IT’S CHILDHOOD CANCER AWARENESS MONTH

HERE’S WHAT MATTERS MOST!

Pediatric glioblastoma is among the most lethal and devastating childhood cancers. My family has lived this journey Documentary Film.mov – Google Drive

The truth is stark: progress has been painfully slow—not for lack of brilliant scientists or promising technologies, but because too many institutions choose competition over collaboration.

Prestigious centers are hoarding patient data, research findings, and funding. Some are even diverting scarce dollars away from hospitals that urgently need support, while building financial reserves far beyond what is required.

The result? Duplication of effort. Fragmented silos. Wasted time. Vast regions of the country without adequate resources. And while institutions protect their turf, children continue to die from a disease that has seen little meaningful progress in decades.

Over the past 50 years, billions of dollars and countless hours have been invested. Yet the return on that investment has been negligible. The culprit is clear: a siloed culture that donors have—often unknowingly—funded. We all know what must change.

If a cure is to be found, collaboration and transparency must replace competition and concealment. Institutions must explicitly commit to collaboration in their mission statements. This demands a culture shift—and donors have the leverage to make it happen.

What You Can Demand as a Donor:

• Open Data Sharing – Require institutions to share patient and research data without delay or restriction.
• Collaborative Trials – Expand access for families by insisting on multi-institutional clinical trials.
• Collective Investment – Direct funding toward shared infrastructure—biobanks, genomic commons, AI platforms—where every dollar multiplies impact.
• Accountability – Ensure donor intent is honored; funds must advance cures, not inflate reserves.

Every child lost is a reminder that fractured efforts cost lives. A cure will not emerge from one laboratory or one hospital; it will come only from a unified front.

Your investment is precious. Make it count—insist that it fuels collaboration, transparency, and urgency. Together, we can demand results. Make your pledge below.

Click to sign the Donor Pledge for Collaboration in Pediatric Brain Cancer Research

CHILDHOOD CANCER AWARENESS MONTH DONORS

IT’S CHILDHOOD CANCER AWARENESS MONTH

RESEARCH INSTITUTIONS ARE THE BARRIER!

NOT THE RESEARCHERS

Pediatric glioblastoma remains one of the most devastating childhood cancers. My family has lived this journey Documentary Film.mov – Google Drive

The greatest obstacle to progress is not science or technology—it is culture. Too many prestigious institutions hoard data, guard funding, and even divert resources from hospitals that struggle to meet the needs of their patients. The result: duplication, silos, wasted resources, and entire regions left without adequate care. While institutions protect their turf, children are dying from a disease that has seen little meaningful progress in decades.

Why Change Is Non-Negotiable

• Rarity demands scale. No single center sees enough cases or holds enough data to solve this disease. Only pooled knowledge and shared resources can yield statistically valid insights.
• Collaboration accelerates progress. Unified trials and infrastructure reduce duplication, broaden patient access, and generate results in years—not decades.
• Resources are scarce. Every dollar consumed by competition or redundant effort is a dollar not spent advancing cures. Shared platforms ensure maximum impact.
• Mission must outweigh prestige. Institutional rivalries cannot be allowed to determine life-or-death outcomes for children.

For fifty years, billions of dollars and countless hours have been invested with negligible return. Progress has been stalled not by lack of ability, but by a siloed, protective culture. We know what must be done:

• Open, unrestricted data sharing across all centers.
• Shared infrastructure for biobanks, genomic data, and AI platforms.
• Joint clinical trials that expand access and accelerate results.
• Transparent research sharing to prevent duplication and expand the data pool.
• Equitable funding distribution that strengthens resource-limited hospitals.

If a cure is to be found, collaboration and transparency must replace competition and concealment. Every institution should embed transparency and collaboration in its mission.

The Path Forward

Change will not occur by good intentions alone. Institutions are unlikely to move independently. What is required is a pediatric glioblastoma summit—a forum where leaders agree on principles, establish timetables, and commit to implementation.

Childhood Cancer Awareness month is the time for each institution to pledge their support for such a summit; a summit that will knock down barriers, one that will advance collaboration, transparency, and urgency. Make your pledge below.

