Cancer and Blood Disorders Research

Cancer is the leading non-accidental cause of childhood death. Yet, thanks to research, since the 1970s childhood cancer deaths have declined 35 percent, and in the case of leukemia by 50 percent. However, the battle is not over. Each year 25 percent of children diagnosed with cancer will die of their disease.

Statistics like these fuel a passion to find cures within physicians and scientists of the Division of Hematology/Oncology of UPMC Children’s Hospital of Pittsburgh. The division has extensive research interests in clinical treatment for and basic mechanisms underlying cancer, ultimately aimed at improving clinical therapies. Its commitment to advancing the science of hematology and oncology includes basic research extending into the areas of stem cell biology and molecular oncology.

With so much at stake, Children’s Hematology/Oncology team ranks first in sheer volume of clinical studies within the divisions of UPMC Children’s Hospital of Pittsburgh. This is due, in part, to its strong relationship with the Children’s Oncology Group, the only pediatric oncology clinical trials group funded by the National Cancer Institute and National Institutes of Health (NIH).

At Children’s, clinical trials are underway related to bone marrow transplantation, treatment of sickle cell disease, cancer control, and late-effects in oncology. An ongoing oncology study at Children’s examines incidence of graft-versus-host disease following unrelated donor stem cell transplantation. Children’s is the only institution in the region participating in Phase 1 clinical trials through the Children’s Oncology Group and the Pediatric Brain Tumor Consortium. These important and innovative studies of new cancer treatments are critical to our progress in achieving the goal of curing all children with cancer.

Ongoing NIH-funded oncology projects include the study of critical biochemical events that control the growth, maturation and death of cells that cause leukemia and the study of a family of genes that cause cancers such as Burkitt’s lymphoma and neuroblastoma.

As leaders in their field, Children’s Hematology/Oncology scientists have access to the most advanced tools and resources. Genetic microarray technology, for example, is being utilized in a study on the mechanisms that cause cells to become cancerous or resistant to anti-cancer drugs. This cutting-edge technique, based on results of the Human Genome Project, allows one to measure simultaneously how much protein is being made by more than 5,000 different genes in a single cell at any given time.

The Hematology/Oncology team has not limited itself to the scientific studies of childhood cancers, but also addresses the special medical and psychosocial needs of cancer patients. Through this broad-spectrum approach Children’s Division of Hematology/Oncology continues to forge ahead in its efforts to explore, diagnose, treat and cure childhood cancers.

Physicians at Children’s Hospital are also active in clinical care and research in diseases of blood other than cancer. Sickle cell disease is a serious chronic illness of the red blood cell that is complicated by anemia, stroke, lung complications and decreased quantity and quality of life. The sickle cell program at Children’s Hospital is one of eight sites around the world that are participating in a clinical trial to determine the prevalence of pulmonary hypertension a serious complication involving the heart and lung. Patients found to have pulmonary hypertension will be treated in a randomized double-blind study of a drug called sildenafil. This groundbreaking study is expected to provide information on a serious and little understood complication of this disease.

Researchers at Children’s Hospital are also leading a study to cure sickle cell disease by a gentler type of bone marrow transplant. This approach has the potential to make this curative treatment to many patients to whom this was not previously available. Research in sickle cell disease also includes approaches on how best to provide comprehensive care, education and counseling to a population that disproportionately affects individuals who belong to minority, underserved groups, by using a comprehensive "medical home" approach and the use of medical information technology.

Edward Prochownik, MD, PhD

Edward V. Prochownik, MD, PhD

Director of Oncology Research, Children's Hospital of Pittsburgh of UPMC
Professor of Molecular Genetics and Biochemistry, University of Pittsburgh School of Medicine

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