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2022 Research Grants

CURE CHILDHOOD CANCER PROUDLY ANNOUNCES MORE THAN $4.7 MILLION IN RESEARCH GRANTS

Research is the key to ensuring children with cancer have the opportunity to thrive long beyond diagnosis. CURE’s research priorities are two-fold. First, we prioritize research that will lead to more effective treatments for children within two to three years. We also prioritize research aimed at solving recurrent and hard-to-treat cancers for which no effective treatment exists. Precision medicine, gene-based therapy, continues to be our highest priority, as we believe this approach to treatment shows extraordinary promise.

In response to CURE’s request for proposals, we received dozens of studies seeking funding. Expert oncologists and academic researchers reviewed and scored each proposal using the same process employed by the National Institutes of Health. Their scores and critiques guided CURE’s board, ensuring we invest funds in the most strategic and prudent fashion.

We are proud to share that our 2022 research grants are focused almost entirely on helping children facing the toughest diagnoses – high-grade brain tumors, aggressive leukemias and solid tumors, and metastatic disease. These 18 grants awarded to top scientists at leading pediatric cancer research institutions across the nation total more than $4.7 million, CURE’s highest disbursement in a single grant cycle in our 47 year history.

“We are so pleased and proud that CURE is providing such a high level of support to very promising research this year,” said Kristin Connor, CEO of CURE Childhood Cancer. “We are urgently focused on getting new treatments to those children with high-risk, difficult-to-treat cancers that currently lack effective treatments. Virtually all our grants this year are aimed at doing that, which gives me so much hope for these children.”

The grant includes funding for these difficult to treat cancers:

  • 5 High-risk blood cancer studies (3 AML, 2 B-cell ALL)
  • 7 High-grade brain tumor studies (3 DIPG, 1 high grade glioma, 3 medulloblastoma)
  • 2 High-risk solid tumor studies (neuroblastoma and Ewing sarcoma)

In an effort to ensure the best and brightest young minds are trained to care for children with cancer and advance research, we also proudly announce our funding of the fellowship training of three pediatric oncology fellows at Emory University’s School of Medicine: Dr. Frank Chien, Dr. Robert Lisac (Sam Robb Fellow), and Dr. Sanyu Janardan (Connolly Family Fellow).

Our 2022 Pediatric Cancer Research Initiative includes the following studies:

Aflac Cancer and Blood Disorders Center at Children’s Healthcare of Atlanta

Aflac Precision Medicine Program

Robert Castellino, MD
Combined molecular targeting to enhance therapy for group 3 medulloblastoma

Tobey MacDonald, MD 
Clinical investigation of cluster-wells in pediatric brain tumors

Christopher Porter, MD
Targeting mechanisms of T cell suppression mediated by Siglec15

Sunil Sudhir Raikar, MD 
Optimizing gamma delta T-cell immunotherapy for acute myeloid leukemia

Renee Read, PhD   
Human organoid models of pediatric high-grade gliomas

Muxiang Zhou, MD
Feasibility study of VERU-111 for precision treatment of pediatric AML

Beckman Research Institute of the City of Hope

Ling Li , PhD
Targeting PRMT1 elicits anti-tumor immunity in childhood leukemia

Qiang Lu, PhD
Developing inhibitors of mitotic kinesin KIF20A for brain tumor treatment

Children’s Hospital of Philadelphia

Michael Chorny, PhD 
Macromolecular prodrug-based therapy for indolent neuroblastoma

Timothy Olson, MD, PhD
Targeting niche inflammation and MSC cell fate in monosomy 7 predisposition

Vinodh Pillai, MD, PhD 
Assessment of bone marrow to predict response to CAR T-cell therapy

Indiana University

Jignesh Tailor, PhD
Discovery of synthetic lethal targets in MYCN neuroepithelial stem cells

Seattle Children’s Hospital

Elizabeth Lawlor, MD, PhD 
Optimizing safety and efficacy of anthracyclines in Ewing sarcoma

