Researchers at Children’s National Hospital identified a vulnerability in a developmental signaling pathway that can be hijacked to drive pediatric low-grade glioma (pLGG) formation, according to a study published in Developmental Cell. The study demonstrated that targeted treatment prevents tumor formation, long before irreversible damage to the optic nerve can cause permanent loss of vision. 

“The evidence presented can inform chemoprevention therapeutic trials for children with NF1-OPG,” said lead researcher Yuan Zhu, “This therapeutic strategy may also be applicable to children with developmental disorders that are at high risk of developing pediatric tumors, such as other RASopathies.”

 In this study, researchers in Zhu’s lab identified cells that were MEK/ERK pathway-dependent and grew during a transient developmental window as the lineage-of-origin for NF1-OPG in the optic nerve. The researchers used a genetically engineered pre-clinical model to design a transient, low-dose chemo-preventative strategy, which prevented these tumors entirely.

Search Antibodies
Search Now Use our Antibody Search Tool to find the right antibody for your research. Filter
by Type, Application, Reactivity, Host, Clonality, Conjugate/Tag, and Isotype.

“When we provided a dose-dependent inhibition of MEK/ERK signaling, it rescued the emergence and increase of brain lipid-binding protein-expressing (BLBP+) migrating GPs glial progenitors, preventing NF1-OPG formation,” the team said. “Equally importantly, the degree of ERK inhibition required for preventing NF1-OPG formation also greatly improved the health and survival of the NF1-deficient model.”