Tag Archive for: pediatric cancer

boy getting eye exam

Expanded partnership with Virginia Tech accelerates pediatric cancer research

boy getting eye exam

The new partnership will advance pediatric health through innovative discoveries and therapies, with an initial focus on pediatric cancers, including brain tumors.

Children’s National Hospital and Virginia Tech are expanding their research partnership, building on a successful collaboration established in 2019. This partnership will advance pediatric health through innovative discoveries and therapies, with an initial focus on pediatric cancers, including brain tumors.

The partnership brings together Children’s National, ranked among the nation’s top pediatric hospitals by U.S. News & World Report, and Virginia Tech, a leading academic research institution. Together, they aim to deliver transformative advancements to enhance outcomes for children facing devastating diagnoses.

The goals of the research-focused partnership include:

  • Accelerating the understanding of the biology, improvements in prevention and treatment of pediatric cancers and other childhood diseases.
  • Developing new diagnostic and therapeutic tools to improve care for children.
  • Training the next generation of scientists and physician-scientists.

What they’re saying

  • “Over the years, our partnership with Virginia Tech has demonstrated the power of combining top-tier research expertise with a shared commitment to improving pediatric health,” said Catherine Bollard, MBChB, MD, senior vice president and chief research officer and director of the Center for Cancer and Immunology Research. “This expansion underscores our belief that by working together, we can accelerate discoveries and develop life-changing therapies for children with cancer and other rare diseases.”
  • “Children’s National Hospital has been an important partner for us in biomedical research and innovation,” said Michael Friedlander, PhD, Virginia Tech vice president for health sciences and technology. “Our collaboration deepened with the launch of Children’s National Research & Innovation Campus in Washington, D.C., and now, as our partnership grows even stronger, we’re poised together to take on some of the biggest challenges in cancer research to contribute to the health of children and adults.”
  • “Partnering with Children’s National connects us to a world-class clinical trial institute that has been a pioneer in treating brain tumors with focused ultrasound technology, and this presents a unique opportunity to help children and families struggling with cancer,” said Cheng-Chia “Fred” Wu, MD, PhD, a member of the Children’s National Brain Tumor Research Institute and a principal investigator in cancer research and faculty member at the Fralin Biomedical Research Institute in Roanoke and in the Virginia Tech Carilion School of Medicine.“I can’t wait to see where this takes us.”

Big picture

The initial focus of the collaboration is pediatric cancers, including brain tumors — among the most challenging childhood diagnoses. By combining Virginia Tech’s leading-edge technology and research infrastructure with Children’s National’s expertise in pediatric care, the organizations aim to make significant strides in understanding these diseases.

An interdisciplinary approach is at the heart of the ongoing strategy. The collaboration first began with the launch of a 12,000-square-foot Virginia Tech biomedical research facility within the Children’s National Research & Innovation Campus, which opened in 2020. Located on a 12-acre portion of the former Walter Reed Army Medical Center in Washington, D.C., the campus was the nation’s first innovation hub focused exclusively on pediatric research.

Attendees at the 2nd annual Cell & Gene Therapy Symposium

Regional powerhouse: Cell and Gene therapy leaders from mid-Atlantic forge connections

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Nearly 200 biomedical leaders from Washington, D.C., Maryland, and Virginia gathered at the Children’s National Research & Innovation Campus for the 2nd annual Cell & Gene Therapy Symposium. The event showcased groundbreaking developments in rare disease treatments and underscored the importance of regional collaboration.

“By targeting diseases at the cellular level, we are on the cusp of breakthroughs in cell and gene therapy that will transform medicine,” said Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research (CCIR) at Children’s National Hospital and a host of the symposium. “Progress will accelerate if we build partnerships beyond our own organizations.”

The big picture

Scientists and clinicians have worked for more than two decades to develop cell and gene therapies aimed at treating diseases on a cellular level. The past few years have been particularly promising as investment in science has led to advancements. Children’s National is at the forefront, as one of the first pediatric hospitals in the world to offer commercial gene therapies for sickle cell disease.

Many more treatments for rare diseases are in development at Children’s National and beyond. Leaders at CCIR are actively building collaborations with companies, academic institutions and enterprises across the mid-Atlantic region to accelerate these efforts.

