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

Apoptosis: Programming Cancer Cell Death To Save A Life

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

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

The Journey is Horrific

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

Those that Survive, Suffer

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

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

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

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

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

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

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

Bridge To A Cure Foundation continues our ambitious mission to unite and transform the childhood cancer research community to reduce childhood cancer deaths 50% by 2030. We have discovered and worked to remove the barriers that, for so long, have held back progress toward cures. We have identified like-minded experts to form partnerships that push the entire research community forward. And now, we have identified three key areas of research that we at Bridge To A Cure believe hold the promise of finding cures. This is the first of three articles introducing groundbreaking research initiatives in our foundation investing: immunotherapy, angiogenesis, and apoptosis.

Immunotherapy: Harnessing the Body’s Ability to Fight Disease

Immunotherapy represents a groundbreaking shift in cancer treatment, offering new hope for curing childhood brain tumors. Unlike traditional treatments that broadly target rapidly dividing cells, immunotherapy works by harnessing the body’s immune system to fight cancer more precisely and effectively.

Patients with a pediatric brain tumor — among the most challenging cancers to treat due to their delicate location — have seen significant advancements in treatment options with the advent of immunotherapy. This treatment approach includes a variety of modes such as checkpoint inhibitors, CAR T-cell therapy, and cancer vaccines, each designed to boost the immune system’s ability to recognize and destroy cancer cells.

Checkpoint inhibitors work by blocking the proteins that cancer cells use to hide from the immune system, essentially “unmasking” the cancer cells and allowing the immune system to detect and eliminate them. CAR T-cell therapy involves collecting the patient’s immune cells, genetically modifying them in a lab to better recognize cancer cells, and then infusing these enhanced cells back into the patient to seek out and destroy cancer. Cancer vaccines, on the other hand, are designed to provoke the immune system to attack specific cancer cells.

One of the key advantages of immunotherapy in treating childhood brain tumors is its potential for specificity.

By targeting the tumor cells while sparing healthy brain tissue, immunotherapy can reduce the side effects associated with conventional treatments like chemotherapy and radiation, which are particularly concerning in growing children due to the risk of long-term cognitive and developmental issues.

Moreover, immunotherapy offers the promise of durable responses, meaning that some patients may achieve long-term control over their cancer, turning it into a manageable chronic condition. This is a significant step forward in improving the quality of life and survival rates for children with brain tumors.

While the application of immunotherapy in childhood brain tumors is still an area of active research, early clinical trials have shown promising results, offering hope to families facing these daunting diagnoses. As researchers continue to understand how best to harness the immune system against these brain tumors, immunotherapy stands poised to transform the landscape of cancer treatment, potentially offering a cure for some of the most challenging cancers affecting children. Bridge To A Cure wants to accelerate progress in this area via a research initiative that draws on the databases now available and the analytical/diagnostic capabilities of artificial intelligence (AI).

Alternative, less toxic treatments warrant the same level of robust research as pharmaceuticals.

The horrific journey a child and family travel as they navigate cancer cries out for us to explore options beyond the current standard of care. Bridge To A Cure Foundation’s approach does just that by expanding the standard approach to attacking cancer cells in two ways:

1. We advocate expanding the field of attack to also address the four pathways disrupted by the cancer that compromise the body’s natural defense systems:
   • Angiogenesis — Growth of new blood vessels that fuel tumor growth.
   • Apoptosis — Normal cell death which cancer evades.
   • Immunity — The body’s mechanisms to detect and destroy
   • Metabolism — Tumors rewire metabolic processes for continuous growth.
2. We advocate expanding the arsenal of treatments to include alternative medicine and other non-traditional methodologies such as:
   • Traditional Chinese medicine
   • Vitamin therapy
   • Diet and Nutrition

By expanding the field of attack and the arsenal of treatments, the toxicity level of the standard of care approach can be reduced and the prospects for cures increased. We can also make the journey more tolerable and the outcome more hopeful for kids facing cancer.

