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Gene | RB1 |
Variant | positive |
Impact List | unknown |
Protein Effect | unknown |
Gene Variant Descriptions | RB1 positive indicates the presence of the RB1 gene, mRNA, and/or protein. |
Associated Drug Resistance | |
Category Variants Paths |
Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
---|---|---|---|---|---|---|---|
RB1 positive | Advanced Solid Tumor | predicted - sensitive | Ribociclib | Phase I | Actionable | In a Phase I clinical trial, Kisqali (ribociclib) demonstrated safety and preliminary efficacy in patients with RB1-positive solid tumors and lymphomas, resulting in partial responses in 2.3% (3/132) of patients and stable disease in 32.6% (41/132) of patients, including 8 patients demonstrating stable disease for greater than 6 months (PMID: 27542767, PMID: 24795392). | detail... 24795392 27542767 |
RB1 positive | glioblastoma | predicted - sensitive | Palbociclib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Ibrance (palbociclib) inhibited proliferation of RB1-proficient glioblastoma cell lines in culture and inhibited tumor growth in intracranial cell line xenograft models (PMID: 20354191). | 20354191 |
RB1 positive | prostate cancer | no benefit | Palbociclib | Phase II | Actionable | In a Phase II trial, addition of Ibrance (palbociclib) to androgen deprivation therapy (ADT) did not improve the rate of PSA less or equal to 4 ng/ml at 28 weeks (80%, 32/40 vs 80%, 16/20, p=0.87), PSA undetectable rate at 28 weeks (43% vs 50%, p=0.5), radiographic response rate (89% vs 89%), or 12-month biochemical progression-free survival (74% vs 69%, p=0.72) in patients with metastatic hormone-sensitive prostate cancer expressing Rb1 as confirmed by IHC (PMID: 33727260; NCT02059213). | 33727260 |
RB1 positive | medulloblastoma | sensitive | Palbociclib | Preclinical - Pdx | Actionable | In a preclinical study, Ibrance (palbociclib) inhibited Rb1 phosphorylation in tumor tissues and improved survival in patient-derived intracranial xenograft models of medulloblastoma (PMID: 27012813). | 27012813 |
RB1 positive | breast cancer | predicted - sensitive | Paclitaxel + Palbociclib | Phase I | Actionable | In a Phase I trial, alternating sequential treatment with Ibrance (palbociclib) and Taxol (paclitaxel) resulted in a median progression-free survival (mPFS) of 209 days in patients with Rb1-positive breast cancer, with a mPFS of 802 days and a clinical benefit rate of 55.6% (5/9) at the recommended phase II dose (PMID: 30635336; NCT01320592). | 30635336 |
RB1 positive | colon cancer | sensitive | Dalpiciclib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, Dalpiciclib (SHR6390) inhibited CDK4/6-RB pathway activation, leading to growth inhibition in an RB1-positive colon cancer cell line in culture, and tumor regression in a cell line xenograft model (PMID: 30724426). | 30724426 |
RB1 positive | Advanced Solid Tumor | sensitive | Dalpiciclib | Preclinical - Cell culture | Actionable | In a preclinical study, Dalpiciclib (SHR6390) inhibited CDK4/6-RB pathway signaling, leading to cell cycle arrest and inhibition of proliferation in a panel of RB1-positive tumor cell lines in culture, and tumor growth inhibition in cell line xenograft models (PMID: 30724426). | 30724426 |