We have a common enemy: childhood brain tumors. The fact is, there have been many amazing oncology professionals, scientists, researchers, and institutions working tirelessly on cures over the past 50 years – half a century! And yet, not one treatment has been developed for childhood brain tumors. Over this time, roughly 750,000 children have endured the painful, cruel journey of the #1 killer of kids by disease.

So what’s missing in our country’s childhood cancer research model that makes progress much slower than it should be?

Collaboration.

Bridge To A Cure greatly respects the work these childhood cancer heroes are doing for childhood cancer patients. We just want them to work together – and to share their data and discoveries to arrive at breakthroughs to cures sooner. We understand that by working together we can accelerate the pace toward cures. And when young lives are waiting for help, and families are holding on to hope, every day matters — every second matters.

There are three major players seeking new therapies for childhood brain tumors:

• Children’s Brain Tumor Network
• Pacific Neuro-Oncology Consortium
• Pediatric Brain Tumor Consortium

Additionally, these groups each include approximately 15 research organizations working on childhood brain tumors under their umbrellas. In other words, there are a lot of experts with a lot of knowledge involved in this race to cures. And most of them are holding their data within their own institutions and consortia rather than sharing with the entire childhood cancer research community.

Consortia Collaboration Will Save Lives

Sharing data and patient information will cut years from research to discovery, to trials to effective use, and to saving lives. We could accomplish in ten years what now takes up to twenty. Think of the thousands upon thousands of lives this can save.

We want these three consortia to:

• Molecularly characterize and sequence all biospecimens they house.
• Share data they have about the patients in a way that protects patient privacy. This includes age, lifestyle, other health issues, family histories, and circumstances.
• Share all data from childhood tumors.
• Provide access for all researchers and organizations working on childhood brain tumors.

Creating teamwork within the system will absolutely speed up the process leading to breakthrough results.

Collecting and sharing data is the first step. Next, we need machine learning and AI to help decipher and analyze everything – there’s just too much data for one analyst or team to ever digest. AI is so powerful that it can process in one hour what it would take a researcher to process in a year.

Once the network for sharing is set up, analysts can tap into it and add content they’ve collected that matches up with a similar patient or type of brain tumor whose data research is further along. In this way, more brilliant minds have better knowledge from which to develop treatments and break through to cures.

Obstacles to Collaboration

There are several reasons that data sharing happens less frequently than it could. We understand some of them, and they make a degree of sense from a business perspective. It’s just that in this instance, the business end is inhibiting the health of children.

Key reasons organizations don’t share include:

• Seeing their project through. Research institutions take pride in their work and progress. It’s human nature to want to see what you started through to the end. Unfortunately, this mentality adds years to a potential breakthrough.
• These groups receive grants and donations based on their progress toward discoveries. If essential data is shared, the fear is the money they need would be reduced because they alone are no longer making progress, so they choose to “protect” their data.
• The dilemma is that if an institution or consortium shares data, another may make that final connection to a treatment or cure. And which group will get the credit and the accompanying bounty? Probably the one who finished the process, not the one who laid the foundation.

Bridge To A Cure’s Collaborative Approach

In 2018, under the leadership of Dr. Ned Sharpless, the NCI started the Children’s Cancer Data Initiative. This ten-year, $500 million program will provide researchers and oncologists access to all data for all types of pediatric cancer.

This much-needed initiative is progressing, but much too slowly because they are trying to tackle all childhood cancers at once. Bridge To A Cure Foundation’s plan first addresses the most deadly form of childhood cancers: brain tumors. By doing this, we can move faster to save lives, and can then apply this model not only to other childhood cancers, but to all kinds of serious childhood diseases and disorders.

We believe we can speed up and be the catalyst for change. We understand the players, the situation, and we have the framework to accomplish our mission unify and transform the childhood cancer research community to reduce deaths due to childhood cancer 50% by 2030. 

Learn more about our progress to open up real-time, collaborative access to the data that leads to cures here.

What we can do to fight the trauma of childhood cancer + COVID-19

Every time 5-year-old Julia Malicki goes to the doctor for cancer treatments, she has to brave having a cotton swab stuck up her nose to the back of her throat to test for coronavirus. The fear and discomfort she endures seems almost unbearable for a child who has already lost her right eye, and 70% of the vision in her left, to a rare form of cancer called retinoblastoma.

It’s anguishing for her and for her mom, Jessica.

