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Each year we have a one week “bootcamp” called a Nanocourse to empower families. At the 2015 nanocourse, Macy Easom’s aunt, Lisa, teamed with other families to write a hepatoblastoma research roadmap, which they published in the peer-reviewed scientific literature. The hepatoblastoma roadmap is available to read here. In this roadmap, the families conveyed that addressing the lack of cell lines and mouse models and need for faster preclinical testing were the key next steps. Lisa’s community via the Macy Easom Foundation funds an engineer in our lab, Samuel Rasmussen, to develop a fast and cost-effective way to screen drugs.
With the engineer in place, Addie’s parents Christina & Cody committed to funding the development of new cell lines and mouse models. To that end we have received 23 mouse models and/or cell lines from a collaborator in France. The next big thing to do was to hire a full-time biologist, Dina Kats, to work full time in parallel to our engineer, Samuel.
The key goals over the first year have been:
- To validate each of the new mouse models and cell lines as being from the patient they are believed to be from, and that each mouse model or cell line is hepatoblastoma.
- To sequence the DNA (exome) and RNA from each mouse model or cell.
- To make cell lines from mouse model tumors.
- To screen each cell line against 60 or more drugs that are being used in clinical trials for other cancers in adults or children.
From these studies, we have identified the PLK1 inhibitor volasertib as a potentially active agent in relapsed and refractory hepatoblastoma that spares normal liver cells. In the coming year, for which we are seeking funding, we will further validate volasertib-plus-irinotecan as a potential therapy for hepatoblastoma, continue DNA & RNA sequencing, and explore the epigenetic susceptibility of hepatoblastoma based upon the cell-of-origin.
Make this second year’s hepatoblastoma research program funding possible through crowdfunding participation here.