Until recently, alternative cancer treatments, also known as Complementary and Alternative Medicine (CAM), focused on palliative care — relieving pain and other symptoms to improve quality of life. As research advances, herbal medicines and some other therapies considered “alternative” are showing promise to bolster the health and immunity of patients undergoing radiation and/or chemotherapy. For instance, extract of viscum album (Korean mistletoe) is used to bolster patients’ immune systems that have been compromised by radiation and chemotherapy.

Some CAM treatments also show promise for directly attacking the disease, and for bolstering the body’s four protective processes that can become pathways for cancer.

Attacking the Disease

Pediatric brain tumors have the highest death rate of all childhood cancers, yet are among the most poorly funded when it comes to research. A new study in the United Kingdom is looking at whether cannabidiol (CBD) can help shrink brain tumors in children. Another study is looking at how CBD promotes apoptosis in glioblastoma, the most common primary malignant brain tumor in adults.

“Numerous investigations of the last decade demonstrated cytotoxic effects of cannabinoids, including non-toxic cannabidiol (CBD) without psychogenic activity, on human and mouse glioblastoma cells,” the study states.

Boosting Apoptosis

Herbal medicines that have been used for thousands of years in China are the subject of research into their effect on promoting death of cancer cells and boosting the immune system. These include ginger, (which contains phenolic compounds that have been shown to be toxic to some tumor cells) and resveratrol (a phytoalexin found in grapes that is known to causes apoptosis in some cancers).

Angiogenesis: Cutting Cancer’s Energy Source

Tumors survive and grow through angiogenesis, forming new blood vessels that supply them with oxygen and nutrients. Research shows that many natural health products inhibit angiogenesis. These include ginger, aloe vera, green tea, garlic, grape seed extract, fish oils containing omega-3 fatty acids, resveratrol found in red wine and grapes, and antioxidants such as vitamins A, C, and E.

Metabolism: Slowing Tumor Growth

The ketogenic diet used to control spikes in blood sugar in diabetics also shows promise in inhibiting glucose that fuels growth of certain malignant tumors. In one study, researchers looked at how the diet restricts growth of glioblastoma tumors. In another, they studied effects of the diet on tumor metabolism in two pediatric oncology patients suffering from advanced stage astrocytoma, a type of brain cancer. The results were among the clearest evidence yet that changes in diet and nutrition can help fight cancer.

“Within seven days of initiating the ketogenic diet, blood glucose levels declined to low-normal levels and blood ketones were elevated twentyfold to thirtyfold. Results of PET scans indicated a 21.8% average decrease in glucose uptake at the tumor site in both subjects. One patient exhibited significant clinical improvements in mood and new skill development during the study. She continued the ketogenic diet for an additional twelve months, remaining free of disease progression.

“While this diet does not replace conventional antineoplastic (chemotherapy) treatments, these preliminary results suggest a potential for clinical application which merits further research,” the study says.

Immunotherapy: Bolstering the Body’s Natural Defense System

The body’s natural immune system is often ineffective in fighting off cancer because it does not recognize cancer cells as intruders. Immunotherapy, also known as biological therapy, helps boost the body’s immune response and train it to identify and destroy cancer cells. It includes stem cell transplants that are helping young patients beat neuroblastoma, leukemia, and lymphoma

Immunotherapy can be used as an alternative to chemotherapy and radiation and the damaging long-term side effects they can cause for children suffering from cancer.

“Immunotherapy has already led to major advances in the treatment of many types of cancers, and has been approved as a first line of treatment for several. Its effectiveness has also been proven against types of cancer that have been historically resistant to chemotherapies and radiation treatments,” states an article from Cancer Research Institute.

Adjusting the Sails

The journey children with cancer and their families travel is horrific and the outcomes often discouraging. At Bridge to A Cure Foundation we want to reduce the agony of the journey and increase the probability for happier outcomes. Expanding the field of attack and the arsenal of treatments to reduce toxicity and improve outcomes offers that potential.

There is a famous quote by William A. Ward: “The pessimist complains about the wind; the optimist expects it to change; the realist adjusts the sails.” Rather than continuing the current standard of care and expecting a different outcome, it’s time we take the realist’s approach. At Bridge to A Cure Foundation, we are working to adjust the sails.

