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Melanoma Therapeutics Foundation
Drug
Discovery and Development Process
Drug discovery and development is a multidisciplinary
process that requires many years of dedicated effort and extensive
financial investment to bring new drugs that benefit patients
to market. Although the efforts are continuous and frequently
iterative, the process can be realized in distinct stages that
require objective decision-making for advancement.
Discovery Research – Beginning
with the study of disease genetics and biology, this discipline
encompasses
the identification and validation of appropriate molecular
targets for therapeutic intervention. Robust assays (biochemical
and biological cell-based) must be developed to screen and
discover compounds or Hits that affect target function. Hits
are further analyzed for their potency and selectivity and
selected based on their ability to demonstrate therapeutic
efficacy in appropriate in vitro and in vivo models of human
disease – Leads.
Preclinical Development – This
is the process of taking a new chemical lead and refining it
in such a way
that allows its testing in humans. Using medicinal chemistry
and pharmacology expertise, extensive Lead Optimization is
undertaken to improve lead compounds’ potency, selectivity
and pharmacological properties, as well as reduce their potential
side effects. Studies are conducted to learn and improve
the lead compound’s interaction with various tissues
within a living organism with respect to how it is absorbed,
distributed, metabolized, and excreted – ADME. This
Pharmacokinetic profile (PK) defines the lifespan of the
drug and its metabolites in the body. Similarly, assays are
developed and studies are done to determine how the drug
affects the molecular target in various tissues, especially
the disease tissue within the organism, Pharmacodynamic
profile (PD). Finally, extensive Toxicology studies are performed
in small and large animal models to identify short and long
term adverse side effects associated with the lead compounds.
Together, the PK, PD and toxicity profiles of the lead compound
are used to decide the appropriate dose and the right regimen
to administer the drug. The synthesis, manufacturing process
and formulation of these selected Development Candidates are
then optimized in preparation for filing an Investigational
New Drug application with the Food and Drug Administration,
FDA.
CLINICAL TRIALS
NEED – ACCELERATED
DEVELOPMENT
LINKS
FDA, Food and Drug Administration http://www.fda.gov/
CEDR, Center for Drug Evaluation and Research http://www.fda.gov/cder/
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Clinical
Trials Scan
Melanoma
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