Presentation on theme: "Stem Cells: Roadmap to the clinic Negar Azarpira, MD Associate professor of Pathology 14 April 2010."— Presentation transcript:
Stem Cells: Roadmap to the clinic Negar Azarpira, MD Associate professor of Pathology 14 April 2010
■■■■ Why it is Important? ■■■■■■■■ Is It As A New Drug?
Stem cell Definition Unspecialized cells in human body that are capable of becoming specialized cells, with new specialized cell function. Very broadly, they divided in two major groups: Embryonal/Fetal SC Adult SC Best example: Hematopoietic stem cells (HSC)
Hematopoietic stem cell Cell surface marker/ Florescent or magnet
Hematopoietic stem cell CD34 + : 0.5-5% of Bone marrow
Hematopoietic Stem Cell Transplantation Outologus or Allogenic HLA matching ■ Source of Hematopoietic cells: Bone marrow (GM-CSF,…) Mobilized peripheral blood Umbilical cord blood
Graft versus Host Disease (liver, Intestine, mucosa) Graft versus Tumor
Clinical Application Treatment of hematological malignancy Inherited disease (SCID), β Thalasemia Mediate tolerance for Solid organ allograft Vehicle for gene Transfer
Future Direction…. ↓ 1. Genetic modification (addition of genes for self renewal, Wnt) 2. Three Dimensional stromal culture 3.Supportive endothelial population or cytokines
Other interesting cells in bone marrow CD133 + : Endothelial precuresure Mesenchymal cell The most common phenotypical markers: CD90, CD73, and CD105
Mesenchymal stem cells - 0.01-0.001% of Bone marrow cells - Has Immunomodulaory properties -Animal study: Skin graft, BMT (GVHD ↓ ) - Human study: Bone marrow graft Crohn’s disease Islet cell transplant
First Stem Cell Drug Prochymal Prochymal is a preparation of mesenchymal stem cells, obtained from the bone marrow of healthy young adults The first SC product approved by the FDA as pharmaceutical product The target disorders of this therapeutic are GVHD and Crohn’s disease For the repair of heart tissue following a heart attack For protection of pancreatic islet cells in patients with type I diabetes For the treatment of Chronic Obstructive Pulmonary Disease (COPD) Osiris Therapeutics, Inc
Reprogramming induced pleuripotent stem cell (iPS) By ectopic expression of transcription factors by retroviral transduction can reprogram somatic nuclei to the pleuripotent state.