Molecular Profile Detail

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Profile Name RB1 loss
Gene Variant Detail

RB1 loss (loss of function)

Relevant Treatment Approaches HDAC Inhibitor

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Molecular Profile Indication/Tumor Type Response Type Relevant Treatment Approaches Therapy Name Approval Status Evidence Type Efficacy Evidence References
RB1 loss retinoblastoma sensitive HDAC Inhibitor Trichostatin A Preclinical - Cell culture Actionable In a preclinical study, Trichostatin A (TSA) inhibited growth of retinoblastoma cell lines in culture (PMID: 18483379), which have been demonstrated to be deficient in RB1 (PMID: 23498719). 23498719 18483379
RB1 loss estrogen-receptor positive breast cancer resistant Palbociclib Preclinical - Cell culture Actionable In a preclinical study, RB1 loss was associated with acquired resistance to Ibrance (palbociclib) in an estrogen-receptor positive breast cancer cell line in culture (PMID: 27020857). 27020857
RB1 loss Advanced Solid Tumor no benefit Palbociclib Preclinical Actionable In preclinical studies, the CDK4/6 inhibitor, Ibrance (palbociclib), was not effective in a variety of solid tumors with Rb1-deficiency (PMID: 26649278). 26649278
RB1 loss retinoblastoma sensitive Sirolimus Preclinical Actionable In a preclinical study, Sirolimus (rapamycin) decreased tumor occurrence, tumor hypoxia and tumor vascularization in a retinoblastoma mouse model with functionally inactivated Rb protein (PMID: 21468343, PMID: 1689463). 21468343 1689463
RB1 loss neuroendocrine tumor sensitive Sirolimus Preclinical Actionable In a preclinical study, Sirolimus (rapamycin) slowed pituitary tumors and decreased the occurrence of thyroid tumors in Rb1+/- mice (PMID: 23454836). 23454836
RB1 loss lung small cell carcinoma resistant Trilaciclib Preclinical - Cell line xenograft Actionable In a preclinical study, RB1-deficient small cell lung cancer cell lines were resistant to Trilaciclib (G1T28) in culture and in xenograft models (PMID: 26826116). 26826116
RB1 loss retinoblastoma sensitive HDAC Inhibitor Entinostat Preclinical - Cell line xenograft Actionable In a preclinical study, Entinostat (MS-275) inhibited growth of retinoblastoma cell lines in culture and inhibited tumor growth in a retinoblastoma cell line xenograft model (PMID: 18483379), which have been demonstrated to be deficient in RB1 (PMID: 23498719). 23498719 18483379
RB1 loss lung small cell carcinoma sensitive Topotecan + Trilaciclib Preclinical - Cell line xenograft Actionable In a preclinical study, the combination of Trilaciclib (G1T28) and Hycamtin (topotecan) inhibited tumor growth in RB1-deficient small cell lung cancer cell line xenograft models, with greater efficacy than Hycamtin (topotecan) alone (PMID: 26826116). 26826116
RB1 loss retinoblastoma sensitive HDAC Inhibitor Vorinostat Preclinical - Cell culture Actionable In a preclinical study, Zolinza (vorinostat) inhibited growth of retinoblastoma cell lines in culture (PMID: 18483379), which have been demonstrated to be deficient in RB1 (PMID: 23498719). 23498719 18483379
RB1 loss Merkel cell carcinoma sensitive LY3295668 Preclinical - Pdx & cell culture Actionable In a preclinical study, LY3295668 resulted in cell growth inhibition and increased cell cycle arrest and apoptosis in Merkel cell carcinoma patient-derived cell lines with loss of Rb1 expression in culture, and resulted in reduced tumor growth in a patient-derived xenograft (PDX) model (PMID: 34359608). 34359608
Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
NCT03360721 Phase II Abiraterone + Apalutamide + Prednisone Study of Apalutamide and Abiraterone Acetate in Castration-Resistant Bone Metastatic Prostate Cancer Patients Suspended USA 0
NCT04592237 Phase II Niraparib Cabazitaxel + Carboplatin + Cetrelimab Cetrelimab + Niraparib Cabazitaxel, Carboplatin, and Cetrelimab Followed by Niraparib With or Without Cetrelimab for the Treatment of Aggressive Variant Metastatic Prostate Cancer Recruiting USA 0
NCT04388852 Phase I DS-3201b + Ipilimumab DS3201 and Ipilimumab for the Treatment of Metastatic Prostate, Urothelial and Renal Cell Cancers Recruiting USA 0
NCT05384119 Phase Ib/II C188-9 + Palbociclib Study of TTI-101 in Combination for Participants With Metastatic Hormone Receptor-Positive and Human Epithelial Receptor 2 (HER2)-Negative Breast Cancer Recruiting USA 0
NCT02873975 Phase II Prexasertib A Study of LY2606368 (Prexasertib) in Patients With Solid Tumors With Replicative Stress or Homologous Repair Deficiency Active, not recruiting USA 0
NCT04116541 Phase II Alectinib Cabozantinib Ribociclib + Siremadlin A Study Evaluating the Activity of Anti-cancer Treatments Targeting Tumor Molecular Alterations/Characteristics in Advanced / Metastatic Tumors. (MegaMOST) Recruiting FRA 0
NCT01976169 Phase I Ado-trastuzumab emtansine + Palbociclib Phase 1b Study of PD-0332991 in Combination With T-DM1(Trastuzumab-DM1) Completed USA 0
NCT03356223 Phase II Abemaciclib Evaluation of ABEMACICLIB Monotherapy in Patients With Locally Advanced/Metastatic Head and Neck Cancer After Failure of Platinum and Cetuximab or Anti-EGFR-based Therapy and Harboring an Homozygous Deletion of CDKN2A, and/or an Amplification of CCND1 and/or of CDK6 (ABORL) Recruiting FRA 0
NCT04469764 Phase II Abemaciclib + Anastrozole Abemaciclib + Letrozole Abemaciclib Abemaciclib for the Treatment of Recurrent Ovarian or Endometrial Cancer Recruiting USA 0
NCT02059213 Phase II Goserelin Palbociclib Bicalutamide Leuprolide A Phase II Study of Androgen Deprivation Therapy With or Without PD 0332991 in RB-Positive Metastatic Prostate Cancer Completed USA 0
NCT04129151 Phase II Ganitumab + Palbociclib Palbociclib + Ganitumab In Ewing Sarcoma Active, not recruiting USA 0