Over this past year, Bridge To A Cure has played a key role in accelerating life-saving discoveries for childhood brain tumors through our partnership with the Children’s Brain Tumor Network (CBTN), a worldwide leader in pediatric data collection, sharing, and collaboration.

In fact, every dollar donated through Bridge To A Cure in 2024 strengthened the vital work conducted by our coalition, empowering hundreds of researchers with the tools and infrastructure they need to unlock cures. It further enforces the collaborative spirit that will result in better treatments and, ultimately, a cure for these deadly diseases.

Together, we’re changing how research is conducted and the speed at which discoveries are made.

We’re thrilled to share some of the biggest highlights of 2024:

Expanding Critical Data: With funding from several sources, including Bridge To A Cure, CBTN processed hundreds of new patient samples in 2024, significantly enriching the Pediatric Brain Tumor Atlas. This invaluable resource allows researchers to delve deeper into the unique biology of these tumors, paving the way for targeted and more effective treatments.

Enhancing Global Collaboration: The CAVATICA software platform, a cornerstone of this partnership’s research efforts, supported over 1,500 global research projects in 2024. Upgrades to this essential tool have improved usability and real-time data access, fostering seamless collaboration among researchers worldwide and accelerating the pace of discovery.

Advancing AI-Driven Solutions: With the resources of CBTN and supported by our coalition, we’ve made significant strides in developing AI-ready datasets and predictive models for some of the most challenging types of pediatric brain tumors, including diffuse midline gliomas (DMG) and medulloblastomas. These innovative approaches hold immense promise for deeper insights and the development of new treatment strategies.

Strengthening Pediatric Proteomic Data: CBTN maintains the largest cohort of pediatric proteomic (proteins) data for brain tumors, expanding it by 30% in 2024. This enriched dataset is crucial for advancing personalized medicine and developing treatments tailored to each child’s unique tumor characteristics.

Building A Data-Driven Research Community

In October 2017, Bridge To A Cure Founder Bob Martin met with National Cancer Institute NCI) Director Dr. Ned Sharpless to share his recommendation to build a national childhood cancer database that was easily accessible by researchers. This idea is realized in the Childhood Cancer Data Initiative (CCDI), authorized by Congress just two years later and funded at $50 million annually for 10 years.

This crucial resource was made possible through harmonizing data using a unified lexicon, an endeavor funded by the Bridge To A Cure Foundation through the Pediatric Cancer Data Commons (PCDC). By uniting data from various sources, including the Kids First Data Resource Center, St. Jude Cloud, and the Treehouse Childhood Cancer Data Initiative, CCDI aims to foster a collaborative research environment where scientists can access more data, tools, and resources than ever before.

A RADIANT Approach to Cures

For years, Bridge To A Cure has called to expedite a united approach to scientific discovery through technological advancement. Now, CBTN stands at the forefront of pediatric cancer research, spearheading initiatives like the government-funded RADIANT project. In collaboration with leading technology companies, including Amazon Web Services (AWS), Valsera, and Mulesoft (a SalesForce company), this ambitious initiative aims to integrate AI seamlessly into all aspects of pediatric cancer research. AI and leading-edge technologies are significant aspects of how Bridge To A Cure will achieve our mission of reducing the childhood cancer death rate by 50% by 2030. That’s why we invested $200,000 to seed this vital program. Through this investment, CBTN created patient data pipelines that seamlessly integrate from local hospital record platforms to the PBTA.

Through RADIANT, CBTN transforms how we approach diagnosis and treatment planning, providing researchers with unprecedented access to AI-ready datasets, advanced tools, and leading-edge resources. With continued support from Bridge To A Cure and others, RADIANT will set a new standard for personalized care, bringing us closer to a future where every child diagnosed with a brain tumor doesn’t just survive but thrives.

What Lies Ahead

Over the past seven years, Bridge To A Cure Foundation has transformed and united the childhood cancer community. We’ve built a powerful network of researchers, doctors, families, and advocates, all working to end childhood brain cancer.

During this time, we’ve seen significant strides in advancing scientific research and fostering an environment where innovation and hope flourish. The accomplishments of 2024 are the culmination of many years of hard work, determination, and a shared belief that we can overcome the challenges that have long hindered the progress that keeps us from ending this brutal disease. Bridge To A Cure has created real, lasting change, from breakthroughs in data-driven research and patient care to new partnerships with leading research institutions, tech companies, and the federal government. We are poised to make even more incredible strides with these achievements as the framework for new advancement.

For 2025 and beyond, Bridge To A Cure is urging foundations and philanthropists that share our vision to invest $100 million in dedicated brain tumor research funds. By mirroring the collaborative success of our research institution partners, these funds will empower scientists to bypass traditional barriers and accelerate groundbreaking therapies and prevention strategies. And, by supporting this groundbreaking research, we’re investing in a brighter future for families. Every contribution, no matter the size, translates into tangible progress. Your generosity fuels real hope that we will conquer childhood brain tumors and protect the health and happiness of our children.

A May Brain Tumor Awareness Month Progress Report

In the battle against childhood brain tumors, Bridge To A Cure Foundation has been relentless in its pursuit of progress and hope. May is Brain Tumor Awareness Month, which gives us pause to consider the remarkable milestones met. Read on to explore the Bridge To A Cure journey as we mark our foundation’s sixth May Brain Tumor Awareness Month. It is our steadfast belief that by working together we will experience the first May that brain tumor cures will be celebrated.

UNLEASHING THE POWER OF DATA

In collaboration with the Children’s Brain Tumor Network (CBTN), Bridge To A Cure has spearheaded an extraordinary data-building effort. The scale of childhood brain tumor data has grown exponentially, opening doors to research that was once deemed unattainable. In a single year, CBTN added data from over 8,000 specimen samples and added hundreds of new patient participants. This wealth of information is a beacon of hope for researchers worldwide, propelling their quest to discover effective treatments and cures.

