October 19, 2020

The Niche

Knoepfler lab stem cell blog

Wait, there’s an FDA stem cell meeting this week too? The scoop

stem cell crystal ball
Stem cell Crystal Ball

There’s a big FDA stem cell meeting coming along next week that has gotten much attention, but many people didn’t realize there’s also an FDA meeting on stem cells this week too in three days on September 8th.

I pasted this week’s meeting schedule at the bottom of this post from the FDA page. The meeting is a workshop entitled, “Public Workshop: Scientific Evidence in the Development of Human Cells, Tissues, and Cellular and Tissue-Based Products Subject to Premarket Approval”.

The meeting next week on September 12-13 getting all the attention is entitled, “Draft Agenda: Part 15 Hearing: Draft Guidances Relating to the Regulation of Human Cells, Tissues, or Cellular or Tissue-Based Products”.

Within the “Human Cells, Tissues, and Cellular and Tissue-Based Products” or HCT/P larger category the ones getting the most attention are stem cells. If you look at the speakers at this week’s meeting including the keynote by Irv Weissman, it’s pretty clear this meeting is focused squarely on stem cells even though it could apply to other products.

What’s the difference between the meetings?

The first meeting seems to be a broader look at the science related to stem cell products and considerations for their pre-market approval. The talks range from potential promise to serious negative outcomes that have been experienced, ethical and policy considerations, and then some other stuff that to me doesn’t quite seem to fit such as the warfare talk.

By contrast, the second meeting occurring next week is specifically focused on the four FDA draft guidances published last year and provides an opportunity for dozens of stakeholders to speak.

What will happen and be the aftermath of these meetings? One would need a stem cell crystal ball to be sure. Why is the FDA doing these meetings? Together they will demonstrate a reasonable effort on the part of the FDA to seriously tackle the stem cell issue on multiple levels, get various perspectives, and form a foundation for future decisions and potential regulatory actions.  I’m hopeful the meetings will lead to a better oversight framework for stem cells that is more nimble, ultimately safer for patients, and better for the stem cell field overall.

September 8th stem cell meeting at FDA.

8:30-8:40: Welcome/Opening remarks: Celia Witten, PhD, MD
8:40-9:45: Session 1: Keynote and Regulatory Scheme

8:40-9:10: Keynote address: Irving Weissman, MD, Stem Cell Biology and Regenerative Medicine

9:10-9:15: Questions to keynote speaker

9:15-9:45: Steven Bauer, PhD,  FDA Perspectives on Scientific Evidence and HCT/P Development

9:45-11:40: Session 2:  Experiences in Product Development

9:45-10:05: Jacques Galipeau, MD, How Mechanistic Studies on Mesenchymal Stromal Cells Inform Design of Human Clinical Trials for Autoimmune Ailments – The Fitness Paradigm

10:05-10:25: Michael Matthay, MD,  Mesenchymal Stem Cells for Treatment of ARDS Patients: Challenges & Lessons Learned in Pre-Clinical Testing, FDA Approval, and Ongoing Clinical Trial

10:25-10:40: Break

10:40-11:00: Gregory Russotti, PhD, Drivers and Methodologies for Making Cell Therapy Process Changes

11:00-11:20: Dennis Clegg, PhD, Development of ES-derived Retinal Pigmented Epithelium on a Scaffold for Age-related Macular Degeneration

11:20-11:40: Christopher Breuer, MD, The Development and Translation of the Tissue Engineered Vascular Graft: From the Bench to the Bedside and Back Again

11:40-12:20: Panel Discussion 1: Galipeau, Matthay, Russotti, Clegg, Breuer, Weisman
12:20-1:20: Lunch
1:20-2:20: Session 3: Views from Professional Societies

1:20-1:40: Jonathan Kimmelman, PhD, Ethics, Evidence, and Regulatory Approval for Cell-Based Interventions

1:40-2:00: Massimo Dominici, MD, Dissecting Unproven Cellular Therapies: The International Society for Cellular Therapy (ISCT) Position

2:00-2:20: Peter Rubin, MD, Clinical Adipose-Based Therapies

2:20-2:40: Session 4: Views from other Government Agencies

2:20-2:30: Kristy Pottol, Delivering Mission Ready Medical Solutions to the Warfighter

2:30-2:40: Martha Lundberg, PhD, Enabling Development of Regenerative Medicine Technologies and Therapies at the NHLBI

2:40-2:55: Break
2:55-4:15 Session 5:  Patient and Society Experience and Expectations

2:55-3:15: Jeffrey Kahn, PhD, Societal Perspectives on Development and Oversight of Novel Cell-Based Therapies

3:15-3:35: Brian Mansfield, PhD, Perspectives of Stem Cell Therapy for Orphan Inherited Retinal Dystrophies

3:35-3:55: Thomas Albini, MD, Severe Visual Loss After Intravitreal Injection of Autologous Adipose Tissue-derived Stem Cells for Age-related Macular Degeneration

3:55-4:15: Michael Miller, MD, PhD, Glioproliferative Lesion of the Spinal cord Arising from Exogenous Stem Cells

4:15-4:50: Panel Discussion 2: Kahn, Kimmelman, Dominici, Rubin, Pottol, Lundberg, Mansfield, Albini, Miller
4:50-5:00: Closing Remarks: Irving Weissman, MD

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