Bill Gates + Bridge to a Cure = Path Forward

In Bill Gates’ 2015 TED Talk titled “The next outbreak? We’re not ready,” Bill warned that we were woefully unprepared to deal with the threat of a pandemic. He was right.

In the Bridge to a Cure Foundation’s March 2017 release, we identified the five barriers contributing to the unacceptably slow development of pediatric cancer treatments and cures.  The same is true for infectious diseases.

Globally the coronavirus has infected millions, killed hundreds of thousands, and decimated national economies and the lives of many families. Bottom line: we weren’t prepared. The next time — and there will be a next time — we must be ready. The Path Forward is now and here’s how we should implement it:

• Develop the response plan proposed by Bill Gates and be ready to implement it to identify infectious disease outbreaks early: Gates proposed we look at how the military prepares for war and use that as a model to prepare for the net pandemic. This includes having trained “soldiers” ready to deploy, reserves we can scale up rapidly, and war games-like training simulations. “Those are the kinds of things we need to deal with an epidemic,” Gates said.
• Ensure that the required numbers of trained human resources are prepared to deploy: This includes epidemiologists ready to study the disease and identify how far it has spread, along with medical teams trained and ready to treat patients. Using the Ebola outbreak as an example, Gates noted we were unprepared in these areas and warned: “A large epidemic would require us to have hundreds of thousands of workers.”
• Advance preparedness for clinical research to combat pandemics: Speaking of our lack of preparedness for Ebola, Gates said, “There was no one there to look at treatment approaches. No one to look at the diagnostics. No one to figure out what tools should be used. As an example, we could have taken the blood of survivors, processed it, and put that plasma back in people to protect them. But that was never tried.” In an article titled “Advancing Preparedness for Clinical Research During Infectious Disease Epidemics,” the authors state: “Clinical research is vital for an effective response to infectious disease epidemics. To be viable, preparations must be made in anticipation of infectious disease epidemics and must address barriers to rapid deployment and implementation.”

To meet these challenges we should follow the same approach developed by the Bridge to a Cure Foundation to modernize pediatric cancer research by unleashing resources already available today. Measures we propose include:

• Develop a robust national database for each microbial threat: As the article notes, “In many instances the point at which an epidemic occurs is the only time to gather data and generate new knowledge regarding disease characterization, prevention, and treatment. Experience from previous epidemics highlights how time and again, the research response is delayed and the narrow window of opportunity for enrolling patients during peak epidemic waves is missed.” We must provide a process and framework to capture the hundreds of millions of pages of research, medical files, and the science behind every treatment, drug and alternative medicine so that this data is available in advance of the next pandemic. To learn more, read our Sept. 24, 2019 blog Dying in the Dark and the April 15, 2019 blog Stop Pediatric Cancer Like We do Terrorists.
• Include alternative medicine/treatments: Develop a methodology to evaluate/approve alternative medicines/treatments. To learn more, read our May 29, 2018 blog Breaking Down Barriers, Bridging to A Cure.
• Capitalize on the proven capability of artificial intelligence: Research institutions have been slow to embrace this technology, trusting more in the mind of the researcher alone. Once a robust national infectious disease database is developed, we need to provide the methodologies and tools for researchers to extract meaningful findings. There are computers that can read and review hundreds of millions of scientific pages in seconds. Algorithms can be written to discover, analyze and predict from the data a robust global database would provide. To learn more, read our Feb. 15, 2019 blog Putting the Pieces Together for the Fight Against Cancer.
• Simplify, sensitize, and modernize the clinical trial process by speeding up and reducing complexity. Some ways to do this include pursuing simulated research, balancing life expectancy and quality of life, and including exemptions to allow terminal patients to participate in trials of experimental drugs. To learn more, read our Jan. 31, 2020 blog Solving the Clinical Trial Debacle and the July 25, 2019 blog The National Cancer Institute Knows How to Cure Pediatric Cancer.
• Align and collaborate across institutions and practitioners: Develop a reward and recognition system unique to infectious disease research — one that reinforces a culture of collaboration and spans across and within nations, institutions, governments and sectors. The article referenced above states, “There are multiple challenges to building equitable and mutually supportive partnerships. These include unequal access to funding, academic reward systems that incentivize competition over cooperation, silo-thinking reinforced by (artificial) disciplinary boundaries and commonly held assumptions regarding mutual capacities or needs. Fragmentation and competition among stakeholder groups, research initiatives, and disciplines represent a lost opportunity for shared expertise and learning, and to strengthen global, national, and regional research preparedness.” To learn more, read our Nov. 25, 2019 blog Why Reward Systems are Barriers to Pediatric Research Breakthroughs & How to Fix it.

