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.

Vincristine Shortage Information


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.

2019-2020 Research Grants


At CURE, we believe that investment in the highest quality research is the key to achieving significant progress in the fight against childhood cancer. Our focus is two-fold: we prioritize research that will reach the bedside within five years and research which will improve the outcomes for the 20% of children not surviving today’s methods of treatment. While we continue our important work to increase survival rates, it is also imperative we increase funding into safer treatments which will not compromise the rest of a child’s life. To that end, we are proud to announce $4.3 million in research grants for CURE’s fiscal year 2019–2020.

Our funding decisions are guided by our Scientific Advisory Council, a group of doctors, scientists, and researchers who conduct a thorough review of research proposals to assess both scientific merit and alignment with CURE’s goals. This year, we selected to fund 21 of the very best proposals submitted by experts at leading institutions across the country. Precision medicine continues to be a strategic focus as we enter the third year of our $4.5 million commitment which created the Aflac Cancer Center Precision Medicine Program at Children’s Healthcare of Atlanta. This unique program has already yielded significant results – giving hope to children for whom standard treatment has failed. Finally, in an effort to ensure the best and brightest minds continue to research cures for children, we will fully fund training for three pediatric oncology fellows and are providing funding to two young investigators for their research.

“We have developed an incredible partnership with CURE in the fight against childhood cancer,” said Dr. Douglas Graham, Chief of the Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta. “We are so thankful for the generous gift from CURE this year to allow us to provide direct assistance to our families, fund new research projects to search for better cancer treatments, and help us train the next generation of physicians specializing in cancer care for children. The gift from CURE will also enable us to continue to develop and expand our Cancer Precision Medicine Program to enable us to provide optimal care to our most difficult to treat cancer patients. This ongoing partnership with CURE is critical to our mission to provide world class cancer care to our pediatric cancer patients.”

Our 2019-2020 Pediatric Cancer Research Initiative includes the following studies:

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

Douglas Graham, MD, PhD
Aflac Cancer Center Precision Medicine Program

Robert Castellino, MD
PPM1D in High-Risk, Non-SHH, Non-WNT Medulloblastoma

Zhihong Chen, PhD
Leveraging MMR Deficiency for Effective Immunotherapy in Childhood High-Grade Glioma

Deborah DeRyckere, PhD
TAM Kinases as Mediators of Chemoresistance in AML

Lubing Gu, MD
Dual Inhibition of MDM2 and XIAP for Treatment of Childhood Cancers

Lisa Ingerski, PhD
HRQOL Outcomes During Molecularly Targeted Therapy for Brain Tumors

Christopher Porter, MD
Targeting Siglec15 for the Treatment of Childhood Cancers

Erwin Van Meir, PhD
Negative Regulation of b1-Integrin Signaling by ADGRB1 in Medulloblastoma

Muxiang Zhou, MD
MYCN Inhibition as a Precision Therapy in MYCN-amplified Neuroblastoma

Augusta University Research Institute

Daquing Wu, PhD
Novel Targeted Therapy for Metastatic Osteosarcoma

Baylor College of Medicine

Alexandra Stevens, MD
A Trial of Atovaquone with Conventional Chemotherapy for Pediatric AML (ATACC AML)

Lisa L. Wang, MD
Optimizing GD2.CAR T Cell Immunotherapeutic Strategies for Relapsed Osteosarcoma and Neuroblastoma

Joanna Yi, MD
Defining the Transcriptional Control of Pediatric AML to Find New Drugs

Children’s Hospital of Philadelphia

Frank M. Balis, MD
GD2 as a Circulating Biomarker and Clinical Trial Endpoint for Neuroblastoma

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

Timothy Olson, MD, PhD
Bone Marrow Niche Targets to Prevent Cancer in Shwachman Diamond Syndrome

LLS PedAL Initiative, LLC

Soheil Meshinchi, MD, PhD
PedAL Initiative

Lurie Children’s Hospital of Chicago

Oren Becher, MD
Unraveling Mechanisms of Resistance to ACVR1 Inhibitors to Treat DIPG

Memorial Sloan Kettering Cancer Center

Estibaliz Lopez Rodrigo, MD
Lung Macrophage Synergy with L-MTP-PE to Target Metastatic Osteosarcoma.

