Childhood Cancer Research

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Children’s Hospital Los Angeles

4650 Sunset Blvd.
Los Angeles, CA 90027
United States

Ewing sarcoma (ES) is a malignant cancer of bone and soft tissues that occurs mainly in children, adolescents and young adults. Currently, combinations of intensive chemotherapy, surgery and radiation are only 70% effective for localized tumors, and these therapies can cause lifelong health problems in survivors. If the tumors spread, fewer than 1/3 of patients will survive. New therapeutic advances are needed that are more effective with fewer toxic side effects.

Diffuse midline glioma (DMG) is a deadly pediatric brain cancer. Despite radiation treatment, nearly all children diagnosed with the disease succumb to it with a median survival of 12 months. There have been few advancements in treatment, and current treatment has no curative intent. Therefore, there is a dire need to develop new therapeutic strategies to improve the terrible outcomes for these children and improve quality of life. Looking at genes that are turned on or off in a particular type of cancer can be helpful to figure out what is causing the cancer to grow.

Rhabdomyosarcoma is the most common type of muscle cancer in children. When this cancer spreads to other parts of the body (a process called metastasis), it becomes much harder to treat, and survival rates drop to as low as 20%. Therefore, understanding how this cancer spreads is crucial for developing better treatments. One type of this cancer, fusion-positive rhabdomyosarcoma (FPRMS), has a special abnormal gene that combines two genes, PAX3 and FOXO1. This type tends to spread more than the other type that doesn't have this fusion gene.

Retinoblastoma (RB) is the most common eye cancer, affecting ~8000 children every year worldwide. This cancer can form in one or both eyes of babies and toddlers. Untreated, RB can lead to death of the child, however even with treatment the tumor may respond poorly and requiring the eye to be removed in 50% of advanced cases. There is a need to better understand the mechanisms behind treatment resistance and relapse for children with RB. For a long time we were limited in understanding this because RB tumors cannot be directly biopsied due to risk of cancer spread outside the eye.

As featured in the 9/2013 ALSF E-News:

“ALSF support was very important for the continuation of our studies on ALL.” – Nora Heisterkamp, PhD

Mentor Name: James Amatruda

Mentor Name: Jessica Tsai

Mentor Name: Jesse Berry

The analysis of cell-free DNA (cfDNA) from liquid biopsies, such as blood or cerebrospinal fluid, is a rapidly growing area for clinical innovation. To date, most clinical assays have been developed for solid tumors in adults, which require large volumes of fluid that cannot be safely obtained from young children.

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