New drugs with a lot of potential
Biopharmaceuticals, medicines based on biological molecules such as proteins and nucleic acids, have already delivered effective treatments for a number of serious, often hard to treat diseases, such as Crohn’s disease and multiple sclerosis, dramatically improving patients’ quality of life.
The pharmaceutical industry is keen to expand its work on these novel drugs, but biopharmaceuticals still have a number of drawbacks which are hampering their broader application. For example, because these molecules tend to be complex and delicate, most biopharmaceuticals have to be injected; if they were administered orally (a more patient-friendly route), they would be destroyed by the harsh environment of the stomach. Furthermore, even once biopharmaceuticals are in the body, their large size means it is hard for them to get to their targets.
The overall aim of the COMPACT project is to overcome the delivery and targeting bottlenecks for biopharmaceuticals. It will do this by tackling a number of key issues. Firstly, it will identify and characterise the main transport routes across biological barriers and through cell membranes that could be exploited for drug delivery purposes. These include the intestinal barrier, skin barrier, and blood-brain barrier.
Secondly, the researchers will devise and characterise formulations to allow the delivery of peptide and protein-based drugs via non-invasive routes (e.g. orally, via the airways, and via the skin). The team will also work to find ways to get these drugs across the blood-brain barrier.
Another goal involves transporting drugs based on nucleic acids (e.g. RNA) into and through the cell. Throughout the project, the team will use advanced imaging techniques to track the movement of biopharmaceuticals at the (sub) cellular, tissue, and whole body level.
Benefits for patients
Biopharmaceuticals have the potential to improve the lives of many patients with diseases and conditions that are currently hard or even impossible to treat. By finding more effective ways of administering these drugs, and improving their ability to travel through the body to where they are needed, COMPACT will allow more patients to benefit from biopharmaceuticals. Furthermore, designing less invasive administration routes and reducing the dose (and therefore the side effects) and frequency of administration will help to improve patient compliance with treatments.