JOHNS HOPKINS ALL CHILDREN’S HOSPITAL

Medulloblastoma (MB), a central nervous system tumor that predominantly affects children, requires aggressive therapy in all cases. It frequently recurs as resistant to treatment and is associated with high morbidity and mortality. In addition, affected individuals often suffer from disorders secondary to therapy. About 20% of all childhood brain tumors are medulloblastomas (MBs) and in a recent single-cell sequencing study, using cells from the developing murine brain (cerebellum) revealed that different molecular subgroups of childhood cerebellar tumors reflect the transcription of cells from distinct, temporally restricted cerebellar lineage.

There are four major molecular subgroups MBs: wingless-type (WNT)-activated MB (10%; children and adults, associated with very good prognosis), sonic hedgehog (SHH)-activated MB (30%; intermediate prognosis; infants and adults), Group III MB (25%; poor prognosis; infants and children), and Group IV MB (35%; intermediate prognosis; children and adults). Unsurprisingly ongoing transcriptomic and genome-wide methylation studies are revealing even greater heterogeneity in these subgroups.

By applying machine-learning classification to publicly available 175 genome-wide transcriptomics (RNA-seq) and 742 Affymetrix microarray data sets, researchers have identified several subgroup-specific MB lncRNAs. Among them, lncRNA HLX2-7 was highly upregulated in Group III MBs compared to other groups.
These proposed studies will hope to provide insights into the interplay between HLX2-7 and its partner molecules and how these interactions are involved in normal early brain development, and how these mechanisms are altered in the development of malignant medulloblastomas. These studies also look to provide novel and general insights into how lncRNAs regulate cellular states through their interactions with target molecules. Ultimately, the data should pave the way for new lncRNA-based diagnostics and therapeutics for human medulloblastomas.

Long-term goal is to improve understanding of tumor metabolism to design more effective therapies.
Long-term goal is to improve understanding of tumor metabolism to design more effective therapies.
New biorepository will provide the technology to grow and store cancer cells for use in brain tumor research.
New biorepository will provide the technology to grow and store cancer cells for use in brain tumor research.
Together, these Atlanta institutions pioneer the use of nanotechnology to gauge tumor size, stop tumor growth, and shrink tumors.
Together, these Atlanta institutions pioneer the use of nanotechnology to gauge tumor size, stop tumor growth, and shrink tumors.
Research focused on uncovering the mechanisms by which gene fusions contribute to tumor formation in pediatric low grade gliomas.
Research focused on uncovering the mechanisms by which gene fusions contribute to tumor formation in pediatric low grade gliomas.
Objective is to determine if an electrical stimulations regimen can arrest the division of dividing brain tumor cells.
Objective is to determine if an electrical stimulations regimen can arrest the division of dividing brain tumor cells.
Working to find biomarkers for brain tumors to make the diagnosis of tumor types easier, track their growth or even, no recurrence.
Working to find biomarkers for brain tumors to make the diagnosis of tumor types easier, track their growth or even, no recurrence.
Research establishing the feasibility of using interstitial infusion for treating pediatric brain stem gliomas. FDA approved clinical trial underway.
Research establishing the feasibility of using interstitial infusion for treating pediatric brain stem gliomas. FDA approved clinical trial underway.
Research focused on proteins which are active in pediatric low-grade gliomas. From this research, NYU hopes to better select molecular targeted drugs directed at these pathways.
Research focused on proteins which are active in pediatric low-grade gliomas. From this research, NYU hopes to better select molecular targeted drugs directed at these pathways.
Research focused on using new brain imaging techniques to improve diagnosis, prognosis and treatment of pediatric brain tumors.
Research focused on using new brain imaging techniques to improve diagnosis, prognosis and treatment of pediatric brain tumors.
Partnership between a physician and research scientist is examining innovative drug delivery methods for children with brain tumors.
Partnership between a physician and research scientist is examining innovative drug delivery methods for children with brain tumors.
This groundbreaking project could be the first step for the development of a variety of attractive modalities targeting therapeutic approaches beyond immunotherapy.
This groundbreaking project could be the first step for the development of a variety of attractive modalities targeting therapeutic approaches beyond immunotherapy.
Discovery regarding cancer cells' ability to hijack the brain's nerves could lead to new treatment avenues for aggressive brain tumors.
Discovery regarding cancer cells' ability to hijack the brain's nerves could lead to new treatment avenues for aggressive brain tumors.
Developing a strategy to ensure rapid translation of new drug candidates into clinical trials of medulloblastomas is a collaborative effort.
Developing a strategy to ensure rapid translation of new drug candidates into clinical trials of medulloblastomas is a collaborative effort.
University of Michigan Researchers aim to treat Pediatric Brain Tumors through the combination of Gene and Immune Therapy.
University of Michigan Researchers aim to treat Pediatric Brain Tumors through the combination of Gene and Immune Therapy.
Researchers at Johns Hopkins All Children’s and Johns Hopkins University hypothesize that a group of lncRNAs, including lncRNA HLX2-7, are key molecular signatures (biomarkers) and therapeutic targets for Group III medulloblastoma in children.
Researchers at Johns Hopkins All Children’s and Johns Hopkins University hypothesize that a group of lncRNAs, including lncRNA HLX2-7, are key molecular signatures (biomarkers) and therapeutic targets for Group III medulloblastoma in children.
Scientists work to establish a new system for targeting oncogenic mutations in pediatric brain tumors.
Scientists work to establish a new system for targeting oncogenic mutations in pediatric brain tumors.