SUPERIOR STEM CELL STORAGE IN AFRICA AND EUROPE

Transcription

1 SUPERIOR STEM CELL STORAGE IN AFRICA AND EUROPE

2 Umbilical Cord Stem Cells are a biological treasure. The importance of their current therapeutic applications and their huge future potential make cryo-preservation of these precious stem cells an option for the future of our children. The Cryo-Save Team. Version 01 June 2014

3 CONTENTS SUPERIOR STEM CELL STORAGE IN AFRICA AND EUROPE Discover who we are and why we care 5 Cryo-Save network 5 Expertise - The leading Private Umbilical Cord Stem Cell Storage Group in Europe and Africa 6 Experience and know-how to ensure your peace of mind 6 Certification and Accreditation 7 Quality & Care 7 Longevity - We will be here for your baby s future 8 Expertise & Technology - Always one step ahead 9 Our medical team at your disposal 10 The most trusted Stem Cell Bank in Europe 11 CORD BLOOD STEM CELLS - SAVE TODAY, PROTECT TOMORROW Becoming a parent is a lifetime responsibility 14 Stem Cells - Life s building blocks 15 Allogeneic Umbilical Cord Blood Stem Cell transplants around the world 16 Storing stem cells: an option for future therapeutic use 16 What the future holds - Expert opinions 17 Reasons to save your baby s Stem Cells 18 It is so simple to safely save 19 Cryo-Save Service Choice 20 UMBILICAL CORD STEM CELLS - A REALITY TODAY, A FUTURE FULL OF POSSIBILITIES Stem Cell - Much more than a Cell 22 Embryonic Stem Cells 22 Adult Stem Cells: Haematopoietic & Mesenchymal 23 Types of Adult Stem Cells 23 Haematopoietic Stem Cell - Decades of therapy 24 Umbilical cord blood actual use in modern medicine 25 Diseases treated 27 Mesenchymal Stem Cells - Umbilical cord tissue 29 Clinical trails 31 Potential uses of Stem Cells 32 Testimonials 33 SUPERIOR STEM CELL STORAGE IN AFRICA AND EUROPE 3

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5 DISCOVER WHO WE ARE AND WHY WE CARE Cryo-Save Europe is accredited as a Licensed Organ & Tissue Establishment for the collection, analysis, processing and preservation of stem cells from Umbilical Cord Blood and Cord Tissue. Cryo-Save is an experienced and technologically advanced Stem Cell Bank in Europe and Africa. Established in 2000, Cryo-Save has been active in the stem cell industry for more than 13 years. Our long-established professionalism and expertise is recognised by the medical community and we have cryopreserved samples from over 40 countries on six continents. We strive to continue improving our facilities and protocols to ensure the best quality and security in cryo-preservation. Our expertise and quality have made it possible that many samples released from our facilities were used to treat different diseases such as Leukemia, Blackfan-Diamond Anemia, Medulloblastoma and Cerebral Palsy. CRYO-SAVE NETWORK Largest in Europe & Africa 40 countries Five stem cell processing labs and cryo-preservation facilities Various Group Accreditations samples stored SUPERIOR STEM CELL STORAGE IN AFRICA AND EUROPE 5

6 EXPERTISE THE LEADING STEM CELL STORAGE GROUP IN EUROPE AND AFRICA Cryo-Save Group is the largest Stem Cell Storage Company in Europe and Africa, one of the Leading Stem Cell Banks of the World. Founding Member of ITERA Life- Sciences Member of CORD BLOOD EUROPE THE CELL FACTORY - Research & Development Involved in the HYPERLAB PROJECT Works in collaboration with GERMAN HEART CENTRE, BERLIN EXPERIENCE THE KNOW-HOW TO ENSURE YOUR PEACE OF MIND More than 13 years of Experience and a team of over 260 professionals looking after your baby s stem cells, let us achieve the highest quality standards in Stem Cell Storage and ensure your peace of mind. Our expertise and quality made it possible that samples released from our facilities were used to treat diseases such as Leukemia, Blackfan-Diamond Anemia, Medulloblastoma and Cerebral Palsy. 6 CRYO-SAVE THE FAMILY STEM CELL BANK

