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February 09, 2015
Stephen Shrewsbury
From the January/February issue of HealthCare Business News magazine

By Stephen B. Shrewsbury

If you thought evolution has had its greatest effect upon homo sapiens, higher mammals or even all vertebrates, think again. Possibly evolution is best-utilized by those invisible microbes we have been waging war against for millennia. Great strides were made in our favour with the understanding of antisepsis. That improved understanding was used by James Lister, Florence Nightingale and others to reduce mortality from communicable disease. Then it seemed we might even have won that war when Alexander Fleming happened upon penicillin.

Alas, despite the numerous antibiotics developed in the last 80+ years, bacteria are gradually fighting back against our best medicines. MRSA (Methicillin resistant Staphylococcus aureus), is perhaps the best known of these resurgent bugs, but by no means the only one. Many bacteria that we thought we had beaten have evolved by acquiring resistance capability from other species, or developing it themselves as a result of evolution — survival of the fittest.

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But what about viruses? The recent —and ongoing — outbreak of Ebola in West Africa caught media attention for a while. But while the media has moved on to other news stories, the death toll in Liberia, Sierra Leone and Guinea continues to rise. As yet, no effective antiviral has been rushed to these countries to treat those afflicted; and no vaccine is currently approved to protect those brave health care workers who don protective gear to treat and nurse the sick, or vaccinate the teams now collecting and disposing of the dead. How can we beat these new viral killers that may jump species and afflict homo sapiens?

There is hope. Actually both bacteria and viruses, like homo sapiens, have genes and from those genes (mostly DNA-based), come messages — messenger RNA. These messages are the blueprints that lead to the manufacture of new proteins. In the case of bacteria, they make the proteins themselves. Viruses are more nefarious; they hijack the machinery of the host (us, homo sapiens) and get our cells to make the proteins they need to replicate and spread.

A new type of medicine, called therapeutic oligonucleotides, oligos for short (or gene patches), are currently in research and early development from a whole range of small companies. These drugs are microscopic snippets of synthetic DNA that can actually bind to and block these RNA messages and halt the disease. Back in 2011, while working at AVI BioPharma (now Sarepta Therapeutics) I led a program testing an anti-Ebola gene patch in human safety testing. The results of that work were recently published and suggest that the drug was well tolerated.

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