Cancer and Blood Diseases Center

Our innovative approach to childhood cancer research

The team at Dana-Farber/Children’s Hospital Cancer Center improves care in many ways. Some changes come from scientific research into childhood cancer. Understanding diseases deeply—even at the cellular or molecular level—leads to new drugs and therapies. Other improvements come from moments spent at the bedside, when doctors and nurses see opportunities to improve current treatment methods.

Innovations play a critical role in our patients’ health, so we’re constantly creating new ways to treat cancer and blood diseases. This is especially true for hard-to-treat conditions like relapsed leukemia, certain brain tumors, neuroblastoma and some inherited genetic conditions like Fanconi anemia and bone marrow failure. An innovation may be “small”—like finding a creative way to help a young patient take her medication. Or it may be big—a discovery that changes the entire field of pediatric hematology/oncology.

 
_{Some inspiring examples from our childhood cancer research programs include:}
 

Gene therapy clinical trials for x-linked Severe Combined Immunodeficiency (SCID)

Gene therapy, which uses a virus to reintroduce a missing gene, holds the promise to cure x-linked SCID, more commonly known as “bubble boy disease.” Clinical trials stalled after five boys with the disease developed leukemia as a result of their treatment in Europe. Now, an international trial—sponsored in the United States by Children’s David Williams, MD, chief of Hematology/Oncology and director of Translational Research and led by Luigi Notarangelo, MD, (Immunology) and Sung-Yun Pai, MD, (Hematology/Oncology)—hopes to revive this potentially life-saving treatment. The new trial, the first to be given the green light by the FDA, will use a completely redesigned gene-delivery vector expected to be much safer than the one used in the European trial. Our team is one of five in the United States and Europe participating in this trial, which, if it succeeds, is expected to pave the way for treating several other single-gene diseases with gene therapy.

Outpatient chemotherapy service

A daily course of chemotherapy no longer automatically means an overnight hospital stay for many pediatric patients. Through our outpatient chemotherapy program, launched by pediatric oncology doctors and nurses at Children’s and Dana-Farber, eligible patients have the option of receiving post-chemo intravenous fluids from a small pump carried in an easy-to-wear backpack. This means they can spend more time outside the hospital environment, sleeping either at home or a home-away-from-home facility, such as the Ronald McDonald House. In 2009, outpatient chemotherapy allowed more patients to spend time receiving therapy at home, instead of in the hospital.

Home Visits, a community outreach program for children with cancer

Newly diagnosed families experience a range of emotions—from relief to fear of being responsible for managing a child’s medical needs—the first time they leave the hospital. To ease the transition, a team of nurses at Children’s developed Home Visits. Since 2007, Home Visits has dispatched a nurse to the home of newly diagnosed patients within the first two days of the child's first inpatient discharge. During the visit, our staff nurse coordinates treatment with the home care nurse, educates the family and helps them become comfortable managing the child's medical needs. In addition to easing family worries, Home Visits also optimizes care by educating home care nurses about pediatric cancer care so patients return to Children's at the earliest sign of a complication.