MicroRNA Profiling Kits Facilitate Drug Toxicity Studies

The objective of a drug developer is to assess toxicology early in the preclinical discovery process, and generate optimized structures with a risk profile that can be advanced into in vivo safety assessment with a reasonable expectation of success.

The Scottish company Sistemic (Glasgow, United Kingdom) offers a highly predictive, relatively simple, and cost-effective way to monitor the toxicity profile of a test compound. Sistemic's "SystemRNA” kits are directed at key therapeutic areas including oncology and inflammation where results they produce will enable better strategic decision making at critical stages of the drug discovery and development process.

The "SistemRNA” platform uses human cells and it is based on changes in the level of microRNA expression upon exposure of the cells to candidate drug compounds. There are only several hundred microRNAs expressed in a cell, whose expression pattern may change after exposure to a bioactive compound. The microRNA response is reproducible, characteristic of compound type, and is a robust early sentinel marker defining the biological interaction between compound and cell. The benefit of "SistemRNA” is that with microRNA profiling, pathway control of the entire biological system of the cell can be analyzed in one assay. Data is kept to a minimum whereas knowledge is greatly enhanced allowing more appropriate decisions to be taken on compound development.

"The validity of our use of microRNA profiling as a sentinel marker of phenotypic effects in model systems is overwhelming, and we are seeing time and time again very strong correlations between microRNA changes and drug effects,” said Dr. Vincent O'Brien, chief scientific officer at Sistemic.

Dr. Chris Hillier, CEO of Sistemic, said, "The strength of Sistemic's approach is that we provide an extremely reliable way to make key decisions on safety and efficacy based on very strong biological information about the properties of your compound without reference to the expected target. This lack of bias produces valuable evidence to support drug development and has the power to create some very exciting opportunities.”

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