“It’s a lot of emotional turmoil as a child to have to go through that fear,” Jessica told USA Today in an article titled “‘Like a new diagnosis’: Cancer families struggle to continue treatment amid coronavirus pandemic.” “The COVID testing really impacted (her).”

Cancer + COVID-19: Traumatizing Kids & Families

Surgery, radiation, and chemotherapy already traumatize families emotionally, psychologically, and financially. Now they face the added fear and worry of contracting another potentially fatal disease.

Since October 2016, Julia’s family has traveled from Wisconsin to New York City every four weeks for her tests and treatments. After driving 17 hours, the family has arrived to find motels closed due to the pandemic. Due to COVID-19 rules, only her mother can be with her during hospital visits, depriving Jessica of the support of her husband or other family.

Disrupting Caregiving Comfort

Some patients have had their treatments postponed or delayed. Lakelynn Markham, a 6-year-old from Holly Springs, N.C., became a candidate for a clinical trial three years ago after doctors discovered a tumor pressing on critical nerves in her neck, disqualifying her from surgery.

For half of her life, Lakelynn and her family traveled to the Dana-Farber Cancer Institute in Boston for her treatment. The pandemic ended those trips. She now sees doctors at a Duke University Medical Center and receives treatments via overnight courier.

It means that she now has to fight for her life without her “superhero,”  a Boston nurse named Shawn, who used to help her through her treatments.

“He has magic,” Lakelynn told Raleigh, N.C. radio station WRAL, explaining that when Shawn prepared her IV, his magical hands made sure the needle didn’t hurt at all.

Children like Lakelynn get to know and trust their doctors and nurses, and depend on routines to help them through medical trauma. Changing routines can increase a child’s fear and anxiety.

They also face struggles that make the inconveniences healthy people face — like having to wear masks and maintain social distance — seem minor. Lakelynn has been doing these things since long before the pandemic began. And while she understands why she can’t play with her friends, it is still heartbreaking for her mom to watch her staring out the window at other kids having fun.

Raising the Stakes of Pediatric Cancer

For children battling cancer and their families, COVID-19 has disrupted surgeries, radiation and chemotherapy treatments, and added to their emotional, financial, and psychological trauma. The same is true for children and families fighting other major medical issues.

A new study in the journal JAMA Pediatrics looked at 48 children in the United States in intensive care with COVID-19. It found that 40 of them had significant medical issues and half of these were categorized as “medically complex,” meaning they were either dependent on technological support for survival or faced serious medical issues such as immunosuppression, usually connected to cancer treatment, the New York Times reports.

“Children with immunodeficiencies or children who have had chemotherapy are at higher risk for serious illness,” the article notes.

Yet children without previous medical problems have also ended up in intensive care, sometimes suffering from a condition related to COVID-19 known as pediatric multisystem inflammatory syndrome. It develops weeks after the initial coronavirus infection and assaults a child’s circulatory system with inflammation rather than directly attacking the lungs.

Treatment Must Go On

Despite the risks posed by the pandemic, experts warn against discontinuing chemotherapy, surgery, radiation, and other therapies for children battling cancer.

“We have observed in our conversations with colleagues from around the world that it is giving us major disruptions, and that children will die or have died, not because of the infection but because of how COVID-19 has affected their (health care) services,” Dr. Carlos Rodriguez-Galindo, told U.S. News.

Rodriguez-Galindo is director of St. Jude’s Global COVID-19 Observatory and Resource Center for Childhood Cancer, a new database of resources for medical professionals specializing in treating pediatric cancer. Similar to the national pediatric cancer database advocated by Bridge to a Cure Foundation, it includes a resource library, a global registry of pediatric cancer patients infected with COVID-19, and a collaboration space for healthcare professionals.

“We have learned from them that even the children that have active therapy for cancer with COVID-19 don’t seem to do that poorly, and that is reassuring.”

As a result of the pandemic, children and families battling cancer and other chronic and terminal diseases and conditions face longer odds and more harrowing battles than they did before. They need our help.

