Download Slide 1: Welcome to my Medicinal Chemistry Site. You`ve come to

Slide 1:
Insights Into
Medicinal Chemistry
Website by Lucas Bohidar
Honors Chemistry
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Welcome to my Medicinal Chemistry Site.
You’ve come to the right place if you’d like to learn about how chemists play a
big role the development of pharmaceutical compounds.
As starting place, I will give you an overview of where medicinal chemistry fits
into the drug development process, and I will also tell you about the different
sections of my website.
Slide 2:
Medicinal Chemistry
• WHY
• WHAT
• WHO
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I chose the topic of Medicinal Chemistry because it shows how important
chemists are in creating drugs that help to improve and save lives worldwide.
Medicinal Chemistry, also sometimes known as Pharmaceutical Chemistry,
focuses on the identification, optimization and formulation of new molecular
entities that are suitable to treat or prevent illnesses and diseases.
Medicinal Chemists are people with diverse backgrounds that combine organic
chemistry, biochemistry, computational chemistry, and pharmacology.
Slide 3:
The Drug Development Process
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The next 3 graphics come courtesy of Johnson and Johnson.
Discovering a new drug has been likened to searching for a “needle in a
haystack”. Hundreds and sometimes thousands of chemical or biological entities
must be made and tested to find one that can achieve the desirable result without
serious side effects.
Going from the laboratory to the patient is a long, expensive and uncertain
process. On average, in order for one medicine to be approved, research must
screen about 10,000 compounds. By the time animal testing, called Pre-Clinical,
is done only 250 compounds have made it through the pipeline. By the time
clinical trial research in humans is done, only 5 compounds have survived, and
out of these only 1 receives approval.
Slide 4:
The Drug Development Process
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If you add up the number of years that are indicated by the red arrows, this
process takes approximately 15 years, averaging 12 years of Research &
Development (R&D) time to bring a drug to market.
Not only is this process time intensive, but extremely costly as well. If you add up
the costs throughout the R&D process, you will find that it costs approximately
$1.7 billion dollars to have that one drug approved.
Slide 5
Focus of Website
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In the long process of drug development, we will focus on role that medicinal
chemists play the research phase.
This is the phase where the compounds are first discovered, then synthesized to
improve their biological properties, and lastly the processes to scale up the
manufacture of the drug formulation are developed.
At the end of this phase the New Molecular Entity declaration means that the
regulatory agencies like the FDA have approved the testing of the drug in
humans.
Slide 6:
· Identify and validate a biological target that has the potential to influence disease
processes.
· Identify “drug-like” molecules that treat the disease without adverse effects and
· In small molecule research, the focus is on the discovery of small molecular drug
candidates or NME’s with the potential to satisfy unmet medical needs. Small molecular
drug candidates are usually synthesized in the laboratory by chemists.
Target Identification
The small molecule research process begins with Target Identification.
The goal is to identify the biological “target” that drives the disease state.
During this phase, biological targets are studied in the laboratory using a series of tests
called assays:
ADD HIGH THROUGHPUT SCREENING HERE
Hit to Lead
The objective of Hit to Lead is to identify and verify the desired activity in the molecular
entity, which means the compound exhibits a desired effect on the disease state.
Keep in mind that a hit demonstrates some activity and a lead demonstrates significant
activity.
Lead Optimization
The purpose of Lead Optimization is to find a single compound that has significant
activity with suitable “drug-like” properties and produces a desired effect on the disease
state, utilizing both in-vitro and in-vivo testing in animals.
Now the work becomes more sophisticated and more people become involved, such as:
o Medicinal Chemists, who design and synthesize molecules in the laboratory.
Using assay results as a guide, they make modifications to the molecules to
optimize desired effect on the target and minimize toxicity
Chemists and biologists work together with a common goal, which is to identify and
optimize a lead and to validate that the lead can influence a disease state that will
eventually be introduced to humans and, ultimately, have commercial value in the
marketplace.
At-Risk Scale-Up
The purpose of at-risk scale-up is to evaluate if the molecule can be made into a form that
can be ingested and absorbed by the body, and then safety testing can be done to
determine if the compound can be considered an NME to develop further.
Larger quantities of the lead substance are required in order to perform large-scale tests.
To accomplish this, we need to develop systems and procedures to produce large
quantities of the lead substance. We must perform experiments to ensure that results
produced at the lab bench are reproducible in large-scale production.
This process is considered occurring “at-risk” because significant investment must occur
to support scale-up and longer-term toxicity studies before the viability of a compound is
determined.
NME Declaration
The purpose of NME Declaration is to determine if the compound meets its
preestablished criteria to transition from Research to initiate testing in humans.
NEED TO POINT OUT THROUGHOUT THE PRESENTATION WHERE THE
INFORMATION FROM THIS WEBSITE FITS INTO THE DRUG DISCOVERY
PROCESS