Childhood Cancer

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December 17, 2018

by Trish Adkins

For Eden, the trouble began when she was 10 years old. The trouble had a name: pain. It seemed normal at first—maybe a side effect from dancing or growing. But then it never went away and then, suddenly, Eden could not dance anymore.

“I knew in my heart it was more than growing pains,” said Eden’s mom, Shannon. 



A MRI revealed the source of the pain: a tumor on her leg. And not just any tumor, it was a tumor so rare that it had never been seen before. They nicknamed it Eden’s Tumor. Eden’s doctor, Dr. Jennifer Foster at Texas Children’s Cancer Center turned to research to find a treatment plan for Eden. Eden went through treatment once and then her cancer returned, this time in her shoulder. 

Eden is now enrolled in a clinical trial for children with relapsed tumors. We spoke with Dr. Foster about Eden’s tumor, her treatment and how innovative clinical trials are propelling the world closer to cures. 

ALSF: What type of cancer does Eden have?
Dr. Jennifer Foster (JF): Eden has a tumor that has not been described before. Our team ended up calling it a “primitive round blue cell primitive neural tumor.” But, really, we just refer to it as Eden’s Tumor, since it is the only one of its kind anyone has ever described. 

ALSF: Wow, walk me through what happens when a tumor like Eden’s lands on your desk?
JF: Until we can describe the tumor, we cannot treat it. This is true for both rare and less-rare tumors. We tapped into our large team of pathologists, solid tumor oncologists, radiologists and other childhood cancer experts across the country to come up with a description of the tumor. 

ALSF: What are the features that make Eden’s tumor so rare?
JF: Eden’s tumor has several features that make it so unusual. First, the tumor did not pick up on an MIBG or bone scan, which are scans used to detect neuroblastoma cells. Yet, the pathologists could tell the tumor had some characteristics of neuroblastoma. The PET scan did pick up the tumor cells in Eden’s leg, shoulder and throughout her body. 

Pathologists looked at samples of Eden’s tumor under the microscope and not only saw the neuroblastoma features, but also saw some features of the common bone cancer Ewing’s sarcoma. When we sequenced the tumor to study its biology, there were no genetic hits against a wide panel of known markers of cancer. 

ALSF: So, how do you treat something that isn’t anything that has existed before?
JF: We heavily relied on the pathology report to create a treatment plan that we knew to be effective against both neuroblastoma and Ewing sarcoma. We chose chemotherapy drugs known to work on both cancers as well as radiation, which we know kills both of these types of cancer cells. The treatment was effective and the tumors responded. Unfortunately, Eden relapsed 1 year off of therapy, this time in her opposite shoulder. 

ALSF: What happens when a child like Eden relapses?
JF: When any child relapses, we know that the standard of care did not work and we have to try something new. Often, tumors are harder to treat after relapse.  So, we turn to clinical trials and research to find potential solutions. For Eden, the biopsy of her relapsed disease showed that the tumor looked exactly the same under the microscope as it did at diagnosis. Since the first round of treatment did not work,  we had to come up with a new plan. For Eden, that plan was a new clinical trial opening both at Texas Children’s Cancer Center and at other select institutions across the country.

ALSF: What is the name of the trial Eden is on?
JF: The official title of the study Eden is a part of is “ADVL1615, A Phase 1 Study of Pevonedistat a NEDD8 Activating Enzyme (NAE) Inhibitor, in Combination with Temozolomide and Irinotecan in Pediatric Patients with Recurrent or Refractory Solid Tumors”

ALSF: How does the drug work?
JF: Pevonedistat is a novel agent that we use in combination with two standard chemotherapy regiments (Temozolomide and Irinotecan). Pevonedistat may work in combination with Irinotecan and temozolomide by blocking some of the enzymes needed for cell growth, thus stopping the growth of cancer cells. 

The pre-clinical lab testing of this combination has shown great promise and this drug has also showed success in clinical trials for adults with cancer. As long as Eden is responding to treatment, she will have up to 17 cycles of therapy over about a year. 

ALSF: Why are childhood cancer research and clinical trials so important?
JF: The trial Eden is on represents 10 years of process—pre-clinical experiments in the lab, adult clinical trials, and the building of the infrastructure necessary to support a clinical trial. We don’t have the full story for most types of childhood cancer. Continued research and clinical trials will help us learn the full story, so we can start making the drugs and agents that can cure future children diagnosed with cancer. 

