Prospectus

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Cell Pharmacy

Course
2024-2025

Admission requirements

Participants are expected to possess knowledge of stem cell biology, cellular and molecular biology at BSc level. Reading material and a general induction will be given to ensure the basics of stem cell biology are covered.

Description

This Caput series focuses on the upcoming revolution of cell therapy. Advanced therapy medicinal products (ATMPs) is a historical innovation in pharmacology, biotech, and medicine, made of permanently modifying living cells. Principle types of stem cell biology and ATMPs will be introduced, including stem cell based therapy for neurodegeneration, gene therapy for inborne disease, and CRISPR-CAS for muscular dystrophy. Selected topics will be introduced by the course coordinator, and talks will be given by external expert speakers. Students will give seminar presentations in conjunctions to expert talks, and present a poster in mini symposium at courses’ end.

Course objectives

This course aims is to provid knowledge on types of ATMPs, and emerging issues in their clinical applications. An emphasis will be given to biological narrative, conceptual design of proposed therapies, and discussion will revolve around scientific justification, risk, quality, clinical outcomes and cost. Attention is paid to biological mechanisms of production, e.g. reprogramming of iPSCs and cellular differentiation illustrated with specific examples from tissues, e.g. pancreatic islet cell therapies for diabetes.

Final qualifications:
Participants understand primary classes of ATMPs and their modes of clinical applications. They understand mechanisms of stem cell differentiation (gene expression, chromatin, signaling cascades), and key techniques used to manufacture cell and gene therapies. They have state-of–the-art in-depth knowledge about specific examples of cell therapies entering the clinic in cancer and degenerative disease.

Timetable

This course is scheduled in semester 2, period 3 and 4.

You will find the timetables for all courses and degree programmes of Leiden University in the tool MyTimetable (login). Any teaching activities that you have sucessfully registered for in MyStudymap will automatically be displayed in MyTimetable. Any timetables that you add manually, will be saved and automatically displayed the next time you sign in.

MyTimetable allows you to integrate your timetable with your calendar apps such as Outlook, Google Calendar, Apple Calendar and other calendar apps on your smartphone. Any timetable changes will be automatically synced with your calendar. If you wish, you can also receive an email notification of the change. You can turn notifications on in ‘Settings’ (after login).

For more information, watch the video or go the the 'help-page' in MyTimetable. Please note: Joint Degree students Leiden/Delft have to merge their two different timetables into one. This video explains how to do this.

A detailed schedule of the course will be published on the Brightspace course module.

Mode of instruction

Lectures, reading, and discussion of articles and presentations

Assessment method

A seminar given by students on selected topics, participation in discussion, and posters

Reading list

Will be provided.

Registration

Every student has to register for courses with the enrolment tool MyStudymap. There are two registration periods per year: registration for the fall semester opens in July and registration for the spring semester opens in December. Please see this page for more information.

Please note that it is compulsory to register for every exam and retake. Not being registered for a course means that you are not allowed to participate in the final exam of the course. If you want to retake the exam of the course without following the lectures, you have to contact the coordinator of the course to see if this is possible (you would have passed the other assignments of the course in the earlier year).
Registration for your (resit) exam participation is possible until ten days before the exam.

Extensive FAQ's on MyStudymap can be found here.

Contact

Coordinator: Prof. M. Drukker (m.drukker@lacdr.leidenuniv.nl).

Remarks

Minimum number of participants: 12

Topic 1. Micha – Introduction somatic and pluripotent stem cell therapies
Topic 2. Melissa van Pel – Introduction manufacturing cell therapies; students MSC treatment
Topic 3. Peter von dem Borne – Somatic bone marrow stem cell therapy; students HSC purification
Topic 4. Niels Geijsen – Introduction genetic editing in (pre)clinical settings; students crispr therapeutics
Topic 5. Ejone Rusha – induced pluripotent stem cell manufacturing; students HLA stem cell immune matching and immunosuppression
Topic 6. Françoise Carlotti – PSC-Islet differentiation; students PSC-beta cell transplantation outcomes
Topic 7. Sebastian Knöbel - PSC-dopaminergic neuron differentiation; students PSC-dopaminergic neuron trial outcomes;
Topic 8. – CART cells; students clinical trials outcomes
Topic 9. Tineke van den Hoorn – ATMP assessment; students risk assessment in PSC therapy
Topic 10. Patient – 1st hand experience somatic stem cell therapy; students health cost reimbursement strategies for cell therapy