We are at a moment in history where it is time to acknowledge the flaws in our approach to medical research and treatments, and how we respond to pandemics. Overcoming the barriers identified above is a good place to start. But that alone isn’t enough. We need a holistic strategic approach that engages participation from the medical community; pharmaceutical and technology companies; investment and investor communities; nonprofits/foundations and donors; and military, state, and local officials and government agencies.

It is time to recognize and value institutions and corporations for their contribution to the global good. The mantra of maximizing shareholder value alone is insufficient. Corporations should do more to better mankind — and they should be rewarded for it. In our May 2019 blog, we introduced the Millennial Organization. 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. Now is the time for the investment community and pharmaceutical industry to redefine the matrices for valuing this industry. Investing in childhood cancer would be a good place to start. To learn more, read our May 28, 2019 blog The Cure to Pediatric Cancer is at Our Fingertips.

This month, we acknowledge those committed to identifying and destroying the most horrific terrorist cells in America – cancer cells!

These terrorist cells kill more children than any other disease. If we are to destroy them, we will need to provide our healthcare professionals with the medical arsenal to fight the fiercest terrorists targeting our children.

Thanks to the insight and input collected by the Bridge to a Cure Foundation from over 120 childhood cancer researchers and practitioners, we now know what our medical professionals need to finally wipe out these terrorist cells that target our children:

• A robust and dynamic national childhood cancer database.
• Algorithms that optimize the full capability of artificial intelligence.
• A simplified, sensitized, and modernized clinical trial process.
• A methodology to evaluate/approve alternative medicines/treatments.
• Collaboration and alignment within and across institutions.
• Pharmaceutical companies motivated to invest in ending childhood cancer.

CHILDHOOD CANCER HEROES

There were many institutions and medical professionals involved in the identification and development of these remedies for which we are appreciative. Today, we would like to give special recognition to those whose counsel and support have made a significant contribution to Bridge to a Cure’s work to identify the armaments we need to win the war on childhood cancer:

• Dr. Darell Bigner – Duke Cancer Institute
• Dr. Saskia Biskup – Center for Genomics and Transcriptomics, Germany
• Dr. Melissa Bondy – MD Anderson Cancer Center
• Dr. Alberto Broniscer – University of Pittsburgh School of Medicine
• Dr. Robert Darnell – The Rockefeller University
• Dr. Ira Dunkel – Memorial Sloan Kettering
• Dr. Henry Friedman – Neuro-oncologist, Duke Cancer Center
• Dr. James Garvin – NY Presbyterian/Columbia University Medical Center
• Dr. Todd Golub – The Broad Institute
• Dr. Dirk Hadaschik – CeGat Germany
• John Kelly III – Senior Vice President, IBM
• Dr. Robert Kerbel – Sunnybrook Research Institute, Toronto
• Dr. Mark Kieran – Dana-Farber Cancer Institute
• Rebecca Lambert – NED Biosystems
• Dr. John Letterio – University Hospital Cleveland
• Dr. Duane Mitchell – University of Florida
• Dr. Filiberto Munoz – San Diego Clinic, Mexico
• Dr. Jeremy Rich – Cleveland Clinic
• Dr. Ned Sharpless – Director, National Cancer Institute
• Dr. Linda Van Aelst – Cold Spring Harbor Labs
• Dr. William Weiss – University of California, San Francisco
• Dr. Ben Williams – Anti-Cancer Alliance

We especially want to give recognition to the institutions and individuals that have advanced the effort to develop the arsenal needed to destroy childhood cancer cells. These are the ones who want to change the status quo — the ones who realize that we cannot continue to repeat what has not worked and who feel the urgency to implement a better approach to childhood cancer research.

IBM’s Watson computer system uses artificial intelligence (AI) to aid cancer research.

