In July 2015, a group of Professor Tomohiro Morio and Associate Professor Hirokazu Kanegane, Department of Pediatrics, Tokyo Medical and Dental University Hospital, performed bone marrow transplantation for children with severe congenital alveolar proteinosis, and succeeded for the first time in the world. Did.After the transplant, her symptoms improved and she was discharged in November.
There are hundreds of millions of small sac-like tissues called alveoli in the lungs, and on the surface of the alveoli, there is a substance containing a protein called surfactant that helps to spread the alveoli.Pulmonary alveolar proteinosis is a disease in which this substance accumulates in the alveolar for some reason. In particular, congenital alveolar proteinosis is an extremely rare hereditary disease in Japan and has been designated as an intractable disease. ..
The patient was a 1-year-old girl who was admitted to the hospital in March 2015 due to immunodeficiency.Based on the test results for alveolar proteinosis caused by the loss of dendritic cells, which are a type of immune cells, hematopoietic cell transplantation has made it possible to cure.Sources of hematopoietic cells include bone marrow, umbilical cord blood, and peripheral blood. The patient underwent umbilical cord blood transplantation in May, but engraftment failure occurred.Therefore, in July, a bone marrow transplant was performed with the father as a donor, and complications such as acute graft-versus-host disease in which the transplanted cells showed an incompatible reaction with the patient's tissue occurred, but recovered by hard treatment in November. I was able to leave the hospital.No serious side effects were observed other than complications, and after transplantation, dendritic cell recovery and alveolar proteinosis were improved.
Since hematopoietic cell transplantation has cured congenital alveolar proteinosis, it is possible that various intractable diseases against the background of primary immunodeficiency due to congenital defects can be cured by hematopoietic cell transplantation. it was done.However, there are some unclear points such as the cause of dendritic cell loss.Currently, we are working with a group of Professor Toshitoshi Oki, Department of Biodefense, Institute of Intractable Diseases, Tokyo Medical and Dental University, to elucidate these causes, and hope to lead to the development of safe and effective treatment methods. increase.
