for bite of science (october 9, 2012) dr. robert kernstock
TRANSCRIPT
For Bite of Science (October 9, 2012)
Dr. Robert Kernstock
You May Be Wondering Why I Am Here?...
• Why Science?• How Did I Get Here?• What is Astellas?• My Job Now?• How are Drugs
made?
Why science, and ‘how did I get here’?
Pharma: From ‘Bad Rap’ to Sought After Field
“Old-School” Thought Current thought• Academic Science
Nobel Aspirations• Pharmaceutical
Science Money-Generating
Current Thinking• Working Together!• Applied Science• Using Each Other’s Strengths• Sharing the Real Rewards
Improving Quality of Life
So Who is Astellas?
At Astellas, our mission is to identify safe and effective medicines for people all over the world
Dermatology
Cardiovascular
Infectious Disease
Urology Immunology
Oncology Central Nervous System
U.S. Therapeutic Franchises
Urology
Immunology
Dermatology
Cardiovascular
Infectious Disease
Oncology
Our Lab Stuff
Ligand BindingGenotyping
Flow Cytometry
CyclesFlu
ore
scen
ce
Industry
Academic Drug DiscoveryMedical Science Liaison
Product Development
Genetic Engineering
Clinical Trial Research
Histology
PharmacodynamicsBiology
Chemistry
Physical Science
Physics
Anatomy
Astronomy
BioPharma: It’s Real World Science!
Science Career Opportunities in Pharma
ScienceDegree
Regulatory&
QA
R&D
Gov’t Affairs
Science Liaison
Business Dev’t
Sales & Marketing
PK Modeling
Pharmaco-vigilance
So How Do Drugs Work?
Cells in our bodies carry out molecular reactions to perform every function, for example:– Digesting your lunch– Moving your finger– Transmitting thoughts in your brain
One molecule interacts with another, which interacts with another, and so on.– These cascades are called chemical pathways
A mistake in one of the steps in a pathway can lead to disease.
Drug molecules interact with a molecule in the pathway, changing their activity.
How Do We Fulfill this Mission?
Drug Discovery then Development
Example Discovered
The Discovery Process
Pre-Discovery:Goal: Understand the disease and choose a target molecule.How: Scientists in pharmaceutical research companies, government, academics and for-profit research institutions contribute to basic research.
3 –
6 Y
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RS
DiscoveryGoal: Find a drug candidate.How: Create a new molecule or select an existing molecule as the starting point. Perform tests on that molecule and then optimize it (change its structure) to make it work better.
PreclinicalGoal: Test extensively to determine if the drug is safe enough for human testing.How: Researchers test the safety and effectiveness in the lab and in animal models.
The Development Process
Phase I Phase II Phase III
Number of Subjects
Preclinical Phase IV
(1 year) (2 years) (4 years)(3-6 years)
Drug Discovery then Development
NUMBER OF SUBJECTS
NDA(2 yrs)
Personalized Medicine
• “The Dream”– Plug and play medicine based on
individual’s gene signature– We are all unique and a ‘conglomerate’ of
our biological relatives
Personalized MedicineEML4-ALK fusions in Lung Cancer
Tyr Kinase
1 1620TM
1 981
Basic WDEML4
Basic
1 1059496
Tyr Kinase
ALK( Anaplastic Lymphoma Kinase )
( Echinoderm microtubule-associated protein like protein 4 )
A fused gene EML4 is connected to ALK (in DNA)ALK fusions exist in 5% of lung cancer. A simple PCR test to detect fusion.
Over 70% of ALK fusion + cancers are cured!
Question & Answer
Special thanks to Astellas and Center of Excellence in Education
www.ibioinstitute.orgwww.finding-your-future.org (SCI: Science Career Investigation)www.scienceworx.org
Mission of a Pharmaceutical Research Company
Take the path from understanding a disease to bringing a safe and effective new therapy to patients.– Select a molecule in a pathway => target– Validate the target– Discover the right molecule to interact with the target– Test the compound in the lab for efficacy and safety– Test the compound in the clinic for efficacy and safety– Gain approval to get the drug to doctors and patients
Our FocusIn the U.S., we are intensely focused on five key therapeutic areas sowe can develop a deep understanding of the disease, our customersand patient needs.
Our ValuesAt Astellas we are committed to providing patients, customers, community and employees with a bright future by changing tomorrow.
Our ApproachAstellas is different, not only because of what we do but because of howwe do it.
What Sets Astellas Apart
A Commitment to Changing Tomorrow
COMMUNITY
Encourage employee involvement and
volunteerism
SCIENCE AND EDUCATION
Building a strong foundation in science to
ignite passion and interest in science
among today’s youth
SUSTAINABILITY
Helping to improve the environment through internal and external
practices
Our efforts align with our strong scientific orientation and a focus on the community we work
and live in
State-of-the-Art R&D Facilities
Astellas Research Institute of America (ARIA)• Based in Illinois, ARIA is committed to strengthening
research in transplantation through:
– Drug targeting research
– Clinical biomarker translation research
– Absorption, Distribution, Metabolism and Excretion (ADME) prediction research
– Collaborations with delegate researchers (KOLs)
Phase 1 - Pharmacokinetics
Time (day)
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0 20 40 60 80 100 120 140
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ASKP1240: PK in 7163-CL-0101 < multiple dose simulation > / model= 720
80 mg (1.0 mg/kg)
Time (day)
0 20 40 60 80 100 120 140
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Day 1, 14, 28, 42, 56
Time (day)
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Time (day)
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Day 1, 14, 28, 56
Time (day)
0 20 40 60 80 100 120 140
0.1
1.0
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Day 1, 7, 14, 28, 56
ASKP1240: PK in 7163-CL-0101 < multiple dose simulation > / model= 720
240 mg (3.0 mg/kg)
Establish Dose and Frequency
The Approval Process
Investigational New Drug (IND):Goal: Obtain FDA approval to test the drug in humans.How: FDA reviews all preclinical testing and plans for clinical testing to determine if the drug is safe enough to move to human trials.Clinical Trials:Goal: Test in human to determine if the drug is safe and effective.How: Candidate drug is tested in clinical setting in three phases of trials, beginning with tests in a small group of healthy volunteers and moving into larger groups of patients.
Review:Goal: FDA reviews results of all testing to determine if the drug can be approved for patients to use.How: The FDA reviews hundreds of thousands of pages of information, including all clinical and preclinical findings, proposed labeling and manufacturing plans. They may solicit the opinion of an independent advisory committee.
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7 Y
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