During the symposium, Eugene Hwang, M.D., chief of Oncology at Children’s National, addressed the urgent need for more effective and less toxic treatments for pediatric brain tumors. He highlighted the potential of combining immunotherapies with innovations like low-intensity focused ultrasound, which can open the blood-brain barrier temporarily to improve drug delivery to tumors.

“With collaboration between the lab and clinic, alongside industry partners and even between hospitals, we can finally make strides I haven’t seen in my entire career,” Dr. Hwang said. “It’s an incredibly inspiring time for all of us.”

Why it matters

Experts from organizations as diverse as MaxCyte, ScaleReady, RoosterBio, PSC Biotech, Qiagen, FujiFilm and the Frederick County Office of Economic Development came together for the daylong conversation.

Michael Friedlander, Ph.D., executive director of the Fralin Biomedical Research Institute at Virginia Tech, emphasized the critical role of regional partnerships in fulfilling the potential of these emerging therapies. He pointed to the collaborative research between Children’s National and Virginia Tech on brain tumors, where bioengineers and cancer researchers are working side-by-side to create new treatments.

“We are now able to begin delivering these leading-edge therapies to patients,” Dr. Friedlander said. “For example, those who live in rural settings often have much less access to such frontline medical innovations. By collaborating with Children’s National and gaining access to urban pediatric populations, as well as patients in our more rural area, we can start to bring these therapies to a much broader audience.”

What’s next

Patrick Hanley, Ph.D., chief and director of the Cellular Therapy Program at Children’s National, observed that other regions in the U.S. are uniting to advance scientific discoveries with the backing of government, academia and industry. He hopes to see similar collaboration across the D.C., Maryland, and Virginia area, known as the DMV. Children’s National is leading an initiative called CHARM – the Capital Health and Mid-Atlantic Regenerative Medicine – to bring regional experts together for webinars, networking events and partnership opportunities.

“There’s significant interest in cell and gene therapy worldwide,” said Dr. Hanley, a symposium host. “I see an even greater interest in creating cell and gene therapy hubs. The time is right for our mid-Atlantic region, and I’m excited to see what unfolds in the next five years.”

A clean room at CNRI.

CellBuilder: A ready-made solution for cell & gene therapy manufacturing

A clean room at CNRI.

With CellBuilder and our global partnerships, Children’s National hopes to expand access to groundbreaking cell and gene therapy treatments as they take off in the next five years.

With cell and gene therapies poised to reshape cancer and rare disease treatments, researchers at Children’s National Hospital are pioneering ready-to-use solutions that will bring these cutting-edge therapies directly to hospitals and other treatment centers, shrinking the distance between doctors and patients.

“The next five years are going to be a period of tremendous growth for cell and gene therapy,” said Patrick Hanley, Ph.D., chief and director of the Cellular Therapy Program at Children’s National. “Currently, there’s no shortage of interest from the medical community, but there’s a shortage of people who can manufacture and administer this care. We’re looking for ways to get these treatments to the patients by providing other institutions the tools they need to launch these programs cost-effectively, safely and efficiently.”

Called CellBuilder, the starter kits for cell and gene therapy programs could transform the landscape for pediatric patients.

The big picture

Dr. Hanley and many members of the Children’s National team have been working in cell and gene therapy for more than a decade, gaining extensive experience in the technical and regulatory hurdles inherent in creating treatments that target diseases at their source.

In cell therapy, a specific cell type is modified and transferred to a patient with a payload that can target a disease or disorder. For example, T cells may be modified and delivered to patients to teach their immune systems to fight cancer.  In gene therapy, a patient’s genetic code is modified to treat or prevent diseases, such as sickle cell disease, cancers and other genetic disorders. This can be done by introducing a healthy copy of a gene, repairing a faulty gene or altering a gene’s function.

Children’s National has become a leader in manufacturing virus-specific T cells, one method of delivering cell therapies, and the Cellular Therapy Program has conducted consortium-led, multi-center trials. Many other healthcare sites across the country want to start programs offering this care at their facilities.

The holdup in the field

Starting a cell and gene therapy program from scratch can take years of effort, training and money. That’s why Jay Tanna, M.S., R.A.C., quality assurance manager of the Cellular Therapy Program at Children’s National, said the team is creating CellBuilder starter kits, which include the manufacturing protocol and the resources necessary to launch a cell therapy program almost instantly.

