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Stem Cells Technology, business, & ethics. By Steve Mushero www.SteveMushero.com Swedish-American Chamber of Commerce & Silicon Vikings. Overview. A little about Stem Cells What are they good for ? Latest news, cool research Swedish contribution Ethics and the big issues
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Stem Cells Technology, business, & ethics By Steve Mushero www.SteveMushero.com Swedish-American Chamber of Commerce & Silicon Vikings
Overview • A little about Stem Cells • What are they good for ? • Latest news, cool research • Swedish contribution • Ethics and the big issues • How can we make money ? • Future Stem Cells - Swedish-American Chamber of Commerce
What are stem cells ? • Stem = Root = Source • Stem cells are the source of all cells • Some stem cells can become any cell type • Eventually differentiate to 200 cell types • Very complex, poorly understood process • Several stages of differentiation • Stem, blood, red blood, . . . • Reversible ? Yes, no, maybe, perhaps, don’t know Stem Cells - Swedish-American Chamber of Commerce
Where are the Stem Cells ? • Two choices – Embryo & Adult • Embryo • Start with one single cell - the fertilized egg !! • Most flexible - can become any cell type • Two choices of embryo source • Fertilized in the usual way (Man & Woman) • Cloned (use your own cells) • Adult • Present in normal tissues, including brain • Have significant and poorly understood limitations Stem Cells - Swedish-American Chamber of Commerce
Getting Embryonic Stem Cells • Need an embryo • Old fashioned way - With egg & sperm • In Vitro Fertilization • Most common • Excess embryos (usually dozens created) • Never implanted, so not aborted • Fetal (Abortion) – Used for some R&D • Therapeutic Cloning • More exciting – allows use of own cells • Needed to eliminate rejection • Not reproductive cloning • Move in Congress to ban therapeutic cloning Stem Cells - Swedish-American Chamber of Commerce
Getting Embryonic Stem Cells • Egg divides - first 2 cells, then 4, then 8 . . . • Becomes “Blastocyst” • Hollow ball with key cells inside • Harvest at 3-5 days – 30 cells in inner mass • Embryo is destroyed in process, key ethical issue • Two crucial embryonic cell characteristics • Not yet differentiated (pluripotent) • Can divide forever (key to culturing) Stem Cells - Swedish-American Chamber of Commerce
Adult Stem Cells • Extracted in VERY small amounts from normal tissues • Key to avoiding rejection issues – your own cells • Traditional rejection • Graft vs. Host Disease – The Stems Attack ! • Key to avoiding embryonic ethical issues • Always partially differentiated (for now) • BUT, research points to plasticity • Liver stem cells to brain, muscle, and liver • Bone marrow to muscle • Brain stem cells to blood and muscle • Not found in many key organs (Pancreas) • Difficult to grow in culture; can’t mass produce • Holy Grail - Is there a real master adult stem cell ? Stem Cells - Swedish-American Chamber of Commerce
Using Stem Cells • Reverse damage • Central Nervous System (CNS) damage • Parkinson’s Disease • Multiple Sclerosis • Heart Damage, Diabetes, Bone/Joint loss • Cancer - Bone marrow transplant avoidance • Fix genetic problems !! • Severe Combined Immune Deficiency (SCID) • Chronic Granulomatous Disease (CGD) • Make good cells as needed ! • Make whole organs as needed ! • Kidney, Liver, Pancreas, maybe a Thumb Stem Cells - Swedish-American Chamber of Commerce
Recent Research • Three ongoing key focal points • Understand basic processes, especially differentiation • Differentiation triggers • Expand uses (usually embryonic & cloning) • Brain – Lots of progress, especially in dopamine, neurons • Blood – Lots of immune system work • Bones / Joints • Pancreas (Diabetes) • Liver – Cells come from marrow ? • Weekly announcements – eyes, veins, heart muscle, skin • Improve Adult Cells - Transdifferentiating / Plasticity • Found a receptor/protein that limits plasticity (GCNF) • Stanford found in Sept they cannot make other cells from adult blood stem cells • How do adult cells work – fusion, division; Cancer risk ?? Stem Cells - Swedish-American Chamber of Commerce
