Demystifying Stem Cells. Brent Bost M.D., CPA, MBA, FACOG

Transcription

1 Demystifying Stem Cells Brent Bost M.D., CPA, MBA, FACOG

2 Disclaimers Author currently serves on Board of Directors of CordTrack Most applications discussed today are not currently FDA approved and are, by definition, considered experimental Must be paid for by the patient Some are becoming common practice

3 Hematopoietic Stem Cells (HSCs) Sources Cord blood Blood filtering National Bone Marrow Registries Autologous BMTs Immunosuppresant therapy not required Recent advances in expansion

4 Mesenchymal Stem Cells (MSCs) Found in Cord Tissue Adipose Collagenous portion of Bone Marrow Aspirate Expand rapidly 1 cm of cord tissue produces 1 billion stem cells after 30 days incubation Immuno priveleged Are not recognized by the immune system

5 Stem Cell Mechanisms of Action Replace dead cells (If they are a sufficiently close genetic match)

6 Stem Cell Mechanisms of Action Replace dead cells (If they are a sufficiently close genetic match) Modify Micro(local)/Macro (global) environment

7 Stem Cell Mechanisms of Action Replace dead cells (If they are a sufficiently close genetic match) Modify Micro(local)/Macro (global) environment Alter local cellular metabolism Eliminate anaerobic metabolites ( inflamma on) Prevent/slow apoptosis

8 Stem Cell Mechanisms of Action Replace dead cells (If they are a sufficiently close genetic match) Modify Micro(local)/Macro (global) environment Alter local cellular metabolism Eliminate anaerobic metabolites ( inflamma on) Prevent/slow apoptosis Mask immune response (via cytokines) Immunologic induced tissue destruction humoral and cell mediated Nonspecific immune response

9 Stem Cell Mechanisms of Action Replace dead cells (If they are a sufficiently close genetic match) Modify Micro(local)/Macro (global) environment Alter local cellular metabolism Eliminate anaerobic metabolites ( inflamma on) Prevent/slow apoptosis Mask immune response (via cytokines) Immunologic induced tissue destruction humoral and cell mediated Nonspecific immune response Produce bactericidal / static substances

10 Stem Cell Mechanisms of Action Replace dead cells (If they are a sufficiently close genetic match) Modify Micro(local)/Macro (global) environment Alter local cellular metabolism Eliminate anaerobic metabolites ( inflamma on) Prevent/slow apoptosis Mask immune response (via cytokines) Immunologic induced tissue destruction humoral and cell mediated Nonspecific immune response Produce bactericidal / static substances Induce angiogenesis at site of injury

11 Stem Cell Mechanisms of Action Replace dead cells Modify Micro(local)/Macro (global) environment Alter local cellular metabolism Mask immune response (via cytokines) Produce bactericidal / static substances Induce angiogenesis at site of injury Induce pericytes in the host tissue vessels to become the tissue specific stem cells required for tissue repair

12 Stem Cell Mechanisms of Action Replace dead cells Modify Micro(local)/Macro (global) environment Alter local cellular metabolism Mask immune response (via cytokines) Produce bactericidal / static substances Induce angiogenesis at site of injury Induce pericytes in the host tissue vessels to become the tissue specific stem cells required for tissue repair ** engraftment of donor stem cells seldom occurs

13 NMDP $50,000 per sample 2 Samples per BMT Bone marrow aspirate Cord blood/tissue Commercial preparations Pharmaceutical pricing model estimate: $20,000 to $30,000 per sample Adipose liposuction $8,000 to $10,000 per sample Other sources Stem Cell Sources

14 Processing Cord Blood 1. Shipped via FedEx/Courier 2. Excess plasma is removed 3. RBCs removed 4. Cryoprotectant added 5. Controlled rate freeze 6. Nitrogen tanks

15 Single bag storage 80:20 bag Traditional Cord Blood Storage

16 Single bag storage Traditional Cord Blood Storage 80:20 bag same processing requirements in private storage vs. public donation

17 Why Umbilical Cord Tissue? Mesenchymal stem cells

18 Why Umbilical Cord Tissue? Mesenchymal stem cells Main functions in regenerative medicine

19 Why Umbilical Cord Tissue? Mesenchymal stem cells Main functions in regenerative medicine stop inflammation mask immune response bacteriocidal induce angiogenesis

20 Why Umbilical Cord Tissue? Mesenchymal stem cells Main functions in regenerative medicine stop inflammation mask immune response bacteriocidal induce angiogenesis *stem cells induce pericytes in host tissue blood vessels to become the tissue specific stem cells required for tissue repair.

21 Why Umbilical Cord Tissue? Mesenchymal stem cells Main functions in regenerative medicine stop inflammation mask immune response bacteriocidal induce angiogenesis *stem cells induce pericytes in host tissue blood vessels to become the tissue specific stem cells required for tissue repair. (Often no engraftment occurs...)

22 Progression of Stroke Ischemic event triggers Anaerobic metabolites accumulate Inflammation Ischemic cell death Apoptosis of damaged but still viable cells

23 Stem Cell Therapy in Stroke Alter metabolism to clear anaerobic metabolites Reduce tissue destruction of inflammation response Prevent apoptosis Stimulate multiplication and migration of indigenous stem cells to affected area to replace dead cells Promote rapid growth of new vascular beds

24 Target Disorders of Stem Cell Therapy Autoimmune Disorders Type 1 Diabetes Multiple Sclerosis Rheumatoid Arthritis Lupus Cardiovascular Diseases Myocardial Infarction: Valve Repair PAD Peripheral Artery Occlusive Disease CHD Neurologic Disorders ALS Amyotrophic Lateral Sclerosis Cerebral Palsy Stroke Spinal Cord Injury Parkinson s Disease Alzheimer s Disease Orthopedic Conditions Degenerative Disc Disease Cartilage, Tendon and Ligament Repair Bone Repair Pulmonary Disorders (COPD) Lung Injury General and Regenerative Medicine Burns, Wounds, Scars Deafness Missing Teeth Blindness, Macular Degeneration and Vision Impairment Baldness Liver Disease Post Mastectomy Cosmetic Breast Reconstruction

25 How Stem Cells Are Retrieved Patient s Physician suggests stem cells as a possible therapy Parents inform MD that they have their child s cord blood and tissue stem cells stored Physician has their treatment center contact Bank to obtain stem cells Bank sends parents the release forms to authorize release Treatment center makes shipping arrangements Cells shipped still frozen Shipping costs are usually included in the treatment costs

26 Donation National Marrow Donor Program (NMDP) Hospital Claim Waivers eliminated 2013 Procedure Decide around week 28 at glucose testing time Federal screening survey is sent and returned Donation kit shipped to patient Blood collection Exactly like private storage» Kit is given back to the patient» Patient calls the number on the kit Only a donation kit may be used for collection Should NEVER cost the patient a penny (there should be no shipping, kit or processing fees)

27 Thousands of donations are collected before even one is sold Government subsidies help these banks survive No weekend donations Shipping costs skyrocket No twin donations 2 kits would be used but volume is never sufficient Volume requirements Donation

28 The Future of Cord Blood Storage 1:3 people will be able to benefit from Stem Cell Therapies in the future Stem cell expansion technology will become more efficient Cost for SCT will rise initially, then fall over time Whole Cell Therapies will be augmented by 1) Targeted therapies 2) Generic, sub cellular technology

29 Questions