Bin them or bank them?

Transcription

1 Bin them or bank them?

2 What are stem cells? Single cells that can Differentiate into any one of 220 different specialised cells in the body or......replicate themselves Stem cells are often called master cells. They form the foundation of your entire body and act as building blocks for the blood, immune system, tissue and organs. They can replicate or regenerate themselves and have the ability to differentiate into any kind of specialised cell in the body. Types of stem cells and where they come from Tissue stem cells Tissue stem cells allow us to develop, grow, heal and replace worn out cells. Embryonic stem cells: These cells are created from the inner cell of a blastocyst. Muscle Blood cells Inner cell mass Blastocyst 150 cells 200 micrometers Cells are collected and grown on plates in a laboratory. ips cells: (induced pluripotent stem cells) Skin Nervous system Cells from a person are genetically reprogrammed in a laboratory. Most tissues have tissue stem cells. They are important at all stages of life. The modified cells begin to gain the ability to self-renew and differentiate in the same way as embryonic stem cells. 2

3 What are stem cells used for? Stem cell therapies, bioprinting and other technologies are transforming the future of healthcare. Stem cells are used to replace dead or diseased cells e.g. blood stem cell transplant. Stem cell engraftment INFUSION Stem cells are used in clinical trials to regenerate tissues and organs for transplantation e.g. Lungs harvested from cadaver donor Cells are removed Stem cells taken from patient recipient are seeded into donor lungs Seeded lungs are transplanted into patient Stem cells are being used to 3D print tissues and organs. Stem cells are used to induce the regeneration of cells. 3

4 What are blood stem cell transplants? New York, 1956 American doctor E. Donnall Thomas did the first successful bone marrow transplant to cure leukaemia. Blood stem cell transplantation has been performed for more than 50 years, with more than ONE million blood stem cell transplants across the world playing an important role in the treatment of bone marrow failures, blood cancers, blood disorders, metabolic diseases, immune deficiencies and autoimmune diseases. Why a blood stem cell transplant may be needed? To treat and/or cure certain types of blood related disease, for example to: replace diseased bone marrow with healthy bone marrow to treat/cure patients with e.g. blood cancer. replace non-functioning bone marrow with healthy functioning bone marrow in patients with e.g. acquired bone marrow failure. regenerate a healthy immune system in patients with e.g. immune deficiencies and autoimmune disease. replace bone marrow with genetically healthy functioning bone marrow in patients with inherited blood related diseases. Blood stem cells (tissue stem cells) are responsible to maintain the blood and immune system and can differentiate into red blood cells, white blood cells and platelets. Platelets are tiny cells that have the important job to stop bleeding. Red blood cells deliver oxygen to all organs and tissues and also remove carbon dioxide from your body, transporting it to the lungs for you to exhale. White blood cells are the cells of the immune system that are involved in protecting the body against both infectious disease and foreign invaders. Blood stem cells Common myeloid progenitor Common lymphoid progenitor Myeloblast Lymphoblast Red blood cells Platelets Basophil Neutrophil Eosinophil Monocyte T Lymphocyte B Lymphocyte White blood cells 4

5 Blood stem cells used in transplants can be collected from: Bone marrow Circulating blood Umbilical cord blood To date: blood stem cell transplants have been done with more than 1/3 using cord blood stem cells What are your chances of finding a blood stem cell donor in SA? 70% of patients in need of a blood stem cell transplant won t have a sibling donor Depending on a person s tissue type, the chance of finding a bone marrow donor may be less than 1 in a Bone marrow transplants require a perfect tissue match in most cases Only healthy donors are registered on the South African Bone Marrow Registry 5

