RNAi Therapeutics and Cancer Research

When there's a will, there is a way.
I will start with the story of accidental finding in Molecular Biology.
Two scientists were experimenting with a special model organism called C. elegans.
This is what they look like.

Scientists have mapped how every cell divides from conception onward (there are exactly 959 cells found in hermaphrodites and 1031 cells in males). However, when these scientists fed the worms something called "Double-Stranded RNA", the cells chewed up that Double-Stranded RNA and other normal RNAs that look like it in the cell. RNAs are used by the cells to make proteins, which carry out the most vital and fundamental processes in the cell. They had unknowingly discovered an old mechanism cells use to combat viral infection, some of which, use Double-Stranded RNA.

Shortly after this discovery, many in the community were intrigued with the idea of selectively degrading transcripts that are abherrantly expressed in cancer cells.

The challenge lies in this: C elegans are made up of around one thousand cells, while humans are made of many more than that. How do you create enough of the RNAs to have an effect on as many human cells? How do you get them to the correct cells (If the original cancer growth started in the liver, say, then any growths that come from that are also more like liver cells they came from than anything else.)

Despite how young the technique is, many of the tissue specificity issues are being addressed and the list of diseases currently being tested are extensive:

-Bevasiranib siRNA by Acuity Pharmaceuticals is being tested to treat macular degeneration. It is currently in phase III trials (As of 2009).
-ALN-RSV01 by Alnylam Pharmaceuticals is being tested to treat Respiratory Syncytial Virus and is in phase II trials.
-NUC B1000 by Nucleonics is being used to treat Hepatitis B. It is in Phase I trials.
-Anti-tat/rev shRNA (small hairpin) by Benitec is being "pilot feasibility" studied to treat AIDS.
-CALAA-01 by Calando Pharmaceuticals is being designed to treat solid tumors and is in phase I trials.

We'll keep our fingers crossed!