Research and Clinical Studies

Our research in the lab and with patients helps us give your child the very best care. Seattle Children's doctors lead research to improve treatment, cure rates and quality of life for children with conditions that affect their brain, skull or spinal cord.

Our doctors and researchers from many specialties work together to answer basic questions and make new discoveries. We build on those findings to understand the causes of neurological conditions and to develop new ways to diagnose and treat them. Our patients can take part in research studies (clinical trials) of promising new treatments. 

Developing and Testing Novel Therapies

Immunotherapy for brain cancer

Seattle Children’s has one of the largest pediatric cellular immunotherapy programs in the world. This experimental treatment “reprograms” a patient’s immune system T cells so they hunt down and destroy cancer cells. The changed cells are called CAR T cells.

The BrainChild group of phase 1 clinical trials is testing CAR T-cell immunotherapies in children and young adults with certain relapsed or difficult-to-treat brain tumors. BrainChild-04 delivers CAR T cells directly to the brain. BrainChild-04 is open to patients with diffuse intrinsic pontine glioma (DIPG), among other childhood brain or spinal cord tumors. At this time there is no cure for DIPG.

Making immunotherapy as safe as possible

The Gust Lab studies how cancer treatment and inflammation affect the developing brain. Seattle Children’s is a leader in pediatric cancer immunotherapy, and the lab’s mission is to develop ways to deliver this therapy as safely as possible. Current research by Juliane Gust, MD, PhD, and her team includes:

• Investigating if biomarkers in blood, cerebrospinal fluid or on brain imaging can predict the risk of nervous system damage (neurotoxicity) and measure brain injury in children receiving cancer immunotherapy. Read more about clinical biomarkers of CAR T-cell neurotoxicity.
• Using a mouse model to study how CAR T-cell therapy for leukemia and lymphoma affects the network of tiny blood vessels in the brain. Read about the mechanism of CAR T-cell neurotoxicity research project.
• Studying how cancer-fighting CAR T cells can efficiently travel to tumors in the brain.   

 The Gust Lab is part of the Norcliffe Foundation Center for Integrative Brain Research.

Clinical trials of new treatment options

In addition to therapies developed at Seattle Children’s, our patients have access to clinical trials of other new treatment options that many hospitals do not offer. This can be especially helpful if your child’s disorder is not well controlled with standard medicines or surgeries.

These are just a few examples of clinical trials that aim to improve care and quality of life for children with neurological conditions.

• Dr. Stephanie Randle is looking at 3 new approaches to treat children with tuberous sclerosis complex (TSC). The approaches include MRI brain imaging and 2 different medicines that control seizures.
• Dr. Aimee Sato is studying how to lessen the need for surgery in patients with neurofibromatosis by using targeted therapies to block a protein that helps tumors grow.
• As a founding member of the Synostosis Registry Group (SynRG), we work on multicenter clinical trials for children with craniosynostosis. Dr. Amy Lee is co-principal investigator at Seattle Children’s. 
• In the Hydrocephalus Clinical Research Network (HCRN), we partner with other leading children’s hospitals on clinical trials to improve hydrocephalus care for children. Dr. Samuel Browd is senior researcher at Seattle Children's.

Improving Patient Care and Outcomes

TMS mapping to protect critical brain areas

Transcranial magnetic stimulation (TMS) activates brain nerve cells (neurons) in a noninvasive way. We use TMS to map the brain areas that control movement and speech. Our research helps us improve how we use TMS to map the brain. This helps us protect critical areas as we plan and carry out surgery.

Researchers also use TMS mapping to identify and better understand the relationship between brain structure and function. TMS passes a pulse of electric current through a coil placed over a patient’s head. This generates a rapidly changing magnetic pulse that activates neurons.

Studying brain injury to improve neurocritical care

Dr. Mark Wainwright and his team study brain injury and how to improve neurocritical care for children. This includes research on:

• Brain injuries in children on extracorporeal membrane oxygenation (ECMO)
• Stroke
• Seizures that last for a long time or happen in clusters (status epilepticus)
• Treatment of seizures in newborns
• Long-term outcomes after COVID-19
• Uses of artificial intelligence (AI) in critical care

Safer brain surgery in hard-to-reach areas

Tumors or lesions around the base of the skull (called the cranial base or skull base) can be life-threatening and hard to reach. In the past, surgeons could not safely access the skull base due to the risk to important nerves, blood vessels and brain regions. Advanced, minimally invasive procedures now enable neurosurgeons to reach some tumors and lesions of the skull base. They use smaller incisions or go through the nostrils, without needing to cut the head or neck. 

