Research

Dr. Jim Hendricks, president of Seattle Children’s Research Institute.

Ranked as one of the top pediatric research centers in the U.S., Seattle Children’s Research Institute has accomplished so much in its 11-year history, and there is much to look forward to in 2018. Here, Dr. Jim Hendricks, president of Seattle Children’s Research Institute shares with On the Pulse what’s in store for the year ahead.  

1. Cancer immunotherapy

Seattle Children’s continued immunotherapy work is going to be very exciting this year.

In 2017, Seattle Children’s opened three new clinical trials offering innovative chimeric antigen receptor (CAR) T-cell immunotherapies for children and young adults with relapsed or refractory acute lymphoblastic leukemia. Currently, there are four Pediatric Leukemia Adoptive Therapy clinical trials underway at Seattle Children’s (PLAT-02, PLAT-03, PLAT-04 and PLAT-05), with others expected to open this year. Read full post »

Audrey, pictured here with her cello before Kawasaki disease caused a large aneurysm to form in her heart.

Whether she’s performing in her school’s jazz band, teaching cello, painting or working as a YMCA counselor, Audrey Wright, 16, seems to do it all. She especially doesn’t let an aneurysm in her heart that developed as a result of Kawasaki disease get in the way of being a busy teenager and her dreams of majoring in studio art.

Audrey was diagnosed with Kawasaki disease, a serious illness that causes inflammation of blood vessels throughout the body, after coming down with what she and everyone else thought was a really bad viral infection. Despite visiting her pediatrician four times in six days, it wasn’t until her blood pressure dipped dangerously low that she was transported to Seattle Children’s. There, doctors in the Pediatric Intensive Care Unit began to unravel the mystery of what was causing her illness.

“Once they got the report back, all of the pediatricians she had seen before coming to the hospital couldn’t believe it was Kawasaki disease,” Karen Wright, Audrey’s mom, said. “They were pretty shocked because she’s not the typical patient.” Read full post »

Ashton Leeds, 8, was treated with larotrectinib at Seattle Children’s for thyroid cancer that had spread to his lungs and lymph nodes.

Dr. Doug Hawkins, division chief of Hematology and Oncology at Seattle Children’s, remembers matching one of the first pediatric cancer patients to an experimental drug that targets a specific set of genetic alterations associated with soft tissue tumors. The drug, larotrectinib, is designed to selectively stop the resulting abnormal tropomyosin receptor kinase (TRK) fusion proteins from promoting cancer cell growth.

“I was so excited to share the test results with the family and present them with the option of enrolling in a clinical trial for this new medication,” Hawkins said. “At the time, I had a pretty good inkling the drug was going to work, but there was very limited evidence of its effectiveness in children. It’s incredibly special that families were willing to take a chance on this drug early on.”

Today, the promising evidence in support of larotrectinib is building. A paper published in The New England Journal of Medicine documents the drug’s effectiveness in treating TRK fusion-positive cancers regardless of patient age or tumor type. The paper includes data from 55 patients, ages 4 months to 76 years and representing 17 different TRK fusion-positive tumor types, treated with larotrectinib. Overall, 75% of patients responded to the treatment and at one year, 71% experienced no disease progression since starting treatment. Read full post »

Auren Satake, 17 months, was born with a congenital heart defect known as hypoplastic left heart syndrome.

Rachael Satake holds a 3D-printed replica of her son’s heart condition in her hands during a recent appointment at Seattle Children’s Heart Center. For the first time since learning about the defect midway through pregnancy, she clearly sees how the surgeries he has undergone are helping his heart work despite having only one ventricle.

Her son, Auren, has a serious congenital heart defect called hypoplastic left heart syndrome (HLHS), which means he was born missing the left ventricle of his heart. His right ventricle works double time to supply blood to both his lungs and the rest of his body. Read full post »

New research suggests inhibiting one group of neurons’ activity may prove to be a highly effective treatment for reducing relapse in recovering addicts.

A new study published by researchers from Seattle Children’s Research Institute reveals how neurons in the brain fuel drug-seeking behavior following compulsive drug use. Their findings, published online in Addiction Biology, suggest inhibiting one group of neurons’ activity may prove to be a highly effective treatment for reducing relapse in recovering addicts.

While the science of addiction is beginning to show how pathological drug use causes the brain’s “go” pathway to become overactive, little is known about what renders some individuals vulnerable to developing addiction and what protects others against it. There are also few effective treatments available to people who develop a drug addiction, or the approximately 90% of individuals who relapse following addiction treatment.

Dr. Susan Ferguson, a principal investigator in the Center for Integrative Brain Research at Seattle Children’s and senior author on the study, describes how her team used an experimental approach called chemogenetic inhibition to probe the relationship between brain activity and behavior in drug addiction. Read full post »

From left to right: Puget Sound Skills Center BioMedical Research and Global Health program students Maryan Farah, Samantha Johnson and Lul Abdinoor. Offered in collaboration with Seattle Children’s Research Institute, the course is the first-of-its kind at a Washington state Career and Technical Education school.

