Severe Combined Immunodeficiency (SCID) Treatment

Also called: bubble boy disease, bubble baby disease 

SCID symptoms

SCID causes

SCID is caused by a change (mutation) in 1 or more genes that are involved in the immune system. 

The most common type of SCID is X-linked severe combined immunodeficiency disease (SCID-Xl). It usually occurs in boys because the mutated gene is on the X chromosome. 

SCID is a genetic condition. When a child has SCID, parents should talk to a genetic counselor about the chances of future children having the same condition.

SCID diagnosis

Screening is the most common way to find out if a child has SCID. Newborn infants are screened for SCID at birth. For children who have not been screened, blood tests can be done to confirm SCID.

SCID treatment

Bone marrow transplant

The most effective treatment for SCID is bone marrow transplant (also known as a stem cell transplant). In this treatment, a child with SCID receives healthy stem cells from a matched donor. This donor is usually a healthy brother or sister. 

The new cells rebuild the immune system of the child with SCID. It is possible for children who receive this type of transplant to be cured. Unfortunately, less than 1 in 4 children with SCID have a matched donor.

Without a matched donor, doctors may use stem cells from an unrelated donor or parent. Children who receive these cells do not do as well as those with fully matched donors. About 1 in 3 people treated with a transplant from partially matched donors will develop immune system problem years later.

A bone marrow transplant has the best chance of working when it occurs in the first few months of a child’s life. That is why an early SCID diagnosis is important. 

Gene therapy

Research at St. Jude offers better treatment options for children with X-linked severe combined immunodeficiency disease (SCID-Xl).  The St. Jude research study called LVXSCID-ND treats SCID-X1 with a new method called gene therapy. In this process, bone marrow stem cells are removed from the child. A corrected (normal) copy of the abnormal gene is added to those bone marrow stem cells. These cells are then transplanted into the child’s body.

Without treatment, children with SCID usually die from infections within the first 2 years of life. Early bone marrow transplant, frequent follow-ups, and prompt treatment for infections increase survival rate.

SCID clinical trials

St. Jude offers clinical trials and research studies for children, teens, and young adults with SCID. Learn more about clinical research at St. Jude.

SCID care at St. Jude

• St. Jude has one of the leading gene therapy programs in the country. They have expertise producing and using gene therapy vectors. These vectors are used to treat blood and immune cell diseases.
• A gene therapy developed at St. Jude has helped patients develop a full complement of immune cells. These cells include T cells, B cells, and NK cells.
• The St. Jude Bone Marrow Transplantation and Cellular Therapy Department is one of the largest pediatric programs of its kind in the world.
• The St. Jude Transplant Program is approved by the Children's Oncology Group (COG), the National Marrow Donor Program (NMDP), the Pediatric Blood and Marrow Transplant Consortium, and the Center for International Blood and Marrow Transplant Research (CIBMTR). The program is certified by the Foundation for Accreditation of Cellular Therapy (FACT).
• St. Jude is part of the federally funded Primary Immune Deficiency Treatment Consortium. This group works to improve treatments for patients with SCID and other inherited immune disorders.
• Doctors in the St. Jude Transplant Program work closely with scientists in the lab to quickly move new discoveries to patient care. This team conducts clinical trials to study new discoveries.
• The Transplant Unit at St. Jude is dedicated to the care of patients receiving bone marrow transplants, stem cell transplants, or cellular therapy. The transplant team includes doctors and nurse practitioners. A pediatric immunologist works closely with the transplant team. They have extensive training and experience in this lifesaving treatment for SCID. The team also includes registered nurses, a pharmacist, clinical dietitian, child life specialist, and social worker.
• Transplant Unit nurses have specific training in the care of patients with SCID.
• Each patient room in the St. Jude Transplant Unit has an adjoining parent room. That room has a bed, bath, TV, and phone. Parents can stay with their child throughout the transplant process.