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Androgen Signaling Drives Lethal Pediatric Ependymoma, Study Finds

The study points to a druggable hormone pathway for a cancer with few options.

Overview

  • The research team, publishing Wednesday in Nature, links male hormone activity to the growth of posterior fossa type A ependymoma in young children.
  • In mouse models and patient-derived tumor cells, added androgens sped tumor growth while drugs that block androgen signaling reduced proliferation.
  • The experiments separate hormones from sex chromosomes, showing the male bias in cases and worse outcomes stems from androgen activity rather than XX or XY status.
  • Scientists highlight the prospect of testing existing anti-androgen medicines used for prostate cancer, with pediatric safety and dosing work needed before clinical trials.
  • The data suggest androgens keep tumor cells stuck in an immature, fast-growing state that appears specific to this rare hindbrain cancer, which is more common in boys and often emerges around age three.