In March 2015, WORLD CORD BLOOD CONGRESS V & INNOVATIVE CELL THERAPIES took place in Monaco. Congress traditionally was organized by Prof. Eliane Gluckman (Paris university, France), who in 1988 performed the world’s first cord blood stem cells transplant to a child with Fanconi anemia.
The Congress discussed hot issues of biology of cord blood stem cells, evolution of umbilical cord blood banking, ethical and legal aspects of the industry, the results of the use of umbilical cord blood in children and adults, methods of engraftment stimulatation, innovative treatments of non-malignant diseases.
Actually more than 35,000 cord blood transplants for the treatment of cancer and other diseases in children and adults were performed worldwide. By the end of 2013, the global registry of public cord blood banks included nearly 731,280 HLA-typed cord blood samples ready for transplantation. Significantly more samples of umbilical cord blood is stored in banks of family type.
Interestingly, that if in the early 90-s of XX century related stem cell transplantations of umbilical cord blood were dominating, from the year 2000 the number of unrelated transplants increased. Thus, as of 2014 unrelated transplants made about 90% of the total number of cord blood transplants performed since 1988. According to these statistics, from 1988 to 2014, 53% of cord blood transplants were performed in children and 43% of operations – in adult patients. However, since 2008 the number of cord blood transplantation in adults each year exceeds this number in children.
The world’s first patient, who was performed cord blood stem cells transplantation in 1988 (25 years after transplant, with his family) http://www.esh.org/
Prof. Gluckman, in her report, “The history and future of cord blood transplantation,” noted that survival rates of recipients of cord blood are compatible with those in bone marrow recipients. Among the advantages of cord blood modern scientists consider the immediate availability of a transplant, no risk to the donor, ethics, applicability both to children and adults, a lower incidence of of “graft-versus-host.” Delayed transplant engraftment, compared to bone marrow is considerd to be a negative factor of cord blood transplantation.
In the light of modern science the value of cord blood is not only hematopoietic stem cells but also other cell populations – very small embryonic-like cells, endothelial colony-forming cells, mesenchymal stromal cells from umbilical cord blood, unrestricted somatic stromal cells. These cell populations are promising for regenerative therapy. Also, in addition to cell transplants, umbilical cord blood is a biological upstream material suitable for the manufacturing of plasma drugs and platelet gel, isolation of immune cells, particularly lymphocytes NK and T reg and transfusion purposes.
Also actual issues of stimulation of cord blood engraftment, prevention and control of infectious complications in recipients, techniques of pretransplantation expansion of cord blood stem cells and lymphocytes, individualization of conditioning regimens in cord blood recipients, current indications for regenerative therapies using cord blood stem cells and the effectiveness of the latter were discussed at the Congress. In particular, the first results of the use of cord blood stem cells in the treatment of bronchopulmonary dysplasia and spinal cord injury were presented.
According to WHO, bronchopulmonary dysplasia is the main cause of death in preterm infants. These positive results of pre-clinical studies in mice have become a prerequisite for conducting Phase 1 clinical trial to study the efficacy of cord blood stem cells in the treatment of bronchopulmonary dysplasia. The two fractions of cord blood stem cells were injected intratracheally into the right and left lung in a dose 1-2×107 MSCs / kg in children aged 7-14 days after birth at 24-26 weeks’ gestation. No side effects of the therapy were observed. Compared with the control group, researchers observed an improvement of respiratory parameters in children receiving intratracheal infusion of umbilical cord blood.
The first clinical experience of the successful application of cord blood stem cells in the treatment of spinal cord injury was described in 2014. As a result of cell therapy, improvement of sensitivity, motor function, self-help skills were noted. Stem cells exert antiapoptic effect in neurodegenerative diseases.
The international study of the team of scientists from Eurocord – Monacord, EBMT and CIBMTR analyzed the results of treatment of 999 patients with sickle cell anemia, 80% of patients were successfully treated.
The team of scientists from the company “Sangramo Biosciences” presented a very interesting report on the high-tech methods of treatment of thalassemia and sickle cell anemia, which include targeted modification of genes in hematopoietic stem cells. Scientists have come to conduct Phase 1 clinical trials. The success of this method of transplantation of autologous stem cells can be used to save thousands of patients with thalassemia from difficult and often fruitless search for the donor of hematopoietic stem cells.
Prof. Jean-Antoine Ribeil (Paris, France), in the report “Gene therapy of beta-globin disorders: first results” presented the results of two clinical trials devoted to the study of gene therapy in the ex-vivo treatment of patients with β-thalassemia major. According to the authors of the study, the safety of this kind of treatment of β-thalassemias is comparable to autologous transplantation, without considering the possible complications of the gene therapy. The advantage of gene therapy of thalassemias is the early achievement of independence from transfusions.
The representative from Brazil shared the experience of the functioning of the program of the public umbilical cord blood banking in the country. So, the public cord blood bank, established at the clinic Hospital de Clinicas de Porto (Brazil) from 2001 to 2014 managed to procure 1300 samples of umbilical cord blood. In general, there are 17 cord blood banks in Brazil.
A colleague from Canada spoke about the experience of the first year of operation of the National Canadian Public Cord Blood Bank. The national public cord blood bank in Canada was created on March 14, 2011 by the Ministry of Health. Canada’s national program of cord blood banking has allocated an investment of 48 million dollars over the 8-year period. During the first year of the bank operation 1,348 samples were collected by “ex utero” method and 720 samples – “in utero”. According to the requirements to the public cord blood banks 346 and 207 samples were suitable for further cryopreservation, respectively – about 25%. Microbial contamination occurred in 0.29% of samples of umbilical cord blood, collected “ex utero” and in 2.42% of “in utero” collected samples. The average volume of cord blood samples collected by the method “in utero” was 96.7 ml and 85 ml – “ex utero”.
Summing up the main messages of the WORLD CORD BLOOD CONGRESS V & INNOVATIVE CELL THERAPIES (Monaco, 2015), we can conclude that industry of cord blood banking is actively developing all over the world. Continuous improvement of methods of expansion of stem cells and conditioning regimens, facilitation of engraftment results in increase of the number and effectiveness of transplantation of cord blood stem cells. The achievements of modern science define new trends in cell therapy – targeted gene therapy, the use of manipulated cells, bioengineering etc.