Pediatric stem cell transplant and cellular therapy: Harnessing the healing power of stem cells

Harvesting stem cells from your child’s own bones or blood.

Harvesting stem cells from the blood or bone marrow of a child's biological brother or sister. There is a 25% chance a sibling's HLA — a protein found on most cells — will match that of the child receiving the transplant.

Harvesting stem cells from the blood or bone marrow of a genetically (HLA) matched, non-family member adult. We find these donors through international registries.

Harvesting stem cells from the blood of a biological parent. Parents are always a genetic half-match for their children.

Harvesting stem cells from a newborn baby's umbilical cord blood. These can come from stored cord blood from a related or unrelated donor.

CAR T cells are a therapy that uses a type of immune cells (T cells) that are modified to specifically attack certain types of cancers. These are usually made from a patient’s own T cells. These are the only FDA-approved type of cell therapy and are offered at American Family Children’s Hospital for patients with specific types of blood cancer. Your child may also qualify for clinical trials testing CAR T cells that have not been FDA approved.

Researchers at UW Health | Carbone Cancer Center are studying cell therapies using other types of immune cells including NK cells and macrophages, or T-cell receptor (TCR) modified T cells. Your child may qualify for clinical trials involving one of these types of cell therapies.

Hematologic cancers form in the bone marrow or the immune system. They include:

• Acute lymphoblastic leukemia

• Acute myeloid leukemia

• Chronic myeloid leukemia

• Hodgkin's lymphoma

• Non-Hodgkin's lymphoma

Solid tumors are abnormal growths in the body that don't contain cysts or liquid. Types include:

• Brain tumor

• Ewing sarcoma

• Neuroblastoma

• Rhabdomyosarcoma

Bone marrow failure syndromes are a rare group of conditions. They prevent the body from making enough blood. Types include:

• Aplastic anemia

• Fanconi anemia

Hemoglobinopathies refer to conditions that affect the production of hemoglobin. Hemoglobin is a protein in red blood cells that transports oxygen in the body. Hemoglobinopathies include:

• Sickle cell anemia

• Thalassemia

Primary immune deficiencies are genetic conditions. They affect the body's ability to fight off infections. Some of these include:

• Chronic granulomatous disease

• Hemophagocytic lymphohistiocytosis

• Severe combined immunodeficiency syndrome

• Wiskott-Aldrich syndrome

Sometimes, cancer doesn't respond to treatments the way we hope it will. For certain cancers, we offer clinical trials that test promising new stem cell transplant therapies. We are the only transplant center in the country to offer some of these treatments. Our research programs aim to improve cancer treatments for all children.

In some cases, children donate their own stem cells. We may collect the cells from your child's blood using an apheresis. In other cases, a donor will provide the stem cells. We store the cells until we're ready to use them.

We will use chemotherapy or radiation to destroy your child's existing, unhealthy bone marrow. This procedure occurs several days before the transplant.

We infuse your child's new stem cells through a tube placed in a large vein, similar to a blood transfusion. Usually, this process is painless and takes minutes to hours.

It will take a few weeks for the stem cells to reach your child's bone marrow and create new blood cells. During this time, we watch your child carefully. We take special care to protect their health and reduce their risk of infection. We will also monitor your child on an ongoing basis for complications.

Many cellular therapies are made from a patient’s own immune cells. These are collected using a process called apheresis. This product is then used to modify the cells to specifically attack the disease.

Usually before cell therapies are administered, chemotherapy medications are given to “make space” for the modified immune cells to be accepted by the patient. This typically takes about a week and may be done outpatient or inpatient.

We infuse the modified immune cells through a tube placed in a large vein, similar to a blood transfusion. Usually this process is painless and takes less than an hour.

After cell therapies, patients are monitored for side effects that can include fevers and other signs of inflammation. Your child will be monitored closely during the first month after cell therapy, with frequent labs and visits, or with an inpatient stay.