Research Archives - CURE Childhood Cancer

Mark Myers In Precision Medicine, Research, Uncategorized

2024 Research Awards

We are excited to share a significant step forward in CURE’s 49-year effort to find cures for childhood cancers as we proudly announce a $5.6 million investment in innovative research studies – the largest amount we’ve ever committed in a single year.

This record-breaking investment represents an important advancement in our ongoing work. It reflects the growing support of our community, the persistent dedication of researchers, and our unwavering commitment to finding better treatments for children with cancer.

These studies are led by top scientists at leading pediatric cancer research institutions nationwide and focus on the most critical needs. Here’s what makes this investment so impactful:

Advancing Precision Medicine: We’re awarding $2 million to the Aflac Precision Medicine Program, reinforcing our leadership in this crucial field. This funding expands access to genetic sequencing for young patients. It accelerates research into targeted therapies, bringing us closer to truly personalized treatments and giving hope to many children with aggressive or recurring cancers.
Funding Diverse, Innovative Research: The remaining $3.6 million funds 11 promising projects carefully selected from over 90 applications. These studies address some of the most challenging childhood cancers with limited or outdated treatments, employing innovative approaches that could significantly improve treatment outcomes.
Bridging Research and Treatment: Time is precious in the fight against childhood cancer. That’s why we’ve prioritized studies with strong potential for rapid clinical translation, potentially bringing new treatments to children faster than ever before.
Harnessing Immune Power: Within every child’s body lies an army of potential cancer-fighting cells. Several projects focus on enhancing cutting-edge immunotherapy approaches, unlocking the full potential of the body’s own defense mechanisms, and offering new possibilities for children battling resistant forms of cancer.
Looking Beyond Remission: The fear of relapse looms heavy for patients and families. By investigating mechanisms of recurrence, these studies aim to improve long-term outcomes and quality of life for survivors – and offer children a chance at a cancer-free future.

As we make these awards, we’re filled with a sense of purpose and hope. While the road ahead may be challenging, each step forward brings us closer to our goal of conquering childhood cancer.

Thank you for being an essential part of this journey.

CURE’s 2024 RESEARCH AWARDS

Early Investigator Awards

Rula Green Gladden, MD, Fred Hutchinson Cancer Center
Redefining residual disease detection in pediatric AML

Elizabeth Young, MD, University of California, San Francisco
Defining determinants of a cGAS-STIGN-mediated anti-tumor inflammatory response in osteosarcoma

Translation to CURE Awards

Eric Sweet-Cordero, MD, University of California, San Francisco
Defining replication stress and DNA damage as a therapeutic vulnerability in osteosarcoma

Pavithra Viswanath, PhD, University of California, San Francisco
Targeting and imaging serine metabolism in the tumor microenvironment in pediatric brain tumors

Michael Andreeff, MD, PhD, University of Texas, M.D. Anderson Cancer Center,
c-MYC protein degradation in therapy-resistant pediatric leukemias

Eugenie Kleinerman, MD, University of Texas, M.D, Anderson Cancer Center
Metabolic reprogramming of the Ewing Sarcoma tumor microenvironment using pramlintide to augment NK cell immunotherapy

Kristopher Bosse, MD, Children’s Hospital of Philadelphia
Development of a GPC2 CAR T cell amplifying RNA vaccine

Alex Huang, MD, PhD, Case Western Reserve University
Effective TGF-beta signaling blockade synergizes cryoablation-induced STING activation in treating refractory and metastatic sarcoma

Jason Yustein, MD, Emory University
Dissecting and targeting PAK4-mediated signaling in Ewing Sarcoma development and metastasis

David Robbins, PhD, Georgetown University
Defining the druggable GLI Interactome in medulloblastoma

Soheil Meshinchi, MD, PhD, Fred Hutchinson Cancer Center
Rapid Transition of B7-H3 Targeted Therapies to High-Risk Childhood AML

Precision Medicine Program, Children’s Healthcare of Atlanta
A program leveraging genomic sequencing for pediatric patients with high-risk tumors, with the goal of identifying alterations that can impact therapies and improve outcomes.

Mark Myers In Childhood Cancer, Children, Precision Medicine, Research

We Finally Have Hope

Easton has been fighting cancer for most of his twelve years. When he was 21 months old, he was constantly sick. His pediatrician noticed that his sickness was abnormal – he vomited more frequently in the mornings and while sitting in his car seat. A scan revealed a large tumor on Easton’s brain stem near the area that controls swallowing and nausea.

“The fact that Easton would get sick after being in his car seat made our pediatrician suspicious,” recalled his mother, Jill. “If the car seat hadn’t put pressure on the tumor, it might have taken longer to find.”

Easton had surgery to remove as much of the tumor as possible, followed by eight weeks of proton radiation in Jacksonville, Florida. His family was thrilled when a follow-up scan showed no presence of the tumor.

“We thought it was over,” said Jill. “We were told that if we had five years of clear scans, we wouldn’t have to worry about it again. Easton had four years of clear scans. But the cancer came back during the fifth year.”

Easton started treatment all over again in 2017 and experienced horrible side effects from the chemotherapy and radiation. Because the tumor grows around the area of the brain that controls swallowing, he has always had challenges eating. The awful mouth sores from his chemo caused him to be on a mostly liquid diet, and he had trouble maintaining weight.

In May 2022, Easton underwent a very risky surgery to try and remove the last part of the tumor. The surgery was unsuccessful. As he started recovering, Easton fell out of bed in the middle of the night. His parents rushed him to the emergency room, where doctors found that Easton’s brain was having a reaction to the glue that was used on his skull after surgery. He would need another risky brain surgery.

