Cord blood is rich in hematopoietic (blood-forming) stem cells (HSCs), which, starting 1970s, have been used for transplantation to treat a whole range of severe diseases. As of today, these are, first of all, oncohematological diseases, a number of blood and immune system diseases, certain inherited diseases. Throughout the world, HSC transplantations (HSCT) are also widely used to restore hematogenesis following high-dose chemotherapy.
Currently, the most commonly used method of transplanting HSCs for treating both children and adults is to make allogeneic (including related donor) transplantations of HSCs from cord blood, bone marrow, and peripheral blood, as well as autologous transplantations of peripheral blood HSCs. The latter are mainly used for treating adults, since personal cord blood banks have existed for just the last 10 years in Russia, and 20 years in the rest of the world. The illnesses that are treated using autologous transplantation most often occur at a later age than the age of today’s cord blood bank users.
First the medical field learned how to draw HSCs invasively from bone marrow, and later – from peripheral blood as well (through migration from bone marrow under the influence of specific stimulants). Starting from 1988, the medical field began to use a new method of obtaining HSCs non-invasively – using the umbilical-placental complex of the newborn. The blood contained in the placenta and in the attached umbilical cord is collected, and subsequently a concentrate of stem cells is extracted from the blood in the laboratory. This concentrate is then stored in deep cold. The non-invasiveness and safety of the procedure are accomplished due to the fact that blood is collected from the material that then is thrown away with the rest of the afterbirth (after the separation of the child).
It’s noteworthy that HSCs obtained from umbilical cord blood is much younger than similar cells from bone marrow and peripheral blood, as they are stored at the beginning of life, and are in a state of high biological activity. It’s been proven that under allogeneic transplantation, cord blood cells are less likely to cause complications such as the graft-versus-host reaction than do cells taken from bone marrow and peripheral blood.
Having a personal stem cell bank makes it possible to conduct transplantation in time and save the life of the close relatives of the child whose cells are kept in storage, namely an ill brother or sister, since for them these HSCs can be HLA-matched (tissue-compatible) donor material.
Moreover, if there is already sick child in the family, whose treatment ought to include HSC transplantation — while no matching donor found – today it is possible to apply the innovative method of giving birth to a healthy sibling to become a donor of HSCs for an older child. This technology was firstly applied in 2000, when using the Preimplantation Genetic Diagnosis (PGD) a pioneer baby, Adam Nash, was born to save the life of an older sister, who had and inherited disease – Fanconi Anaemia. Without HSC transplantation the girl, Molly Nash, would not have lived to see her 10th birthday. As Molly’s parents failed to find a fully compatible donor match for her, they decided to undergo in vitro fertilization using PGD to select an embryo free of the genetic mutation casing Fanconi Anemia and also histocompatible. Upon birth, cord blood stem cells from Adam were collected and stored. Afterwards they were transplanted to Molly and saved her life.
In Russia this technology that combines preimplantation genetic diagnosis (PGD), IVF, and transplantation of hematopoietic cord blood stem cells was firstly implemented by HSCI. The patient was a girl with Shwachman-Diamond syndrome. Using PGD, HSCI’s specialists helped the family give birth to a healthy boy (in 2014), for which it was chosen for implantation the embryo who was not only free of the genetic mutation causing the disease of his older sister, but also 100% HLA-matched with her. Therefore, on the one hand, HSCI helped the family to get a healthy child; while on the other hand, fully compatible hematopoietic cord blood stem cells were collected at his birth, which hematologists used for treating the Shwachman-Diamond syndrome of the elder child: the successful transplantation was made in 2016. For details see: Bone Marrow Transplantation advance online publication 27 March 2017; doi: 10.1038/bmt.2017.46 (First experience of hematopoietic stem cell transplantation treatment of Shwachman–Diamond syndrome using unaffected HLA–matched sibling donor produced through preimplantation HLA typing).
As for autologous transplantations, the practice of employing preserved cord blood HSCs will only grow over time. In particular, as was noted earlier, these cells can replace the person’s own peripheral blood cells for treating the complications of cancer chemotherapy.
The spectrum of diseases which experts see the possibility of treating using cord blood is expanding together with the progress of research. This relates primarily to the field of regenerative medicine. Presently, for example, the practice of using cord blood cells in the treatment of ICP (Infantile Cerebral Paralysis) is expanding universally, and Gemabank® has already provided a number of HSC samples for this purpose (both for allogeneic and autologous transplantations).
Furthermore, clinical studies worldwide are using cord blood cells for treating diseases of various kinds and origins, including diseases of the internal organs. Particularly promising is the use of the method of transplanting cord blood cells in the treatment of autoimmune diseases (multiple sclerosis, etc.), liver cirrhosis, Alzheimer’s disease, acquired deafness, and autism.
In addition, a series of preclinical studies (on animal models) has shown the effectiveness of cord blood cells in the treatment of ischemic heart disease and stroke.