University Hospitals Rainbow Babies & Children’s Hospital

John Letterio, MD 
Development of a CNS-penetrant synthetic oleanane triterpenoid for DIPG

University of Texas Health Science Center at San Antonio

Raushan Kurmasheva, PhD 
PEGylated talazoparib for pediatric malignant rhabdoid tumor therapy

Virginia Polytechnic Institute and State University

Jia-Ray Yu, PhD 
Pharmacological screening of a new class of NSD1 inhibitor

Washington University in St. Louis

Jason Weber, PhD  
Development of p14ARF-based therapies to treat CDKN2A-deficient pediatric cancers

Advancing Science Together

As you read this, CURE’s Peer Review Committee is evaluating 44 research proposals from scientists at 27 institutions who are seeking funding. Based on their feedback, we will select the life-saving research that we fund in our next award, and you can have a direct impact on the process.

These proposals have the potential to significantly improve treatments for pediatric cancers where current options are not adequate and, if funded, to be in clinical testing or treatment phases within 2-3 years.

The committee is reviewing cutting-edge research and will score proposals the researchers hope will lead to better treatments for cancers that affect children. These proposals include new methods to fight the most common cancers like leukemia and lymphoma. They also seek to advance science that will impact children diagnosed with the hardest to treat brain tumors, sarcomas, and metastatic disease.

These proposals have come in from leading research institutions across the country.

Each submission is being reviewed by two committee members who have specific knowledge and interest in the cancer type of the proposal. In May, we will host a meeting during which every submission will be discussed by the entire committee. Members will have the opportunity to ask questions and vote to accept or change the overall score. These final scores will be ranked and presented to CURE’s Board of Directors on June 21, and final funding decisions will be made.

This is where you come in. Before the funding decisions are made in June, you can increase the amount of funding available for these projects by renewing your gift by May 31. Additionally, because of a $100,000 matching gift from a generous donor, your impact could be doubled. This is a chance for you to make a direct impact on the way we treat childhood cancer.

The Long Journey from Idea to Bedside

Dr. Michael Jensen of Seattle Children’s Hospital is one of the leading authorities on CAR T-cell therapy. He has been working on CAR T-cell therapy for many years, and his work is proving to be very beneficial for pediatric cancer patients. This type of therapy provides hope that someday children can receive far less-toxic medicines that lead to a cure.

Dr. Jensen started his career in the late 1990s at City of Hope National Medical Center in Los Angeles, where his research looked at the technology to take immune cells from a cancer patient and genetically modify them to recognize and attack cancer cells. This is something the patient’s natural immune system can’t do. His research was groundbreaking at that time, and his lab had to build everything from scratch.

“This was similar to creating the Apollo rocket and trying to land on the moon,” Dr. Jensen said. “We had to invent and create every step of the process and make sure the quality was sufficient. Once the first cells were infused, it was literally like landing on the moon and hoping that everything went right.”

It took over a decade of small trials to work out each and every kink, and obtaining funding for something so revolutionary was challenging. One source of consistent funding came from Lauren’s Run. From the beginning, Lauren’s parents were looking for groundbreaking research that would help children with cancer. After learning of Dr. Jensen’s research, they decided that it was exactly what they were looking for and made sure proceeds from the race supported his work.

His breakthrough came in 2014, when doctors using the patient’s own immune system through CAR T-cell therapy started seeing dramatic remissions in children with leukemia who were otherwise out of options.

“Without a functioning immune system, cancer would be much more common,” explained Dr. Jensen. “Think of cancer cells as a semi-truck on a freeway down a mountainside with the brakes broken, and the gas pedal stuck on full. On the way down, the cells acquire genetic programs and mutations and become uncontrolled. The human immune system is challenged because this isn’t a virus that came from outside the body. It doesn’t help that the cancer cells create deflector screens to fool the immune system into peaceful coexistence when we would like for the immune system to attack.”

