RARE DISEASE
Rare hepatic and biliary tract tumor
Rare hepatic and biliary tract tumor
Rare hepatic and biliary tract tumor
Drug discovery
2
drugs
With orphan designations
Overview
Rare hepatic and biliary tract tumors, including cholangiocarcinoma (CCA) and gallbladder carcinoma, are aggressive malignancies with variable anatomical subtypes (intrahepatic, perihilar, extrahepatic). Risk factors include primary sclerosing cholangitis, liver fluke infections, and congenital bile duct anomalies [1][5][8]. Diagnosis is often delayed due to nonspecific symptoms, leading to advanced-stage presentation and poor prognosis [5][17].
Burden
5-year survival for advanced CCA: <10%; gallbladder cancer: median survival 3 months [6][11][12].
High morbidity from biliary obstruction (jaundice, cholangitis) and liver failure [5][8][14].
Limited clinical trial data due to rarity, necessitating multidisciplinary care at specialized centers [3][14][16].
Therapies
Curative resection for localized disease; liver transplantation for select perihilar CCA [3][8][13].
First-line chemotherapy: gemcitabine + cisplatin (median survival 11.7 months) [3][16].
Emerging targeted therapies (e.g., FGFR2 inhibitors, IDH1 inhibitors) and adjuvant capecitabine post-resection [3][16][19].
Categories: rare hepatic diseases, rare neoplastic diseases
Research Papers
126 drug discovery papers about Rare hepatic and biliary tract tumor, with 1 first-in-class and 2 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
126 drug discovery papers about Rare hepatic and biliary tract tumor, with 1 first-in-class and 2 next-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2026-05-27 | Dual BRAF/MEK inhibition in BRAF V600E –mutated biliary tract cancer with exploratory histology-dependent response: A systematic review and meta-analysis.
4108 Background: The BRAF V600E mutation defines an actionable subset of rare cancers. Metastatic BTC carries a poor prognosis, with median survival under one year on standard chemotherapy. Given the rarity of BRAF V600E-mutated BTC and the absence of RCTs, rigorous evidence synthesis is needed; existing data for dabrafenib plus trametinib come only from single arm basket trials such as ROAR and NCI-MATCH. The low prevalence of this mutation limits the feasibility of large randomized studies. This SRMA aimed to quantify the efficacy of dual BRAF/MEK inhibition in BTC and explore potential histology dependent differences in response. Methods: We performed a systematic review and meta-analysis (SRMA) pooling single arm Phase II basket trial cohorts (N = 53 BTC; N = 17 CNS). Seven case reports were summarized qualitatively and excluded from pooled analyses. The primary endpoint was pooled overall response rate (ORR). A random effects model was used. Subgroup and heterogeneity analyses were performed given the tissue-specific behavior of V600E-driven tumors. Histology was evaluated as an exploratory moderator and heterogeneity was quantified. Results: In the pooled cohort of 70 patients (53 BTC and 17 CNS), the overall ORR was 45.3% (95% CI: 24.5-66.9%). Heterogeneity for the BTC subgroup was moderate (I 2 = 62.6%), while overall pooled heterogeneity across all studies was low (I 2 = 14%). ORR in BTC was numerically higher than in Central nervous system (CNS) cohorts (34.9%) but subgroup differences did not reach statistical significance (P = 0.3386). The observed median OS of 13.5 months in the ROAR BTC cohort compares favourably with historical outcomes commonly reported in the 6-12 month range for advanced BTC. The consistency of pooled estimates, despite non-randomized designs, suggests a stable treatment signal while acknowledging that causality cannot be inferred from uncontrolled data. Median progression-free survival was 9.0 months in BTC and 5.5-8.0 months in CNS cohorts. Median OS in the ROAR BTC cohort was 13.5 months (95% CI: 10.4-17.6), exceeding historical expectations. The pooled rate of Grade ≥3 AEs was 59.2%, consistent with the known safety profile of dabrafenib plus trametinib; no treatment-related deaths were reported. Qualitative data from case reports confirmed regression of CNS metastases and feasibility in patients with severe hepatic dysfunction. Conclusions: Dual BRAF/MEK inhibition shows clinically meaningful activity in BRAF V600E-mutated BTC, with survival exceeding historical expectations. Exploratory analyses indicate histology-dependent differences, supporting tumor-specific therapeutic stratification. These findings align with growing tissue-agnostic evidence for MAPK inhibition and reinforce the value of routine molecular profiling, though conclusions are limited by small cohorts and single arm studies.
