Research and Clinical Trials

Our experts play an active role in innovative and prestigious clinical trials to advance the science of pediatric cancer care. We offer your child access to novel treatments for complex conditions, some of which are only available at Children’s National.

Cancer Research and Clinical Trials at Children’s National

With research interests covering nearly every aspect of pediatric cancer care, our work is making great advancements in childhood cancer. For example, some blood cancers that were once fatal now have an average survival rate of 90 percent. This means that nine in 10 children will live a long and happy life.

Highlights of our research efforts include:

  • Access to new treatments: As a designated Children’s Oncology Group (COG) Phase I institution, we are one of only 20 hospitals in the country with access to the latest cancer fighting medications.
  • Advancing the science of cancer care: With more than 100 clinical trials exploring every aspect of pediatric cancer care, all of our patients have an opportunity to help advance the care available for their disease.
  • Seamless care: If your child participates in a clinical trial, he or she will not need to change doctors. Trial leaders work with your child’s existing care team to seamlessly coordinate trial activities into your child’s care.


Since its inception, Children’s National has played a prominent role in COG. Supported by the National Cancer Institute, COG is the world’s largest cancer research group. We are also a proud partner and leader in many other elite cancer research groups, such as the Collaborative Ependymoma Research Network (CERN).

Leukemia and Lymphoma Research

Our Leukemia Lymphoma Program research interests include:

  • Reducing the incidence of relapse: We are looking to improve treatment options for children whose leukemia returns after treatment (relapse).
  • Developing new treatments for high-risk leukemia: We are exploring intravenous (IV) treatments for children with advanced forms of leukemia. This research includes care for acute lymphoblastic leukemia.
  • Experimental therapeutics: This treatment area includes new medications (therapeutics) for cancers that are not responsive to other treatments. Some of these medications are only available in select programs across the country. Learn more about our experimental therapeutics program.
  • Research leadership: Children’s National oncologist, Anne Angiolillo, MD, is the principal investigator of the COG Experimental Therapeutics program. Her work gives our team additional expertise in finding new treatment pathways for complex cancers.

Sarcoma Research

Our Solid Tumor Program’s research interests include:

  • Magnetic resonance-guided high-intensity focused ultrasound (MR-HIFU): We are the first children’s hospital offering noninvasive, experimental treatment for bone and soft tissue tumors. Learn more: Children’s National First in U.S. to Destroy Bone Tumors with Incisionless Surgery.
  • Pathology: Children’s is home to one of the leading pediatric oncology diagnostic specialists (pathologists) in the country, D. Ashley Hill, MD. Dr. Hill has a special interest in rare soft tissue sarcomas affecting the airway and lungs (Pleuropulmonary blastoma).
  • Experimental therapeutics: We are exploring new medications (therapeutics) for cancers that are not responsive to other treatments. Some of these are only available in select programs across the country. Learn more about our experimental therapeutics program.
  • Research leadership: Our sarcoma experts lead a number of national, multi-center trials on behalf of the Sarcoma Alliance for Research through Collaboration (SARC).

Brain Tumor Research

Our Brain Tumor Institute’s research interests include:

  • Early intervention: We are exploring ways to help children with brain tumors receive treatments (interventions) earlier in their disease.
  • Targeted therapies: This work is helping us understand the effects of certain cancer medications on brain development. Our goal is to improve safety by making medications less toxic.
  • Neurofibromatosis: Our long history of researching this condition and its neurological effects on adults and children has brought about important advances in care. Learn more about our Neurofibromatosis Institute.
  • Research leadership: We are one of only 11 sites in North America participating in the Pediatric Brain Tumor Consortium.

Blood and Marrow Transplant Research

Our Blood and Marrow Transplant research interests include:

  • Innovative stem cell sources: One of the biggest challenges with blood and bone marrow transplants is finding the right match for each patient. We are closing this gap by exploring new stem cell sources, such as a child’s biological parent.
  • Cellular therapy: This treatment offers a lifeline to children who relapse after transplant, as well as children who have severe viral infections for which other therapies are not working. Our research explores short- and long-term outcomes in the hopes of making this treatment more widely available in the future. Learn more about cellular therapy.
  • Extra corporeal photopheresis: We are one of few centers in the world offering extracorporeal photopheresis. This treatment helps children who experience dangerous post-transplant complications, such as graft vs. host disease. This treatment was developed in part by one of our physicians, David Jacobsohn, MD.

Collaboration with Children’s Research Institute

We work side by side with colleagues from the Children's Research Institute, the academic division of Children’s National Health System. Children’s Research Institute specializes in uncovering the basic science that behind many of our research interests.

Learn more about clinical research programs at Children’s National.

Contact Us

Meet the team or for more information, call us at 202-476-2800.