Only a small fraction of today’s pediatric treatments were developed for — or tested in — children. This means physicians must give children therapies that were designed for adults. But children’s developing minds and bodies have unique needs and are uniquely vulnerable to side effects.
Our goal is to develop therapies that are tailored to children and teens with autoimmune diseases – therapies that are more effective than current treatments, with fewer side effects.
Our research includes:
Juvenile Diabetes Research
Drs. David Rawlings and Andrew Scharenberg
Diabetes occurs when the body’s immune cells turn against it and attack the pancreas. Drs. David Rawlings and Andrew Scharenberg are using gene editing to engineer regulatory cells that could halt these attacks – a groundbreaking approach that could lead to cures.
Pediatric Multiple Sclerosis Research
Dr. Mohamed Oukka is identifying the mechanisms behind multiple sclerosis (MS) and developing therapies that could stop it from causing disabilities in children.
MS occurs when the body’s immune cells attack the myelin sheath around nerves in the brain and spinal cord. Today’s MS drugs overcome this by dampening immune activity. This reduces MS symptoms but opens the door to potentially fatal brain infections.
Oukka was part of a team at Harvard University that identified the specific T cells, called Th17 cells, that attack the myelin sheath. Now his lab is investigating drugs that suppress Th17 cells while leaving other T cells free to protect the brain.
Watch Oukka discuss how Seattle Children’s research is developing better therapies for children and teens with multiple sclerosis:
Immunotherapy for Multiple Sclerosis
Oukka is also working with Rawlings and Scharenberg to genetically reprogram immune cells to find and destroy Th17 cells. This could spare children with MS from taking medication on a regular basis – the modified cells would do all the work.
Searching for Genes That Cause Multiple Sclerosis
Oukka is working with Dr. Troy Torgerson and others to sequence the DNA of children with MS. Their goal is to identify which genes work together to cause the disease, opening the door to gene therapies that could cure it. The goal is to correct the genes so the immune system behaves normally.
Testing a New Drug for Autoimmune Diseases
Dr. Anne Stevens
Our researchers, including Oukka and Dr. Anne Stevens, are joining forces with Kineta Inc. to pursue new therapies for autoimmune diseases. The collaboration – the Alliance for Children’s Therapeutics – is testing a new drug candidate called dalazatide. It could be a safer, more effective treatment for autoimmune diseases like lupus, multiple sclerosis and juvenile arthritis.
Searching for Genes That Cause Autoimmunity
Regulatory T cells police the immune system and tell other immune cells when to calm down and call off their attacks. Torgerson and his colleagues discovered that a variety of mutations in the gene FOXP3 can stop the body from producing regulatory T cells, triggering a severe autoimmune condition called immune dysregulation polyendocrinopathy enteropathy X-linked (IPEX).
Now Torgerson’s team is sequencing the genes of patients who have IPEX-like symptoms but don’t have FOXP3 mutations. The researchers hope to identify other gene mutations that affect regulatory T cells. This could lead to insights about the fundamental causes of many autoimmune disorders.
Food Allergy Research
Our researchers are investigating links between autoimmunity and food allergies. We are part of the Seattle Food Allergy Consortium (SeaFac), a collaboration that studies what causes food allergies and tests new treatments. These treatments include a patch that delivers a peanut protein into the skin of people with peanut allergies. The goal is to train the immune system to develop a tolerance for peanuts.
SeaFac unites our researchers with investigators from the University of Washington, the Benaroya Research Institute at Virginia Mason, the ASTHMA Inc. Clinical Research Center and the Northwest Asthma and Allergy Center.