NIH launches program to offer molecular characterization of childhood cancers

In support of President Biden’s Cancer Moonshot℠ goal of fostering data sharing in cancer research, the National Cancer Institute, part of the National Institutes of Health, has launched the Molecular Characterization Initiative for pediatric tumors.

The program offers tumor molecular characterization, also called biomarker testing, to children, adolescents, and young adults with newly diagnosed central nervous system tumors who are being treated at hospitals that are affiliated with the Children’s Oncology Group (COG)(link is external), an NCI-supported clinical trials group that includes more than 200 hospitals and institutions that treat most children diagnosed with cancer in the United States.

The Molecular Characterization Initiative is offered through NCI’s Childhood Cancer Data Initiative, which was launched in 2019 to promote data sharing and collection of new data among researchers who study childhood cancers.

Children, adolescents, and young adults diagnosed with a central nervous system cancer across the United States will be eligible to receive molecular characterization of their tumors free of charge through this voluntary program. DNA and RNA from tumor and blood samples will be analyzed to help make an accurate diagnosis and to understand what is causing or driving the cancer. The Molecular Characterization Initiative will expand later in 2022 to include soft tissue sarcomas and other rare tumors.

“The ultimate dream has really been for every child with cancer to have a state-of-the-art diagnosis and the safest and most effective therapy,” said Brigitte C. Widemann, M.D., special advisor to the NCI director for childhood cancer. “The Molecular Characterization Initiative is a transformative collaboration that will entail participation of the entire community.”

Having a precise diagnosis based on the molecular characteristics of a patient’s tumor can help doctors choose the most effective and potentially least toxic treatment for each child. Data on the molecular changes seen across childhood cancers can also help researchers better understand the molecular causes of childhood cancers and accelerate the development of new, more effective, and potentially less toxic treatments, especially for rare childhood cancers for which treatment options are limited. 

“The game changer for patients is that we’re going to understand the patient’s disease precisely and comprehensively in a way that we’ve done piecemeal so far,” said Douglas S. Hawkins, M.D., group chair of COG.

Previously, comprehensive tumor molecular characterization was available to children enrolling in some clinical trials or to those being treated at larger institutions with internal resources to offer such state-of-the-art diagnostics. Data on tumor biomarkers were stored exclusively at the hospital or institution where a child was treated, with limited sharing of data among institutions. The new program will make tumor molecular characterization broadly available for children throughout the country. Moreover, the data collected will be available in a central location so that childhood cancer researchers can learn from the data and use it to inform future studies.

“We can help make molecular characterization available throughout the country so that it will be a standard of care that every child can get,” said Maryam Fouladi, M.D., COG’s central nervous system tumor disease committee leader. “An accurate molecular diagnosis can inform optimal treatment for every child.”

For example, Dr. Fouladi explained, some childhood cancers, such as gliomas, can be misdiagnosed. “We can apply molecular diagnostics to a child diagnosed with a high-grade glioma and find out that it is actually a low-grade glioma or an entirely different tumor, which may need very different treatments and have a very different outcome,” she said. “Molecular diagnostics can really contribute to getting the correct diagnosis, offering the optimal treatment and, ultimately, improving the patient’s outcome.”

In addition to providing detailed information about a cancer to use in making an accurate diagnosis, the data can also be used to determine whether a child is eligible for a clinical trial. Molecular characterization can reveal, for example, whether a child has a specific cancer subtype that is eligible for a clinical trial evaluating a new treatment explicitly designed for that subtype.

Enrollment in the Molecular Characterization Initiative is initially offered through participation in Project:EveryChild(link is external), a childhood cancer registry maintained by COG (APEC14B1). Initial participants will include newly diagnosed children, adolescents, and young adults ages 25 years and younger at the time of diagnosis. Young adults over the age of 25 who are being screened for eligibility into a COG clinical trial may also be included.

