Regulatory Science Symposium: “Innovations in Regenerative Medicine Products” - Session 5: The Marriage of Ophthalmology and Bioengineering

Syllabus/Topics

1. Objective: Macular Degeneration Therapy
   1. Leading cause of blindness
   2. 196 million people have macular degeneration, which is expected to be in 288 million by 2040.
   3. Global cost of $300 Billion ($255 Billion to direct health care costs)
2. Description of eye and retina
3. Age-Related Macular Degeneration (AMD)
   1. Affects central vision
   2. Dry-type (Atrophic) - present in 80 to 90% of the population
   3. Wet-type (Exudative - bleeding happens under retina) - present in 10 to 20% of the population
4. Damaged Cones
5. Types of stem cells used
   1. Pluripotent cell: Can give rise to most cell types
      1. Embryonic
      2. Induced Pluripotent
   2. Multipotent: Can give rise to a few cell types
      1. 1. Adult stem cells (fat, bone marrow), 2. Perinatal stem cells (amniotic fluid, umbilical cord fluid)
   3. Caution that stem cells from autologous fat has led to blindness when injected into eye of patients with AMC; risk of tumor formation and rejection
6. Study in Japan - Yamanaka Factors
7. Stem Cell Suspension or Sheet?
   1. Various institutes are researching either suspension or sheet
   2. Stem cell-derived RPE Patch:
      1. Parylene C-Substrate was used because it was highly valuable in making of the patch
   3. Synthetic nonporous biomimetic membrane
8. First in Human Studies
   1. FDA approval for Phase 1/2a clinical trial
      1. Funded through California Institute for Regenerative Medicine (CIRM)
   2. Animation of surgical procedure
   3. Conclusion: Stem cell derived RPE Implant can restore vision in patients with Geographic Atrophy by tropic and direct graft effect
9. Secretome Program:
   1. For people who are not ready for an implant but just starting to have a problem
   2. Process of Secretome Program
10. Summary & Future Direction:
    1. Pluripotent stem cells in early clinical trials to address unmet need of blindness
    2. CPCB Team (Dr. Humayun’s team) conducted largest Phase 1 clinical trial of stem cell implant to data showing improvement in patients with AMD
    3. Future Direction:
       1. Starting Phase 2B trial
       2. Starting pre-IND effort to develop the Secretome
11. Stem Cell Team
12. Video testimonial of a patient with restored vision
13. Questions
    

Acknowledgement
Accompanying text created by Roxy Terteryan, RKS Project Administrator, SC CTSI (atertery@usc.edu)