7 ACCREDITATIONS THE ONLY STEM CELL BANK IN EUROPE WITH GMP AND ISO CERTIFICATION AND AABB ACCREDITATION Our Lab in Niel, Belgium is the most advanced Stem Cell Bank in Europe. It was built in 2009 for the purpose of storing one million umbilical cord stem cell samples under the highest quality and security conditions. Our State-of-the-Art technology and the most demanding protocols have allowed us to obtain strict international certification and accreditation. In addition, each of our laboratories around the world adheres to our strict criteria making us a highly credible group of laboratories. QUALITY & CARE New and tailor-made processing kit for Cryo-Save called DeNovo 520A for Sepax2. Sepax2 equipment with unique software options only for us to allow better processing of very small and very large samples. Dual Storage system: Stem Cells are stored independently in two separate storage tanks for additional safety. Cryo-Save launched the Board of Parents in This programme gives parents the opportunity to visit a Cryo-Save lab and learn more about our family Stem Cell Bank. We care about families and children. With our Cost Free Donation Program we offer free of charge storage to families wishing to store their newborn s umbilical cord blood stem cells for a direct family member diagnosed with a life-threatening disease treatable by stem cells. For more information about our labs, we invite you to visit our labs website at com/labs. SUPERIOR STEM CELL STORAGE IN AFRICA AND EUROPE 7

8 LONGEVITY WE WILL BE HERE FOR YOUR BABY S FUTURE The Cryo-Save Group is a strong and solvent company. Our main goal is to protect your baby s future health in the next 20 years, 50 years or even longer. This is why we strive to ensure our financial strength and our solvency. We also follow a full transparency policy. We are a listed company on the Amsterdam and Johannesburg stock markets, therefore all of our information is public, and our Lab is open to visitors. 8 CRYO-SAVE THE FAMILY STEM CELL BANK

9 EXPERTISE & TECHNOLOGY ALWAYS ONE STEP AHEAD Our scientists and lab technicians are constantly improving our Stem Cell Processing and Storage protocols and techniques to improve the quality of the samples we store. Some of the unique advantages our Lab offers are: In collaboration with Biosafe, we exclusively use the DeNovo-520A processing kit that was specifically developed for us. It improves sample processing and allows us to perform a better stem cell separation while processing the samples. The cryoprotectant DMSO Solution, used to protect stem cells during the cryopreservation process, is exclusive to Cryo-Save, with a much lower cytotoxicity, thus making the sample safer for later therapeutic use, as well as minimizing cellular loss. We perform a complete testing process for every sample and maternal blood collected, including a safety serology testing for viruses such as HIV 1, 2; HBV; HCV; CMV and for Syphilis. The risk of contamination is remote and the entire process is fully traceable. In collaboration with the Cell Factory in Belgium, Cryo-Save developed a revolutionary method to assess and confirm cell viability in the tissue of the Umbilical Cord itself. This is the first of its kind in Europe and Africa and ensures that your samples are viable prior to storage. SUPERIOR STEM CELL STORAGE IN AFRICA AND EUROPE 9

10 OUR MEDICAL TEAM AT YOUR DISPOSAL Dr. Albert Ramon Director de ITERA (Bélgica) Dra. Elena Nikiphorou Hospital Univ. Cambridge (Reino Unido) Marcin Jurga Doctor en Ciencias Médicas (Bélgica) Dra. Roxana Macrin Universidad de Bucarest (Rumanía) Dra. Sally Snyman Cryo-Save Group (Bélgica) Dra. Amira Fazlagic Clínica Medison Plus (Serbia) Dr. Dimitar Borrisov Universidad de Sofia (Bulgaria) Dr. Vuk Devrnja Cryo-Save Group (Serbia) Prof. Stefano Grossi Cryo-Save Group (Italia) Prof. Luis Madero Hospital Niño Jesús (Spain) OUR MEDICAL TEAM WORKS WITH SPECIALISTS THAT WILL HELP YOUR FAMILY IF YOUR SAMPLE IS REQUIRED FOR TREATMENT. Dra. Danica Alacka Universidad Skopje (Macedonia) Dr. Renald Blundell Universidad de Malta (Malta) Dr Robert Crookes Cryo-Save Group (South Africa) Dr. Markus Riek Medicina Interna (Suiza) 10 CRYO-SAVE THE FAMILY STEM CELL BANK

