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Slide 1: Insights Into Medicinal Chemistry Website by Lucas Bohidar Honors Chemistry Welcome to my Medicinal Chemistry Site. You’ve come to the right place if you’d like to learn about how chemists play a big role the development of pharmaceutical compounds. As starting place, I will give you an overview of where medicinal chemistry fits into the drug development process, and I will also tell you about the different sections of my website. Slide 2: Medicinal Chemistry • WHY • WHAT • WHO I chose the topic of Medicinal Chemistry because it shows how important chemists are in creating drugs that help to improve and save lives worldwide. Medicinal Chemistry, also sometimes known as Pharmaceutical Chemistry, focuses on the identification, optimization and formulation of new molecular entities that are suitable to treat or prevent illnesses and diseases. Medicinal Chemists are people with diverse backgrounds that combine organic chemistry, biochemistry, computational chemistry, and pharmacology. Slide 3: The Drug Development Process The next 3 graphics come courtesy of Johnson and Johnson. Discovering a new drug has been likened to searching for a “needle in a haystack”. Hundreds and sometimes thousands of chemical or biological entities must be made and tested to find one that can achieve the desirable result without serious side effects. Going from the laboratory to the patient is a long, expensive and uncertain process. On average, in order for one medicine to be approved, research must screen about 10,000 compounds. By the time animal testing, called Pre-Clinical, is done only 250 compounds have made it through the pipeline. By the time clinical trial research in humans is done, only 5 compounds have survived, and out of these only 1 receives approval. Slide 4: The Drug Development Process If you add up the number of years that are indicated by the red arrows, this process takes approximately 15 years, averaging 12 years of Research & Development (R&D) time to bring a drug to market. Not only is this process time intensive, but extremely costly as well. If you add up the costs throughout the R&D process, you will find that it costs approximately $1.7 billion dollars to have that one drug approved. Slide 5 Focus of Website In the long process of drug development, we will focus on role that medicinal chemists play the research phase. This is the phase where the compounds are first discovered, then synthesized to improve their biological properties, and lastly the processes to scale up the manufacture of the drug formulation are developed. At the end of this phase the New Molecular Entity declaration means that the regulatory agencies like the FDA have approved the testing of the drug in humans. Slide 6: · Identify and validate a biological target that has the potential to influence disease processes. · Identify “drug-like” molecules that treat the disease without adverse effects and · In small molecule research, the focus is on the discovery of small molecular drug candidates or NME’s with the potential to satisfy unmet medical needs. Small molecular drug candidates are usually synthesized in the laboratory by chemists. Target Identification The small molecule research process begins with Target Identification. The goal is to identify the biological “target” that drives the disease state. During this phase, biological targets are studied in the laboratory using a series of tests called assays: ADD HIGH THROUGHPUT SCREENING HERE Hit to Lead The objective of Hit to Lead is to identify and verify the desired activity in the molecular entity, which means the compound exhibits a desired effect on the disease state. Keep in mind that a hit demonstrates some activity and a lead demonstrates significant activity. Lead Optimization The purpose of Lead Optimization is to find a single compound that has significant activity with suitable “drug-like” properties and produces a desired effect on the disease state, utilizing both in-vitro and in-vivo testing in animals. Now the work becomes more sophisticated and more people become involved, such as: o Medicinal Chemists, who design and synthesize molecules in the laboratory. Using assay results as a guide, they make modifications to the molecules to optimize desired effect on the target and minimize toxicity Chemists and biologists work together with a common goal, which is to identify and optimize a lead and to validate that the lead can influence a disease state that will eventually be introduced to humans and, ultimately, have commercial value in the marketplace. At-Risk Scale-Up The purpose of at-risk scale-up is to evaluate if the molecule can be made into a form that can be ingested and absorbed by the body, and then safety testing can be done to determine if the compound can be considered an NME to develop further. Larger quantities of the lead substance are required in order to perform large-scale tests. To accomplish this, we need to develop systems and procedures to produce large quantities of the lead substance. We must perform experiments to ensure that results produced at the lab bench are reproducible in large-scale production. This process is considered occurring “at-risk” because significant investment must occur to support scale-up and longer-term toxicity studies before the viability of a compound is determined. NME Declaration The purpose of NME Declaration is to determine if the compound meets its preestablished criteria to transition from Research to initiate testing in humans. NEED TO POINT OUT THROUGHOUT THE PRESENTATION WHERE THE INFORMATION FROM THIS WEBSITE FITS INTO THE DRUG DISCOVERY PROCESS