6 Why should we store life-saving umbilical cord blood stem cells? There is no public umbilical cord blood bank in South Africa 1 in 217 The chances of needing a stem cell transplant in a lifetime of 70 years > cord blood transplants performed in almost 30 years >80 diseases treated >5 million cord blood units stored in private banks 1. Healthier stem cells. Cord blood stem cells are healthy cells that can be stored for stem cell therapy when needed. 2. A newborn s stem cells are young and flexible. They are young and active (a maximum of nine months old), compared to stem cells from other sources. This means that they can differentiate faster, displaying an impressive multiplying potential, and potentially have greater regenerative potential than bone marrow stem cells. 3. Ready to use. Cord blood is collected in advance, tested, cryopreserved and stored ready to use when needed. Unlike bone marrow, there is no need to take time to locate a possible donor and then determine whether he or she is still willing and able to donate if a transplant is required. 4. Increased probability of finding a suitable donor. Studies have shown that cord blood transplants can be performed in cases where the donor and the recipient are only partially matched, which increases the potential of finding a suitable donor. In contrast, bone marrow grafts require a perfect match in most cases. 5. Improved transplant outcomes. Cord blood transplants show significantly lower rates of graft vs host disease, improving outcomes. The immune cells in cord blood are less likely to attack the recipient s own tissues and cause the transplantrelated complication of GVHD. 6. Reduced risk of viral contamination. Cord blood is rarely contaminated with underlying viruses resulting in greater acceptance of cord blood stem cells in comparison to those from bone marrow. 7. New developments and treatment possibilities. Cord blood has been shown to induce regeneration of the central nervous system which may potentially be used in treating diseases like autism and cerebral palsy in future. 6

7 Who can use banked umbilical cord blood? The goal of a bone marrow transplant/blood stem cell transplant is to treat and/or cure certain types of blood related diseases. Banked umbilical cord blood can potentially provide easier access to a stem cell transplant. Studies have shown that cord blood transplants can be performed in cases where the donor and the recipient are only partially matched, depending, for example, on the number of stem cells in the collection. In contrast, bone marrow transplants require a perfect match in most cases. Your baby s cord blood stem cells will always be: 100% perfect match At least a half match Autologous: using your own stem cells Syngeneic: using stem cells from an identical twin Haploidentical: using stem cells from biological parents 25% chance of being a perfect match for 1: chance of being a perfect match Allogeneic: using your sibling s stem cells Allogeneic: using an unrelated donor s stem cells (recruited through the South African Bone Marrow Registry) The good news is that with umbilical cord blood stem cells, you don t always need a perfect match. This increases your chances of finding a matching donor. 7

8 What have cord blood stem cell therapies done so far? YEAR blood-related disease was treated more than cord blood transplants have been performed to date YEAR types of diseases were treated YEAR types of diseases were treated YEAR 2020 Who knows what the future still holds, but it looks promising Sample releases from Cryo-Save for treatment Blood stem cell transplants have been performed for more than 50 YEARS and have been used to treat or cure more than 80 potentially life threatening blood related diseases like leukaemia, lymphoma and bone marrow failures. Within the Cryo-Save Group internationally, there have been 18 stem cell releases to date - to centres such as Duke University North Carolina, USA, Kliniek Pediatrische Hematologie en Oncologie Frankfurt, Germany and Kinderspital Zurich, Switzerland. One cord blood unit was released from Cryo-Save SA, which was for diagnostic genetic testing for acute lymphoblastic leukaemia. This confirms that the Cryo-Save South Africa laboratory aligns with international standards, validating the standards of our cord blood products. 14/18 have been for autologous (own stem cells) & sibling transplants 4/18 have been for clinical trails for cerebral palsy 8

