Functional Interrogation of Nuclear Export Inhibition in SMARCB1-Deficient Cancers
Abstract
Dr. Hong is a pediatric oncologist at the Dana-Farber/Boston Childrens Cancer and Blood Disorders Center. He has spent the past decade training as a pediatrician and then as a pediatric oncologist in Boston. Over the past several years, he has undertaken laboratory research under the mentorship of Dr. Willaim C. Hahn at the Dana-Farber Cancer Institute and Broad Institute to identify new medicines that may benefit our children and young adults diagnosed with cancer. The Peer Reviewed Cancer Research Program Career Development Award will enable Dr. Hong to pursue his goal of transitioning to an independent physician-scientist leading the charge on a set of rare cancers that affect children and young adults. Although our active duty Service members and their beneficiaries are young and healthy, cancers occur. While we have made progress in cancers like acute lymphoblastic leukemia, we have a long way to go with brain tumors like atypical teratoid/rhabdoid tumor, kidney cancers like renal medullary carcinoma or rhabdoid tumor, or muscle cancers such as epitheliod sarcomas. Interestingly, all of these cancers share a common theme – loss of a gene called SMARCB1. Currently, we treat these patients with aggressive medicines and radiation therapy, but our outcomes remain very poor. New therapies and targets are sorely needed. Our recent work has identified a class of medicines currently in early phases of testing in other types of cancers such as leukemias. This class of medicines affects how the cancer cell is able shuttle proteins between different parts of the cell. By changing the dynamics of what proteins are in what compartment of the cell, we can cause the cancer cells to die. We believe we can utilize this shuttling process to our advantage in this group of cancers. Our proposal will aim to shed light on how the shuttling of different proteins leads to cell death. We will then see if we can combine this medicine with another medicine we have identified recently to improve outcomes in our pre-clinical models. This will pave way to developing a clinical trial to determine if we can improve outcomes by using this regimen compared to our current standard of care. Our ultimate goal of the proposed research is to identify new medicines that can be given to our active duty Service members or their beneficiaries if they are diagnosed with this set of cancers.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Nov 19, 2019
- Source ID
- W81XWH1910281
Entities
People
- Andrew L Hong
Organizations
- Dana–Farber Cancer Institute
- United States Army