Targeting protein folding in N-Myc-driven medulloblastoma

Selective targeting of N-Myc-driven Sonic hedgehog (SHH) medulloblastoma has been a challenge for many years and, despite decades of research, few targeted therapy opportunities exist. Recently, Kuzuoglu-Ozturk et al. characterized the translatome of N-Myc-driven medulloblastoma as a promising therapeutic target. The study showed that N-Myc controls a subset of members of the protein folding machinery that could be inhibited pharmacologically and validated a subset of Hsp70 functions as required for medulloblastoma progression in vitro and in vivo.

Automated summary

Selective targeting of N-Myc-driven Sonic hedgehog (SHH) medulloblastoma has been a long-standing challenge, with limited targeted therapy opportunities. A recent study by Kuzuoglu-Ozturk et al. characterized the translatome of N-Myc-driven medulloblastoma and identified potential therapeutic targets. The study demonstrated that the protein folding machinery controlled by N-Myc could be pharmacologically inhibited, and validated the necessity of certain Hsp70 functions for medulloblastoma progression in vitro and in vivo.