All Articles in the Category ‘Research’

BrainChild-04 Study Is Enrolling Patients With Brain and Spinal Cord Tumors

About BrainChild-04

In May 2023 Seattle Children’s began enrolling patients in a new study of a novel treatment for certain types of brain and spinal cord tumors in children and young adults. These include diffuse intrinsic pontine glioma (DIPG),  diffuse midline glioma (DMG) and other recurrent or refractory central nervous system tumors that carry a poor prognosis with limited survival.

The BrainChild-04 study is investigating Seattle Children’s Therapeutics’ first CAR T-cell product that targets four antigens simultaneously: B7-H3, EGFR806, HER2 and IL13-zetakine.  This is the first known CAR T-cell product in the world to target four antigens at the same time by delivering CAR T cells directly to the brain.

A long-standing obstacle to successfully treating brain tumors has been the blood-brain barrier, which stops therapeutic medications from reaching their intended targets.

Up to 72 participants between the ages of 1 and 26 are expected to enroll in this phase 1 clinical trial. Read full post »

How Genetic Epilepsy Research Is Offering Pediatric Patients New Options: A Q&A With Drs. Ghayda Mirzaa and Jay Hauptman

In Part 1 of our Epilepsy Q&A, Dr. Jay Hauptman discussed advances in neurosurgery and the many new options available for children with intractable epilepsy. In Part 2, we hear from Dr. Hauptman and Dr. Ghayda Mirzaa about the genetics of epilepsy and how research is leading to new nonsurgical treatment options.

Dr. Mirzaa is a Seattle Children’s clinical and molecular geneticist at the Center for Integrative Brain Research and Seattle Children’s Epilepsy Program. Dr. Hauptman is a neurosurgeon with Seattle Children’s Epilepsy Program.


Q: What do we know about the role of genetics in epilepsy?

DR. HAUPTMAN: We know genetics contribute significantly to many types of epilepsy. Roughly a third of cases have an underlying genetic cause. We are still learning how and why genetic mutations cause epilepsy in kids.


Q: What is the focus of recent research?

DR. MIRZAA: We’ve made great progress in the last few years, at Seattle Children’s Center for Integrative Brain Research and elsewhere. Our Seattle Children’s team has traced focal cortical dysplasia, which is among the leading causes of intractable epilepsy, to mutations in a family of genes that control important pathways, such as the mammalian target of rapamycin (mTOR) pathway. Now we are exploring if drugs that are known to inhibit this pathway, which are already being used or tested to treat cancer, can be effective for epilepsy too.

Read full post »

Seattle Children’s Research Featured in Worldwide Study on COVID-19 Vaccine Safety in Young Children

Written by Casey Egan and Empress Rivera-Ruiz, On the Pulse, February 16, 2023.

New findings from Seattle Children’s Research Institute’s Center for Clinical and Translational Research reveal that a three-dose primary series of Pfizer’s COVID-19 vaccine was safe and effective in children 6 months to 4 years of age, even when a new variant (Omicron) was circulating.

The findings were released on Feb. 16 in the New England Journal of Medicine by an international team conducting the study, including Seattle Children’s lead study investigator, Dr. Janet Englund.

“No data on a three-dose series of Pfizer mRNA vaccine has been previously published in young children,” said Dr. Englund.

Dr. Englund also noted that the findings demonstrate that three doses of the Pfizer vaccine offer better protection than two doses against the Omicron variant in this age group.

Studies of bivalent booster doses of the Pfizer mRNA vaccine are now ongoing at Seattle Children’s. This research continues under Seattle Children’s Pediatric Infectious Disease Research Group, directed by Dr. Englund.

Learn more here: COVID-19 Vaccine Found Safe, Effective for Youngest Children (