“With a suitable knowledge base, institutions can start their own cellular therapy program at the point of care, using our manufacturing protocols, vetted reagents and other key elements of the process,” Tanna said. “Of course, interested institutions would have to meet regulatory requirements and establish a clean room to manufacture these therapies. If they want to use CellBuilder to run a clinical trial, they can do that. If they want to take it to become a licensed product, they can do that, too.”

Children’s National has worked with more than five institutions to build their virus-specific T-cell program and is now using the kits to accelerate and commercialize the process to increase patient access. The lab has also entered into a memorandum of understanding with the Tokyo-based Hitachi Global Life Solutions, Inc., an innovative modular clean room manufacturer, with the goal of offering a bundled solution.

Why we’re excited

Dr. Hanley and his colleagues say that the partnerships Children’s National is forging as they consult with other experts in this field will expand access to cell and gene therapy across the country—and hopefully around the world.

“It used to be that, to get a CAR T cell, you would collect the cells at Children’s National, ship them to a company like Novartis, have the therapy manufactured there and then shipped back,” said Michael Keller, M.D., who co-led a first-of-its kind immunotherapy trial as the Translational Research Laboratory director at the Children’s National Cell Enhancement and Technologies for Immunotherapy Program. “It was expensive, time-consuming and limited patient access. Now, there’s growing interest in manufacturing at each site, just like you would with a bone marrow transplant.”

With CellBuilder and our global partnerships, Children’s National hopes to expand access to groundbreaking cell and gene therapy treatments as they take off in the next five years, extending lifetimes and improving the quality of life for children suffering from rare disorders.

“We’re trying to capture the momentum underway in the field by providing this kit so that institutions don’t have to know how to do everything,” Dr. Hanley said. “We provide all the knowledge, a reagent list and everything else they need—and they provide the care.”

Leaders from pediatric healthcare, federal organizations, academia, industry and patient advocacy groups gathered at the Children’s National Research & Innovation Campus.

Access4Kids: A new model to pay for pediatric cell and gene therapies

Science is pioneering cures for pediatric rare diseases in a coming wave of new cell and gene therapies. However, the biopharmaceutical industry’s insistence on large patient populations and high profit margins may prevent these life-saving treatments from reaching the children who desperately need them. When successful therapeutics fail to see commercialization, experts say they have fallen into the “Valley of Death.”

To address this, leaders from pediatric healthcare, federal organizations, academia, industry and patient advocacy groups gathered at the Children’s National Research & Innovation Campus. Their objective: build a new framework to deliver these transformative drugs to clinics worldwide.

Meet the team forming Access4Kids, a nonprofit whose mission is to build new pathways to pay for cures and provide hope to children with life-limiting diseases. This group is working to change medicine and how we pay for it, under the leadership of Catherine Bollard, M.D., M.B.Ch.B., director of the Children’s National Center for Cancer and Immunology Research, Crystal Mackall, M.D., director of the Stanford Center for Cancer Cell Therapy, Julie Park, M.D., Oncology Department chair at St. Jude Children’s Research Hospital, and Alan Wayne, M.D., pediatrician-in-chief at Children’s Hospital Los Angeles.

illustration of diseased kidneys

The future of Wilms tumor therapies: Q&A with Jeffrey Dome, M.D., Ph.D.

illustration of diseased kidneys

Dr. Dome’s mission is to come up with other therapies to treat Wilms tumor.

Conducting Wilms tumor research has placed Children’s National Hospital as the leader in this space. Jeffrey Dome, M.D., Ph.D., talks about the future of Wilms tumor therapies and what excites him about being able to offer different treatment options to this group of patients.

Q: What’s next for Wilms tumor therapy treatments?

A: Something we’ve learned is that we’re reaching the maximum benefit that we can achieve with conventional chemotherapy and have hit a plateau with some of the high-risk Wilms tumors.

If you look at the history of Wilms tumor, we’ve ratcheted up the cure rate by adding more and more chemotherapy agents. We’re now at a point in which patients can’t tolerate having more toxic chemotherapy added to their regimen. Our mission is to come up with other therapies that work differently.

Q: What have you learned that can lead to new therapy options?

