Facebook Tracking Image

National Comprehensive Cancer Network

Members Only
NCCN Oncology Research Program (ORP)
NCCN Lilly Abemaciclib Research Grant Opportunity: Submit by Friday, July 10, 2020
Study Proposal Submission-NCCN iEnvision
NCCN Pfizer Axitinib Research Grant Opportunity: Submit by Wednesday, August 12, 2020
ORP Recent News
ORP Scientific Publications
ORP for Industry
ORP Investigator Steering Committee
ORP Testimonials
Clinical Trials at NCCN Member Institutions
Find NCCN ORP Funded Clinical Trials at NCCN Member Institutions
ORP Grant History
Informed Consent Language Database
Points to Consider on the Best Practices for Biorepositories, Registries and Databases
NCCN Shared Resources
ORP Disclosure Policy
NCCN Collaboration with the National Business Group on Health
NCCN Health Information Technology Licensees
NCCN Policy Priorities

NCCN Research & Business Resources

Find a clinical trial >

Pediatric Oncology


The Pediatric Oncology Program stands at the forefront of childhood cancer research and patient care. All Pediatric Oncology faculty members are board certified or eligible and are internationally recognized as leaders in their field. Other pediatric sub-specialists participate closely in both investigative work and clinical care with colleagues in surgery and neurosurgery, urology, gastroenterology, otolaryngology, orthopedics, critical care, psychiatry, and radiation oncology. Their programs also interface with the cutting edge work being done in medical oncology at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, particularly in the areas of leukemia, Hodgkin lymphoma, brain tumors, bone marrow transplantation, sarcomas, histiocytosis, and the development of novel differentiation therapies and cancer vaccines. The Pediatric Oncology and Hematology Programs at Johns Hopkins has also programmatically partnered with the Pediatric Oncology Branch at the National Cancer Institutes at NIH, most notably with a joint fellowship training program in Pediatric Hematology/Oncology. The faculty emphasizes translational laboratory and clinical research, which has had significant impact on cancer therapy. The Pediatric Oncology Program is a division of The Johns Hopkins Children's Center and The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins.

The pediatric oncologists have expertise in the management of all childhood cancers and are world-renowned experts in the areas listed below.

Special Expertise

· Aplastic Anemia and Autoimmune Disorders

· Bone and Soft Tissue Sarcomas

· Bone Marrow Transplantation

· Histiocytosis

· Hodgkin disease

· Immunology of Cancer

· Late Effects and Survivorship

· Leukemia

· Neuro-Oncology

· Non-Hodgkin Lymphoma

· Chemotherapy Resistant Cancers

General Information

Ages Treated

Children and adolescents through age 21

Pediatric Oncology Daytime Line


General Information


24 hour Referral & Information

800.765.JHHS (800.765.5447)

410.955.9444 - The Hopkins Access Line (HAL)

410.283.2333 - Clinical Fellow


CMC 8 - Inpatient Unit

600 North Wolfe Street

Baltimore, Maryland 21287

Facility Description

The Pediatric Oncology Program at Johns Hopkins Kimmel Cancer Center offers a 20 private room unit that is completely HEPA-filtered and has short stay and outpatient capabilities in the Children's Center. The unit is staffed by specialized nurses trained in the special needs of children and adolescents with cancer as well as those undergoing blood or bone marrow stem cell transplantation for cancer and/or a wide variety of inherited disorders including immunodeficiency, sickle cell anemia, storage diseases, and autoimmune and histiocytic disorders. The outpatient clinic is in a connecting building. Both inpatient and outpatient units have been completely renovated in 2005.

The Pediatric Oncology and Transplantation Unit is a special wing of the Harriet Lane Pediatric Service and Children's Center. This unit is especially designed to provide a positive and comfortable experience for the pediatric oncology patient and family by staff devoted to and trained in pediatric hematology, oncology, and transplantation.

Travel Assistance

Johns Hopkins USA provides out-of-town patients with toll-free access to services from appointment coordination to guidance on hotels, transportation, and preferred routes of travel. Call 800.225.2201 or 410.614.1911, Monday through Friday, 9:00 AM to 5:00 PM, Eastern, or fax to 410.614.5196.