CAR T-cell therapy takes the T-cells out of the body and supercharges them. The supercharged cells are then put back into the body as a surprise attack on the cancer cells, and the deflectors are not always effective in turning off the immune response. Ideally, within a week or two, the patient goes into remission. The cells then continue to move to all parts of the body, hunting and eliminating any remaining cancer cells. Over 90% of leukemia patients whose initial treatment didn’t work go into remission when given CAR T-cell therapy!

Leukemia was the first target, but the goal is to get CAR T-cell therapy to work against other childhood cancers. CAR T-cell therapy represents a major step toward a safer cure for children. It started as an idea in the mind of brilliant researchers years ago and is now a frontline treatment for many children. But it didn’t happen overnight. It was a long process, and Lauren’s Run was with them every step of the way.

Using our Immune Systems to Cure Cancer

By Chris Porter, MD
Pediatric Hematologist/Oncologist
Aflac Cancer & Blood Disorders Center of Children’s Healthcare of Atlanta

 

Researchers have learned a great deal about how the human immune system can function as physiologic superheroes – able to prevent and cure diseases, including cancer. Like particularly cunning comic book villains, some cancers devise intricate plans to weaken their superhero foes – think kryptonite to Superman. In our lab, we have been investigating how blood cancer cells evade the immune system and have discovered that the cancer cells use a molecular kryptonite, called Siglec15, to weaken the immune cells. Fortunately, there may be ways to shield our immune cells and new ways to strengthen them, which we are actively pursuing every day.

Cancers derived from blood cells, including leukemia and lymphoma, are the most common cancers in children. Thanks to research, cure rates are better than ever. But cancer remains the leading cause of illness-related death in children. Thus, there is a need for better therapies, and understanding how cancer cells behave is critical to developing better medicines.

Dr. Chris Porter

A couple of years ago, our lab found that leukemia cells depend on a protein called calcineurin to evade immune cells. In the cell, calcineurin helps control the expression of a number of other proteins. One of the proteins that calcineurin controls is a signaling molecule, IL-12. This molecule is a member of a family of signaling proteins called cytokines. Researchers have known for a long time that IL-12 very strongly stimulates immune cells to kill cancer cells. However, IL-12 has not been an effective therapy, in part because it can cause several side effects. To get around this problem, we are collaborating with scientists at Emory University and the Georgia Institute of Technology. Together we hope to develop a nanomedicine to deliver IL-12 exactly to where it needs to be, where immune cells interact with cancer cells. This will effectively give additional super-powers to help the immune cells eliminate the cancer cells.

In addition, we found that calcineurin controls the expression of Siglec15, a protein of which not much is known. Leukemia and lymphoma cells seem to make a lot of Siglec15, which we know can inhibit immune cells. In fact, children with leukemia have much higher levels of Siglec15 in their blood than healthy individuals. Now we are trying to understand how the leukemia and lymphoma cells make it and how it inhibits the immune cells. In addition, we are working with a drug company that makes a medicine that can block Siglec15, which could act as a forcefield for the immune cells.

Thus, with funding from CURE, we are making great strides in figuring out ways to strengthen and protect immune cells, which should let these superheroes destroy the villainous cancer cells.

 

 

2021 Research Grants

CURE CHILDHOOD CANCER PROUDLY ANNOUNCES MORE THAN $3 MILLION IN RESEARCH GRANTS

When CURE’s fiscal year began, we were squarely in the midst of the COVID-19 pandemic and couldn’t begin to predict how the year ahead would unfold. As circumstances became more dire for many families we serve, we quickly realized we needed to invest more funds in supporting our patients and families. At the same time, revenue was declining as the pandemic forced us to cancel events and hold others virtually. While we were confident that our priority needed to be helping families through these extraordinary times, we did not want to let up on research support. How would we be able to do it all, we wondered? Our community answered by giving generously to ensure critical research could move forward even in the pandemic.

Thanks to you, CURE is delighted to announce more than $3 million in research grants for the 2021 fiscal year!

Our research priorities are clear. First, we prioritize research that is likely to reach the bedside within five years. Second, we focus on research that will improve the outcomes for the 20% of children not surviving today’s methods of treatment. In furtherance of these goals, we continue to prioritize precision medicine and advancing the Aflac Precision Medicine Program at Children’s Healthcare of Atlanta.

