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| Molecular Profile | CDKN2A loss |
| Therapy | Palbociclib |
| Indication/Tumor Type | chordoma |
| Response Type | sensitive |
| Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|
| CDKN2A loss | chordoma | sensitive | Palbociclib | Preclinical - Cell culture | Actionable | In a preclinical study, Ibrance (palbociclib) inhibited expression of phosphorylated Rb and growth of chordoma cell lines with CDKN2A loss and loss of p16 protein expression in culture (PMID: 26183925). | 26183925 |
| CDKN2A loss | chordoma | sensitive | Palbociclib | Phase II | Actionable | In a Phase II trial (NCT PMO-1601), Ibrance (palbociclib) treatment resulted in modest activity with a disease control rate of 39% (11/28, all stable disease), median progression-free survival of 5.6 months, median overall survival of 24.6 months in patients with advanced chordoma with CDKN2A or p16 loss and retained CDK4/6 and RB1 expression (PMID: 40627883; NCT03110744). | 40627883 |
| PubMed Id | Reference Title | Details |
|---|---|---|
| (26183925) | Preclinical Characterization of Novel Chordoma Cell Systems and Their Targeting by Pharmocological Inhibitors of the CDK4/6 Cell-Cycle Pathway. | Full reference... |
| (40627883) | CDK4/6 inhibition in advanced chordoma: final results of the NCT PMO-1601 trial. | Full reference... |