“This time, I felt like it was getting away from us and it was the beginning of the end,” said Jill. “He had been on some form of treatment for five years, and the tumor always found its way around it.”

But thanks to funding from CURE Childhood Cancer, Easton’s doctors have a new tool in their toolbox. CURE’s funds would pay for Easton’s tumor to be genetically mapped to see if his cancer involved any genetic mutations that could be targeted.

The genetic mapping revealed that a protein was feeding Easton’s tumor, causing it to grow. Doctors found an open clinical trial using a chemotherapy to inhibit this specific protein to prevent further tumor growth. Easton was immediately enrolled in the trial, and the results have been astounding. After four months, a scan showed that the inside of Easton’s tumor appeared to be dying. Three months later, the tumor is much smaller and is collapsing in on itself.

“The best news is that the tumor is dying. But also, he has no side effects. He can do the things a twelve-year-old should do while on this treatment,” shared Jill. “We’re still early in the process, but it has saved his life – at least at this point. I finally have hope for the first time in years. We are so encouraged and thankful to his doctors and to CURE for investing in precision medicine.”

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

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Mark Myers In Research

2023 Research Grants

CURE CHILDHOOD CANCER PROUDLY ANNOUNCES $5 MILLION IN RESEARCH GRANTS

Research has led to significant improvements in survival rates for many childhood cancers over the past few decades. For example, the five-year survival rate for all childhood cancers combined has increased from less than 60% in the 1970s to more than 80% today. However, there is still much work to be done, and continued research is crucial for further progress.

To that end, CURE is proud to announce that we are directing $5 million, the most CURE has ever funded in a single year, to research aimed at solving childhood cancers that remain so hard to cure!

In this year’s grant cycle, we were extremely pleased to receive more than 70 grant applications. Following a rigorous grant review process with a committee of researchers and scientists, CURE is funding 12 targeted projects at leading institutions across the country. 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.

This is the most significant grant cycle we’ve ever had for several reasons. For example:

MORE EFFECTIVE – All of the research studies we are funding aim to make more effective therapies that are better tolerated, with a special emphasis on relapsed and refractory disease.
LESS TOXICITY – Five of the research projects we are funding are specifically aimed at harnessing the immune system’s power to fight cancer instead of using today’s toxic chemotherapies.
FASTER DELIVERY – We are addressing both near-term work that is anticipated to enter clinical trials in the next 2-3 years and funding work that contributes to long-term cancer biology understanding, which will impact research for decades.
BREAKING BARRIERS – Crossing the blood-brain barrier to deliver chemotherapy drugs directly to a brain tumor is challenging. Two studies we are funding focus on better delivery of drugs to address this issue.
PINPOINTING PROBLEMS – We continue to prioritize precision medicine. With our funding, researchers are getting closer to knowing which drugs are most likely to be effective against some of the most difficult childhood cancers.

Because bright young physicians often leave pediatric research for more lucrative pursuits, we expanded our research funding to include three early investigator awards to get these scientists started.

Another way we work to support young doctors and ensure they are trained to care for children with cancer and advance research is to fund the fellowship training of three pediatric oncology fellows at Emory University’s School of Medicine: Dr. Toni Chanroo, Dr. Robert Lisac (Sam Robb Fellow), and Dr. Jason Stevenson (Connolly Family Fellow).

Our 2023 Research Initiative includes the following studies:

Early Investigator Awards

Kyle L. MacQuarrie, MD, PhD, Lurie Children’s Hospital of Chicago
Biological consequences of altered chromosomal organization in rhabdomyosarcoma cells

Shubin Shahab, MD, Emory University
PBK and let-7 in Group 3 medulloblastoma treatment resistance

Kristen Van Heyst, DO, University Hospitals Rainbow Babies & Children’s Hospital
Tumor microenvironment modulation as an effective therapy for osteosarcoma

Translation to CURE Awards

Precision Medicine Program, Children’s Healthcare of Atlanta

Yana Pikman, MD, Dana Farber Cancer Institute
Targeting RAS pathway mutations for pediatric acute leukemia therapy

Pietro Genovese, PhD, Dana Farber Cancer Institute
Empowering pediatric immunotherapies by HSC engineering

Kelly Goldsmith, MD, Emory University
Second generation gamma delta T-cell therapy for neuroblastoma and osteosarcoma

Praveen B Raju, MD, PhD, Icahn School of Medicine at Mount Sinai
Nanotherapeutic targeting of PPM1D inhibitors across the blood-brain barrier for DIPG

Agnieszka Czechowicz, MD, PhD, Stanford University
Development of receptor tyrosine kinase-targeting chimeric antigen receptor T-cells as dual hematopoietic stem cell transplantation conditioning and immunotherapeutic agents for cure of pediatric acute myeloid leukemia

Elizabeth Lawlor, MD, PhD, Seattle Children’s Hospital
Augmenting the efficacy of BET inhibitors for metastatic Ewing sarcoma

Erwin Van Meir, PhD, University of Alabama at Birmingham
Small molecule targeting of epigenetic reader MBD2 for medulloblastoma therapy

Beau Webber , PhD, University of Minnesota
Genetically engineered gamma delta T-cells for treatment of metastatic osteosarcoma

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

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Mark Myers In Research

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

Mark Myers In Childhood Cancer, Research

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.

DOUBLE YOUR IMPACT

Mark Myers In Research

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.

Mark Myers In Research

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.

Mark Myers In Research

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

Mark Myers In Childhood Cancer, Children, Research

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.”

Mark Myers In Research

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.

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