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应该是真的，只是在2012年，还是很有希望：
ALN-VSP: Liver Cancers
Alnylam has advanced its RNAi therapeutic, ALN-VSP, into the clinic for the treatment of liver cancers and potentially other solid tumors with liver involvement.
ALN-VSP targets two key genes each involved in the disease pathway of liver cancer: kinesin spindle protein, or KSP is involved in cancer proliferation, and vascular endothelial growth factor, or VEGF, is involved in the growth of new blood vessels that feed tumors. We believe that a dual-target approach in cancer increases the potential for a significant therapeutic benefit.
In April 2009, Phase I clinical trials were initiated for ALN-VSP. Results of this trial were presented at the American Society of Clinical Oncology (ASCO) 2011 Annual Meeting early June 2011. ALN-VSP is Alnylam's first systemic RNAi program and the Phase I trial results represent an important milestone in the advancement of RNAi therapeutics. Data demonstrate for the first time both clinical activity and RNAi mechanism for an RNAi therapeutic.
The Phase I study showed that ALN-VSP was generally well tolerated. ALN-VSP was administered to 41 patients at doses ranging from 0.1 to 1.5 mg/kg; a total of 209 doses have been administered, with a range of 1 to 28 doses per patient. Based the safety data, the recommended dose for advancement of ALN-VSP into Phase II studies is 1.0 mg/kg. In this study, 29 tumor biopsies were obtained voluntarily from 15 patients across multiple dose levels (from 0.4 to 1.5 mg/kg) using a CT-guided procedure; these included hepatic (liver) tumor biopsies from 11 patients and extra-hepatic tumor biopsies from four patients. The two siRNAs targeting VEGF and KSP that comprise ALN-VSP were detected in nearly all of the biopsy samples evaluable for drug levels at siRNA concentrations ranging from 0.3 to 142 ng/g tissue. These levels of siRNA are pharmacologically relevant since pre-clinical studies have shown that siRNA tissue levels of 1 ng/g are associated with 50% target gene silencing (Landesman et al., Silence, 1:16, 2010).
In June 2012, results from our Phase I extension study were presented at the ASCO Annual Meeting. These data include safety and tolerability of multiple doses of ALN-VSP, as well as evidence for anti-tumor activity in this very advanced, heavily pre-treated cancer patient population. Multiple patients achieved stable disease or better, including a patient with endometrial cancer metastatic to the liver who achieved a complete response. Results from the extension study also give us increased confidence in long-term chronic dosing with RNAi therapeutics delivered via LNPs, as patients have received drug twice a month for up to nearly two years, and approximately 11 months on average. In this study, chronic dosing of up to 23 months with ALN-VSP was found to be generally safe and well tolerated.
The drug is formulated using a first generation lipid nanoparticle developed by Tekmira Pharmaceuticals Corporation. The ALN-VSP program is partnered with Ascletis for HCC in China, and Alnylam will retain all rights in the rest of the world. |
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我勤劳节约的外公外婆 (2010-2-18) 公主 
发表于 2013-9-13 21:57
