Miller shares how pediatric cancer treatment advances bring new hope

In a 2018 interview, Dr. Barbara A. Miller, chief of the Division of Pediatric Hematology and Oncology at Penn State Health Children’s Hospital, discussed the significant improvements in the treatment of pediatric cancer over the past four decades that have boosted survival rates.

Dr. Barbara A. Miller is the chief of the Division of Pediatric Hematology and Oncology at Penn State Health Children’s Hospital.

How have treatments for pediatric cancer improved since 1974?

• The treatment regimens are much more complex. Multi-drug regimens are very common, often with higher drug doses and incorporating new or improved drugs.
• Risk-directed therapies are more commonly used. Patients are now often stratified into “low” risk, meaning that their disease responds better to current therapies, and they require less intensive therapy, compared to “high” risk, meaning they are at high risk for a relapse and require more intensive therapies to give a better chance of cure. The genetic and biological criteria for designing therapies are continually being better defined.
• New drugs are available that are biologically targeted and have fewer side effects, and more of these are currently being developed. However, we still need to learn a lot about how to use them, including what diseases they are effective in, how to use them with conventional chemotherapy and with each other, and for how long.
• We are developing genetic testing so that it may precisely characterize the tumor of each patient, so that one day a more individualized approach to all cancers may be possible. We are currently involved in a research study in which the tumors of patients with relapsed or refractory disease – cancer that doesn’t respond to treatment – undergo extensive genetic analysis and for these patients, for whom no effective therapy is known, a targeted therapy using new drugs and based on biological and genetic characteristics of the tumor is designed. This has been possible only in the last few years.
• Supportive care including new antibiotics and the quality of our blood products has improved tremendously.

With these treatments, the overall survival of pediatric cancer patients is 80 percent at five years, but for some subgroups, such as low-risk acute lymphoblastic leukemia, the survival is now 90-95 percent, Miller noted.

What are the biggest challenges of treating cancer in children?

• It takes time to confirm discoveries made in the laboratory and to translate them to patient care. That can be frustrating.
• Relapsed patients can have very refractory disease, and the hope is that one day, we can use the new discoveries up front. The biggest challenge is also our main goal – to be able to cure all children with cancer and to reduce the side effects that current therapies sometimes have.

What do you like the best about your work?

• Patient outcomes and survival are continually improving. This is a very gratifying result of our research, and we thank Four Diamonds and THON for their support.

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