Workshop: Measurement Assurance Strategies for Cell Therapy Products

Purpose:

• Would you like to improve the reproducibility of your potency assay?

• Would you like to have more confidence in your measurements of critical quality attributes?

• Do you want work as a team with your colleagues to improve confidence in the assays that are critical to getting your cell therapy products to market?

As the saying goes, "If you can't measure something, you can't improve it." So please join us as we examine approaches for improving confidence in measurements critical for cell therapy products! 

Goal: Examine approaches for improving confidence in the measurements that are necessary for bringing cell therapy products to market.

Scope: Challenges in achieving reproducibility, accuracy and comparability in biological assays have been identified as major roadblocks to development, manufacturing and regulatory approval of cell therapy products (CTPs). Improving confidence in measurements for CTPs involves understanding and controlling sources of variability, development of appropriate reference materials to enable traceability and providing objective evidence to validate results. The workshop will focus on strategies to achieve measurement assurance for assays that may contribute to characterizing critical quality attributes and/or potency, and will include breakout sessions focused on specific measurements. 

Content: The first morning will cover strategies for achieving confidence in measurements, such as traceability, reference materials, validation, uncertainty analysis & inter-laboratory testing. In the afternoon, we will breakout into sessions focused on specific measurements, such as cell counting, viability and functional tests. On the second morning, report-outs will lead discussion on the key steps that were identified for improving the confidence in the cell measurements that were examined.

AGENDA (pdf of agenda, participant overview, choice of breakout sessions)

Monday, May 11, 2015, 9:00am – 5:30pm

7:30: Coffee & pastries; NIST cafeteria also has a full breakfast bar for purchase

9:00 – 9:10: Anne Plant (Chief, Biosystems & Biomaterials Division), Opening remarks

• Slides

9:10 – 9:15: Laurie Locascio (Director, Material Measurement Lab), Welcome to NIST

9:15 – 9:45: Marc Salit (NIST), "Systematic Elements of Metrology, & How They Work"

• Slides

9:45 – 10:15: John Elliott (NIST), "Protocol Design & Inter-Laboratory Testing"

• Slides

10:15 – 10:45: Chris Wiwi (Celgene), "Analytical Considerations for Cell Therapy Manufacturing"

10:45 – 11:05: Coffee Break

11:05 – 11:35: Mahendra Rao (Q-Therapeutics), "Developing & Using Reference Materials for Stem Cells"

• Slides

11:35 – 12:05: Ivan Rich (Hemogenix), "Validating a Cell Viability Measurement"

• Slides

12:05 – 12:30: John Elliott, Charge for breakout sessions: Discuss how to improve confidence in the assigned measurement. Generate a draft summary of challenges and opportunities that may include: sources of variability, cause & effect diagram, flow chart of a robust assay protocol, sensitivity testing, needs for reference materials, positive & negative controls, inter-laboratory testing.

• Slides
• Breakout Templates   (pptx)  (pdf)

12:30 – 1:30: Lunch (for purchase at NIST cafeteria)

1:30 – 1:45: Continue charge for breakout sessions (Green Auditorium)

1:45 – 4:30: Parallel Breakout Sessions:

• Breakout #1: Cell Counting (Lecture Room E); Moderators: Janet Davis (Janssen) & Sumona Sarkar (NIST)
  • Slides
• Breakout #2: Cell Viability (Lecture Room D); Moderators: Ivan Rich (Hemogenix) & John Elliott (NIST)
  • Slides
• Breakout #3: Functional Cell Assay - In Vitro Angiogenesis (Lecture Room A); Moderators: Claudia Zylberberg (Akron Biotech), Eytan Abraham (Lonza), Juliana Woda (Juventas), Sagi Nahum (Pluristem), Carl Simon (NIST), Michael Halter (NIST), Yael Porat (BioGenCell), Sandro Matosevic (Akron Biotech)

4:30 – 5:00: Reconvene: Preliminary reports from breakout sections

5:00: Break for the day

5:30: Shuttle back to Holiday Inn (for those not going to Brasserie Beck)

Tuesday, May 12, 9:00am – 12:15pm

7:30: Coffee & pastries;NIST cafeteria also has a full breakfast bar for purchase

9:00 – 9:10: Sheng Lin-Gibson (Deputy Chief, Biosystems & Biomaterials Division), Welcome back

• Slides

9:10 – 10:40: Full reports from breakouts showing cause and effects diagrams, flow charts, needs for reference materials, potential for inter-laboratory studies, etc.

9:10 – 9:40: Breakout#1 Report: Cell Counting

• Report

9:40 – 10:10: Breakout #2 Report: Cell Viability

• Report

10:10 – 10:40: Breakout #3 Report: Functional Cell Assay

• Report

10:40 – 11:00: Coffee Break

11:00 – 12:00: Panel Discussion; Moderator: Carl Simon

Michael Mendocino (Mesoblast), Pam Robey (NIH), Chris Wiwi (CelGene), Steven Oh (FDA), Mahendra Rao (Q-Therapeutics), John Elliott (NIST), Jessica Carmen (ARM), Ivan Rich (Hemogenix)12:00 –

12:15: Concluding remarks

Workshop Output: 

• Workshop Summary
• Workshop Report: Simon Jr CG, Lin-Gibson S, Elliott JT, Sarkar S, Plant AL (2016) Strategies for achieving measurement assurance for cell therapy products. Stem Cells Translational Medicine 5, 705-708.   https://doi.org/10.5966/sctm.2015-0269

Contributors: Carl Simon, Sheng Lin-Gibson, Anne Plant, Sumona Sarkar, John Elliott