A NEW ERA OF FUNDING SUPPORT

After years of stagnation, pediatric cancer research has received a much-needed boost in government investment. This encouraging shift is a testament to the tireless advocacy of childhood cancer foundations, including Bridge To A Cure and St. Baldrick’s Foundation. While progress has been made, increased funding still falls short of what is necessary to combat childhood brain tumors effectively. Bridge To A Cure Foundation remains resolute in its commitment to lobby for increased investment, as the number one killer of children by disease in the U.S. demands urgent attention.

ACCELERATING RESEARCH: COLLABORATION AND DISCOVERY

The power of collaboration and data sharing has revolutionized the pace of research in critical areas of rare childhood brain tumors. Thanks to the pioneering efforts of CBTN and Bridge To A Cure, breakthroughs have been accelerated by as much as 20 years. This remarkable acceleration underscores the promise of open science and collaboration, not only in the realm of childhood cancer but also in the pursuit of solutions for other devastating diseases. Through their unwavering dedication, researchers are reshaping the landscape of pediatric oncology, offering hope to countless families.

FORGING A GLOBAL NETWORK

The journey towards a cure requires a united front, transcending borders and institutions. Bridge To A Cure Foundation has played a pivotal role in fostering a global collaboration among leading childhood brain tumor research and treatment centers. In the past year alone, six new institutions have joined the network, amplifying the impact of their collective expertise and resources. With 34 member institutions working hand-in-hand, researchers gain a comprehensive understanding of childhood brain tumors, paving the way for discovering more effective therapies. Together, they form an unstoppable force for progress.

POWERING UP THE PROMISE OF AI

Artificial intelligence (AI) is grabbing headlines these days. Essentially, AI enables the completion of complicated tasks using powerful, advanced computing technology. In the children’s cancer research space, it’s arguably become the biggest buzzword of the year for good reason. Because of the tremendous efforts of many in the Bridge To A Cure Foundation network, progress is picking up pace in terms of:

• Near-automatic detection of brain tumors on MRI scans
• Providing subtype predictions that can lead to faster, more accurate diagnoses
• Helping doctors identify the best treatment options for each child
• Assisting in detecting disease progression

Our collaborators at CBTN, powered by the Center for Data-Driven Discovery in Biomedicine (D3b) at the Children’s Hospital of Philadelphia, have partnered with Amazon Webs Services (AWS) to find new ways to use the power of high-powered, cloud-based computing and CBTN data and resources to impact the way healthcare is delivered, managed, and accessed for children with brain tumors.

To dive deeper into how AI promises to bring more effective treatments and cures to childhood cancer patients, read our February article, “Artificial Intelligence Offers Massive Potential for Progress.”

SHINING A SPOTLIGHT ON SCIENTIFIC BREAKTHROUGHS

While strides have been made in treating certain childhood cancers, many deadly forms, including the 30 known types of pediatric brain and central nervous system cancers, still lack effective treatments. Bridge To A Cure Foundation shines a spotlight on these scientific challenges, highlighting the urgent need for innovative solutions. By addressing the long-term side effects of current treatments and driving research into novel therapies, Bridge To A Cure remains unyielding in their commitment to saving young lives.

This May Brain Tumor Awareness Month is marked by significant progress, hope, and unwavering commitment at Bridge To A Cure Foundation. Through a data-driven approach, increased funding support, collaborative research acceleration, global network adoption, and a focus on scientific breakthroughs, we are closer to a future where childhood brain tumors are no longer a devastating reality. However, the journey is far from over.

This month, you are called upon to join Bridge To A Cure Foundation in our mission. Together, we can make a life-altering impact in the lives of thousands of children and families affected by brain tumors. Let us stand united in support; providing hope, healing, and a brighter future for generations to come. Together, we can accelerate breakthroughs and build bridges to cures for childhood brain tumors.

Congress is slated to renew a bipartisan bill that extends vital funding for America’s only pediatric cancer research database of its kind — but it’s been needlessly put at risk.

Last year, Bridge To A Cure Foundation announced our emphatic support of renewal of the Gabriella Miller Kids First Research Act. The original bill, which passed in 2014, revolutionized pediatric research and cleared the way for unprecedented progress in childhood cancer and structural birth defect disease spaces by:

• Generating one of the largest inventories of molecular and clinical datasets for childhood cancer and structural birth defects research. To date, more than 93,000 samples have been used to generate DNA and RNA data that is paired with imaging and clinical data, all made available to freely empower collaborative research through a centralized, cloud-based data portal.
• Receiving wide support from patients and families. The National Institutes of Health (NIH) Kids First Data Resource Center, administered by the Children’s Brain Tumor Network (CBTN), created the infrastructure to house data sets from more than 30,000 participants across the pediatric cancer and structural birth defects landscape.
  
• Accelerating breakthroughs. Kids First Data Resource Center has advanced more than 500 pediatric cancer and structural birth defect research projects and freely provided access to more than 3,200 users.

Bridge To A Cure Foundation understands from direct experience the barriers scientists and researchers face in accessing enough data to make meaningful progress. After all, Bridge To A Cure has helped the Kids First Data Resource Center build-out through roles on both the CBTN Executive Council and its Executive Board, as well as through grant funding. Kids First does nothing less than ensure that children are no longer left behind when it comes to medical advancement. And now, assets are fully in place and being accessed by thousands of users — an explosion of scientific discovery is at the ready! However, the decade-long commitment to put kids first ends soon. 

Funding for the NIH Kids First Data Resource Center will expire if Congress does not act by end of year to pass The Gabriella Miller Kids First Pediatric Research Act renewal. 

The Kids First Data Resource Center has been a key component in the creation of a national childhood cancer database—which is at the top of the list in the Bridge To A Cure Foundation Action Plan to reduce deaths due to childhood cancer 50% by 2030. Long-time ally CBTN has been identified by the National Institute of Health (NIH) as the model for open science in data-driven discovery. The impacts of Congressional failure to this bill will be devastating for researchers and scientists, kids and families around the world:

• $126 million already invested in this successful, one-of-a-kind resource will be wasted. 
• The Kids First Data Resource Center will be discarded. 
• Critical funding needed to fulfill Kids First’s life-saving mission will be cut off. 
• The opportunity to again create an initiative like this one is unlikely. 
• While much of the data can be transferred to a different data platform, access and types of use of the data become much more limited. 
• Researchers would once again be forced to analyze adult data to study and treat kids’ diseases blindly. 
• Potential for effective personalized medicine that can save lives becomes greatly diminished. 