Stanford University

Maria-Grazia Roncarolo, MD
Innovative Cell Therapy for Pediatric AML

Kathleen Sakamoto, MD, PhD
Niclosamide for Relapsed/Refractory Pediatric AML

Beyond Survivorship

Many people have come to know September is National Childhood Cancer Awareness Month. But fewer realize June is National Cancer Survivors Month. We should definitely celebrate survivors! After all, the journey to survivorship is arduous, and these brave children are heroes. At CURE, we love to see cancer’s youngest warriors ring the bell at the end of treatment and walk out of the hospital, shoulders back and heads held high, ready to conquer the world.

However, life after treatment for these young survivors can be tough, and most people don’t realize the price they pay to achieve survivorship. By price, we don’t mean the financial cost, but the toll of months – sometimes years – of treatments involving toxic drugs which cause damage to organs and healthy cells at the same time they kill cancer cells.

When CURE was founded in 1975, the survival rates for children diagnosed with cancer were very low. There were few survivors. With focused and collaborative research, those rates have steadily risen to above 80%. However, as is usually the case, this statistic doesn’t tell the whole story. There’s another important statistic to note: more than 95% of childhood cancer survivors will have  significant health issues by the time they are 45 years old. For those who are poor at math, 95% is only a little shy of all of them.

From heart and lung damage to secondary cancers to serious cognitive deficiencies, childhood cancer survivors enter adulthood far differently than their peers. Some of their challenges are obvious – like managing with prosthetic limbs, wheelchairs, or canine assistants. Others, less so – such as cognitive and emotional challenges. For some, survivorship can mean  moving from the confines of treatment to a different sort of cage. It’s our responsibility, as parents, friends, and adults, to ensure we do everything we can to free these survivors from this cage.

While we continue our important work to increase survival rates, it is imperative we change our thinking and increase funding into treatments which will not compromise the rest of a child’s life. CURE’s investments in precision medicine and immunotherapies hold much promise as safer cures.

Precision medicine seeks to treat the child based on his or her own genetic makeup. By analyzing the child’s DNA, doctors can match treatment to the child more precisely and avoid toxic chemotherapies that are not needed or aren’t likely to work. Fighting cancer more precisely can lead to fewer short and long-term effects. Likewise, immunotherapies work by programming one’s own immune system rather than using toxic drugs and compounds to destroy cancer cells.

These treatments offer hope for safer cures that will one day allow every child the opportunity to reach for their dreams without a cancer diagnosis standing in their way.

To learn more about CURE’s investment in groundbreaking research and see how you can join us, please click here.

Maylee and Precision Medicine

Maylee has always been kind, intelligent, and slightly feisty. Since her birth in Lagrange, Georgia, she has also been a picture of health. So when she got a violent stomach virus at the age of three, her mother, Kristin, knew it was more than just a bug.

“I never expected she would go through life without being sick,” Kristin said. “But I knew right away this was different and something much worse.”

Mom was right. As the emergency room staff began giving Maylee fluids, they also tested to see if she had meningitis. Those tests found something much more sinister; Maylee’s blood had leukemia cells in it. After confirming the diagnosis, her doctor sent her by ambulance to Children’s Healthcare of Atlanta where she would spend the next six weeks.

“I had such a wide range of emotions when the doctor said my baby had cancer,” Kristin recalled. “I was confused, overwhelmed, and scared. But I also had a strange peace because I felt the Lord say, ‘I created this child and I will take care of her.’”

Maylee’s chemotherapy treatments were very aggressive. Every child responds differently to treatment, and her little body had difficulty rebounding, which forced longer hospital stays. She also had a very unusual emotional response.

“Chemo made her angry,” Kristin recalled. “She understood what was going on – that the chemo led to her feeling bad – and she took it out on the doctors. So she wouldn’t tell us when she felt bad or hurt because that would mean more doctors in the room.”

Fortunately, Maylee reached remission soon and found a routine in the hospital, although her feistiness never let her totally forgive the doctors. After seven months, she finished treatment and was declared cancer-free.

