5,000 Children Enrolled in the Children’s Brain Tumor Network

Pediatric brain tumors are rare yet devastating, and it is essential to collect tumor tissue to unravel their biology and explore potential therapies. With over 125 types of childhood brain tumors and new subtypes continually being discovered, collecting these tumors for research is crucial. CBTN converts these precious resources into reusable large-scale data, empowering researchers to gain a deeper understanding of these complex diseases.

“The research conducted by the Children’s Brain Tumor Network, of which Bridge To A Cure is part, will influence the development of better treatments and interventions for childhood brain tumors. By leveraging the data and insights gained, clinical trials and drug development can be tailored to individual patients, leading to more effective treatments for our most vulnerable.”- Jena Lilly, CBTN Executive Director of Research Operations and Strategic Planning

At the Bridge To A Cure Foundation, we stand at the intersection of hope and loss, driven by a deep commitment to transforming the landscape of pediatric brain tumor research. Today, we reflect on a momentous milestone achieved by our collaborators at the Children’s Brain Tumor Network (CBTN): the enrollment of over 5,000 children as subjects to donate precious data samples. This landmark accomplishment, unprecedented in its scale, carries deep significance and underscores the urgency of the cause. It’s a revelation that may surprise some: in the pursuit of better treatments and cures, this unprecedented endeavor had not been fully realized until now.

As an organization that intimately understands the devastating impact of pediatric brain tumors, Bridge To A Cure is profoundly grateful for this milestone and the unwavering courage of the children, their families, and our entire network of scientists, researchers, and activists. Together, we forge ahead to find better treatments and cures, fueled by a shared determination and a commitment to honoring the lives of those we have lost to childhood cancer.

This milestone represents a profound partnership that goes beyond numbers. These 5,000 children and their families who have made the decision to share their clinical data and biospecimens to help find cures for other kids like them. Their legacy is our hope, and their resilience inspires us to keep pushing to unite and transform the childhood cancer research community to reduce childhood cancer deaths 50% by 2030.

The Transformative Approach to Childhood Brain Tumor Research

CBTN pairs the collected tissue with clinical data collected over the course of treatment, and, whenever possible, multiple types of molecular data, to create a robust and holistic dataset for researchers. This comprehensive approach allows for significant advancements in our understanding of childhood brain tumors. Recognizing the exponential impact of donated tissue and biospecimens, CBTN stewards the largest collection of childhood brain tumor biospecimens in the world. Moreover, they strive to make all specimens, data, and resources freely accessible to the global community of cancer researchers. This open collaboration and research-sharing model not only accelerates scientific discoveries but also significantly shortens the time required to launch new research projects.

A Call to Unity and Collaboration

At Bridge To A Cure Foundation, we believe progress accelerates through unity. As we observe this milestone, we pause to appreciate the collaborative efforts of CBTN as well as its dozens of partner institutions, hundreds of researchers, and advocates globally who have joined forces to end pediatric brain tumors. We look to the future with confidence and resolution. We envision a day when pediatric brain tumors are no longer a devastating diagnosis, but a treatable – or preventable – condition.

The knowledge gained from the research made possible because of these courageous donors, coupled with the passion and dedication of organizations like Bridge To A Cure, guide us toward that future. By fostering a culture of collaboration and sharing resources, we transform the dreams of children and families into tangible advancements. Furthermore, we commit to leveraging this milestone to bring attention, resources, and support to pediatric brain tumor research.

“We won’t stop. We will do everything in our power to find better therapies and cures through their very precious donation. We’re just not going to stop.” -Jennifer Mason, CBTN Director of Biospecimen and Clinical Research Operations

Bridge To A Cure Foundation and our allies are united in our commitment to honor the lives of the children we have lost and to create a brighter outcome for those still fighting. Together, we can bridge the gap between hope and cures.

You can help at bridgetoacure.org/donate.

There’s never been a single drug developed for the number one killer of children. This must change now – Enough is Enough!

How is it possible that there hasn’t been one drug developed for the number one killer of children – brain tumors? It’s not because we don’t have the best minds dedicated to finding a cure – we do. No, it’s because these great minds don’t have access to the critical data essential to discovery. At Bridge To A Cure we are working to change that.

Here’s how. There are three components to make critical data available:

• Data collection
• Data storage
• Data access

In this month’s blog, we’ll address Data Collection. Bridge To A Cure is working to establish what data needs to be collected and in what format. The Pediatric Cancer Data Commons (PCDC) is one of the most important organizations working toward Bridge To A Cure’s goal to build a robust database to help beat childhood cancer. Through this effort, we’re creating a data dictionary for childhood brain tumors so that the healthcare community uses a signal, unified vocabulary in recording data.

