Autoimmune Diseases Pediatric Research and Clinical Trials

The pace at which our understanding of the immune system—and, along with it, autoimmune diseases—has increased dramatically in the past few decades. So, too, has the pace of diagnostics and treatment.

Here at Boston Children’s Hospital, the Samara Jan Turkel Clinical Center for Pediatric Autoimmune Diseases is helping to pioneer some of the most promising new therapies for these illnesses, including the use of biologics: protein-based drugs made from living cells cultured in a lab. 

Our physicians have participated in a number of multicenter studies that focus on biologics, including: 

  • Rituximab in Myositis (RIM), which examined rituximab—a man-made antibody initially used to treat certain types of cancer—as a therapy for juvenile dermatomyositis, an autoimmune disease marked by muscle weakness and skin rash that affects an estimated 3,000 to 5,000 children in the U.S.
  • Trial of Early Aggressive Therapy (TREAT) in JIA, which involved etanercept (a.k.a. Enbrel), one of a key group of biologics called tumor necrosis factor (TNF) blockers that counteract high levels of inflammatory proteins in the body. In this study, doctors looked at both etanercept and the more well-established drug methotrexate as early treatments for juvenile idiopathic arthritis, an autoimmune disease that affects about 50,000 children in the U.S.

Learn more about research at Boston Children's.

Clinical trials

There are many ways in which your child might benefit from Boston Children’s Hospital’s medical research program. Boston Children’s doctors and scientists have made many breakthrough discoveries about diseases like polio and leukemia; our ongoing research continues to push the boundaries of the way pediatric medicine is practiced. 

It’s possible that your child will be eligible to participate in one of Boston Children’s current clinical trials. These studies are useful for a multitude of reasons: Some trials are designed to evaluate the effectiveness of a particular drug, treatment or therapy on a specific disease; others help doctors to better understand how and why certain conditions occur. At any given time, Boston Children’s has hundreds of clinical trials under way. 

Alternately, your child’s doctor might ask you to participate in a registry, a list of patients with JIA that is matched to an archive of biospecimens (such as serum and DNA) to serve as a resource for research into this disease.

And participation in any clinical trial or registry is completely voluntary: We’ll take care to fully explain all elements of the treatment plan prior to the start of the trial, and you may remove your child from the medical study at any time.