Protecting children from the most toxic forms of cancer treatment

Neuroblastoma is a devastating form of cancer that develops in the nervous system and mostly affects children less than five. Current treatment options aren’t good, and include high-dose chemotherapy, radiation and surgery. The research of physician scientist Dr. Mark Applebaum focuses on a genetic biomarker that can help predict the cancer’s aggressiveness. Knowing the severity, Applebaum can create personalized treatment regimens that insulate children from the most toxic forms of treatment. View Halo Profile >>

Tell us about your research…

I treat children with neuroblastoma, a type of cancer that forms in certain types of nerve tissue. The treatments for these children include high-dose chemotherapy, surgery, stem cell transplants, radiation, and painful immunotherapy. Treatment lasts almost a year and half with much of that time spent in the hospital. My long-term goal is to develop “cancer specific” targeted strategies to improve outcomes while minimizing risk from toxicity. There has been an explosion of new technology and knowledge regarding cancer genomics. I want to harness these innovations to refine and personalize therapy for my patients.

I treat children with neuroblastoma, a type of cancer that forms in certain types of nerve tissue. The treatments for these children include high-dose chemotherapy, surgery, stem cell transplants, radiation, and painful immunotherapy.

Tell us about your research…

We are focusing on one particular epigenetic biomarker as a determinant of cancer aggressiveness. We are exploring how we can use this epigenetic marker to determine how aggressive a child’s tumor is in order to recommend the most appropriate treatment strategy. We have shown that epigenetic profiling of neuroblastoma tumors can serve as sensitive biomarkers for risk stratifying children and improves our understanding of tumor biology. We are using the same approach to evaluate neuroblastoma epigenetic profiles using blood samples. In contrast to biomarkers that require tumor tissue, liquid biopsies are minimally invasive, clinically convenient, and enable dynamic monitoring.

We have shown that epigenetic profiling of neuroblastoma tumors can serve as sensitive biomarkers for risk stratifying children and improves our understanding of tumor biology.

How could it someday impact patient lives?

Early results from blood samples show we can detect metastatic disease burden in children with high sensitivity and this approach has the potential to identify children who are likely to relapse from otherwise clinically undetectable disease. We are developing this test to enable us to determine how aggressive the tumor is at diagnosis, how well it’s responding to chemotherapy, and inform our treatment options based on the biology of the tumor. This methodology has the potential for being rapidly integrated into the clinic, allowing patients to be easily profiled at any time point during treatment.

Early results from blood samples show we can detect metastatic disease burden in children with high sensitivity and this approach has the potential to identify children who are likely to relapse from otherwise clinically undetectable disease.