Click to sign the Institution Pledge to Drive Collaboration and Transparency

CHILDHOOD CANCER AWARENESS MONTH RESEARCHERS

Bridge To A Cure’s reimagined approach to childhood brain tumor cancer was presented to the National Cancer Institute on October 5, 2017. The core imperatives driving the approach are:

• Data: Fuel a robust, multiomic brain tumor data ecosystem shared freely among all researchers globally.
• AI: Employ generative AI (artificial intelligence) throughout the research process.
• Nontoxic Treatments: Develop nontoxic treatments that target pediatric brain tumor cancer cells via the immune system, angiogenesis or apoptosis.

Thanks to NCI’S support and advocacy, this approach has been enthusiastically embraced by the broader pediatric cancer community, with our partners at the Children’s Brain Tumor Network (CBTN) leading the pack.

The team at CBTN, led by Dr. Naqvi, leveraging data and AI has successfully found a solution to targeting tumor cells without harming normal brain cells. This was just published by the Cell Report, a prestigious research journal that publishes meaningful breakthroughs that the entire research community has open access to.

Scientists studying aggressive childhood brain tumors (pediatric high-grade gliomas, or pHGGs) face a problem: there aren’t many unique “flags” on the tumor cell surface that can be targeted by treatments without harming normal brain cells.

To find new treatment targets, researchers looked at the differences in how cancer cells and healthy brain cells read and edit genetic instructions. They noticed that in tumor cells, some tiny bits of genetic code—called microexons—were missing from certain outer-layer proteins. Many of these proteins help nerve cells connect and talk to each other, including one called NRCAM.

NRCAM is a protein that sits on the surface of nerve cells and acts like Velcro, helping them stick to and communicate with each other so the brain’s wiring works properly.

In nearly every tumor sample tested, two specific microexons (numbers 5 and 19) were missing from NRCAM. This altered version of NRCAM wasn’t just different—it was critical for the tumor’s ability to spread and grow. When researchers made an antibody that specifically recognized this altered NRCAM, they could “mark” the tumor cells. Then, specially engineered immune cells were able to find and destroy them.

This discovery suggests that the altered form of NRCAM—and possibly other similar proteins—could be highly precise targets for new cancer treatments that train the immune system to attack only the tumor cells.

Childhood cancer is a life-altering diagnosis for families. Facing it requires a multi-pronged approach, and thankfully, new technologies are emerging to offer glimmers of hope. One such innovation is the concept of digital twins, virtual replicas of a patient’s biology used to personalize treatment plans and potentially revolutionize pediatric oncology.

What are Digital Twins in Healthcare?

Imagine a virtual version of yourself, a computer model incorporating your unique genetic makeup, tumor characteristics, and treatment response. This is the essence of a digital twin in healthcare. These models create a dynamic representation of a patient’s health by integrating various data sources like medical imaging, genetic analysis, and real-time biometrics collected from wearable devices.

In the context of childhood cancers, digital twins hold immense potential. These “virtual patients” can be used to:

• Simulate Treatment Responses: Doctors can run simulations on the digital twin, testing different treatment options and predicting their effectiveness. This allows personalized therapy plans tailored to the specific cancer and the child’s unique biology.
• Predict Treatment Side Effects: Digital twins can forecast potential side effects of various treatments, enabling doctors to choose the most efficacious option with minimal adverse effects, improving a child’s quality of life during treatment.
• Develop New Therapies: Researchers can utilize digital twins to test the efficacy and safety of new drugs in a virtual environment before clinical trials. This reduces risks and accelerates the development of more effective treatments.

New Technologies Propelling the Digital Twin Revolution

The power of digital twins lies in their ability to leverage a confluence of cutting-edge technologies:

• Artificial Intelligence (AI): AI algorithms analyze vast amounts of patient data, identifying patterns and more accurately predicting treatment outcomes.
• Machine Learning (ML): ML models learn from existing data on childhood cancers and treatment responses, continuously improving the predictive power of digital twins.
• Big Data Analytics: The ability to collect, store, and analyze vast datasets, including genomic information, medical imaging, and treatment response data, is crucial for building robust digital twins.
• High-Performance Computing: Simulating complex biological processes within digital twins requires immense computational power, which high-performance computing provides.

