The main aim of translational medicine (TM) is to use academic, medical and industries
specialist knowledge in order to forward our understanding of human healthcare. Increasing our
understanding of human healthcare will get us in good stead for developing the most effective
therapies for diseases and as such will advance the healthcare of society (Parks & Disis, 2004).
As the CNS diseases market is one of the largest and fastest growing markets in the
pharmaceutical industry (Business Insights, 2008) there will be a huge amount of unmet medical
needs if there is to be a barrier to progression of these therapies due to the way in which drugs
are developed. It is important for us to overcome these unmet medical needs by using TM as a
means to do this; to improve the process of drug discovery and through this learn more about the
mechanism of the disease. It will reduce the number of late stage clinical failures which are a
huge cost to pharmaceutical companies and will hopefully reduce the safety concerns due to the
clinical specificity of many CNS drugs (Business Insights, 2008; Riley, 2008). TM will not only
reduce costs but make more effective drugs. In order to do this, knowledge transfer between
specialists in academia, medicine, and industry must happen.
Parkinson’s disease (PD), like most CNS diseases, requires scientists and clinicians who
are highly specialised to understand and deal with the disease. TM exploits these specialised
skills and bridges the gap between these fields in order to make an environment where
information can be shared in order to develop our understanding of the disease (Parks & Disis,
2004; Mintzberg, 1981). This collaboration, in addition to that with industry, may overcome some
of the high risk strategies used today to develop effective therapeutics for PD. It will mean moving
from a ‘Walmart Principle’ of low margins and large volumes, to a ‘Hermes Principle’ of quality
and margins at the expense of volume (Fitzgerald, 2005). An example of improvement of the
quality of therapeutics through TM is the development of biomarkers which would exploit our
understanding of the disease in non-human models in order to predict the outcome of new
compounds in humans (see Fig. 1) (Horig et al., 2005).
It must be noted that although TM can potentially speed up the process of understanding
about PD by using effective knowledge transfer, we are still a long way from a preventative
method for PD. PD research needs to overcome a few big hurdles in order to make effective
progress and these include understanding the cause, early detection and finally progression of
the disease (PD society, 2009).
Finally, in a country where healthcare is nationalised we think we do not have to pay the
cost of drug development. However, not only has PD got ramifications for quality of life and
mortality, but it’s costs for hospitalization, drugs and social services puts a huge strain on the
NHS which in the long run puts costs onto us as a taxpayer (Lindgren, 2004). TM can not only
help us understand about PD in more ways than one, it will help us develop effective detection,
treatment and prevention which will reduce these huge social and economic costs.
Figure 1.
Biomarkers can predict outcome of new
compounds.
Can be applied at early clinical stage of
development indicating any problems which
may occur in later stages.
Reduced costs.
Help with go-no go decisions.
Help optimizes clinical dose.
Individual therapies may become possible
due to increased capabilities to understand
the causes of individual differences in
response to therapies.
Do this with phenotyping (genomics and
proteomics), biomarkers, and correlating this
information with disease aetiology.
Help also to refine dose selections.
Help understanding of drug efficacy and
safety.
Mechanism of the disease
understood in more detail due to
genomic and proteomic screens of
tissue or fluid.
Space in the market for
TM to make its mark as
an efficient process for
therapeutic
development, therefore
more funding for this
type of research due to
effective outcomes.
PARKINSON’S
DISEASE
Screening of tissue or fluid removed
after clinical trial in patients at all
stages of therapeutic development.
Have better understanding of genetic
factors influencing response to
certain compounds.
Productive testing and validation of
these new compounds through basic
science research.
Genetic and functional biomarkers predictive of
genetic risk, capable of early detection, and/or
providing accurate prediction of response to treatment.
Figure 1. Diagram describing the possible benefits of translational medicine in developing our
understanding of and development of therapeutics for Parkinson’s disease (Horig et al., 2005;
Fitzgerald, 2005; Tucker, 2005).
Parks M. R. & Disis M. L. Conflicts of interest in translational research. Journal of Translational
Medicine 2004. 2: 28
Business Insights. The CNS Market Outlook to 2013: Parkinson’s disease. Business Insights Ltd
2008; pp. 47-51
Riley S., The Pharamaceutical Market Outlook 2018: Regulatory hurdles. Business Insights Ltd
2008; pp. 29-32.
Fitzgerald G. Anticipating change in drug development: the emerging era of translational
medicine and therapeutics. Nature Reviews Drug Discovery 2005; 4:815-818
Horig H., Marincola E. & Marincola F. Obstacles and Opportunities in Translational Research,
Nature Medicine 2005, 11:705-708.
Mintzberg H. Organizational design: Fashion or fit? Harvard Business Review 1981. 59: 103-116.
Tucker J. The CNS Market Outlook to 2010: Parkinson’s disease. Business Insights Ltd 2005; pp.
41-44.
Lindgren P, Economic eviodence in parkinson's disease: a review. Eur J Health Econom Suppl 1,
S63-6, 2004