11 THE MOST TRUSTED STEM CELL BANK IN EUROPE More than 260,000 mothers and fathers have chosen to rely on Cryo-Save, to trust their baby s stem cells in our expert hands. More than 8,000 doctors collaborate with us in the 40 countries where we are present. For Cryo-Save this Trust is the reward for more than 14 years of a work well done. Kirsten and Robert South Africa Arno and Lizanie South Africa When Kirsty fell pregnant, we started finding out about stem cell storage. We wondered about taking matters into our hands and playing God by storing Makenna s stem cells in order to provide a buffer and insurance in the event that she may fall ill. But then we realised if we could contribute towards insurances which safeguard our material possessions, then we could certainly make the allowance to protect our little girl, storing her stems cells to maximise our chance of fighting a disease should Makennna fall ill, especially with both of our family history of different types of cancers. Rather than just discarding umbilical cord blood, we have decided to store our baby s stem cells for private use. We chose Cryo-Save as they play an active role in making stem cell storage possible by offering the opportunity to provide an additional means of assistance and coverage against possible future disabling illnesses. We really hope that we never need to make a phone call to request our son s stem cells but at least we know that we stand a fighting chance. It s our insurance policy. Miguel and Mara Spain Luca and Bozhidara Italy We believe in stem cell storage, not only because of the actual uses of stem cells, but more importantly because of all the clinical trials that are going on and that will bring new therapies for the future; hopefully for more common diseases like diabetes or cerebral palsy. Stem cell storage is something you have to choose carefully for it lasts 20 years. We have chosen Cryo-Save because of its good reputation among qualified operators in the health sector. Cryo-Save laboratories are located in the heart of Europe and are compliant with the European legislation. SUPERIOR STEM CELL STORAGE IN AFRICA AND EUROPE 11

12 12 CRYO-SAVE THE FAMILY STEM CELL BANK

13 CORD BLOOD STEM CELLS - SAVE TODAY, PROTECT TOMORROW

14 We stored our children Stem Cells, because as parents we want to give as much as we can. BECOMING A PARENT IS A LIFETIME RESPONSIBILITY Having a baby is one of the most emotional and fascinating moments in life. You may start asking yourself many questions about the future of your family. You may wonder how to provide the best for your child and how to ensure his/her health for the years ahead. Some of the recent medical innovations, like Stem Cell storage can offer new possibilities to address future medical medical needs. 14 CRYO-SAVE THE FAMILY STEM CELL BANK

15 STEM CELLS TREMENDOUSLY SMALL & INCREDIBLY POWERFUL SPECIALISATION REGENERATION SELF- OF TISSUES, ORGANS RENEWAL & FUNCTIONALITY STEM CELLS LIFE S BUILDING BLOCKS Stem Cells are precious because they have the ability to develop into different kinds of cells within the human body. This unique process of SPECIALISATION is what builds our complex organism. Some of these Stem Cells remain in our body as a reserve and play a key role in REGENERATION in the case of damaged or aged tissue. These important Stem Cells, can be found in the Umbilical Cord Blood and Umbilical Cord Tissue. They have great and effective ability to produce different human tissues. Furhermore, Stem Cells from the Umbilical Cord Blood have numerous advantages: they are the youngest and most effective Haematopoietic Stem Cells that can be found in the human body and relatively unaffected by diseases and genetic mutations accumulating throughout lifetime. Umbilical Cord Blood Stem Cells are called Haematopoietic Stem Cells (HSCs). Umbilical Cord Tissue Stem Cells are called Mesenchymal Stem Cells (MSCs). WHAT IS... Autologous: For own use Allogeneic: A transplant where the donated stem cells comes from a different individual (possibly related) CORD BLOOD STEM CELLS - SAVE TODAY, PROTECT TOMORROW 15

