Transcript Translational Medicine - PEER

Translational Medicine
Turning Basic Research into
Medicines and Treatments
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
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
Illustration of the Process
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.
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).
Now We Have a Target for
Developing a New Blood
Pressure Medicine
Strategy: develop a chemical that
1. activates the enzymes that
make H2S, OR
2. stimulates formation of the body
chemical that naturally activates the
H2S enzymes (calcium-calmodulin)
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
Clinical Trials at TAMU College of Veterinary
Medicine
Veterinary Drug Development
Dr. Heather Wilson
Veterinary Small Animal Clinical Sciences
College of Veterinary Medicine and Biomedical
Sciences
Texas A&M University
Preclinical Trials
Must be done in experimental animals.
Purpose:
1. Confirm expected effects.
2. Get an idea on proper doses.
3. Establish toxicity.
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