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Inside Pediatrics, Pulmonology

Using Mobile Health To Improve Cystic Fibrosis Care

Gabriela Oates, Ph.D., assistant professor pulmonary and sleep medicine at UAB and Children’s of Alabama, is working to develop a mobile app to to help young children, adolescents and their families manage cystic fibrosis.

It’s not easy living with cystic fibrosis (CF). The multitude of medications, therapies and nutritional supplements that children and adolescents with the disease require can be exhausting and overwhelming, leading to high rates of nonadherence, particularly in adolescents. That’s why Gabriela Oates, Ph.D., an assistant professor of pulmonary and sleep medicine in the University of Alabama at Birmingham (UAB) Department of Pediatrics at Children’s of Alabama, is working to create a mobile health application designed to bridge the gap between what is and what should be when it comes to managing the disease.

The application builds on one developed in Sweden, which 65 to 87% of CF families in that country now use. Called Genia, the app is used to track symptoms, activities and aspects of daily care and share them with the clinical team.

In modifying Genia for the U.S., Oates and her team didn’t rely on what they thought it should look like. They turned to the experts, holding five focus groups with adolescents with CF, families of younger children with CF and the clinicians who care for them. The approach is called “health care service coproduction” and its central tenet is getting buy-in from both clinicians and patients. It’s part of the movement away from the paternalistic view of health and medicine in which the doctor alone drives the process.

The focus groups showed differences between what the clinicians wanted the app to do and what the families and patients asked for, something Oates said the team expected. “The clinicians don’t have the lived experience of managing the disease on a daily basis; our patients and families provided that. However, the clinicians told us what’s necessary to make it work in the clinic; capturing just the patient perspective would have led to an app that’s not workable in the clinical setting.”

Among the changes the focus group participants requested and the team implemented were adding a mental health tracker, making other trackers (i.e., nutrition, medication, physical activity) more specific, syncing the app with calendars and customizing the app with avatars, images and colors.

One thing parents wanted that the adolescents didn’t: the ability to see their child’s entries. On this point, the team decided that the account holder gets to determine the privacy setting and each family decides who the account holder is. “This is normal,” said Oates. “Our children are supposed to become independent and take over the management of their own health condition.”

She and her team are currently conducting a pilot study about the impact of the app’s use on clinical outcomes, with results expected later this year. So far, they are getting high praise from physicians, families and patients. For instance, the patients/families love that they can use the app to submit pre-visit reports instead of completing long paper forms in the waiting room. The clinical team, on the other hand, appreciates having a detailed view of patients’ symptoms or struggles before seeing them in clinic; it helps them understand what’s going on in their patients’ lives and better tailor treatment plans. The app also flags potential problems and shares them with the CF care team on a weekly basis, which allows for earlier intervention.

With the help of the Children’s CF care team, Oates hopes to transition Genia from research settings to a standard of care. Recently, the app was translated to Spanish and made available for both Android and Apple devices. This will allow it to reach a larger segment of CF families to help them manage daily care and better integrate their experiences in treatment plans.

Inside Pediatrics, Pulmonology

When It Takes A Village: A Unique Multidisciplinary Team For Aerodigestive Disease And Complex Conditions

Children’s of Alabama pediatric pulmonologist Tom Harris, M.D., left, and Reed Dimmitt, M.D., MSPH, right, director of the UAB Division of Pediatric Gastroenterology, are among the multidisciplinary team members who make up Children’s Aerodigestive Clinic.

Break down the word “aerodigestive” and you can understand why the condition is so complex and intertwined. Children with aerodigestive conditions often have overlapping disorders involving the upper airway, larynx, trachea, esophagus and lungs, all of which originally developed embryonically from a common source.

One patient may have symptoms affecting multiple systems. At most pediatric hospitals, each condition is treated separately; subspecialists typically focus on the organ of their expertise and not the child holistically.

At Children’s of Alabama’s Aerodigestive Clinic, families don’t need to make multiple appointments and repeated trips to the hospital to see a slew of medical professionals. Instead, they can see a multidisciplinary team of pediatric subspecialists during a single clinic visit and receive coordinated care. Children’s Aerodigestive Program is the only one in Alabama and one of just a few around the country.

“We felt there was a big gap in care, and that led to frustrations that maybe we weren’t meeting their needs,” explained clinic coordinator Ashley Chapman of why Children’s started the program. Chapman focuses on improving communication and facilitating access.