The Children's Center House is across the street from Hopkins. Also, there is the Lucas Livingston suite available at a nearby hotel for Pediatric Oncology patients. A Ronald McDonald House across town is served by shuttle bus. The Children's House at St. Casimir is five to seven minutes by car. Social workers assist in lodging arrangements for families.

The Children's House at Johns Hopkins

1915 McElderry Street

Baltimore, MD 21205


One block from Hospital's main entrance.

The Ronald McDonald House

635 W. Lexington Street

Baltimore, MD 21201


Accessible by Metro subway or by Hopkins shuttle van; call
410.502.6880 for arrangements.

The Children's House at St. Casimir

2712 O'Donnell Street

Baltimore, MD 21224


Five to seven minute drive by car.

Social Support

Psychological, social, and practical services are comprehensive and tailored to meet specific needs throughout treatment, rehabilitation, and recovery. Rehabilitation medicine, child life, and social work services are offered to help families and their children with special problems during treatment.

Every pediatric oncology patient has a social worker assigned to him or her to meet individual and family needs. Child Life Specialists staff both in- and outpatient facilities and provide educational and recreational programs for patients and siblings from preschool age through adolescence. Support groups are also available. Patients can participate in Camp Sunrise, the American Cancer Society camp operated for children with cancer.

Home Health Care

"Pediatrics at Home" uses Johns Hopkins-trained pediatric oncology nurses to provide appropriate medical services at home.

Clinical and Research Information

Pediatric Oncology Director

Donald Small, MD, PhD

Multidisciplinary Teams

Each patient is assigned a primary pediatric oncology physician, primary nurse, social worker, and physician's assistant who work as a team with the patient and family from diagnosis through long-term follow-up. Colleagues from Pathology, Radiology, Surgery, Radiation Oncology, and Infectious Disease participate in the weekly Leventhal Pediatric Oncology Case Conference for comprehensive treatment planning.

Cooperative Group Membership

· Childrens' Oncology Group (COG)

· Pediatric Oncology Experimental Therapeutics Investigators Consortium (POETIC)

· Pediatric Blood and Marrow Transplant Consortium (PBMTC)

· National Marrow Donor Program (NMDP)

· Blood and Marrow Transplant Clinical Trials Network (BMT CTN)

Cooperative Group Activities

Participation in COG committees include:

· Bone Tumors

· Soft Tissue Sarcoma

· Germ Cell

· Hodgkin Disease

· Late Effects

· Acute Myeloid Leukemia

· Acute Lymphoblastic Leukemia

· Juvenile Myelomonocytic Leukemia

· Osteosarcoma

· Central Nervous System Tumors and Pathology of Brain Tumors

· Pathology Discipline

· Stem Cell Transplantation Discipline

· Radiation Oncology Discipline

· Biopathology/Translational Research Committee (BTRC)

· Developmental Therapeutics

· Adolescent/Young Adult

Oncologists are national study chairs on numerous studies and hold leadership roles in several disease and discipline committees.

Average Number of Pediatric Clinical Trials


Pediatric Clinical Trial Coordinator

Tammy Scott, RN - 410.614.5990

Research Efforts

The Division of Pediatric Oncology is committed to the discovery and translation of new knowledge into more effective and less toxic therapies for children and adolescents with cancer. Research on the basic biology of how cancer develops and how cancer cells grow and avoid recognition by the immune system should lead to novel and more specific targets to treat cancer. The extensive interactions of Physician-Scientists in the Division of Pediatric Oncology with members of Medical Oncology at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Johns Hopkins Medical and Graduate School Campuses, and colleagues at the National Institutes of Health optimize the ability to rapidly translate new discoveries into treatments.

Investigators are working to gain an understanding of how the survival, proliferation, and differentiation of normal and malignant stem cells are regulated. Researchers hope to translate their findings into improved therapies, specifically blood and bone marrow transplantation and gene therapy. Researchers are also gaining molecular insights into the normal function of blood cells and how they proliferate in order to form a better understanding of how leukemias originate so they may develop novel therapies for this common type of childhood cancer.