The Impact on Childhood Cancer Treatment

The potential benefits of digital twins for childhood cancers are multifaceted:

• More Targeted Therapies: Digital twins pave the way for precision medicine, where treatments are customized based on a child’s needs. This can lead to higher cure rates and improved long-term outcomes.
• Reduced Treatment Burden: Doctors can choose gentler therapies by predicting side effects, minimizing the child’s physical and emotional toll.
• Faster Drug Development: Simulating new drugs in a virtual environment allows researchers to identify promising candidates faster, ultimately leading to more effective therapies reaching children in need sooner.
• Improved Patient Care: Digital twins empower doctors to make more informed decisions, improving patient care and family communication.

Challenges and the Road Ahead

While digital twins hold immense promise, there are hurdles to overcome:

• Data Security and Privacy: Safeguarding sensitive patient data used to create digital twins is paramount. Robust data security protocols are essential to earn patient trust.
• Model Development and Validation: Building accurate and reliable digital twins requires extensive data and ongoing validation to ensure their efficacy.
• Infrastructure and Accessibility: Not all healthcare institutions might have the resources and infrastructure needed to implement digital twin technology. Ensuring equitable access will be crucial.

Despite these challenges, the potential benefits of digital twins in treating childhood cancers are undeniable.

As research progresses and technology advances, Bridge To A Cure Foundation expects digital twins to become a cornerstone of childhood cancer treatment, offering a brighter future for children battling this disease.

The Future of Digital Twins in Healthcare

The impact of digital twins extends beyond childhood cancers. This technology has the potential to revolutionize the entire healthcare landscape, and Bridge To A Cure is helping to foster its use. Imagine a future where digital twins are used to manage chronic diseases, predict health risks, and even prevent illnesses. While this vision might seem distant, the strides in digital twin technology in pediatric oncology offer a glimpse into a future where personalized medicine empowers doctors to fight diseases more effectively, leading to a healthier future for all.

The nonprofit sector in the United States boasts a staggering number of over 1,540,000 organizations, each dedicated to various causes. But not all nonprofits are equal. The disparate nonprofit landscape underscores the challenges for small nonprofits: only 3% generate revenue above $5 million. These nonprofits are large national institutions, many with revenue in the billions, and with tremendous influence. Most of the remaining 97% raise less than $99,000 a year.

Why Smaller Foundations Fail

While size is one reason many nonprofits struggle to achieve meaningful impact there are other perhaps more important reasons. The challenges that plague the sector, and why a substantial portion of these organizations are not effective are attributed to operational shortfalls and inapt strategies.

Operational Shortfalls

Most nonprofits are started by someone touched by a personal tragedy or motivated by an injustice. While their hearts are in the right place, their skill set may not provide what is necessary to build, operate, and grow the organization in a way required to be successful. Effective leaders are visionary, focused, and create a culture of innovation: important attributes to be successful. Conversely, inexperienced or unqualified leadership can drive a nonprofit into trouble from the outset.

Many smaller nonprofits lack the knowledge or funding to integrate efficient technology solutions into internal workflows, fundraising, communications, and finance operations. As a result, they don’t have the time to focus on the initiatives that help them achieve meaningful progress. Further, those foundations focused on medical cures should include machine learning (AI) as a core element of their discovery process. Very few have this capability or are considering it, leading to a failure to optimize operations.

Inapt Strategies

Unfortunately, many smaller nonprofits have not taken the time to develop a mission statement, or they’ve drafted amission that is not audacious or measurable. One of the primary issues facing nonprofits is the lack of clear challenging goals and measurable outcomes. Without well-defined objectives, organizations often find themselves adrift, unable to demonstrate tangible results to their stakeholders. Additionally, an overemphasis on fundraising can divert attention from the core mission, turning nonprofits into entities more focused on sustaining themselves than effecting change.