The result of better care has improved both patient and provider satisfaction. “Families appreciate that they don’t feel ‘bounced’ around the medical system,” said pediatric gastroenterologist Reed Dimmitt, M.D.

“Our team is a fun, collaborative group,” added pediatric pulmonologist Tom Harris, M.D. “Previously, we each treated the patients in a general clinic but were limited by our individual skill set. This approach allows us to lean on one another’s expertise, asking, ‘How can we improve care by working together?’”

Consider the triple endoscopy (direct laryngoscopy bronchoscopy, flexible bronchoscopy and esophagogastroduodenoscopy) with the ENT surgeon, pulmonologist and gastroenterologist all in the procedure room. Before, patients would need three separate procedures, requiring three times under general anesthesia with separate interpretations. Now, the three physicians meet together with the family post-procedure to discuss findings and deliver the management plan.

“It’s an additive model, but there’s also a synergy that occurs with everyone meeting together, which contributes to better outcomes,” said Dimmitt. “Collaborating with the multidisciplinary team,” he said, “pushes me to think outside the GI box.” The aerodigestive coordinator, speech pathologist and dietician are central to management plans, providing additional non-M.D. layers of coordination, expertise and pragmatic considerations.

The clinic offers in-person as well as telemedicine visits, and is growing fast, with referrals doubling in the past two years. “That’s also led to more complex patients,” added Dimmitt. “To meet these many needs, we spend a lot of time with the parents.”

The team receives referrals from a variety of sources, including inpatient subspecialists, community pediatricians and speech pathologists, among others. “My favorite referrals,” Harris said, “are families telling friends. That’s when we know we have succeeded.”

“What we hear from parents is that they are so thankful for the collaborative care, the time everyone spends and that their concerns are heard,” said Chapman.

Inside Pediatrics, Pulmonology

New Faculty Expands Pulmonary and Sleep Medicine Division

The UAB Division of Pediatric Pulmonary and Sleep Medicine at Children’s of Alabama welcomed two new faculty in July 2020.

The University of Alabama at Birmingham (UAB) School of Medicine Division of Pediatric Pulmonary and Sleep Medicine at Children’s of Alabama continues to expand, with two new faculty joining in July.

Pedro Anis Nourani, M.D., anassistant professor in the division, earned his medical degree at the University of São Paulo and completed his pediatric residency at Texas A&M’s Driscoll Children’s Hospital. He finished his sleep medicine and pulmonary fellowships at UAB and decided to stay.

“It’s definitely the people that work here,” he said of his decision to remain in Birmingham. “The collaboration between everyone, the excellent support from not just the physicians, but everyone in the department. This group has extensive expertise on subspecialities within pulmonary medicine, which provides good support for someone just starting their career.”

He was drawn to pulmonology medicine, he said, because it’s the “best that pediatrics has to offer.” That includes long-lasting relationships with patients; continuity of care; a strong inpatient presence and the ability to perform procedures. And he was drawn to UAB for his fellowship because of its large pediatric sleep lab. He interviewed at other institutions upon finishing his fellowships, “but there just wasn’t the support and excellence.”

He and his wife have three children, the youngest of whom was born at UAB.

Guillermo Beltran Ale, M.D., an assistant professor in pulmonology, was born and raised in Peru, where attended Universidad Peruana Cayetano Heredia medical school. He completed his pediatric residency at Cincinnati Children’s Hospital Medical Center, then stayed for his pediatric pulmonary fellowship. “I’ve been interested in pulmonology forever,” he said. First because of asthma and cystic fibrosis, with its complex management system and physiology. Then because of the strong bronchoscopy program at Cincinnati and its relationship with chronic ventilator management.

He chose Children’s because it offered a place to grow his skills and continue his research on the pathology of long-term ventilation. He moved to Birmingham with his wife and dog in the summer and started his new career at Children’s July 1.

COVID-19 has, of course, changed the experience of moving to a new city. “We meet a lot of people but we can only see their eyes,” he said. Nonetheless, “we enjoy Birmingham so far.”

Hematology and Oncology, Inside Pediatrics, Neurology & Neurosurgery

Children’s of Alabama Launches Second Groundbreaking Trial of Viral Treatment for Brain Tumors

Children’s of Alabama and the University of Alabama at Birmingham are leading studies using a genetically re-engineered herpes virus to treat pediatric high-grade gliomas.

“A uniformly dismal prognosis.” That’s how Children’s of Alabama neurosurgeon James M. Johnston, Jr., M.D., describes what children with recurrent malignant brain tumors face, with an average lifespan of six months given a lack of effective treatments.