Earlier this year, Bridge To A Cure Foundation celebrated when the House of Representatives passed the Gabriella Miller Kids First Pediatric Research Act renewal, designating $125 million to creating a truly comprehensive, open-access, shared-data resource that will benefit the entire childhood cancer and rare diseases research community.

But, the Senate must approve the bill by the end of this year or all of the progress toward better treatments and cures will be lost.

Since this open-access, data-driven model is what the NIH calls the standard, it’s hard to fathom that inaction by just one Senator can dismantle ten years of work by an entire research community focused on cures for America’s #1 killer of kids by disease. But that is what’s happening now. Bridge To A Cure and its coalition met with dozens of Congressional members, and more than 50 have recognized the importance of funding this bipartisan bill through co-sponsorship.

But that isn’t enough to save Kids First. If Senate leaders choose to not include this bill in its end-of-year appropriation package, Congress won’t get the chance to renew the Gabriella Miller Kids First Pediatric Research Act. These few key Senators — Schumer (D-NY), McConnell (R-KY), Murray (D-WA), and Burr (R-SC) — have the power to move the bill forward. They must ensure it does. After all, the lives of hundreds of thousands of sick kids and their families hang in the balance. 

Implications of Failure

The significance of the need for reauthorization of the Gabriella Miller Kids First Pediatric Research Act cannot be overstated. Since the pace of data generation and analysis moves so quickly today, a lapse in even just a couple years means the biggest halt to progress ever experienced in the childhood cancer research community. In addition, data left unused becomes increasingly less usable. Millions of dollars will have been invested for nothing. Leaders in collaborative research models like the CBTN will lose the capacity for collaborative research and precision medicine. Most tragic of all, children will suffer and die needlessly.

Join the Twitter Campaign to Put Kids First

Bridge To A Cure Foundation, along with dozens of other childhood cancer advocate groups, are pushing for the Gabriella Miller Kids First Pediatric Research Act to be included in the Senate’s end-of-year appropriations package. We ask for your help and support, now more than ever, to join our efforts in getting through to key Senators. 

It’s time to put Kids First—again.

Below you’ll find a 5-day posting plan to insist that our elected officials include the S. 1521 Kids First Pediatric Research Act in their end-of-year funding package. We’ve outlined two different ways you can use Twitter to reach Senate leadership:

 OPTION 1: VIDEO MESSAGE
Record a video message to share along with your tweets.

• Record your video message using a smart phone or computer camera. Call on Congress to include the Gabriella Miller Kids First Pediatric Research Act act in their end-of-year package. 
• Place the video link as noted in each tweet below as indicated.
• Copy the entire tweet into your Twitter account. 
• Send.one tweet each day for 5 days.

DAY 1

.@SenSchumer @LeaderMcConnell @PattyMurray @SenatorBurr Please put S.1521 Kids First Research Act 2.0 in your end of year package & put #KidsFirst in #ChildhoodCancer & #RareDisease Research and Data Collection. @smashingwalnuts

DELETE THIS AND PLACE YOUR VIDEO LINK HERE.

DAY 2

Please @SenSchumer @LeaderMcConnell @PattyMurray @SenatorBurr put S.1521 Kids First Research Act 2.0 in your end of year package & put #KidsFirst in #ChildhoodCancer & #RareDisease research and Data Collection. @smashingwalnuts

DELETE THIS AND PLACE YOUR VIDEO LINK HERE

DAY 3

Please put S. 1521 The KIDS FIRST RESEARCH ACT 2.0 in your end of year package @SenSchumer @LeaderMcConnell @PattyMurray @SenatorBurr Lets place #KidsFirst in #ChildhoodCancer & #RareDisease research and Data Collection. @smashingwalnuts

DELETE THIS AND PLACE YOUR VIDEO LINK HERE

DAY 4

Let’s place #KidsFirst in #ChildhoodCancer & #RareDisease research and Data Collection. Please put S. 1521 The KIDS FIRST RESEARCH ACT 2.0 in your end of year package @SenSchumer @PattyMurray @LeaderMcConnell @SenatorBurr @smashingwalnuts

DELETE THIS AND PLACE YOUR VIDEO LINK HERE

DAY 5

#ChildhoodCancer kids are always last in line for new drugs and therapies. Make #KidsFirst by passing S. 1521 Kids First Research Act 2.0. Put it in your end of year package. @SenSchumer @PattyMurray @LeaderMcConnell @SenatorBurr @smashingwalnuts

DELETE THIS AND PLACE YOUR VIDEO LINK HERE

OPTION 2: CHILD’S IMAGE
Share an image of a beloved child that has experienced childhood cancer or structural birth defect.

1. Upload a child’s image as noted in the tweet below
2. Select the version of copy appropriate for your situation. Copy the entire tweet into your twitter program and send.
3. Send one each day for 5 days.