A: I was a co-investigator on a National Cancer Institute-funded project called the TARGET study, which entailed sequencing several hundred Wilms tumors to identify genetic mutations that could be druggable. While a wealth of knowledge was gained on the nature of the biology of Wilms tumor, only a minority of Wilms tumors have targetable mutations. We understand what the mutations are, but most are not mutations that lend themselves to drug therapy.

Therefore, we must think about other forms of therapy for Wilms tumor, such as immunotherapy. That’s where the work with the T cells targeting the WT1 protein Wilms tumor cells come into play.

Q: What do the new therapies look like?

A: I think the future of Wilms tumor therapy will be combining the classic chemotherapy and radiation therapy that we’ve used for many years but also adding another component for the patients with the highest risk of relapse, such as immunotherapy.

Up until now, the studies that we’ve conducted using immunotherapy have introduced this treatment very late as a last resort after everything else has failed. We have seen early signals of efficacy and if we can corroborate this in current studies, I believe the future will be to introduce immunotherapy earlier in the treatment course.

There has been success using immunotherapy in other pediatric solid tumors such as neuroblastoma. I believe the prospects for Wilms tumor are also exciting.

 

histological image of Wilms Tumor

Leading Wilms tumor research nationwide: Q&A with Jeffrey Dome, M.D., Ph.D.

histological image of Wilms Tumor

Children’s National has become a resource for patients and families with Wilms tumor.

During the past year, Children’s National Hospital saw nearly 100 patients with Wilms tumor and other less common kidney cancers of childhood, far more than most centers in the country. This is largely due to the reputation the hospital has established for specializing in these diseases. While most patients with Wilms tumor have excellent outcomes, a significant minority of children with kidney cancer do not fare well. Children’s National has become a resource for patients and families with these challenging cancers.

Behind this reputation is Jeffrey Dome, M.D., Ph.D., senior vice president of the Center for Cancer and Blood Disorders and division chief of Oncology at Children’s National, and the team of researchers he leads. For over a decade, he chaired the Children’s Oncology Group Renal Tumor Committee, an opportunity that gave him and his work great exposure.

Dr. Dome shares more on how Children’s National is leading in this space and what the future holds for new, exciting Wilms tumor treatment options.

Q: How is Children’s National leading in this space?

A: The good news is that for the most common type of childhood kidney cancer, Wilms tumor with “favorable histology,” the survival rate is more than 90%, which is an incredible success story. But approximately 25% of children and teens with other types of Wilms tumor and other kidney cancers do not fare as well. We specialize in kidney cancers that are harder to treat, such as anaplastic Wilms tumor, relapsed favorable histology Wilms tumor, bilateral Wilms tumor, clear cell sarcoma of the kidney, malignant rhabdoid tumor and renal cell carcinoma. Because we see a relatively large number of patients, we can draw on our prior experience and observations to recommend the best treatment options.

Q: What’s unique about this research?

A: We have several early-phase clinical trials that are of interest for children with relapsed kidney tumors. Some of these trials are part of research consortia, such as the National Cancer Institute-funded Pediatric Early Phase Clinical Trials Network (PEP-CTN). Other studies have been developed in-house at Children’s National, including a couple of studies using T cells to target pediatric solid tumors. The T cells that have been engineered by the Children’s National Cellular Therapy Laboratory are of particular interest for Wilms tumor because they target a protein called WT1, which is expressed in most Wilms tumors. In fact, WT1 was named after Wilms tumor. We have now had more than 25 patients with relapsed Wilms tumor come from around the country to participate in these studies. Based on early successes, we are continuing this line of research and trying to improve the technology in the current generation of studies.

Schistosoma haematobium egg

For hemorrhagic cystitis, harnessing the power of a parasite

Schistosoma haematobium egg

“Urogenital Schistosoma infestation, which is caused by S. haematobium, also causes hemorrhagic cystitis, likely by triggering inflammation when the parasite’s eggs are deposited in the bladder wall or as eggs pass from the bladder into the urinary stream. S. haematobium eggs secrete proteins, including IPSE, that ensure human hosts are not so sickened that they succumb to hemorrhagic cystitis,” says Michael H. Hsieh, M.D., Ph.D.