Creating a central database to serve as a one-stop shop for all childhood cancer researchers starts with developing uniform standards for data collection and labeling for participating researchers to follow.

We’re taking an important first step in creating a data dictionary for childhood brain and central nervous system tumors thanks to the expertise of Dr. Sam Volchenboum and the PCDC team at the University of Chicago. The dictionary is a detailed list of data fields with definitions and metadata details, such as data type, source, and units of measurement. These universal standards will:

• Ensure that researchers can query the data with reliable results.
• Speed up future data collection by enabling automated and secure data extraction from electronic health records.
• Allow researchers to combine and analyze data from multiple sources — even across oceans and language barriers.

The total cost to create the pediatric brain tumor dictionary is estimated at $540,257 over three years. PCDC estimates it will take this amount of time to reach the fundraising goal and complete this project.

What is a data commons?

A data commons is defined as a cloud-based infrastructure that includes storage for data and the computational resources and tools to analyze it. The research community can submit, request or download data and collect and analyze it on the commons infrastructure.

“By facilitating these tasks, the presence of a data commons relieves the need for the researcher to purchase and manage local storage, compute, or processing tools,” the American Society of Clinical Oncology notes.

Why a data dictionary is critical

We recently spoke with Dr. Samuel L. Volchenboum, pediatric oncologist and principal investigator at the Volchenboum Lab at the University of Chicago that hosts the PCDC, to learn more about the data dictionary. Dr. Volchenboum is also a co-principal investigator on the NCI’s Center for Cancer Data Harmonization (CCDH), which is developing the data model.

As we’ve noted, several organizations working toward creating the database that we believe is a key to curing childhood cancers and finding more humane and effective treatments. They include the National Cancer Institute’s Childhood Cancer Data Initiative (CCDI).

Volchenboum is advocating for the Pediatric Cancer Data Commons and the Center for Cancer Data Harmonization to work together closely. For instance, the harmonized data model CCDH is creating, with significant involvement of the UChicago team, will cover all types of cancer — both pediatric and adult. CCDI has also contracted with UChicago to integrate PCDC data dictionaries into the harmonized CCDH data model. This demonstrates the NCI’s “commitment to utilizing our data dictionaries for this national effort,” Volchenboum says.

Currently, data from children with central nervous system cancers is spread across several registries. Volchenboum and his UChicago teams are working with these registries, exploring ways to remove patient-identifying information from datasets so they can be imported into the data commons.

Developing a global data language

Developing a data dictionary is a time-consuming but critical step toward creating a data commons. According to Volchenboum, getting international consensus on data elements is a cornerstone of the childhood brain tumor dictionary project’s success.

“Prior to COVID, the PCDC team hosted international meetings where disease experts convened to debate and discuss every aspect of these data elements,” he said. “This work has transitioned to online meetings, but the format remains the same.”

Facilitated by the University of Chicago team, participants from countries around the world discuss each proposed data element. Once the dictionary is built, there will be an open comment period followed by a vote to adopt the dictionary.

“This entire process can take up to a year — sometimes more, depending on the complexity and size of the dictionary,” Volchenboum said.

After that, the PCDC team will work with the NCI’s terminology team to keep everything up to date so that the work can be propagated forward to future clinical trials.

“The ultimate goal of building the commons is to lower barriers to research, leading to new discoveries and better outcomes for children with cancer,” he says.

To hear from Bridge To A Cure Foundation Founder Bob Martin on our approach to accelerating brain tumor cure advancement, click here.

Conventional metrics for gauging nonprofits fall short; it’s time to reward them based on collaboration and getting the job done.

We are a generous society. We Americans donate an estimated $400 billion of our hard-earned money each year to more than 1.5 million nonprofits that employ more than 10% of our workforce. Seems good, but are we getting our money’s worth?

People often choose to support a particular nonprofit based on a personal connection. Or it may be a cause that captures their imagination and emotion. Rarely do they research whether or not a charity is worth investing in.

The decision-making criteria we apply for investing in nonprofits is not the same as the criteria we apply for investing our savings in a company. Yet we can learn a lot from the way investors evaluate corporations.

Evaluating the merit of investing in a specific charity is harder than evaluating a corporation due to lack of reliable agencies that can provide meaningful and actionable data. There are only a few agencies that rate nonprofits. The largest has 20 employees and 12 analysts. That translates to about 75,000 nonprofits for each analyst. As a result they rely on one data point — percent of overhead. Yet this overly simplistic measure fails as a meaningful metric for several reasons.