Dr. Jake Ruzevick performs these complex surgeries and studies the outcomes of surgery via standard craniotomy and minimally invasive endoscopic approaches. Understanding how well each surgical approach works helps our doctors choose the safest, most effective surgery for your child.

Neurosurgeons and engineers across Seattle Children’s and the University of Washington are working to develop even safer, more precise neurosurgical methods. These collaborations bring together experts in oncology, endocrinology, ophthalmology, head and neck surgery and engineering. 

Enhancing recovery after surgery

Dr. Hannah Goldstein and colleagues work to improve care before, during and after surgery. Her quality improvement team looks at data, others' research and how our patients do after surgery. The goal is to make your child's recovery as easy as possible.  

The team also studies access to care and works to identify and better understand the health disparities that affect quality of life. 

Insights Into Genetic Disorders of the Nervous System

Pinpointing genes that cause relentless seizures

Dr. Ghayda Mirzaa and her team offer comprehensive genetic testing for children who have genetic disorders that affect the nervous system. These disorders can cause brain malformations, epilepsy and developmental delays. By testing brain tissue removed from children undergoing surgery for intractable focal seizures, the Mirzaa Labb has pinpointed certain genes. These genes contribute to brain disorders that are a leading cause of hard-to-treat epilepsy in children. The disorders include hemimegalencephaly and focal cortical dysplasia. 

The team traced the disorders to mutations in several genes in an important network of cells (called the mTOR pathway). Our doctors are studying if drugs that inhibit the mTOR pathway will treat epilepsy in patients who don’t respond to standard therapies or surgery. 

Watch Dr. Mirzaa describe how her research leads to new therapy options. (Video 1:54)

The Mirzaa Lab is part of the Norcliffe Foundation Center for Integrative Brain Research.

Linking genetic changes to disease pathways

Kimberly Aldinger, PhD, and her team study genetic disorders that cause changes in brain structure, harm cognitive function and lead to epilepsy in children. The researchers use genomic sequencing and advanced computing to learn how changes in genes and cells lead to specific symptoms and diseases.

The team is interested in changes in the cerebellum — the part of the brain that helps coordinate and regulate many functions and processes in the brain and body. Their discoveries may open the door to more effective and targeted therapies for children with rare genetic forms of epilepsy. 

The Aldinger Laboratory is part of the Norcliffe Foundation Center for Integrative Brain Research.

Understanding the mechanisms that drive epilepsy

The Kalume Laboratory studies what causes seizures and other problems in genetic epilepsies that do not respond to treatment. Current research projects in the lab are centered on Dravet syndrome and Leigh syndrome. Dravet syndrome is a severe and life-threatening form of epilepsy. Leigh syndrome is a mitochondrial disease that causes severe seizures and early death. 

Franck Kalume, PhD, and his team use several genetic mouse models and state-of-the-art neuroscience research tools to:

• Learn what causes sudden unexpected death in epilepsy (SUDEP) and find new therapies to prevent these deaths
• Figure out why the ketogenic diet helps reduce seizures that are not controlled by medicines (intractable)
• Understand the underlying causes of disrupted sleep in patients with Dravet and Leigh syndromes
• Develop genetic-based therapies for Dravet and Leigh syndromes

The Kalume Lab is part of the Norcliffe Foundation Center for Integrative Brain Research.

Ways to Help

Donations from people like you help researchers pursue ideas that could lead to lifesaving treatments. Email askus@seattlechildrens.org to learn more. Or you can donate today and designate the area you’d like to support.

Contact Us

If you would like an appointment, ask your child’s primary care provider to refer you. If you have a referral, call 206-987-2016 to make an appointment. 

Providers, see how to refer a patient. 

If you have questions, contact us at 206-987-2016 or 844-935-3467 (toll-free).