16 MORE THAN 30,000 CORD BLOOD STEM CELLS TRANSPLANTATIONS HAVE BEEN PERFORMED IN THE WORLD SO FAR Haematopoietic Stem Cells from Umbilical Cord Blood have been used, for allogeneic transplants, for more than 25 years to treat more than 70 different diseases. Umbilical Cord Blood is becoming a preferred source of Hematopoietic Stem Cells because: They are Younger than any other adult Stem Cell, which means they have a better ability to multiply and specialize. The rate of Graft vs Host Disease is lower. This is a complication (also called rejection) after a Stem Cell transplant. Their Collection is a very easy and risk-free process. Mesenchymal Stem Cells (MSCs) found in Umbilical Cord Tissue are responsible for the formation of bone, cartilage and other types of connective tissue Cells in our bodies. MSCs have been involved in regenerative medicine, with the end goal to replace, repair or support regeneration of living functional Cells or tissues. Stem Cells are actively being researched for the treatment of numerous diseases, such as cerebral palsy, type 1 diabetes, brain disorders, heart conditions, auto-immune disorders, liver disorders, neurological diseases and others. STEM CELLS ARE TOO VALUABLE TO BE WASTED Nowadays, an estimated 95% of Cord Blood is discarded after birth. Cryo-Save is convinced that the Cord Blood and Cord Tissue should be saved, either with a family bank or made available for public use or research. Thanks to the thoughtful parents and Cryo-Save s high quality cryopreservation, several Stem Cells samples have been used to treat children with Aplastic Anemia, Congenital immunodeficiency, Celebral Palsy, Acute Lymphoblastic Leukemia, Medulloblastoma and Cerebral Edema. 16 CRYO-SAVE THE FAMILY STEM CELL BANK

17 4,000 TRAILS ON STEM CELLS WHAT THE FUTURE HOLDS EXPERT OPINIONS Over 4,000 trials involving Stem Cells with over 200 of these trials using Umbilical Cord Blood Stem Cells. Clinical trials analyse new medical treatments for their safety, efficiency and potentially adverse effects in humans. Dr. Marcin Jurga R&D Manager at Cryo-Save s flagship lab, Niel, Belgium Apart from current therapies using Haematopoietic Stem Cells, from Umbilical Cord Blood, there is an incresing number of clinical trials using Mesenchymal Stem Cells. These are used in researching a broader scope of applications, such as treatments of: Nervous system diseases, Graft-vs-Host disease, orthopedic injuries and disorders, cardiovascular illnesses, diabetic patients, but also in tissue engineering applications where entire organs can be produced. The future of Stem Cell applications is yet to show its full potential. Dr. Luis Madero Director of Oncohematology, Niño Jesús Hospital, Madrid, Spain There are clinical trials for the use of Cord Blood Cells for non-hematopoietic diseases in children for cases of cerebral palsy and diabetes. The information reported on the cerebral palsy trials has given some positive preliminary results. The experience in diabetes is still limited and more data need to be gathered before any conclusion can be drawn. Prof. Dr. Daniel Surbek Department of Obstetrics and Gynecology, Inselspital Bern, Switzerland Evidence from recent studies suggests that transplantation of Stem Cells from the Umbilical Cord may be used as treatment for severe perinatal brain damage due to preterm delivery or birth asphyxia. Transplanted Stem Cells may regenerate damaged neural tissue and central nervous system function. Several clinical trials in children affected by cerebral palsy are ongoing. Dr. Csaba Papp Associate professor, medical geneticist, Semmelweis University, Buda pest, Hungary Together with the Umbilical Cord Blood, the preservation of the Cord Tissue is not only a possibility for obtaining mesenchymal Stem Cells, but for yielding other Cells that are important for future Stem Cell therapies. CORD BLOOD STEM CELLS - SAVE TODAY, PROTECT TOMORROW 17

18 REASONS TO SAVE YOUR BABY S STEM CELLS Stem Cell storage from a newborn s Umbilical Cord and Cord Blood is increasing among families. Banking your child s Cord Blood Stem Cells can be valuable for the child itself, but could also be a source of transplantable Stem Cells for a family member in medical need. Cryo-Save offers high quality family storage for the following reasons: Providing opportunity for autologous or allogeneic grafts. Autologous: Matching is guaranteed. Siblings could benefit from the same sample: they have a one-in-four chance of being a perfect match. For cord blood, transplants using a partially matched donor/s are often used. In case of related allogeneic use the rate of Graft vs Host Diseases is low. More than 70 blood related diseases and immune system disorders have been treated with allogeneic stem cell transplants. Stem Cells stored in private bank remains the property of the child under the guardianship of the parents. The sample is immediately available. Cryopreservation for at least 20 years. They are Younger 25 years 70 diseases 30,000 transplants Easy to Collect Low Contamination Risk UMBILICAL CORD IS PREFERRED SOURCE OF STEM CELLS BECAUSE: 18 CRYO-SAVE THE FAMILY STEM CELL BANK