Four years ago, our funding made the Aflac Precision Medicine Program a reality. The results in these four short years have been dramatic. Since its clinical introduction, genetic sequencing of tumors has provided actionable results in 78% of the patients – meaning that the information obtained through this sequencing impacted the treatment of 78% of the children in some way. What an extraordinary impact this sequencing makes. CURE’s investment of $1.8 million this year will allow the Aflac Precision Medicine Program to provide genetic sequencing for more children in addition to going deeper with the genetic testing – in other words, analyzing even more genes that may be contributing to tumor growth. CURE’s Precision Medicine grant will also enable research into racial and ethnic differences which may affect a child’s outcome. With our funds, scientists will explore and identify the immune factors that contribute to racial and ethnic differences in outcome. This exciting research will significantly impact children of all races diagnosed with cancer now and in the future.

We are again funding the training of three future pediatric oncologists through our Fellowship Program: Dr. Frank Chien, Dr. Robert Lisac (Sam Robb Fellow), and Dr. Sanyu Janardan (Connolly Family Fellow). By funding their training, we remove financial impediments and allow the fellows to focus on research and care of children fighting cancer as they begin their careers.

Your generosity allowed us to do even more than we believed possible. In addition to our precision medicine initiative and our investment in the fellows, CURE is also funding eight individual research projects aimed at solving difficult to cure cancers. Our 2020-2021 Pediatric Cancer Research Initiative includes the following studies:

Aflac Cancer and Blood Disorders Center at Children’s Healthcare of Atlanta

Waitman Aumann, MD     
The role of SIX1 in CALM-AF10 and other t-cell leukemias

Deboray DeRyckere, PhD
Nanoparticle delivery of MRX-2843 for treatment of pediatric leukemia

Robert Castellino, MD
Identifying and targeting therapeutic vulnerabilities in DIPG

Henry Curtis, PhD
Delineating the impact of anti-Galectin-9 immunotherapy on t-cell all epigenetics and survival

Shahab Shubin, MD, PhD 
Deciphering the oncogenic potential of LIN28B in group 3 medulloblastoma

Karen Effinger, MD, MS
Evaluation of vestibular dysfunction in survivors of childhood cand adolescent cancer treated with platinum-based chemotherapies

Swati Bhasin, PhD  
Therapeutic targeting of single cell RNA Seq derived t-all blast signatures

Children’s Hospital of Philadelphia

Michael Chorny, PhD
Combination Therapy of Neuroblastoma Using Co-drug Impregnated Nanocarriers

Raegan’s Playground

Raegan lived life on her own terms. She was sweet and spunky and loved unicorns. Her parents never knew how much of a fighter she was until she had to be.

She was thriving in kindergarten when she began suffering from minor illnesses that wouldn’t go away. After shuffling back and forth to appointments and different kinds of testing, Raegan was finally diagnosed with a pediatric brain tumor called DIPG.

“To say your life can change in the blink of an eye, four letters, DIPG, completely devastated our family,” recalled Raegan’s father, Marc. “We had never heard of this disease, and what we learned broke our hearts.”

Marc and his wife, Andrea, would soon learn that DIPG is a rare brain cancer for which there is no known cure. As if that wasn’t bad enough, Raegan had the worst possible genetic mutation and was given a prognosis of 6-9 months. Both Marc and Andrea work in the medical field and began scouring the internet for information and potential clinical trials.

“We shut down everything,” said Marc. “Our entire focus became about saving Raegan’s life.”

They found three clinical trials at St. Jude’s in Memphis, but by the time they arrived, two of them had closed and the third had little information available. So they packed up and came to Children’s Healthcare of Atlanta, where there were open trials that looked promising. There they met CURE.

“When we first got admitted to the Aflac Cancer Center, we were given one of the CURE tote bags,” said Andrea. “We were going through so much right then that I was very touched to feel like someone was thinking of us.”

