How Tissue Donation Saves Lives in Childhood Cancer and Rare Diseases

For families facing the life-altering news that their child is diagnosed with cancer or a rare disease, hope can feel scarce. Traditional treatments often have limitations, and finding effective therapies for these complex conditions can be a long and arduous journey.

This is where tissue donation steps in, offering a beam of light in a seemingly dark tunnel. Among the many ways that Bridge To A Cure works towards accomplishing its mission is by strongly supporting this practice. Donated tissue samples become invaluable tools for researchers, allowing them to unlock the secrets of these illnesses and develop life-saving treatments.

Why Tissue Donation Matters for Childhood Cancers and Rare Diseases

Childhood cancers and rare diseases are different compared to adult cancers. They are often more aggressive, have unique genetic mutations, and respond differently to treatments. Due to their relative rarity, research funding and readily available tissue samples can be scarce, further hindering progress.

Donated tissue plays a critical role in this fight

Understanding the Disease: Tissue samples allow researchers to study the disease at a cellular and molecular level. This helps them identify the genetic mutations causing the illness and understand how it progresses.

Developing New Therapies: With a deeper understanding of the disease, researchers can develop targeted therapies. These therapies can be more effective for specific types of childhood cancers and rare diseases, with fewer side effects for young patients.

Testing Existing Treatments: Tissue samples allow scientists to test the efficacy of existing treatments and identify which ones work best for specific patients. This personalized approach to medicine can significantly improve treatment outcomes.

Developing Diagnostics: Tissue analysis can help develop new diagnostic tools to identify these diseases earlier and more accurately. Early detection is crucial for improving survival rates in children with cancer and rare diseases.

The Gift of a Lifetime

Without the unwavering support from organizations like Bridge To A Cure, the Children’s Brain Tumor Network (CBTN) couldn’t display the transformative power of tissue donation. Established in 2011, CBTN is the world’s largest biobank dedicated to childhood brain tumors, and Bridge To A Cure plays a vital role in its success.

With over 5,500 patients and 2,500 family members enrolled, CBTN provides a rich resource of tissue samples for researchers worldwide. This network, supported by Bridge To A Cure and other powerful organizations, operates through hospitals and research institutions across the globe. When a child undergoes surgery or a biopsy for a brain tumor, families have the option to donate a small portion of the removed tissue to CBTN.

Bridge To A Cure’s contribution to childhood brain tumor research is far-reaching through this collaborative effort. Thanks to CBTN’s vast collection of tissue samples, generously donated by families and facilitated by advocacy, researchers have made significant strides in understanding the biology of these tumors, leading to the development of new and more effective treatment strategies. This collaboration between organizations like Bridge To A Cure and CBTN is among the reasons the goal of reducing the childhood cancer death rate by 50% by 2030 is even remotely possible.

The Future of Tissue Donation

The story of the power of tissue donation is being told daily, as evidenced by the pace of advancing research for childhood cancer and rare diseases. As tissue donation programs grow and expand, we can expect even greater breakthroughs in the fight against these devastating illnesses.

Ways You Can Contribute to the Cause

Spread Awareness: Talk to your family and friends about the importance of tissue donation. You can also share information and resources from organizations like Bridge To A Cure on social media and engage with posts about these important topics.

Consider Tissue Donation: If you or someone you know has a child facing a diagnosis of cancer or a rare disease, inquire about tissue donation options at your hospital.

Give to Support Enrolled Families: Tissue donation requires the cooperation of families, doctors, coordinators, labs, and biobanks. This complex but critical web is needed to successfully make use of this fragile tissue. When you give to Bridge To A Cure, proceeds support this worldwide network and the amazing children who make this research possible.

By working together, we can ensure that tissue donation remains a beacon of hope for children facing cancer and rare diseases, offering them brighter futures and a chance to thrive.

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

Transformative change requires a bold challenge.

