Conditions & Treatments
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Stem cell transplant (bone marrow transplant)

Disease Information

Research & Innovation

Dana Farber/Children’s Hospital Cancer Center is a world leader in pediatric stem cell transplantation and stem cell research.

Our doctors work with national research groups, including the Children’s Oncology Group, Blood and Marrow Transplant Clinical Trials Network and the Pediatric Blood and Marrow Transplant Consortium to develop innovative clinical trials and expand transplantation to new patients.

Some of our current research activities include:

Researchers are investigating the use of gene therapy to improve stem cell transplantation. Gene therapy is a method of altering the genetic information stored in cells and thus correcting a defect. Our research focuses on the use of gene therapy and stem cell transplant for children with severe combined immunodeficiency (SCID) and adrenoleukodystrophy, and we hope to expand these techniques to treat other conditions in the future.

We are expanding the use of stem cell transplants to treat non-cancerous conditions. Both sickle cell disease and thalassemia can be cured through stem cell transplantation. We are currently working to make stem cell transplant safer for patients with these diseases and to make the therapy available to a broader patient population. Your child’s hematologist can talk with you about whether stem cell transplantation is possible therapy for your child.

We are very involved in research investigating supportive care of patients before, during and after transplant including evaluating the role of vitamin D in stem cell transplantation, post-transplant fatigue and pulmonary complications following transplant.

For more information about our research, visit our stem cell research website.

Children speak: What’s it like to be a medical research subject?
View a day in the life of Children's Clinical and Translational Study Unit.

Stem cell transplant saves “bubble boy” from Argentina
Can you imagine how you’d feel if you couldn’t hold your child? Agustin Caceres’s parents could not come in physical contact with him when he had Severe Combined Immunodeficiency, better known as “bubble boy disease.”Read how Children’s and the Dana Farber’s Cell Manipulation Core Facility were able to use stem cell transplant treatment so he could be in touch with his family and the world again, literally and figuratively. Read more about the international gene therapy trial.

Stem cell transplant saves “bubble boy” from Argentina

Can you imagine how you’d feel if you couldn’t hold your child? Agustin Caceres’s parents could not come in physical contact with him when he had Severe Combined Immunodeficiency, better known as “bubble boy disease.”Read how Children’s and the Dana Farber’s Cell Manipulation Core Facility were able to use stem cell transplant treatment so he could be in touch with his family and the world again, literally and figuratively. Read more about the international gene therapy trial.

“Imprinted” genes help control stem cells
Carla Kim, PhD, of the Stem Cell Research Program studied stem cells in the lung and the role of “imprinted” genes, which help regulate the growth of stem cells. Learn more about the imprinted gene research on Children’s newsroom.