Question: What are the top priorities for drug development at the moment? ...How are these priorities determined?

Fiona Scott answered on 7 Mar 2019: last edited 8 Mar 2019 12:22 am

There are so many things to take into account when developing a drug. I guess the main things are we want our drugs to be 1. potent – that they work well without having to give a patient too much of them. 2. specific – we know exactly how it works and 3. safe, causing as few side effects as possible.

Potency is determined by just measuring the effect of giving the drug to a cell/animal/patient and plot a “dose response curve” graph where you work out what the ideal dosage is that maximises the desired effect of the drug without causing any toxicity.

Specific – this is the hard one. We have about 40,000 different enzymes/proteins in our bodies and it is currently impossible to check if your drug only hits the one that’s involved in your disease against every other protein. For example, there are believed to be 518 kinase enzymes but I’ve only been able to test my drugs against 400 of them using the most advanced kinase test techniques. All we can do is test against as many of the important ones (e.g. hERG that affects the heart, kinases involved in cell division etc.) in a test tube before giving them to an animal/person.

Safe – this is done in different ways. Some overlap with above e.g. if a drug affects the hERG enzyme by a certain amount, its progress to market is immediately halted because it is not safe to interfere with a patient’s heart, regardless of what disease you’re treating. I guess this is done by observing all the symptoms of animals/patients as the drugs progress through the development process and as soon as any red flags occur to investigate and fix them asap before allowing a drug to progress further!