Updated: May 24
Written by: Christina Stiverson (Addie's Mom)
We are grateful to be partners with The Children’s Cancer Therapy Development Institute (cc-TDI), a non-profit biotech lab researching answers to the most challenging problems. One thing we have learned while studying a rare disease, which only affects a small population, is that it can be very costly and difficult due to the lack of funding. However, cc-TDI challenges those barriers with affordable and revolutionary research.
For us, it’s not only about helping kids with Hepatoblastoma but sharing our research and contributing to translational solutions that search for mutations, targets, and cures across all rare pediatric cancers.
Over the past few years with our grassroots fund raising, Addie’s Research has been able to contribute funding for several ‘proof of concept’ projects, which has now enabled the lab to submit two National Institute of Health (NIH) research grant proposals specific to Hepatoblastoma totaling over 4 million dollars! We hope to collaborate on a study about the ‘liver cell of origin’ to explore how Hepatoblastoma develops, so as a community we can better understand how to target the disease. Also, if awarded, there is the possibility to continue work on developing the Quail Assay. CC-TDI has been using this ground-breaking pre-clinical testing model to test high-risk Hepatoblastoma cell-lines against possible drug targets in living organisms. The amazing thing about this engineering discovery is a quail egg study costs $700, whereas a mouse model showing similar results costs over $25K! Through our Hepatoblastoma research we have found two additional drugs that specifically target high-risk Hepatoblastoma and we have successful data testing in a petri-dish on high-risk HB cell lines, so we plan to move to quail egg testing next month!
In theory, these drugs will target and attack the cancer only, with less toxicity going to healthy cells, but we must prove it during the next steps of research. We are pursuing relationships with the pharmaceutical companies who own the drugs, and Cody (Addie's Dad) is on the board for a newly formed biopharma company that can bring the drugs into clinical trials if the research supports our theory.
After publishing our first scientific paper last year, we continue our research with Volasertib and Irinotecan as a possible life extension combination for Hepatoblastoma.
Even though this drug may not kill the cancer, it can delay the cancer cells from growing/spreading, which many of us know can lead to a surgical option for kids with Hepatoblastoma. We continue to research more into genetic mutations and why it showed better efficacy on some high-risk models than others. The high-risk make-up of HB tumors is varied and very complex, which is why these children don’t respond to the prescribed treatment. Also, upon relapse or metastasis, we know cancer mutates and becomes more difficult to treat. However, we are trying to outsmart the cancer using science!
In addition, last year we started The Hepatoblastoma Resource Network (Hepatoblastoma.org) and spearheaded the parent stakeholder portion of the first global Hepatoblastoma Community Conference in Feb 2020.
We are currently planning the next conference for May 2021, led by Dr. James Gellar at Cincinnati Children’s Hospital, to bring parents, researchers, and clinicians together to focus on long-term survivorship challenges, relapse/refractory disease research, and creating a formal parent support network.
We continue to initiate and collaborate on projects that we find meaningful to advance the Hepatoblastoma community. Thank you all for your steadfast support over the past 4 years to help us do so.
Collage Photo Credit: Sharleigh Stevenson