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Dr Susan Winks

What does it take to advance a new drug from the laboratory bench to the patient’s bedside? Historically, drugs were discovered serendipitously and were usually isolated from natural sources. Today, drug discovery efforts take many years and cost hundreds of millions of dollars. They also require the input of dozens of specialists from a multitude of backgrounds to ensure that therapeutic molecules are efficacious and safe.

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What does it take to advance a new drug from the laboratory bench to the patient’s bedside? Historically, drugs were discovered serendipitously and were usually isolated from natural sources. Today, drug discovery efforts take many years and cost hundreds of millions of dollars. They also require the input of dozens of specialists from a multitude of backgrounds to ensure that therapeutic molecules are efficacious and safe.

This four-week course takes a high-level tour of what it takes to research and develop a new drug. We will meet different scientists and professionals across the drug discovery value chain as we explore the different stages of discovery and development, including the different tools and technologies that are influencing how we think about new medicines today.

Starting off with the needs of the patient and the early phases of drug discovery, we will cover drug metabolism, pharmacokinetics and drug safety all the way to clinical development and beyond. This course is relevant for scientists who are new to drug discovery research or already have some experience, health professionals, or anyone who is interested in knowing what had to happen to ensure that their medicine is effective and safe.

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What's inside

Syllabus

The starting point for drug discovery: The needs of the patient
This week covers an introduction to the drug discovery and development process. It starts off by considering the patient, as the ultimate beneficiary of new and improved drugs, to ensure that they are kept at the centre of and integrated into all subsequent decision-making. Through a case study, the concept of an unmet medical need is introduced and provides a backdrop for understanding further critical concepts which underlie drug discovery and development. An overview of various drug discovery strategies is presented, along with approaches to screening and hit discovery. Various scientific, medical and humanitarian considerations need to be taken into account if effective delivery of new and improved drugs is to be achieved. Some of the challenges are presented, and a panel discussion highlights different, and similar, perspectives in the academic and industry approach to drug discovery projects.
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The early phases of drug discovery
This week explores the early phases of drug discovery. As outlined previously, the starting point is always the needs of the patient and their biomedical realities. Understanding disease biology is a critical next step and sets the scene for mapping the context for immune response and the origins of ill health and how the progression of disease can be controlled or arrested. Case studies, particularly in the context of tuberculosis (TB), demonstrate the rationale behind molecular biology studies of the pathogen and the host. Key concepts such as the essentiality and vulnerability of targets and their 'druggability' are introduced. Medicinal chemistry drives the chemical modification of drug-like molecules to improve their activity and safety. The various tools and approaches that the medicinal chemist has at their disposal, including the value of the screening cascade, will be discussed.
Drug metabolism, pharmacokinetics and drug safety
This week picks up the drug discovery and development story at the point at which promising drug candidates have already been identified. The crucial importance in this phase of investigating the pharmacokinetics and pharmacodynamics of potential drugs is introduced, as these are key parameters for the effectiveness of a new drug and a positive patient experience. These properties have a bearing on bioavailability and metabolic processes and provide a backdrop for animal model studies, and the extent to which the biological properties of the drug are retained in mammalian systems. The ethical considerations and technological advances pertaining to in vivo testing are discussed. Drug testing, safety profiling, and toxicology and efficacy in preparation for clinical trials are presented, and an introduction to process optimization and API manufacturing is also discussed.
Development phase
This week outlines and examines the clinical development phase. Once a drug candidate has successfully progressed through the phases outlined previously, it qualifies for filing as an Investigational New Drug (IND) and a Clinical Trial Application (CTA) and the accompanying review process. An overview is given of the regulatory framework governing this, and a description of the various phases of clinical trials. The extensive teamwork that is required during the clinical trial is outlined, as well as the various role-players in this process, and some of the critical aspects, such as the submission and approval process, protocols that need to be established, informed consent and site management, are described. Finally, we look to the future and consider how new technologies are changing the face of drug discovery and development and how this may ultimately bring newer and safer medicines to the patient more rapidly.

Good to know

Know what's good
, what to watch for
, and possible dealbreakers
Develops knowledge and tools for professionals across the drug discovery value chain
Provides a comprehensive overview of drug discovery and development
Taught by Dr Susan Winks, a recognized expert in drug discovery
May be too high-level for those with extensive experience in drug discovery
Requires some knowledge of biology and chemistry
Does not cover advanced topics in drug discovery