Special Expertise/Descriptions

Faculty Member

Areas of Expertise

Robert J. Arceci, MD, PhD

Pediatric Oncology, Development of molecular and immunotherapeutic targets for leukemias and histiocytic disorders

Michael Borowitz, MD

Hematopathology, Leukemia

Patrick Brown, MD

Molecularly targeted therapies for leukemia

Peter Burger, MD


Benjamin Carson, MD


Allen R. Chen, MD, PhD

Clinical and translational studies in BMT

Curt Civin, MD

Pediatric Hematology, Normal and malignant hematopoiesis, stem cell biology

Kenneth J. Cohen, MD, MBA

Pediatric Oncology, Clinical and translational studies in Neuro-Oncology

Paul Colombani, MD

Pediatric Surgery

Anne Fischer

Pediatric Surgery

Frank Frassica, MD

Orthopedics, Sarcomas

Alan D. Friedman, MD

Pediatric Oncology, Transcriptional Regulation of Myeloid Differentiation and Action of Myeloid Oncoproteins

Meghan Higman, MD

Pediatric Oncology, EBV Associated Malignancies and BMT

George Jallo, MD

Pediatric Surgery

Henry Lau, MD

Pediatric Surgery, Liver Tumors

David M. Loeb, MD, PhD

Pediatric Oncology, Molecular Biology of AML

Steven Lietman

Orthopedics, Sarcomas

Charles Paides

Surgery, Sarcomas

Ido Paz-Priel, MD

Transcriptional Regulation in Leukemia

Elizabeth Pearlman

Pathology, Wilms' Tumor, Germ cell tumors, Ewing's Sarcoma

Donald Small, MD, PhD

Pediatric Oncology, Molecular Biology of Normal Hematopoiesis and Leukemia, Development of molecularly targeted therapies for ALL and AML

Kristy Weber, MD

Surgery, Orthopedics, Sarcomas

John Weingart, MD


Moody Wharam, MD

Radiation Oncology, Rhabdomyosarcoma

Elias Zambidis, MD, PhD

Pediatric Oncology, Human Developmental Hematopoiesis and Embryonic Stem Cell Biology

Adjunct Teaching Faculty

Areas of Expertise

Frank M. Balis, MD (NIH)

Clinical pharmacology and drug development

Stephen J. Chanock, MD (NIH)

Immunocompromised host defenses and genetic risk factors for infection

Lee J. Helman, MD (NIH)

Molecular pathogenesis and immunotherapy of sarcomas

Gregory Kato, MD (NIH)

Translational studies in sickle cell disease

Javed Khan, MD (NIH)

Pediatric cancer genomics

Crystal Mackall, MD (NIH)

Immune reconstitution, immunotherapy

Carol J. Thiele, PhD (NIH)

Molecular biology of neuroectodermal tumors, regulation of cellular differentiation pathways

Alan S. Wayne, MD (NIH)

Clinical trials in hematopoietic malignancies

Thomas J. Walsh, MD (NIH)

Diagnosis and treatment of infections in immunocompromised hosts, antifungal pharmacology

Brigitte Widemann, MD (NIH)

Clinical pharmacology and new drug development for children with cancers and neurofibromatosis type 1

Jon Wigginton, MD

Immune-mediated antitumor mechanisms and biological therapy of pediatric cancers

Aplastic Anemia and Autoimmune Disorders

In collaboration with Medical Oncology, a novel approach to treating patients with these disorders using high-dose cyclophosphamide, rather than using bone marrow transplantation, is being tested. Thus far, this approach has shown excellent results.


Bone and Soft Tissue Sarcomas
Johns Hopkins operates a multidisciplinary program led by national experts in the chemotherapy, radiation oncology, orthopedic surgery, and pathology of bone sarcomas. New approaches to therapy are being developed. In collaboration with Radiation Therapy, a protocol has been developed using the radioactive isotope Samarium 153 to treat patients with osteosarcoma.

Bone Marrow Transplant

The Pediatric Bone Marrow Transplantation Service is an international leader, attracting patients from around the world, and is renowned for performing mismatched and unrelated transplants in children. Johns Hopkins performs BMT for solid tumors such as neuroblastoma, Wilms' Tumor, Retinoblastoma, Ewing's sarcoma, hepatoblastoma, and hematologic malignancies as well as for the range of non-malignant diseases of cells derived from the hematopoietic stem cell. Pediatric BMT patients have access to the same services provided in the adult program, including the Cell Therapy Laboratory and the Graft vs. Host Disease Clinic.