Maylee and her family began to settle into a new routine of normalcy. She welcomed her second brother into the world and began school. But during the summer of 2018, she began to snore. It might seem innocuous, but Kristin’s mother’s intuition struck again because she had been told sleep apnea can be a sign of relapse. As they rode to the hospital for doctors to evaluate Maylee, Kristin began steeling herself for another fight against cancer, feeling the familiar mix of fear, faith, and comfort.

Something new awaited Maylee at the hospital, though. Since her original diagnosis and with a $4.5 million grant from CURE, the Aflac Precision Medicine Program had launched. This program allows doctors to look at the genetic makeup of a child’s cancer and then search for a treatment designed specifically to treat the genetic abnormality at issue. Once doctors confirmed her cancer had returned, they looked at the genetics of Maylee’s leukemia and found very interesting results.

The genetic testing revealed that Maylee has a gene called CEBPA, which causes her to be predisposed to developing leukemia. Strangely enough, doctors were happy to find this gene because they know it responds well to treatment. If genetic testing had been available when Maylee was originally diagnosed and this gene discovered, Maylee’s treatment would have been different. Rather than the months of chemotherapy, Maylee would have immediately received a bone marrow transplant. Thankfully CURE’s funding allowed the program to launch in 2017, in time to help Maylee when her cancer returned not to mention so many other children yet to be diagnosed.

Maylee has since had what is called an allogenic stem cell transplant and is recovering nicely. She is once again in remission and focusing on getting well.

The discoveries from her genetic testing didn’t end there, however. Doctors also found that her leukemia is familial and that her two brothers share the gene that predisposed Maylee to it. Maylee’s brothers were immediately enrolled in the new genetic predisposition clinic.

“We had many different options available to us when they found out,” explained Kristin. “We chose to monitor the boys and watch for any symptoms. There is no guarantee that they will ever have leukemia. We just need to look for things out of the norm, and since they are enrolled in the predisposition clinic, we can have bloodwork done immediately.”

Maylee has completed treatment and is home growing stronger each day. She ultimately forgave her doctors but was thrilled to leave them behind. While she gets better, Maylee dreams of a return to normal life with no more hospitals, cancer, or chemo. And as each day passes, she inches closer to lacing up her cleats and playing soccer without a care in the world besides the goalie in her way.

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

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. Maylee’s story shows how important this program has become in only its second year.

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. “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, more than 40 relapsed or high-risk patients have been enrolled in the Aflac Cancer Center’s Precision Medicine Protocol.

At CURE’s Board of Directors meeting in October 2018, Dr. Daniel Wechsler, Director of Pediatric Oncology at the Aflac Cancer Center, shared, “We have received sequencing results back so far on 36 patients. Several patients have had ambiguous diagnoses confirmed as a result of (genetic) sequencing, which has helped us choose appropriate therapeutic protocols. A couple of patient tumors have shown mutations that have directed us to specific clinical trials for targeted agents.”

Fully funded by a three-year grant from CURE Childhood Cancer, the APMP is well ahead of schedule. In this second year, thirty patients were expected to be enrolled in the program.  However, Dr. Graham and Dr. Wechsler report they have enrolled many more than that. The groundwork has now been laid for more and more children to benefit from this new area of science.

While information is the key to the program, it is also the most significant limiting factor. The sheer complexity of the human genome is overwhelming, and not many are people trained to interpret genetic data. Biological informatics is a growing field of study due to this demand, and the APMP is working with the best resources available.

Also, there are currently only a handful of pediatric cancer centers across the country which precision medicine programs and the volume of information being recorded by each is significant. Finding ways to share information is a challenge to progress. This is changing, however. Doctors from the APMP are working with Memorial Sloan Kettering in New York, Cincinnati Children’s Hospital, and other leading centers to develop collaborations and efficient ways to share data.


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.

