For 22 years, umbilical cord blood cells have saved the lives of people with leukemia, congenital blood diseases, and immunodeficiencies. Currently, intensive research is underway on the possibility of using them in the treatment of many other diseases, e.g. cardiovascular or nervous system.
The latest state of knowledge on this subject was presented by scientists during the World Cord Blood Congress, which was held in Marseille on November 4-7.
The history of cord blood stem cell transplantation began in 1988, when the parents of the American boy Matthew Farrow, suffering from a serious blood disease, decided to trust the French hematologist prof. Eliane Gluckman. Under the guidance of a specialist, in Paris, the boy underwent the world’s first transplant of cord blood cells that came from his sister and were identical in terms of HLA antigens.
Matthew suffered from Fanconi anemia, a genetically determined disease that leads to bone degeneration and an increased predisposition to cancer. Today he is a fully healthy man, husband and father, and the number of life-saving cord blood transplants – according to the latest data – has exceeded 21 worldwide. In 2009, for the first time in the US, the number of cord blood cell transplants was greater than that of bone marrow transplants.
Today we know that the survival of patients who are transplanted with umbilical cord blood cells is the same as after a bone marrow stem cell transplant. We also know that although their hematopoietic system regenerates more slowly, they are less exposed, for example, to a graft versus host reaction harmful to the recipient – said Prof. Gluckman, who chaired the congress in Marseille.
According to Dr. n. med. Dariusz Boruczkowski, a specialist in children’s diseases and clinical transplantation from the Polish Stem Cell Bank, the advantages of cord blood include the fact that it is easily available, and the method of its collection is completely non-invasive. Transplantation requires fewer tissue antigens to match than cells from bone marrow or peripheral blood, and there is less risk of transmission of any infection.
Its biggest disadvantage is that the period of the hematopoietic system recovery after transplantation is about two weeks longer than after bone marrow transplantation – said the specialist in an interview with PAP. Dr. Boruczkowski was in the team of doctors who were the first in Poland to use bone marrow stem cells to treat an inherited disease called adenoleukodystrophy.
As prof. Gluckman, currently the most common indications for cord blood transplantation are: leukemias, hemoglobinopathies (genetically determined diseases), aplastic anemia with bone marrow failure (e.g. Fanconi anemia), complex immunodeficiencies and congenital metabolic disorders.
However, scientists are conducting intensive research on the use of umbilical cord blood in the treatment of other diseases, including damage to the nervous system, heart, liver, autoimmune diseases (multiple sclerosis, type 1 diabetes, Crohn’s disease) and many others. This is due to, inter alia, from the fact that – like bone marrow – umbilical cord blood can be not only a source of hematopoietic stem cells, but also cells capable of transforming into other tissues, e.g. cartilage, heart muscle or nervous tissue.
Some of these studies are very intriguing, but there is no convincing evidence yet that these therapies work. That is why it is very important for scientists to carefully transfer laboratory tests to clinical practice, Prof. Hal Broxmeyer of Indiana State University, who is one of the pioneers in cord blood transplantation.
Prof. Joanne Kurtzberg from the Duke University Medical Center in Durham (USA) started research on the effectiveness of transplanting own umbilical cord blood (the so-called autologous transplant) to children with cerebral palsy.
I use umbilical cord blood cells to correct genetically determined metabolic conditions like mucopolysaccharidosis that interfere with brain development. If these cells are transplanted early, neurological changes in children can be prevented – told PAP Prof. Kurtzberg.
In this case, the therapeutic effect is mainly due to the fact that the transplanted cells produce the correct enzyme that replaces the absent or defective version. These cells cross the blood-brain barrier, unlike the enzyme when it is administered as a drug.
As we have some evidence that these cells function also with other mechanisms, such as the fact that they secrete a number of growth factors and can inhabit the damaged brain, we decided to test them in children with acquired nerve damage such as cerebral palsy. This is the beginning of research and it is difficult to predict what the results will be, the researcher said.
According to her, umbilical cord blood stem cells are more effective in the treatment of metabolic disorders than bone marrow cells. We suppose that one of the reasons may be that they have a greater development potential – she explained.
From Marseille Joanna Morga (PAP)