Further, there is prevalent absence of collaboration and partnerships. All nonprofits should collaborate and build meaningful partnerships that place the goal ahead of the institution’s drive for recognition. This is as true for the 3% of the largest nonprofits as it is for the 97% that are minimally funded. No matter the size, it should be a strategic imperative for nonprofits. Sadly, it is not. For smaller nonprofits, they become burdened with excessive overhead and have zero influence. While the founders and supporters feel good about what they advocate, they achieve little.

As a result of operating shortfalls and inapt strategies, combined with the larger nonprofits dominating the nonprofit community, half of the smaller nonprofits fail, and those that survive have minimal impact, if any.

Why Bridge To A Cure Has Flourished

The progress highlighted in our  2022 annual report  took many by surprise; we welcomed the accolades. Accompanying the accolades were often followed with the same question: Given Bridge To A Cure’s modest revenue stream, how has it been able to help propel advancements in pediatric cancer, specifically childhood brain tumor cancer? Or how is it that the Bridge to a Cure Foundation is dramatically overperforming versus the status quo?

Simply, Bridge To A Cure excels at each of the operational and strategic imperatives identified above.

Operational Excellence

Bridge To A Cure Foundation has been built by qualified personnel: seasoned leadership, board of directors members, and advisors from industry and childhood cancer research with a track record of success. Bridge To A Cure executives apply years of experience competing, developing strategic advantages, optimizing resources, creating cultures of innovation, building strategic partnerships, responding quickly to challenges and opportunities.

Robust technology also is crucial to Bridge To A Cure success. We use multiple cloud-based applications to manage what is “backroom” operations: finances, fundraising, communications, and market research. This allows us to focus on building partnerships that will advance our mission and influence key decision-makers to drive the change needed to transform the childhood cancer community’s approach to research.

Effective Strategies

Our mission inspires us emotionally and creatively, it serves as a guideline for collaborating and partnering with like-minded organizations and targeting the right audience for support. Our strategies rely on strong strategic partnerships, with the goal of getting the right data and the right technology in the right hands. And, we are focused, we will cure childhood brain tumor cancer.

We have a mission that is audacious and measurable: To unite and transform the childhood cancer community’s approach to research with the goal of cutting childhood cancer death rate 50% by 2030.

Collaboration and strategic partnerships have been strategic imperatives since the begging. Today, we collaborate with over 20 other childhood brain tumor cancer nonprofits and several of the largest nonprofit institutions, including a partnership with the largest childhood brain tumor organization in the world. We have attracted board of director members and board of advisors that are leaders in industry and childhood cancer research. We have the loyal support of 400 donors and over 2,000 followers that are champions of our mission and appreciative our progress.

In our determination to end childhood cancer, Bridge To A Cure Foundation, in collaboration with its partners, has made remarkable advancements this past year. Through a multifaceted approach that emphasizes collaboration, open science, and the application of groundbreaking technologies, the foundation has brought together well-positioned individuals, forged vital partnerships, harnessed the power of data-driven research, and achieved transformative breakthroughs. These accomplishments have improved patient care and treatment outcomes and propelled advancements in the broader healthcare research field.

Exponential Impact in 2023

While a $250,000 foundation might seem modest, Bridge To A Cure has exponentially multiplied our impact in 2023 by targeting research and nonprofit organizations that share our mission and have the scientific resources and government influence to achieve it. And, we contributed strategic planning and communication skills found at senior executive levels within the business community. Over the past few years, these targeted organizations have come to rely on us to play this role. In doing so, Bridge To A Cure has become a crucial player in the childhood cancer research landscape, facilitating progress far beyond its financial size.

Empowering Key Individuals

The foundation supported our former executive director, Wendy Payton, as she transitioned to an executive role at the Children’s Brain Tumor Network (CBTN). Bridge To A Cure Foundation plays an active role at CBTN as an Executive Council member. In addition, Foundation president Robert Martin sits on its Executive Committee. Wendy’s transition to CBTN further enhances the foundation’s impact of bringing its vision and strategic objectives to a wider audience within the CBTN, fostering collaboration and pushing for progress. Wendy continues her involvement with Bridge To A Cure as a member of our board of directors.

