Science Corner: A Provider's Reflections on the 5th International Pediatric Liver Tumor Conference 2025
- Sarah Lansing
- Oct 13
- 6 min read
Jim Geller, MD is a renowned pediatric oncologist with more than 20 years of experience in his field. He has served as Director of the Liver and Renal Tumor Programs at Cincinnati Children's Hospital, has published over 170 peer-reviewed articles, and has chaired and sponsored numerous multidisciplinary committees and research trials for Children's Oncology Group as well as pharmaceutical companies and regulatory entities. Dr. Geller is currently affiliated with Rady Children's Hospital in San Diego, CA.
For three packed days in June of 2025, international experts from all perspectives related to pediatric and adolescent liver cancer convened in San Francisco, California, to share knowledge and outline opportunities for the future. Such international conferences typically occur every other to every third year, with the prior conferences being in 2023 (Boston, Massachusetts), 2020 (Cincinnati, Ohio), and 2018 (Houston, Texas). Like other such conferences, representation in San Francisco was notable from all medical disciplines including oncology, surgery, pathology, biology, radiology, epidemiology, radiation oncology, as well as parent/patient stakeholders. Coordinated representation from clinical and research investigators from the three large international cooperative groups- Children’s Oncology Group (COG), Liver Tumor Strategy Group of the International Society of Pediatric Liver Tumors (SIOPEL) and the Japanese Children’s Cancer Group/Liver Tumor Group (JCCG/JPLT) were integrated, which along with parent perspectives, shaped the conference.
[This event] speaks to the promise of an army of clinicians and scientists devoted to the cause.
Overall, there were over 70 oral presentations over the 3 days, incorporating an array of topics. The first two days including scientific presentations. Clinical trial updates were presented from the Pediatric Hepatic International Tumour Trial (PHITT; AHEP1531), a world-wide trial aiming to improve outcomes and reduce toxicities overall, from the perspective all three participating cooperative groups (COG, SIOPEL, JCCG). Greater than 1000 children with liver cancers (hepatoblastoma (including hepatocellular carcinoma not otherwise specified) and hepatocellular carcinoma (including fibrolamellar carcinoma) enrolled through the three cooperative groups. Data is starting to be released and analyzed, with conclusions likely to be presented to the public in the form of publications in the next several years.
New concepts were presented for children with high risk metastatic hepatoblastoma (SIOPEL/Explorer trial) which ultimately aims to include an HDAC (histone deacetylase) inhibitor (panobinostat) in addition to cisplatin/doxorubicin backbone therapy (not yet approved/open). A trial using an oral HDAC inhibitor (vorinostat, SAHA) in combination with cisplatin and sodium thiosulfate (for hearing protection) was also discussed, now accruing eligible children with refractory or relapsed hepatoblastoma.
An important session on hepatocellular carcinoma not otherwise specified (HCN NOS), which is not a specific diagnosis but rather a catch-all phrase used to describe tumors that harbor both hepatoblastoma and hepatocellular carcinoma features, led by our pathology discipline experts, offered the opportunity to educate as well as plan for future consolidation of knowledge to be gained from HCN-NOS data from the PHITT/AHEP1531 trial.
A session focusing on advancing novel biomarkers of liver cancer, mostly but not exclusively defined via molecular study of such tumors, were presented from all three cooperative group liver tumor biology subcommittees, with a number of key opportunities for enhanced risk stratification modeling discussed. Such biomarkers ultimately will enable a more personalized therapy, either via lightening or intensifying therapy, as indicated by risk profile, leading to improved cure with less long-term sequelae.
The collaborative international spirit emanating from leaders and members of the three large liver tumor cooperative groups was obvious.
Additional translational (pre-clinical model research) and biological presentations covered various topics including developmental biology signals in hepatoblastoma; use of organoid models to define hepatoblastoma subtype and potential drug targets; use of bioengineered tissues to model hepatoblastoma; single cell analytics of hepatoblastoma; study of the tumor microenvironment and its interplay in regulating the immune response to hepatoblastoma; mechanistic models for predicting drug efficacy against pediatric liver tumors; mechanisms of hepatoblastoma evolution to carcinoma- like biology and/or treatment resistance; epigenetic mechanisms of drug resistance in liver cancer cells; correlating liver cancer features to genetic syndromes; a review of pediatric hepatocellular adenomas (pre-malignant tumors); proteomics and pediatric liver cancer; and use of multityrosine kinase inhibitors for the treatment of pediatric liver tumors.
