In the past years, buzzwords such as ‘precision medicine’ and ‘personalized medicine’ have
been echoing in numerous congresses and articles. It designates a fascinating field that
doesn’t want to believe that health is a matter of individual luck, but a matter of applying
medicine in an individualized and precise way, tailored to both the disease’s mechanism and
the patient’s characteristics, respectively. For instance, immunotherapies are the direct
result of our better understanding of the molecular mechanisms at fault of diseases, saving
the lives of cancer patients and making life bearable for those affected by chronic debilitating
diseases such as rheumatoid arthritis. Personalized medicine is already possible as we
already understand how our genetics can determine how our drug-metabolizing enzymes
behave and impact a drug’s effect. Hitherto, a DNA passport specifically for
pharmacogenetics has already been in use in the Netherlands: a quick check on your
passport may guide your physician in his/her treatment’s choice. Well then, this must be the
era of personalized precision medicine, right?
The truth is, that we are not yet in this era. In fact, certain observations beg the question
whether we will ever enter this glorious period.
The DNA passport, which provides pharmacogenetic information, has found disappointingly
little use into everyday clinical practice. This failure does not originate in the quality of the
scientific research that led to this tool. The Human Genome project’s goal was to generate
tools to better interpret a patient’s genetic make-up in order to scientifically guide treatment
decisions. The DNA passport in itself is practical: it is in the form of a card which easily fits in
your wallet, next to your identity and bank cards. The information and, more importantly,
interpretation can be read from the card. It has widespread use in the psychiatry field and
oncology. It is, however, not used in primary care, although the majority of drugs are
metabolized by 5 CYP-enzymes for which mutations are known to affect the metabolizing
rate. So where did we go wrong? Somewhere in the implementation process obviously.
This example shows us that there are still barriers for such tools to be implemented in
today’s clinical practice; even if it may enable personalized medicine. If we want
personalized medicine to be readily used, we need to better identify these barriers and find
solutions for absolute implementation. This starts early on, while we gather knowledge: how
do we proceed with this knowledge, who are the end-users, what are their practical needs
and how can we meet these? Of course, genotyping every citizen is too expensive. But is it
really? This depends on the advantages personalized medicine has to offer? The patient
gets better treatment from the start and doesn’t have to go through the ‘trial-and-error’
phase. So it really depends on how we define the ‘return on investment’.
In oncology, personalized and precision medicine has recently become an unprecedented
reality. Recently, chimeric antigen receptor-T cell therapies were developed and approved
by the FDA in the battle against various cancers. This treatment is the perfect example of
precision and personalized medicine: the patient’s own immune cells are used to target
tumor-specific antigens through genetic manipulation. With an astonishing survival rate, this
new therapy may dramatically change the oncology landscape. That is, if we let it. What’s
the issue here? The equally unprecedented costs: both tisagenlecleucel and axibactagene
cost 475 000 and 373 000 USD per treatment, respectively. The current payer models are
not in place to deal with such daunting numbers, and will only be complicated if these
therapies will be approved for other indications as well.
It is worrying that we already have the know-how for personalized and precision medicine,
but practical issues block our entry into this new and exciting era. It is like a never-ending
ride to Disney World, where the kids in the back ask that nagging question: are we there
This article is not meant as a negative statement, but rather an encouragement to those who
have the privilege to work in the field of medicine to join the debate. Whether you’re a
physician, a scientist or a regulatory affair specialist; ask yourself regularly the following
questions: How can we prevent a premature halt of revolution in medicine? What do we
need to make personalized and precision medicine feasible and affordable? How do we
define return on investment to enable personalized and precision medicine?
Asking these questions during scientific research is what truly enables translational medicine
and makes the utilization of scientific results more likely. Concomitant with the era of
personalized and precision medicine, we should focus on translational medicine to ensure
implementation of our knowledge as actual bench-to-bedside science.
About the author:
Lynda Grine completed her Master in Biochemistry and Biotechnology in 2010 at the Ghent
University. Right aftewards, she pursued a PhD at the Flemish Institute of Biotechnology
(VIB) with an IWT-grant. Today, she’s doctor-assistant at the Ghent University, an FWO-
project manager and senior researcher at UZ Gent. In 2018, she successfully attained the
‘Translational Medicine’ certificate at the ULB. Next to her daytime job, Lynda remains
passionate about science and wants to let the world know about scientific innovation, today’s
challenges and tomorrow’s opportunities.