ALSF: How can studying a rare, one-of-a-kind tumor help other kids battling cancer?
JF:  Every piece of knowledge we gain from rare tumors like ‘Eden’s tumor’ will build and help guide us in future treatment decisions for other children; just like how the knowledge has guided us to this point of being able to help Eden.

Our Q&A with Dr. Jennifer Foster is the second in a series about Eden’s incredible story.  If you missed the first installment, an interview with Eden, just head here.
 

YOU CAN MAKE A DIFFERENCE for children, like Eden, who want more opportunities to receive potentially lifesaving treatments and hope for a brighter, healthier future.
YOUR GIFT will fund the most promising, innovative scientific projects with the likelihood of making an impact.
YOUR SUPPORT allows Alex’s Lemonade Stand Foundation to continue to fund researchers around the country to develop less-toxic treatments and more cures for kids with cancer.
DONATE, today. 

 

 

 

 

 

 

 

 

 

December 14, 2018

by Trish Adkins

Children with certain types of hard-to-treat childhood cancers just got another huge dose of hope. Last week, the U.S. Food and Drug Administration (FDA) granted accelerated approval to a drug called Vitrakvi (also known as larotrectinib), making the treatment available to children with cancers that are NTRK fusion-positive. 

NTRK is a gene that is present in certain types of pediatric cancers, as well as some adult cancers.

ALSF funded-researcher Dr. Steven DuBois at Dana-Farber Cancer Institute played a critical role in the research that led to the breakthrough. In a clinical trial led by Dr. DuBois over 75-percent of patients treated with larotrectinib responded positively to the drug and their tumors either shrunk or disappeared. 

“Our goal is to make discoveries and disseminate these discoveries, especially when there is a therapy with such a high response rate and tolerability,” said Dr. DuBois.

The trial was held at multiple sites, including Dana-Farber Cancer Institute, which ALSF funds through its Center of Excellence (COE) program. Dr. DuBois, together with Dr. Wendy London, managed the ALSF grant funds to help sustain the infrastructure of the Developmental Therapeutics Center, collaborate with other institutions, train physician scholars in drug development and build a pediatric oncology developmental therapeutics program. The center actively participates in over 100 oncology studies at any given time.

Each of the patients in the larotrectinib trial had cancers that had the NTRK gene present. NTRK occurs when genes fuse together abnormally, resulting in the growth of abnormal cells, which become cancer. While NTRK is rare, it does occur across a range of cancer types including infantile fibrosarcoma, soft-tissue sarcoma and types of colon, lung and thyroid cancers. 

Prior to the FDA approval of larotrectinib, children with this type of cancer had no effective options for treatment.

This is the second time the FDA has approved a cancer treatment based on a common genetic feature across different types of cancer rather than the specific tumor type. The approval marks a continued effort to develop treatment protocols that could work for several different types of cancer—versus the old paradigm where cancer was treated based on the type rather than the genetic marker.

In Dr. DuBois’ trial, a total of 55 patients, ranging in age from 4 months to 76 years were treated. His findings were published in the February 22, 2018 edition of the New England Journal of Medicine. The article, “Efficacy of Larotrectinib in TRK Fusion-Positive Cancers in Adults and Children,” concluded that larotrectinib, had marked durable anti-tumor activity in patients with TRK fusion-positive cancer, regardless of the age of the patient or of the tumor type. The findings led to the recent FDA approval of the drug. The full article is available here

In addition to the support from ALSF, the trial was supported by Loxo Oncology and by grants from the National Institutes of Health, the Cancer Prevention and Research Institute of Texas and the National Center for Advancing Translational Sciences.


About the ALSF Center of Excellence grants program 
ALSF supports the advancement of clinical trials through its entire research grant program. By funding all stages of research from early career to advanced laboratory studies to our clinical trial programs, ALSF ensures that the breakthrough science happens now, so children can have cures for tomorrow.

Center of Excellence grants are given to institutions over a 5-year period to support research as well as the acceleration of clinical trials through funding for infrastructure needs and training the next generation of research scholars. In addition to Dana-Farber Cancer Institute, ALSF funds Children’s Hospital of Philadelphia, University of California San Francisco and Baylor College of Medicine through the Center of Excellence grant program.