Now Johnston, in collaboration with Greg Friedman, M.D., associate professor of pediatric oncology and director of Developmental Therapeutics at Children’s, is leading groundbreaking studies designed to shift that trajectory. The team recently completed a Phase 1 immunotherapy clinical trial of genetically re-engineered herpes virus G207 to treat pediatric high-grade gliomas. Their work builds on adult research on the viral treatment pioneered by James Markert, M.D., MPH, who chairs the Department of Neurosurgery at the University of Alabama at Birmingham (UAB), as well as Friedman’s laboratory work, which showed the virus was more effective against pediatric brain tumors than adult tumors.

In the past three years, 11 patients with high-grade gliomas have traveled to Children’s from throughout the country, Mexico and Canada to participate in the study. They receive special screening to pinpoint the tumor location, which is then biopsied. Johnston then places three to four catheters in the tumor. The next day, Friedman and his team infuse the genetically-modified virus into the brain through the catheters.

“We think the virus works by directly killing the tumor cells,” Johnston said, as well as activating the immune system to destroy any remaining cells. “Brain tumors have a way to hide from the immune system by making themselves immunologically ‘cold,’” he explained. The herpes virus turns a “cold” tumor into a “hot” tumor and generates the immune response. Indeed, months after the surgery tests show that immune cells have infiltrated the tumor and continue killing tumor cells.

The initial phase 1 trial in patients with high-grade gliomas was designed to demonstrate safety and wasn’t powered to show efficacy. Nonetheless, Johnston said, “our median survival was significantly longer than the historical six months,” with several children now long-term responders.

In late December 2019, the team received a three-year, $750,000 R01 grant from the U.S. Food and Drug Administration for a Phase 1 trial in malignant cerebellar brain tumors, which may be even more sensitive to the virotherapy than the gliomas. At the same time, they are submitting grants for a Phase 2 multicenter trial of the virus therapy for recurrent malignant supratentorial tumors.

Johnston stresses that the research is a team effort, involving basic scientists, oncologists, surgeons, nurses and intensivists. “It’s an ‘all-hands-on-deck’ kind of thing,” he said.

Inside Pediatrics, Neurology & Neurosurgery

Reducing Spasticity, Improving Quality of Life – One Child at a Time

Children’s of Alabama neurosurgeon Brandon Rocque, M.D., left, is part of a multidisciplinary team that offers surgical and medical therapies for children with cerebral palsy and severe spasticity.

Take orthopedic surgeons, neurosurgeons, rehabilitation medicine specialists, physical and occupational therapists, and specially trained nurses and what do you have? A multidisciplinary team that makes Children’s of Alabama one of the few pediatric medical centers in the U.S. to offer state-of-the-art surgical and medical therapies for children with cerebral palsy and severe spasticity, whether that be selective dorsal rhizotomy or intrathecal Baclofen pump therapies.

“We all work together to determine the best option for the child,” said Children’s neurosurgeon Brandon Rocque, M.D. But the medical professionals aren’t the only ones who play a role in the decision. “The most important factor is the family’s goals for the child,” Rocque said, which typically focus on some improvement in walking.

The two main options are rhizotomy, in which the surgeon exposes, stimulates and then severs nerve roots in the spinal cord, or a Baclofen pump, which continuously releases the muscle relaxer through the implanted pump.

“There are an infinite number of ways to stimulate the nerves (for rhizotomy) and make decisions about what to cut and not cut,” Rocque said. “Every program is unique.” To bring a more objective approach to the procedure, Children’s participates in the Cerebral Palsy Research Network (CPRN), a group of 25 medical centers committed to planning and executing high quality clinical research and quality improvement protocols.

“The idea of CPRN is to pull clinical data from the electronic medical record from children with cerebral palsy into a single data repository to understand it better,” Rocque said. That includes identifying children most likely to benefit from rhizotomy as well as the most appropriate surgical approach based on their condition.

For children who don’t require the extensive rhizotomy surgery, orthopedic surgery to release tight hamstrings or Achilles tendons might be used to reduce spasticity. And for those with more severe spasticity or dystonia, there’s the Baclofen pump.

More recently, the team has been performing palliative rhizotomy for children who would typically be considered for a Baclofen pump. While the pump remains an option, Rocque said, it is an implanted device, has to be refilled and carries a risk of infection and mechanical complications. Palliative rhizotomy, while not restoring the ability to walk, improves muscle tone in the legs. “Families are thrilled with how much looser the kids are, how much more comfortable and easier to take care of.”

