Development of Chromosome 1q+ Specific Treatment for Highest Risk Pediatric Posterior Fossa Ependymoma.

Andrea M Griesinger Annaliese J Calzadilla Enrique Grimaldo Andrew M Donson Vladimir Amani Angela M Pierce Jenna Steiner Soudabeh Kargar Natalie J Serkova Kelsey C Bertrand Karen D Wright Rajeev Vibhakar Todd C Hankinson Michael Handler Holly B Lindsay Nicholas K Foreman Kathleen Dorris

Published in: Clinical cancer research : an official journal of the American Association for Cancer Research (2024)

These results are the first to identify a chromosome 1q+ specific therapy approach in 1q+ PFA. Existing phase I studies have already established single-agent pediatric safety and dosages of 5FU and ATRA, allowing for expedited clinical application as phase II trials for children with high-risk PFA.

Keyphrases

phase ii
clinical trial
open label
copy number
young adults
randomized controlled trial
gene expression
double blind
mesenchymal stem cells
bone marrow
cell therapy