Course Syllabus

 

Translational Medicine: The Power of Biomedical Research

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Draft syllabus

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CC 2.0, John Voo, lungs-viruses-bacteria-disease, Dec 9, 2015

 

Semester & Location:

Fall 2020 - DIS Stockholm

Type & Credits:

Core course - 3 credits

Core Course Study Tours:

Sweden, London

Major Disciplines:

Biology, Biomedicine / Biotechnology, Pre-Medicine / Health Science

Faculty Members:

Natalia Landázuri, Ph.D.

Program Director:

Susana Dietrich

Time & Place:

TBD

Course Description

Translational medicine aims to obtain a new understanding of disease mechanisms and strives to design and develop novel diagnostic and therapeutic tools. It covers the cyclical journey linking unmet patient needs, laboratory research, and clinical trials. The major emphasis of the course lies on the dynamic interplay between the world of in vitro and in vivo laboratory-based preclinical research and the world of clinical research. During the course, you interact with interdisciplinary teams of researchers who collaborate towards translating biomedical research discoveries into medical practice.

During field studies and study tours students will observe the process of translational research as performed by scientists and clinicians at biomedical research institutions and hospitals. In addition, they will learn about the healthcare systems and translational medicine approaches in Sweden and the UK.

 

Course Structure

Module 1

Students learn how researchers design new therapies based on patient data analysis, and how they test their design using in vitro cell culture models and in vivo animal models in a preclinical setting. Students discover how encouraging preclinical results transition to a number of clinical trials and potentially lead to FDA-approval of new therapies. Topics include

  • identification of novel therapeutic targets (for example through handling of patient material and OMICs-e.g. genomics, transcriptomics, proteomics)
  • use of preclinical models to understand human physiology and pathophysiology: in vitro cell culture-based systems and in vivo animal-based models
  • transition from preclinical research to clinical trials and various phases of clinical trials

Students learn about the importance of working in multidisciplinary teams to complete the above-described cycle and the importance of maintaining an open line of communication between the world of preclinical research and the clinic to effectively reach and benefit patients.

Module 2

Students obtain a deeper appreciation of the concepts covered in module 1 through exposure to analysis of real-life scenarios in some of the most innovative fields of translational medicine. Topics include

  • implementation and use of biobanks (collections of tissue samples from patients) to investigate new disease mechanisms
  • development of personalized medicine using stem cell therapy through reprogramming of patient cells, including efforts to activate tissue regeneration through exploiting and enhancing the body’s capacity to repair itself
  • gene therapy strategies to help cure a disease (permit editing the human genome to correct mutations, upregulate of downregulate expression of proteins) and lessons learned from failures and successes
  • major breakthroughs within cell and gene therapy (e.g. in 2017, the FDA approved, for the first time, gene therapies to treat cancers and to cure a type of retinal disease that leads to vision loss)
  • reasons for major drawbacks within the field, especially due to unexpected deaths or the development of adverse side effects during certain clinical trials

Final project

This assignment consists of writing a letter of intent for a funding agency for a research proposal within the field of translational medicine as specified below. Students are expected to articulate their hypotheses, propose sound experimental designs, predict potential outcomes and justify the scientific merit of the research proposed, the latter requiring a substantial literature review.

Students must propose innovative approaches to treat a disease that today has no cure (e.g. spinal cord injury, stroke, diabetes). To accomplish this task, students need to identify an underlying cause of a disease they would like to treat, research the state of the art therapeutic options for that particular disease and propose a cell-based and/or gene-based approach to treat the disease. Students should explain the steps they would take to test a proposed therapy in a preclinical and clinical setting.

 

Expected Learning Outcomes

Upon successful completion of this course, students will be able to:

  • Describe major concepts of human disease and explain abnormal biology underlying certain diseases
  • Distinguish between preclinical and clinical research leading to the development of novel patient-oriented solutions
  • Describe in vitro and in vivo preclinical approaches and explain how these are developed, implemented and utilized for the development of novel patient-oriented solutions
  • Describe different phases of clinical trials leading to the translation of investigational therapies to the clinic
  • Analyze and interpret translational case studies leading to real-life personalized medicine regimens
  • Reflect on the importance of translational medicine and implications to clinical practice and scientific advancement
  • Describe future trends in the field of translational medicine

 

Faculty

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Natalia Landázuri

Ph.D. in Biomedical Engineering, Georgia Institute of Technology, Atlanta, GA, USA (2005). Assistant Professor, Karolinska Institutet, Stockholm, Sweden. Research background in genetic engineering, cardiovascular disease, regenerative medicine, and cancer. With DIS since 2017.

