Paradigms in Developmental Therapeutics: New Therapeutic Approaches and a New Way of Thinking

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Oncology Update

In the past, early phase clinical trials of cytotoxic agents enrolled children with a variety of refractory or recurrent cancers. As new agents become more specific and target molecular characteristics of specific cancers, clinical trial enrollment may be restricted to children with relapsed cancers that harbor selected mutations.

The physicians and scientists of the Developmental Therapeutics Program at The Children’s Hospital of Philadelphia want to encourage a change in thinking about caring for children with cancers that are metastatic at diagnosis, have high-risk clinical phenotypes or have a high likelihood of recurring or being refractory to standard therapy.

“When a child is diagnosed with a high-risk tumor or a cancer that is likely to relapse, it’s important to consider what options they may need in the future,” says Elizabeth Fox, MD, who heads the Developmental Therapeutics Program at CHOP. “Because new agents for childhood cancer are beginning to target the molecular changes that cause specific cancers, we are considering new agents earlier in the course of therapy for children with cancer.”

Currently, the Division of Oncology at The Children’s Hospital of Philadelphia has many open studies of new therapeutic approaches for cancer in children and adolescents. We encourage physicians to contact us about any patient with resistant, relapsed, or recurrent leukemia or metastatic solid tumor. In our opinion, for high-risk patients, these discussions are never premature. Preliminary discussions may help preserve options for your patient in the future or facilitate referral for enrollment in a clinical trial, if needed.

Our clinical trial of CTL019 (or CART19) in children and adolescents with relapsed leukemia is an example of a study that requires early discussion and planning. For this clinical trial, T cells are collected from each patient by apheresis and engineered ex vivo to express a CD19- directed chimeric T cell receptor. In order to harvest an adequate number of T cells, the apheresis must occur before T cells are depleted by standard cytotoxic reinduction chemotherapy. Early planning is essential.

Our Developmental Therapeutics Program would like to partner with physicians, patients and families to consider potential options for clinical trials earlier in the course of standard therapy. We must work together to ensure that the most promising new anti-cancer strategies are available to children and adolescents and to identify patients with advanced or metastatic cancer who may benefit from innovative approaches.

If you have questions or are considering a referral, please call 888-ONC-CHOP (888-662-2467) to speak to an oncologist.