Nonprofits, like for-profit corporations, need to invest in people and technology to be effective. But nonprofits are also different and need to be rewarded accordingly.

For instance, shouldn’t they receive high marks for collaborating, rather than competing with other nonprofits, to get things done? What should be the criteria for assessing the merits of nonprofits?

At Bridge To A Cure Foundation, we have been researching what differentiates nonprofits that have been successful from those that have not. Among our findings, the most effective nonprofits:

• Identify the problem in a fact-based, clearly articulated, and compelling way.
• Have an audacious and clearly stated goal that is measurable and inspirational. Too often goals are written that are not measurable — “helping kids do better” is not a goal.
• Set a deadline. They declare a specific time horizon to achieve the goal and each milestone along the way. Too many nonprofits avoid timelines for fear of losing funding if they don’t deliver. Well, they should!
• Rely on sound strategy. They map out processes in a holistic and systematic way, identifying key players, bottlenecks, issues and opportunities. They track and report on milestones. When it comes to resources, they understand there is no substitute for allocating and dedicating the resources and talent required to deliver the goal. They don’t dilute their plans for fear that the investment required will be rejected.
• Collaborate. They don’t just look at what they are doing but at the collective impact of all the players/entities seeking the same goal. They come together with a common agenda to define the problem and create a shared vision to solve it. Too many nonprofits don’t work together. This not only dilutes progress, it forgoes opportunities for cost-savings.

Today, 86% of donors believe that low overhead defines good charities. Only 6% of donors want to know if their donation is making a difference.

These are the donors who take the time to understand the nonprofit’s goal, strategies and progress, and how they learn from failures and reach goals. Yet many nonprofits are not making the difference they could when it comes to addressing society’s longstanding problems. Consider:

• Poverty has been stuck at 11-15% since the 1970s.
• There are five million more kids living below the poverty level now than there were in the 1960s when LBJ launched the war on poverty.
• One in five Americans is functionally illiterate, and it’s been that way for 30 years.
• The suicide rate has increased almost 30 percent since the 1990s.
• About 16% of America’s children live in households below the poverty line; for kids age 3 and younger, it’s 21%.
• In the past 40 years only four cancer treatments have been developed specifically for children.

In our next blog we will apply this criteria to explain the flaws within the childhood cancer nonprofit community and our plan to correct them. In the interim, please know that when it comes to achieving an audacious and challenging goal like cutting the childhood cancer death rate 50% by 2030, we at Bridge To A Cure Foundation will keep pushing to challenge the status quo that has left too many children and families without hope. We are thankful for our supporters, and ask for your continued support in the year to come as we push for a more effective approach to childhood cancer research and funding.

Young lives are lost daily because we’ve put blinders on research;
a National Pediatric Database will break down silos and shine light on finding cures

The cures for pediatric cancer and other terminal and chronic diseases are out there waiting to be discovered. Do we have the vision to find them?

As we examine what it will take to end the scourge of pediatric cancer and other “incurable” illnesses that strike kids, we’ve looked at what the following entities must do to change:

• Investors
• Donors
• Government (the National Cancer Institute)

Today we’ll look at research being conducted to fight pediatric diseases through public and private institutions. This is a vast area that encompasses work at major cancer research centers, universities, and children’s hospitals across the US.

There are hundreds such institutions working on different aspects of childhood health and disease – so many that no one seems to know exactly how many there are. For instance, there are more than 220 children’s hospitals where various forms of research are ongoing.

Yet despite all the research that’s going on, and billions of dollars spent, “the causes of most childhood cancers remain unknown,” according to NCI. This is one reason why cancer remains the leading cause of death from disease among children and adolescents in the US.

“Although substantial progress has been made in the treatment of several types of childhood cancer over the past five decades, progress against other types has been limited,” NCI states.

Research on childhood cancers and other diseases tends to be narrowly focused on specific drugs, treatments, and genetic or other causal factors. For this reason, there is good chance we may be missing the forest for the trees when it comes to finding cures.

For instance, the National Institutes of Health announced last year it had formed a new Trans-NIH Pediatric Research Consortium to coordinate pediatric research programs across its institutes and centers to “advance pediatric research on a global level.”

“The new consortium aims to harmonize these activities, explore gaps and opportunities in the overall pediatric research portfolio, and set priorities” among the 27 NIH institutes and centers that fund some aspects of child health research.

Yet when we at Bridge to a Cure contacted the consortium to find out if they were participating in building a National Pediatric Cancer Database, a spokesman referred us to NCI, saying, “This is an initiative of the National Cancer Institute.”