DAY 1

My child (or use first name) didn’t (get sick or die) in vain. @SenSchumer @LeaderMcConnell @PattyMurray @SenatorBurr Please put S.1521 Kids First Research Act 2.0 in your end of year package & put #KidsFirst in #ChildhoodCancer & #RareDisease Research and Data Collection. @smashingwalnuts

DELETE THIS AND PLACE CHILD’S IMAGE LINK HERE

DAY 2

Please @SenSchumer @LeaderMcConnell @PattyMurray @SenatorBurr put S.1521 Kids First Research Act 2.0 in your end of year package & put #KidsFirst in #ChildhoodCancer & #RareDisease research and Data Collection. My child (or use first name) didn’t (get sick or die) in vain. @smashingwalnuts

DELETE THIS AND PLACE CHILD’S IMAGE LINK HERE

DAY 3

Please put S. 1521 The KIDS FIRST RESEARCH ACT 2.0 in your end of year package @SenSchumer @LeaderMcConnell @PattyMurray @SenatorBurr Place #KidsFirst in #ChildhoodCancer & #RareDisease research and Data Collection in (honor or memory) of my child (or use first name). @smashingwalnuts

DELETE THIS AND PLACE CHILD’S IMAGE LINK HERE

DAY 4

Let’s place #KidsFirst in #ChildhoodCancer & #RareDisease research and Data Collection. Please put S. 1521 The KIDS FIRST RESEARCH ACT 2.0 in your end of year package @SenSchumer @PattyMurray @LeaderMcConnell @SenatorBurr @smashingwalnuts

DELETE THIS AND PLACE CHILD’S IMAGE LINK HERE

DAY 5

#ChildhoodCancer kids are always last in line for new drugs and therapies. Put #KidsFirst by passing S. 1521 Kids First Research Act 2.0 in your end of year package. Remember (or honor) my child (or use first name). @SenSchumer @PattyMurray @LeaderMcConnell @SenatorBurr @smashingwalnuts 

DELETE THIS AND PLACE CHILD’S IMAGE LINK HERE

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.

Congress is considering a bipartisan bill to extend and increase funding for childhood cancer research – without the need for taxpayer investment.

It’s not every day we get the chance to support bipartisan legislation to speed up research for cures to childhood cancer. So we at Bridge To A Cure are excited to support the Gabriella Miller Kids First Research Act 2.0.

In 2014, Congress passed the original Gabriella Miller Kids First Pediatric Research Act to fund a 10-year program to advance research into rare childhood diseases. The legislation has provided $88 million of non-taxpayer funding to the National Institutes of Health over seven years.

With this funding, NIH established the very successful Gabriella Miller Kids First Pediatric Research Program (Kids First), which has funded many important studies into the genetic basis of childhood cancers and structural birth defects. The legislation is expected to provide another $38 million before it expires.

With the current law set to sunset before long, we need to extend and expand it to keep dollars, data and momentum flowing in the fight against childhood cancer. The Gabriella Miller Kids First 2.0 legislation will do that (learn more and sign a petition to Congress in support of it here.)

The bill would greatly increase funding for childhood cancer research through the NIH without using any taxpayer dollars. It would do this by channeling the funds paid by companies that break the law directly into critical medical research.

As Congress continues partisan battles on many fronts, the Gabriella Miller Kids First 2.0 enjoys wide bipartisan support with more than 40 representatives and seven senators cosponsoring it. More than 250 foundations and organizations —including Bridge To A Cure Foundation — plus more than 1,200 advocates in all 50 states have signed a letter of support for the new legislation.

The bill’s chief advocate is Ellyn Miller, Gabriella’s mom. At age 9, Gabriella was diagnosed with Diffuse Intrinsic Pontine Glioma (DIPG), an inoperable brain tumor deemed terminal upon diagnosis. Gabriella quickly became a global advocate for children with cancer.

Less than a year after her diagnosis, Gabriella passed away. An interview filmed two weeks before her death led to her name being added to the first act allocating $126 million for childhood disease research.

Now the Gabriella Miller Kids First Act 2.0 legislation is working its way through Congress, and Bridge To A Cure is joining other partners in urging the public to support it. The new legislation strongly supports the third pillar of our action plan, to implement a new approach to childhood cancer research funding and increase funding allocated for research.

This is critical when we consider that cancer remains the single leading cause of death by disease among American children, yet currently only 4% of the National Cancer Institute’s $4.9 billion budget goes towards development of cures and treatments for childhood cancer. Because of limited research in the past 30 years, only four drugs have been developed exclusively to treat childhood cancer.

The new legislation is a also an important key to creating a national childhood cancer database, number one on Bridge To A Cure’s action plan. Funding from the legislation will help to develop a truly comprehensive shared-data resource for scientists researching the majority of pediatric cancers and structural birth defects; it will also support development of computational tools to analyze these very large, complex genomic and clinical data sets.

The Gabriella Miller Kids First Research Act 2.0 (HR 623) was introduced on Jan. 28 of this year by representatives Jennifer Wexton (D-VA10) and Tom Cole (R-OK4). In April, Senators Tim Kaine (D-VA) and Jerry Moran (R-KS) introduced a bipartisan Senate companion bill (S 1521). Both would take penalties that the U.S. Securities and Exchange Commission collects from pharmaceutical, cosmetic, food supplement, and medical device companies that violate the Foreign Corrupt Practices Act, and redirect them to critical research into rare pediatric diseases. Under the Gabriella Miller Kids First Research Act 2.0, these penalties would be perpetually designated for childhood cancer and disease research, instead of going into general treasury as they do now.

Join us in calling on Congress to pass the Gabriella Kids First Research Act 2.0. Learn more and sign the childhood cancer community letter of support at the Bridge To A Cure Foundation Kids First 2.0 landing page.

We need a national healthcare strategy to cut waste and redirect billions, especially toward the fight against childhood cancer.

The United States’ bloated healthcare spending is projected to surpass $4 trillion this year — up from $3.81 trillion last year, according to a report from the Centers for Medicare & Medicaid Services. Most startling of all, those projections “do not account for spending related to the coronavirus epidemic.”

Even before the pandemic, our nation was spending 18% of GDP on healthcare — almost twice as much per capita as other high-income countries. At least a quarter of all that spending is waste, according to a study published in the Journal of the American Medical Association (JAMA).

If we were to redirect even 1% of that wasted money  — $1 billion — to fighting childhood cancer, that would be double the $500 million the government plans to spend over the next 10 years to build a National Childhood Cancer Database.

Building that database is our top priority at the Bridge To A Cure Foundation in our fight against the most common cause of death by disease for children in America. Yet the $50 million the government plans to spend annually is peanuts compared to what we need to truly accelerate the search for cures and save children’s lives.