In addition, we welcomed new board member Nan Smith, who contributes valued perspectives from her expertise as a cancer patient advocate and research patron. Smith brings fundraising acumen built over 30 years of philanthropic projects, including the Hackers for Hope charity golf tournament, which raised over $20 million for cancer research programs. Her experience includes leadership positions at innovative nonprofit organizations Americares and Changing Our World. The addition of Nan to the board amplifies the caliber and expertise inherent within Bridge To A Cure Foundation leadership.

Strengthening Partnerships

CBTN expanded our reach in 2023 by welcoming two new pediatric healthcare member institutions, Children’s Healthcare of Atlanta and Maria Fareri Children’s Hospital, part of the Westchester Medical Center Health Network. And, CBTN is working to align with six additional institutions in the onboarding stage of membership. This exponential growth signifies the increasing recognition of the collaborative model and the trust bestowed upon CBTN to drive this strategy.

This year we marked the milestone of 5,000 child patient enrollees into CBTN, accompanied by the additional participation of now over 2,500 family members. Such levels of engagement have fortified the CBTN with an extensive dataset crucial for researchers across the globe. We do not celebrate this achievement because it means we have not yet accomplished our mission. But we honor these courageous children and families for their belief that what we are all doing together will help us end childhood cancer once and for all. Through this collective effort, Bridge To A Cure is helping to revolutionize childhood cancer research and providing researchers with the tools they need to advance progress.

Additionally, a significant milestone was achieved through a partnership forged between CBTN and Amazon Web Services (AWS) to drive AI innovation in patient care. Leveraging AWS’s cutting-edge technologies, the world’s largest data repository of its kind at CBTN, this powerful collaboration is working to develop individualized treatment protocols for doctors and their patients while empowering scientists and researchers worldwide with new cloud-based tools for data-driven discovery.

Harnessing Technological Expertise

Recognizing the potential of cloud-based computing and artificial intelligence (AI), the experts within the CBTN network have applied these technologies to not only drive childhood cancer research but also revolutionize broader healthcare research. By leveraging the power of AI and cloud computing, researchers are unlocking new insights into disease mechanisms, genetic variations, and potential treatment pathways. This interdisciplinary synergy is poised to deliver transformative outcomes in the quest for cures.

Influential Voices in Research

CBTN’s Co-Executive Director, Dr. Adam Resnick, has emerged as a prominent figure in data-driven bioscience discovery. He regularly makes appearances at patient advocacy and scientific conferences, including participation in the White House Cancer Moonshot gathering this past fall. His colleague Dr. Allison Heath, CBTN Director of Data Technology and Innovation, and others from CBTN also led engaging and thought-provoking presentations within the scientific research community this past year. This included participation in a prominent panel discussion at the AWS Summit in Washington, D.C. in September, where she joined leaders from AWS, the NIH National Cancer Institute, and the White House Cancer Moonshot. These discussions gained significant traction around the topics of how cloud-based open science research models are eliminating silos and allowing scientists across the globe to analyze – in real-time – medical records and other data to accelerate research and aid in the development of new therapies.

Transformational Research Progress

Significant progress has been made through transformative research initiatives in various domains. Notably, an imaging study utilizing AI has provided three-dimensional tumor images, aiding in the detection of tumor changes faster than traditional radiology methods alone. This breakthrough expedites diagnosis and treatment decisions, resulting in improved patient outcomes as timely and accurate interventions become possible. It signifies a pivotal step towards personalized, effective treatments and realizing the foundation’s overarching goals. It’s just a glimpse of what is possible through AI technology and open science.

In its collaborative efforts, Bridge To A Cure Foundation has delivered remarkable achievements in 2023, vastly outperforming what many thought possible for a smaller foundation such as ours. The alignment of key individuals, strengthening partnerships, technological advancements, amplification of influential voices, and transformational research breakthroughs reflect the foundation’s unwavering commitment to driving cures for children with cancer as we move forward into 2024.

