Stem Cell Transplantation

US News - Cancer 2016-2017

Hematopoietic stem cell transplantation can be a curative therapy for children and young adults with immunological, hematological and metabolic diseases as well as for patients with cancers who have not responded to standard chemotherapy.

The Pediatric Stem Cell Transplantation program was established at Lucile Packard Children’s Hospital Stanford in 1986. Since then, we have transplanted more than 900 patients.

The program is housed in a 12-bed Stem Cell Transplant Unit with HEPA-filtered air and a dedicated nursing staff on the first floor of Lucile Packard Children’s Hospital Stanford. The program is growing and will move to a new 24-bed unit. Now that the Stanford Laboratory for Cell and Gene Medicine is open, the program is capable of producing cells required for both cellular and gene therapies.

The program is an active member of the Children’s Oncology Group, the Pediatric Bone Marrow Transplantation Consortium and the Bone Marrow Transplantation Clinical Trials Network. Our patients are treated on either standard of care protocols or investigational protocols for diseases and conditions that have no established therapies. The investigational protocols have all been approved by both the Food and Drug Administration (FDA) and the Stanford Investigational Review Board. The program’s attending physicians are all board-certified physicians with accreditation in hematology, oncology and/or immunology, and all are certified by the Foundation for the Accreditation of Cellular Therapy (FACT).

Our principal goals are:

  • Improving the effectiveness and safety of stem cell transplantation
  • Developing approaches to treat patients and diseases that cannot currently be successfully treated by stem cell transplantation

We are currently developing several innovative theories, including:

  • An antibody-mediated preparative regimen for hematopoietic stem cell transplantation that does not require either chemotherapy or irradiation, thereby eliminating the side effects associated with the use of chemotherapy and irradiation
  • Regulatory T lymphocytes (TR1 cells) that are being evaluated for their ability to prevent acute graft-versus-host disease in mismatched stem cell transplantation
  • Innovative preparative regimens with reduced or no doses of cyclophosphamide to reduce or eliminate the side effects associated with cyclophosphamide

With the opening of the Stanford Laboratory for Cell and Gene Medicine, clinical Phase 1, proof-of-concept trials using gene editing and gene transfer techniques are under development to treat patients with diverse diseases, including:

  • Patients with sickle cell anemia, who will be treated by the transplantation of autologous hematopoietic stem cells that have been edited to replace the sickle cell anemia gene with the normal hemoglobin gene
  • Patients with IPEX syndrome, a genetic immunodysregulatory disease, who will be treated by the infusion of autologous T lymphocytes that have been transduced with a lentiviral vector containing the normal FOXP3 gene followed by the transplantation of autologous hematopoietic stem cells transduced with the same FOXP3-containing lentiviral vector
  • Patients with acute myelogenous leukemia, who will receive TR1 cells as a means to reduce acute graft-versus-host disease and also as immunotherapy for their acute myelogenous leukemia


Pre-clinical and developmental research has been undertaken by the members of the Division of Stem Cell Transplantation and Regenerative Medicine and other scientists at Stanford University. The Stanford Laboratory for Cell and Gene Medicine makes it possible for pre-clinical research to be directly translated into Phase 1, proof-of-concept clinical trials at Lucile Packard Children’s Hospital Stanford.

The clinical stem cell transplant of each patient is determined by the patient’s disease, the source of the hematopoietic stem cells used for transplantation, the stem cell donor and the patient’s prior clinical history. The stem cell donor can be the patient themselves (autologous) or someone else (allogeneic). The stem cells can come from bone marrow, peripheral blood or cord blood. The patient’s disease and clinical history will determine the specific therapy that he or she receives prior to transplantation. Patients are usually hospitalized for 1 to 2 months depending upon their clinical status. After their discharge, patients will have follow-up visits in the outpatient clinic, including the long-term follow-up clinic.
Patient and family support

We recognize that transplantation places enormous emotional, financial, psychological and logistical burdens on patients and families. In addition to a dedicated inpatient and outpatient nursing staff, the program provides multifaceted support for patients and families, including dedicated social workers and child life specialists; a hospital-based school; physicaloccupational and speech therapy; and spiritual support. Our social workers also conduct group therapy sessions for families. The hospital-based school is a unique collaboration between the hospital and the Palo Alto Unified School District that permits young patients to continue their schoolwork during transplantation and aids in their transition back to their original school.

In addition, the newly renovated Ronald McDonald House is available for housing patients and families. A shuttle is available to transport patients and families between the house and the hospital.

Contact us online or call (650) 497-8953 for more information.