KEY INITIATIVES

• A robust and dynamic national childhood cancer database — The National Cancer Institute’s (NCI) Childhood Cancer Data Initiative (CCDI) aims to make it easier for researchers to learn from each of the approximately 16,000 children and adolescents diagnosed with cancer in the US each year. CCDI is the beginning of realizing Bridge to A Cure’s top objective: creating a robust national database that contains everything we know about childhood cancers. CCDI aims to maximize every opportunity to improve treatments and outcomes for children with cancer; build a connected data infrastructure to enable sharing of childhood cancer data from multiple sources; identify opportunities to make data work better for patients, clinicians, and researchers; and develop and enhance tools and methods to extract knowledge from data.

• Algorithms that optimize the full capability of artificial intelligence — We are beginning to see AI used to guide cancer management, with the most prominent example being IBM’s Watson for Oncology (WFO). Each month Watson ingests about 10,000 new scientific articles and data on 100 new clinical trials to keep up-to-date on new findings. While some point to the system’s limitations, it is important to keep in mind how new the technology is, and to recognize those who are adapting it to the real world fight against cancer. Other childhood cancer institutions experimenting with the potential of AI include: Lucile Packard Children’s Hospital Pediatric Molecular Imaging Program and the Department of Pediatrics, Hematology/Oncology Section, both at Stanford University School of Medicine; Memorial Sloan Kettering Cancer Center; The University of Texas MD Anderson Cancer Center; the Department of Pediatrics, UNC School of Medicine; and the Sherry and John Woo Center for Big Data and Precision Health at Duke University.

• A simplified, sensitized, and modernized clinical trial process — Organizations such as The New York Genome Center are using state-of-the-art genomic technologies and collaborating with leading research institutions to investigate the origins, diagnosis, and evolution of cancer. This includes working with The Broad Institute to simulate pediatric tumors in the laboratory to the map the capacity of tumors to develop resistance to drugs and drug combinations.

• A methodology to evaluate/approve alternative medicines/treatments — Opposition to alternative treatments is one of the barriers we’re working to remove in the fight against pediatric cancer. Some efforts worthy of recognition include research studies in Complementary and Alternative Medicine (CAM) to reduce symptoms and improve well-being for children with cancer. While most of these have been limited to single institutions, publications such as The Journal of Alternative and Complementary Medicine are providing peer-reviewed studies to evaluate and integrate of CAM into mainstream medical practice. Medical marijuana is another promising area. Research by Yale Cancer Center showed a majority of pediatric cancer providers endorse its potential use for children with advanced cancer, although clinicians want to see much stronger clinical evidence that marijuana treatments can help relieve symptoms such as nausea and pain.

• Collaboration and alignment within and across institutions — We see increasing recognition of our goal of increasing collaboration among groups such as Stand Up To Cancer. SU2C funds “multidisciplinary and multi-institutional collaborative research projects that address critical problems in cancer prevention, diagnosis, and treatment to deliver near-term patient benefit.”

• Motivating pharmaceutical companies to invest in ending childhood cancer — As millennials become the dominant force in corporate culture, they demand companies work to improve the common good and not just the bottom line. Impact investing is one way to achieve both financial and social returns. Although impact investing is a relatively new strategy, its global market size is estimated to be $228 billion and growing, with 75% of investments generated from private investing strategies. Last year the CEO of BlackRock, the world’s largest asset manager with over $6 trillion in investments, called on CEOs of the world’s largest public companies to not just deliver profits but to fulfill their responsibility to make “a positive contribution to society.” The time is right to capitalize on the movement to value companies for their contributions to improving the human condition in addition to sustainable profit growth.

These are just a few of the innovations we want to highlight for International Children’s Cancer Day, which took place on February 15th. There are many others. As we raise awareness of the children battling cancer, we must focus and accelerate the momentum toward achieving the breakthroughs kids and their families so desperately need.

In closing, we want to dedicate International Childhood Cancer Day to all the childhood cancer researchers and child oncologist professionals for their dedication, caring, and drive to make a difference. Their commitment to saving children is a challenge both intellectually and emotionally. These dedicated professionals are motivated by the desire to save lives and to stop the diseases that remain the most common cause of death among children in America.