9 Umbilical cord blood approved stem cell therapies First used Cord blood stem cells were first successfully transplanted in 1988, and now it is used to treat over 80 blood-related diseases. BLOOD CANCERS Acute Lymphoblastic Leukaemia (ALL) Acute Myeloid Leukaemia (AML) Chronic Myeloid Leukaemia (CML) Myelodysplastic Syndrome (MDS) Multiple Myeloma Hodgkin's Lymphoma Non-Hodgkin's Lymphoma Bone marrow failure syndromes Severe Aplastic Anaemia, Unspecified Fanconi Anaemia BLOOD DISORDERS Sickle Cell Disease Thalassemia IMMUNODEFICIENCIES Severe Combined Immune Deficiency (SCID) Wiskott-Aldrich Syndrome INHERITED METABOLIC DISORDERS Hurler Disease (MPS type IH) Osteopetrosis Adrenoleukodystrophy Krabbe Disease AUTOIMMUNE DISEASES Systemic Lupus (SLE) Umbilical Cord blood research Clinical trials where children use their own (autologous) cord blood stem cells Clinical trials where children use donor (allogeneic) cord blood stem cells Cerebral palsy Autism Neonatal oxygen deprivation Acquired hearing loss Encephalopathy (neonatal) Hypoplastic left heart syndrome Stroke (pre/peri-natal) Traumatic brain injury Type 1 Diabetes Alzheimer s disease Bronchopulmonary dysplasia Cartilage repair Cerebral palsy Critical limb ischemia Global development delay Graft versus host diseases HIV Fertility Intraventricular haemorrhage Liver cisshosis Neurodegenerative disorders Stroke Sweat gland regeneration Type 1 Diabetes 9

10 Umbilical cord blood - not just a bag of stem cells Cord blood therapies have shown highly promising results to treat diseases for which there are currently no available therapies. Results using autologous (own) cells in babies and children with HIE, cerebral palsy and autism spectrum disorder are safe and results are promising. The safety of partially or fully matched sibling cord blood infusions has also been demonstrated preliminarily. 1 Featured advanced cell therapy trial: Expanded access cord blood therapy for autism and cerebral palsy The Duke University Medical Center has received permission from the FDA to offer cord blood therapy for conditions like autism spectrum disorder and cerebral palsy under an expanded access clinical trial. This protocol establishes an umbrella clinical trial, registered on 31 October This trial enables children who have these neurological disorders to receive therapy with their own cord blood or cord blood from a sibling, regardless of whether they qualify for a targeted clinical trial. The registration of this clinical trial opens the door for many children who are afflicted with an acquired neurological disorder to travel to Duke University for cord blood therapy, provided they have a sufficiently matching cord blood unit in a family bank. Sibling therapy only requires a partial match, not a perfect match. 2 Reference: 1. WCBD17 Treating children with cerebral palsy and autism spectrum disorder with cord blood. Joanne Kurtzberg, MD 2. Cell Trials Data is a project of Parent s Guide to Cord Blood Foundation, a 501(c)(3) non-profit organization that is headquartered in the United States at Georgia Ave., Brookeville MD For more information visit 10

11 Cerebral palsy Joanne Kurztberg: Children who received a high dose of cord blood cells had statistically significant changes in their motor function, more than 30% above where they would have been predicted to be without cord blood therapy. These children made new connections in their brain and that s why their function was better. 3 1 in 326 children are affected Autism spectrum disorder Joanne Kurztberg: Some children, who were not speaking very much, had big increases in their vocabulary and their functional speech. Many children were able to attend to play and have meaningful communication in a way that they weren t before. Some children had less repetitive behaviours than they did when they came onto the study. 4 1 in 68 children are affected UCB has been shown to induce regeneration of the central nervous system. UCB contains a rich and diverse mixture of stem and progenitor (source) cells that have the potential to generate a variety of cell types. Umbilical cord blood cells improve motor function in experimental systems via paracrine signaling. Paracrine signaling is a form of cell-tocell communication in which a cell produces a signal to induce changes in nearby cells, altering the behaviour of those cells. 3. Cerebral Palsy: 1. Cellular Horizons Day 1 - Cord Blood for Treatment of Brain Injury Jessica M. Sun, Allen W. Song, Laura E. Case, Joanne Kurtzberg. Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial. Stem Cells Translational Medicine 2017;00: Autism: 1. Cellular Horizons Day 1 - Cord Blood for Treatment of Brain Injury Geraldine Dawson, Jessica M. Sun, Katherine S. Davlantis, Joanne Kurtzberg et al. Autologous Cord Blood Infusions Are Safe and Feasible in Young Children with Autism Spectrum Disorder: Results of a Single-Center Phase I Open-Label Trial. Stem Cells Translational Medicine 2017;6:

