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 years
• Median 24 years

Source Science. 321 (2008)1298
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Illustration of the Process
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Identify a Target
For 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 that
• activates the enzymes that make H2S, OR
• 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 Wilson
• Veterinary Small Animal Clinical Sciences
• College of Veterinary Medicine and Biomedical
  Sciences
• Texas AM University

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Preclinical Trials

• Must be done in experimental animals.
• Purpose
• Confirm expected effects.
• Get an idea on proper doses.
• Establish toxicity.

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The Clinical Trial Process

• Four 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