Pluripotent Stem Cells in Translation: Preclinical Considerations

The National Institutes of Health and the Food and Drug Administration are collaborating on a series of three workshops on moving pluripotent stem cell therapies into the clinic. The first meeting, “Pluripotent Stem Cells in Translation: Early Decisions” took place last year in March. The second meeting, “Pluripotent Stem Cells in Translation: Preclinical Considerations” will be held on the NIH campus at Natcher Auditorium on July 10 and 11, 2012. The focus of this meeting is preclinical animal studies and the issues inherent in performing these studies with cells. As before, the talks will be a mix of science and quite practical matters.

The meeting has participation from industry, academic and clinical scientists, the FDA, and the NIH. The meeting will also be webcast at a link to be announced later.
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* Tuesday, July 10, 2012
*
8:00 AM - 8:30 AM
REGISTRATION

8:30 AM - 9:20 AM
Session 1: Introduction
NIH Welcome and Summary of 1st Workshop

FDA Welcome and Summary of 1st Workshop

The product development timeline: aligning manufacturing, preclinical testing and clinical trials




9:20 AM - 10:30 AM
Session 2: Turning Experimental Ideas into Therapeutic Products
Changing Your Mindset: The Promise and the Therapeutic Challenges

Understanding FDA Expectations for Preclinical Studies (FDA)

10:30 AM - 11:00 AM
BREAK

11:00 AM - 12:30 PM
Session 3: Session 3: Model/Species Choice for Preclinical Testing
General considerations in animal species/model selection

Effects of disease/injury in proof-of-concept (POC) evaluation - the microenvironment and stage of disease

12:30 PM - 1:30 PM
LUNCH ON YOUR OWN

1:30 PM - 3:30 PM
Session 4: Cell Delivery and Tracking
Value of identifying mechanism of action (MOA) from POC studies

Tracking cell distribution and fate

Assessing the effectiveness and safety of the cell delivery route and system

3:30 PM - 4:00 PM
AFTERNOON BREAK

4:00 PM - 4:30 PM
Panel Discussion
What are some of the challenges in conducting preclinical POC studies for pluripotent stem cell-derived products and how can these challenges be addressed?


* Wednesday, July 11, 2012
*
7:45 AM - 8:15 AM
Day 2 Registration if needed

8:15 AM - 8:20 AM
Welcome Back

8:20 AM - 10:15 AM
Session 5: Safety and Preclinical Study Design
Best practices for preclinical study design (FDA)

Effects of disease in pivotal safety evaluation

Achieving cell engraftment/survival in preclinical safety testing

Testing for tumorigenicity and ectopic tissue formation

10:15 AM - 10:45 AM
BREAK

10:45 AM - 12:15 PM
Session 6 : Case Studies: Lessons Learned

Investigator experiences with transitioning from concept to clinical trial

Panel Discussion on Lessons Learned in Bridging to Clinical

12:15 PM - 1:15 PM
LUNCH ON YOUR OWN

1:15 PM - 2:30 PM
Session 7: Combination Products
Regulation of combination products (FDA)

Testing the effectiveness and safety of a cell/scaffold combination product

Driving cell fate with adjunct factors

2:50 PM - 4:20 PM
Session 8: Emerging Areas
The 3R’s/in vitro testing

Humanized mouse models for immunogenicity testing

Large animal models (e.g., transgenic pigs)

4:20 PM - 4:40 PM
Wrap-up and Summary of Meeting

4:40 PM - 4:40 PM
ADJOURN

10 Jul - 11 Jul 2012
Bethesda
United States of America
meeting website