Foundation Medicine's Molecular Information Supports Discovery of Precision Therapeutics for Pediatric Cancers
- Data from a Comprehensive Genomic Dataset of 1,239 Pediatric Cancers Presented Today in a Late-Breaking Poster Session at the AACR Annual Meeting -
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These findings were presented in a late-breaking poster titled "Genomic
profiling of 1239 diverse pediatric cancers identifies novel discoveries
across tumors" by
The poster presentation details genomic profiles from 1,239 pediatric tumors (ages 0-18). Multiple studies are actively investigating clinically relevant genomic alterations in common tumor subtypes for therapeutic exploitation. As demonstrated by the identification of novel fusions and mutations, this data set offers significant discovery potential and can be used to generate hypotheses, validate rare findings, and investigate the genomic landscape of rare tumors in a pediatric population for which only small studies currently exist. Key findings include:
- Genomic profiles from 51 disease subtypes representing sarcomas (26.6 percent), other blastomas (22.4 percent), brain tumors (20.4 percent), hematological malignancies (19.5 percent), carcinomas (9.8 percent) and gonadal tumors (1.4 percent).
- Alterations with proven clinical actionability in pediatric cancers (BRAF V600E and ALK, NTRK1 and ABL1 fusions) were found in 3.9 percent of samples across brain, sarcoma and hematologic cases.
- Three novel ALK fusions were identified in a neuroblastoma (BEND5-ALK), a soft tissue sarcoma (IGFBP5-ALK) and an astrocytoma (PPP1CB-ALK), respectively. Two novel BRAF fusions were also found in an astrocytoma (BCAS1-BRAF) and a ganglioglioma (TMEM106B-BRAF).
- This large data set also challenges the paradigm of "disease-specific" alterations as previously characterized fusions involving ALK, NTRK1 and PAX3 were observed in novel diseases from which they were originally reported.
Pediatric cancers are a rare and diverse collection of diseases.
Childhood cancer rates have been rising slightly for the past few
decades, with The
Cautionary Note Regarding Forward-Looking Statements
This press release contains "forward-looking statements" within the
meaning of the Private Securities Litigation Reform Act of 1995,
including, but not limited to, statements regarding the ability of data
collected from comprehensive genomic profiling of pediatric cancers,to
support the discovery of precision thereapeutics for pediatric cancers,
All such forward-looking statements are based on management's current
expectations of future events and are subject to a number of risks and
uncertainties that could cause actual results to differ materially and
adversely from those set forth in or implied by such forward-looking
statements. These risks and uncertainties include the risk that the
molecular information contained in the pediatric dataset is not suitable
for research purposes, the pediatric dataset is not accessible,and the
risks described under the caption "Risk Factors" in
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