A recently published scientific article shows how just a few years of personal involvement & grass roots fundraising by family-led organizations moved our medical community closer to a treatment option for rare pediatric cancer. Hepatoblastoma (liver cancer) is a disease with very few cases each year. Thus, patients suffer the same fate as many other rare diseases: under-funded research leading to limited treatment options. Patients with recurring or spreading disease have even fewer options and endure a horrifically low rate of survival. A few key parents and family-based foundations took this to heart and engaged with a revolutionary approach to finding a solution with the The Children’s Cancer Therapy Development Institute (cc-TDI) in Beaverton, Oregon. Scientists at the lab (championed by Dina Katz with mentorship from Dr Charles Keller) built on collaborative efforts with a lab in Europe on a path of discovery. Dr James Geller, from the Cincinnati Children’s Hospital and a liver committee chair for the Children’s Oncology Group has also followed and assisted this process of treatment development.
The words "there's nothing more we can do" are a cancer parent's worst nightmare. Knowing research is being done that would provide more options for children with aggressive hepatoblastoma gives us hope that fewer parents will have to hear them. ~Katie & Kevin Gott, Henry’s Parents
In general, medical treatment discovery starts with basic science to understand the normal function of cells, and then pursues deeper insight into how the disease behaves—what makes cancer stand out and how the cancer is vulnerable to intervention. What kills cancer while not harming healthy parts of the body? This takes mountains of foundational knowledge and samples (cell lines) of the disease in order to find effective treatments. That process involves petri dish testing (called in vitro) to find something that slows or stops the cancer’s growth. In this case, the scientists focused on a specific abnormality: the process of hepatoblastoma cell division showed an abundance of a protein called polo-like kinase 1 (PLK1). This protein expression occurs in normal liver cell division as well, but at a much lower rate. Building on that knowledge, the team focused on a targeted drug which could safely be delivered and bind to PLK1, hence causing the cancer cell to die while not adversely impacting normal liver cells. The team did enough research in petri dishes to move into the in vivo (living-model tests) phase; and the scientists found that the PLK1 inhibitor drug worked best in combination with an existing chemotherapy for Hepatoblastoma.
cc-TDI allowed parent-led organizations to help guide the research, jointly prioritizing experimentation, and ultimately focus the grass roots funding—capital which is required for research to happen.
Supporting research where you know people genuinely want to find that new drug means so much to parents like us. Our child wasn’t to be saved, but hopefully one day, many more children in the same situation as Dawson can be. ~Wendy Willcock, Dawson’s Mom
It’s also critical to highlight that none of this could happen without the generous gifts of patient tissue—a purposeful tribute by children and their families, which provides the foundation for future treatment development. More work is required to move the new drug into clinical trials for children with relapse hepatoblastoma. However, this great success which was made possible by doctors and scientists around the globe and urgently pressed forward by families and their dedicated supporters, stands as a beacon for the power of community. Big thanks go out to the hundreds of donors who generosity enabled the logistics, imagination, ingenuity, and eventual success of this amazing step forward.
We watched Sam fight that nasty disease, and as devastated as we are to have lost him, we love that his memory can help fund such an important and amazing group of people that also want to end childhood cancer! The fact that parents can be involved and that our kiddos pictures are on the wall at cc-TDI means so much. ~Lee Ann Biggs, Sam’s Mom & Founder of The Sam Biggs Memorial Foundation
Our sincere appreciation goes out to: Children’s Cancer Therapy Development Institute (cc-TDI), Foundation for Addie’s Research, Willcock Family, Gott Family, Sam Biggs Memorial Foundation, Arctic Explorers Preschool, Owls For Avery Foundation, Macy Easom Cancer Research Foundation, Mid City Grill, and Gracie’s Candles.
For the full article published in Oncotarget, please visit: