Fundamental knowledge helps us understand and fight disease
Proteins play an important role in the many complex processes in the human body. Knowledge of these processes and the role of proteins in them helps us understand what goes wrong in the body when a person becomes ill. Once chemists have found out which proteins play a crucial role in certain disease processes, they are able to make small molecules that inhibit or alternatively activate these proteins. If we can ensure that these molecules work in an extremely targeted fashion, they can serve as an efficient treatment with the minimum side-effects.
Molecules as building blocks for new drugs
When chemists develop molecules that alter the function of proteins, they draw as much inspiration as possible from nature. This is because it is known that substances that occur naturally are soluble – an essential condition for medication – and that they can survive in cells without disrupting essential bodily processes. This makes the likelihood of success that much greater than with molecules that chemists develop without first knowing what properties they will have. In the next stage of the process, promising molecules are further developed into usable drugs.
Efficient drug development process
Developing suitable molecules into drugs is a process that takes years and that spans from the test tube to testing on healthy test subjects and patients. During this process scientists regularly find that a substance that seemed promising at an early stage falls by the wayside later on, because it does not work as well on living organisms or because it causes side-effects. Better predictive testing is therefore necessary to indicate at an early stage whether a certain molecule will be effective as a medication for humans. Researchers in Leiden use advanced research methods for this, such as the organ-on-a-chip technology that was developed by the Leiden Academic Centre for Drug Research. Another method is to test drugs on model organisms such as the zebrafish. This provides an accurate prediction of the effectiveness of drugs.
Leiden University and the LUMC possess knowledge of the whole chain of drug development from fundamental research into suitable molecules right up to the clinic. With the Leiden Bio Science Park just a stone’s throw away, the setting could not be more favourable to work together to make the drug development process as effective as possible.