19 IT IS SO SIMPLE TO SAFELY SAVE Enroll yourself on Internet You receive your Cryo-Save KIT directly at home Do not forget to bring the Cryo-Save KIT to the hospital! Right after the delivery, call our team! The KIT is sent to a specialized Cryo-Save Laboratory As soon as the KIT reaches the laboratory the processing starts... Your child s stem cells will be preserved in one of our high tech Liquid Nitrogen Cryopreservation Tanks. After successful Cryopreservation you will receive a Certificate of your child s stem cell storage. CORD BLOOD STEM CELLS - SAVE TODAY, PROTECT TOMORROW 19

20 CRYO-SAVE SERVICE CHOICE All service choices are subject to a minimal annual fee for the duration of the Storage. 1. LOCAL CORD BLOOD 2. LOCAL CORD BLOOD & TISSUE 3. INTERNATIONAL CORD BLOOD 4. INTERNATIONAL CORD BLOOD & TISSUE 5. DUAL CORD BLOOD 6. DUAL CORD BLOOD & TISSUE PAYMENT OPTIONS 1. ELECTRONIC FUNDS TRANSFER 2. CREDIT CARD 3. DIRECT DEBIT LONG TERM STORAGE GUARANTEE With 13 years of international experience, Cryo-Save takes our contractual responsibility to our valued clients very seriously. We know that over the next 20 years international regulations and standards will advance significantly. We can only imagine what the stem cell environment will look like in the future but we guarantee that we will remain at the forefront of this technology protecting your valuable assets. For this reason, the world s largest and most successful stem cell storage facilities provide unmatched peace of mind through their annual storage fees. It is through our annual fees that the Cryo-Save Group does not need to rely on business growth to fulfil our contractual responsibility to our clients. You get this priceless peace of mind for only R200 per year (local storage) and only R500 per year (International storage). You pay this amount once a year on the birth date of your child these payments begin on their 1st birthday. 20 CRYO-SAVE THE FAMILY STEM CELL BANK

21 UMBILICAL CORD STEM CELLS - A REALITY TODAY, A FUTURE FULL OF POSSIBILITIES

22 STEM CELL MUCH MORE THAN A CELL Stem Cells are the Building Blocks of our Body. They are capable of becoming other types of Cell and Regenerate our Tissues, Organs and Functionality thanks to their two unique abilities: Self-Renewal. Specialization into other cell types. There are two types: Embryonic and Adult Stem Cells. What is a Stem Cell? A single cell that s capable of... SELF RENEWAL: REPLICATE THEMSELVES Specialisation: differentiate into many cell types EMBRYONIC STEM CELLS Embryonic Stem Cells (ESCs) can only be collected from the Blastocyst, a preimplantation embryo that develops 5 days after fertilization. The Embryo is destroyed when they are collected and thus they are subject to ethical controversy. Use of these Stem Cells is in early stages of investigation and they aren t currently used in therapies at all, especially because they have been proven to potentially cause cancer in the patient. Cryo-Save is not involved in ESC collection or utilization. 22 CRYO-SAVE THE FAMILY STEM CELL BANK

23 ADULT STEM CELLS HEMATOPOIETIC & MESENCHYMAL Adult Stem Cells (ASCs) are Multipotent Stem Cells that can be found after birth, in different parts of the body such as Umbilical Cord Blood, Bone Marrow or Fat Tissue. They have a more limited specialization capability compared to the Pluripotent ESCs, but their collection isn t subject to any ethical concerns, and they have been used in Medical Therapies for over 40 years. Cryo-Save is involved in Umbilical Cord Stem Cell collection and cryopreservation since TYPES OF ADULT STEM CELLS Among Adult Stem Cells there are two types that are at the core of Cell Therapy and will become the future of Regenerative Medicine. Haematopoietic Stem Cells (HSCs): Are responsible for rebuilding our Immune System and the different blood cells. These can be collected from Umbilical Cord Blood. Mesenchymal Stem Cells (MSCs): Are capable of specializing in many different Cell types such as Heart Cells, Cartilage Cells, Neurons, etc. We collect these from Umbilical Cord Tissue. Umbilical Cord Stem Cells are the youngest Adult Stem Cells Umbilical Cord Stem Cells have a high potential to proliferate Umbilical Vein Umbilical Cord Blood Cells Umbilical Artery Wharton s Jelly Umbilical Cord Tissue Stem Cells UMBILICAL CORD STEM CELLS - A REALITY TODAY, A FUTURE FULL OF POSSIBILITIES 23