2026-04-20 | IgG4-sclerosing cholangitis masquerading as cholangiocarcinoma: a case report of an unresolved preoperative diagnosis.
Immunoglobulin G4-related disease (IgG4-RD) is an immune-mediated chronic fibroinflammatory condition that can affect multiple organ systems. IgG4-related sclerosing cholangitis (IgG4-SC) is its manifestation involving the biliary tract. Due to atypical clinical presentations in some cases and insufficient awareness among clinicians, IgG4-SC is frequently misdiagnosed as cholangiocarcinoma, and multiple instances of inappropriate treatment as a result have been documented. Here we report a case of IgG4-SC that presented diagnostic challenges preoperatively and was followed by rare pancytopenia after surgery. The patient was a 50-year-old man who sought medical attention due to elevated transaminases for eight months, without obvious clinical symptoms. Among serum tumor markers, the level of protein induced by vitamin K absence/antagonist-II (PIVKA-II) was elevated. Examinations including magnetic resonance cholangiopancreatography (MRCP), contrast-enhanced abdominal computed tomography (CT), and positron emission tomography-computed tomography (PET-CT) all suggested malignant stricture at the hepatic hilum. A needle biopsy indicated dysplastic changes but was inconclusive for cholangiocarcinoma. Although IgG4-SC was considered, multiple serum IgG4 measurements remained within the normal range. With an initial clinical diagnosis of cholangiocarcinoma, the patient underwent surgical resection. Intraoperative frozen section analysis indicated an inflammatory process. Postoperatively, the patient developed pancytopenia that responded poorly to conventional supportive treatment. The condition was ultimately diagnosed as IgG4-SC based on postoperative histopathology. Corticosteroid therapy led to the normalization of the patient's blood counts, transaminases, and bilirubin levels. IgG4-SC can present with a spectrum of atypical features, such as isolated biliary strictures, absence of characteristic symptoms, normal IgG4 levels, and imaging findings resembling cholangiocarcinoma. Therefore, in cases clinically suspicious for cholangiocarcinoma, differentiating IgG4-SC warrants serious consideration. Every effort should be made to complete preoperative biopsy and histopathological assessment. For cases where differentiation remains difficult despite comprehensive evaluation, surgical intervention with intraoperative frozen section biopsy is necessary to establish a definitive diagnosis and avoid delayed treatment. Furthermore, this case suggests that IgG4-SC may also involve the hematopoietic system, manifesting as pancytopenia, which can respond effectively to corticosteroid therapy.
2026-04-13 | Neoadjuvant Treatment Strategies in Unresectable Gallbladder Cancer (GBC) in a Regional Cancer Centre in India: a Prospective Cohort Study.
BACKGROUND: Since GBC usually presents as unresectable disease, we conducted a prospective observational study to evaluate the effect of neoadjuvant strategies (NAT) on radiologic downstaging and resectability. MATERIALS AND METHODS: Patients with locally advanced GBC were treated with neoadjuvant chemotherapy (NACT). Those who were unresectable after 4–6 cycles NACT were offered consolidation chemoradiotherapy [CTRT] (45 Gy along with weekly concurrent cisplatin, 5FU [prior to 2014] and thereafter concurrent capecitabine@1250mg/2). Radiological assessment of response to chemotherapy was done (by CT angiography and PET-CT) to evaluate for resectability. Features affecting resectability were evaluated. Those found suitable for resection underwent radical surgery. RESULTS: 217 patients were evaluated (January 2012 to December 2022) (NACT:60%, NACT followed by CTRT:40%,). Pretreatment CT scans revealed involvement of liver>2 cm (75%), duodenum (42%), colon (29%), CBD (36%), CHD/primary confluence (45.6%), Hepatic Artery (32%), portal vein (21%), N0 (24.5%) N1 (17%), N2 (17%), retroperitoneal LN ( 40%). After NACT considerable radiological downstaging was evident in liver, duodenum and colon involvement (52%, 40% and 37%), while downstaging in CBD, CHD/confluence was relatively rare (14%, 7%). The proportion of nodal downstaging was evident from increase in proportion of N0 and N1 (37.5%, 20%), and decrease in proportion of N2 and RPLN (8%, 20%). Only 22 patients (10%) underwent surgical resection (EC n = 21, SC n = 1, after NACT:12, after CTRT:10). All except two were R0 (91%) and 48% had ypN0 disease. The median OS of those who underwent resection is 49 months (95% CI 26.8–69 mo.) versus 9 months (95% CI 8–10 mo.) in those who did not. CONCLUSIONS: NAT in unresectable GBC results in 10% resectability rate. Patients with liver involvement, duodenal involvement and lymphadenopathy had greater possibility of resectability after NAT, whereas those with biliary tree or vessel involvement had least possibility of resection. This approach has a potential of achieving R0, node negative disease leading to improved survival rates and should be actively explored in patients without biliary and vessel involvement.
