When funded next April, the St. Jude grant is estimated to exceed $9 million.
The grant proposal includes five local projects focusing on stem cell transplantation, pneumococcal infection in sickle cell patients, combination drug therapy, gene therapy and molecular biology of hemoglobin development. The proposal also includes a multi-center project examining the effectiveness of the drugs hydroxyurea and magnesium used in combination to combat the clinical complications of sickle cell disease.
As a result of the grant, St. Jude sickle cell patients will have more opportunities for new therapies and participation in groundbreaking clinical trials.
"This is the first step toward the development of a national clinical trials network for sickle cell disease," said Winfred Wang, M.D., director of the St. Jude Hematology Division. "This is an approach that has been successful in improving outcomes for pediatric cancers and thalassemia (a genetic blood disorder) but has not been available for sickle cell disease."
Wang said the grant will make possible several planned initiatives, including evaluating patients to determine if they fall into high-risk categories and tailoring therapies accordingly. Wang also hopes to give sickle cell patients from St. Jude Domestic Affiliate clinics the opportunity to participate in clinical trials and come to Memphis regularly for evaluation. Additionally, the St. Jude sickle cell program will develop closer ties to centers treating adult sickle cell disease, sharing research and treatment findings.
Most pediatric sickle cell patients in the Mid-South will continue to receive their primary care at the St. Jude sickle cell clinic, which is located at Le Bonheur Children's Medical Center in Memphis. Clinical research activities will take place mainly on the St. Jude campus.
"This grant is important because it raises awareness that St. Jude is very much involved with sickle cell disease and has been for a very long time," Wang said. "Among sickle cell caregivers and investigators, people know that St. Jude is a leader, but people outside that community aren't necessarily aware of the institution's contributions."
The other institutions to receive NHLBI grants are:
- Children's Hospital and Research Center at Oakland
- Cincinnati Children's Hospital Medical Center
- University of Southern California
- University of Texas Southwestern Medical Center at Dallas
- Boston University
- The Children's Hospital of Philadelphia
- Thomas Jefferson University
- Albert Einstein College of Medicine
- Duke University
Sickle cell disease is an inherited disorder of red blood cells that causes the cells to become sickle-shaped and firm, instead of round and flexible. These abnormal blood cells clog blood vessels, causing episodes of severe pain, organ damage and eventually premature death. Sickle cell disease occurs primarily in African-Americans but also affects people of Hispanic and Middle Eastern descent. If both parents have the sickle cell trait there is a one-in-four chance that their child will be born with sickle cell disease. About one in 375 African-Americans is born with a form of the disorder. More than 70,000 Americans have sickle cell disease.
St. Jude Children's Research Hospital, in Memphis, Tennessee, was founded by the late entertainer Danny Thomas. The hospital is an internationally recognized biomedical research center dedicated to finding cures for catastrophic diseases of childhood. The hospital's work is supported through funds raised by ALSAC. ALSAC covers all costs not covered by insurance for medical treatment rendered at St. Jude Children's Research Hospital. Families without insurance are never asked to pay. For more information, please visit www.stjude.org.