Acquiring Targets in the Fight Against AML

A childhood cancer diagnosis can turn complete strangers into intimate friends very quickly. Because each type of cancer is unique, parents tend to fragment by diagnosis to share knowledge, side-effect remedies, and treatment options. Three Atlanta families met that way; while their children were all fighting Acute Myeloid Leukemia (AML). Those beautiful children – Lake, Mary Elizabeth, and Melissa – fought bravely but ultimately succumbed to the disease. The bonds built by their families during treatment didn’t break after their deaths. They strengthened.

Together they formed a Named Fund at CURE called United for a CURE. They decided to work together in honor of their children with a vow to never stop fighting until there is a cure for children battling AML. Since then, they’ve worked tirelessly, and thanks to the support of friends, family, and colleagues they have raised over $350,000 in just two years. With that sum, they chose to support a project called Target Pediatric AML.

“We chose Target Pediatric AML because leading researchers agree that this project has the highest potential for a cure in the next five years,” said Joe Depa, Melissa’s father. “All children fighting AML have their tumor samples banked, and when we spoke with the project’s lead researcher, Dr. Soheil Meshinchi, he become emotional as he connected the samples he was studying with our families and our children. We could feel his passion immediately.”

Target Pediatric AML aims to facilitate genomic sequencing for every child fighting AML, searching for unique identifiers and vulnerabilities of the disease in young patients. Once these factors are identified, the hope is that the information will allow existing agents to be delivered in the right combination to effectively treat children fighting the disease. This knowledge will enable expansion of the treatment toolkit and jumpstart more informed, individualized therapy.

The project has already had some key wins:

  • The project has created the world’s largest database of genetic data for pediatric cancer patients. The team has provided genetic sequencing (mRNA) for more than 1200 AML patients.
  • A new therapeutic target has been identified and funding has been secured for clinical trial. Mesothelin was identified as a therapeutic target in AML. Typically found in lung cancer patients, genetic profiling revealed this protein in AML patients, as well. Bayer Pharmaceuticals has an anti-mesothelin drug for lung cancer and has agreed to participate in a clinical trial in 2018 to test its effectiveness against pediatric AML.
  • Additional targets are under development. Based on the RNA sequencing, over 100 potential targets have been identified and are undergoing verification for therapeutic development.
  • The information and database is shared publicly. All health institutions, pharmaceutical companies, and researchers can access and leverage the genetic data acquired with the hopes that collaboration will lead to provide more targeted therapies.

The underlying fact is that all children are unique and their cancers are also very different. If we can find common targets in the genomic make-up of AML, we might be able to fight these targets with existing therapies or therapy combinations.

“There are many different subtypes of AML,” explained Joe. “In fact, each of our three children had a completely different type. And yet, today’s treatments are basically the same for every subtype. This project is searching for individual therapies for every child to improve their survival rate. It’s too late for Lake, Mary Elizabeth, and Melissa. But we hope someday their names are attached to a cure for AML so that other families win their fight. We owe them that much.”

To learn more about the United for a CURE Fund, please click here:

CURE Childhood Cancer Announces over $4 million in Research Awards

CURE Childhood Cancer is increasing its impact into research that will develop effective treatments for the 20% of children not surviving current methods. In the past ten years alone, CURE has raised more than $35 million to fund cutting-edge research and provide critical support to families dealing with a cancer diagnosis. Our unwavering commitment is to find a cure for childhood cancer in our lifetime.

To that end, CURE announces funding in excess of $4 million for our fiscal year 2017-2018. This amount includes our largest grant ever – a $1.5 million award for the development of the Aflac Cancer Center Precision Medicine Program. The program, which will be led by Douglas K. Graham, M.D., Ph.D., director of the Aflac Cancer Center and professor of pediatrics at Emory University School of Medicine, envisions personalized, non-toxic and curative cancer therapy for all children.

“We are so grateful to CURE for this generous gift and their continued support of our patients and researchers as we work to develop new treatments for childhood cancer,” says Dr. Graham. “Through the systematic implementation of integrated, comprehensive tumor profiling and the development of novel strategies to identify individual tumors’ vulnerabilities, the Precision Medicine Program will provide state-of-the-art care for children with the highest risk tumors. Our hope is that we will be able to share these treatments with centers around the country as our new approaches are adopted elsewhere.”

