Cord blood cured 44 children from genetic diseases

Since the first use of umbilical cord blood stem cells in 1988, more than 35,000 transplants of this valuable biological material have been performed worldwide. The range of diseases in which umbilical cord blood stem cells are used is constantly expanding, and today includes about 100 nosological units.

Researchers from UPMC Children’s Hospital of Pittsburgh (USA) recently reported that they safely and effectively treated 44 children from a variety of serious genetic diseases with the help of umbilical cord blood stem cells. These include sickle cell anemia, thalassemia, Hunter’s syndrome, Crabbe’s disease, metachromatic leukodystrophy, and a number of immunodeficiencies. This is the largest clinical study of its kind using umbilical cord blood conducted to date. The results of the treatment of patients were published in the journal Blood Advances.

Umbilical cord blood stem cells were administered intravenously to patients. Stem cell transplantation was preceded by low-dose chemotherapy and immunosuppressive drugs. Doctors tried to create a therapeutic protocol that would actually be used in different centers at a low cost of treatment. According to this scheme, patients after the first dose of stem cells and their “integration” with the recipient’s body were administered a small subsequent dose. It is important to note that the developed method of treatment did not require strict immunological compatibility between donor and recipient.

The developed method of treatment of genetically determined diseases is especially important for ethnic minorities, because due to the high variability of antigens on the cell surface, children from parents of different nationalities can find it very difficult to find a compatible bone marrow donor.

In treated patients, complications from umbilical cord blood stem cell transplantation were relatively minor. No patient developed an acute form of graft-versus-host disease, and the mortality rate from viral infections was 6%, which is significantly lower than in previous clinical trials.

In all 30 children with genetic metabolic disorders, in which the patient accumulates harmful toxins, a year after treatment, normal levels of enzymes and improvement of neurological symptoms were observed.

The most common disease for which transplantation was performed was leukodystrophy. Patients with this disease usually die within a few years after the first symptoms. Even with umbilical cord blood transplantation, only 60% of children previously lived 3 years after treatment. When using the new protocol described above, more than 90% of patients were alive 3 years after transplant.

None of the previously known treatments has provided such a high level of safety, efficacy and versatility in the treatment of various diseases. This transplant scheme is effective in at least 20 diseases.

The new method of treatment is already being successfully used in adults.


In Ukraine, future parents since 2003 have the opportunity to preserve perinatal tissues (umbilical cord blood, placenta, umbilical cord) at the birth of a baby in the Ukraine’s first Cryobank of the Institute of Cell Therapy in accordance with international standards for cryopreservation of biomaterial. Preserved cells and tissues can be used in the future in the treatment of diseases.