Every year, hundreds of thousands of U.S. patients – and even more throughout the world – are prescribed cyclophosphamide or ifosfamide. These two chemotherapy drugs can be life-saving for a wide range of pediatric cancers, including leukemias and cancers of the eyes and nerves. However, these therapies come with a serious side effect: Both cause hemorrhagic cystitis in up to 40 percent of patients. This debilitating condition is characterized by severe inflammation in the bladder that can cause tremendous pain, life-threatening bleeding, and frequent and urgent urination.

Infection with a parasitic worm called Schistosoma haematobium also causes hemorrhagic cystitis, but this organism has a fail-safe: To keep its host alive, the parasite secretes a protein that suppresses inflammation and the associated pain and bleeding.

In a new study, a Children’s-led research team harnessed this protein to serve as a new therapy for chemotherapy-induced hemorrhagic cystitis.

“Urogenital Schistosoma infestation, which is caused by S. haematobium, also causes hemorrhagic cystitis, likely by triggering inflammation when the parasite’s eggs are deposited in the bladder wall or as eggs pass from the bladder into the urinary stream. S. haematobium eggs secrete proteins, including IPSE, that ensure human hosts are not so sickened that they succumb to hemorrhagic cystitis,” says Michael H. Hsieh, M.D., Ph.D., senior author of the study published April 3, 2018, by The FASEB Journal. “This work in an experimental model is the first published report of exploiting an uropathogen-derived host modulatory molecule in a clinically relevant model of bladder disease, and it points to the potential utility of this as an alternate treatment approach.”

S. mansoni IPSE binds to Immunoglobulin E (IgE), an antibody produced by the immune system that is expressed on the surface of basophils, a type of immune cell; and mast cells, another immune cell that mediates inflammation; and sequesters chemokines, signaling proteins that alert white cells to infection sites. The team produced an ortholog of the uropathogen-derived protein. A single IV dose proved superior to multiple doses of 2-Mercaptoethane sulfonate sodium (MESNA), the current standard of care, in suppressing chemotherapy-induced bladder hemorrhaging in an experimental model. It was equally potent as MESNA in dampening chemotherapy-induced pain, the research team finds.

“The current array of medicines we use to treat hemorrhagic cystitis all have shortcomings, so there is a definite need for novel therapeutic options,” says Dr. Hsieh, a Children’s National Health System urologist. “And other ongoing research projects have the potential to further expand patients’ treatment options by leveraging other urogenital parasite-derived, immune-modulating molecules to treat inflammatory bowel diseases and autoimmune disorders.”

Future research will aim to describe the precise molecular mechanisms of action, as well as to generate other orthologs that boost efficacy while reducing side effects.

In addition to Dr. Hsieh, Children’s study co-authors include Lead Author, Evaristus C. Mbanefo; Loc Le and Luke F. Pennington; Justin I. Odegaard and Theodore S. Jardetzky, Stanford University; Abdulaziz Alouffi, King Abdulaziz City for Science and Technology; and Franco H. Falcone, University of Nottingham.

Financial support for this research was provided by National Institutes of Health under award number RO1-DK113504.

Catherine-Bollard-SIOP

Advancing cures for pediatric cancer: Highlights from leading Children’s National experts at SIOP 2017

In mid-October 2017, nearly 2,000 clinicians, scientists, nurses, health care professionals and cancer patients and survivors gathered in Washington, D.C., for SIOP 2017, the Annual Congress of the International Society of Paediatric Oncology. For four days, attendees heard from world-renowned experts while exchanging ideas and information, all in the name of advancing cures for childhood cancer.

Hosted in the hometown of Children’s National Health System and chaired by Jeffrey Dome, M.D., Ph.D., Vice President of the Center for Cancer and Blood Disorders and Chief of Oncology at Children’s National Health System, more than 20 doctors and nurses from Children’s National made an impact on participants through a series of widely attended sessions and addresses, including:

  • Symposium lecture on the latest approaches in anti-viral T-cell therapy to improve patient outcomes, given by Catherine Bollard, M.D., M.B.Ch.B.
  • Keynote lecture on DICER1 mutations in pediatric cancer, given by Ashley Hill, M.D., whose study of a rare childhood lung cancer and gene mutations set the stage for a better understanding of microRNA processing gene mutations in the development of pediatric cancer.
  • Education session on new therapies for sarcomas, led by AeRang Kim, M.D., Ph.D., and Karun Sharma, M.D., Ph.D., sharing research on new approaches for local control of sarcomas, such as surgery, radiation and other ablative measures.
  • Education session on new therapies for gliomas, led by Roger J. Packer, M.D., with presentations on immunotherapy from Eugene Hwang, M.D., and targeted therapy by Lindsay Kilburn, M.D.
  • Podium paper presentation on a new method to measure cancer treatment toxicities as reported by the child by Pamela Hinds, Ph.D., RN, FAAN, as well as an education session on advanced care planning, led by Hinds with a presentation from Maureen E. Lyon, Ph.D.

