• Childhood Cancer and Blood Disorder Research and Innovation

    Rani George, MD 

    At Dana-Farber/Boston Children's Cancer and Blood Disorders Center, we improve care in many ways. Some changes come from scientific research into childhood cancer and blood disorders. 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.

    Continue reading to learn about our history of innovation and our ongoing contributions to the field of pediatric hematology and oncology. And take the time to learn about the research and innovation taking place within each of our clinical centers: Blood Disorders Center, Brain Tumor Center, Hematologic Malignancy Center, Solid Tumor Center and our Stem Cell Transplant Center.

    Clinical Trials

    Clinical trials, or research studies evaluating new treatment approaches, are a major offering at Dana-Farber/Boston Children's. Many of our studies have built the foundation for new and improved cancer and blood disorder therapies used today. At any time there is an average of 60 unique clinical trials accepting new patients at Dana-Farber/Boston Children's. We offer enrollment on several independent as well as cooperative treatment studies, many of which are available only at our center. Learn about our current clinical trials.

    Innovative Therapies

    Our history of research and innovation has resulted in many of the diagnostic and therapeutic techniques that are widely used today. In our labs and research facilities, we are advancing the therapies of tomorrow. Tomorrow’s therapies will not only be more effective in treating cancers and blood disorders, but they will be less invasive and require shorter recovery times. 

    Currently, we are among the world’s leaders in the development of three innovative new therapies: Gene Therapy, Immunotherapy and Vaccine Therapy. Each of these innovations holds the promise of improving outcomes and reducing side effects and recovery times for children with cancer and blood disorders. 

    Advanced Technology

    Patients coming to Dana-Farber/Boston Children's Cancer and Blood Disorders Center gain access to the combined resources of both a renowned cancer treatment and research institution and a top children’s hospital. These resources are not limited to the expertise of our doctors; our state-of-the art treatment facilities allow our doctors and nurses to provide the safest and most effective care possible. Some of the advances we offer:

    • Therapeutic I-131 MIBG is a special treatment for children with refractory or relapsed neuroblastoma. Children receiving this treatment must stay in a special protective room at Boston Children’s Hospital - one of only a handful of centers in the United States equipped for this type of treatment.
    • Our pediatric stem cell transplant team, one of the most experienced in the United States, performs more than 90 transplants each year for children in our special HEPA-filtered unit. The unit is built with needs of stem cell transplant patients in mind, offering maximum safety without completely restricting the patient to his/her room.
    • Children with brain tumors often need surgery as part of their treatment. At Children’s, our pediatric neurosurgeons use an intra-operative magnetic resonance imaging (MRI) machine to guide surgeries, ensuring that they remove as much of the tumor as possible, while sparing healthy brain tissue. For many children, this means fewer surgeries and a better outcome. 
    • Diagnostic imaging tests are important before, during and after treatment. Boston Children’s has many types of imaging machines, including a brand new pediatric Positron Emission Tomography (PET) scanner. 
    • For Fanconi anemia, advanced genetic testing at Children’s Hospital’s CAP/CLIA-certified laboratory and DEB testing and retrovirus complementation analysis at Dana-Farber guide our team in making the best treatment decisions. 

    Scientific Achievements

    More than 60 years ago, Sidney Farber, MD, founded the Childhood Cancer Program, the first pediatric cancer program in Boston. Dr. Farber refused to accept that childhood cancer was untreatable, and his determination led to the development of chemotherapy and the first remissions of childhood leukemia. 

    Since then, we have been carrying on Dr. Farber’s legacy through research that has yielded many of the advances in care used today. Here are some highlights of our team’s accomplishments:

    • introduction of multiple-agent treatment (combination chemotherapy) for leukemia in 1972. With successful improvements, we attained childhood leukemia cure rates of 80 percent or more.
    • A molecular switch called BCL11a may hold the key to forcing the red blood cells of patients with sickle cell disease to stop making the mutated adult form of hemoglobin and instead produce the fetal form, which is unaffected by the sickle cell mutation.
    • Leadership in developing the first effective chemotherapy for acute myelogenous leukemia (AML).
    • Gene therapy holds great promise for correcting Fanconi anemia (FA), a rare, inherited bone marrow failure disease.
    • New insights into why cells from patients with FA resist efforts to reprogram them into induced pluripotent stem cells could help bring the promise closer to reality.
    • Leadership in developing a new drug called defibrotide as a promising new treatment for hepatic veno-occlusive disease, a major complication following stem cell transplant.
    • Patients with genetic immunodeficiencies like X-linked severe combined immuodeficiency syndrome (X-SCID) or Wiskott-Aldrich syndrome are also benefitting from new research into gene therapy techniques. Translational and clinical research is making this form of treatment safer and more effective than ever before.
    • Leadership in developing national standards of care for pediatric cancer survivors and pediatric palliative care.
    • One of the main clinical features of sickle cell disease is the intense pain associated with veno-occlusive crises. A clinical trial based on a new model of how these crises occur could result in a new, targeted way to put a halt to the pain.
    • A new trial is investigating stem cell transplant without the use of irradiation of the recipient in Fanconi anemia patients with bone marrow failure.
    • The first published experience with double autologous peripheral blood stem cell transplant for high-risk neuroblastoma.
    • Leadership in clinical trials for Ewing sarcoma; five-year event-free survival rates now at about 70 percent.
    • Initiation of the first antiangiogenic treatment trials for pediatric brain tumors.
    • The first pediatric hospital installation of a mobile “intraoperative MRI,” which allows surgeons to obtain images while the patient is still under anesthesia and to perform further procedures as needed.
    • Leadership in advancing survival rates for hard-to-treat tumors like medulloblastoma and atypical teratoid rhabdoid tumor (ATRT).
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