Tumor and blood samples from participants will be sent to an accredited lab for analysis, and the results will be available to patients and their families within 21 days. The molecular data will also be aggregated into a database available to researchers for future studies, such as those exploring why some tumors become resistant to therapies they initially responded to or what factors increase the risk of treatment-related side effects. Personal information that could be used to identify a participant will be removed before data are put into the database.

Dr. Widemann said the Molecular Characterization Initiative is a program the childhood cancer community can easily get behind. “To be able to apply the best tools to make the most accurate diagnosis so that the most effective treatment can be prescribed, that’s a goal that I think physicians and families can all align around,” she said.

WHO and St. Jude to dramatically increase global access to childhood cancer medicines

The World Health Organization and St. Jude Children’s Research Hospital today announced plans to establish a platform that will dramatically increase access to childhood cancer medicines around the world.

The Global Platform for Access to Childhood Cancer Medicines, the first of its kind, will provide an uninterrupted supply of quality-assured childhood cancer medicines to low- and middle-income countries. St. Jude is making a six-year, US$ 200 million investment to launch the platform, which will provide medicines at no cost to countries participating in the pilot phase. This is the largest financial commitment for a global effort in childhood cancer medicines to date. 

“Close to nine in ten children with cancer live in low- and middle-income countries,” said Dr Tedros Adhanom Ghebreyesus, WHO Director-General. “Survival in these countries is less than 30%, compared with 80% in high-income countries. This new platform, which builds on the success of the Global Initiative for Childhood Cancer launched with St. Jude in 2018, will help redress this unacceptable imbalance and give hope to many thousands of parents faced with the devastating reality of a child with cancer.” 

Affordable, good quality and uninterrupted cancer medicines for children

Each year, an estimated 400 000 children worldwide develop cancer. The majority of children living in low- and middle-income countries are unable to consistently obtain or afford cancer medicines. As a result, nearly 100 000 children die each year. 

The new platform aims to provide safe and effective cancer medicines to approximately 120 000 children between 2022 and 2027, with the expectation to scale up in future years. This platform will provide end-to-end support  ̶  consolidating global demand to shape the market; assisting countries with the selection of medicines; developing treatment standards; and building information systems to track that effective care is being provided and to drive innovation. 

“St. Jude was founded on the mission to advance research and treatment of childhood cancer and other catastrophic pediatric diseases. Nearly 60 years later, we stand with the World Health Organization, partner organizations and our Global Alliance collaborators to expand that promise for children worldwide,” said James R. Downing, M.D., president and CEO of St. Jude. “With this platform, we are building the infrastructure to ensure that children everywhere have access to safe cancer medicines.”

This innovative approach will open a new chapter in access to cancer care by addressing medicine availability in low- and middle-income countries that is often complicated by higher prices, interruptions in supply and out-of-pocket expenditures that result in financial hardship.

According to a WHO Noncommunicable Disease Country Capacity survey published in 2020, only 29% of low-income countries report that cancer medicines are generally available to their populations compared to 96% of high-income countries. By consolidating the needs of children with cancer globally, the new platform will curtail the purchasing of sub-standard and falsified medicines that results from unauthorized purchases and the limited capacity of national regulatory authorities.

“Unless we address the shortage and poor quality of cancer medicines in many parts of the world, there are very few options to cure these children,” said Carlos Rodriguez-Galindo, M.D., executive vice president and chair of the St. Jude Department of Global Pediatric Medicine and director of St. Jude Global. “Health-care providers must have access to a reliable source of cancer medicines that constitute the current standard of care. We at St. Jude, with our co-founding partners at WHO and many vital partners around the world, can help achieve that.” 

“WHO, St Jude and partners will spare no efforts to get children’s access to cancer medicines on track,” added Dr Bente Mikkelsen, Director of the Department of Noncommunicable Diseases at WHO. “WHO is on the ground, working with governments to deliver support and services to ensure that all children have access to the best cancer treatment possible.”

