Pediatric Stem Cell Transplantation Treatment and Clinical Trials

What is a stem cell transplant?

How stem cells are obtained

Stem cells used for transplantation can be obtained from bone marrow, umbilical cord blood, or mobilized peripheral blood. Stem cells can be obtained from the bone marrow in the operating room under general anesthesia or from the peripheral blood by a process called apheresis, where the stem cells are collected and the rest of the blood is then returned to the donor. Cord blood is collected at the time of delivery from volunteer mothers and frozen until it is used.

Allogeneic stem cells

Allogeneic stem cells are collected from a related or unrelated donor. The allogeneic donor is chosen to be as genetically close to your child as possible. The best donor is a brother or sister who has the same HLA antigens (genes that make proteins that help the body differentiate between “self” and “other”) as your child. Siblings have a 25 percent chance of being such a donor. If a brother or sister cannot be a donor, we contact the National Marrow Donor Program (NMDP) to see if an unrelated volunteer donor has HLA typing similar to that of your child, and therefore, could be used as a stem cell donor. In cases when an unrelated donor cannot be identified, the father or mother (who have 50 percent of the same genes as the patient) can be used in a haploidentical transplant or unrelated cord blood can be used as a source of stem cells.

Autologous stem cells

For some forms of cancer, your child’s own stem cells can be collected from the bone marrow or peripheral blood and used for transplant.

Pre-transplant therapy

Before transplantation, your child will receive therapy to destroy the cancer or abnormal stem cells and allow normal stem cells to grow. The specific therapy that is administered will depend upon your child’s disease and clinical history. Our doctors are testing new approaches to use antibodies as an alternative to chemotherapy to prepare your child’s body for transplant.

Hospital stay

After the completion of pre-transplant therapy, the stem cells are infused into your child’s vein where they can travel to your child’s bone marrow. Your child must remain in the hospital's Stem Cell Transplant Unit until the donor stem cells have grown enough to permit your child to safely leave the hospital, usually 1 to 2 months after the transplant.

Stem cell transplantation and gene therapy clinical trials

Our doctors and researchers are constantly working to make stem cell transplantation safer and available to more people—especially patients suffering from conditions without a cure. After years of testing in the laboratory, we can bring potential treatments to our patients for testing in clinical trials.

Some of our unique clinical trials include:

  • A new approach to stem cell transplantation for patients with blood cancers and blood diseases that removes the immune system’s “fighter cells” from the donor’s cells before they are transplanted into the recipient. This approach aims to reduce the risk of graft-versus-host disease and enable patients without a fully matched donor to still have access to a potentially curative stem cell transplant. Another related trial also adds a cell therapy called T-allo10. This cell therapy is made from the cells of the same person who donated stem cells for the transplant. The cell therapy contains T regulatory cells that tell the recipient’s immune system to leave the transplanted stem cells alone. We are testing whether this approach can help the recipient’s immune system rebuild itself after transplant. Both trials are only available at Stanford.
  • Gene therapy for patients with immunodysregulation polyendocrinopathy enteropathy X-linked (IPEX) syndrome, adding a normal copy of the gene that is malfunctioning in patients with IPEX syndrome. This trial is only available at Stanford.
  • A new approach to stem cell transplantation for patients with Fanconi anemia, which aims to make transplantation safer using antibodies instead of chemotherapy to prepare the patient’s body for transplant, and removing certain immune cells from the donor cells to reduce the risk of graft-versus-host disease. This trial is only available at Stanford.
  • Gene therapy for patients with severe sickle cell disease, which aims to correct the genetic mutation that causes sickle cell disease and decrease the production of sickle-shaped red blood cells. Lucile Packard Children’s Hospital Stanford is the only hospital in the Western United States to offer this trial.
  • Gene therapy for patients with pyruvate kinase deficiency (PKD), which involves inserting a normal version of the gene that has malfunctioned to cause PKD (the PKLR gene) into a patient’s own stem cells and transplanting those cells back into the patient. Lucile Packard Children’s Hospital Stanford is the only hospital in the United States to offer this trial.

To learn more or make an appointment, please call (650) 502-7179.