“We are very excited to grow our long-standing relationship with the Aflac Cancer Center by fully funding the new Precision Medicine Program,” says Kristin Connor, CURE Childhood Cancer’s Executive Director. “CURE’s mission is to drive innovative childhood cancer research that will move the needle closer to therapies with fewer side effects for children with cancer and, eventually, cures. We believe bringing Precision Medicine capabilities to Atlanta is a very important step in advancing our mission.”

In addition to the Precision Medicine Program, CURE is awarding another $1.26 million to projects at the AFLAC Cancer Center and $1.3 million at centers of excellence around the country.

CURE’s full awards are as follows:

AFLAC Cancer & Blood Disorders, Emory University

Graham, Douglas K. MD, PhD, Aflac Cancer Center Precision Medicine Program

Spencer, H. Trent  Ph.D, Manufacturing of a GMP compliant T-cell product to treat high risk neuroblastoma

Porter, Christopher C. MD, Targeting Siglee15 for the treatment of childhood leukemia

MacDonald, Tobey J. MD, Combined CSF-1R and STAT3 Inhibition as a Novel Immunotherapeutic Strategy for Medulloblastoma

Gu, Lubing MD, MDM4-TOP2A interaction as potential target for treatment of pediatric cancers (Renewal of 2016-2017 CURE funded project)

Kenney, Anna PhD/Dey, Targeting YB1 to prevent post-radiation medulloblastoma recurrence

Zhou, Muxiang M.D., Targeting MYCN mRNA for treatment of MYCN-amplified neuroblastoma of children

Van Meir, Erwin, G PhD, Investigating the tumor suppressor function of BAI3 in WNT medulloblastoma

Graham, Doug and Deborah DeRyckere, MERTK Inhibitor Combination Therapy for Treatment of AML

Thomas Cash, MD, MSc, A Phase I Study of 131I-MIBG with Dinutuximab for Relapsed/Refractory

Himalee Sabnis, MD, MSc, ENCERT: A Phase 1 Trial using Everolimus in combination with Nelarabine, Cyclophosphamide and Etoposide in Relapsed T cell Lymphoblastic Leukemia/Lymphoma

Children’s Oncology Group & Fred Hutchinson Cancer Research Center

Meshinchi, Soheil MD, PhD, Target Pediatric AML

Children’s Cancer Therapy Development Institute

Keller, Charles MD, Prediction & Validation of a Novel Drug Combination Against Anaplastic Wilms’ tumor

Dana-Farber Cancer Institute

Steven DuBois, MD MS, Phase 1 Trial of Dual PI3K/BRD4 Inhibitor for Children with Neuroblastoma

Seattle Children’s Hospital

Leslie Kean, MD, A First-in-Disease Phase II Trial of T cell Costimulation Blockade for GVHD Prevention

The Children’s Hospital of Philadelphia

Felix, Carolyn MD, Mechanism-Based Prevention of TOP2-Poison Related Leukemia

The University of Utah

Jones, Kevin B. Epigenetic Drivers of Clear Cell Sarcoma

An Effective Two-Part Treatment Against Deadly Cancer

Dr. Thomas Cash has never forgotten the precocious two year-old cancer patient and his family. He had gotten close to them while treating the toddler’s cancer.

But he couldn’t save the little boy. “I realized I had two ways to go: I could stay really sad,” he said. “Or this could drive me forward in finding better treatments – so that other children and their parents would never have to go through this.”

Dr. Cash, a pediatric hematologist and oncologist at the Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta, now focuses on neuroblastoma, one of the most common cancers seen in children. It’s also one of the most deadly if a cancer patient doesn’t respond to treatment or the cancer returns after initial rounds of therapy.

“Relapsed/refractory neuroblastoma is incurable in the majority of cases,” explains Dr. Cash. “This is why there is such an urgent need for novel therapeutic approaches.”

Statistically, neuroblastoma comprises eight percent of all childhood cancer cases, and it is responsible for 12 percent of cancer deaths in children under 15 years of age. In addition, one in two neuroblastoma patients are classified as high-risk, and half of these children will die from their disease despite intensive treatments.

To combat those statistics, Dr. Cash is developing a new attack plan – taking two effective fighters against neuroblastoma and combining them into one powerful treatment.