This year, the four-Center of Excellence institutions announced the opening of a multi-site phase 1 clinical trial for a drug that shows promise for reactivating a gene that typically acts as a tumor suppressor. Called p53, the gene is often missing or mutated in several types of resistant childhood cancers including lymphoma, refractory leukemia, Ewing sarcoma, rhaboid tumors, retinoblastoma, hepatoblastoma and other cancers.  Subscribe to the ALSF blog for more details on this Phase 1 study. 

December 3, 2018

by Adam Paris

Eden is a 13 year old who loves to shoot photos and smile for her own snapshots. After being unable to walk or practice her favorite activity, dancing, for nearly a year, she was diagnosed at age 10 with a cancer no doctor had ever seen before. They tried standard chemotherapies for two different types of solid tumors similar to her cancer and after many months doctors deemed her cancer-free in March 2016.

Two years later, the cancer relapsed in her shoulder. Now, she and her tight-knit family continue to fight the cancer they named after her, Eden’s tumor, while waiting for a potential cure. 
As researchers learn more about Eden’s cancer, we wanted to give you a glimpse into Eden, the person. Check out our conversation below to learn more about this inspirational cancer fighter. 

ALSF: If you could have a superpower, what would it be and why?
Eden Green (EG): I would like to fly and be invisible. Flying is cool, but also scary. I like invisibility because I could hear conversations without being noticed in the other room. 

ALSF: What’s your go-to song to sing?
EG: I like to sing Francesca Battistelli. She’s a Christian artist so I sing her songs a lot, and then Lauren Daigle too. 

ALSF: I know you love taking photos! What do you like to photograph? 
EG: I like shooting nature a lot. I do a lot of flowers and nature shots and they turn out pretty good actually. 

ALSF: What is your favorite 2018 memory so far?
EG: My family went on a cruise in January. We went to Cozumel, Jamaica and Puerto Rico. I didn’t get to do as much this year because of my relapse. 

ALSF: What do you want to be when you grow up?
EG: It changes, but I kind of want to be a travel agent like my mom. I changed my mind from nursing because I don’t really want to do that anymore. Maybe a social worker at the hospital, but I don’t even know if I want to work at the hospital.

ALSF: What did you miss most while you were in treatment?
EG: I missed church, and we go to church a lot, so I missed going there. I like to see my friends and everything, so I didn’t get to see them really when I was diagnosed again. That was hard. I got to spend a little bit of my summer with my friends though, especially my one friend Rachel. She’s at my house like every day. I also missed going on trips that I was supposed to go on. 

ALSF: How has your family supported you?
EG: I don’t know, I’m asleep most of the time! My whole family is really funny though, but my dad he’s really funny. He’ll pull up things like the In My Feelings challenge videos to cops lip syncing stuff so he’ll watch those and start dying from laughter!
My sister Mya provides entertainment and my mom does too. Mom has to get her Fitbit steps in. When doctors come to help me I say, “Okay mom, you get your Fitbit steps in.”

ALSF: What does Mya do that helps you out? 
EG: She’s a competitive gymnast, so that helps me because I’m at the gym almost every day with her, watching their whole team. They practice every day for three hours. Most of the time before I relapsed, I was on the floor encouraging them during their routines. I actually got an award for being their team mascot. I’m like their group’s big sister because their siblings usually don’t come and watch them, so I’m always there. 

ALSF: What advice do you have for kids with cancer in the hospital?
EG: Stay strong and you’ll get through it. I’ve done it once and I’m doing it a second time so you can do it, I know you can. Keep having your faith and keep doing what you’re doing to help get through it.  

Our Q&A with Eden is the first in a series about her incredible story. Stay tuned next week for a discussion with her doctor, ALSF-funded researcher Dr. Jennifer Foster. 
 

YOU CAN MAKE A DIFFERENCE for children, like Eden, who want more opportunities to receive potentially lifesaving treatments and hope for a brighter, healthier future.

YOUR GIFT will fund the most promising, innovative scientific projects with the likelihood of making an impact.

YOUR SUPPORT allows Alex’s Lemonade Stand Foundation to continue to fund researchers around the country to develop less-toxic treatments and more cures for kids with cancer.

