STEM CELLS : A THERAPEUTIC REVOLUTION JACQUES KADOCH ROBERT HEMMINGS MARINELA MANDRA

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1 STEM CELLS : A THERAPEUTIC REVOLUTION JACQUES KADOCH ROBERT HEMMINGS MARINELA MANDRA OVO CLINIC I 8000 BLVD DECARIE, MONTREAL QC H4P 2S4 I I OVOCLINIC.COM

2 2 a therapeutic revolution As the achievements in Stem Cell therapy research are growing each year, and with it the number of successful transplantations, the recommendations and the approach of the medical authorities has changed, focusing mostly on the physician s educational role to offer accurate and scientific based information. OVO BIOSURANCE has the mission to offer to health care providers and parents accurate information regarding Stem Cell Therapy In 2008, the American Congress of Obstetricians and Gynoecologists (ACOG) changed its position regarding the "Umbilical Cord Blood Banking" affirming that : «If a patient requests information on umbilical cord blood banking, balanced and accurate information regarding the advantages and disadvantages of umbilical cord storage should be provided.» 1 In October 2009, the World Stem Cell Summit, affirmed: «Recent developments in the autologous use of cord blood support the argument that parents should be allowed to make an educated decision concerning the storage of their child s cord blood 2.» (WORLD STEM CELL SUMMIT STEM CELL BANKING THE GROWTH OF PUBLIC AND PRIVATE CORD BLOOD BANKS).

3 A scientific revolution? stem cells 3 WHAT ARE STEM CELLS? Stem cells are the body's "master" cells that regenerate and get specialized in all types of cells that build up the tissues, organs, and systems found in the human body. Stem cells have 2 main abilities: 1- They can give rise to specialized cell types such as blood cells, cardiac cells, nerve cells or liver cells. 2- They are capable of renewing themselves through cell divisions. Each new cell has the potential either to remain a stem cell or to become another type of cell with a more specialized function, such as a muscle cell, a red blood cell, or a brain cell. THERE ARE 3 TYPES OF STEM CELLS 1- Embryonic Stem Cells; 2- Adult Stem Cells: are found in the blood, bone marrow, liver, kidney, cornea, dental pulp, umbilical cord, brain, skin, muscle, salivary gland, testis, ; 3- Umbilical Cord Stem cells (blood, umbilical cord tissue and placenta).

4 4 applications STEM CELL APPLICATION THROUGH THE YEARS HISTORY OF CORD BLOOD TRANSPLANTS yr-old boy with acute lymphoblastic leukemia received cord blood units from 8 different unrelated donors, untested for any HLA compatibility.this was the first transplantation in cord blood ever tried. - Ende M, Ende N. Hematopoietic transplantation by means of fetal (cord) blood. Virginia Med Monthly 1972;99: yr-old boy cured of Fanconi anemia after receiving blood from his baby sister. It was the first successful cord blood transplantation - Gluckman et al New York placental blood program was opened - New York placental blood program First public UCB banks in New York, Milan, Dusseldorf, UK Cord Blood Bank became the first public umbilical cord blood bank in Canada. 33 cord-blood registries worldwide

5 applications 5 HOW MANY TRANSPLANTS HAVE BEEN DONE? Since the first successful umbilical cord blood stem cell transplantation in 1988, we have now seen approximately 20,000 transplants in total (according to the latest Stem Cell Summit Report in October 2009). Currently about 3,000 transplants are performed each year, meaning that we can safely speak of a medical standard. 4 HEMATOPOIETIC STEM CELL ALLOGENIC TRANSPLANTATIONS (bone marrow, peripheral blood and umbilical cord) Current indications for allogenic transplantations, include 4 : Acute myeloid leukemia Acute lymphoblastic leukemia Chronic myeloid leukemia Chronic lymphocytic leukemia Myeloproliferative disorders Myelodysplastic syndromes Multiple myeloma Non-Hodgkin lymphoma Hodgkin disease Aplastic anemia Pure red cell aplasia Paroxysmal nocturnal hemoglobinuria Fanconi anemia Thalassemia major Sickle cell anemia Severe combined immunodeficiency (SCID) Hemophagocytic lymphohistiocytosis (HLH) Inborn errors of metabolism (eg, mucopolysaccharidosis, Gaucher disease, metachromatic leukodystrophies and adrenoleukodystrophies)

6 6 applications HEMATOPOIETIC STEM CELL AUTOLOGOUS TRANSPLANTS There are over 206 autologous transplantations done all over the world, and the list is increasing each year. The graphic below shows the distribution of the autologous transplantation through the years, beginning from 1998, from which 150 were done in the last 2 years. 5 NB. OF TRANSPLANTATIONS The distribution of transplantations from 1998 The diseases treated until now with Autologous transplantation include: Cerebral palsy Diabetes type 1 Autism Aplastic anemia Neuroblastoma YEARS

7 applications 7 Approximately 20,000 transplants and currently about 3,000 transplants are performed each year The recommendations for the autologous transplantations include: 4 Multiple myeloma Non-Hodgkin lymphoma Hodgkin disease Acute myeloid leukemia Neuroblastoma Germ cell tumors Autoimmune disorders Systemic lupus erythematosus (SLE), systemic sclerosis Amyloidosis WHAT IS THE LIFETIME PROBABILITY OF A PERSON USING HIS STEM CELLS? The first and the most accurate prediction to date of the probability that a person will have a stem cell transplant was published in March 2008 by Nietfeld.This probability was estimated to be 1 in 435 or 0.23%, that a person will require an autologous transplant (his own stem cells) and becomes 1 in 217 if considering either autologous or allogenic transplant (from a donor). 6-7 This probability becomes higher as the list of recommendations increases each year.