At the same time they were facing this monumental battle, friends and fire departments around the country began to “Rally for Raegan” by posting pictures of support and contributing to a fund meant to help with Raegan’s medical expenses.

The first clinical trial worked well for seven months. But in January 2020, Raegan began to show symptoms of progression. When they came back to Atlanta for radiation, the COVID-19 pandemic hit, and the family was forced to stay in a hotel for twelve weeks. During this time, Raegan started a new clinical trial funded by CURE. She continued this treatment for five months until her tumor again showed progression.

“This all showed us how much of a fighter Raegan was,” said Marc. “She had already lived way beyond her diagnosis, and her doctor said, ‘I can tell you what we expect, but this is Raegan we’re talking about.’”

Sweet Raegan passed away at home during a tropical storm on November 8, 2020. She outlived her original diagnosis by nearly a year. And, oh how she lived.

“Raegan never stopped smiling and laughing,” said Marc. “She turned everything into her playground. The trials we went through weren’t failures because they bought us time to be with her, and every moment was precious.”

Marc and Andrea returned to Atlanta recently to pay it forward for the support they received during Raegan’s treatment.

They wanted to pay it forward to the organizations that cared for them during Raegan’s treatment. So they split the remaining funds between CURE, the Aflac Cancer and Blood Disorders Center at Children’s Healthcare of Atlanta, and the Winship Cancer Institute where she received radiation. Despite losing Raegan, they are dedicated to advancing research that will lead to cures for children with cancer.

After their experience, Andrea shared advice for parents of newly diagnosed children.

“When your child is diagnosed, it knocks the air out of you,” she said. “Once you catch your breath, start doing your own research. There may be other options out there and new treatments on the horizon. I have no regrets. The trials gave us 18 good months, much of which we wouldn’t have had. Keep fighting every day.”

Madeline and Precision Medicine

At the age of two, Madeline’s fine motor skills were in the lower 5% range. She had more baby fat than most of her little peers, and an awkward gate when she tried to run. Her family was referred to several doctors, but never discovered the cause behind her issues.

“She fell down a lot and would cry no matter how hard the fall,” recalled her mother, Bethany. “We just thought she was sensitive, but her motor skills continued to fall behind children her age.”

Just after her third birthday, Madeline had terrible leg pain and refused to walk, so Bethany took her back to the hospital. Madeline’s blood work showed that inflammation markers were very high, so doctors ordered a total body scan. They found a football-sized mass in her abdomen that had smashed her bladder and pushed into her intestines, kidney, and liver.  A biopsy of the mass confirmed a diagnosis of neuroblastoma.

“The signs were there if we look back,” Bethany said. “Madeline wasn’t sensitive. She was probably the toughest kid around. Every one of those falls hurt her badly.”

Madeline began chemotherapy right away.  After two rounds, she was able to walk again. The chemo that proved effective at first slowly stopped working to shrink the tumor. A long surgery allowed doctors to extract 85% of what remained. Unfortunately, the tumor was too intricately woven into Madeline’s spinal column for complete removal.

Madeline was enrolled in the Aflac Precision Medicine Program (APMP) funded by CURE, and the results of her genetic testing were both interesting and useful. Madeline’s tumor had a gene mutation often associated with an aggressive form of neuroblastoma. But fortunately, the remnants of Madeline’s tumor have been stable. Knowing that Madeline has that particular genetic mutation will help doctors in the future should her tumor become active.

Another part of the APMP is the genetic predisposition program, which provides care for children who are at risk for developing cancer due to a cancer predisposition syndrome or a family history of cancer. Madeline’s younger sister, Sedona, has a known genetic disorder called hemihypertrophy. So she was referred to the genetic predisposition clinic to see if her genetic mutation was the same as Madeline’s. If a genetic link between the two was revealed, it might indicate that Sedona had a high risk of developing cancer in the future.

Although no link was found, the fact that both of their children have genetic disorders had an impact on the family as a whole.

“We’ve decided not to risk having additional biological children,” Bethany said. “If we want to grow our family in the future, there are certainly other ways to do so.”