Much has been written about why the U.S. healthcare system costs so much and rates so poorly compared with healthcare in other industrialized nations.

Last year healthcare spending skyrocketed to $3.65 trillion, an increase of $250 billion in just two years. That’s more than the GDPs of many countries – including Brazil, the U.K., Mexico, Spain, and Canada – and by far the highest in the developed world.

“The national doctor bill dwarfs anything else we spend money on, including food, clothing, housing, or even our mighty military,” states a 2017 analysis in The Atlantic. It found that five percent of patients, including people with chronic diseases and victims of accidents and violence, account for 50 percent of all medical costs.

“For most people, the vast majority of all the health care they’ll ever get comes near the hour of death. Hundreds of billions of dollars each year are spent treating Americans who are in the last weeks, or days, of life.”

Medical research and development accounted for $171.8 billion in 2016, according to Research!America, less than 4.9 percent of the total $3.5 trillion healthcare bill that year. Hospital and surgery costs accounted for 32 percent, $1.1 trillion. Physician care accounts for about 20 percent and prescription drugs about 10 percent among medical spending categories.

People age 55 and over were responsible for 56 percent of health spending, according to a Kaiser Family Foundation analysis. It found that 36 percent of spending went toward people age 65 and older, while young people 18 and under comprised only 10 percent. Adults age 19 to 64, the largest group, made up 54 percent of spending.

The bottom line: we’re spending only about five percent of our total medical costs on research and 95 percent on everything else. This formula has not served us well when it comes to discovering cures for cancer and other chronic and terminal conditions, including those that affect children.

This fact weighs on my mind and informs our work at Bridge to a Cure. It’s especially acute for me when October rolls around. Oct. 8 is the second anniversary of the day we lost my granddaughter Clara to brain cancer. Her positive attitude and courage never wavered. Her memory inspires me every day to challenge the status quo and to address the barriers that prevent researchers from delivering the breakthroughs our children expect and deserve.

Are we spending that ever-growing $3.65 trillion annually for healthcare as wisely as we should? The answer is an obvious and emphatic “No!” It’s clear to me we would be better off if we reallocated a large chunk of the money we are paying out for hospitalizations, surgery, and end-of-life care and put it into research – beginning with the building of a robust national pediatric cancer database.

Many of the treatments we use today to fight cancer and other diseases, such as radiation and chemotherapy, have horrific side effects. Then there are those hundreds of billions of dollars we spend each year treating folks in their final months, weeks, or days of life. It’s no wonder our healthcare system is so costly, so inefficient, and so ineffective. It’s like we are pouring money into a deep hole, and just keep doing it because it’s the way we have always done it.

We have to find a better way to reallocate more spending to research, and to ensure we spend those dollars as wisely as possible. We advocate a process that focuses research dollars into the five areas we believe hold the most promise when it comes to finding cures and effective treatments for pediatric cancer and other conditions affecting children:

1. Develop a robust national database for each pediatric disease and disorder. Provide a process and framework to capture the hundreds of millions of pages of research, medical files, and other information.
2. Align and collaborate across institutions and practitioners. Develop a reward and recognition system unique to pediatric research – one that reinforces a culture of collaboration and spans from researcher to investor.
3. Include alternative medicine/treatments. Provide an assessment process to qualify as a treatment option in database.
4. Capitalize on the proven capability of artificial intelligence. Provide methodology and tools for researchers to extract meaningful findings from the robust database.
5. Simplify, sensitize, and modernize the clinical trial process. Streamline processes, pursue simulated research, and balance life expectancy and quality of life, including exemptions to allow terminal patients to participate.

Healthcare spending continues to grow at an alarming rate and is expected to approach 20 percent of GDP in the coming decade. The time is now to for us to demand that we redirect more of that spending to research. It’s the key to curing disease and preventing it in the first place – and to begin reining in costs of our out-of-control system.
To read our prior posts providing solutions to improving our approach healthcare 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 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.

Remember the “Cancer Moonshot” that former Vice President Joe Biden launched? It’s now called “Cancer Breakthroughs 2020,” and aims to find vaccine-based immunotherapies against cancer. More than 50 leading cancer doctors recently released a report recommending 13 priority areas to speed up the process.

