INTERFERING with our genetic machinery could prove an effective way to fight leukaemia.
The form of the disease called chronic lymphocytic leukaemia (CLL) is characterised by large numbers of abnormal white blood cells triggered by over-expression of a gene called Bcl-2. So Santaris Pharma of H酶rsholm in Denmark has developed a drug that switches off Bcl-2 by binding to its messenger RNA (mRNA) strands and destroying them before they can be translated into protein; this is called RNA interference (RNAi).
Most RNAi-based drugs rely on double-stranded pieces of RNA to destroy mRNA, but these can be difficult to get into cells and often have to be injected locally. Santaris has now created smaller single-stranded molecules termed 鈥淩NA antagonists鈥�, which cross cell membranes easily. This means they can be injected into the bloodstream, and so can be used in a broader range of diseases.
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In early human trials, an RNA antagonist for Bcl-2 led to rapid falls in the white cell blood count, without serious side effects. The results were presented at a meeting of the American Society for Clinical Oncology in Chicago last week.