With the mass removed from her stomach and cancer treatment behind her, Madeline is a completely different child. She loves climbing on the playground and using her body now that it works better for her. She rarely cries when she falls down, which proves she is one of the toughest four-year-old kids around.

Take the next step to support research that will help kids fighting cancer… Kids like Madeline.

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What is Precision Medicine?

In 2017, CURE made an unprecedented $4.5 million commitment to the Aflac Cancer Center of Children’s Healthcare of Atlanta to launch the Aflac Precision Medicine Program (APMP). With this award, the Aflac Cancer Center would become one of only a small handful of pediatric cancer centers nationwide able to offer this cutting-edge treatment approach to children with cancer.

An easy way to understand precision medicine is to think of it as “personalized medicine.” Although we know that every child is unique, today’s childhood cancer treatment does not take into account the genetic differences of each child. Rather, a child’s cancer is treated according to disease type. But often, children with the same type of cancer respond differently to the same treatment. A chemotherapy which is effective for some may fail altogether for others because of the genetic differences at play. That is where personalized medicine comes in.

Over the past twenty years science has made incredible leaps in discovery by finding what is referred to as genetic barcodes – our DNA and RNA. We now understand what healthy cells look like and can often find triggers or markers in a tumor where something went wrong with a gene. By locating and isolating that problem and finding chemotherapies or other treatments proven effective against the genetic problem, doctors hope to improve survival while also minimizing exposures to toxic treatments which are not likely to work.

“There are really four outcomes when we look at genetic information taken from a child’s tumor,” explains Dr. Douglas Graham, Director of the Aflac Cancer Center. “The first is the perfect storm – we find a target that has a drug which is known to be effective against it and that drug is approved for children. We also may find a target with a matching drug that is not approved for children and would have to petition for access. The other options are not as optimistic. We may find a target with no drug known to work against it or we may find no target at all.”

The first step in the process is getting the child’s genetic information. Since July 2018, CURE has funded the genetic sequencing of more than 200 children with high risk or refractory cancers who would not have otherwise received the sequencing through another source.

 For 78% of the children sequenced, their treatment was impacted by the genetic information obtained!

CURE Childhood Cancer remains determined that precision medicine is one of the most promising methods for improving survival rates in children. And we steadfastly believe that our children deserve the best and safest options available.

A CURE-funded Study Moves to Clinical Trial

Acute myeloid leukemia (AML) is a devastating and aggressive blood cancer that affects nearly 500 children in the United States every year. AML symptoms develop so rapidly that most children feel well just weeks before diagnosis. Despite intensive research over the last 50 years, there are still only a few drugs available to treat this disease, and the survival rate is only 50%. The treatment for AML entails extremely high, toxic doses of chemotherapy. Side effects of chemotherapy include profound impairments to immune function, cardiac toxicity which can lead to heart damage, and kidney and liver dysfunction that can cause permanent, profound disability or death. In fact, current AML drug dosages put children’s organs and immune systems under so much stress that nearly 10% of AML patients die from complications of treatment rather than the disease.

Dr. Alexandra Stevens

While we can temporarily control and improve the symptoms of AML so that it becomes undetectable in the blood, the disease returns with a vengeance in nearly 50% of children. When AML recurs, it is often resistant to the few drugs effective against it, making second remissions and cures particularly challenging. To improve survival of this aggressive cancer, it is critical that scientists identify more effective, better-tolerated drugs with fewer side effects that can be safely incorporated into existing treatment regimens.

While reviewing scientific literature, Dr. Alexandra Stevens of Texas Children’s Hospital read about an antibiotic drug called atovaquone that killed a blood cancer found in adults. In reading how the drug worked, Dr. Stevens believed it would be effective against pediatric AML cells. Dr. Stevens found this drug to be particularly attractive for several reasons:

  • It is already FDA-approved and has known dosing recommendations for pediatric patients;
  • It has no appreciably serious side effects, which makes it ideal to combine with intensive AML therapy; and
  • It is already used to prevent a type of pneumonia, known as PJP pneumonia, that children with AML are at risk of acquiring and treated for prophylactically as a matter of course. This means that incorporating atovaquone into existing AML treatment should be seamless; physicians could simply use atovaquone for PJP prevention so that children could also reap the drug’s potential anti-leukemia benefits in addition to warding off PJP pneumonia.

