Title:Childhood Medulloblastoma: Current Therapies, Emerging Molecular Landscape and Newer Therapeutic Insights
Volume: 16
Issue: 7
Author(s): Soumen Khatua*, Anne Song, Divyaswathi Citla Sridhar and Stephen C. Mack
Affiliation:
- Department of Pediatrics, MD Anderson Cancer Center, The University of Texas Health Science Center at Houston, Pediatrics Houston, Texas,United States
Keywords:
Childhood medulloblastoma, targeted therapy, molecular subtypes, epigenetic machinery, newer treatment strategies,
therapeutic resistance.
Abstract: Background: Medulloblastoma is the most common malignant brain tumor in children,
currently treated uniformly based on histopathology and clinico-radiological risk stratification leading
to unpredictable relapses and therapeutic failures. Identification of molecular subgroups have
thrown light on the reasons for these and now reveals clues to profile molecularly based personalized
therapy against these tumors.
Methods: Research and online contents were evaluated for pediatric medulloblastoma which included
latest information on the molecular subgroups and their clinical relevance and update on
efforts to translate them into clinics.
Results: Scientific endeavors over the last decade have clearly identified four molecular variants
(WNT, SHH, Group 3, and Group 4) and their demographic, genomic, and epigenetic profile. Latest
revelations include significant heterogeneity within these subgroups and 12 different subtypes of
MB are now identified with disparate outcomes and biology. These findings have important implications
for stratification and profiling future clinical trials against these formidable tumors.
Conclusion: With the continued outpouring of genomic/epigenomic data of these molecular subgroups
and evolution of further subtypes in each subgroup, the challenge lies in comprehensive
evaluation of these informations. Current and future endeavors are now needed to profile personalized
therapy for each child based on the molecular risk stratification of medulloblastoma, with a
hope to improve survival outcome and reduce relapses.