From the Clinic to the Lab, Understanding Medulloblastoma Relies on Molecular Profiling

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Scientists at St. Jude Children’s Research Hospital and their colleagues have published a detailed account of SJMB03, a clinical trial for pediatric patients with medulloblastoma. Additionally, they report results of the largest analysis of matched primary and relapsed medulloblastoma tumors to date. In both the clinic and the lab, results underscore the need for integrated molecular assessment of these tumors.

Clinical trial confirms subgroups, highlights need for additional analysis

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Medulloblastoma is among the most common malignant pediatric brain tumors. Previous research by St. Jude scientists and others has classified medulloblastoma into four distinct molecular groups: WNT and SHH (which are driven by their namesake genetic mutations), Group 3 and Group 4. Prognosis for medulloblastoma is different for each molecular group. Retrospective analyses have shown that WNT tumors respond the best, with 95% five-year survival. SHH and Group 4 tumors have approximately 75% five-year survival, and Group 3 has 60%.

“This was a large clinical trial that really incorporated biology into the analysis,” said first author Amar Gajjar, MD, chair of the St. Jude Department of Pediatric Medicine. “This allowed us to interpret the outcomes based on clinical and molecular risk features.” 

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