The consortium is made up of NIH officials. NCI is part of NIH. Yet they did not seem to be aware of what we believe is the most promising avenue for results in curing pediatric cancers and other diseases. This despite the fact that in FY 2017, NIH funded pediatric research grants and projects totaling almost $4.2 billion out of its annual budget of about $37 billion.

The main purpose of the NIH consortium is “to identify opportunities for collaborative research, identify gaps that need to be addressed, and try to harmonize the overall research effort at NIH directed toward child health.” So they should be deeply involved in work to create a National Pediatric Database that would combine everything we know about childhood cancers.

As we’ve pointed out, we need a National Pediatric Database now to empower researchers to:

• Access to all available information on the diseases, drugs and treatments to identify patterns that will help them prevent, diagnose, treat, and ultimately cure cancer and other diseases.
• Use the power of Artificial Intelligence and Machine Learning to search for synergies among existing conventional and alternative treatments and new ways to attack disease.
• Overcome hurdles posed by the existing patchwork of data, including data from unstructured electronic health records and other other sources collected for disparate purposes. Most existing databases “do not have sufficient quality to be used by AI algorithms to achieve the quality standard required for clinical trials.”

We have researchers with great minds spending mountains of money, yet there are many children’s diseases for which we don’t have a cure. This is unacceptable when we consider:

• In the United States in 2019, an estimated 11,060 new cases of cancer will be diagnosed among children from birth to 14 years, and about 1,190 children are expected to die from the disease.
• The most common types of cancer diagnosed in children ages 0 to 14 years are leukemias, brain and other central nervous system (CNS) tumors, and lymphomas.
• Ten to twenty million children and adolescents in the United States have some form of chronic illness or disability.

I believe the reason we don’t yet have cures or effective treatment for so many childhood illnesses lies in the fact that research today still lacks the unified database needed to tie together everything we know about these diseases.

It’s time for leadership of research institutions – whether they are cancer centers, universities, children’s hospitals, or other entities – to realize how a comprehensive national pediatric database could be a game-changer.

I am totally focused on building a national database that has everything we know about the specific diseases and every drug or treatment that could be used to attack those diseases, including:

• Causes of disease (still unknown for many forms of childhood cancer).
• Weaknesses in the diseases themselves (the approach most research centers take today.
• Bolstering the immune system and other systems such as angiogenesis and apoptosis to defend against disease.
• Fighting the energy sources that fuel disease processes.

To be truly comprehensive, a National Pediatric Database must also include data from every medical file of every child that has a specific disease, and information on non-traditional treatments and how they interact with conventional medicine. Right now we don’t have this data on conventional – let alone alternative – treatments. This is the system we must build if we are serious about stopping the diseases that rob children of their lives and their futures.

Our research institutions are full of skilled and dedicated people who want to achieve these breakthroughs. We must give them the data and the technological support they need to make sense of it. Artificial intelligence and quantitative analysis will give us the ability to hunt for synergies in drugs and treatments that hold the keys to cures.

Think about it: These cures could be staring us in the face. Will we have the vision to see them?

As a grandparent, I’m always trying to think of new ways to connect with my grandkids—new ways to encourage them, help them grow and thrive, and support them in having a happy, fulfilling life.

When my granddaughter Clara was diagnosed with terminal brain cancer, I dedicated my efforts to supporting her parents’ tireless journey to find a cure. At first, we dove into research to find out what exactly was out there currently in terms of a cure for cancer. Sadly, I discovered that there really isn’t a viable cure available right now. On the other hand, though, we found a lot of promising research that is on the cusp of a cure.

We live in exciting times, indeed, when a cure for cancer feels within reach!

The problem, however, is that these exciting scientific breakthroughs still largely amount to a cure that is years away, perhaps even a decade or more. For Clara and other kids and their families who are struggling with the disease now, those years can feel insurmountable… yet all the research has been pointed solely at finding a cure. We couldn’t find any institutions working on a way to sustain the lives of these kids who just need more time.

Inspired by our SuperClara, we decided this had to change.

To that end, I am so excited to be able to announce the creation of the Bridge to a Cure Foundation. Its sole purpose is to fund research for life-sustaining treatment for terminally ill kids, and it is the first-ever effort of its kind. Presentations by many renowned research institutions on how they would support the effort have already begun and will continue throughout the year.

Perhaps most exciting of all, Bridge to a Cure is a tangible way that all of us who care about these children to put our energy into something that has a direct impact on their well-being. It’s a chance for us to let go of the fear and hopelessness that so often take hold and instead focus on life and hope.

Hope paired with positive action can change the world. Will you join us?