Basic and Translational Research Program in Sickle Cell Disease

Principal Investigator: Victor R. Gordeuk, MD

The Basic and Translational Research Program (BTRP) in Sickle Cell Disease includes Howard University, a historically-black teaching institution in the nation's capital, and Children's Research Institute, the premier provider of healthcare to children with sickle cell disease (SCD) in the area. The components of the project are:

Component 1: Neurodevelopmental Deficits among Infants and Toddlers with Sickle Cell Disease (SCD) (“Translational II”). We are undertaking a 4-year mixed cross-sectional longitudinal study of early neurodevelopmental status in children younger than 4 years with SCD.

Specific Aims:

  1. Characterize the prevalence and nature of the developmental deficits in infants and toddlers while controlling for Socioeconomic Status (SES).
  2. Examine the moderating effects of sickle cell phenotype, hematologic variables, disease severity, and parent characteristics on developmental outcome.
  3. Improve patient outcomes by including routine parent education about normal development and behavior during each patient visit at the SCD Clinic.

Component 2: Sickle Cell Summer for Science Program (“Administrative Core”). The main focus of the Administrative Core is to ensure general BTRP oversight and manage the Summer for Science Program at Howard University. This program will expose high school students to a laboratory research environment in a medical center and should be designed to cultivate continued interest in scientific research related to sickle cell disease.

Specific Aims:

  1. Publicize, solicit, review and select applications from area high school students interested in the program.
  2. Develop didactic curriculum for the program to include background information about sickle cell disease, exposure to modern clinical research methodologies and regulations and introduction to current treatments and therapies for managing sickle cell disease.
  3. Assign each student a research mentor/adviser who will provide a specific research project to work on and present upon completion of the summer program.

Component 3: ("Sickle Cell Scholar Program") Dr. Naomi Luban, from the Children's Research Institute in Washington, DC, in conjunction with the physicians and scientist of BTRP, will develop a carefully planned, individualized research training program designed to ensure that the chosen Scholar becomes an independent physician and/or biomedical researcher focused on critical translational and clinical questions related to sickle cell disease.

Specific Aims:

  1. Promote the development of clinical and translational research skills of physicians and physician-scientists with a dedicated interest in the pathophysiology and treatment of sickle cell disease.
  2. Encourage promising young Ph.D. biomedical researchers to focus their efforts on the biochemical, genetic and genomic approaches to the complications of sickle cell disease, including, but not limited to, vasculopathy, immunogenetics and iron metabolism.
  3. Facilitate interactions between young clinical and translational researchers who will become future leaders in investigations leading to improved treatment of sickle cell disease and related disorders.