One is a compound called Metaiodobenzylguanidine (MIBG), which can be combined with a radioactive iodine that delivers targeted radiation to the cancer cells. The other is Dinutuximab, which is an antibody that binds to GD2, a tumor surface marker which is prevalent in neuroblastoma. Dr. Cash believes if they are combined as a “one-two punch,” it will be a powerful new tool.

A key reason is that MIBG is already known to be effective in “sneak attacks” against cancer. MIBG, which mimics a natural hormone, can be absorbed by certain tissues, including some tumors. But then,radiation can then attack the tumors in a classic bait-and-switch.

Dr. Cash believes that if MIBG is paired with Dinutuximab, the approach will be even stronger. “We believe that both working together could be a very effective combined force. That’s because once the MIBG is in the patient’s body, we think it revs up the patient’s own immune system, and then like pouring gas on a fire, when we add the Dinutuximab, the patient’s own immune system will attack the tumors and kill them.”

Several factors will have to be examined in the study, including the toxicity of this new therapy (previous studies have proven it is safe to combine MIBG with other chemotherapies), and the right dosages of these two therapies when they are given together.

The Phase 1 study, funded by CURE, will enroll mostly children, but patients up to 30 years old can enroll. It will involve between six and eight weeks of the novel treatment.

“I think the most exciting thing for families who are dealing with this is this study takes two treatments that we know are effective in treating neuroblastoma and combines them together,” Dr. Cash said. “And we here at the Aflac Cancer Center are the only ones doing it.”

And most importantly, the study allows Dr. Cash to continue to honor a family and a little boy who meant so much to him by exploring new options which could keep the littlest of patients alive.

“A cure for most neuroblastoma patients whose cancer has relapsed is not currently an achievable goal,” he said. “But I think if we can turn the cancer into a chronic disease and give these kids many more good years, then that’s also a big win – not just for us, but mainly our patients and their families.”

Staggering yet Stagnant Innovation

What are the greatest innovations of the last forty years? Going back to our bicentennial – 1976, can you come up with the top scientific, technological, and medical discoveries that have impacted society? I had the opportunity to speak to a middle school class in Decatur, GA last week and posed that very question. Although they’ve only been alive for one decade, hands shot up all over the classroom and I began to field their responses:

“The Internet!”

Correct! There was no internet or social media. We talked to each other.

“Cell phones!”

I mentioned rotary phones but lacked the Pictionary skills to make them understand rotary dialing.

“Mobile devices!”

Another good answer. I explained the huge shoulder boom boxes but since they can store a thousand songs on a device the size of a quarter, it was lost on them.

“Color Television!”

No, we had color tv’s but only three channels with no cable or satellite.

Mouths dropped.

“Space travel!”

Well, a man walked on the moon in 1969 but you are right, there has been a great deal of space innovation since. We’ve even sent a rover to Mars.

“The Gyro Rocketter!”

I suppose so. I’m sorry, I don’t know what that is.

“You couldn’t know, I only invented it just now.”

A shy hand went halfway up nearby. With the others bemoaning how hard life must have been in the paleo-internet age, I called on the young man who tentatively asked:

“I don’t mean to offend you, but did you fear from dinosaurs back then?”

Thus ended the question and answer portion of the presentation.

When you think about it, the innovation of the last forty years has been staggering. Yet in some areas, it remains unbelievably stagnant. While we have made huge leaps of progress in the fight against many forms of cancer, there are still some childhood cancers that have eluded treatment advances altogether. I discovered this fact when my daughter was diagnosed with Ewing’s Sarcoma at the age of twelve. We were given a treatment protocol which I was told would have been nearly identical to what I would have been given had I gotten the same cancer at twelve. I am not quite as old as dinosaurs, but I was twelve in the 70’s and find it unbelievable that during this age of discovery, children are being left behind.

