Data mine your DNA to develop better drugs

from by by Liat Clark

A team of chemists has developed a new computational drug discovery technique that targets microRNAs using genome sequencing. To demonstrate the technique, which the research team believes presents a new and efficient, general-use approach to targeted drug discovery, they developed a drug that caused cancer cells to attack themselves.

“With our program, we can identify compounds with high specificity,” said Sai Pradeep Velagapudi, a graduate student at The Scripps Research Institute and lead author on a paper describing the technique, termed Inforna. “In the future, we hope we can design drug candidates for other cancers or for any pathological RNA.”

Typically, oligonucleotides have been used to target RNA and research tailored drugs. These are short nucleic acid polymers — single-stranded DNA or RNA molecules. Because they readily bind to their opposing nucleotide, they are often synthesised in the lab to use in detecting specific DNA or RNA. In 2012, CEO of RaNA Therapeutics Art Krieg called oligonucleotide therapeutics the “third major drug-development platform”, after small molecules and biologics. MicroRNAs have, in recent years, come to the fore though, and this latest technique has focused on the burst of innovation in the field and promises to aid better small molecule drug delivery. Only discovered in the 90s, they are short molecules, which function as gene controllers — they bind the genes to stop them becoming proteins.

Velagapudi SP, Gallo SM, Disney MD. (2014) Sequence-based design of bioactive small molecules that target precursor microRNAs. Nat Chem Biol [Epub ahead of print]. [abstract]