The program sees about 100 children a year, most referred through rehabilitation medicine specialists, and hopes to expand from a bimonthly to a monthly clinic.

Inside Pediatrics, Neonatology

NICU Care Coordinators: Getting Babies Ready for Discharge from the Day of Admission

Infants admitted to Children’s of Alabama’s Neonatal Intensive Care Unit (NICU) are closely monitored by care coordinators who begin planning discharge from the day of admission.

When a baby is admitted to the neonatal intensive care unit (NICU) at Children’s of Alabama, a special team of nurses has already begun planning the care required to send them home. Called care coordinators, their job is to ensure everything is in place for a positive outcome after discharge. “The doctor, bedside nurses and nurse practitioners are mainly focused on acute care of the patient,” said NICU Medical Director Carl H. Coghill, M.D., who, together with Children’s nursing administration, helped developed the discharge program when he became medical director in 1998. “The coordinators are focused on the long-term. They are the ones that see a patient from start to finish.”

 “They are a face parents learn to know and trust,” he said.

From routine immunizations, retinopathy of prematurity screenings, hearing evaluations and other newborn screenings, the discharge planners oversee the health maintenance of these tiny patients. They also round with the medical team every day to stay abreast of the most current plan for the baby, then incorporate that information into the discharge planning.

“As medicine has evolved, more preemies are, thankfully, surviving,” said Brenda Voulgarides, RN, who helped shape the position as the first care coordinator 20 years ago. “But they often go home with complex medical needs.” For instance, she still remembers the first time a patient was discharged with a gastrostomy tube or tracheostomy.

Today, as discharge nears, the team ensures that all pending consults, studies and lab work are followed; work to ensure that all consulting teams agree to the final treatment plan; and arrange all outpatient follow-up. They also provide individual education to the families on the baby’s care, including instruction on all equipment and medications. An important part of this is ensuring that parents learn how to be their child’s advocate and know when and who to call for help. After discharge, they provide the baby’s outpatient pediatrician with a comprehensive overview of the hospital course, medications, and plans to facilitate care transition.

The complexity of the patients means that the care coordinators have to be adept at working through many barriers during the education process. For instance, Voulgarides once taught a blind mother how to administer her baby’s medications and care for the infant’s tracheostomy.

The challenges of the babies mean that “the parents have to really understand their child’s diagnosis, reasons for the medicines and need for all of the follow-up,” said care coordinator Andrea Walding,RN. And that takes time. “It can’t be done over two days.”

The care coordinators also work to ensure that the baby’s team of physicians all agree on the discharge plans, said Voulgarides, so when the babies are seen in the outpatient clinic, “everyone’s philosophy aligns” and there are no major changes that require significant re-education of the parent or doubt about their prior care.

Coghill knows how important the coordinators are. “Often, parent satisfaction with the NICU has more to do with the care coordinators than with the doctors and bedside nurses,” he said.

Inside Pediatrics, Neonatology

Neurodevelopmental Multidisciplinary Care

Children’s of Alabama’s Neonatal Intensive Care Unit (NICU) developed a neuroprotective team two years ago in efforts to improve an infant’s neurodevelopmental care.

The first three years of life are a crucial time period for rapid brain growth and normal development. This growth and development relies heavily upon the surrounding environment and positive external stimulation. Now imagine a baby spending months, if not years, in the neonatal intensive care unit (NICU), an environment filled with noxious stimuli—loud monitors, bright lights, painful procedures and if a patient is acutely ill, limited positive physical interactions. A great obstacle, then, is how to provide babies the best care to ensure normal neurodevelopmental growth.

“Many of our NICU patients are already at particularly increased risk due to their underlying diagnoses and prolonged hospitalizations,” said neonatologist Allison Black, M.D. “Some patients stay in the unit up to two years,” she said. Black, along with therapists, nurses, and educators who care for the babies, felt they could do more to optimize their environment, care, and, ultimately, their neurodevelopmental growth and long-term outcomes.

They created a neuroprotective team two years ago to provide the initiatives needed to improve an infant’s neurodevelopmental care. This multidisciplinary team, composed of occupational, physical and speech therapists, care coordinators, physicians, neonatal nurse practitioners, bedside nurses, and nursing educators, is designed to improve communication and alignment among the multitude of health care providers who work with the infant.