24 HAEMATOPOIETIC STEM CELL OVER 40 YEARS OF THERAPY Haematopoietic Stem Cells (HSCs) are responsible for regenerating the different blood cells such as Red Blood Cells, White Blood Cells and Platelets as well as for rebuilding our immune system. In the past, the only way to use HSCs in therapy was through bone marrow transplants, which is an uncomfortable and invasive procedure and, in order for a transplant to be successful, the donor and recipient must be genetically similar. If they are too genetically different it could induce Graft vs. Host disease, a common transplant complication, or the transplant could be rejected. Scientists have developed ways to derive HSCs from other sources such as Peripheral Blood. Umbilical Cord Blood is becoming a preferred source of HSCs, especially for treatment of paediatric patients. STEM CELL MYELOID PROGENITOR HEMATOPOIETIC STEM CELL LYMPHOID PROGENITOR BASOPHILS ERYTHROCITES PLATELETS MONOCYTE EOSINOPHILS NEUTROPHILS T LYMPHOCYTES B LYMPHOCYTES NK CELLS 24 CRYO-SAVE THE FAMILY STEM CELL BANK

25 UMBILICAL CORD BLOOD ACTUAL USE IN MODERN MEDICINE The first Umbilical Cord Blood (UCB) Stem Cell transplant was performed in 1988 and, since then, more than 30,000 transplants have been performed worldwide. Around the globe, hundreds of clinical trails are being undertaken to evaluate the use of umbilical cord blood in treating hematologic and non-hematologic diseases. UCB Stem Cells can be used in Autologous (same person s stem cells) or Allogeneic (donor s stem cells) transplants, including Stem Cells from a sibling or other family member. In 1997 a study proved that one-year survival rate is more than twice as high if patient receives a cord blood stem cell transplant from a relative (63%) vs. an unrelated donor (29%). (Gluckman et al, 1997) SURVIVAL RATE: UMBILICAL CORD BLOOD TRANSPLANTS 100% 80% 60% 40% 20% 0% 63% Related Umbilical Cord Blood Transplantation 29% Unrelated Umbilical Cord Blood Transplantation E. Gluckman et al. (1997) N. Engel, J.Med GRAFT vs HOST: UMBILICAL CORD BLOOD TRANSPLANTS 100% 80% 60% 40% 20% 0% 3% 42% Related Unrelated Kutzberg (1996), Wagner (1995), Laghlin (2001) UMBILICAL CORD STEM CELLS - A REALITY TODAY, A FUTURE FULL OF POSSIBILITIES 25

26 UMBILICAL CORD BLOOD ACTUAL USE IN MODERN MEDICINE - continued Year after year Umbilical Cord Blood has become a preferred source of Stem Cells, especially in paediatrics. The use of allogeneic Umbilical Cord Blood Stem Cells has increased especially in paediatric patients since Half of the transplants in 2011 were performed using Umbilical Cord Stem Cells Bone Marrow Peripheral Blood Umbilical Cord Blood Paediatric Transplant (under-18 patients), registered by National Marrow Donor Program in 2012 UMBILICAL CORD BLOOD TRANSPLANTS GROW EVERY YEAR Paediatric Transplant (under-18 patients), registered by National Marrow Donor Program in CRYO-SAVE THE FAMILY STEM CELL BANK

27 DISEASES TREATED Leukemia and lymphomas Cancer of the lymphocytes, a type of blood cell that forms part of the immune system, also called cancer of the blood. Acute lymphoblastic leukaemia Acute myelogenous leukaemia Chronic lymphocytic leukaemia Chronic myelogenous leukaemia Hodgkin lymphoma Non-Hodgkin lymphoma Juvenile myelomonocytic leukaemia Multiple myeloma and other plasma cell disorders Cancer of the plasma cells in the bone marrow. Severe aplastic anaemia and other marrow failure states A group of disorders that result in the failure of normal functioning of cells in the bone marrow. Amegakaryocytosis/congenital thrombocytopenia Fanconi anaemia Paroxysmal nocturnal hemoglobinuria(pnh) Pure red cell aplasia Severe aplastic anaemia SCID and other inherited immune system disorders Diseases in which children are born without an immune system or with an incomplete immune system. Severe combined immunodeficiency (SCID, all sub-types) Wiskott-Aldrich syndrome Hemoglobinopathies Genetic defects that result in abnormal structure of one of the globin chains of the haemoglobin molecule that is responsible for carrying of oxygen in the blood. Beta thalassemia major Sickle cell disease UMBILICAL CORD STEM CELLS - A REALITY TODAY, A FUTURE FULL OF POSSIBILITIES 27