2026-04-03 | Abstract 2667: TM4SF1 as a novel tumor-associated antigen in biliary tract cancers targetable by immune effector cell therapy
Abstract Biliary tract cancers (BTC) are a rare set of genetically heterogeneous and aggressive malignancies associated with late presentation, poor prognosis and limited effective therapies. Thus, there is an unmet clinical need for the development of novel therapeutic strategies. Identification of uniquely expressed cell-surface tumor-associated antigens (TAA) holds promise in epithelial tumors more generally, as they can serve as ligands for a variety of therapeutics, including CAR-T cell therapy. However, TAA identification in BTCs has been limited by expression in normal liver tissue. Here, we identify the cell-surface protein transmembrane 4 L six family member 1 (TM4SF1) as a potential BTC TAA targetable by CAR-T therapy. We find TM4SF1 expression is upregulated in BTC relative to normal hepatic/biliary tissue at the mRNA level in the TCGA dataset. Correspondingly, we find that TM4SF1 in primary BTC archival tissue is upregulated at the protein level using immunohistochemistry. To validate TM4SF1 as a targetable TAA in BTC, we show that TM4SF1-directed CAR-T cells demonstrate robust, dose-dependent growth inhibition of human-derived BTC cell lines in vitro and significant anti-tumor activity in a heterotopic BTC cell line-derived xenograft model in vivo. Together, our data provide support for TM4SF1 as a promising TAA in BTCs that can be used for the rational development of therapeutic modalities targeting TM4SF1, including CAR-T cells, in a patient population with significant unmet need. Citation Format: Lorraine Nuniz, Franklin Huang. TM4SF1 as a novel tumor-associated antigen in biliary tract cancers targetable by immune effector cell therapy [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2026; Part 1 (Regular Abstracts); 2026 Apr 17-22; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2026;86(7 Suppl):Abstract nr 2667.
2026-02-19 | Perihilar Biliary Neuroendocrine Carcinoma Treated With Perioperative Chemotherapy and Radical Liver Surgery: A Thought-Provoking Case.
BACKGROUND Biliary neuroendocrine neoplasm (NEN) arising from the extrahepatic bile duct is rare, with an incidence of 0.2% among gastroentero-hepatopancreatobiliary NEN cases. Biliary neuroendocrine carcinoma (NEC) is an extremely rare high-grade malignancy that requires multidisciplinary treatment, including surgery, chemotherapy, and radiation. However, there have been only a few reports on the combined therapy for biliary NEC. CASE REPORT A 74-year-old man was referred to our hospital with a chief concern of obstructive jaundice. He was diagnosed with perihilar biliary NEC. The tumor primarily involved the confluence of the hepatic and left hepatic ducts. After 4 courses of systemic chemotherapy with cisplatin and etoposide (EP regimen), the tumor showed partial shrinkage. The patient underwent left and caudate hepatectomy with combined resection and reconstruction of the extrahepatic bile duct. Intraoperatively, strong adhesion between the right hepatic artery and the dorsal surface of the tumor was observed, requiring combined resection and reconstruction of the right hepatic artery. Postoperatively, the patient was treated with adjuvant chemotherapy (EP regimen) for 6 months. Ten months after surgery, he was diagnosed with multiple aggressive recurrences that were refractory to multimodal therapies. Eighteen months after the initial diagnosis and 12 months after surgery, he died of liver failure due to cholangitis. CONCLUSIONS This report presents the first case of perihilar biliary NEC that underwent perioperative chemotherapy and radical liver surgery. We believe that the introduction of perioperative chemotherapy is essential to achieve a better prognosis for perihilar biliary NEC, given its oncological malignancy and surgical invasiveness.
