By Helen Young
Scientists at The Scripps Research Institute have developed a new drug candidate that lowers the growth rate of tumor cells in animal models1 in one of the most difficult cancers to battle: triple negative breast cancer. This is the first time that scientists have been able to take a genetic sequence and design a drug candidate that is effective against this type of cancer. What makes the molecule so unique is its ability to destroy only the cancer cells that express the cancer gene, leaving healthy cells intact. The research is poised to totally change the way we identify drugs: by working with the genetic makeup of an illness.
The compound designed by the scientists is called Targaprimir-96; it causes breast cancer cells to destroy themselves by targeting a specific RNA that sparks the cancer precisely. In the past, scientists were forced to test millions of possible drug candidates to find a few that successfully affected target areas. The new study makes the process much speedier and more effective. It relies on an approach called Inforna, which is centered on creating compounds that bind to RNA folds. They are particularly effective at targeting microRNAs.
MicroRNAs are a recently discovered class of non-coding RNAs2 that play an important role in regulating gene expression. They typically work as ‘dimmer switches’ for genes, binding to their transcripts and interfering with the protein production process. In the new study, the new compound was tested on animals over a 21-day period. Results showed lesser production of microRNA-96 (which promotes cancer by stopping programmed cell death from occurring) and increased programmed cell death. Because the compound was so precise in its targets, healthy cells were left intact. Scientists expressed that in the future, this strategy might be used to treat other harmful RNAs, which lead to everything from aggressive cancers to powerful viruses such as Ebola and Zika.
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