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 yearsMedian: 24 years

Source: Science. 321 (2008)1298

Illustration of the Process

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.

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 that1. 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 WilsonVeterinary 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 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