There are various factors contributing to this sad fact. Although incidence rates are increasing, childhood cancers are still classified as rare compared to adult cancers such as lung, breast, and prostate. Funding from the government and large cancer charities lean heavily toward the cancers that affect more people without taking age into consideration. Yet our children are counting on us to do better. Certainly there are good doctors and researchers working hard to uncover safer and more effective treatments, but there is still a long way to go. That is why CURE is so vitally important. We focus our funding on projects aimed at improving outcomes for the 20% of children not surviving today’s methods of treatment. In addition, we fund projects for developing therapies that will preserve a child’s quality of life after treatment.

Click here to see a list of the research projects we are currently funding and join our search for a CURE

CURE Welcomes Dr. Douglas Graham as the new Director of the Aflac Cancer and Blood Disorders Center at Children’s Healthcare of Atlanta

We are very excited to welcome Dr. Douglas Graham to the Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta, and introduce him to the CURE community. We enjoy a strong partnership with the Aflac Cancer Center, providing funding for promising research conducted by its researchers, annual funding of two fellows (soon to be three!), and serving patients and families – often within its four walls – cared for its the skilled doctors and nurses. We are proud of and grateful for this partnership. In the letter below, Dr. Graham shares insight and his vision for the Aflac Cancer Center. We look forward to continue to work in partnership for the good of childhood cancer patients and their families and all who care for them.

Screen Shot 2016-02-10 at 11.34.31 AMAs the recently appointed Director of the Aflac Cancer and Blood Disorders Center at Children’s Healthcare of Atlanta, it is a privilege to become a part of the CURE Childhood Cancer family. Thank you for your remarkable support which has enabled the Aflac Cancer Center to become one of the premier centers in the country for children to receive every level of cancer care.  Your financial support has been critical to our research to develop better cancer treatments, our fellowship program to train the next generation of pediatric cancer doctors and for our clinical care, which includes family emergency funds.

Kristin Connor, CURE’s Executive Director, graciously provided me with this opportunity to give you a little more insight into care and research offered by the Aflac Cancer Center, and a few exciting programs that lie ahead.

The Aflac Cancer Center has made enormous strides with support such as CURE’s, and our advances have been recognized nationally. Last year we were ranked by US News and World Report as the number one program in the state of Georgia to treat pediatric cancer patients and the number nine program in the entire country!  We now have 80 pediatric specialists who are hematology/oncology/BMT faculty, and we are one of the five largest pediatric oncology programs in the nation. This is significant because we encounter every type of childhood cancer, and we are able to provide the most sophisticated care for the sickest of children with cancer, while simultaneously being a top-rated research center.  We are a major referral center for the country, having cared for children from 43 states.

We participate nationally in the Children’s Oncology Group (COG), which is a collaborative group of cancer doctors across the country who take care of more than 90% of all children with cancer in the US. Within COG, we have been selected as one of only 21 sites (out of 223 COG member institutions) to be given access to the newest of cancer drugs in early phase clinical trials through the COG Phase I Consortium. Our goal is to continue to offer new therapies through COG as well as to move new treatment ideas into the clinic developed by our cancer physicians and researchers.  Some new research discoveries both in gene therapy and in new cancer drug development made by our faculty are the basis for new clinical trials which are planned to be launched in the coming year in brain tumors and in leukemia.

Two examples of innovative therapies we have initiated at the Aflac Cancer Center are our MIBG program for neuroblastoma and our CAR-T immunotherapy program for relapsed childhood leukemia. We are one of the few places in the country to offer MIBG therapy, and the COG is including the Aflac Cancer Center as one of the treatment sites for an upcoming MIBG clinical trial.  A new MIBG treatment room is being planned to accommodate this anticipated increase in patient referrals.

The CAR-T therapy for relapsed leukemia involves harnessing one’s own immune system to combat cancer.  In this type of therapy, immune cells (T cells) are taken from the leukemia patient and modified in cell culture to be able to seek out and kill leukemia cells. The Aflac Cancer Center is one of only a handful of centers in the country to be selected as a treatment site. This therapy is successful in offering hope to children with resistant leukemia who have no other treatment options resulting in cures not previously possible.

Thank you, again, for your remarkable support of cutting edge research to cure more kids, and for your dedication to provide family centered care for kids with cancer. I look forward to continuing our strong partnership so that we can provide the best care possible for children with cancer, both today and tomorrow.