“These sessions and lectures provided a glimpse into the groundbreaking work by SIOP attendees from around the world,” says Dr. Dome. “Children’s National is proud to play an active role in the development of life-saving treatments for children with cancer and our clinicians look forward to another year of revolutionary developments.”

For more on this year’s SIOP, see the Children’s National press release.

  • Jeffrey Dome, M.D., Ph.D., addresses a group of international colleagues at a reception at Children’s National.

    Jeffrey Dome SIOP

  • Catherine Bollard, M.D., M.B.Ch.B., addresses a group of international colleagues at a reception at Children’s National.

    Catherine-Bollard-SIOP

  • Lindsay Kilburn, M.D., engages with peers from around the world at a reception at Children’s National.

    Lindsay-Kilburn-SIOP
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Javad Nazarian

Advancing pediatric cancer research by easing access to data

Javad Nazarian

“This is a tremendous opportunity for children and families whose lives have been forever altered by pediatric cancers,” says Javad Nazarian, Ph.D., M.S.C., principal investigator in the Center for Genetic Medicine Research and scientific director of the Brain Tumor Institute at Children’s National.

Speeding research into pediatric cancers and other diseases relies not only on collecting good data, but making them accessible to research teams around the world to analyze and build on. Both efforts take time, hard work and a significant amount of financial resources – the latter which can often be difficult to attain.

In a move that could considerably advance the field of pediatric cancer, the National Institutes of Health (NIH), a body that funds biomedical research in the United States, recently awarded a public-private research collective that includes Children’s National Health System up to $14.8 million to launch a data resource center for cancer researchers around the world in order to accelerate the discovery of novel treatments for childhood tumors. Contingent on available funds, five years of funding will be provided by the NIH Common Fund Gabriella Miller Kids First Pediatric Research Program, named after Gabriella Miller, a 10-year-old child treated at Children’s National.

As principal investigators, researchers at Children’s Hospital of Philadelphia will lead the joint effort to build out the “Kids First” Data Resource Center. Children’s National in Washington, D.C., will spearhead specific projects, including the Open DIPG project, and as project ambassador will cultivate additional partnerships with public and private foundations and related research consortia to expand a growing trove of data about pediatric cancers and birth defects.

“This is a tremendous opportunity for children and families whose lives have been forever altered by pediatric cancers,” says Javad Nazarian, Ph.D., M.S.C., principal investigator in the Center for Genetic Medicine Research and scientific director of the Brain Tumor Institute at Children’s National. “From just a dozen samples seven years ago, Children’s National has amassed one of the nation’s largest tumor biorepositories funded, in large part, by small foundations. Meanwhile, research teams have been sequencing data from samples here and around the world. With this infusion of federal funding, we are poised to turn these data into insights and to translate those research findings into effective treatments.”

Today’s NIH grant builds on previous funding that Congress provided to the NIH Common Fund to underwrite research into structural birth defects and pediatric cancers. In the first phase, so-called X01 grantees—including Eric Vilain, M.D., Ph.D., newly named director of the Center for Genetic Medicine Research at Children’s National—received funding to sequence genetic data from thousands of patients and families affected by childhood cancer and structural birth defects.

This new phase of funding is aimed at opening access to those genetic sequences to a broader group of investigators around the globe by making hard-to-access data easily available on the cloud. The first project funded will be Open DIPG, run by Nazarian, a single disease prototype demonstrating how the new data resource center would work for multiple ailments.

DIPG stands for diffuse intrinsic pontine glioma, aggressive pediatric brain tumors that defy treatment and are almost always fatal. Just as crowd sourcing can unleash the collective brainpower of a large group to untangle a problem swiftly, open data sharing could accomplish the same for childhood cancers, including DIPG. In addition to teasing out molecular alterations responsible for making such cancers particularly lethal, pooling data that now sits in silos could help to identify beneficial mutations that allow some children to survive months or years longer than others.