We need to boost that funding, and reining in waste is a good place to start.

The JAMA study found that approximately 25% of healthcare spending — between $760 billion and $935 billion annually — “can be characterized as waste.” Some key areas where money is being wasted, and estimated ranges of the amount wasted, include:

• Failure of care delivery, $102.4 billion to $165.7 billion
• Failure of care coordination, $27.2 billion to $78.2 billion
• Overtreatment or low-value care, $75.7 billion to $101.2 billion
• Pricing failure, $230.7 billion to $240.5 billion
• Fraud and abuse, $58.5 billion to $83.9 billion

Despite potential waste close to $1 trillion, the report identified just $191 billion to $286 billion in savings from interventions to address the problem.

Another report found that 10% of healthcare spending in the U.S. goes toward end-of-life care, typically defined as care in the last six months of life. Of the $3.65 trillion Americans spent on health care in 2018, $365 billion of it went to end-of-life care.

There is also a well-documented disparity in money raised to fight different forms of cancer. For instance, breast cancer gets the lion’s share of funding at $460 million, accounting for a third of all cancer-specific nonprofit revenue. Childhood cancer accounts for $177 million, about 13%. The large amount of money focused on breast cancer has resulted in many advances in treatments.

The average age at diagnosis of breast cancer is 61. In contrast, the average age that a child is diagnosed with cancer is 10. This calculates to 67 years of life lost when a child dies from cancer, compared to an average of 16 years lost on average to breast cancer.

We are not accustomed to thinking of mortality in such terms. Our nation has not faced the hard conversations to decide where our healthcare priorities should be. We spend 10% of healthcare dollars to keep people alive for the last six months of their lives and consider it an essential part of healthcare.

We can and must do much more for sick children who deserve an opportunity to live their lives and chase their dreams.

Much of the waste in our healthcare system is due to our lack of a national healthcare strategy. We have no unified policy that governs how we spend healthcare dollars. Developing such a strategy would help us to define our priorities and provide a blueprint for how we should be spending (and not wasting) our money.

A national healthcare strategy is a document that outlines a country’s priorities, budgetary decisions, and course of action to improve and maintain the health of its people. Rather than having such a comprehensive policy, the US relies on national health initiatives, strategies, and action plans, a series of plans to address specific diseases, conditions, and issues. But this siloed approach does not help us answer the big questions about how to prioritize healthcare spending, which is often driven by a “squeaky wheel gets the grease” mentality rather than overall vision.

We need to stop doing what we’ve done, and move toward finding lasting solutions for childhood cancer and many other diseases. We won’t do that by looking at each disease in isolation, but by taking a holistic look at what our nation needs to help people live healthier lives.

When we get serious about weeding out waste in our healthcare system, and making tough choices about how much we should spend on end-of-life care, we will free up billions or even trillions of dollars. With a national strategy, we could target those savings to initiatives that make the most difference — such as building a National Childhood Cancer Database, streamlining the clinical trials process, and unleashing the power of artificial intelligence to fight disease.

Children battling cancer and their families have been forced to abandon the idea of a “comfort zone.” We need to get out of ours, break out of the status quo of waste and inefficiency, and give researchers the tools they need to finally conquer the diseases that rob kids of their childhoods and their lives. We can do this through Bridge To A Cure Foundations’ strategic imperative to implement a new approach to funding childhood cancer research.

Where there is a will, there is a way. The question is, do we have the will?

Five-year survival rates don’t begin to tell the story of the cruel toll cancer inflicts on innocent kids — and all who love them.

“No. No, this isn’t possible. This is wrong. All wrong. Someone run another test. Get me another doctor. For Christ’s sake, someone punch me and wake me up from this nightmare.”

This is how one mom described her feelings upon getting the cancer diagnosis that would lead eight days later to the death of her baby girl.

Losing a child has been called the ultimate loss — the deepest, darkest hole of grief into which a human can plunge.

Yet childhood cancer is so inexplicably painful, so agonizing, so brutal, that cancer charities, researchers, and oncologists take heart where they can. For many, it’s in statistics such as an increased five-year survival rate for kids with cancer.

In recognizing June, Cancer Survivors Month, The American Association for Cancer Research touts “spectacular advances in cancer research” that have lengthened lives. The American Cancer Society says 84% of children with cancer now survive five years or more, a huge increase since the mid-1970s, when the five-year survival rate was about 58%.

They are measuring childhood survival rates like a batting average in baseball. But a child’s struggle against cancer is a journey, not a single event. Both the distance traveled and the quality of the journey is how we should measure success. And right now, that journey remains fraught with massive physical, mental, and emotional suffering for kids and their families.

The statistics overlook the trauma routinely inflicted in the standard of care treatments — the trembling fear of the child anticipating her next visit, the family’s economic devastation, the stress and emotional anguish of watching your child suffer through painful and debilitating treatments that can last for years. All these can leave lifelong scars for those fortunate enough to survive to adulthood.

 Cancer Traumatizes Everyone in the Family

The horrors that kids and families experience in battling cancer are cruel and agonizing, as anyone who has lived through this ordeal can attest. The pain and suffering children endure as they undergo standard cancer treatments spread devastation like tornadoes as they tear through parents, siblings, grandparents and other relatives, friends, and caregivers.

It starts with having to try to explain to a child that he or she has a potentially deadly disease — that the headaches, nausea, tiredness, swelling, lump, or aches in their bodies can’t be kissed or wished away. How can you as a parent answer when your child asks you: “Why me, Mom? Did I do something wrong? Is God mad at me?”

 Kids’ Suffering Brings Tears to Their Eyes and Their Parents’

Kids undergoing chemotherapy are constantly poked with needles and injected with powerful chemicals that destroy their immunity, making them more susceptible to other potentially fatal diseases, including COVID-19. The drugs are so toxic that we would probably not use them if there were any other choice. Cytostatic antibiotics, for instance, are among many chemotherapy drugs that are “cardio toxic” and can damage the heart if used too long.

