About the Developmental Therapeutics Program

Pediatric cancer researchers at CHOP study all types of childhood cancer, from the rare malignancies to the most common cancers found in children.

They conduct basic, translational and clinical research. This means that our experts are involved in every stage of investigation required to develop a new treatment.

We have more than 50 investigators from a variety of programs including oncology, hematology, pathology, neurology, neurosurgery and radiation oncology. Your child's entire care team is involved in pediatric cancer research; that includes physicians, nurses, psychologists, scientists and other health professionals.

Clinical trials for newly diagnosed and relapsed cancer

What is a clinical trial?

Clinical trials are controlled tests of new drugs or treatments. These studies evaluate the effectiveness of new treatments and therapies for diseases. Clinical trials are among the fastest and safest ways to find treatments that work against pediatric cancer.

Studies have shown that children's survival rates are increased by 20 to 40 percent if their cancer care is coordinated by pediatric oncologists who follow established clinical trial protocols.

The majority of our oncology patients are enrolled in some sort of clinical trial, many of which are developed right here by CHOP's own cancer researchers. In many cases, the same people who developed the trial will also be caring for your child.

Clinical trials at CHOP's Cancer Center

At CHOP, clinical trials for children whose cancer has relapsed (come back) or not responded to standard therapy are managed by the Developmental Therapeutics Program. The team includes some of the world's leading experts in pediatric cancer research and drug development and is part of our Center for Precision Medicine for High-Risk Pediatric Cancer. In partnership with the Division of Genomic Diagnostics, we offer comprehensive tumor profiling to identify the mutations that drive each individual patient’s tumor.

We partner closely with our Cancer Immunotherapy and Cellular Therapy Programs to develop a customized treatment plan for each child, which may include several different types of clinical trials including precision targeted therapies, immunotherapy, and cellular therapies. The goals of the Developmental Therapeutics Program include:

  • Improving the outcomes of children, adolescents and young adults with cancer through clinical trials of new investigational agents and regimens
  • Working with patients, families and their medical team to determine if participation in a clinical trial of an investigational agent or new treatment approach is right for your child

Eligible patients and families enrolled in clinical trials at CHOP for relapsed or refractory cancer have access to the GlaxoSmithKline Hope for Families Fund, to assist with travel and living expenses associated with treatment.

The Developmental Therapeutics Program is focused on improving the efficacy and safety of anti-cancer drug therapy. Preclinical studies and clinical trials are ongoing, with a diverse portfolio of cytotoxic and molecularly targeted anti-cancer drugs. We typically have dozens of clinical trials open to accrual.

  • We are a member institution of national consortia for drug development in childhood cancer, including the Children’s Oncology Group Phase 1/Pilot Consortium and New Advances in Neuroblastoma Therapy.
  • We conduct industry-sponsored clinical trials of new agents. • We develop investigator-initiated clinical trials.
  • We focus on molecularly targeted therapies and novel clinical trial designs with new endpoints and correlative studies, including clinical pharmacokinetics and pharmacodynamics.

Leaders in childhood cancer research

The Cancer Center at CHOP is proud of its long-standing commitment to and leadership in pediatric cancer research. Driving this commitment is the Center for Childhood Cancer Research (CCCR), a Center of Emphasis at The Children’s Hospital of Philadelphia Research Institute.

  • CHOP's designation as a Children’s Oncology Group (COG)/Pediatric Early Phase Clinical Trial Network Phase I and Phase II institution helps us advance our mission to develop more effective, less toxic treatments for childhood cancers by letting our researchers fast-track clinical trials of new treatments for all types of childhood cancer.
  • 100 percent of our cancer physicians are researchers. These clinicians are constantly thinking of ways to immediately apply their research findings to improve treatments for their patients.
  • Children's Hospital of Philadelphia is one of only 21 institutions nationwide conducting COG Phase I clinical trials.
  • Our physician-scientists hold leadership positions in nationally and internationally renowned clinical associations and lead multiple international clinical trials of precision targeted therapies
  • Host to one of the world's largest pediatric cancer sequencing programs. CHOP’s state-of-the-art clinical tumor sequencing laboratory has sequenced more than 4,000 tumors. Our lab utilizes a custom sequencing panel targeted to pediatric cancers, which distinct from many commercial labs that have been developed for cancers in adults. This is critical to detecting genetic changes that drive individual tumors and may be targeted by novel therapies.
  • We partner closely with the Center for Single Cell Biology, a center of emphasis at CHOP and leader in single-cell analysis of pediatric cancers. Researchers in the Center uses a variety of single-cell sequencing and imaging technologies to understand how tumor heterogeneity contributes to cancer development and treatment response.
  • We partner closely with the Center for Data Driven Discovery in Biomedicine and CCCR Precision Medicine Biospecimen Repositories. These groups are working to improve outcomes for children with cancer by collecting and integrating data and biospecimens.
  • Our robust fellowship program will train the next generation of physician-scientists dedicated to pediatric oncology and pediatric cancer research.

Partnering to find a cure

While some of the best pediatric cancer clinicians and researchers in the world call CHOP their home, to ensure we are bringing the latest research and clinical trials to our patients, our physicians, lead, collaborate and participate with many cooperative groups, including:

  • Children's Oncology Group (COG)
  • National Institutes of Health (NIH)
  • American Association for Cancer Research (AACR)
  • American Society of Clinical Oncology (ASCO)
  • American Society for Clinical Investigation (ASCI)
  • American Society of Hematology (ASH)
  • American Society of Pediatric Hematologists/Oncologists (ASPHO)
  • New Approaches to Neuroblastoma Therapy (NANT)
  • Pediatric Brain Tumor Consortium (PBTC)
  • Society for Pediatric Research (SPR)
  • Therapeutic Advances in Childhood Leukemia & Lymphoma (TACL)


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