Seattle Children’s Therapeutics, a venture at Seattle Children’s, bringing cutting edge, curative technologies and therapies to defeat pediatric cancer and other diseases that impact children, today announced a collaboration with Cellevolve Bio, a development and commercialization company focused on cell therapies, aimed at developing and commercializing a suite of novel multiplex chimeric antigen receptors (CARs) for the treatment of pediatric central nervous system (CNS) malignancies.
Under the exclusive agreement, Seattle Children’s Therapeutics will conduct early-stage and pre-clinical discovery, and Phase 1 clinical trial development. Cellevolve will lead Phase 2 and subsequent clinical development with key Seattle Children’s Therapeutics involvement. Read full post »
The low-cost bCPAP device combines room air with oxygen and delivers it to the baby’s nose. The tubing carrying the oxygen ends submerged in water, which creates the pressures in the system and makes bubbles when the air comes out. The bubbles create a vibration that helps to keep the lungs open and working better. (Photo: PATH)
Each year, hundreds of thousands of babies born prematurely in low- and middle-income countries die because medical facilities there cannot afford the equipment that could help babies survive those crucial first few weeks after birth.
Many of these deaths are caused by respiratory distress syndrome.
In sub-Saharan Africa alone, some 6 million preterm babies are born every year with immature lungs. Their lungs aren’t fully developed, and they have trouble staying inflated, so they collapse. While medical institutions in high-income countries have bubble continuous positive airway pressure machines to help them breathe, those bCPAP units cost thousands of dollars—making them prohibitively expensive for many low-income nations. Of those 6 million babies, 800,000 of them are born at mid-level facilities that require bCPAP devices but likely don’t have them.
The bCPAP devices keep the lungs from deflating and also deliver blended oxygen into them—a critical step because breathing 100% oxygen can cause blindness in premature babies.
Medical providers in some low-resource countries use improvised bCPAP kits assembled from parts they already have in their clinics and use them to help preterm babies survive. However, these kits do not have the ability to provide blended oxygen for babies.
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Amanda and her daughter, Amelia, have both been treated at Seattle Children’s for Alagille syndrome.
When Amanda Thorlacius found out she was pregnant with a little girl, she was overjoyed. But she wondered if her daughter would inherit the same genetic condition that robbed her of a normal childhood.
“Give me all the diseases in the world, but don’t give Alagille syndrome to my children,” Amanda said.
Alagille syndrome (ALGS) is a rare, inherited condition in which children may have too few bile ducts in the liver. This causes problems with the way bile moves and makes it hard for the body to remove toxins.
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Dr. Burt Yaszay is the new chief of Orthopedics and Sports Medicine at Seattle Children’s.
Seattle Children’s is excited to welcome Dr. Burt Yaszay as the new chief of Orthopedics and Sports Medicine at Seattle Children’s. Yaszay comes to Seattle Children’s with a bright vision for the future as well as a deep respect for the roots in which Seattle Children’s was founded.
We sat down with Yaszay to learn more about his extensive expertise and vision for the program.
Yaszay earned his medical degree at Stanford University School of Medicine and did his residency in general and orthopedic surgery at the University of Washington and a fellowship at NYU Hospital for Joint Diseases. Yaszay most recently spent 14 years at Rady Children’s Hospital in San Diego, which is nationally recognized as one of the best programs in the country.
He is excited to bring his expertise to Seattle Children’s and foster an environment of innovation and collaboration.
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Dr. Nicolas Fernandez, pediatric urologist at Seattle Children’s
Hypospadias (pronounced hype-oh-spay-dee-us) is a birth defect where the opening of the urethra, the tube that carries urine from the bladder to the outside of the body, is not located at the tip of the penis. Depending on the severity of the condition, it can affect the penile function and one’s self-perception. It is a common condition that can be treated with surgery in some cases; however, a decision to proceed with surgery needs to be carefully thought through and discussed among the provider, parents and patients.
The current standard treatment for hypospadias starts with the provider defining the severity of the condition. The severity is based on the location of the urethral meatus, the external opening of the urethra, and the penile curvature. Based on the severity, some patients may be offered surgery as a next step. Surgery involves using local tissue from one part of the patient’s body to relocate the urethral opening to the correct position at the tip of the penis. To date, assessment of the tissue is subjective, and therefore, it is hard for surgeons to repeat the process moving forward.
Dr. Nicolas Fernandez, a pediatric urologist at Seattle Children’s and surgeon scientist at the University of Washington, is working on a research project that aims to reduce this subjectivity and explore new approaches to better assess structural and genetic components of the tissue used for surgery. He is doing so by proposing a novel approach to evaluate hypospadias.
