Pharmacology

Pharmacology (BMS 610)

Midterm Exam

Fall 2014

100 points

1. Drug Discovery and Approval: You are a drug discovery researcher working with a major pharmaceutical company to identify novel therapeutic targets for the treatment of Alzheimer’s disease. Discuss the steps necessary, from target identification through final FDA approval process for a novel therapeutic agent designed to prevent disease progression associated with chronic disease. Include an estimate of the amount of time necessary to complete each step. (20 points)

Drug discovery must begin with a pre-discovery period in which scientists research the disease, try to examine the underlying causes, and then try to understand the damage of cellular activity. In Alzheimer’s patients, it has been found that there are protein build-ups called beta-amyloid in the brain that can damage the cell-to-cell communication. There are also tau proteins that twist into abnormal tangles inside the brain cells, which can lead to failure of internal support or lack of nutrients transported to cells. Knowing this biological abnormality of the disease is crucial to the next step of drug discovery: identifying the drug’s molecular target. Since MRI scans and further research has shown that the primary cause of Alzheimer’s is accumulation of beta-amyloid tangles in the brain, we can begin to explore a drug mechanism that can antagonistically decrease beta-amyloid production or remove beta-amyloid from the brain through immune system response. Then, we can start screening for compounds or molecules in plants, chemicals, hormones or genetically engineered entities towards a leading compound.

Once a promising compound is created, we must test it’s pharmacokinetics to make sure that it can be absorbed into the bloodstream, distributed to the proper site in the body, it can efficiently and effectively be metabolized, and it can successfully be excreted from the body without becoming toxic. This...