Grant Awarded to Rho's Federal Systems Division for Pediatric Sickle Cell Research

Chapel Hill, NC

The National Heart, Lung, and Blood Institute (NHLBI) has awarded a five-year grant to Rho’s Federal Systems Division, a division of Rho, Inc., to conduct “TCD with Transfusion Changing to Hydroxyurea (TWiTCH),” a Phase III clinical trial of a novel therapy to reduce the risk of stroke and prevent iron overload in children with sickle cell anemia (SCA). This grant is supported by NHLBI Award Number R01HL095511. St. Jude Children’s Research Hospital will host the TWiTCH Medical Coordinating Center and Rho will host the TWiTCH Statistics and Data Management Center (SDMC).

Patients will be recruited, treated, and evaluated at about 25 clinics in the United States. Almost 10% of U.S. children under the age of 18 with SCA suffer strokes, a devastating clinical event that results in severe motor and neurocognitive problems. Previous studies have shown that the risk of stroke is closely related to the velocity of blood flow in arteries in the neck, which can be measured by transcranial Doppler (TCD) ultrasound. Children with high velocities are treated proactively to prevent stroke. The standard therapy involves monthly transfusions of normal red blood cells to prevent buildup of sickle cells and reduce clotting, thus reducing the risk of a stroke. While transfusions are effective at preventing stroke, over several years the iron from the transfused red blood cells accumulates in critical organs, including the heart, often leading to disability or death from iron overload.

The novel therapy initially developed by Dr. Russell Ware, then at Duke University and now at St. Jude Children’s Research Hospital, with the assistance of UNC Professor Emeritus and Rho cofounder Dr. Ronald Helms, is two-pronged. Hydroxyurea, a generic drug that increases the percentage of non-sickling red blood cells, is used to reduce stroke risk, and a monthly phlebotomy procedure that withdraws a unit of blood from the body reduces iron accumulated from previous transfusions. The study’s hypotheses are that the novel treatment will be comparable to transfusions for controlling stroke risk and will reduce iron overload more than the standard iron chelation treatment.

The TWiTCH SDMC continues Rho’s long history of working with the NHLBI and in sickle cell disease research. Dr. Helms served as the principal investigator (PI) for the coordinating center of the NHLBI-funded “Safety of Hydroxyurea in Children with Sickle Cell Anemia (HUG-KIDS)” study that first demonstrated that hydroxyurea is safe for use in children. He was also PI of the SDMC for the “Comprehensive Sickle Cells Centers Program (CSCC),” providing research services to 10 internationally prominent sickle cell research centers, including a combined Duke-UNC center. Dr. Helms and Dr. Nancy Yovetich, who currently serve as the PI and co-PI on the NHLBI-funded SDMC at Rho for the “Stroke With Transfusions Changing to Hydroxyurea (SWiTCH)” Phase III study, will also serve as PI and co-PI for the new TWiTCH SDMC.