12 What are umbilical cord tissue stem cells? First used Clinical trials using MSCs started in 1995, with more than 200 trials currently underway. Umbilical cord tissue stem cells, also called mesenchymal stem cells (MSCs) are able to differentiate into certain specialised cells. Liver cells Nerve cells Bone and cartilage cells MSCs Mesenchymal stem cells found in Warton s Jelly, bone marrow asipose (fat) tissue and tooth pulp. Muscle cells Skin cells Fat cells Fat Bone marrow Mesenchymal stem cells can be collected from Umbilical cord tissue Clinical trials using MSCs for treatment of: Neurologic Disorders Autism (Phase 2) Cerebral palsy (Phase 2) Hearing loss (acquired sensori-neural) (Phase 2) Hypoxic Ischemic Encephalopathy (HIE) (Phase 1) Spinal cord injury (Phase 2) Cardiovascular Critical Limb Ischemia (Phase 3) Ischemic stroke (Phase 2 placenta) (Phase 3) Myocardial Infarction (Phase 3) Cardiomyopathy (Phase 3) Auto-Immune Disorders Crohn's Disease (Phase 3) Diabetes, Type 1 (Phase 2) Graft-versus-Host Disease (GvHD) (Phase 3) Kidney plus stem cell transplant (Phase 2) Systemic Lupus (SLE) (Phase 2) Multiple Sclerosis (Phase 1) Rheumatoid Arthritis (Phase 2) Scleroderma (Phase 2) 12

13 How are cord blood and tissue collected? Collecting umbilical cord blood and tissue is QUICK, PAINLESS and NON-INVASIVE posing no medical risk to mother or baby. After birth, the umbilical cord is clamped and cut before the baby is taken away for care. The blood is collected from the umbilical cord. A 20-25cm segment of the umbilical cord is collected in a quick, painless and non-invasive procedure. Cord blood and tissue collection is usually done within 10 minutes of giving birth Umbilical cord blood collection The average labour for a first time mom is about hours. Greater volume of blood = more stem cells. For a collection to be potentially successful, a minimum collection of 80ml of cord blood is recommended COURIER The sample is secured in a collection kit and retrieved by a medical courier COLLECT The cord blood is drawn into a collection bag. The tissue is cut and saved. 10 min CLAMP & CUT The umbilical cord is clamped and detached from the baby. Then it is cleaned. Speak to your doctor or midwife about the importance of collecting enough cord blood. Stage 2 Labour Cord blood collection takes place after birth and only once the umbilical cord has been clamped and cut. It is a painless procedure that poses no risk to mother or baby. In-utero collection With in-utero collection, the cord blood is collected immediately after the umbilical cord has been cut and before the expulsion of the placenta. This extraction mode requires about 10 minutes and is faster because uterine contractions allow cord blood to flow more easily. Ex-utero collection Ex-utero collection takes place after the expulsion of the placenta and takes about 25 minutes to complete. 13

14 1 We will deliver the kit to your home or office. 2 On the day of birth, BRING ALONG YOUR COLLECTION KIT. What happens after registration? 3 Once the baby is born, the umbilical cord, which is attached to mom and baby, will be clamped and cut. The baby will receive care away from the collection procedure. 6 Blood samples will be drawn from mom and placed inside the kit. 5 The doctor or midwife will complete the collection record form. 4 The doctor or midwife will draw the blood from the clamped cord into a special collection bag. 7 Dad will need to call the Cryo-Save laboratory. A specialised medical courier will collect the kit from mom s room and deliver it to the Cryo-Save laboratory. 8 At the laboratory, the cord blood and tissue are processed according to international standards. Viability, cell counts and sterility tests will be done. 9 The cord blood stem cells and tissue will be kept in a liquid nitrogen storage tank (between -196 and -150 degrees Celsius) inside our secured facility. 14

