FDA Awards UNC Researcher $2M Grant to Study Kidney Disease in Sickle Cell Anemia

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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The U.S. Food and Drug Administration (FDA) has awarded Dr. Kenneth Ataga, a $2 million grant over five years to support a prospective study in sickle cell anemia to determine biomarkers of specific cellular function changes in chronic kidney disease (CKD).

Ataga, a researcher with the University of North Carolina at Chapel Hill, published the results of a Phase 2 SUSTAIN clinical trial (NCT01895361) in December 2016. The trial demonstrated that the anti-P-selectin antibody crizanlizumab (SEG101), developed by Selexys Pharmaceuticals and Novartis, reduced the median annual rate of sickle cell-related pain crises by 45.3 percent, when compared to placebo.

Researchers observed these results in both patients with and without hydroxyurea therapy, which reduces rate of painful attacks in sickle cell disease (SCD). Kidney involvement in SCD boosts a patient’s likelihood of also developing CKD. Endothelial cells detect physical and chemical changes in the blood vessels and release various factors to counter these changes to maintain homeostasis, or internal stability.

Endothelial dysfunction is characterized by off-balanced blood vessel function, increased oxidative stress and inflammation, abnormal smooth muscle cell proliferation, and a deficient repair mechanism. CKD patients usually have endothelial dysfunction.

A prospective study is a type of investigation that watches for outcomes like disease development and then relates that observation with other factors, such as suspected risk or protection factors.

In this case, Ataga will look for biomarkers in CKD endothelial function changes in patients with sickle cell anemia – a type of sickle cell disease that occurs when a person has two hemoglobin S genes, Hemoglobin SS — often the most common and most severe type of SCD.

The award was part of the FDA’s larger Orphan Products Grants Program, which awarded six grants worth a combined $6.3 million for natural history studies in rare diseases — defined as illnesses affecting no more than 200,000 Americans.


To boost the number of grants awarded through this program, the National Center for Advancing Translational Sciences (NCATS) — a unit of the National Institutes of Health — contributed additional funding through its Therapeutics for Rare and Neglected Diseases program.

“One of the challenges we encounter developing therapies for rare diseases is the lack of natural history data to guide the design of successful clinical trials,” Dr. Nora Yang, director of portfolio management and strategic operations in NCATS’ division of pre-clinical innovation, said in a press release. “We are pleased that we can collaborate with the FDA to fund two natural history studies this year that will help NCATS develop novel treatments for patients who suffer from these devastating diseases.”

The aim of the Orphan Products Grants Program is to inform medical product development or to eventually use natural history to increase the use of placebo arms in studies of drugs that target very rare disease, where trial recruitment can be a challenge.