1. Translational Medicine Turning Basic Research into Medicines and Treatments

2. 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

3. 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

4. Illustration of the Process

5. 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.

6. H 2 S: 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).

7. Now We Have a Target for Developing a New Blood Pressure Medicine Strategy: develop a chemical that 1.activates the enzymes that make H 2 S, OR 2.stimulates formation of the body chemical that naturally activates the H 2 S enzymes (calcium-calmodulin)

8. 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

9. Clinical Trials at TAMU College of Veterinary Medicine

10. Veterinary Drug Development Dr. Heather Wilson Veterinary Small Animal Clinical Sciences College of Veterinary Medicine and Biomedical Sciences Texas A&M University

11. Preclinical Trials Must be done in experimental animals. Purpose: 1.Confirm expected effects. 2.Get an idea on proper doses. 3.Establish toxicity.

12. 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