2026-05-27 | Dual BRAF/MEK inhibition in BRAF V600E –mutated biliary tract cancer with exploratory histology-dependent response: A systematic review and meta-analysis.
4108 Background: The BRAF V600E mutation defines an actionable subset of rare cancers. Metastatic BTC carries a poor prognosis, with median survival under one year on standard chemotherapy. Given the rarity of BRAF V600E-mutated BTC and the absence of RCTs, rigorous evidence synthesis is needed; existing data for dabrafenib plus trametinib come only from single arm basket trials such as ROAR and NCI-MATCH. The low prevalence of this mutation limits the feasibility of large randomized studies. This SRMA aimed to quantify the efficacy of dual BRAF/MEK inhibition in BTC and explore potential histology dependent differences in response. Methods: We performed a systematic review and meta-analysis (SRMA) pooling single arm Phase II basket trial cohorts (N = 53 BTC; N = 17 CNS). Seven case reports were summarized qualitatively and excluded from pooled analyses. The primary endpoint was pooled overall response rate (ORR). A random effects model was used. Subgroup and heterogeneity analyses were performed given the tissue-specific behavior of V600E-driven tumors. Histology was evaluated as an exploratory moderator and heterogeneity was quantified. Results: In the pooled cohort of 70 patients (53 BTC and 17 CNS), the overall ORR was 45.3% (95% CI: 24.5-66.9%). Heterogeneity for the BTC subgroup was moderate (I 2 = 62.6%), while overall pooled heterogeneity across all studies was low (I 2 = 14%). ORR in BTC was numerically higher than in Central nervous system (CNS) cohorts (34.9%) but subgroup differences did not reach statistical significance (P = 0.3386). The observed median OS of 13.5 months in the ROAR BTC cohort compares favourably with historical outcomes commonly reported in the 6-12 month range for advanced BTC. The consistency of pooled estimates, despite non-randomized designs, suggests a stable treatment signal while acknowledging that causality cannot be inferred from uncontrolled data. Median progression-free survival was 9.0 months in BTC and 5.5-8.0 months in CNS cohorts. Median OS in the ROAR BTC cohort was 13.5 months (95% CI: 10.4-17.6), exceeding historical expectations. The pooled rate of Grade ≥3 AEs was 59.2%, consistent with the known safety profile of dabrafenib plus trametinib; no treatment-related deaths were reported. Qualitative data from case reports confirmed regression of CNS metastases and feasibility in patients with severe hepatic dysfunction. Conclusions: Dual BRAF/MEK inhibition shows clinically meaningful activity in BRAF V600E-mutated BTC, with survival exceeding historical expectations. Exploratory analyses indicate histology-dependent differences, supporting tumor-specific therapeutic stratification. These findings align with growing tissue-agnostic evidence for MAPK inhibition and reinforce the value of routine molecular profiling, though conclusions are limited by small cohorts and single arm studies.
2026-04-20 | IgG4-sclerosing cholangitis masquerading as cholangiocarcinoma: a case report of an unresolved preoperative diagnosis.
