BioTech Buzz: New Study Calls RNAi Into Question
Mello's discovery draws scrutiny from researchers in Kentucky
Ribonucleic acid, or RNA, transmits genetic data, usually for cells to take action of some kind. RNAi can tell a gene to be silent. And that ability to silence genes leads some to hope RNAi will help cure all sorts of maladies, including cancer.
Cottage Industry
Worcester's RNAi industry most notably includes biomedical companies like RXi Pharmaceuticals Corp., at 60 Prescott St., co-founded by Dr. Craig Mello, who is a local celebrity for being a co-discoverer of RNAi and a Nobel Prize winner in 2006.
But Mello's research and notoriety has spawned even more activity. At the University of Massachusetts Medical School on Lake Avenue, there are professors and students immersed in a variety of research, including RNAi. In fact the school, where Mello is also a professor, has hired additional RNAi researchers to build up its faculty and research.
While researchers here and elsewhere are focusing on using RNAi to silence specific genes, a researcher at the University of Kentucky says his group's research has determined that two drugs using RNAi that are in clinical trials actually work by triggering the immune system in general, not by targeting and silencing specific genes. Neither of the drugs he studied are from Worcester companies or from work done here.
Researchers and drug companies have matched gene sequences in the body with sequences in RNAi to silence a specific gene. One use has been silencing a gene that creates weak blood vessels in the back of the eyes that can lead to macular degeneration, generally an age-related disease. The disease is most recognizable by the large black sunglasses its sufferers wear.
The theory has been that a specific bit of RNAi can stop genes from making these weak blood vessels. But Dr. Jayakrishna Ambati, the Kentucky researcher, found that the blood vessel creation can be stopped by using any of a number of RNAi sequences, including those not in the two drugs studied. That runs completely counter to the drug makers' claims.
And that finding is a warning that RNAi researchers need to design their research so they can prove beyond any doubts that the effect is due to a specific gene sequence and not the immune system.
While many researchers do research through rigorous design levels, the truth is that many do not, according to Ambati.
"The potential promise of gene interference research is tremendous, " he said, but that promise may not be realized unless researchers are rigorous about how the results are created.
Harvard Medical School professor and senior investigator Dr. Judy Lieberman, who has conducted immune disease research with RNAi, said Ambati's research is something fellow researchers should be aware of.
"I think it's an important paper that highlights a potential off-target effect of RNAis," she said. When scientific research problems like this crop up, researchers are equally skilled in solving them, she said. One answer could be encapsulating the RNAi in a complex material so it reaches the exact location that it should.
But you've got to admire the way scientific minds work, including their ability to see lemonade in what was potentially a pile of lemons. It's part of what makes science so exciting. Dr. Ambati explains it best:
"It has been a very busy month and there has been tremendous interest on the part of the press and the scientific community. They are wondering if that response could be broadened beyond the eyes to other organs. There are over 70 diseases where blocking blood vessel creation would be helpful, like cancer, arteriosclerosis and others, so they are wondering if it can applied in some way to combat them."
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