Dicerna Pharmaceuticals Initiates Phase 1 Study of DCR-MYC in Patients with Solid Tumors and Hematological Malignancies

“Dicerna Pharmaceuticals, Inc. DRNA +0.67% , a leader in the development of RNAi-based therapeutics, today announced the initiation of a Phase 1 dose-escalating clinical study of DCR-MYC, (also known as DCR-M1711), in patients with solid tumors, multiple myeloma, or lymphoma. DCR-MYC, Dicerna’s first drug candidate to enter clinical testing, is a Dicer Substrate siRNA (DsiRNA) that targets the driver oncogene MYC, which is central to the growth of many hematologic and solid tumor malignancies. Dicerna is investigating DCR-MYC in a variety of tumor types with the initial focus on hepatocellular carcinoma.”

Editor’s note: This new drug may hold promise for people with lung cancer or melanoma, as well as other cancer types.


Dicerna Pharmaceuticals Initiates Phase 1 Study of DCR-MYC in Patients with Solid Tumors and Hematological Malignancies

“Dicerna Pharmaceuticals, Inc. DRNA +0.67% , a leader in the development of RNAi-based therapeutics, today announced the initiation of a Phase 1 dose-escalating clinical study of DCR-MYC, (also known as DCR-M1711), in patients with solid tumors, multiple myeloma, or lymphoma. DCR-MYC, Dicerna’s first drug candidate to enter clinical testing, is a Dicer Substrate siRNA (DsiRNA) that targets the driver oncogene MYC, which is central to the growth of many hematologic and solid tumor malignancies. Dicerna is investigating DCR-MYC in a variety of tumor types with the initial focus on hepatocellular carcinoma.”

Editor’s note: This new drug may hold promise for people with lung cancer or melanoma, as well as other cancer types.


Enhancing Tumor Cell Response to Chemotherapy through Nanoparticle-Mediated Codelivery of siRNA and Cisplatin Prodrug

“The development of acquired chemoresistance is often a clinical problem limiting the successful treatment of cancers. RNAi is showing promising results in human clinical trials. The combination of chemotherapy with RNAi approaches to suppress the expression of proteins involved in the emergence of drug resistance represents a promising synergistic strategy to circumvent or reverse acquired chemoresistance. Such combination therapy approaches require specific delivery vehicles to encapsulate and deliver chemotherapy and siRNA therapeutics simultaneously in a controlled manner. Herein, we describe a nanoparticle technology to codeliver a DNA-damaging chemotherapeutic and siRNAs that impair the cell’s ability to repair the DNA damage. This combination therapeutic approach can sensitize cancer cells to chemotherapeutics and shows superior tumor inhibition compared with monochemotherapy.”


Elevation of Receptor Tyrosine Kinases by Small Molecule AKT Inhibitors in Prostate Cancer Is Mediated by Pim-1

The PI3K/AKT pathway is hyperactivated in prostate cancer but its effective therapeutic targeting has proven difficult. In particular, the antitumor activity of AKT inhibitors is attenuated by upregulation of receptor tyrosine kinases (RTKs) through an uncharacterized feedback mechanism. In this report, we show that RNAi-mediated silencing or pharmacological inhibition of Pim-1 activity curtails AKT inhibitor-induced upregulation of RTKs in prostate cancer cells…”


First-in-Humans Trial of an RNA Interference Therapeutic Targeting VEGF and KSP in Cancer Patients with Liver Involvement

RNA interference (RNAi) is a potent and specific mechanism for regulating gene expression. Harnessing RNAi to silence genes involved in disease holds promise for the development of a new class of therapeutics. Delivery is key to realizing the potential of RNAi, and lipid nanoparticles (LNP) have proved effective in delivery of siRNAs to the liver and to tumors in animals. To examine the activity and safety of LNP-formulated siRNAs in humans, we initiated a trial of ALN-VSP…”


First-in-Humans Trial of an RNA Interference Therapeutic Targeting VEGF and KSP in Cancer Patients with Liver Involvement

RNA interference (RNAi) is a potent and specific mechanism for regulating gene expression. Harnessing RNAi to silence genes involved in disease holds promise for the development of a new class of therapeutics. Delivery is key to realizing the potential of RNAi, and lipid nanoparticles (LNP) have proved effective in delivery of siRNAs to the liver and to tumors in animals. To examine the activity and safety of LNP-formulated siRNAs in humans, we initiated a trial of ALN-VSP…”


First-in-Humans Trial of an RNA Interference Therapeutic Targeting VEGF and KSP in Cancer Patients with Liver Involvement

RNA interference (RNAi) is a potent and specific mechanism for regulating gene expression. Harnessing RNAi to silence genes involved in disease holds promise for the development of a new class of therapeutics. Delivery is key to realizing the potential of RNAi, and lipid nanoparticles (LNP) have proved effective in delivery of siRNAs to the liver and to tumors in animals. To examine the activity and safety of LNP-formulated siRNAs in humans, we initiated a trial of ALN-VSP…”


RNA Interference Drug Demonstrated Activity and Safety in Phase I Trial

WASHINGTON, D.C. — Early results from a phase I, first in-human study indicate that a potential new class of drugs, RNA interference (RNAi) drugs, can be safely administered in humans, according to a researcher who presented data on the safety and…