On the day On the Pulse visited the BioMedical Research and Global Health program at Highline Public Schools’ Puget Sound Skills Center (PSSC), the students were preparing to extract DNA from plant specimens in order to learn about a process used by scientists for studying DNA.

Instructor, Dr. Noelle Machnicki, reviewed the protocol, including a detailed description of lysis – a process the students would be using to break open the cells – and then sent them to their benches to get started.

Machnicki, a biologist with a doctorate degree, skilled educator and a member of the Science Education Department at Seattle Children’s Research Institute, was immediately drawn to the opportunity to teach the first-of-its-kind yearlong program offered in a partnership between Seattle Children’s and the PSSC.

“The program intends to create a strong foundation in biological sciences for high school juniors and seniors through extensive hands-on laboratory experience and other educational and leadership opportunities,” said Machnicki. “It provides research training beyond what a student would get in a typical high school science class.” Read full post »

As the countdown to 2018 begins, we can’t help but look back on all of the amazing stories from Seattle Children’s that inspired readers in 2017. With over 100 stories of hope, care and cures posted on our blog this year, here are the top seven most-read posts of 2017.

1. Novel Diet Therapy Helps Children With Crohn’s Disease and Ulcerative Colitis Reach Remission

Adelynne, with her mom here, was diagnosed with Crohn’s when she was 8 years old. With the help of a special diet, Adelynne has been in clinical remission for more than two years.

A first-of-its-kind-study led by Dr. David Suskind, a gastroenterologist at Seattle Children’s, published in the Journal of Clinical Gastroenterology, found a special diet called the specific carbohydrate diet (SCD) could bring pediatric patients with active Crohn’s and ulcerative colitis into clinical remission.

The findings support the use of SCD – a nutritionally balanced diet that removes grains, dairy, processed foods and sugars, except for honey – as a sole intervention to treat children with inflammatory bowel disease. Read full post »

Dr. Jane Buckner of the Benaroya Research Institute at Virginia Mason and Dr. David Rawlings at Seattle Children’s Research Institute are leading research to develop an immunotherapy for type 1 diabetes.

Advances in engineering T cells to treat cancer are paving the way for new immunotherapies targeted at autoimmune diseases, including type 1 diabetes. Now, researchers are also investigating therapies that reprogram T cells to “turn down” an immune response, which may hold promise for curing type 1 diabetes, as well as a number of diseases where overactive T cells attack a person’s healthy cells and organs.

“Instead of stimulating the immune system to seek and destroy cancer cells, treating autoimmune conditions will require programming a patient’s own T cells to tell rogue immune cells to calm down,” said Dr. David Rawlings, director of the Center for Immunity and Immunotherapies at Seattle Children’s Research Institute and chief of the Division of Immunology at Seattle Children’s Hospital.

Harnessing gene-editing techniques pioneered by Seattle Children’s, Rawlings and colleagues have already made headway in equipping T cells with the instructions needed to potentially reverse type 1 diabetes. In a new $2 million research project funded by The Leona M. and Harry B. Helmsley Charitable Trust, researchers will leverage these recent successes using this new form of T-cell immunotherapy into first-in-human clinical trials. Read full post »

Primary human B cells could offer the next promising cell therapy. Credit: Human B Lymphocyte by NIAID (CC by 2.0)

Scientists at Seattle Children’s Research Institute have unlocked the ability to engineer B cells, uncovering a potential new cell therapy that could someday prevent and cure disease.

In a paper published in Molecular Therapy, the research team describes how they genetically reprogrammed primary human B cells to act as cell factories capable of delivering sustained, high doses of therapeutic proteins. The gene editing techniques used reprogrammed B plasma cells to secrete a protein that could treat patients with hemophilia B.

Dr. Richard James, a principal investigator in the Center for Immunity and Immunotherapies at Seattle Children’s Research Institute and an author on the paper, discusses the significance of their discovery.   Read full post »

Genetic testing helped diagnose Nolan Wood, 3, with KCNQ3 epilepsy.

Even though Nolan Wood hadn’t experienced a seizure in more than two years, his parents still had questions about their son’s future.

“We wondered if there were others out there that have what Nolan has,” said Emily Wood, Nolan’s mom. “If so, what does their life look like?”

The Woods’ search for answers began when Nolan, 3, was diagnosed with infantile spasms and regression of his motor skills when he was 6 months old. Before receiving seizure medications, Nolan had hundreds of daily subtle, reflex-like seizures. Due to the regression of his motor skills, he had stopped rolling over, smiling and crying. A condition known as cortical visual impairment had also rendered him legally blind. Read full post »