At Monroe Carell Jr. Children's Hospital at Vanderbilt
At Turner's Heroes, the Discovery Grants that we fund are focused on pediatric cancer research. The panel that chooses which grant will be funded will consist of Noelle and Randy Cockrell, those associated with Turner's Heroes, and donors to Turner's Heroes. Your donations will go towards a specific and innovative grant that has the potential to do amazing in our world so that no family must endure another cancer diagnosis.
With common adult-onset cancers, recent clinical guidelines, combined with approval of efficacy-tested, molecularly-targeted therapies, highlight the importance of tumor (somatic) and normal tissue (germline) DNA testing. In pediatric oncology, while somatic testing is being conducted more commonly, the incorporation of germline testing is not well established. There are emerging data showing that the presence of germline mutations in pediatric cancer patients is not as rare as previously thought. Identification will affect treatment and surveillance and may have a significant impact for other family members.
This growing need in pediatric oncology comes at a time when there is a national shortage of providers trained in cancer genetics, resulting in long wait times for patients and families to undergo genetic testing. Therefore, our goal is to expand access to germline genetic testing by developing algorithms for germline genetic testing in pediatric patients and point of care testing.
We will develop and pilot test an algorithm for referral of pediatric, adolescent ad young adult cancer patients, ages 0 – 21 years, for germline genetic testing and provide that testing in the pediatric oncology clinic, with strong interaction with the hereditary cancer clinic. We will assess barriers and facilitators to the process and assess the impact of the processes on patients, parents and providers. Once established, we can provide this service to pediatric oncology patients outside of Vanderbilt who would otherwise not have access to this service and will pilot direct-to-consumer approaches using telemedicine.
Disordered ribosome biogenesis and function is the underlying cause of a group of diseases called ribosomopathies and this may lead to leukemia. We have discovered that a potential therapeutic target for leukemia arising from disordered ribosomes is WDR5 and furthermore have developed some inhibitors of WDR5.
We seek to investigate this further by identifying ribosomal proteins implicated in adult and pediatric leukemia the determine in the laboratory the efficacy of inhibition in the treatment of leukemia with ribosomal protein mutations.
The aims of this project are to better understand the role of ribosomal protein expression in adult and pediatric myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) as well as examine the role of novel inhibitors of WDR5 for treatment of myeloid neoplasms arising from disordered ribosomes. These studies may provide insight into the role of ribosomal protein expression in the pathogenesis of MDS and AML, and will contribute to larger efforts at Vanderbilt exploring WIN site inhibition as a novel treatment for myeloid neoplasms. This has the potential for the discovery of new treatments for leukemia.
The Turner's Heroes Kids Cancer Endowment Fund will continually and indefinitely contribute to the cancer care of patients at the American Family Children's Hospital in Madison Wisconsin. The Endowment Fund will also contribute to the pediatric cancer research at the UW Carbone Cancer Center. Thank you to our friends in Madison, Wisconsin and the University of Wisconsin - Madison for helping us create this impactful fund!