Treatment Progress • Modest to date – Very early in clinical process • Parkinson’s injections • Mixed, but promising results • Last week - Got neural stem cells to make dopamine • Multiple Sclerosis • Treat by killing own rogue stem cells • Israel in June • 22 month old infant with immune system failure (SCID) • Cured via repaired bone marry stem cells • Mouse research this year • Extract and fix cell DNA • Clone cells to get embryonic stem cells • Inject new stem cells to fix immune system Stem Cells - Swedish-American Chamber of Commerce
Fixing an Immune System • Baby with damaged immune system • Remove bone marrow stem cells • Fix genetic problem in DNA • Culture cells to get useful volume • Inject new immune cells into baby • Normal immune system results !! • Works when you have stem cells • Mouse Immune Research - Cloned embryonic cells • Remove ANY cell • Fix DNA problem • Clone cells to produce embryo/blastocyst • Harvest stem cells • Coax to differentiate into immune cells • Inject new cells • Crucial when body has no usable stem cells Stem Cells - Swedish-American Chamber of Commerce
Swedish Stem Cell Research • Leading stem cell research center • Over 30 teams, 300 people • 38% of NIH approved cell lines (25 lines) • Main centers - Karolinska, Sahigrenska, Lund • Work in all areas – embryonic, adult, cloning • Legally favorable • Therapeutic cloning allowed • No embryonic issues • Many research firsts • Adult neural stem cell discovery • First human embryonic stem cell cultures • Number 1 or 2 per capita in patents, startups, biotechs, R&D spending, researchers, funding Stem Cells - Swedish-American Chamber of Commerce
Swedish Companies • Cell Therapeutics – Therapies and broad stem cell applications • Neuronova – Adult neural stem cells for Parkinson’s • NsGene – Neural stem cell therapies • Vitrolife – IVR and fertility system supplier Stem Cells - Swedish-American Chamber of Commerce
Ethics – What’s Wrong ? • Most issues surround Embryonic Stem Cells • Embryo cannot survive cell extraction • When does life begin ? What is an Embryo ? • Does it matter if it’s cloned (no sperm) ? • Does it matter if not implanted (not aborted) ? • Is it okay if IVF embryos discarded anyway ? • Abortions also a source and worry • Will we harvest humans ? • Already having kids for marrow matching • But, must abort for stem cells (cord blood ?) Stem Cells - Swedish-American Chamber of Commerce
President Bush Chooses • Balance scientific needs versus ethical & religious issues • Federally funded scientists may work only with existing lines • Cannot create any new stem cell lines (kill no embryos) • Bottom Line - 78 cell lines are now in registry (27 in USA) • 14 approved sources in 6 countries - UCSF has 2 lines • 25 lines in Sweden, mostly in early stages (3+ ready) • University of Gotenborg had 50 other Blastocysts, but rejected because embryo not yet destroyed • 90% of mouse work over 20 years with only 5 lines • Controversial • Sufficient diversity • Quality and purity issues • Licensing, access issues • Federal funding issue only • Allows considerable private research and new lines • Co-mingling decision – Allows mixed use in same lab – Big deal Stem Cells - Swedish-American Chamber of Commerce
Business / Future • Significant funding for basic research • Very International – Sweden, US, India, Israel, Korea • Massive promise in many of the most serious diseases • Opportunities all along the development tree, in many specialties • Suppliers, tools, equipment, reagents • Stem cell sources (extraction from fat, etc.) • Registries for matching (bone marrow) • Processes for differentiating • Actual therapies using cells (like drugs) • Actual therapies targeting cells • Entirely new views of the world Stem Cells - Swedish-American Chamber of Commerce
Future of Stem Cells • Immense promise, though much work remains • Therapies beginning to emerge – SCID, Parkinson’s • Embryonic cells from cloning • No actual embryo, reduced ethical issues • Adult stem cells will work eventually • Eliminate need for embryonic cells & ethical issues • Tremendous progress with applications • Repaired genetic defects, maybe in womb • New therapies for non-genetic diseases (Heart) • New organs • Repairing genetic defects will be common • Change brown hair to blonde ? Ethics, ethics, ethics . . . Stem Cells - Swedish-American Chamber of Commerce
Thanks & Questions Steve Mushero Steve@SteveMushero.com www.SteveMushero.com Swedish – American Chamber of Commerce & Silicon Vikings Stem Cells - Swedish-American Chamber of Commerce