And just as Bridge To A Cure could not make these advancements alone, neither could we continue to operate without the giving and advocacy of our friends and donors. It takes all of us doing our part, and we are so grateful for your support and encouragement over the past 6 years. Through the application of cutting-edge technologies and the global collaboration facilitated by Bridge To A Cure Foundation initiatives and upheld by you, hope shines brighter than ever for the future of childhood cancer research and beyond.

In this, the third of our three-part series on leading-edge research in childhood cancer, Bridge To A Cure informs of a novel approach to research in which we are investing. We’ve taken a look at scientific approaches in the areas of immunotherapy and angiogenesis. Now, let’s explore what else researchers are uncovering within kids’ own bodies to combat challenging and often aggressive childhood brain tumors, apoptosis.

Apoptosis: Programming Cancer Cell Death To Save A Life

Often called “programmed cell death,” apoptosis is a crucial process in developing and maintaining healthy tissues in the body. It allows the body to remove cells that are no longer needed or that could potentially become harmful, such as damaged, diseased, or cancerous cells. In the context of childhood brain tumors, leveraging apoptosis has emerged as a promising therapeutic strategy.

Childhood brain tumors represent a diverse group of diseases that vary significantly in their prognosis and treatment options. Traditional treatments, such as surgery, radiation, and chemotherapy, often referred to as “standard of care,” has been devastating to the children with brain tumors and their parents:

The Journey is Horrific

• Frightful, excessive, and severe chronic pain
• Excruciating and harmful emotional strain
• Work disruption/Financial devastation
• Ongoing deep psychological damage

Those that Survive, Suffer

• 95% who survive have significant health-related issues later in life
• The emotional strain continues
• Occupational/Financial recovery is a serious challenge

The strategy of inducing apoptosis in cancer cells offers a targeted approach to therapy, aiming to minimize harm to healthy tissues while effectively eliminating cancer cells.

Cancer cells often evade apoptosis, which is one of the reasons they can grow uncontrollably. By understanding the molecular mechanisms that underlie this evasion, researchers will be able to develop drugs and therapies that can specifically trigger apoptosis in cancer cells.

One approach to induce apoptosis in childhood brain tumors is with targeted therapies that home in on specific genetic mutations or pathways that are active in cancer cells but not in healthy cells. For example, some drugs can target the proteins involved in the pathways that regulate cell survival and death, effectively reactivating the apoptosis process in cancer cells.

Another promising strategy involves immunotherapy, which enhances the body’s immune response against cancer cells. Certain types of immunotherapies can help recognize and destroy cancer cells by triggering apoptosis, offering a more personalized and effective treatment option for pediatric brain tumor patients.

Gene therapy is also being explored to correct the genetic mutations that prevent apoptosis in cancer cells. By repairing or replacing the defective genes, this approach seeks to restore the natural process of programmed cell death, thereby reducing tumor growth and potentially leading to a cure.

The use of apoptosis in treating childhood brain tumors represents a significant shift towards more targeted and less toxic treatments. While research in this area is ongoing, early clinical trials and studies have shown promising results, offering hope for more effective and safer treatments for young patients with brain tumors. Bridge To A Cure wants to accelerate progress in this area via a research initiative that draws on the databases now available and the analytical/diagnostic capabilities of artificial intelligence. The goal is to replace the horrific standard of care approach used today with an approach that is nontoxic and that cures.

Our goal for a childhood brain tumor cancer cure by 2030 continues to progress. Our enthusiasm and optimism about the potential of artificial intelligence (AI) in revolutionizing the search for a cure for childhood brain tumor cancer is why. The intersection of advanced technology and medical research holds immense promise, and AI stands out as a powerful tool in this endeavor.

AI algorithms can significantly enhance the analysis of vast and complex datasets related to childhood brain tumors. The ability of AI to process and identify patterns in large sets of genetic, clinical, and imaging data allows researchers to unravel intricate connections that may contribute to the development and progression of these cancers. This, in turn, accelerates the identification of potential therapeutic targets. Thanks to our alliance partner at the Children’s Brain Tumor Network (CBTN), these data sets are now available to researchers worldwide.

Indeed, AI-driven image analysis plays a crucial role in the field of medical imaging, aiding in the early detection and precise monitoring of brain tumors.

