Leukaemia Busters’ Scientific Director, Dr David Flavell answers your questions about biomedical research and how it has worked so successfully over the years to improve medical treatments.

The enormous progress that has been made in the development of new treatments for a wide variety of human diseases has been entirely due to medical research conducted over many decades. But how exactly does the research process work and what does it actually entail? To many people this remains a bit of a mystery. This series of eight most frequently asked questions and answers is intended to throw a spotlight on key parts of the process and help demystify the way in which biomedical research actually works.

Q1. How do you know where to start with your research?

A. This is the question I am asked most frequently. The most important starting point is to identify the medical need, what might be needed to meet it and then, very importantly, to understand the subject area inside out and what other researchers have done or are doing. Where to start the research then falls into place naturally. Usually there are several different ways of approaching the research question to tackle the same problem.

Q2. Where do you get your ideas to do research?

A. This question really follows on from the one above. Having acquired a critical mass of knowledge and gained an insight into a medical need, ideas about how to tackle this problem follow naturally to any inquisitive scientist with appropriate training. It is also very much a question of learning from what others have done previously (see Q4) and then building on that to conduct additional focussed research that is hoped will lead to new discoveries and treatments.

Q3. How do you let everybody know the results of your research?

A. It is every scientist’s professional responsibility to publish the results of their research for all to see. This is usually achieved by publishing their original results as research papers in international scientific journals (most of which are now available over the internet) but also through chapters in specialist books and presentations at large and small national and international gatherings of fellow scientists. Communicating results in all its different forms is an essential part of the research process that lets the world community of researchers know exactly what has been done by you and of course vice versa what has been done by others. Often different groups of scientists working in different institutions or even different parts of the world discover that each is pursuing the same research interest and this often leads to close collaborations between the two groups that can result in an acceleration of the discovery process.

Q4. How do you know you’re not just repeating something that somebody else has already done?

A. Another frequently asked question to which there are at least two answers. Firstly as alluded to in the previous question, the scientist’s mantra is to publish their research results. I regularly scan the medical literature in my area of interest constantly looking for new discoveries and facts, probably spending at least 50% of my working time doing so. Any scientist worth his salt should know if something has been done or not through this process of keeping abreast of published and unpublished work. There are some circumstances where it is desirable to repeat somebody else’s work if nobody else has done so before. This is in order to provide important independent confirmation that the research finding is for real and not just an experimental artefact.

Q5. What is meant by hypothesis led research?

A. Most research is said to be hypothesis led. So for example when an observation is made about something, for instance that cells respond to a drug in a certain way that can be measured in lab experiments but it is not understood how or why this occurs then it is the scientist’s job to create a hypothesis in an attempt to explain why. Experiments can then be designed and carried out to prove or disprove the hypothesis. In this way the truth slowly emerges as experiments allow the scientist to build a picture of what is or is not correct. This process leads eventually to a full understanding of that which is under investigation.

Q6. How do the results of your research get put to practical use?

A. Laboratory research findings are translated into practical benefits for patients through a complex and expensive process that involves drug design, laboratory‑based drug testing for safety and efficacy, drug manufacture and finally testing in patients enrolled onto clinical trials. Only after the drug has been used in many hundreds or even thousands of patients under very stringent clinical testing conditions can a reliable judgement be made as to the new drugs safety and worth in treating the disease it was designed for. Many new drugs fail this process in the early phases of clinical trial testing and then for the scientists involved it’s back to the drawing board.

Q7. Why does it take so long to do the research and get the practical benefits from it?

A. The whole process described in the Q6 above gives an insight into why this complex and involved process takes as long as it does. It’s also important to realise that both scientists and physicians proceed with their work very cautiously in order to avoid at all costs any serious damage to patients. On average from beginning to end it takes fifteen years to develop a new drug for treatment, a time that is thought to be too long by some and which scientists and doctors are always striving to reduce. Add to this that up to ninety percent of promising new drugs fail in clinical trials, usually because they are too toxic for the patient then you begin to understand why this is such a long process.

Q8. Why is research so expensive?

A. As you’ll have gathered from all the answers above, the research process, if done properly, is quite slow by its very nature. A meticulous approach is needed; it is very easy to get false results that are of no use to anyone if the approach is sloppy or experimental work is inadequately controlled. It is also necessary to repeat the same experiment more than once, often three or four times to demonstrate that the results obtained are real and not falsely misleading. Take this together with the cost of buying and maintaining expensive laboratory equipment, the cost of salaries for fully trained scientists, the cost of the reagents used in experimental work and all the other overhead costs associated with running a complex laboratory environment, then it becomes clear why the research endeavour is such an expensive one.

Q9. What is the difference between pure research and translational research?

I’ll bet that many of you have heard the words pure research and translational research somewhere or other and wondered how these might differ. Allow me to demystify this by explaining the important differences between the two and how in reality they actually complement each other during the process of making major advances in medicine.

Pure research is considered to be the pursuit of experimental investigations to uncover new facts about some unknown or poorly understood phenomenon without any obvious direct practical application. To put it simply it is the pursuit of knowledge for knowledge’s sake. An example here might be to understand the mechanism by which normal cells repair themselves when they have been damaged in some way. There might not be any obvious practical application for this at the time the research is carried out but the facts that emerge may have use in future.

Translational research on the other hand is the pursuit of research that is focused on having some immediate practical benefit. An example here would be research to develop a new drug for the treatment of cancer. Of course to undertake translational research there has to be a strong starting point and this is where pure research comes in because it is this that provides the necessary facts to move forward on.

You can see that the two types of research are bound to overlap with translational research feeding off the discoveries of pure research. It might be many years or even decades between a discovery made in the laboratory from pure research before other scientists working on  a whole variety of different diseases devise ways of utilizing this “new” knowledge for the practical benefit of patients. The good news here is that the time period between discovery and practical application is speeding up as modern research techniques, computers and the science known as informatics (meaning the storage and analysis of very large amounts of information) has developed in recent years That is why the sharing of new information is such an important vehicle in enabling us to make the advances that are so urgently needed.

It is so important that we continue to pursue pure research on the understanding that the findings that arise are not always immediately obvious. At the Simon Flavell Leukaemia Research laboratory we pursue both types of research in our quest for new and improved treatments for all patients with leukaemia and other blood cancers that includes lymphoma. Of course we also borrow the research findings reported by other scientists and build on these in pursuit of our life saving objective. Most importantly our scientists always publish the results of our research in high quality international medical journals so that it is always available to other scientists and doctors in the international biomedical research community. By supporting us you are contributing directly to this process and helping us to forge ahead with progress that is set to benefit future generations.