Watching a child suffer has been called a living nightmare for parents. There’s nothing more agonizing than listening to your child cry “Stop, please, stop!” when the pain gets too much for them to bear. You feel so helpless as you watch them lose their hair, their appetite, and their energy and struggle through pain, nausea, rashes, sores, kidney and bladder problems, anemia, and bleeding. These are all among the long list of side effects kids endure through our current inadequate arsenal of chemo, radiation, and other standard treatments.

Then there is the sadness you feel in trying to comfort a child who feels isolated from their friends and sad as they miss out on doing things other kids can do. Cancers upset family dynamics, as siblings have to step up and help out parents facing exhaustion, and give up part of their childhoods as they wrestle with their own fears about the illness.

Financial Stress and Your Worst Fears

With all that you do to try to comfort your child, you face the added stress of wondering how you’ll be able to pay off massive medical bills that pile up as you are forced to take time off from work to drive, often long distances, for treatments. At the very time you need to be focused on caring for you child, you must wrestle with thoughts like: “I can’t keep missing work….What’s going to happen to my job? How am I going to pay off these thousands of dollars on my credit card?” All the while you struggle to keep up a hopeful and cheerful outlook for your child, while pushing down fears of losing your baby.

For too many families, there is no happy ending — just a never-ending string of days filled with loss, of dreams destroyed and milestones that will never be measured.

“The death of a child is considered the single worst stressor a person can go through,” Deborah Carr, chair of the sociology department at Boston University, says in Fatherly. “Parents and fathers specifically feel responsible for the child’s well-being. And they’re not just losing a person they loved. They’re also losing the years of promise they had looked forward to.”

Dread, Uncertainty and Lifetime Scars

As we applaud the fact that more children are living beyond five years, we can’t overlook the struggles they endure to reach that milestone, or the continued pain and suffering that may await them the rest of their lives.

Long-term side effects (called late effects) include damage to the heart, lungs, brain, nerves, kidneys, thyroid gland, or reproductive organs that can impact growth and development, emotions, thinking, learning, and memory. Adults who experienced childhood cancer have twice the suicide risk as the general population. The impact to mental as well as physical health is substantial.

Kids and teens that have undergone some types of chemotherapy may face higher risks of developing other cancers as they grow up. They often live with anxiety, depression, and fear that the cancer will come back. As survivors of childhood trauma, they may carry hidden scars that never go away.

“Because childhood cancer survivors are living longer, they are having more late effects after cancer treatment,” the National Cancer Institute says. “Survivors may not live as long as people who did not have cancer.”

The most common causes of death in childhood cancer survivors are:

1. The primary cancer comes back.
2. A second (different) primary cancer forms.
3. Heart and lung

When you consider the pain, suffering and agony cancer inflicts on families kids — even those fortunate to survive five or more years — it’s clear that how far we have to go before we can claim victory over this enemy.

We must clear a path to finding cures and treatments that don’t subject survivors to lifelong suffering, and give every child a chance to chase their dreams. To do that, the Bridge To A Cure Foundation proposes a radical change in the way we conduct clinical trials.

The clinical trial process was created for adults — not children. As a result, 50% of medicines used to treat children have been extrapolated from adult data without data to support safe and effective dosing for kids. The performance criteria for clinical trials is also based on adults, and fails to consider the child’s horrific suffering, the emotional strain on parents and siblings, the potential for deep psychological scars, or the debilitating impact of the financial burden.

Children are not small adults, and their bodies’ physiology and chemistry react differently to cancer treatments. We need a new clinical trial protocol tailored to children, and performance standards that protect their quality of life as they and their families go through this journey. Next month, we’ll look at this in more depth.

The mission of the Bridge to a Cure Foundation is to increase the pace and success of childhood cancer research. In order to do that, we need to remove barriers to finding cures and effective treatments. The clinical trial process is one such barrier. Without clinical trials, there are no new drugs. In this blog we examine:

• Why we need childhood cancer clinical trials.
• Proof that today’s clinical trials are a debacle.
• The reasons why the current process is failing.

Why we need childhood cancer clinical trials

• To ensure that the drug(s) are effective. “Children and adolescents are not small adults, and their bodies’ physiology and chemistry react very differently to illness and disease. Thus, pediatric clinical trials are critical to developing treatments and cures for childhood disease and illnesses.”
• To identify harmful side effects and “provide reliable evidence of treatment effects by rigorous controlled testing of interventions on human subjects.“

Proof that today’s clinical trials are a debacle

• In the last 25 years, the FDA has received only 30 applications for new drugs for use in pediatric-specific oncology.
• Few drugs have been developed — Since 1980, fewer than 10 drugs have been developed for use in children with cancer.
• The few drugs that have been developed have not delivered a cure and have been appallingly harmful and ineffective. As a result:
  • Cancer remains the number one cause of death by disease among children.
  • There has been no change to the survival rate for children with brain and other nervous system cancers for the past 20 years.
  • Even with survival defined as a child living for a mere five years, only 75% of childhood cancer patients meet that yardstick.
  • More than 95% of childhood cancer survivors will have a significant health-related issue by the time they are 45 years of age.
• Many clinical trials for children are abandoned and their results never published.
• Few clinical trials are conducted in childhood patients compared to the number of industry-supported clinical drug trials conducted in adults, a 2012 study found.

