You are viewing AANS Neurosurgeon Volume 27, Number 3, 2018. View our current issue, Volume 27, Number 4, 2018

AANS Neurosurgeon | Volume 27, Number 3, 2018

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Targeted treatment shrinks deadly pediatric brain tumors

Chemotherapy and radiation are effective cancer treatments because they kill rapidly dividing cells, including tumor cells. But for children—whose tiny bodies are still growing—these treatments can cause lifelong damage. This is particularly true for children with brain cancer, and researchers are working hard to find treatments that reduce side effects while remaining effective.

Now, scientists at Sanford Burnham Prebys Medical Discovery Institute (SBP) have reported that a targeted therapy that blocks a protein called LSD1 was able to shrink tumors in mice with a form of pediatric brain cancer known as medulloblastoma. LSD1 inhibitors are currently under evaluation in clinical trials for other cancers, which could speed their potential path to children with medulloblastoma.

“Subjecting a developing child to chemotherapy and radiation of the head and spine can leave devastating long-term effects. Some children even become intellectually disabled as a result of the treatment, and aren’t able to go to college, live on their own or achieve other important milestones,” says Robert Wechsler-Reya, Ph.D., senior author of the paper, professor and director of the Tumor Initiation and Maintenance Program at SBP and program director of the Joseph Clayes III Research Center for Neuro-Oncology and Genomics at the Rady Children’s Institute for Genomic Medicine. “Our lab is working to understand the genetic pathways that drive medulloblastoma so we can find better ways to intervene and treat tumors. This study shows that a personalized treatment based upon a patient’s specific tumor type might be within our reach.”

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