By using technology to detect small genetic differences that can lead to big changes in an individual’s physical characteristics and by applying artificial intelligence on patients with hypospadias, providers can improve clustering of individuals with similar characteristics to ensure a more accurate prediction for treatment and surgery for this condition. This ultimately leads to better, more impactful outcomes for the patient and family.
Dr. Fernandez spoke with On the Pulse about his research.
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When the worst pandemic of the century struck, a group of nine Seattle Children’s Research Institute’s scientists teamed up with researchers from Rockefeller University to innovate powerful tools for diagnosing and treating a virus that has claimed over 5 million lives.
In a recent study published in E-life, scientists from Seattle Children’s Aitchison, Sather, Myler and Debley labs, in collaboration with Rockefeller University’s Chait, Rout and Bieniasz labs, demonstrate how a unique group of antibodies, known as nanobodies, could become an exceptional resource for superior COVID-19 protective and therapeutic interventions, as well as bring hope for effective treatment of diseases most commonly found in low-resource countries.
Antibodies are produced by our bodies as a protective response to bacteria, viruses and foreign substances in our blood. Nanobodies are fragments of antibodies produced by llamas and other members of the camelid family. Nanobodies are ten times smaller than human antibodies, they are remarkably robust and they are readily “humanized” for use in diagnostic and therapeutic treatments. Nanobodies bind to viral-producing antigens in places not accessible to human antibodies and are considerably more thermo-resistant. Read full post »
For the past 15 years, Seattle Children’s Research Division has been at the forefront of breakthrough innovations. From new drugs to treat cystic fibrosis, to first-in-the-nation use of laser ablation for epilepsy and brain tumors to remove unwanted cells, the research division is advancing our mission to provide hope, care and cures to help every child live the healthiest and most fulfilling life possible.
Here, we take a look at some of the achievements of the past decade-and-a-half.
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Recent breakthroughs in cell and gene therapy research within Seattle Children’s Research Division, which includes both Seattle Children’s Research Institute and Seattle Children’s Therapeutics, has contributed to the creation of new technologies, new companies and – researchers hope –will lead to a range of new treatments and cures for pediatric diseases down the line.
Cell and gene therapy has become an increasingly popular form of therapeutic treatment and research in recent years.
“It has really become a third pillar of therapeutic treatment behind small molecules and monoclonal antibodies – cell therapy is here to stay and it’s growing exponentially as more capabilities are developed,” said Dr. Brian Phillips, director of the Intellectual Property Core at Seattle Children’s, which aims to help Seattle Children’s researchers and clinicians commercialize their intellectual property. Read full post »
The phone call came at 2 a.m. It was a neonatologist calling about Kimberly Aldinger and Scott Houghtaling’s son, Grayson. Kimberly had given birth to premature twins a month earlier and both babies were in the neonatal intensive care unit (NICU) at Swedish Hospital. The new parents had returned home to get a much-needed night of sleep when the doctor left a message on their voicemail.
Kimberly Aldinger and Scott Houghtaling’s son, Grayson (pictured here), began having seizures when he was just 24 days old. Now, his parents are using their scientific expertise to try to find the cause of his epilepsy.
“I’m really worried about Grayson,” the doctor said. “He’s having a massive seizure. You need to come down here.”
Scott immediately feared the worst. “I thought, the only reason they’d call in the middle of the night was if they were preparing for the worst outcome — Grayson not surviving.”
Thankfully, the medical team was able to stop Grayson’s seizure that night, but it was just the beginning of Kimberly and Scott’s journey to understand the severity of their son’s brain damage and how it would shape all their lives.
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An innovative clinical trial led by Dr. Nicholas Vitanza, a neuro-oncologist at Seattle Children’s, shows promise that delivering cancer-fighting chimeric antigen receptor (CAR) T cells directly to the brain for children and young adults with recurrent or refractory brain and central nervous system (CNS) tumors may be feasible and tolerable.
The results, published today in Nature Medicine, are the initial findings from Seattle Children’s Therapeutics’ BrainChild-01 immunotherapy clinical trial. BrainChild-01 is the first of three such trials seeking to comprehensively target all types of pediatric brain and spinal cord tumors.
Seattle Children’s Therapeutics is a unit in the research division at Seattle Children’s and is taking promising CAR T cell immunotherapies forward to the first clinical trials of their kind for children. As a novel non-profit therapeutic development enterprise, it is devoted to envisioning and testing next-generation cell and gene therapies for pediatric diseases, so children have the medicines they deserve.
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