Dr. Stevens began conducting studies testing atovaquone on pediatric AML cells in the lab, eliciting promising results: this well-tolerated drug performed as well in petri dishes as did the toxic chemotherapy that forms the current basis of pediatric AML therapy.

In response, Dr. Stevens’ team immediately began preclinical studies to confirm that incorporating atovaquone into AML treatment regimens would be safe and effective. In two short years, the team opened a limited-institution trial to identify potential issues with co-administering atovaquone with standard, upfront AML chemotherapy.

The preclinical work was recently published, and the clinical trial has already achieved more than 70% enrollment. Importantly, the next Children’s Oncology Group trial for pediatric AML will collect data on which patients receive atovaquone for PJP prophylaxis and enable researchers to use that data to help determine whether atovaquone reduces the frequency of relapse in a real-world setting.

“Our research team looks forward to continuing their work to learn how best to harness atovaquone’s effects,” said Dr. Stevens. “With the instrumental support of CURE Childhood Cancer, we hope to improve outcomes in patients with pediatric AML.”

 

Why We’re Bullish on Precision Medicine

Could a massive leap forward for cystic fibrosis patients help children with cancer?

In 1989, a research team unlocked a valuable piece of information about a disease that affects an estimated 30,000 people in the United States. After years of study, researchers discovered the gene defect that causes cystic fibrosis (CF). That discovery launched an all-out war against the disease. Patient advocate groups funded research in academic laboratories where incremental discoveries began to unravel the basic biology of the disease.

In a CF patient, there is a malfunctioning protein that doesn’t do its job of balancing salt and water in the lungs. Over time, researchers found ways to correct the error in the protein for the most common problem. In 2019, the Food and Drug Administration approved a three-drug combination that could benefit 90% of patients who suffer from the disease. It is a modern breakthrough of science that began with a single genetic discovery.

“Finding the gene responsible for CF was a ‘needle-in-a-haystack’ problem,” said Francis Collins, the director of the National Institute of Health, and director of the team that found the needle. “But thirty years along, with many bumps along the road and so many people waiting and hoping that something like this would happen – here we are.”

How does this apply to children with cancer?

The model of discovery for this leap forward offers a compelling study into research and drug development for other diseases, including childhood cancer. To understand why, we must explore the differences between cancer in adults and children.

In adults, lifestyle-related risk factors, such as smoking, being overweight, not getting enough exercise, eating an unhealthy diet, and drinking alcohol play a major role in many types of cancer. But lifestyle factors usually take many years to influence cancer risk, and they are not thought to play much of a role in childhood cancers.

Most childhood cancers are the result of DNA changes that happen early in the child’s life, sometimes even before birth. Every time a cell divides into 2 new cells, it must copy its DNA. This process isn’t perfect, and errors sometimes occur, especially when the cells are growing quickly. The causes of DNA changes in most childhood cancers are not known but are likely to be the result of random events that sometimes happen inside a cell, without having an outside cause.

The only way to find the cause of these changes is through genetic testing (also called DNA sequencing). While the primary goal of precision medicine is to bring a therapy to a child that matches the genetic error fueling their cancer, there is also a broader use. By building a large data bank and comparing the genetic errors expressed in children across the world, scientists hope to unlock that “needle-in-a-haystack” for pediatric cancer.

Because there are many types of childhood cancers, there are many gene defects to find. To do so will take steady, ongoing research funded dollar by dollar over a period of time. The road may seem long and grueling, but we believe patience and persistence will yield significant results in the future.

In the meantime, precision medicine is already proving a worthwhile investment as doctors are able to tailor therapies to children on an individual basis. There are children who are alive today because of the findings of genetic testing and precision medicine!