28 DISEASES TREATED - continued 70 DISEASES TREATED WITH ALLOGENEIC STEM CELLS Leukemia is the most commonly treated disease. Transplants registered by Eurocord in paediatric patients, Leukaemia, 47% Hematopoietic disorders, 10% Lymphomas / Solid tumors, 2% Bone Marrow Failure, 11% Hemoglobinopathy, 7% Immunodeficiencies, 11% Metabolic Disorders, 9% Histiocytosis / other, 3% Hurler s Syndrome and other inherited metabolic disorders A group of inherited diseases in which the lack of an enzyme affects various organs and tissues, including the brain. Enzymes are proteins that play many roles, including metabolizing (breaking down) substances in the body. Adrenoleukodystrophy Hurler s syndrome (MPS-IH) Metachromatic leukodystrophy Myelodysplastic and myeloproliferative disorders A group of diseases that affect the bone marrow and blood. It can also develop into a fast-growing, severe leukaemia called acute myelogenous leukaemia. Agnogenic myeloid metaplasia (myelofibrosis) Chronic myelomonocytic leukaemia Refractory anaemia (all types) Familial erythrophagocytic lymphohistiocytosis and other histiocytic disorders Diseases that occur when there is an over-production of white blood cells known as histiocytes that can lead to organ damage and tumor formation. Other malignancies 28 CRYO-SAVE THE FAMILY STEM CELL BANK

29 MESENCHYMAL STEM CELLS UMBILICAL CORD TISSUE The Tissue that surrounds the umbilical vein and arteries, called Wharton s Jelly, is an incredibly rich source of Mesenchymal Stem Cells (MSCs), a different type of Stem Cell that has become the Future of Regenerative Medicine. While HSCs can only specialize into Blood Cells, MSCs are capable of specialising into many different Cell types such as Heart Cells, Cartilage Cells, Neurons and many others. These Stem Cells can be found in different parts of the human body such as fat tissue and bone marrow. Wharton s Jelly is also a rich source of MSCs which is easily collected at birth. Current Use of MSCs: Combined with Haematopoietic Stem Cells they have been shown to reduce the severity of Graft vs. Host Disease, improve engraftment of transplants and achieve a better regeneration of bone marrow. Future Use of MSCs: Given their immense capability to differentiate into many different Cell types and to regenerate different tissues and organs, MSCs are the core of all investigations in Regenerative Medicine, aiming to regenerate tissues that can t self-regenerate such as cartilage, bone or neurons. MSC The Future of Regenerative Medicine Muscle Cells Nerve Cells Bone and Cartilage Cells Epithelial Cells Pancreatic and Liver Cells Heart Cells Lung Cells UMBILICAL CORD STEM CELLS - A REALITY TODAY, A FUTURE FULL OF POSSIBILITIES 29

30 MESENCHYMAL STEM CELLS UMBILICAL CORD TISSUE - continued The Mesengenic Process Mesenchymal Stem Cell (MSC) MSC Proliferation PROLIFERATION OSTEOGENESIS CHONDROGENESIS MYOGENESIS MARROW STROMA TENDOGENESIS ADIPOGENESIS OTHER COMMITMENT Transitory Osteoblast Transitory Chondrocyte Myoblast Transitory Stromal Cell Transitory Fibroblast Preadipocyte LINEAGE PROGRESSION Osteoblast Chondrocyte Myoblast Unique Micro Niche Early Adipocyte DIFFERENTIATION MATURATION Osteocyte Hypertrophic Chondrocyte Myotube Stromal Cell Fibroblast Adipocyte Dermal and other Cells BONE CARTILAGE MUSCLE MARROW TENDON LIGAMENT ADIPOSE TISSUE CONNECTIVE TISSUE 30 CRYO-SAVE THE FAMILY STEM CELL BANK

