Making the most of the first time a medicine is administered to humans

Collecting as much information as possible about administering a new medicine to people can save a lot of money.

Collecting as much information as possible about administering a new medicine to people can save a lot of money in the further development of a medicine and increase the safety for patients. Moreover, it quickly becomes clear if a promising substance does not in fact make a suitable medicine. The Centre for Human Drug Research (CHDR) in Leiden has developed methods to reach these goals.

How do you measure the desirable and undesirable effects of a potential new medicine using healthy volunteers and patients? How do you translate the available knowledge from the laboratory to safe and informative experiments with human test subjects? Which measurements, tests or scans provide the most trustworthy information? This is the sort of question that CHDR’s clinical pharmacologists are looking to answer. Medicines are tested here on a daily basis, usually commissioned by sponsors from the pharmaceutical industry but also very often in collaboration with researchers from LUMC and Leiden University. In the past, new medicines were only developed by the pharmaceutical industry, but a growing number of medicines are being developed directly in the LUMC.

'Painful' tests

CHDR researches medicines for various complaints and illnesses: from pain to dementia, from multiple sclerosis to heart disease and vascular conditions, from psychiatric disorders to thrombosis. A new medicine is usually tested first on healthy volunteers. Even though they are not ill and have no pain, focused tests can still yield a lot of useful information. It then becomes clear whether the medicine reaches the place where it needs to work (for example, the brain), and various effects can also be identified, for example if the test subject becomes less alert after taking the medicine. With imaging techniques such as PET scans and MRI it is possible to see where the medicine ends up, and whether it causes changes. In order to measure the effect of a painkiller, the CHDR has a number of ‘painful’ tests, such as alternating heat and cold. The clinical pharmacologists also check at intervalsto see if there is a connection between changes in the test subject and the concentration of the medicine in the blood. If that change (for example pain relief, or a side effect such as dizziness) becomes more noticeable as the amount of medicine increases, it is probably an effect of that medicine.

Healthy or ill test subjects

In recent years CHDR has done more and more research with patients, and there is room enough for that in the new building in the Leidse BioScience Park, where the whole of the top floor is dedicated to healthy or ill test subjects. Studies are sometimes also conducted with patients in the LUMC or the VUmc in Amsterdam. In all cases the studies are concerned with small numbers of patients, who are monitored to see whether the new medicine can live up to its promise in any way. CHDR focuses mainly on these early phases of the development of new medicines, although over the last few years the research institute in Leiden has also conducted comparative studies with hundreds of patients. Such a large study is a significant investment for pharmaceutical companies, so it is a great advantage if, thanks to the research at CHDR, it becomes clear at an early stage if a promising substance is not appropriate after all, or if a better estimate can be made of the safest and most effective dose. For patients the benefit is just as clear: an effective treatment with as few side effects as possible.