But until all children diagnosed with cancer can benefit from it, there is still work to do.

Previously, the estimated life span of a cystic fibrosis patient was 44 years. For most, the recent discoveries will likely turn what was a deadly disease into a treatable condition.

Children with cancer deserve the same odds and outcomes. We believe precision medicine is the best method to make a similar drastic improvement in the survival rates. To read more about our precision medicine initiative, please click here.

Take the next step to support research that will help save kids with cancer.

Vincristine Shortage Information

LATEST NEWS

As of 10/23, Pfizer has received vincristine and made shipments to hospitals. Pfizer will work under a “controlled distribution” until they reach full recovery. This means that they will hold a buffer so that no location runs out of vincristine and no child will go without their necessary treatment.

The full recovery dates are anticipated as follows:

1 mg dose – January 2020

2 mg dose – December 2019

If you are a patient family and are told you will not get a full dose of vincristine, please contact the FDA immediately at [email protected].

We will post the COG’s webinar here as soon as it is available as well as any appropriate action steps that the childhood cancer community can take to ensure this doesn’t happen again with any drug needed by children.

Please watch this informative webinar hosted by the Coalition Against Childhood Cancer and the Alliance for Childhood Cancer and led by Dr. Peter Adamson, Chair of the Children’s Oncology Group. Dr. Adamson shared information the COG has received along with a perspective on childhood cancer drug shortages, including the current situation with vincristine. The two organizations offered this webinar so that the community could have a better understanding of the shortage, how to work with care providers, how to contact the FDA when problems arise from the shortage, and advocacy steps being taken.

The recent news about a shortage of vincristine is most alarming. This is because vincristine is the chemotherapy drug most widely used to fight pediatric cancers. It has been approved to treat children with cancer for over 50 years and is a critical component of treatment regimens for children with leukemias, lymphoma, brain tumors, bone tumors, neuroblastoma, Wilms tumor, and rhabdomyosarcoma.

Parents of children with cancer are rightfully concerned with the shortage because there is no alternative or substitute for the drug. This means that hospitals and doctors are being forced to ration the drug by lowering dosages for some patients or having them skip administration of it completely.

Drug shortages are not uncommon in the United States. But a shortage of the most commonly used chemotherapy drug for children represents a crisis. Currently, the drug has only one manufacturer in the United States, making almost all pediatric cancer patients completely dependent on their supply. Any disruption of their manufacturing process in the future could create the same situation.

CURE Childhood Cancer is a part of the Coalition Against Childhood Cancer (CAC2), who has created a working group in conjunction with the Alliance for Childhood Cancer. This group is intended to guide action through this crisis, as well as work to ensure it doesn’t reoccur. We feel it is imperative that the childhood cancer community work together as one voice to combat this issue.

Dr. Peter Adamson, Chair of the Children’s Oncology Group has responded to our concerns in this letter.

At this time, the most important call to action is solely for families affected by the shortage: Families immediately impacted should contact the Food and Drug Administration (FDA) at [email protected]. If you are not directly impacted by the shortage, please do not use this email. Use of the email for purposes of complaint or advocacy will only delay action to the parents who need access.

For the longer term, Dr. Adamson writes:

In my view, as related efforts are developed for longer term, economic policy solutions, we need to focus on solutions for today’s children with cancer. In the upcoming days and weeks, I am hopeful we can arrive at focused action items for advocacy solutions that have the goal of guaranteeing cancer drug supplies for children in the United States. Proposals that could be enacted in a reasonable time frame for today’s children that merit consideration include but are not limited to (1) establishment and maintenance of a national stockpile of key cancer drugs used for the treatment of children with cancer and (2) US government purchasing contracts that provide a guaranteed buyer and may help stabilize a fragile market.

CURE Childhood Cancer is committed to keeping you up to date on the progress of the vincristine shortage, and through our partners at CAC2, we will work to ensure all children fighting cancer have an adequate supply of necessary medication. As we learn appropriate advocacy steps, we will share them here and on Facebook and Twitter.

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