kenneth Ataga was the principal investigator for an investigational drug called Crizanlizumab. The trial involved 198 participants in a multi-center, randomized trial.

The “Sustain Trial” results were published online by the New England Journal of Medicine.

The results were so significant that the FDA will now review the findings and discuss whether the drug can be approved based on the study data or if it requires more research.

Sickle cell disease is an inherited blood disorder that affects one out of every 500 African Americans.

Patients can experience extreme pain, leading to emergency department visits or hospital admissions.

“And so when there is obstruction to blood flow, there is ischemia. This is what we think causes the pain episodes,” Ataga said.

Hydroxyurea has been the only medication available for patients with the most common form called Sickle Cell Anemia.

“Patients who got the high dose treatment got a significantly reduced frequency of painful crisis and we actually found a decrease in the frequency of pain of up to 45 percent,” he said.

Ataga said the drug, manufactured by Novartis, helps with all forms of sickle cell disease. It increased the time between pain crises and reduced the rate of days patients spent in a hospital by 42 percent.

“This drug represents a novel, potentially novel agents that can help to decrease the frequency of painful crisis and improve the quality of life in patients with sickle cell disease,” Ataga said.

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