Broad topics of interest also included a review of the epidemiology of pediatric liver tumors; a status update on radiomics (the ability for radiology modeling to determine tumor features); advances in detecting minimal residual disease (evidence of tumor cells) from the blood in the form of circulating tumor DNA (ctDNA) which will ultimately help tailor duration and intensity of therapy needed for a given child as well as enhance diagnostics; aids to enhancing surgical feasibility such as 3D pre-surgery reconstruction using radiology tools enabling a better estimate of surgical complexity; the use of ICG (indocyanine-green) fluorescence-assistance during surgery for pediatric liver tumors; and presentations from medical interventionalists reviewing ‘interventional procedures’ such as trans-arterial chemoembolization (TACE) and trans-arterial radiotherapy (TARE).
A lengthy and detailed session addressing the ‘liver transplant’ vs non-transplant ‘extreme resection’ challenge was presented from many perspectives, as were presentations addressing peri-liver transplant issues such as immune suppression, living donor liver transplantation, salvage (rescue) liver transplantation for relapse, and multivisceral (liver plus other organ(s)) transplantation for hepatoblastoma. How to systematically get at refining our collective understanding, approach, and possible advancement of scientifically designed prospective future research addressing these opportunities were discussed, in part in considering a future ‘PHITT2’ trial.
Rare liver tumors, in addition to benign adenomas mentioned above, also received multidisciplinary attention, with a session including four presentations on fibrolamellar carcinoma (FLC), and a session including four presentations on undifferentiated embryonal sarcoma of the liver (UESL). Dr. Sandy Simon, both an investigator (founder of the FLC Registry) as well as parent stakeholder delivered important perspectives on the investigation of rare pediatric and adolescent liver cancer, followed by presentations by others of several trials including both vaccine-based approaches (targeting the FLC hallmark DNAJB1-PKAc fusion) as well as targeted therapy emerging from Dr. Simon’s work (targeting BCLxl in combination with chemotherapy). A trial targeting ‘sarcomas’ in general inclusive of UESL was also presented.
Day 3 was a highlight for many, wherein the day was split between scientific talks and patient/parent stakeholder events, with a collaborative tone set by Christina Stiverson, hepatoblastoma parent and founder of the Hepatoblastoma Resource Network, who opened the day by presenting the Hepatoblastoma Resource Network and its many efforts. At a scientific level, presentations included focus on late effects (survivorship) ranging from guideline proposals to psychosocial care to management of hearing loss. An ambitious effort to better explore key issues pertinent to ‘survivors’, building off prior stakeholder events at such international conferences, was presented in detail, as were challenges related to health equity disparities and social adversity. Guidelines for the management of hepatoblastoma and hepatocellular carcinoma, assembled by experts from COG, SIOPEL and JCCG, provided as ‘interim guidelines’ pending data output from the PHITT trial, were recently published and presented by its authors (read here: https://www.sciencedirect.com/science/article/pii/S2772610X25000236)
Updates from the CHIC (childhood hepatic international collaboration) were provided wherein the prognostic value of certain clinical features are assessed from historic trials, as was an update on ARIA, an international resource-focused guideline for the global care of hepatoblastoma.
Presentations of various US-based registries included relapsed hepatoblastoma (rrHBL Registry; https://www.rrhblregistry.org ) and hepatocellular carcinoma (Count Me In; https://pedihccproject.org/ ), each aiming to advance collective knowledge enabling improvement in standards and outcomes for affected children and families. In addition, data from the PSORC (pediatric surgical oncology research collaborative) were presented as well as updates from the German Pediatric Liver Tumor registry and Japanese Liver Tumor registry. Finally, at the scientific level, exciting new trials were presented highlighting the potential for immunotherapy to have a role in pediatric liver tumor treatment, including a trial using Engineered TCR (T-cell receptor) for Relapsed/Refractory HB, HCN NOS, and HCC and a trial using CAR-T cells targeting GPC3/IL-15.
On a personal level, the highlight for me, was the patient panel discussion occuring on Day 3, introduced and led by Julie Chessell, including invaluable perspectives and dialogue. Conference attendees were blessed with a speech by Brock Chessell, hepatoblastoma survivor, who enlightened the room with his candor and critical perspective. He then joined on stage nearly a dozen other children and adolescents who have braved the battle against liver cancer themselves, all providing a sobering and touching opportunity for attendees to hear their perspectives on all issues related to their cancer journey.
In sum, the three-day meeting left me feeling hopeful. The very packed nature of the conference, including such outstanding investigators sharing their work and ideas, with open dialogue, including many young investigators early in their career, speaks to the promise of an army of clinicians and scientists devoted to the cause. The collaborative international spirit emanating from leaders and members of the three large liver tumor cooperative groups was obvious. Interest from those not traditionally included in such cooperative group committees was also present, broadening our attention to less thoroughly explored opportunities not limited to survivorship.
Perhaps most importantly, the robust and moving participation from stakeholders of all kinds at the conference adds to a growing reality that old borders that may have existed between ‘care providers’ and ‘patients/families’ is dissipating, with improved perspective and dialogue that will surely enable more positive, meaningful progress in the days and years to come.
Written by Jim Geller, MD
Rady Children's Hospital