Why the current childhood clinical trial process is failing

• The criteria for approval is insufficiently vigorous — Children are considered survivors if they live a meager five years. If a child surviving five years is a goal researchers can celebrate and be rewarded for, then we should not be surprised that that’s what our solutions deliver. The five-year measurement fails to acknowledge the child’s horrific suffering, the emotional strain on parents and siblings, or the financial impact on the family.
• Lack of participation — One study identified 30 potential barriers to participation in childhood clinical trials including a relatively small population of available participants; high cost and lack of incentives for pharmaceutical companies to perform drug trials; potential legal risk to the pharmaceutical sponsor; ethical concerns regarding participation of children in trials; and a lack of adequately trained pediatric investigators.
• Lack of Funding — Pharmaceutical companies won’t invest. “One significant challenge in developing drugs for children is the perspective that pediatric medicines do not provide industry with similar financial rewards as adult medicines,” said Benjamin Ortiz, MD, who formerly served as Medical Officer in the Office of Pediatric Therapeutics at the FDA.
• The pharmaceutical industry “may have been reluctant to study medicines in children” for several reasons, another NCBI study says in an obvious understatement. “The market for the sale of many drugs for children is smaller than that for adults, and therefore investment in childhood drug testing might be less attractive financially. Other reasons cited in this report include ethical difficulties, problems with blood sampling, and difficulties in recruiting sufficient numbers of children.”
• Pharmaceutical companies won’t provide drugs — “Access to drugs and the ability to explore them are the biggest barriers to changing standard of care for pediatric patients,” says childhood cancer expert Patrick Brown, Associate Professor of Oncology and director of the Pediatric Leukemia Program at John’s Hopkins. “We can’t mass-produce drugs, so we usually rely on drug companies to provide drugs for clinical trials,” says Brown. “The much smaller number of patients with childhood cancers compared to adult cancers and the risk of something going wrong with a drug being tested in children can make pharmaceutical companies reluctant to provide drugs.”
• The current clinical trials process favors research into diseases that affect adults over those that afflict children — There are over 300,000 clinical trials available today worldwide, of which there are only 26 childhood clinical trials in the U.S. (https://pediatrictrials.org).
• Ethical concerns — “Pediatric trials are more challenging to conduct than trials in adults because of the paucity of funding, uniqueness of children, and particular ethical concerns,” says a study from the National Center for Biotechnology Information (NCBI), which is under the National Institutes of Health.

We need a new national strategy and commitment to improve the clinical trials process for childhood cancer drugs and therapies. In part 2 of this blog, we will explore what needs to be done.

To read our prior posts providing solutions to improving our approach childhood cancer research, visit Bridge to a Cure.

The Bridge to a Cure Foundation is the lead advocate for modernizing our approach to childhood cancer, with a priority on building a childhood cancer database to speed up the search for cures. We have met and gained the support of over 120 institutions and practitioners, including the former NCI Director Ned Sharpless, who has become a forceful proponent and driver of this initiative in Washington. To learn more about the foundation and add your support, visit Bridge to a Cure.

Government regulations and controls hinder discovery, treatment, and affordability

The mission of the Bridge to a Cure Foundation is to increase the pace and success of pediatric cancer research. In order to do that, we need to remove barriers to finding cures and effective treatments. In this blog we will look at:

• How government red tape hinders cancer research.
• Costs of childhood cancer to families and society.
• Why we need a national strategy to align priorities and streamline the search for cures.

1. Government red tape and cancer research.

The cost of government regulations is well known to the industries that pay the burden. One study put the cost of new regulations implemented in the Obama era at $100 billion. These costs are not just born by manufacturers and utilities, but also in the research arena where agencies such as the Food and Drug Administration have stymied development of drugs to treat cancer. Thousands of families have suffered as a result of government red tape.

Dr. Vincent DeVita, who developed the first curative chemotherapy protocol for adult cancer, argues that the FDA has held up vital cancer treatments through its cumbersome and outdated approval process. In his book The Death of Cancer, he says that bureaucracy is the only thing standing in the way of transforming cancer from a killer to a “chronic but survivable illness.”

DeVita argues that the FDA should not be approving drugs for treating cancer because it treats them like traditional drugs, not taking into account the fact that cancer patients may die without trial medicines. FDA approvals, he argues, are one of the biggest obstacles to patients receiving life-saving treatments.

“The FDA is approving drugs based on outdated methods,” he said in an interview. “It should approve new cancer drugs based on their safety profile and their ability to hit important molecular targets. And I think cancer centers have matured enough to handle all the early trials by themselves.”

The National Cancer Institute (NCI) and FDA should delegate responsibility for all phase I and II trials to NCI-approved cancer centers, he says. This would increase the speed of developing new drugs tenfold, and cost a lot less than the current system.

“Guidelines are backward-looking in a rapidly changing field like cancer,” DeVita says. “But what worries me more are the constraints on innovation. New approaches to treatment in this era of targeted therapy need maximum flexibility to mix and match treatments in order to control the wily cancer cell.” He also calls for greater collaboration between cancer treatment centers.

2. Government and the high costs of childhood cancer.

Since the early 1900s, medical special interests have been lobbying politicians to reduce competition. By the 1980s, the U.S. was restricting the supply of physicians, hospitals, insurance and pharmaceuticals, while subsidizing demand. “In too many areas of the economy — especially health care — free enterprise has given way to government control in ‘partnership’ with a few large or politically well-connected companies.” (Ryan 2012).

Meanwhile, a National Children’s Cancer Society survey of 449 families coping with childhood cancer found that 95 percent reported their child’s cancer caused a financial burden on the family. Among families who were not poor when their child was diagnosed, 10 to 15 percent became impoverished as a result of medical bills. A quarter of families reported losing more than 40 percent of their income to help pay the bills, and this did not even count out-of-pocket expenses like travel to hospitals and childcare.

These findings are not surprising when you consider that the average cost associated with each case of childhood cancer is $833,000. The average cost of hospitalizations for pediatric cancer is $40,000 — nearly five times as much as hospitalizations for other pediatric conditions. And let’s not forget that cancer is just one of the pediatric diseases that rob family finances. An estimated 30 million children in America struggle with a chronic disease or psychological disorder.

The economic cost to families and our nation is staggering. The emotional stress on families is not quantifiable — nor is what our nation forfeits from not having many of these children achieve their potential.

Disturbingly, the culprit contributing to this emotional and economic devastation is the medical industry’s antiquated approach to childhood cancer research and their influence on government.

3. Why we need a national strategy to align priorities and streamline the search for cures.

Government regulations should be linked to an overarching strategy that improves our individual and national health, safety, and economic conditions. Economic return must be part of the strategy.