How You Can Help

• Follow COVID-19 guidelines including wearing a mask and staying at least six feet away from people when in public and washing your hands frequently. Parents of kids battling cancer appreciate when we do our part to stop the spread and help keep their kids safe.
• Support families who have lost children to cancer and those still in the fight. Some families who have lost children are experiencing PTSD and anguish over what other childhood cancer families and patients now face in the pandemic. Some practical ways mentioned in this article include contacting families via social media and asking how you can help — financially, through donations of supplies, or by offering to help with shopping or other errands. Visit via phone and video and be there to listen and provide emotional support.
• Donate blood and blood products such as platelets to help combat shortages due to blood drives that have been canceled due to the pandemic. Cancer patients desperately need these, so contact your local blood bank to schedule an appointment.
• Join the National Bone Marrow Donor Registry. Donated bone marrow and stem cells are a critical part of cancer treatment that help save lives. Learn more and join the national database/registry at DKMS.org or BeTheMatch.org.
• Make your voice heard through Bridge To A Cure. The Bridge To A Cure Foundation is working to help kids with cancer and their families by advocating for greater efficiency, collaboration and data sharing to find effective treatments and cures. Stay updated on our efforts, comment on the fight, and learn about other ways to help when you visit BridgeToACure.org and sign up to receive updates.

Social media posts:

Children with immunodeficiencies and those who have had chemotherapy are at higher risk for serious illness due to COVID-19.

Families going through surgery, radiation, and chemotherapy now face added trauma of possibly contracting another potentially fatal disease.

Lakelynn has to fight for her life without her “superhero,”  a Boston nurse named Shawn who used to help her through her treatments.

Children fighting cancer get to know and trust their doctors and nurses. Changing caregivers and routines adds to their fear and anxiety.

Despite risks posed by the pandemic, experts warn against discontinuing chemotherapy, surgery, radiation and other therapies.

Support families who have lost children to cancer and those still in the fight. Visit www.BridgeToACure.org.

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.

In our last blog, we explored why the clinical trial process is one such barrier, including the fact that few new drugs have been developed for treating children with cancer, and that the few drugs that have been developed have not resulted in cures, but have been appallingly harmful. In this blog we examine some possible solutions.

Acknowledgement of the problem and its cause

Rectifying persistent unsolved problems begins with the acknowledgement that there is a problem, and agreement as to the root cause of the problem. Our prior blog provided evidence that:

• Our childhood clinical trial approach is a serious problem.
• We know the root causes contributing to the problem.
• Those participating in the clinical trial process are aware of the magnitude of the problem and its causes.

Why it has not been resolved

If the medical community knows this, why hasn’t the problem been resolved? There are three overarching reasons:

1. Methodology – It relies on a problem/solution approach whereas what is needed is a process improvement approach.
2. Money – The population of children with cancer is too small for pharmaceutical companies to invest in. Funding for clinical trials is not typically covered by federal grants, and federal laws meant to encourage drug companies to invest in pediatric clinical trials have not been very effective, according to an article titled “Drug Discovery: A Challenge for Pediatric Cancers,” published by Johns Hopkins Medicine. Bottom line, the money isn’t there. “We are completely at the mercy of the drug company as to whether or not it will provide the drug for this study,” pediatric cancer expert Dr. Patrick Brown says in the article. “If it says no, which is the most likely answer, the study doesn’t happen.” This has hindered the efforts of the Children’s Oncology Group, a collaborative of oncologists from 220 institutes that does most large clinical trials.
3. Matriculation – The investment, government and nonprofit communities are not fully immersed as critical components in an effort to fix our approach to childhood clinical trials.

The solution

The following proposed solutions would hopefully encourage the medical, investment, government and nonprofit communities to make fixing the childhood clinical trial process a shared priority, with a clearly stated mission, strategy, and plan.

Methodology
1. Set vigorous performance standards. There are two problems with the current performance metric of extending a child’s life five years:

• It assumes that extending a child’s life by five years justifies the pain and hardships faced by the child and family. This measurement needs to be revisited. A robust analysis that measures the benefit vs. the risks (pain, financial burden, psychological impact, quality of life) is long overdue.
• It encourages the same past practices that have produced too few breakthrough cures and treatments. We need to set standards that will require greater collaboration and the expanded use of machine technology.

2. Capitalize on the enormous potential of existing and emerging technology:

• Today, laboratory analysis of a child’s cancer is limited to the few samples available from the tumor. Simulating the child’s tumor in the laboratory will provide an unlimited ability to analyze and test treatments. The Broad Institute has had some success and we need to invest more in this.
• The population of children with cancer, as is true for many other childhood diseases and disorders, is too small to benefit from the analytical power of artificial intelligence (AI). Without AI, the treatment and cure possibilities are limited. To address this problem, we need to invest money and minds to establish a dynamic methodology to translate the results from adult clinical trials to inform childhood treatments and cures. This would be a singular funded initiative focused only on this goal.

