MTL-CEBPA, a Small Activating RNA Therapeutic Upregulating C/EBP-α, in Patients with Advanced Liver Cancer: A First-in-Human, Multicenter, Open-Label, Phase I Trial.
Debashis SarkerRuth PlummerTim MeyerMikael H SodergrenBristi BasuCheng Ean CheeKai-Wen HuangDaniel H PalmerYuk Ting MaT R Jeff EvansDuncan R C SpaldingMadhava PaiRohini SharmaDavid J PinatoJames F SpicerSarah HunterVineet KwatraJoanna P NichollsDavid CollinRobert NutbrownHelen GlennySonia FairbairnVikash ReebyeJon VoutilaStephanie DormanPinelopi AndrikakouPeter LloydSteve FelsteadJenni VasaraNagy A HabibChris WoodPål SaetromHans E HuberDavid C BlakeyJohn J RossiPublished in: Clinical cancer research : an official journal of the American Association for Cancer Research (2020)
MTL-CEBPA is the first saRNA in clinical trials and demonstrates an acceptable safety profile and potential synergistic efficacy with TKIs in HCC. These encouraging phase I data validate targeting of C/EBP-α and have prompted MTL-CEBPA + sorafenib combination studies in HCC.
Keyphrases
- clinical trial
- open label
- phase ii
- phase iii
- study protocol
- cancer therapy
- double blind
- endothelial cells
- electronic health record
- signaling pathway
- induced pluripotent stem cells
- pluripotent stem cells
- big data
- randomized controlled trial
- drug delivery
- placebo controlled
- squamous cell carcinoma
- case control
- rectal cancer