“Unite Transform Cure” is the theme of our 2020 annual report (you can read it here). It reflects our commitment to speed up not only the search for effective treatments, but to actually end the scourge of childhood cancer.

Most doctors won’t use the word “cure” when discussing cancer, as there is yet no cure, although some treatments may be able to cure some people of some cancers.

We use the word “cure” as part of our theme because we know that an audacious goal requires an audacious challenge. Think of President John F. Kennedy’s challenge on Sept. 12. 1962: to land a man on the moon and return him safely to Earth by the end of the decade. Would that goal have been achieved without a bold challenge that at the time seemed out of reach? Doubtful.

When it comes to childhood cancer, most medical professionals avoid talking about cures because they don’t want to spark “unrealistic” hopes. The fight against childhood cancer has gone on for so long, many people have given up hope they will live to see a cure.

It’s time to jump-start hope.

JFK spoke of the moon challenge as “one we are unwilling to postpone.” He said we accept such challenges “not because they are easy, but because they are hard,” and because they “serve to organize and measure the best of our energies and skills.”

That’s what we seek to do as part of our mission to unite and transform the childhood cancer community’s approach to research. Our goal: to cut the childhood cancer death rate 50% by 2030.

To do that, we must demand more from the healthcare community, nonprofits, private industry, and our government. A key component of our action plan is to align and unite this diverse set of stakeholders toward this common goal.

We know this won’t be easy. We also know it is absolutely essential. We’ve heard the dreaded diagnosis and experienced the tragic journey of a 6-year-old girl bravely facing brain cancer, inspiring her family with hope to the end. We’ve felt the sense of helpless frustration watching a disease that has thwarted the best medical minds in the world take another young life, stalling hope again and again.

To win this fight will take a new approach. That’s why we’re working to transform a system that is built on competition to reap the benefits of collaboration. We serve as a lead catalyst for modernizing the nation’s approach to childhood cancer research, working with the healthcare community and childhood cancer foundations to fix a broken system.

In our first full year
operating as an independent 501(c)(3) nonprofit charitable organization, we’ve supported the National Cancer Institute Childhood Cancer Database Initiative (CCDI). This year our goal is to be represented on the CCDI development commission to ensure that the database is optimized and that patient families are represented.

Bridge To A Cure Foundation acts as a catalyst to connect the people, information, and assets needed to propel new treatments and cures for childhood cancer.
We are building consensus around our unique, multifaceted approach with some of the country’s most well-respected voices in the childhood cancer community, including nonprofits, patient families, and the public.

From our founding in 2017, we have been focused on tearing down barriers to effective cancer treatments. Applying principles from the most successful private sector companies, we identified the need for the following:

• A robust database containing everything we know about childhood cancer.
• Streamlined clinical trials to speed the development of lifesaving treatments.
• Collaboration between government agencies, private companies, and key research institutions.

As the pandemic disrupted lives and consumed vast energy in our healthcare system, we have relentlessly kept our focus on the changes needed to beat childhood cancer. This includes working with America’s leading institutions to identify and understand the barriers they face.

We’ve collected input from Duke Cancer Institute, Memorial Sloan Kettering Cancer Center, Dana-Farber Cancer Institute, Children’s Hospital of Philadelphia, and other leading cancer centers. Our goal is to ensure that the changes we advocate serve those working to develop the most promising new treatments — those that may lead to cures.

We’ve worked in the past year to identify attributes of the most successful nonprofits. These include targeting a compelling problem; setting an audacious, inspirational and measurable goal and a timeline for accomplishing it; and collaborating with others working toward the same ends.

In the coming year and beyond we will continue forging relationships and partnerships with change-makers to align their resources, talents, initiatives, and expertise toward our unified goal of reducing the death rate of childhood cancer 50% by 2030. With the continued help of our engaged partners, donors, volunteers, and supporters, we will keep our eye on the prize: curing childhood cancer — once and for all.

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.