Precision Medicine for Pediatric Brain Tumors
In contrast to adult tumors, recent surveys of pediatric tumors have shown that most are driven by relatively few mutational events, many of which may be targetable with personalized clinically available agents. In collaboration with the U of M MiOncoseq Study (Dr Arul Chinnaiyan and Rajen Mody, PIs), we have an ongoing clinical sequencing study to better understand the role of personalized genomic characterization and precision oncology in the management of children with high-risk brain tumors. We discuss these results in a multi-disciplinary and multi-institutional brain tumor precision medicine conference, with a special emphasis on discussion of the CNS penetration of potential targeted agents.
Our work thus far has revealed a potentially actionable germline or tumor alteration in two-thirds of children with high-risk brain tumors which has resulted in an impact on treatment or change of diagnosis in over half of enrolled patients (Koschmann et al, JCO Precision Oncology, 2018). Ongoing collaborative work with the Koschmann lab and the MiOncoseq program is exploring tying tumor sequencing results to spinal fluid tumor DNA analysis.