Guest lecturer

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Cecilia Österholm Corbascio

Ph.D. in Experimental Surgery, Lund University, Sweden. Assistant Professor at the Department of Molecular Medicine and Surgery,  Karolinska Institutet and Visiting Research Professor at the Cell Therapy Institute/Center for Collaborative Research, Nova Southeastern University, FL, USA.  Research background in transplantation, vascular matrix biology, regenerative medicine and atherosclerosis. With DIS since 2016.

 

Required Readings

  • Principles of Translational Science in Medicine. From Bench to Bedside, 2nd ed. Martin Wehling (2015) Chapter 1
    • Chapter 2.1.1-3, 2.1.5-6
    • Chapter 3.7.1
    • Chapter 4.1
    • Chapter 5
  • Scientific Publications
    • Viral Vectors for Gene Therapy: Translational and Clinical Outlook, Annu. Rev. Biomed. Eng., 2015.
    • Gene Therapy 2017: Progress and Future Directions, Clin. Transl. Sci., 2017
    • Role of Homeostasis in Human Physiology: A Review, Journal of Medical Physiology & Therapeutics, 2017.
    • Biomanufacturing of Therapeutic Cells: State of the Art, Current Challenges, and Future Perspectives, Annu. Rev. Chem. Biomol. Eng., 2016
    • Cell vehicle targeting strategies, Gene Therapy, 2008
    • Visualization and analysis of gene expression in tissue sections by spatial transcriptomics, Science, 2016
    • Multipotent mesenchymal stromal cells and the innate immune system, Nat. Rev. Immunol, 2012
    • Mesenchymal Stromal Cells: New Directions, Cell Stem Cell, 2012
    • Epidemiology of Heart Failure, Circ Res, 2013

 

 Field Studies

Through field studies, students will obtain a perspective that complements the biomedical theoretical background and research presented in class and during study tours.

Approach to Teaching

Classes may contain a mixture of lecture-based teaching, discussions, critical analysis of readings and research, and group exercises. Students are expected to engage actively in classroom discussions, oral presentations and group work/exercises. In addition, students will participate in short and long study tours, and in field studies.The course does not provide regular medical training corresponding to that of medical students and does not include shadowing of doctors or physical examination of patients.

Study Tours

Study tours are integral parts of the core course. The classroom is “on the road” and theory presented in the classroom is applied in the field. Students will travel with classmates and DIS faculty/staff on one study tours to London.

Students are expected to

  • participate in all activities
  • engage in discussions, ask questions, and contribute to achieving the learning objectives
  • be respectful to the destination/location, the speakers, DIS staff, and fellow classmates
  • represent self, home university and DIS in a positive light

While on a program study tour, DIS will provide hostel/hotel accommodation, transportation to/from the destination(s), approx. 2 meals per day and entrances, guides, and visits relevant to your area of study or the destination. You will receive a more detailed itinerary prior to departure.

Travel policies: You are required to travel with your group to the destination. If you have to deviate from the group travel plans, you need approval from the program director and the study tours office.     

Expectations of the Students

  • Laptops may be used for note‐taking, fact‐checking, or assignment in the classroom, but only when indicated by the instructor. At all other times laptops and electronic devices should be put away during class time.
  • Reading must be done prior to the class session; a considerable part of the course is dependent on class discussions.
  • Students need to be present and participating to receive full credit. The final grade will be affected by unexcused absences and lack of participation. Remember to be in class on time!
  • Classroom etiquette includes being respectful of other opinions, listening to others and entering a dialogue in a constructive manner.
  • Students are expected to ask relevant questions in regards to the material covered.

 

Evaluation and Grading

To be eligible for a passing grade in this class, all of the assigned work must be completed.Late assignments may be accepted, but the grade for the assignment will be reduced by 10 points (over 100) per day.The factors influencing the final grade and the weight of each factor is detailed below: 

Component Weight
Participation 10%
2 tests (15 % each) 30%
Study tour assignment 10%
 Case studies 20%
Final Project 30%

 

Participation: 

  • Class attendance
  • Level of preparation and ability to answer questions asked in class
  • Involvement in class and group discussions
  • Level of individual research and contribution to fruitful discussions

Tests:

  • In class, closed-book tests to evaluate the students' knowledge and understanding of material covered in class.

Study Tour Assignment:

  • Generate questions to be asked during academic visits of the Study Tour
  • Preparation and presentation of group work based on academic visits

Case Studies:

  • Written report and oral presentation of case study analysis

Final Project:

  • Written letter of intent (4 pages) in the form of a research project proposal and oral presentation.

Academic Regulations  

Please make sure to read the Academic Regulations on the DIS website. There you will find regulations on:

DIS - Study Abroad in Scandinavia - www.DISabroad.org 

 

DIS Contacts

Mark Peters, Academic Coordinator, mpe@disstockholm.se 

Natalia Landázuri, Faculty

Susana Dietrich, Science & Health Program Director, sd@dis.dk

 

Course Summary:

Date Details Due