Pilot phase in 12 countries

During an initial two-year pilot phase, medicines will be purchased and distributed to 12 countries through a process involving governments, cancer centers and nongovernmental organizations already active in providing cancer care. Discussions are already ongoing with governments to determine the countries which will participate in this pilot phase. By the end of 2027, it is expected that 50 countries will receive childhood cancer medicines through the platform.  

Kathy Pritchard-Jones, president of the International Society of Paediatric Oncology, said; “We look forward to working with St. Jude and WHO on this journey to ensure all children, everywhere, have access to quality cancer medicines. The platform is bringing forth a dream of our more than 2600 global members.” 

João Bragança, president of Childhood Cancer International, added: “Cancer should not be a death sentence, no matter where a child lives. By developing this platform, St. Jude is helping families get access to lifesaving medicines for their children. Working together, we can change the outcome for cancer-afflicted children around the world.” 

Meet Zach Sobiech | My Last Days

Meet Zach, a forever 18 year old osteosarcoma warrior. Zach was a compassionate and loving soul with a passion for living life to the fullest.

In 2009, Zach went on a run with his sister after a lazy summer. When he came home, he was complaining of a sore hip. His mother, Laura said if it persisted, they’d go to the doctor. After a few weeks, the pain was still there so Zach and his mom went to the doctor. The doctor had an x-ray performed, ruled out anything major and sent him home. After the pain got worse, Laura decided to take him back to the doctor where an MRI was ordered. Zach’s parents, Laura and Rob both accompanied him to the MRI where they found out that he had a tumor. The doctor wasn’t completely sure what type of tumor and said it could be one of three possibilities – lymphoma, fibrous dysplasia or osteosarcoma. “Osteosarcoma is the worst of the three,” Rob said, in the book, “Clouds, a memoir.” 

Zach was officially diagnosed after a biopsy was performed. The doctor confirmed Laura and Rob’s worst fears, osteosarcoma. “The words hit me with the percussion of a bomb; I couldn’t breathe and my ears rang. As much as I had prepared myself to hear those words, I wasn’t ready for their impact” remembers Laura. 

Credit: American Childhood Cancer Organization

Nearly half of childhood cancers worldwide undiagnosed

Nearly half of all childhood cancers are not being diagnosed globally, according to a new modeling study led by Harvard T.H. Chan School of Public Health.

A mother and baby in Burkina Faso.

The study found that, in 2015, there were 397,000 cases of childhood cancer worldwide, but only 224,000 were diagnosed. And if health systems around the world don’t improve, the researchers estimate that 2.9 million out of 6.7 million projected childhood cancer cases—43%—will be missed between 2015 and 2030.

The study was published February 26, 2019 in Lancet Oncology. Watch a video with the authors.

“Our model suggests that nearly one in two children with cancer are never diagnosed and may die untreated,” said lead author Zachary Ward, a doctoral student in health policy at Harvard Chan School. “This new model provides specific estimates of childhood cancer that have been lacking.”

Accurate estimates of childhood cancer incidence are needed to inform health policies, but many countries don’t have cancer registries that quantify this incidence. In addition, existing registries may underestimate the true incidence of childhood cancer, according to the authors.

In the new study, researchers developed a model to simulate childhood cancer incidence for 200 countries and territories worldwide. The model included data from cancer registries in countries where they exist. It also took into account trends in population growth and urbanization, geographical variation in cancer incidence, and health system barriers to access and referral that contribute to underdiagnosis.

The prevalence of undiagnosed cancer cases varied widely across regions, from just 3% in western Europe and North America to 57% in western Africa, the study estimated. In south Asia, 49% of cases were undiagnosed. The researchers said that 92% of new cases of cancer are occurring in low- and middle-income countries, a higher proportion than previously thought.

The authors hope that their findings will help guide health systems in setting new policies to improve diagnosis and management of childhood cancers.

“Health systems in low-income and middle-income countries are clearly failing to meet the needs of children with cancer,” said Rifat Atun, professor of global health systems and senior author of the study. “Universal health coverage, a target of United Nations Sustainable Development Goals, must include cancer in children as a priority to prevent needless deaths.”

