PhotoA new development in drug design could pave the way for a new generation of drugs that could be used to treat a range of diseases. By precisely targeting genes and sequences that allow diseases to proliferate, the researchers believe that they can stop or slow their progression to make them more manageable.

While the development has only been tested in rat models, researchers have been able to design a drug that decreases the growth of tumor cells in triple negative breast cancer, one of the most difficult forms of the disease to treat.

“The study represents a clear breakthrough in precision medicine, as this molecule only kills cancer cells that express the cancer-causing gene – not healthy cells,” said Prof. Matthew Disney, a researcher at The Scripps Research Institute where the drug was designed. “These studies may transform the way the lead drugs are identified – by using the genetic makeup of a disease.”

Targeting the disease structure

This method for fighting disease involves inserting certain compounds or molecules into the disease’s structure to disrupt harmful activity. For example, the drug created to fight triple negative breast cancer, called Targaprimir-96, causes breast cancer cells to kill themselves by targeting a specific RNA.

The researchers have named this sort of approach Informa; by creating compounds that specifically attach onto RNA folds – usually specific ones called microRNAs – the activity of dangerous genes can be suppressed. MicronRNAs can be thought of as a sort of dimmer switch for certain genes, and they are not always helpful.

For example, the MicroRNA that was targeted for triple negative breast cancer actually promoted cancer by discouraging cell death, which invariably allowed cells to grow out of control and form tumors. By attaching a compound to this microRNA, scientists are able to allow cell death to happen so that tumor formation decreased.

Opening the door for new treatments

The researchers believe that this new method of developing precise drug candidates can be a huge improvement over existing therapies, which can kill cells indiscriminately or cause harmful side effects. It may also open the door to treating diseases that were considered untreatable up until this point.

“In the future we hope to apply this strategy to target other disease-causing RNAs, which range from incurable cancers to important viral pathogens such as Zika and Ebola,” said Sai Pradeep Velagapudi, first author of the study.

The full study has been published in the Proceedings of the National Academy of Sciences

Share your Comments