8 8 applications CUMULATIVE PROBABILITY % AGE (YEARS) SOURCE: BIOPHYLAXIS.COM 6 STEM CELL THERAPY AND REGENERATIVE MEDICINE Regenerative Medicine is a rapidly growing field of biomedicine that seeks to create substitute tissues and organs for the human body, to repair or replace those whose function is lost through illness, injury, aging or congenital anomaly.

9 future 9 STEM CELL S FUTURE STEM CELLS AND NEURODEGENERATIVE DISEASES CEREBRAL PALSY - there are over 124 cases of children diagnosed with cerebral palsy and treated with their own stem cells. The continued interest in cord blood as a potential treatment for brain injury was highlighted by several research centers such as Duke University, which reports that some children who have undergone cord blood re-infusion for brain injury are developing more skills than they otherwise would have. Most of the autologous cases compiled between 2005 and 2009 were treated by Dr. Joanne Kurtzberg at Duke University, in Durham, North Carolina, USA. 8 A first FDA-approved clinical trial for evaluating the efficiency of the therapy with the cord stem cell in Cerebral palsy was announced in February 2010 by the Medical College of Georgia and led by Dr. James Carroll. 9 MULTIPLE SCLEROSIS (MS) - A successful first clinical trial in patients with MS was effectuated by the British scientists and published at the beginning of this year. This is an early stage clinical trial carried out only in 21 patients, but which gives courage to those suffering from this disease. 10 PARKINSON S DISEASE - There are studies trying to evaluate the possibility of using adult stem cells to replace dopamine neurons in Parkinson s disease. 11 STEM CELLS AND DIABETES -The first clinical trial was led by Dr. Michael J. Haller at Shands Hospital, University of Florida and the study was published in June 2007 by the American Association of Diabetes Based on the results in the US, a second trial was approved in Germany.

10 10 future STEM CELLS AND HEART DISEASE There are a lot of recent studies done to show the ability of Stem Cells to regenerate the damaged heart tissue after a myocardial infarction and can help in building human heart valves. One very interesting study, published in April 2010, by the team of University of Montreal, evaluated the evolution of patient s suffering from coronary ischemic diseases and who received treatment with adult stem cells, in order to increase the regeneration of the injured cardiac muscle. 14 STEM CELLS AND ORGAN TRANSPLANT Surgeons in Spain have carried out the world's first tissue-engineered whole organ transplant - using a windpipe made with the patient's own stem cells. The groundbreaking technology also means for the first time tissue transplants can be carried out without the need for anti-rejection drugs. 15 A second successful tissue-engineered windpipe transplant wasin 2010, this time for a 10-year-old British boy. 16 STEM CELLS AND AUTOIMMUNE DISEASE An observational study from the European Group for Blood and Marrow Transplantation (EBMT) showed that autologous hematopoietic stem cell transplantation HSCT can induce sustained remissions for more than 5 years in patients with severe Autoimmune Diseases. This study was published on September 22nd, 2009, and included patients form with autoimmune diseases such as Crohn s disease, Rheumatoid arthritis, etc. 17 STEM CELL THERAPY AND BONE REPAIRS British surgeons are pioneering a new technique which uses stem cells to repair damaged bones. The procedure could prevent thousands of people from needing to have an artificial hip fitted. 18

11 parents choices 11 WHAT ARE FUTURE PARENTS CHOICES? WHAT INFORMATION SHOULD I GIVE TO MY PATIENT? Cord blood can be donated to a public bank for use by a patient in need or stored at a family cord blood bank for the baby s or family s use. Donating it to a public bank or storing it for private use is a personal decision that the parents must make after being fully informed by their health care providers or by doing their own research. PUBLIC CORD BLOOD BANKS At this time, there is only one public cord blood bank available to accept cord blood donations from babies born in Quebec. They are Héma-Québec and you can find detailed information about their service in their web site. 19 Public cord blood banks save and use cord blood for transplants to help people with one of the treatable diseases or for scientific research. Transplants are anonymous and no information about the baby s or mother s identity are given. Patient has to give a blood sample to assess their genetic profile. Only a small number of individuals will eventually be accepted to donate stem cells. Donated cord blood becomes the property of the public cord blood bank.

12 12 patient choices FAMILY CORD BLOOD BANKS By storing stem cells with a private bank, the cord blood is owned by the family and is the family who controls its use. Studies show that there is less graft versus host disease complications and a better survival rate when cord blood from a related source is transplanted. Family banking, in Canada, usually cost a first fee between 1000$- 1200$ at the time of the delivery, and a annual storage fee between 120$ -150$

13 references 13 REFERENCES: Umbilical _Cord.37.aspx

14 14 legislation LEGISLATION TO ENCOURAGE CORD BLOOD EDUCATION In United States, currently 20 states, covering over two-thirds of U.S. births, have enacted legislation to encourage prenatal care providers to educate expectant parents about their cord blood banking options. This information should be given early enough in pregnancy so that parents can make an informed decision about whether to privately bank their child s newborn stem cells or donate it to a public bank