Immunoglobulin G4-related disease (IgG4-RD) is an immune-mediated chronic fibroinflammatory condition that can affect multiple organ systems. IgG4-related sclerosing cholangitis (IgG4-SC) is its manifestation involving the biliary tract. Due to atypical clinical presentations in some cases and insufficient awareness among clinicians, IgG4-SC is frequently misdiagnosed as cholangiocarcinoma, and multiple instances of inappropriate treatment as a result have been documented. Here we report a case of IgG4-SC that presented diagnostic challenges preoperatively and was followed by rare pancytopenia after surgery. The patient was a 50-year-old man who sought medical attention due to elevated transaminases for eight months, without obvious clinical symptoms. Among serum tumor markers, the level of protein induced by vitamin K absence/antagonist-II (PIVKA-II) was elevated. Examinations including magnetic resonance cholangiopancreatography (MRCP), contrast-enhanced abdominal computed tomography (CT), and positron emission tomography-computed tomography (PET-CT) all suggested malignant stricture at the hepatic hilum. A needle biopsy indicated dysplastic changes but was inconclusive for cholangiocarcinoma. Although IgG4-SC was considered, multiple serum IgG4 measurements remained within the normal range. With an initial clinical diagnosis of cholangiocarcinoma, the patient underwent surgical resection. Intraoperative frozen section analysis indicated an inflammatory process. Postoperatively, the patient developed pancytopenia that responded poorly to conventional supportive treatment. The condition was ultimately diagnosed as IgG4-SC based on postoperative histopathology. Corticosteroid therapy led to the normalization of the patient's blood counts, transaminases, and bilirubin levels. IgG4-SC can present with a spectrum of atypical features, such as isolated biliary strictures, absence of characteristic symptoms, normal IgG4 levels, and imaging findings resembling cholangiocarcinoma. Therefore, in cases clinically suspicious for cholangiocarcinoma, differentiating IgG4-SC warrants serious consideration. Every effort should be made to complete preoperative biopsy and histopathological assessment. For cases where differentiation remains difficult despite comprehensive evaluation, surgical intervention with intraoperative frozen section biopsy is necessary to establish a definitive diagnosis and avoid delayed treatment. Furthermore, this case suggests that IgG4-SC may also involve the hematopoietic system, manifesting as pancytopenia, which can respond effectively to corticosteroid therapy.
2026-04-13 | Neoadjuvant Treatment Strategies in Unresectable Gallbladder Cancer (GBC) in a Regional Cancer Centre in India: a Prospective Cohort Study.
BACKGROUND: Since GBC usually presents as unresectable disease, we conducted a prospective observational study to evaluate the effect of neoadjuvant strategies (NAT) on radiologic downstaging and resectability. MATERIALS AND METHODS: Patients with locally advanced GBC were treated with neoadjuvant chemotherapy (NACT). Those who were unresectable after 4–6 cycles NACT were offered consolidation chemoradiotherapy [CTRT] (45 Gy along with weekly concurrent cisplatin, 5FU [prior to 2014] and thereafter concurrent capecitabine@1250mg/2). Radiological assessment of response to chemotherapy was done (by CT angiography and PET-CT) to evaluate for resectability. Features affecting resectability were evaluated. Those found suitable for resection underwent radical surgery. RESULTS: 217 patients were evaluated (January 2012 to December 2022) (NACT:60%, NACT followed by CTRT:40%,). Pretreatment CT scans revealed involvement of liver>2 cm (75%), duodenum (42%), colon (29%), CBD (36%), CHD/primary confluence (45.6%), Hepatic Artery (32%), portal vein (21%), N0 (24.5%) N1 (17%), N2 (17%), retroperitoneal LN ( 40%). After NACT considerable radiological downstaging was evident in liver, duodenum and colon involvement (52%, 40% and 37%), while downstaging in CBD, CHD/confluence was relatively rare (14%, 7%). The proportion of nodal downstaging was evident from increase in proportion of N0 and N1 (37.5%, 20%), and decrease in proportion of N2 and RPLN (8%, 20%). Only 22 patients (10%) underwent surgical resection (EC n = 21, SC n = 1, after NACT:12, after CTRT:10). All except two were R0 (91%) and 48% had ypN0 disease. The median OS of those who underwent resection is 49 months (95% CI 26.8–69 mo.) versus 9 months (95% CI 8–10 mo.) in those who did not. CONCLUSIONS: NAT in unresectable GBC results in 10% resectability rate. Patients with liver involvement, duodenal involvement and lymphadenopathy had greater possibility of resectability after NAT, whereas those with biliary tree or vessel involvement had least possibility of resection. This approach has a potential of achieving R0, node negative disease leading to improved survival rates and should be actively explored in patients without biliary and vessel involvement.