DONATE, today. 

 

 


 

 

November 29, 2018

by Trish Adkins

In September, Alex’s Lemonade Stand Foundation (ALSF) called over 90 top scientists, pediatric oncologists and researchers from around the world to gather in Philadelphia to discuss the big question:

How can we cross the finish line and find cures for all children fighting cancer?

The meeting kicked off the Crazy 8 Initiative—ALSF’s commitment to identifying obstacles, knocking down roadblocks and developing a comprehensive, achievable plan to foster research, collaborate and accelerate cures for all children

For a long time, the childhood cancer world has focused on the major obstacle—lack of funding for research. Less than 4-percent of the federal cancer research budget is allocated to childhood cancers—requiring support from private foundations to make research possible. 

However, if money was no object, what else gets in the way of finding cures for the 36 children who are diagnosed with one of the hundreds of types of childhood cancer every day in the U.S.? 

That is what the 90 researchers participating in the Crazy 8 Initiative began to answer.

What is the Crazy 8?

“All of us in this room have a fire in our belly and a need to make things change,” said Dr. Nada  Jabado, of McGill University and co-chair of the Crazy 8 Initiative, at the opening session of the three-day meeting. 

The meeting consisted of working groups tackling one of eight areas of pressing concern in the pediatric oncology world. Six groups discussed hard-to-treat cancers—neuroblastoma, embryonal brain cancers, high grade gliomas, fusion positive sarcomas, fusion negative sarcomas and leukemia. The other two groups discussed umbrella topics that affect all types of childhood cancer treatment and research—the use of big data and the acceleration of clinical trials. 

Through small group discussions and full-session cross-topic talks, the researchers identified major obstacles to progress, most notably the need for more high-quality models for testing treatments as well as identifying more genetic drivers of cancer.

“Discovery and research are critical. While we make progress every day, we’ve probably discovered less than 1-percent of all targets,” said Dr. John Maris, of Children’s Hospital of Philadelphia and co-chair of the Crazy 8 Initiative. Dr. Maris is referring to the specific genes, mutations and other biological traits that make cancer develop and grow. By identifying those targets, doctors could match therapies that kill the drivers and in turn, stop the cancer. 

But, there is still work to be done to get there. Members of each working group are currently finalizing the first plan to tackle these eight areas. The phase one plans will be available in December 2018. Then specific grant projects will be developed for funding and ALSF can begin to follow the Crazy 8 roadmap straight toward the destination we all dream of: cures. 

Born from Alex’s Legacy. 
The idea for the Crazy 8 Initiative was born from the legacy ALSF founder Alexandra “Alex” Scott left for all of us. Alex was treated for neuroblastoma nearly her entire life. When one treatment left Alex feeling good for the first time that she could remember, she told her parents that she wanted to host a lemonade stand to help her hospital make other kids feel better, too. 

After that lemonade stand, Alex felt even more dedicated to helping kids like her. When her mother, Liz Scott, suggested that the money raised go toward neuroblastoma research, Alex replied: “But mom that would be selfish.”

Alex knew that all children needed cures. Her legacy compels ALSF to make that dream a reality. You can support the Crazy 8 Initiative and follow along here.
 

October 16, 2018

by Trish Adkins

Alex’s Lemonade Stand Foundation (ALSF) has declared a war on childhood cancer.

Every day of every month of every year, the equivalent of a classroom of children is diagnosed with cancer in the United States—36 sons, daughters, brothers and sisters—all facing the fight of their lives. 

ALSF has worked to find cures—and has already made incredible strides in improving standards of care, increasing the number of childhood cancer clinical trials, helping families access cutting-edge treatments and funding breakthroughs like CAR T cell immunotherapy. Until there are cures for all children, more work must be done.

Here are 10 facts you need to know about the war on childhood cancer:

1. Children are dying.

Childhood cancer is the leading cause of death by disease for children in the United States. One in five children diagnosed with cancer will die within five years. And every year, an estimated 80,000 children die from childhood cancer around the world. 

2. The deadliest of all childhood cancers are brain tumors.

Brain tumors bumped leukemia out of the top spot in 2016. This isn’t because brain tumors are harder to treat, but because research has made enormous progress in treating several types of pediatric leukemia. 