The ability to isolate stem cells, the cells in the bone marrow that give rise to all blood cells, was made possible through a discovery made by Pediatric Oncology researchers. This new technology has improved the success of bone marrow transplants and reduced both recovery time and potentially fatal complications, such as bleeding and infection. Their oncologists also developed multiple approaches for autologous and allogeneic bone marrow transplant, including non-total body irradiation preparative regimens, CD34+ stem cell purification for resistant pediatric solid tumors, and CD34+ stem cell purification for T-cell depletion in allogeneic bone marrow transplant. New programs using nonmyeloablative approaches for treating patients with nonmalignant conditions as well as cancer are currently being tested. Novel clinical trials are ongoing to make unrelated and haploidentical bone marrow transplantation safer and more effective. Expansion of progenitor cells from cord blood for transplantation is also being explored.

Histiocytic Disorders

The division has a comprehensive program of consultation and care for treatment of patients with Langerhans Cell Histiocytosis (LCH) and other forms of histiocytosis. In addition, research is being directed toward the development of antibody-targeted approaches for diagnosis and treatment of patients with LCH.

Hodgkin Disease

Recognized as national experts in treating Hodgkin disease with chemotherapy and radiotherapy, Johns Hopkins places emphasis on long-term survivors and late effects of treatment. Investigators at Johns Hopkins are leading the national Children's Oncology Group trial testing immunomodulation after bone marrow transplantation inpatients with Hodgkin disease. Additional research involves the immunologic responses to EBV antigens in HD patients and investigations of the Biology of this disease.

Immunology of Cancer

Researchers are developing molecular/immunologic detection of cancer cells and prognostic features. Vaccination approaches for leukemia, lymphomas, and solid tumors are actively being developed and tested.

Late Effects and Survivorship

The Sidney Kimmel Comprehensive Cancer Center at Hopkins operates a long-term childhood cancer survivors follow-up program as a multidisciplinary regional resource. Research studies in long-term effects of cytotoxic effects are ongoing. Specific emphasis includes cardiac function in survivors of Hodgkin's disease, mechanisms of fatigue in long-term survivors, neuro-cognitive effects of ALL therapy, and design of follow-up programs for adult survivors of childhood cancers.


The first studies on the use of timed sequential therapy for the treatment of patients with acute myelogenous leukemia (AML) took place at Johns Hopkins Kimmel Cancer Center and set the foundation for the current clinical trials using this approach. Members of the Division have been instrumental in the discovery of genes involved in the regulation of growth, programmed cell death, and drug resistance of leukemia cells. This work has contributed to the development of several novel treatment approaches including antibody directed targeting, novel approaches to differentiation therapy, and vaccination approaches for patients with AML. Novel clinical trials using discoveries made by Johns Hopkins investigators are being tested in patients with highly resistant leukemia.


Pediatric brain tumors are the group of tumors with the highest mortality rate of any tumors as a group in children. Johns Hopkins has pioneered many upfront approaches to the most deadly brain tumors, which has been widely adopted by other cancer facilities and in cooperative group settings. Combined modality approaches using surgery, chemotherapy, and irradiation have greatly reduced relapses for many types of solid tumors, including brain tumors.

Non-Hodgkin Lymphoma

Vaccine development for the treatment of EBV- (Epstein-Barr Virus) related lymphoma is a major focus of the center. In addition, the use of autologous transplantation and non-myeloablative transplant approaches are being tested.

Chemotherapy Resistant Cancers

Investigators are developing new treatment strategies to overcome resistant cancers through the identification of specific mechanisms that lead to resistance to chemotherapy and radiation therapy. Phase I and II trials testing new approaches are available.



Percent of children treated in each age range

Age Range





Percent Treated





Pediatric Oncology Program Fiscal Year 2008

No. of Inpatient Beds

No. of Oncologists

No. of Admissions

Average Length of Stay (days)

No. of New Outpatients

Total Outpatient Visits

No. of Bone Marrow Transplants