3. Implement reward and recognition programs that:

• Foster collaboration within and across institutions and industries.
• Include objectives designed specifically to improve the clinical trial process.
• Are linked to a more vigorous performance standard.

Both behavior and performance are most influenced by goal expectations. If we want greater collaboration and more effective/compassionate treatment solutions, the performance goals need to clearly articulate what’s expected.

Money
We will need more than just a fresh approach to generate the funds required to finance the testing of potential cancer cures for our children. In our May 2019 blog we introduced the Millennial Organization to do just that. It revolutionizes the criteria for valuing organizations. The blog provides detailed evidence to support that the time has come for the Millennial Corporation – the organization whose stock value reflects not only sustainable profit growth but also its contributions to improving the human condition. Some reasons we cited to justify this approach include:

• Investors want it: Individuals everywhere are concerned about our country, the world, its people and the environment. The horrors of pediatric cancer are at the top of the list of concerns for many Americans. “For these and other reasons, more people are investing their money to get back more than just a monetary return on their investment. Many are investing to make a positive impact in our country and around the world as well as to feel that societal concerns should be made an important part of their investment focus.” (1)
• Investment companies want it: “Our global goal is to develop a comprehensive, industry-leading platform of research, advice, and products that is dedicated to sustainable investing and philanthropy.” (2)
• It’s already happening: “Over the last two years, SRI (Socially Responsible Investing) has grown by more than 22% to $3.74 trillion in total managed assets, suggesting that investors are investing with their heart, as well as their head. In fact, about $1 of every $9 under professional management in the U.S. can be classified as an SRI investment.” (1) Signatories of the UN-PRI (The United Nations-supported Principles for Responsible Investment) manage $59 trillion.

Children continue to suffer the effects of toxic chemotherapies because drug companies refuse to provide promising immunotherapy drugs. This is an example where investors can make a difference, by demanding that pharmaceutical companies invest in clinical trials as part of a move toward socially responsible investing. Now is the time for the investment community and pharmaceutical industry to redefine the metrics for valuing this industry. Pediatric cancer would be a good place to start.

Matriculation
To reinvent the clinical trial process, and to significantly increase the availability of new drugs and treatments for childhood cancer, will require participation and commitment from all cancer related sectors, including:

1. Pharmaceutical and investment communities: Develop criteria for valuing a pharmaceutical company’s contribution to improving childhood cancer. This will require shared leadership from both sectors such as the Security and Exchange Commission and the Pharmaceutical Research and Manufactures of America Foundation  (www.phrma.org )
2. Childhood Cancer Nonprofits and Donors: This community funds both “care” to children/families fighting cancer and childhood cancer research. The “care” support they provide is important and effective. It should be continued. However, the funding of childhood cancer research is not. Instead they should redirect these funds to efforts that will greatly advance discovery by removing barriers to meaningful breakthrough. Fixing the childhood clinical trial process is a good place for them to start.
3. Government: Government laws and policies are among barriers to an effective and sensitized clinical trial process. Legislators should assess all such rules to assure they are not “suffocating, discouraging or preventing the research they seek to regulate.”
4. Tech companies: As noted above, fixing the clinical trial process will need to include artificial intelligence.

While each sector needs to take responsibility for their contribution to improving the clinical trial process, a holistic approach is still required. Fixing the childhood clinical trial process demands a coordinated effort framed by a shared mission, strategy and plan. We recommend that Dr. Ned Sharpless, NCI Director, lead this initiative.

In conclusion, our clinical trial process is broken. We need a new approach. One that is:

• Sensitized by the horrific journey, modernized by machine technology, and optimized to reward higher performance standards and collaboration.
• Built to reward pharmaceutical companies for investing in childhood cancer research.
• Holistic, with active participation and commitment from each sector.

To read our prior posts providing solutions to improving our approach pediatric cancer research, visit BridgeToACure.org.

The mission of Bridge to a Cure Foundation, a nonprofit fund, is to accelerate the pace and success of childhood cancer research by unlocking the full potential of resources already available today. Our vision is happier, healthier lives for children who face the challenges of this debilitating disease.To learn more about the foundation and add your support, visit BridgeToACure.org.

(1)  Forbes, April 24,2013 – Socially Responsible Investing: What You Need To Know
(2) UBS editorial in their March 2015 newsletter.