3. Childhood cancer is biologically different than adult cancers.

Even though they share the same name, childhood and adult cancers do not necessarily share the same treatment protocol. 

4. Traveling for treatment can leave families with impossible choices.

The average cost of one hospital stay for a child with cancer is $40,000—five-times more than the cost of hospitalization for other pediatric health issues. Add another cost of traveling for treatment and families can be left choosing between putting gas in their tank and food on their table. In 2017, the ALSF Travel for Care program helped over 500 families access treatment by funding over 300 flights, 1,000 nights of lodging and 820,000 miles in gas cards.

5. Just 4-percent of the federal budget for cancer research is allocated towards children.

Imagine being told you can only eat 4% of your meal. Or you can only have 4% of your paycheck each week. For cures to become a reality, private research funding, from organizations such as ALSF, is required. 

6. The number of cures that researchers are searching for is infinite.

Childhood cancer is not just one disease or even a dozen diseasesthere are hundreds of subtypes. Researchers are searching for genetic targets within tumors and then developing and matching drugs to attack those targets, making cures a reality.

7. Big data could hold one of the keys to cures.

As researchers continue to discover targeted therapies, they can turn to data for assistance. The Childhood Cancer Data Lab, funded by ALSF, provides researchers with faster, easier access to the wealth of childhood cancer data available.

 8. It’s not just cures we need. We also need safer treatments.

Seventy-five percent of childhood cancer survivors are left with serious side effects for the remainder of their lives. Late effects of childhood cancer treatment affect organ and tissue function, growth and development, learning and memory and psychological adjustment. Treatment can also leave survivors at a higher risk of secondary cancers.

9. Accelerating the rate of clinical trials is key to accelerating cures.

Clinical trials provide two important functions: providing scientists with data and offering hope to children battling relapsed cancer. Clinical trials are expensive and time-consuming—but through the ALSF Infrastructure and Center of Excellence grant programs, institutions can access funds that support and speed up the establishment of clinical trials. 

10. Innovative collaboration among researchers will lead to cures.

The childhood cancer research community is working together to find cures. Through the ALSF Crazy 8 Initiative—a groundbreaking effort to build a roadmap to cures—researchers are working in a coordinated effort to ensure childhood cancer is just a memory. 

Learn more about how ALSF is funding breakthroughs and finding cures for childhood cancer and get more childhood cancer facts here. 


 

July 9, 2018

by Trish Adkins

In 2000, the first draft of the map of human genome—a mosaic representation of characteristics of what makes our biology uniquely human—was released. The map paved the way for more genomics research in several fields ranging from human biology to agriculture and gave scientists models of genetically normal cells which they could compare to abnormal cells, like those cells that make childhood cancer so deadly.

Now, in 2018, an ALSF funded-research project has resulted in the release of over 270 genetic sequences of 25 different types of childhood cancer used routinely by the National Cancer Institute’s Pediatric Preclinical Testing Consortium (PPTC). Each unique tumor model and its biological characteristic data is available to all academically qualified petitioners—opening the door for breakthroughs in childhood cancer research.

Keep reading to learn how cures are getting closer, one childhood cancer genome at a time. 

The story behind the 270 models begins with the PPTC

Founded in 2015 and funded by the National Cancer Institute, the consortium works to develop reliable preclinical testing data for potential pediatric cancer drugs. There are hundreds, maybe thousands of potential cancer drugs—making the study of each drug in a pediatric clinical trial impossible. The PPTC narrows down the list, providing researchers with reliable drug effectiveness data that they can use to accelerate research from “bench to bedside;” bringing science out of the lab and into the clinic. The models studied are directly derived from childhood cancers at diagnosis or relapse, and thus are directly representative of the types of cancers treated in clinical trials.

However, while there is a large pool of potential drugs, there was not a large pool of accurate pediatric tumor models for which to test the drugs. This has long been a struggle for the pediatric oncology research community. Over 14.1 million people are diagnosed with cancer each year worldwide, but only 250,000 of those cases are pediatric cancer. The pool of potential tumors to model is small and obtaining viable tumor cells is difficult, especially for some types of pediatric cancers like spinal cord tumors where securing tissue samples is tricky because of the tumor’s location. 

