Skip to main content
Premium Trial:

Request an Annual Quote

Alnylam Starts Phase I Study of siRNA Liver Cancer Drug


Alnylam Pharmaceuticals announced last week that it has initiated a phase I study of its siRNA-based liver cancer therapy ALN-VSP.

The study, the company said, will enroll an estimated 55 patients with advanced liver cancers, including hepatocellular carcinoma and other solid tumors with liver involvement, who are refractory to the standard of care.

The study is designed to evaluate the intravenous drug's safety, tolerability, pharmacokinetics, and pharmacodynamics.

"Other exploratory objectives include the assessment of tumor response through Response Evaluation Criteria for Solid Tumors, a set of published guidelines that define when cancer patients' disease improves, stabilizes, or progresses during treatment; change in tumor blood flow or vascular permeability measured by DCE-MRI; and change in plasma biomarkers of angiogenesis," Alnylam said.

"In addition, the analysis of pharmacodynamic effects of ALN-VSP on tumors will be measured in patients electing to proceed with voluntary pre- and post-treatment biopsies," the company added.

ALN-VSP, formerly ALN-VSP01, comprises two siRNAs: one targeting vascular endothelial growth factor, which is associated with angiogenesis, and the other targeting kinesin spindle protein, which has been linked to cell proliferation in various cancers. It is also formulated in a lipid nanoparticle developed by Alnylam partner Tekmira Pharmaceuticals.

Earlier this year, Alnylam released preclinical data showing that the drug inhibited tumor growth as measured by serum levels of alpha-fetoprotein. It was also found to significantly improve median survival in treated animals versus controls, and boost survival in treated animals compared with ones receiving the approved liver cancer treatment Nexavar (see RNAi News, 2/19/2009).

The preclinical data also suggested that combining ALN-VSP with Nexavar provided an even greater increase in survival benefit, Alnylam said at the time.

The Scan

Positive Framing of Genetic Studies Can Spark Mistrust Among Underrepresented Groups

Researchers in Human Genetics and Genomics Advances report that how researchers describe genomic studies may alienate potential participants.

Small Study of Gene Editing to Treat Sickle Cell Disease

In a Novartis-sponsored study in the New England Journal of Medicine, researchers found that a CRISPR-Cas9-based treatment targeting promoters of genes encoding fetal hemoglobin could reduce disease symptoms.

Gut Microbiome Changes Appear in Infants Before They Develop Eczema, Study Finds

Researchers report in mSystems that infants experienced an enrichment in Clostridium sensu stricto 1 and Finegoldia and a depletion of Bacteroides before developing eczema.

Acute Myeloid Leukemia Treatment Specificity Enhanced With Stem Cell Editing

A study in Nature suggests epitope editing in donor stem cells prior to bone marrow transplants can stave off toxicity when targeting acute myeloid leukemia with immunotherapy.