Brian Rood, M.D.
بررسی اجمالی تحقیق
We are studying medulloblastoma (MB), a common malignant brain tumor in children that originates in the cerebellum. Current treatments, including surgery, radiation and chemotherapy—are not always effective for high-risk subgroups, such as metastatic and recurrent cases.
In our research, we’ve used a novel proteomics approach called Super-SILAC to study MB, which complements genomic data and provides a more comprehensive understanding of tumor biology. This method is particularly valuable for group 3/4 MB, whose molecular characteristics are not well understood.
We identified 157 significantly overexpressed proteins in group 3/4 MB compared to other subgroups, and then discovered nine of these proteins are essential in the continued growth/spread of cancer in this group.
One of these proteins, PTBP2, is a splicing factor involved in neuronal differentiation (turning neural stem cells into neurons). When we knocked it out in MB cells, we observed reduced growth and spread of cancer cells, along with increased neuronal differentiation.
Our project aims to determine PTBP2’s function in group 3/4 MB differentiation and validate whether PTBP2 knockout impairs tumor establishment or progression in the body. We propose that targeting PTBP2 could stop cancer cells from dividing and reduce malignancy. We believe our study highlights the potential of proteomics-based approaches to identify novel therapeutic targets for MB, particularly for the challenging 3/4 subgroup.
My “Why”
During my pediatric hematology/oncology fellowship, I had the opportunity to experience lab research. I was surprised by the amount of creativity required to successfully answer challenging questions in cancer biology. I also recognized the unique opportunity to help in a way that was completely distinct from providing clinical care. I enjoy the ability to help pediatric patients in multiple, complementary ways. Research provides a great deal of intellectual stimulation and control over the process, while clinical care is extremely rewarding in a humanistic way. Combining these two endeavors makes for a very fulfilling professional life.
چرا تامین مالی مهم است
The funding [from the Prevent Cancer Foundation] will significantly advance our research on medulloblastoma (MB) by allowing us to investigate the role of PTBP2 in group 3/4 MB.
Additionally, funding will enable us to expand our proteomics-based approach to identify more therapeutic targets and translate our findings into preclinical studies, ultimately improving treatment options for high-risk MB patients.