Funding for the study came from Boston Children’s Hospital, Dana-Farber Cancer Institute, Harvard T.H. Chan School of Public Health, Harvard Medical School, the National Cancer Institute, SickKids, St. Jude Children’s Research Hospital, and the Union for International Cancer Control.

WHO kicks off global initiative to treat children with cancer

WHO has announced a new Global Initiative for Childhood Cancer – with the aim of reaching at least a 60% survival rate for children with cancer by 2030, thereby saving an additional one million lives. This new target represents a doubling of the global cure rate for children with cancer.
Cancer is a leading cause of death for children, with 300,000 new cases diagnosed each year among children aged 0-19 years.

According to the International Agency for Research on Cancer (IARC) latest estimates on the global burden of cancer, the global cancer burden is estimated to have risen to 18.1 million new cases and 9.6 million deaths in 2018.

One in 5 men and one in 6 women worldwide develop cancer during their lifetime, and one in 8 men and one in 11 women die from the disease. Worldwide, the total number of people who are alive within 5 years of a cancer diagnosis, called the 5-year prevalence, is estimated to be 43.8 million – IARC

The GLOBOCAN 2018 database, accessible online as part of the IARC Global Cancer Observatory, provides estimates of incidence and mortality in 185 countries for 36 types of cancer and for all cancer sites combined.

An analysis of these results, published in CA: A Cancer Journal for Clinicians, highlights the large geographical diversity in cancer occurrence and the variations in the magnitude and profile of the disease between and within world regions.

The increasing cancer burden is due to several factors, including population growth and ageing as well as the changing prevalence of certain causes of cancer linked to social and economic development. This is particularly true in rapidly growing economies, where a shift is observed from cancers related to poverty and infections to cancers associated with lifestyles more typical of industrialized countries.

Cancer occurs in people of all ages and can affect any part of the body. It begins with genetic changes in a single cell that then grows out of control. In many cancers, this results in a mass (or tumour). If left untreated, cancer generally expands, invades other parts of the body and causes death.

According to the WHO, current data suggest that approximately 10% of all children with cancer have a predisposition because of genetic factors. Ongoing research is needed to identify factors impacting cancer development in children.

Unlike cancer in adults, the vast majority of childhood cancers do not have a known cause. Many studies have sought to identify the causes of childhood cancer, but very few cancers in children are caused by environmental or lifestyle factors. Cancer prevention efforts in children should focus on behaviours that will prevent the child from developing preventable cancer as an adult.

Some chronic infections are risk factors for childhood cancer and have major relevance in low- and middle-income countries. For example, HIV, Epstein-Barr virus and malaria increase the risk of some childhood cancers. Other infections can increase the child’s risk of developing cancer as an adult, so it is important to be vaccinated and other pursue other methods such as early diagnosis or screening to decrease chronic infections that lead to cancer, whether in childhood or later.

 

New trans-NIH consortium aims to advance pediatric research on a global level

The National Institutes of Health has formed the Trans-NIH Pediatric Research Consortium to coordinate pediatric research programs across its institutes and centers.

Nearly all of the 27 NIH institutes and centers fund some aspects of child health research. In fiscal year 2017, this support totaled more than $4 billion. The new consortium aims to harmonize these activities, explore gaps and opportunities in the overall pediatric research portfolio, and set priorities.

“NIH-funded research has resulted in tremendous advances against diseases and conditions that affect child health and wellbeing, including asthma, cancer, autism, obesity, and intellectual and developmental disabilities,” said Diana W. Bianchi, M.D., director of the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), the lead NIH institute for the consortium. “This consortium aims to capitalize on this momentum by enhancing crosstalk between scientific disciplines to address the wide range of health conditions experienced by children in this country and around the world.”

The new consortium will be led by the NICHD Director. In addition to project-based interactions, the full consortium will meet several times a year to discuss scientific opportunities and potential new areas of collaboration, including efforts to enhance training for the next generation of pediatricians.

