C3a-Like Small Molecule To Replace Expensive Protein Therapeutics

Researchers in Australia have pioneered a drug development technique that could pave the way for a new class of low-cost medicines.

AsianScientist (Dec. 12, 2013) – Researchers in Australia have pioneered a drug development technique that could pave the way for a new class of low-cost medicines.

The research study, published in the journal Nature Communications, was led by Professor David Fairlie and Dr. Robert Reid from the University of Queensland’s Institute for Molecular Bioscience (IMB). They designed a technique that reduces large proteins to small molecules suitable for use as drugs.

Professor Fairlie said the result was a smaller, more affordable version of a powerful human inflammatory protein, complement protein C3a, that helps defend against disease.

C3a costs thousands of dollars per milligram to manufacture commercially and degrades in minutes in blood, making it too expensive and unstable to be easily used in medicines. In this study, the researchers designed a small molecule that retains the same potent activities of C3a but is much cheaper and more stable for drug development.

“Despite the importance of proteins to nearly every function in the body, their use in science, industry and medicine is significantly restricted by their high cost and instability,” said Professor Fairlie.

“A holy grail in chemistry has been to find a way to reduce large proteins down to much smaller, simpler and cheaper molecules with the same activities. We have done exactly that, opening up exciting new avenues for chemists to downsize valuable human proteins and obtain affordable new diagnostics and drugs for the detection and treatment of human diseases.”

The article can be found at: Reid R et al. (2013) Downsizing a human inflammatory protein to a small molecule with equal potency and functionality.

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Source: The University of Queensland.
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