31 CLINICAL TRAILS This is a summary of Clinical Trials currently taking place with Umbilical Cord Stem Cells aiming to treat nonhaematologic diseases. Number in brackets indicates the number of Clinical Trials currently investigating that disease Type 1 Diabetes (7) Other Non-Malignant/Metabolic Diseases (7) Cerebral Palsy (3) Epidermolysis Bullosa (3) Osteopetrosis (3) Deficient Severe Combined Immunodeficiency and ADA-deficiency (3) Neonatal Hypoxic-ischemic Encephalopathy (2) Hypoplastic Left Heart Syndrome (2) Acquired Hearing Loss (2) Refractory or Relapsed Malignant Solid Tumors (2) Autism (2) Stroke in Children (1) Wiskott-Aldrich syndrome (1) Traumatic Brain Injury (1) Preterm Neonates (1) Type 2 Diabetes (1) Mucopolysaccharidosis (1) Hurler syndrome (1) High Risk Lysosomal and Peroxisomal Disorders (1) Autoimmune diseases (1) Neuroblastoma (1) GATA 2 Mutations (1) Alzheimer s Disease (1) Bronchopulmonary Dysplasia (1) Acute Burn (1) Knee Articular Cartilage Injury (1) Chronic Ischemic Stroke (1) Graft-versus-host-disease (1) Alopecia Areata (1) Cancers (1) Premature Ovarian Failure (1) UMBILICAL CORD STEM CELLS - A REALITY TODAY, A FUTURE FULL OF POSSIBILITIES 31

32 POTENTIAL USES OF STEM CELLS Stroke Traumatic brain injury Learning defects Alzheimer s disease Parkinson s disease Baldness Blindness Deafness Missing teeth Wound healing Bone marrow transplantation (current established) Spinal cord injury Osteoathritis Rheumatoid arthritis Amyotrophoc lateral-sclerosis Myocardial infarction Muscular dystrophy Diabetes Multiple sites: Cancers Crohn s disease 32 CRYO-SAVE THE FAMILY STEM CELL BANK

33 TESTIMONIALS 4-year-old Pedro was treated with his sister s UCB Stem Cells. Patient: Pedro Age: 4 years old Disease: Blackfan-Diamond Anemia Stem Cell Source: Allogenic Date: May 2013 Mother s Testimonial: Up until the transplantation our life was bound to these transfusions. We could hardly be happier, because now our son will be able to lead a completely normal life. Our daughter will be very happy when she learns that her brother got better thanks to her. Doctor s Testimonial: Blackfan-Diamond anemia is a congenital disease that prevents the bone marrow from producing the red blood cells needed to carry oxygen throughout the body. The transplantation of Umbilical Cord Blood Stem Cells, especially between siblings, is now a recommended treatment option for certain types of hematological diseases, such as this anemia. The benefits versus other treatments for this disease are clear. Spanish girl with Brain Cancer treated with her own Umbilical Cord Stem Cells. Patient: Alba Age: 2 years old Disease: Medulloblastoma Stem Cell Source: Autologous Date: March 2009 Father s Testimonial: Our investment in storing Alba s Stem Cells was our best decision. I had no doubt: we were going to store her Umbilical Cord Stem Cells, they might save her life one day. Alba s mother said: Storing Umbilical Cord Stem Cells is saving for your children s future, you don t know if you will ever need them, but they can change your life. Doctor s Testimonial: When we learnt that she had her own Stem Cells stored at birth we decided to use them instead of peripheral blood, which was the usual treatment, we opted for this technique to accelerate the procedure. I like to say that this Autologous transplant helped curing her. Cerebral Palsy treated with Umbilical Cord Blood Stem Cells in a 4-year-old girl. Patient: Claudia Age: 4 years old Disease: Cerebral Palsy Stem Cell Source: Autologous Date: December 2013 Doctor s Testimonial: Although this kind of treatment is still in it s early stage, Regenerative Medicine therapies are believed to be a great option to regenerate nervous tissue and repair brain damage. UMBILICAL CORD STEM CELLS - A REALITY TODAY, A FUTURE FULL OF POSSIBILITIES 33

34 studio112.co.za S-1504 HEAD OFFICE Ground Floor Acacia House Green Hill Village Office Park Cnr Botterklapper & Nentabos Street The Willows, 0041, South Africa S, E T: +27 (0) F: +27 (0) E: info@cryo-save.co.za Local and International Stem Cell Storage