The team first focused on providing education and increased awareness about neurodevelopmental care to staff and families through a “carnival,” – hands-on skills labs, simulations, learning modules and didactic education. Topics included safe sleep practices, wound and skin care, kangaroo care, feeding, developmental and sensory issues in premature infants, and the use of developmental products such the Dandle WRAP™, which can promote neuromuscular development and self-regulatory ability. More than 200 nurses participated.

The team continues to work on other initiatives that target bedside care providers and encourage family involvement including:

  • A book cart where parents can obtain books for themselves, their other children and their hospitalized infant.
  • A sensory book for each room with pictures and lullaby words so parents can read and sing to their infant.
  • Adult coloring books for reducing stress.
  • Ongoing and regular education with staff, including reminder cards with a summary of important “take home” points from the latest education, review of recent journal articles and monthly educational topics that have both been converted to virtual sessions during the coronavirus pandemic.

In 2019, the multidisciplinary team expanded to include audiologists and rehabilitation medicine as well as NICU physicians, nurse practitioners and bedside nurses, and began holding regular NICU neurodevelopmental rounds. “During weekly rounds, each patient is systematically discussed,” Black said, “including their current medical and neurodevelopmental care plan, specific short- and long-term goals as well as long-term overall prognosis.”

The discussions may lead to changes in the care plan, such as starting physical therapy earlier. “It’s helpful to have a team-based approach to developing the neurodevelopmental goals, discussing parents’ expectations and providing the space for an open forum to discuss each patient’s long-term medical prognosis,” she said.

This team encourages parents and other caregivers to be involved with their infant’s therapy, working alongside the rehabilitation physicians they will see in the outpatient setting. “Involving the rehab medicine team early in the course helps ease the transition once our patients are discharged home,” Black said. “I think the frequent discussion, early engagement and involvement of the multidisciplinary team and care providers will help us accomplish the ultimate goal of improving the overall neurodevelopmental care and outcomes for our patients.”

Hematology and Oncology, Inside Pediatrics

Charting New Ground in Treating Blood Clots in Infants

UAB assistant professor of pediatrics and Children’s of Alabama hematologist-oncologist Hope Wilson, M.D., is researching long-term anticoagulation for high-risk children thanks in part to a American Society of Hematology fellowship.

Venous thromboembolism (VTE) is an increasing problem in pediatrics and can be associated with significant morbidity. Although most children are at low risk for recurrence, some have ongoing risk factors, which increases their risk for having further thrombosis. Yet there is no specific guidance on the optimal duration of treatment for such patients.

Well, not yet, anyway. But if research from Children’s of Alabama hematologist-oncologist Hope Wilson, M.D., is successful, that question will have an answer.

This year, Wilson was named one of 23 American Society of Hematology (ASH) Clinical Research Training Institute (CRTI) participants, a prestigious award that will help fast track her research on long-term anticoagulation for high-risk children. The fellowship is a unique, year-long education and mentoring program for hematology fellows and junior faculty at academic medical centers with the opportunity to interact “with the finest in the field,” Wilson said.

Throughout the year, Wilson will receive intense review and critical feedback on her project. The goal is to have a polished proposal to submit for external funding at the end of the program. But the proposal is just the beginning of what the grant means for her career. “There is a lot of greatness that can happen as a result of the exposure,” she said, including lasting mentorship relationships and critical networking opportunities for potential future collaborations.

The CRTI program kicked off virtually in August, focusing on the foundation, methodologies, and application of patient-oriented clinical research. Participants will meet regularly throughout the year concluding in May 2021 at ASH headquarters in Washington, D.C.

Wilson’s interest in this topic came, in part, from the patients she sees in Children’s new pediatric thrombosis clinic, which she helped develop. The overall goal of the thrombosis program is to improve care for children affected by VTE. To this end, the team has worked to develop protocols and algorithms to standardize treatment for these children, who come from throughout Alabama as well as neighboring states. In just the past year, more than 100 patients have passed through the clinic, many with recurrent clots. 

That’s the population she’s focused on in her research. “Findings from this research will help fill this knowledge gap and provide critical data to inform clinical decision making, changing the way that we manage children who require long-term anticoagulation,” she said.

Hematology and Oncology, Inside Pediatrics

Addressing the Unmet Needs of Adolescent and Young Adult Patients With Cancer

Julie Wolfson, M.D., assistant professor of pediatrics at UAB and Children’s of Alabama, helped develop the Adolescent and Young Adult (AYA) Oncology Program.

There’s the pediatric cancer population, those younger than 15. And the adult cancer population, those 40 and older. And then there’s the “in-between” patients, those ages 15 to 39, defined as adolescents and young adults (AYA), who sometimes seem as if they’ve left behind when it comes to the dramatic improvement in cancer outcomes.