The priorities include worthy goals such as “a sped-up drug discovery process” including “expansion of the cancer center drug pipeline and reduced development costs through partnerships,” and “expanded patient access to new drugs through larger clinical trials, including increased participation of pediatric and minority patients.” Good ideas, but they are unlikely to succeed unless we change our whole approach to fighting cancer.

As part of the “moonshot,” Congress appropriated $1.8 billion last year to fund cancer research over the next seven years. No one has voiced opposition because the cause is noble. But it is will be waste of money unless we take action to tear down the barriers that have so far kept us from conquering cancer.

Think about it: December 23 will mark 46 years since President Richard Nixon signed the National Cancer Act of 1971, launching the so-called “War on Cancer.” Including the $1.6 billion the federal government earmarked at that time, the National Cancer Institute has spent an estimated $115 billion on research and development of treatments since then. Yet almost 40 percent of us will be diagnosed with cancer at some point during our lives, and of that 40 percent, 36 percent will die within five years.

Why does curing cancer continue to elude our best medical minds? Does anyone think this “moonshot” will fare any better?

The search for effective treatments and cures will take forever if we don’t fundamentally change the way we are doing things. For instance, continuing to rely heavily on clinical trials is folly because of the snail’s pace of this process. We need to take action.

We can start by tearing down these 5 deadly barriers impeding the timely development of cancer treatments and cures:

1. Absence of a robust national database. Building such a database must be our first priority.

2. Inability of even the most brilliant scientific mind to digest the millions of pages of research and medical files from which to assess and treat patients.

3. Inadequate collaboration within the scientific community.

4. Clinical trials are costly, slow and complex.

5. Opposition to alternative treatments.

Tearing down these barriers is now our primary mission at Bridge to a Cure with a special focus on pediatric cancers. It’s time to shift the mindset and culture that have kept us from beating cancer. It’s time for academia and government to adopt the practices that have made corporate America successful – practices that I know well from my work turning around companies and divisions.

In the days ahead we will delve into each of the barriers, and what we must do to overcome them. Until we meet the challenge, medical science will be “moonstruck” in its search for cures.

What’s preventing our great medical minds from developing effective cancer treatments?

What all successful organizations have in common that is missing in medical research is the following:

1. Access to information.

Even the medical researchers find it frustrating, if not unbelievable, that there isn’t a single robust database from which they can access all the information there is on any disease. A single database should include:

• All known data on the disease itself (i.e., causes, energy sources, symptoms, molecular structure, compromised pathways).
• Patient history on all known victims of the disease.
• Data on all known therapies and their recorded outcomes (including traditional medical and pharmaceutical treatments, as well as alternative therapies).

2. Fast, reliable product development processes.

The development, testing, and launching of new products is what keeps great companies great. The best companies continually review their processes to find ways to bring better products to market faster. Not so with medical research. They continue to rely on clinical trials despite their poor history.

While protecting the welfare of patients is the intent of the clinical trial process, it is too costly, slow, bureaucratic, complex, and undersubscribed. With the establishment of a robust database, supported by Artificial Intelligence (AI) and predictive algorithms, our best medical research minds will have the opportunity to dramatically improve the process and the speed in which life-saving treatments are brought to market. Technology companies such as IBM and Tempus have already made tremendous progress in this area. Now it’s up to all medical research centers and government agencies to align their processes to capitalize on their technology.

3. A culture of collaboration.

Successful corporations not only excel at collaborating within their own organization, but they also proactively collaborate with their strategic partners; they willingly share data and solutions with the single goal of exceeding customer expectations. While some progress is being made in the medical research arena, they have a long way to go.

Unfortunately, my sense is that they think they are already there. Not so! There are far too many research centers, foundations, and government agencies that are spending billions on initiatives that would be better served by working together. Not only would this generate better ideas, it would cut costs by eliminating so much of the unnecessary overhead that exists today. There needs to be alignment across research centers, non-traditional treatment practitioners, corporations, government agencies (NIH)and foundations.

Learn more about my mission to improve cancer research by visiting www.BridgeToACure.org.