Inhalable therapy may produce drugs in the lungs

Genetic therapies have long been seen as having great potential, but have been difficult to achieve in practice. The concept generally involves using the body’s own refined systems for generating proteins to build large and complex molecules to treat diseases that resist conventional drugs, by inserting customised stretches of genetic code into the nuclei of cells in affected tissues. One problem has been that the immune system resists such invasion. The MIT team, at the Koch Institute for Integrative Cancer Research and Institute for Medical Engineering and Science, has designed an inhalable form of messenger RNA (mRNA) that could be administered directly to the lungs to treat diseases like cystic fibrosis.

In the body, mRNA conveys genetic information from DNA, the medium that encodes all genetic information, to the ribosome, the molecular machine inside living cells where proteins are synthesised. In genetic therapy, it is the most common medium used to try to “trick” cells into making therapeutic proteins. The problem for genetic engineers has so far been to find safe and efficient ways to deliver mRNA into target cells. It is easily broken down within the body, so needs to be transported inside some kind of protective carrier. The MIT team has been working on materials to stabilise RNA during aerosol delivery, so that it can be inhaled in the same way that asthmatics inhale drugs like salbutamol.