By leveraging machine learning algorithms, medical professionals can obtain more accurate and efficient results from imaging studies. This expedites the diagnosis and facilitates timely interventions, thereby improving the prognosis for young patients.

In one promising study by CBTN, 250 brain tumor subject scans were used to generate an AI algorithm. This algorithm generated 3-dimensional segmentations automatically, making note of any tumor progression. This model effectively captured tumor progression earlier than the traditional method in more than half of the cases.

Additionally, the application of AI in drug discovery is a game-changer for developing targeted therapies. By simulating and predicting the interactions between various drug compounds and specific cancer biomarkers, AI expedites the identification of potential drugs that could effectively combat childhood brain tumors. This approach not only reduces the time and resources required for drug development but also increases the likelihood of finding treatments tailored to the unique characteristics of each patient’s cancer. In fact, time has been reduced from 12 years to 4 years. Truly a game-changer.

AI enables the personalization of treatment plans through the analysis of individual patient data. This tailoring of therapies based on a patient’s genetic makeup, response to treatment, and other relevant factors enhances treatment efficacy while minimizing potential side effects. The era of precision medicine, powered by AI, offers a more targeted and less invasive approach to treating childhood brain tumors.

The integration of artificial intelligence into the realm of childhood brain tumor cancer research holds immense promise. From deciphering complex datasets to improving diagnostic accuracy, streamlining drug discovery, and enabling personalized treatment strategies, AI stands as a catalyst for innovation in the quest for a cure. As we continue to explore the vast potential of AI in healthcare, we at Bridge To A Cure Foundation are hopeful that these advancements will bring us closer to a future where childhood brain tumors are not only treatable, but ultimately curable.

It’s a Big Step, But it’s Still Just a Step.

This past month Bridge To A Cure partner Children’s Brain Tumor Network received government support for the genetic research of childhood tumors.

Finally.

As we all know too well, very little federal funding and support (only $1.7 billion of an annual NIH budget that exceeds $42 billion) has been allocated to childhood cancer in the past.

Now, with this government support, the day is drawing closer to when we will unlock the genetic mysteries behind pediatric brain tumors, accelerating the research process.

What does this new support look like?

It doesn’t look like money, but it does look like a hope for an improved future for childhood cancer patients. It looks like progress. It looks like the opportunity we’ve needed to accelerate our search and discovery of new and more effective treatments explicitly designed for kids.

Rather than a monetary grant, the X01 Sequencing and Genotyping Resource Access program will provide critical NIH-supported resources. These additional resources will allow scientific investigators to better conduct and share their research and studies.

As the NIH X01 program recipient, Children’s Brain Tumor Network (CBTN) can now unleash information concealed in thousands of tissue samples across all pediatric brain tumor types. These samples have been collected from 26 CBTN member institutions since 2011.

Thanks to this new support, we can now use these samples to attain molecular characterization for thousands of these brain tumor samples.

This will provide an unprecedented level of insight and pave the way for future therapeutic interventions.

This characterization process, named “Project Accelerate,” will prioritize the most aggressive tumor types, supporting research for those cancers where there is a lack of existing data.

The two primary goals of this project are to:

1. Empower Discoveries
2. Advance Personalized Medicine

CBTN and its partners will reach these goals by connecting, harnessing, and empowering the world’s diverse expertise through collaboration. And this collaboration will lead to accelerated discoveries in research and enable new clinical trials for clinical impact.

Better data access leads to comprehensive data-driven research.

Cancer research has always been data-driven, but the data hasn’t always been available to all investigators. Now, with this influx of government support and the additional resources available, researchers will be able to access, analyze, and share CBTN data on internationally recognized platforms.

This critical government support facilitates a coming together of childhood cancer learnings worldwide. Its access will transform research globally, leading to more progress at faster rates than we’ve ever experienced.

Accelerating breakthroughs.

The following comparison is based on a CBTN investigator’s scientific discovery project timeline. It shows what can now be a reality.

Children’s Brain Tumor Network Story.

The CBTN is a network of researchers, clinicians, patients, and foundations from around the globe dedicated to creating a world where no child dies or suffers from a brain tumor.

