Jacek Jemielity, Joanna Kowalska, Edward Darżynkiewicz and team (Poland)

Invention: Stabilised mRNA for new therapies for cancers and genetic defects

The discovery of more stable messenger ribonucleic acid (mRNA) compounds by a group of Polish researchers paves the way for new therapies for cancers and inherited genetic diseases. Jacek Jemielity, Joanna Kowalska, Edward Darżynkiewicz and their team invented the so-called beta-S-ARCA and beta-B-ARCA compounds that are now finding their way into vaccines and anti-cancer medications.

Thanks to research spanning nearly four decades, the Polish scientists have injected fresh vigour into the field of protein biosynthesis for personalised medicine with their methods for stabilising mRNA.

Their more stable mRNA is not a treatment itself but offers an improved delivery system for therapies using the body's genetic communication channels (its mRNA). The patented invention enables delivery of modified mRNA that can withstand the human body's enzymes. The more stable mRNA is five times more effective and lasts three times longer within a cell than naturally occurring mRNA molecules.

Societal benefit

While survival rates for cancer have greatly improved over the past decade, treatment of the disease can take a heavy toll on patients, especially because of the side effects of chemotherapy. With projections that two out of every five people can now expect to get cancer in their lifetime, personalised medicine could be key to saving lives.

The therapeutic potential of mRNA opens up the possibility of "programming" the human immune system to produce proteins to help fight specific diseases without directly altering a patient's DNA - so far a relatively risky and difficult endeavour. The team's invention may prove to be a powerful asset as scientists unlock the full potential of human DNA. Currently, more than 1 800 disease genes have been identified and more than 2 000 genetic tests have become available, yet there are a total of 20 000 genes in the human genome.

Economic benefit

The scientists at the University of Warsaw (UW) were ahead of the curve in researching more stable forms of mRNA as a vehicle for therapeutics: their research extends back to the 1980s. After discovering promising mRNA compounds, their findings were confirmed, and the invention refined, by a team at Louisiana State University Health Sciences Centre, USA, led by Prof Robert E. Rhoads and Dr Ewa M. Grudzian-Nogalska. They filed for key European patents for mRNA technology in 2008 and established a partnership with German biopharma company BioNTech to bring their patented mRNA stabilisation method to market.

Clinical trials began in 2010, and in the following years BioNTech went on to license mRNA technology to major pharmaceutical companies, including French multinational Sanofi and Swiss multinational Roche's US-based Genentech. Joining forces with Genentech, BioNTech is testing the technology as a stand-alone treatment, as well as in combination with Roche's anti-cancer drug Tecentriq.

According to experts at Market Research Future, the global personalised medicine market is expected to reach EUR 72 billion by 2022, more than doubling in value from EUR 32 billion in 2015. North America is leading the market, followed by Europe. The main drivers are increased patient involvement in healthcare, integrated data from a wider range of sources, integration of wireless technologies with portable healthcare devices and an increase in genetic diseases.