Stem Cell Transplant & Cellular Therapies

Generally, stem cell transplantation and cellular therapy involves collecting cells, processing or modifying these cells in a laboratory, preparing the body to receive the cells, and returning the cells through infusion (intravenously, or through an IV). Nemours offers a variety of treatment options for kids with blood disorders.

Stem Cell (Bone Marrow) Transplantation

• Autologous transplants, using a child's own stem cells, collected and stored during remission. This approach reduces the risk of rejection and is often used for solid tumors and some blood cancers. Our advanced cell processing laboratory ensures the highest quality cell preservation.
• Allogeneic transplants, using cells from a matched donor, like a sibling or unrelated volunteer. We have access to the national donor registry and sophisticated matching methods to find the best possible donor. This option is often used for leukemia, lymphoma, and bone marrow failure conditions.
• Cord blood transplants, using blood saved from umbilical cords, which offers a rich source of healthy stem cells. This option can be especially helpful for children who don't have a matched donor. 
• Haploidentical transplants, using partially matched family donor cells, often from a parent. We’ve developed approaches to reduce complications, making transplant possible for children who lack a fully matched donor.

CAR-T Cell Therapy

CAR-T cell therapy involves collecting and modifying a child’s own immune cells to fight cancer. We're among the first centers nationwide to offer this breakthrough immunotherapy. Our program:

• Uses the latest techniques to minimize side effects.
• Provides expert management of immune responses.
• Offers this treatment for specific types of leukemia and lymphoma.
• Participates in clinical trials to expand treatment options not available everywhere.

Gene Therapy

Gene therapy targets the underlying genetic causes of diseases like sickle cell, thalassemia, and bone marrow failure syndromes. It works by introducing healthy genes to replace or modify ones that don’t work as they should. Our program:

• Offers access to clinical trial treatments often years before they become widely available.
• Provides therapies that can offer long-term benefits from a single dose.
• Uses advanced methods to precisely deliver genes to targeted cells.
• Combines gene therapy with other treatments for the best results.

Other Advanced Cellular Therapies

We stay at the forefront of cellular therapy development. We offer different therapies, such as: 

• Donor lymphocyte infusions (DLI). Targeted immune cell therapy to prevent or treat disease relapse after stem cell transplant. We use precise cell selection methods to maximize effectiveness while minimizing side effects.
• Mesenchymal stem cell therapy. Specialized cells that can help repair tissues and reduce inflammation. Often used to treat graft-versus-host disease and promote healing after transplant.
• Natural killer (NK) cell therapy. Enhanced immune cells that can fight cancer directly. Our program offers both standard and experimental NK cell treatments through clinical trials.
• Virus-specific T cell therapy. Custom-made immune cells that target specific viral infections, important for patients after transplant. We're one of few pediatric centers offering this specialized treatment.