JOSEPHINE HADUONG, M.D.

Audrey Coon is a beautiful little girl who has faced more than her share of challenges in her life. But, thanks to the availability of extraordinary medical care and cutting-edge research, Audrey’s story has a happy ending.

In honor of the care team that made her outcome possible, the Coon Family has designated a special fund to forward-thinking researchers in Audrey’s name. The collaborative nature of this project speaks to the importance of working together in the fight to end pediatric cancer. These two leaders in their field represent a cross-country effort to advance treatments and cures for children facing cancer.

The Project:

Recurrent CNS malignancies are almost universally lethal diseases. Novel and targeted treatment is needed to address the high morbidity and mortality of pediatric CNS malignancies. The standard of care for the most common pediatric brain tumor, medulloblastoma (MB), is a balancing act of preserving neurocognitive function while optimizing long-term survival outcome. This leads to avoiding radiation in younger patients to improve morbidity at the expense of overall event free survival, and risk of early, and lethal tumor recurrence. Medulloblastoma has a 30%-60% relapse rate following initial treatment depending on the initial risk status and prognostic features. These children not only suffer significant morbidity as a sequelae of their treatment, but the relapsed tumor is often treatment-resistant. The neurotoxic treatment regimen devastates their quality of life with sequelae including neurological deficits, secondary malignancy, endocrine dysfunction, cognitive difficulties, and psychological and social problems. The limited treatment options for recurrent MB consist of more high-dose chemotherapy, radiation, and experimental therapeutics within clinical trials. Currently, there are no FDA approved drugs for recurrent medulloblastoma, and clinical trials have had little success in changing long term outcomes. New studies are urgently needed to advance therapeutic agents that both preserve quality of life and improve overall survival. That is the goal of the research outlined here.

Patient tissue will be collected in the operating room at CHOC at the time of tumor resection.  This will provide pediatric medulloblastoma tumor samples that then be used to establish pediatric brain tumor cell cultures and xenograft models used for screening.  Our goal is to establish a pediatric medulloblastoma testing pipeline that will lead to the most promising compounds can be translated into early clinical trials for pediatric patients with recurrent brain tumors..

Clinical and treatment data for individual patients will be studied in collaboration with the molecular characteristics of the tumor tissue collected at the time of surgery. The combination of molecular characteristics of primary tumor cultures, including resistance data, imaging characteristics (on MRI and PET scan studies) will serve to discover new biomarkers, the development and validation of novel diagnostic assays, and/or new potential therapies.