NICHD conducts and supports research in the United States and throughout the world on fetal, infant and child development; maternal, child and family health; reproductive biology and population issues; and medical rehabilitation.

The nation’s medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases.

INTERNATIONAL CHILDHOOD CANCER DAY, 15 FEBRUARY 2018

PRESS RELEASE

INTERNATIONAL CHILDHOOD CANCER DAY, 15 FEBRUARY 2018.

Childhood Cancer International

Advancing Cures, Transforming Care, Instilling Hope

Rowaca Cancer Group – Sierra Leone

Freetown, Sierra Leone, 15 FEBRUARY 2018: International Childhood Cancer Day (ICCD) is celebrated around the world each year on February 15th. Originally commemorated in 2002, ICCD is a day founded by Childhood Cancer International (CCI), a global network of 188-member organizations in 96 countries – including Rowaca Cancer Group – Sierra Leone, Childhood Cancer International is committed to advancing cures, transforming care, and instilling hope for all children and adolescents diagnosed with cancer in the world, wherever they may live.

Childhood Cancer International is not alone in recognizing the devastating impact of childhood cancer on children and families around the globe. In September 2011, the United Nations (UN) General Assembly issued a Political Declaration recognizing four major Non-Communicable Diseases/NCDs (cancer, cardiovascular disease, diabetes and chronic respiratory disease) as the greatest killers of adults and children.

Sadly, childhood cancer continues to be the leading cause of non-communicable related death in children throughout the world. Globally, more than 300,000 children are diagnosed with cancer each year, and in Sierra Leone more cases are being reported. Approximately 80 percent of our world’s children live in low-middle-income countries (LMICs) where more than 80 percent of these children die of their disease. In developed countries like the United States, Canada, United Kingdom, Japan and others, more than 80 percent of children survive cancer with hope to live productive and meaningful lives.

On July 6, 2017, the United Nations General Assembly adopted a global indicator framework for achievement of the Sustainable Development Goals (SDGs) and 2030 Global Health Targets. Sustainable Development Goal 3.4 challenges countries to: “By 2030, reduce by one-third of premature mortality from Non-Communicable Diseases through prevention and treatment, and promote mental health and well-being.” Childhood Cancer International agrees that making childhood cancer a national and global child health priority is a critical first step towards reducing premature child mortality 30 percent by 2030, providing a crucial milestone for countries to obtain this United Nation’s goal.

Childhood cancers are often curable but too many children and adolescents have no hope to overcome their disease simply because they were born in a country entrenched in poverty resulting in late diagnosis, lack of access to life-saving essential medicines and appropriate treatment.

There can be no more ‘but.’ All children in the world deserve hope for a cure – no matter where they live – not more excuses. We can no longer sweep this issue “under the rug.” Children are the future of our country and our world. Their vitality is the heartbeat of our world, a shared passion that can unite us because our future as a global community depends on it.

On International Childhood Cancer Day, all members of Childhood Cancer International stand united to make childhood cancer a national and global child health priority to ensure there are adequate resources to meet the basic rights of children with cancer. We believe those basic rights for all children diagnosed with cancer include:

• The right to early and proper diagnosis;
• The right to access life-saving essential medicines;
• The right to appropriate and quality medical treatments, and;
• The right to follow up care, services and sustainable livelihood opportunities for survivors.

Furthermore, if a cure is not attainable, CCI stands by the right of the child to experience a pain-free death. While unfathomable in developed countries, the shocking reality for a majority of low-middle income nations is that children suffering from cancer will die excruciating deaths without any supportive care or pain management.

There can be no more ‘but.’ United together towards a shared vision we can advance cures, transform care, and instill hope. Together we must take action to reduce premature child cancer mortality.

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Rowaca Cancer Group – Sierra Leone, 17 Off Kingharman Road, Brookfields, Freetown.  Email: rowacacancersl@gmail.com