Two Cancer Drug Advances Show Promise in Early Research
Early trial of Rezatapopt shows 20% response rate in advanced cancers with p53 mutations, while researchers identify AMD3100 as potential solution to enable immunotherapy in rare liver cancer.
Fibrolamellar carcinoma is a rare primary liver cancer that typically occurs in adolescents and young adults, often without underlying chronic liver disease. It is biologically distinct from conventional hepatocellular carcinoma and has unique clinical and molecular features. Many cases present late because early symptoms can be minimal.
Early trial of Rezatapopt shows 20% response rate in advanced cancers with p53 mutations, while researchers identify AMD3100 as potential solution to enable immunotherapy in rare liver cancer.
Study finds FDA-approved drug AMD3100 can mobilize immune T cells to attack fibrolamellar carcinoma, a rare liver cancer affecting children and young adults that has resisted immunotherapy treatment.
Researchers find that AMD3100, an existing FDA-approved drug, can reverse T-cell exclusion in fibrolamellar carcinoma, potentially allowing immunotherapy to fight the rare and fatal liver cancer that primarily affects children and young adults.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT06638931 |
Agnostic Therapy in Rare Solid Tumors |
RECRUITING | PHASE2 |
| NCT06541652 |
A French Multicenter Observational Retrospective Study of Rare Primary Liver Cancers |
RECRUITING | |
| NCT06521567 |
A Study of Cobolimab Plus Dostarlimab in Pediatric and Young Adult Participants With Cancer |
ACTIVE_NOT_RECRUITING | PHASE1/PHASE2 |
| NCT05603572 |
KAT-101 in Subjects With Hepatocellular Carcinoma (HCC) |
SUSPENDED | PHASE1/PHASE2 |
| NCT05468359 |
Safety and Efficacy of Cyclophosphamide, Sorafenib, Bevacizumab, and Atezolizumab in Pediatric Solid Tumor Patients |
RECRUITING | PHASE1/PHASE2 |
| NCT04134559 |
Checkpoint Inhibition In Pediatric Hepatocellular Carcinoma |
RECRUITING | PHASE2 |
| NCT03860272 |
Fc-Engineered Anti-CTLA-4 Monoclonal Antibody in Advanced Cancer |
ACTIVE_NOT_RECRUITING | PHASE1 |
| NCT03533582 |
Cisplatin and Combination Chemotherapy in Treating Children and Young Adults With Hepatoblastoma or Liver Cancer After Surgery |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT02159989 |
Sapanisertib and Ziv-Aflibercept in Treating Patients With Recurrent Solid Tumors That Are Metastatic or Cannot Be Removed by Surgery |
COMPLETED | PHASE1 |
| NCT01642186 |
Study of (1) Everolimus, (2) Estrogen Deprivation Therapy (EDT) With Leuprolide + Letrozole and (3) Everolimus + EDT in Patients With Unresectable Fibrolamellar Hepatocellular Carcinoma (FLL-HCC) |
COMPLETED | PHASE2 |