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Activities

Be better prepared before your course. Deepen your understanding during and after it. Supplement your coursework and achieve mastery of the topics covered in Introduction to Small Molecule Drug Discovery & Development with these activities:
Find a Mentor in the Drug Discovery Industry
Seek guidance and support from experienced professionals in the drug discovery industry to enhance your learning and career prospects.
Browse courses on Mentorship
Show steps
  • Identify potential mentors through networking events, conferences, or online platforms
  • Reach out to potential mentors and express your interest
  • Establish a mutually beneficial mentoring relationship
  • Regularly meet with your mentor for guidance and advice
Become a Mentor for Aspiring Drug Discoverers
Share your knowledge and experience by guiding and supporting others who are interested in pursuing careers in drug discovery.
Browse courses on Mentorship
Show steps
  • Identify platforms or organizations where you can offer mentorship
  • Create a profile or reach out to potential mentees
  • Provide guidance, support, and advice on career paths and research projects
  • Stay connected and follow the progress of your mentees
Read 'Pharmaceutical Biotechnology' by Walsh
Gain a deeper understanding of the principles and applications of pharmaceutical biotechnology, which is essential for drug discovery and development.
Show steps
  • Read the assigned chapters
  • Take notes on key concepts and ideas
  • Summarize the main points of each chapter
  • Discuss the content with classmates or colleagues
Five other activities
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Show all eight activities
Practice Drug Metabolism Problems
Practice solving drug metabolism problems to reinforce your understanding of the principles and concepts covered in the course.
Browse courses on Drug Metabolism
Show steps
  • Review the principles of drug metabolism
  • Identify the different enzymes involved in drug metabolism
  • Solve practice problems involving drug metabolism
  • Analyze the results of your calculations and draw conclusions
Contribute to an Open-Source Drug Discovery Platform
Gain practical experience and contribute to the advancement of drug discovery by participating in open-source projects.
Browse courses on Open Source
Show steps
  • Identify open-source drug discovery platforms
  • Choose a project that aligns with your skills and interests
  • Contribute to the project by writing code, testing, or providing documentation
  • Collaborate with other contributors and maintain the project
Participate in a Drug Discovery Hackathon
Challenge yourself and collaborate with others to develop innovative solutions for drug discovery-related problems through a hackathon.
Browse courses on Drug Discovery
Show steps
  • Find a relevant hackathon
  • Form a team or join one
  • Brainstorm ideas and develop a solution
  • Build a prototype or create a presentation
  • Present your solution to the judges and get feedback
Attend a Drug Discovery Workshop
Network with experts and gain practical insights into the drug discovery process through attending a dedicated workshop.
Browse courses on Drug Discovery
Show steps
  • Research and identify relevant workshops
  • Register for the workshop
  • Attend the workshop and actively participate
  • Take notes and ask questions
  • Follow up with speakers or attendees after the workshop
Develop a Drug Discovery Proposal
Apply your knowledge and skills to create a comprehensive proposal outlining a research plan for a new drug discovery project.
Browse courses on Drug Discovery
Show steps
  • Define the unmet medical need and target disease
  • Research potential drug targets and mechanisms of action
  • Design and outline a research plan
  • Write the proposal, including an introduction, background, methods, expected outcomes, and budget
  • Present the proposal to peers or mentors for feedback

Career center

Learners who complete Introduction to Small Molecule Drug Discovery & Development will develop knowledge and skills that may be useful to these careers:
Biomedical Scientist
This course can help you build a foundation in the drug discovery and development process, with a focus on the needs of the patient. This knowledge is essential for Biomedical Scientists, who are involved in all aspects of drug development, from research and development to clinical trials and marketing.
Regulatory Affairs Specialist
This course provides an overview of the regulatory framework governing drug discovery and development. This knowledge is essential for Regulatory Affairs Specialists, who are responsible for ensuring that new drugs are developed and marketed in compliance with all applicable regulations.
Pharmacokineticist
This course provides an overview of pharmacokinetics and its importance in drug discovery and development. This knowledge is essential for Pharmacokineticists, who are responsible for studying the absorption, distribution, metabolism, and excretion of drugs in the body.
Medicinal Chemist
This course introduces the concepts of medicinal chemistry and discusses the tools and approaches used to modify drug-like molecules to improve their activity and safety. This knowledge is essential for Medicinal Chemists, who are responsible for designing and synthesizing new drugs.
Clinical Research Associate
This course provides an overview of the clinical development phase of drug discovery and development. This knowledge is essential for Clinical Research Associates, who are responsible for managing clinical trials and ensuring that they are conducted safely and ethically.
Pharmacologist
This course provides an overview of pharmacology and its relevance to drug discovery and development. This knowledge is essential for Pharmacologists, who are responsible for studying the effects of drugs on the body.
Toxicologist
This course provides an overview of toxicology and its importance in drug discovery and development. This knowledge is essential for Toxicologists, who are responsible for evaluating the safety of new drugs and ensuring that they are safe for patients.
Drug Safety Scientist
This course provides an overview of drug metabolism, pharmacokinetics, and drug safety. This knowledge is essential for Drug Safety Scientists, who are responsible for evaluating the safety of new drugs and ensuring that they are safe for patients.
Health Economist
This course provides an overview of the economic aspects of drug discovery and development. This knowledge is essential for Health Economists, who are responsible for evaluating the cost-effectiveness of new drugs and ensuring that they are affordable for patients.
Drug Discovery Scientist
This course provides an overview of the drug discovery process, from target identification to clinical trials. This knowledge is essential for Drug Discovery Scientists, who are responsible for identifying and developing new drug candidates.
Science Writer
This course may be of interest to Science Writers, who are responsible for writing and editing scientific content related to drug discovery and development.
Statistician
This course may be of interest to Statisticians, who are responsible for analyzing data related to drug discovery and development.
Medical Writer
This course may be of interest to Medical Writers, who are responsible for writing and editing scientific documents related to drug discovery and development.
Patent Attorney
This course may be of interest to Patent Attorneys, who are responsible for protecting intellectual property rights related to drug discovery and development.
Project Manager
This course may be of interest to Project Managers, who are responsible for managing drug discovery and development projects.

Reading list

We've selected six books that we think will supplement your learning. Use these to develop background knowledge, enrich your coursework, and gain a deeper understanding of the topics covered in Introduction to Small Molecule Drug Discovery & Development.
Classic textbook of pharmacology and would be a valuable resource for students who want to learn more about the pharmacology of drugs.
Provides a comprehensive overview of the field of pharmaceutical chemistry. It covers the different aspects of drug discovery, from target identification to clinical trials.
Provides a comprehensive overview of the field of medicinal chemistry. It covers the different aspects of drug discovery, from target identification to clinical trials.
Covers the basic principles of medicinal chemistry and would be a good starting point for students who are new to the field. It provides a clear and concise overview of the different aspects of medicinal chemistry.
Covers the basic principles of pharmacology and would be a good starting point for students who are new to the field. It provides a clear and concise overview of the different aspects of pharmacology.

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