15 World class technology and service, affordable pricing* options for everyone 18 releases for treatment 17 years experience 30 countries 6 laboratories No.1 in Europe > stored samples Local and international accreditations The Cryo-Save and Salveo storage offering: Unmatched financial security for your investment. JSE:ECS AMS:ESP Different storage options to fulfil your needs. LOCAL storage in SA Different storage options to fulfil your needs. INTERNATIONAL storage in Europe Unmatched service from collection to release. REIMBURSEMENT OF SHIPPING AND CORD BLOOD-RELATED TESTS UPON RELEASE Transport of stem cells is outsourced to experts to safeguard your baby s stem cells. HEALTHCARE COURIER Assurance that your cord tissue has been properly tested and verified to be suitable for long-term storage. CORD TISSUE VIABILITY AND PHOTOGRAPHIC EVIDENCE We take responsibility. COST FREE DONATION PROGRAMME 12 and 24 months interest free payment plans. 60 month interest free payment plan. * Invest in hope from R273 per month. Terms and conditions apply. 15

16 Why Biotech? Advances in the global medical biotechnology industry are changing the face of medicine. These advances are strongly driven by the developments in personalised medicine - a paradigm based on providing treatments best suited to a patient s unique condition, genetic disposition and other health characteristics. Today, medical treatments are largely chemically based, like pain relievers, antibiotics, antidepressants and chronic medications to manage conditions such as high blood pressure, elevated cholesterol levels or thyroid imbalances. Biotechnology on the other hand, is allowing healthcare practitioners to learn more about the causes of disease at a cellular level. These unique insights into DNA and the genetic causes of illnesses are helping patients and doctors to identify and prevent illnesses before they occur, or to find the correct treatment for an individual. About Ecsponent Biotech As one of the specialised divisions in Ecsponent Limited s equity holdings stable, Ecsponent Biotech invests in products and services that promote the adoption of personalised and regenerative medicine among South Africans to improve their quality of life. Additionally, Ecsponent Biotech focuses on the advancement of children s health, by providing access to affordable, lifesaving technologies to everyone who wants to make responsible and informed choices for their children. Investments in several international and local brands combine the technical excellence of leading biotech organisations with Ecsponent s local business expertise. These investments include Cryo-Save (Europe s largest stem cell bank) and Salveo Swiss Biotechnology (Geneva-based leader in predictive medicine). World-class laboratory The Cryo-Save South Africa laboratory is at the heart of these operations. This laboratory, accredited to the highest international standards, is based in Pretoria and has stored thousands of umbilical cord blood and tissue samples for parents since its establishment in With no public stem cell bank in Africa, the Cryo-Save laboratory provides access to private stem cell processing and storage services in southern Africa. Offerings by Ecsponent Biotech In addition to stem cell banking through leading global brands, Ecsponent Biotech offers a growing range of complementary products and services to advance health and quality of life. Umbilical cord blood and tissue storage for parents invested in the future of regenerative medicine and tissue engineering. Putting world class, life-saving stem cell technology within the reach of all South African parents. Professional nurse assists with in-utero and ex-utero collection of cord blood to ensure adequate collection volumes. Verify a mother s omega-3 levels in the third trimester of pregnancy. Omega-3 is linked to improved brain, eye and cognitive development of babies. Verify a mother s omega-3 levels for optimum quality breast milk. Omega-3 levels are dependent on dietary intake - making this test vital for all breastfeeding mothers. Personalised therapy to improve the quality of life of people suffering from severe ocular surface disorders. Information provided by Ecsponent Biotech ( on behalf of Cryo-Save South Africa and Salveo Swiss Technologies South Africa. This is for information and educational purposes only, and should not be construed as healthcare advice. Please discuss this information with your healthcare professional. A full list of references is available on the website and at our offices. Data on file. DOC SA V4 Effective date: 20 November 2017 Office hours: Please phone us at ecsbio.com