“Even though they may have the same diagnosis as the 14-and-under group and the 40-and-older group, we haven’t seen the same improvement in survival as the other groups,” said Julie Wolfson, M.D., assistant professor of pediatrics in the University of Alabama at Birmingham (UAB) School of Medicine Institute for Cancer Outcomes and Survivorship.

In talking to adolescents and young adults with cancer, she said, “you learn that they’re not getting things that are important to them.” This includes financial and psychosocial support, as well as information on fertility preservation. They’re also not enrolling in clinical trials, “and we know that outcomes correlate with clinical trial enrollment.”

To address the problem, the hematology/oncology teams at UAB and Children’s of Alabama developed the Adolescent and Young Adult Oncology program for these “in-between” patients.

The team received seed funding in 2018 from Hyundai Hope on Wheels to develop the program’s infrastructure. The Vestavia Hills High School Rebels Impact through Service and Engagement (RISE) program, through the O’Neal Comprehensive Cancer Center, helped raise money for the program, enabling its expansion.

Nearly 200 AYAs have participated so far. Patients are matched with potential clinical trials and receive fertility preservation counseling and a psychosocial screen from the program’s adolescent and young adult social worker. An online support group for those 25 and younger is available.

A group of specialists from each cancer type meet in a monthly (now virtual) tumor board. “Patients really benefit from us all sharing our knowledge,” Wolfson said. Indeed, research shows improved outcomes with greater collaboration between adult and pediatric health care providers.

The feedback from the health care teams and the patients and families has been very positive, she said. “Patients express relief that someone ‘gets it,’” Wolfson said. “That they’re not a 5-year-old who wants to look at the goldfish and they’re not a 65-year-old with grandkids. There’s a lot of emotion involved and to process all that with someone at their developmental level is really important for them.”

None of this would have been possible without the leadership from the pediatric and adult hematology/oncology programs, she said. “Every AYA program looks different but ours is one of the more comprehensive I’ve seen across the country because we have amazing, collaborative clinicians. If people weren’t willing to play together in the ‘sandbox’ the way they are it wouldn’t be as successful.”

Hematology and Oncology, Inside Pediatrics

Xenograft Program Discovering New Therapies For Pediatric Cancer

Elizabeth Beierle, M.D., center, and Jamie Aye, M.D., right, run one of the few pediatric xenograft programs in the U.S. out of Children’s of Alabama.

Although the story of pediatric cancer over the past 20 years is one of success, there are still areas where the news remains grim, particularly for pediatric solid tumors. In addition, the therapies used to treat pediatric cancers can have long-lasting consequences and even increase the risk of other cancers.

Which makes identifying novel agents with better outcomes and fewer side effects so critical. Enter the xenograft approach, in which cancer tissue taken from the child is implanted into an animal model, allowing for more precise targeting of potential therapies.

“There are xenograft models in the adult population to find novel agents, but the pediatric model is very rare,” said Elizabeth A. Beierle, M.D., a pediatric surgeon at Children’s of Alabama who focuses on pediatric surgical oncology. She, along with pediatric hematologist-oncologist Jamie M. Aye, M.D., run one of the few pediatric xenograft programs in the country out of Children’s.

“These patient-derived xenografts more closely mimic what’s going on in the human,” said Beierle. Otherwise, researchers have to use cells from tumors gathered more than 50 years ago and cultured under artificial conditions. “A lot of those tumors lost the genetic and phenotypic characteristics from the original tumor,” she said. “So when we try to investigate new drugs, we often see that what happens in the tissue culture is not what’s happening in humans.”

Plus, testing compounds in a petri dish misses the reality of a cancer in a living model, including the cells that surround the tumor and the blood vessels.

The xenograft process takes time: six months to a year before the tumors grow and experiments can begin. But seven years after the Children’s initiative began, several therapies are showing promise, with some ready to move into Phase 1 clinical trials.

One is a viral therapy for pediatric solid tumors that infects tumor cells and releases an inflammatory cytokine that attracts other inflammatory cells to attack the tumor cells. Another inhibits a protein found in pediatric liver tumors.

The barrier to clinical trials, said Beierle, is funding. While the program has received strong support from community organizations, more is needed.

One thing that isn’t a problem is obtaining the biopsy tissue for the xenograft bank. “We’ve never had a family say no,” said Aye. “They understand that the research may not affect their child, but they hope it will help other children.”