The PPTC had an idea for a new major effort, the Pediatric Preclinical Genomic Characterization Project, which sought to characterize the tumor samples being used in drug testing. These patient-derived xenograft (PDX) childhood cancer models were being used routinely, but the majority did not have detailed genetic data available. 

The potential was enormous: with a critical mass of PDX models made available to the scientific community, the PPTC could accelerate the route to clinical trials much more rapidly than ever before, bringing potentially lifesaving treatment to children waiting desperately for cures.

There was one catch: there was no funding available for a PDX sequencing project. That’s when ALSF entered the picture. 

The Foundation learned about the PPTC and its desire to generate high-quality PDX genetic data to streamline science’s understanding of why novel treatments work in some cases, but do not work in others, and immediately recognized its promise. 

“ALSF has a legacy of filling critical research and family services gaps in the childhood cancer community,” said Liz Scott, Co-Executive Director of ALSF.  “We knew that funding the PPTC’s genomic sequencing project had the potential to spark long-lasting impact, collaborative efforts and ultimately advance the pace of finding cures for all kids with cancer.”

Legacies of Hope
With the ALSF funding, the PPTC could characterize the stored samples that had been donated by children battling pediatric cancer. Some donations came while a child was in treatment, with an institution’s requested permission to use extra tumor tissue that was not needed for diagnosis or treatment protocol, for research.  

Other donations came from families eager to find cures even when it was too late for their own child. These profound gifts, given at the time of death, left behind a legacy of hope waiting to be unlocked.  

The PPTC has access to over 400 samples representing 25 different types of childhood cancer, stored at -80℃ in its five locations at institutions in the United States and also in Australia, and continues to generate more, often in collaboration with Dr. Patrick Reynolds who receives ALSF funding for the Childhood Cancer Repository where many genetic models are generated. The vast majority of the samples represent relapsed disease and have the promise of modeling childhood cancers at the time that many new investigational treatments are tried in the clinic in Phase 1 trials.

While the PPTC could have tried to establish the tumor lines in a test tube or dish, the researchers leading the project knew from prior experience that growing tumors in artificial environments could lead to the generation of different mutations in revolt to their new homes. These mutations would lead to inauthentic cell lines and muddy the search for drugs that could work. 


Accelerating the Clinical Trial Process
Bringing the right drugs to the clinic has long been a struggle for pediatric oncology researchers.  

The first priority is to ensure a patient’s safety in a clinical trial by adhering to specific safeguards before the trial begins and during the trial. But a safe drug is not necessarily effective and can offer false hope to patients who are enrolled in clinical trials after one relapse—or several.  

Using the PDX models, researchers could discover the “good drugs”—the drugs most likely to be safe and effective in killing cancer cells, and also discover the “bad drugs”—those that are not effective and those that might even result in resistant disease.

The models also give researchers the opportunity to continue to move away from treating diseases by name and begin treating the specific genetic lesions that might drive cancer growth. It is the literal meaning of “killing two birds with one stone”— two different types of cancers may share a genetic trait and in turn, could be sensitive to the same drug. 

“With good models, we can begin designing experiments more robustly and begin getting the right drugs to the clinic and to children quickly,” said Dr. John Maris, MD, of ALSF’s Scientific Advisory Board and Children’s Hospital of Philadelphia’s neuroblastoma representative in the PPTC.

ALSF’s contribution allowed the PPTC, in collaboration with Baylor College of Medicine and Nationwide Children’s Hospital (led by David Wheeler and Julie Gastier-Foster), to genomically characterize over 270 PDX models with four different genomic tools—each tool giving researchers more clues to how the genes and proteins drive cancer growth.

Researchers worked to filter out any noise or irregularities in the final data, using existing cancer cell knowledge and past research. They have ensured the models matched their cells of origin and have retained known cancer driver mutations over time. 

The PPTC began using the data immediately—fulfilling its mission of matching drugs to genetic targets and testing in advance of human clinical trials. 

Now, eighteen months after the PPTC commenced the PDX project, other scientists now have the same opportunity. The data, which was released on July 9, 2018, is available to all academically qualified petitioners through the PedcBioPortal for Childhood Cancer Genomics (pedcBio portal). Raw characterization data will be available on the database of Genotypes and Phenotypes (dbGaP) in the coming months. Tissue samples will be available by request—for just the cost of postage to ship. 