“It’s a question of numbers,” Dr. Vilain says. “The bottom line is that making sense of the genomic information is significantly increased by working through large consortia because they provide access to many more patients with the disease. What is complicated about genetics is we all have genetic variations. The challenge we face is teasing apart regular genetic variations from those genetic variations that actually cause childhood cancers, including DIPG.”

Nazarian predicts some of the early steps for the research consortium will be deciding nuts-and-bolts questions faced by such a start-up venture, such as the best methods to provide data access, corralling the resources needed to store massive amounts of data, and providing data access and cross correlation.

“One of the major challenges that the data resource center will face is to rapidly establish physical data storage space to store all of the data,” Nazarian says. “We’re talking about several petabytes—1,000 terabytes— of data. The second challenge to address will be data dissemination and, specifically, correlation of data across platforms representing different molecular profiles (genome versus proteome, for example). This is just the beginning, and it is fantastic to see a combination of public and private resources in answering these challenges.”

Cancer update: tumor targeting, neurofibromatosis type 1 symptoms

June 6, 2016 –Targeting tumors more precisely, with fewer lasting side effects for kids
Pediatric patients with cancer are often treated with a cocktail of therapies to attack the disease through a variety of mechanisms. While this approach has been instrumental in saving children’s lives, the life-saving therapies can be accompanied by acute side effects, and the treatments may have lingering impacts as cancer survivors enter adulthood. Magnetic resonance-guided high-intensity focused ultrasound (MR-HIFU) holds the promise of surgically removing large tumors without exacting the same array of harsh side effects. Ultrasound relies on high-frequency sound waves to make diagnostic images, and those same sound waves can be used therapeutically to destroy tumors. Layering on MR imaging gives clinicians the ability to precisely guide the ultrasound therapy in real time. A study led by Children’s National Health System researchers and clinicians is using MR-HIFU for the first time in children to examine its safety and feasibility.

May 11, 2016 – Quantitative MRI criteria for optic pathway enlargement in neurofibromatosis type 1
Symptoms of neurofibromatosis type 1 (NF1) vary widely, but the condition is characterized by changes in skin pigmentation and growth of tumors along nerves. The research team sought to determine quantitative size thresholds for enlargement of the optic nerve, chiasm, and tract in children aged 0.5 to 18.6 years with NF1. The study, published in Neurology, found that quantitative reference values for anterior visual pathway enlargement will enhance development of objective diagnostic criteria for optic pathway gliomas secondary to NF1.

Allergy and immunology update: asthma care, microbial signatures

June 16, 2016 – Increased identification of the primary care provider as the main source of asthma care among urban minority children
The research team used electronic communication between an asthma specialty clinic and short-term care coordination to encourage parents of urban youth with asthma to identify their primary care provider as the key source for episodic asthma care – rather than the emergency department. Guardians of 50 children were enrolled in the prospective cohort study, whose findings were published in Journal of Asthma. The youths’ median age was 5.8 years; 64 percent were male, 98 percent were African American. At three and six months after the intervention, 85 percent and 83 percent, respectively, reported that the primary care provider was their child’s primary asthma healthcare provider, compared with 70 percent at baseline. 

June 16, 2016 – Two sampling methods yield distinct microbial signatures in the nasopharynges of asthmatic children
The nasopharynx acts as an anatomical reservoir from which pathogenic microbes spread to the lower and upper respiratory airways, causing respiratory infections. A team led by Children’s National researchers used targeted 16S rRNA MiSeq sequencing and two techniques – nasal washes and nasal brushes – to characterize the nasopharyngeal microbiota in 30 children with asthma aged 6 to 17. The authors report in Microbiome that the children’s nasopharyngeal microenvironments contain microbiotas with different diversity and structure.

Nov. 30, 2015 – Alex’s Lemonade Stand Foundation grant to develop immune-based therapy
Physician-scientist Conrad Russell Y. Cruz, MD, PhD, was awarded a $450,000, grant from the Alex’s Lemonade Stand Foundation to develop novel cell-based therapies to combat pediatric cancer. The “A” grant encourages scientists to develop innovative treatments and cures that impact children with cancer and will provide Dr. Cruz and his team funding for three years.