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.


Inhalable Chemotherapy May Offer Superior Lung Cancer Treatment

A new drug delivery system attaches chemotherapy drugs to nanocarriers–microscopically tiny particles–to create an inhalable lung cancer treatment. Because this approach delivers drugs directly into the lung, it minimizes their effects on the rest of the body, potentially reducing side effects. The drugs also reach the lungs in their most potent form, without being degraded after injection into the bloodstream during transport to the lung. In addition to chemotherapy, the nanocarriers can also deliver another type of small particle, called siRNA, designed to combat drug resistance. In a recent study, this inhalable combination treatment drastically shrank lung cancer tumors implanted in mice, and resulted in 83% of the drug being delivered to the lung, compared to 23% when the drug was injected. However, further testing is necessary before this treatment can be investigated in human clinical trials.


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…”


Diacylglycerol kinase alpha is a critical signaling node and novel therapeutic target in glioblastoma and other cancers

While Diacylglycerol kinase alpha (DGKα) has been linked to several signaling pathways related to cancer cell biology, it has been neglected as a target for cancer therapy. The attenuation of DGKα activity via DGKα-targeting siRNA and small-molecule inhibitors, R59022 and R59949, induced caspase-mediated apoptosis in glioblastoma cells and in other cancers, but lacked toxicity in non-cancerous cells. We determined that mTOR and HIF-1α are key targets of DGKα inhibition…”