2026-04-03 | Abstract 2667: TM4SF1 as a novel tumor-associated antigen in biliary tract cancers targetable by immune effector cell therapy
Abstract Biliary tract cancers (BTC) are a rare set of genetically heterogeneous and aggressive malignancies associated with late presentation, poor prognosis and limited effective therapies. Thus, there is an unmet clinical need for the development of novel therapeutic strategies. Identification of uniquely expressed cell-surface tumor-associated antigens (TAA) holds promise in epithelial tumors more generally, as they can serve as ligands for a variety of therapeutics, including CAR-T cell therapy. However, TAA identification in BTCs has been limited by expression in normal liver tissue. Here, we identify the cell-surface protein transmembrane 4 L six family member 1 (TM4SF1) as a potential BTC TAA targetable by CAR-T therapy. We find TM4SF1 expression is upregulated in BTC relative to normal hepatic/biliary tissue at the mRNA level in the TCGA dataset. Correspondingly, we find that TM4SF1 in primary BTC archival tissue is upregulated at the protein level using immunohistochemistry. To validate TM4SF1 as a targetable TAA in BTC, we show that TM4SF1-directed CAR-T cells demonstrate robust, dose-dependent growth inhibition of human-derived BTC cell lines in vitro and significant anti-tumor activity in a heterotopic BTC cell line-derived xenograft model in vivo. Together, our data provide support for TM4SF1 as a promising TAA in BTCs that can be used for the rational development of therapeutic modalities targeting TM4SF1, including CAR-T cells, in a patient population with significant unmet need. Citation Format: Lorraine Nuniz, Franklin Huang. TM4SF1 as a novel tumor-associated antigen in biliary tract cancers targetable by immune effector cell therapy [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2026; Part 1 (Regular Abstracts); 2026 Apr 17-22; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2026;86(7 Suppl):Abstract nr 2667.
2026-02-19 | Perihilar Biliary Neuroendocrine Carcinoma Treated With Perioperative Chemotherapy and Radical Liver Surgery: A Thought-Provoking Case.
BACKGROUND Biliary neuroendocrine neoplasm (NEN) arising from the extrahepatic bile duct is rare, with an incidence of 0.2% among gastroentero-hepatopancreatobiliary NEN cases. Biliary neuroendocrine carcinoma (NEC) is an extremely rare high-grade malignancy that requires multidisciplinary treatment, including surgery, chemotherapy, and radiation. However, there have been only a few reports on the combined therapy for biliary NEC. CASE REPORT A 74-year-old man was referred to our hospital with a chief concern of obstructive jaundice. He was diagnosed with perihilar biliary NEC. The tumor primarily involved the confluence of the hepatic and left hepatic ducts. After 4 courses of systemic chemotherapy with cisplatin and etoposide (EP regimen), the tumor showed partial shrinkage. The patient underwent left and caudate hepatectomy with combined resection and reconstruction of the extrahepatic bile duct. Intraoperatively, strong adhesion between the right hepatic artery and the dorsal surface of the tumor was observed, requiring combined resection and reconstruction of the right hepatic artery. Postoperatively, the patient was treated with adjuvant chemotherapy (EP regimen) for 6 months. Ten months after surgery, he was diagnosed with multiple aggressive recurrences that were refractory to multimodal therapies. Eighteen months after the initial diagnosis and 12 months after surgery, he died of liver failure due to cholangitis. CONCLUSIONS This report presents the first case of perihilar biliary NEC that underwent perioperative chemotherapy and radical liver surgery. We believe that the introduction of perioperative chemotherapy is essential to achieve a better prognosis for perihilar biliary NEC, given its oncological malignancy and surgical invasiveness.
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Drug Discovery Landscape
2 orphan drug designations for Rare hepatic and biliary tract tumor.
2 orphan drug designations for Rare hepatic and biliary tract tumor.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
humanized IgG1 monoclonal antibody against annexin-A1 | antibodies | FDA | 2025-09-26 | — | Medannex Ltd |
transforming growth factor beta trap/anti-programmed death ligand-1 antibody bifunctional fusion protein | antibodies | FDA | 2018-12-07 | — | EMD Serono Research & Development Institute, Inc. |
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