Perelman School of Medicine at the University of Pennsylvania

Case Studies in Translational Research

Perelman School of Medicine at the University of Pennsylvania

Guidelines and Outline

                                     Case Studies in Translational Research (CSTR)
                                                                    Fall 2014

Course description


A student-led, faculty-supported exploration of the role of physician-scientists in bench to bedside translational medicine. The course will examine 5 case studies of translational research using investigators from Penn and Industry as preceptors. The course design, which is entirely new this year, requires active involvement by all of the students on all of the case studies, working in small teams in which students will rotate as team leader. Preceptors will choose the topics, but the team leaders will be responsible for selecting and managing deliverables.


Course goals


Physician-scientists are especially well-suited for guiding the development of new therapeutic and diagnostic modalities from the research setting to the clinic, but may not be trained in all of the skills needed to accomplish this. The challenges attendant with accomplishing this successfully are diverse and in some respects very different from the day to day issues of running a basic science research lab (or a PhD thesis project). The goal of CSTR2.0 is to teach you about these challenges and how to overcome them in the context of translational science.


Among the useful skills that we will focus on in this course are:


1) Setting research goals and meeting milestones along the way to achieving those goals.

2) Selecting outcomes and producing deliverables.

3) Protecting intellectual property and managing conflict of interest. .

4) Performing preclinical and clinical studies.

5) Organizing, leading and working in teams that include people with different skills.

6) Writing coherent research proposals.

7) Understanding  statistical analysis as applied to preclinical studies as well as trials. .


Lead and Support Team organization


There are 21 second year MD/PhD students taking the course this year. There will be a sign-up sheet in Maggie’s office as of Monday August 18. By Monday August 25, please sign up to be on one of the 5 teams. One team will have 5 people; the rest will have 4. Each team will be the lead team on one of the 5 translational cases, and a support team on each of the other 4.


Course organization


The class will meet 11 times in the Fall semester:  one introductory session and 5 double sessions (one double session for each case study).


September 3: Introduction, planning and a lecture on intellectual property.

Sept 24 and Oct 1:        Lead team A

October 8,13:                Lead team B

October 22, 29:             Lead team C

November 5,12:             Lead team D

December 3, 10:            Lead team E


Case study preceptors and general topics


Dan Rader - Drug development for hypercholesterolemia and related disorders; beyond statins


Tal Zaks (Sanofi) and Don Bergstrom (Mersana Therapeutics, Inc) -  development of cancer drugs from an industry perspective


Jean Bennett - Gene therapy for congenital eye diseases


John Maris - Identification of cancer targets plus drug development


Ali Naji - Restoring pancreatic function:  islet cell transplantation


Team roles


Each team acts as the lead team for one case study and as a support teams for each of the other 4 case studies. One way to do this is to have each member of the lead team act as temporary leader for one of the support teams, helping to select the deliverable chosen for that support team. A list of possible deliverables is shown below.


Each double session requires at a minimum a slide presentation by the lead team plus at least one deliverable from each support team. Work by the lead team in advance of Week 1 should include selection of the appropriate primary articles (basic and translational) with the help of the preceptor and the course directors. Week 1 class time can be used for an overview presentation by the lead time, selection of the deliverable and/or team work on the deliverables. Week 2 is used for presentation and discussion of the deliverables.


Preceptor’s roles


Provide the topic and a suggested clinical and basic article. Meet with the Lead Team in advance of Week 1 at least twice. Advise on additional papers and on deliverables. Attend Week 1 of the double session if possible. Attend Week 2 for sure.


Some possible deliverables (check with the course directors if you want to add to this list)


Each support team does one deliverable for each double session topic. Suggested page lengths can be exceeded, but be careful.


1. A patent application (2 pages)

2. An IRB protocol for a proposed clinical trial associated with the case study (3 pages of trial design plus consent form).

3. An IACUC protocol for animal studies (2 pages)

4. An ITMAT-style pilot grant application (2 pages plus budget). ITMAT grants are for collaborative translational research involving faculty in more than 1 department. Information about this pilot program can be found on the ITMAT website.

5. An NIH R21 pilot study (2 pages plus budget). Information about R21’s can be found on the NIH website.

6. A Small Business Administration (SBA) proposal (2 pages plus references)

7. A proposal for industry-sponsored research (2 pages plus references)

8. A business plan for development of a new diagnostic or producing a new therapy.

9. A MATLAB (or other) computational routine for analyzing anticipated data.

10. A review of the relevant literature establishing clinical need (3 pages plus references)

11. Something else (please check with the course directors for approval)




This course is an experiment in education and, therefore, will require regular evaluation and, if needed, modification as we go along. Therefore, the last 15 min of each Week 2 session is reserved for whole class feedback and evaluation. Letter grades will be assigned by the course directors to everyone in the course at the end of the term.



• Last updated: 09/08/2014