Severe Combined Immunodeficiency (SCID)
Research & Innovation
At Children’s Hospital Boston, our care is informed by our research, and our discoveries in the laboratory strengthen the care we provide at each child's bedside. Children’s scientific research program is one of the largest and most active of any pediatric hospital in the world.
Children’s has now opened an exciting new gene therapy trial for the X-linked form of SCID (known as SCID-X1). The worldwide, multicenter study is sponsored in the United States by David Williams, MD, chief of Hematology/Oncology and director of Translational Research at Children's.
The study is currently open for enrollment, and aims to recruit a total of 20 patients with SCID-X1.
Read the story of one trial participant.
Gene Therapy: Two years and holding!
For the Cáceres family of Argentina, it’s a joyous holiday homecoming. Agustín, who received gene therapy at 5½ months of age, journeyed with his family to Boston for a check-up and got a clean bill of health.
ABOUT CLINICAL TRIALS
Children’s coordinates hundreds of clinical trials at any given time. Clinical trials are studies that may involve:
- evaluating the effectiveness of a new drug therapy
- testing a new diagnostic procedure or device
- examining a new treatment method for a particular condition
- taking a closer look at the causes and progression of specific diseases
Taking part in a clinical trial at Children’s is entirely voluntary. Our team will be sure to fully address any questions you may have, and you may remove your child from the medical study at any time.
|Touching lives from Argentina to Children’s|
|Can you imagine how you’d feel if you couldn’t hold your child? Agustin Caceres’s parents could not come in physical contact with him as a baby because he had Severe Combined Immunodeficiency, better known as “bubble boy disease.”Read how Children’s and the Dana Farber’s Cell Manipulation Core Facility were able to use gene therapy so he could be in touch with his family and the world again, literally and figuratively. Read more about how Agustin was treated through an international gene therapy trial.|