Translational Medicine
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Transcript of Translational Medicine
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Translational Medicine
Turning Basic Research into Medicines and Treatments
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From Idea to Pill or Device
The odds are not good
Between 1979 and 1983, 101 discoveries reported that could lead to a medical treatment. Only 5 received license for clinical use. Only 1 was a popular treatment.
Source: Am. J. Med. 114 (2003) 477
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Even When an Idea Is Translated, It Takes a Long Time
From journal report or patent to product:
Range: 14 to 44 yearsMedian: 24 years
Source: Science. 321 (2008)1298
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Illustration of the Process
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Identify a TargetFor medical devices and appliances: IDENTIFY YOUR TREATMENT OBJECTIVE
Examples: make more effective or longer lasting artificial joints, develop an artificial heart,
For pharmaceuticals: IDENTIFY A BIOCHEMICAL REACTION THAT CONTROLS THE BODY FUNCTION THAT NEEDS TREATMENT.
Examples: insulin regulates blood sugar, leptin controls appetite.
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H2S: A Target Example
Yes, we do mean that smelly gas from rotten eggs.
The human body makes it too, and it is a signaling molecule that relaxes blood vessels (and lowers blood pressure).
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Now We Have a Target for Developing a New Blood
Pressure Medicine
Strategy: develop a chemical that1. activates the enzymes that
make H2S, OR
2. stimulates formation of the body chemical that naturally activates the H2S enzymes (calcium-calmodulin)
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The Development Process
• Understand the relevant science• Develop a prospective treatment• Develop an assay system (How
will you measure the treatment effect?)
• Test it in a suitable animal model. Preclinical trials.
• Begin a formal clinical trial process
• Get approval fromFDA to market
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Clinical Trials at TAMU College of Veterinary Medicine
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Veterinary Drug Development
Dr. Heather WilsonVeterinary Small Animal Clinical Sciences
College of Veterinary Medicine and Biomedical Sciences
Texas A&M University
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Preclinical Trials
Must be done in experimental animals. Purpose:
1. Confirm expected effects.2. Get an idea on proper doses.3. Establish toxicity.
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The Clinical Trial ProcessFour Phases:I test a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
II test a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
III large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments
IV post marketing studies delineate additional information including the drug's risks, benefits, and optimal use