Regulations on automobile safety and emissions are as example. A study by the Institute of Transportation Studies at the University of California, Davis found that added costs from such regulations are significant, but “represent only a modest part of overall vehicle cost increases.” Regulations “had little discernible effect on industry performance and activities,” with cost increases “largely accommodated within normal business and market planning processes of companies.”

The same cannot be said of government regulations restricting new cancer drugs, since they actually prevent patients who have been diagnosed with terminal diseases from accessing medications that are experimental in nature.

I am not arguing for eliminating all regulations, but rather for eliminating those that create barriers for research into cancer and other pediatric diseases — the kind outlined in a study by the Regulatory Transparency Project:

“Poorly designed regulations may cause more harm than good; stifle innovation, growth, and job creation; waste limited resources; undermine sustainable development; inadvertently harm the people they are supposed to protect; and erode the public’s confidence in our government,” the study found.

We should also work to eliminate regulations that drive up the cost of research and treatment in response to legal risk. On the other hand, we need new regulations to address the imbalance in funding for childhood vs. adult diseases. Here’s the dilemma:

• The 60+ age group is huge and they face many medical issues. It’s an attractive market for the pharmaceutical industry and other research institutions. But our youth are the future of our nation, the “seed corn” that hold the key to productivity and a better life.
• Despite their tremendous potential, children under 18 make up less than a quarter of the total population. Those suffering from serious diseases are an even smaller percentage. About 11,060 children in the United States under the age of 15 will be diagnosed with cancer this year. This accounts for less than 1 percent of all cancers. Yet while their numbers are small, the costs to families and society are enormous.

When it comes to new regulations, we should adopt ones that encourage collaboration, investment and promising new approaches. At the top of the list is building a national pediatric cancer database — one that will allow us to tap into the power of artificial intelligence to end the scourge of childhood cancer.

What regulations can be put in place to encourage government, research institutions, and private industry to do the right thing: collaborate and invest in our future? If the government can demand that carmakers invest in seatbelts, why can it not demand that pharmaceutical companies invest more in pediatric research?

It’s time to review our country’s regulatory strategy to eliminate regulations that are a barrier to research, and to add regulations and incentives to accelerate meaningful breakthroughs.

Redefining the role of government in pediatric cancer research is essential to achieving the breakthroughs so many children are counting on. To read our prior posts providing solutions to improving our approach pediatric cancer research, visit www.bridgetoacure.org.

The Bridge to a Cure Foundation is the lead advocate for modernizing our approach to pediatric cancer, with a priority on building a pediatric cancer database to speed up the search for cures. We have met and gained the support of over 120 institutions and practitioners, including the former NCI Director Ned Sharpless who has become a forceful proponent and driver of this initiative in Washington. To learn more about the foundation and add your support, visit www.bridgetoacure.org.

The resources are available today to cure pediatric cancer. We’ll explore this in the third installment of a five-part series that provides the framework necessary to unlock these resources. The key drivers are:

• Investors (See May blog)
• Donors (See June blog)
• Government
• Practitioners
• Educators

Today’s blog focuses on government, specifically the National Cancer Institute (NCI) – what it needs to do better and what it needs to stop doing. Let’s start by looking at the National Cancer Institute’s FY 2020 Annual Plan and Budget Proposal. It calls for funding in four areas to:

• Develop the workforce of cancer investigators.
• Reaffirm our commitment to basic science to drive novel approaches and technologies.
• Innovate the design, administration, and analyses of clinical trials.
• Increase data aggregation and interpretation to speed work across the cancer enterprise.

The first two areas are what the NCI should stop doing. The latter two are what the NCI needs to do better and soon.

What NCI needs to stop doing

1. NCI, like other government institutions, has grown so large and diverse in its responsibilities that it is inefficient and ineffective. Like the failed model of corporate conglomerates that were once popular, NCI is trying to do too many things. The result is an unfocused approach to fighting cancer with incremental results.
2. Like all large bureaucracies, NCI’s focus is on maintaining and increasing the flow of taxpayer dollars to its programs. The institute’s 2020 budget proposal calls for spending more than $6.5 billion, with $662 million in new spending, including an additional $70 million for “understanding the mechanisms of cancer” and an additional $35 million for “strengthening the cancer research enterprise.”

Why should this be the role of the federal government, when the private sector, universities, and medical institutions can do the job better? It should be up to each individual to decide which institutions they want to donate to or invest in, not the government. Government should not be in the business of human resource development, or investing taxpayer money in the private sector. This is a distraction that helps explain why we still don’t have a robust national pediatric database, nor a clinical trial process designed specifically for pediatrics.

What NCI needs to do better

1. Improve the clinical trial process. This is and should be the responsibility of NCI. It is important that all research follow a standard protocol. However, what we have today is outdated and was never designed for pediatric cancer research. Studies have uncovered flaws in the clinical trials process, including the FDA setting benchmarks that are outdated by the time trials start, and testing inadequacies that lead to experimental drugs failing in the final and most costly phases of trials. We should hold NCI accountable for delivering a faster and more effective clinical trial process and monitoring the results.
2. The fourth area in the latest budget, data aggregation and interpretation, is by far the most important. Translated, this means building a robust national pediatric cancer database – one that includes information from every single institution involved in pediatric cancer, including research, patient data, clinical studies on drugs, and research into promising alternative therapies. This will require addressing many of the barriers that prevent organizations from collaborating at the level required, including addressing privacy laws and technology to convert handwritten patient records into digital formats.

Our government is too large; as a result, it is inefficient, ineffective, and poorly governed. It reminds me of the corporate conglomerates that dominated business in the 1960s and 70s, but disappeared in the 1980s because they were too large and unfocused.

Most private corporations today recognize the value of specializing in doing a few things well. It’s time for government to do the same, introducing the U.S. Millennial Government – where technology, focus, and organizational effectiveness generate results above expectations. In this case, NCI as a part of the U.S. Millennial Government needs to revisit its budget with a focus on building a robust national pediatric database, and modernizing and streamlining the clinical trial process.