Their purpose is to unlock the genetic mysteries behind pediatric brain tumors and accelerate the research process. And the hope is that this research will lead to more effective treatments and cures.

The Children’s Brain Tumor Network drives innovative discoveries, pioneers new treatments, and accelerates open science to improve the health of every child and young adult diagnosed with a brain tumor.

Bridge To A Cure Foundation’s role.

As a CBTN partner, our role continues to be 3-fold:

1. Help unite and transform the childhood cancer community approach to research by making these millions of data points readily and available to investigators.
2. Design a clinical trial process specifically for children.
3. Implement a new approach to research funding.

Speaking of funding.

We still need your help. While the NIH has stepped up and offered incredible access to its resources and data, we need money to help facilitate this process.

How much? $1.3 million. Over the next 12 months.

Our cause is more than just a cause. Our cause is the lives of our children, grandchildren, friends, and family. And it’s by all working together that we’ll keep this wheel rolling to our goal of cutting the childhood cancer death rate by 50% by 2030.

Click here to become a donor. 

Learn more about Project Accelerate on our website.

We must unify behind families devastated by childhood cancer.

Pharmaceutical companies here in the U.S. invest millions of dollars in cancer drug development, and advances over the past few years from giants like AstraZeneca, Johnson & Johnson, Merck, and Roche are pushing the boundaries on combination therapies, cell therapies, and other novel approaches. This in turn, has pushed biotechnology firms into the realm of investor darlings. With this influx of investor cash, these companies are able to spend millions investing in even rare cancers.

Good news — for adults across the country. Unfortunately, this market dynamic leaves childhood cancer patients out of the picture. Because, although childhood cancer remains the number one cause of death by disease among kids in the U.S., there simply are not enough of these cases to capture investment.

And the federal government has not done much better. Over the past decade, they’ve invested just 4% of the National Cancer Institute’s budget for research into childhood cancers — just 2/10ths of a percent in healthcare spending overall.

Some may assume that breakthroughs for adult cancers are equally effective for those found in children. But childhood cancers are different in origin and presentation, so the therapies developed to cure adult cancers do not translate to childhood forms of the disease.

In addition, the toxic treatments used to cure cancers of, say, a 65-year-old, may work okay to extend that person’s life for few years, which is the goal of that treatment. However, the toxic effects of the treatment on a growing body can be devastating — to future growth and development, reproduction, brain function, nervous system, mental health — and on and on.

In fact, 95% of all childhood cancer “survivors” will experience lasting, sometimes life altering, effects from noxious treatments they were given that were developed back in the 1950s and 60s. This is the sad reality that families and children diagnosed with cancer face: outdated, adult treatment options with horrifically painful side effects. And a whispered truth in childhood cancer: a child may die “cancer-free” from the effects of the harsh treatments that did in fact, eliminate the cancer.

Of those kids who survive, 95% are likely face chronic health conditions, secondary cancers, cognitive impairment, and a shortened lifespan. And for their families? At nine times the cost of the average childhood hospital stay, caretakers of childhood cancer patients also may contend with financial ruin.

That’s why parents and grandparents, aunts, uncles, and other loved ones feel compelled to step up in any number of ways in desperate need to advance treatments and find cures. Families have founded more than 200 childhood cancer foundations in the U.S. Many of these fund specific research projects or give aid to other families financially and emotionally devastated by a childhood cancer diagnosis.

Some have become activists who lobby Congress for better funding, often sharing their stories of pain, disappointment, and struggle with any official or aide who will listen. Some hold local fundraisers from bake sales to car washes for cancer fighting organizations. And some simply share their stories their friends, neighbors, and colleagues — many of whom have no idea that this is the reality of a childhood cancer diagnosis.

The fact is, apart we will not be as effective as we can be together. That’s why Bridge To A Cure Foundation — founded by a grandfather who lost a young granddaughter to a brain tumor — is calling on childhood cancer foundations to unite. We’re calling on the childhood cancer research community to transform the way it conducts research and shares information. And we’re challenging the federal government to do better when it comes to funding childhood cancer research.

We’ve had enough.

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.