“When childhood cancer relapses, it can become lethal,” said Dr. Maris. “But today, the scientific community has open access to deep genetic profiling that will help overcome some of the major problems we have when treating childhood cancer. We’ve now accelerated years ahead in our search for cures.”

Read more about the PPTC project, as well as other innovative ALSF research here. 

April 20, 2018

by Trish Adkins

Childhood lymphoma arises from the immune system cells and occurs when those cells grow in an uncontrolled and uncoordinated way. While it shares some similarities with leukemia, lymphoma often settles in the lymph nodes of the body, instead of in the bloodstream.

Each type of lymphoma is named by its cell of origin and this dictates treatment protocol. The good news: the most common types of lymphoma (Hodgkin, Non-Hodgkin, Burkitt) have relatively high cure rates as compared to other types of childhood cancer.  

The bad news: cure rates are not 100-percent and even those children who are cured of primary lymphoma can face a lifetime of side effects including cardiac dysfunction and even secondary cancers. 

But, there is hope on the horizon. Alex’s Lemonade Stand Foundation (ALSF) funded researchers so they could continue to search for safer treatments and cures for children battling lymphoma. Meet some of these childhood cancer heroes and the researchers making a difference every day:

Miracle Cure: Zach

When Zach was 5 years old, he was diagnosed with anaplastic large cell lymphoma (ALCL), an extremely rare type of non-Hodgkin lymphoma. While still in treatment, Zach’s lymphoma relapsed. As Zach became sicker and his prognosis worsened, his parents felt they were out of options. Traditional chemotherapy was not working and, in fact, was making Zach sicker. Zach was running out of time. 

Zach’s lymphoma tested positive for ALK, an abnormal gene that fuels certain types of cancer. ALSF-funded research made a clinical trial for a new drug possible, bringing hope to his family. This drug, known as crizotinib, targets and turns off the ALK gene. Days after taking the drug, Zach was feeling better and his cancer was disappearing. 

Now Zach is 12 years old and cancer-free. 

Breakthrough Research: Dr. Yael Mosse, Children’s Hospital of Philadelphia 

Funded by an ALSF Reach grant, Dr. Yael Mosse led the research team at Children’s Hospital of Philadelphia that made Zach’s treatment possible. Her team theorized that since crizotinib worked in ALK-positive lung cancer in adults, it could be the hope children battling ALK-positive cancer needed. 

And since crizotinib is a targeted therapy that just attacks one gene, it has fewer side effects as compared to chemotherapy. 

Dr. Mosse’s clinical trial enrolled 30 children--including Zach. Zach was seven out of the eight children with the same type of lymphoma that received the same miraculous cure. The trial also tested crizotinib in children battling ALK-positive neuroblastoma with successful results. 

Hero (and Boss Lady) Sydnie 

The day Sydnie, age 13, was diagnosed with Hodgkin lymphoma she cried for a moment and then decided no one else was allowed to cry in front of her.

Then, Sydnie asked her mom to dye her blonde hair purple, pink and blue—because she knew she was going to lose it anyway. When she lost her hair as a result of chemotherapy, she wanted to shave her head right away because, as Sydnie, declared, cancer was not going to determine if she was bald or not. It was up to her.

“I won’t let cancer be the boss of me,” Sydnie said. 

Hodgkin lymphoma has a relatively high cure rate (80-90% in most cases) but requires immediate chemotherapy and radiation. Sydnie endured nine months of chemotherapy and 10 days of radiation. 

Today, she is cancer-free.
 
Translating Research into Treatments: Dr. Helen Heslop, Baylor College of Medicine 

Dr. Helen Heslop, a researcher at Baylor College of Medicine (an ALSF Center of Excellence) and Texas Children's Hospital focuses her work on translational research, which combines testing in the lab with patient studies. This type of research can help accelerate new therapies to clinical trials and someday, as new standards of care. Of particular interest to Dr. Heslop is how immunotherapy treatments, like CAR T cell immunotherapy, could help children with hard-to-treat lymphoma and also lead to safer frontline treatments. 
 
“The grants that ALSF provides support early career research, innovative, high-risk ideas and early phase trials that are critical for clinicians and scientists as they test approaches to improve outcomes,” said Dr. Heslop. 
 
Learn about lymphoma research and meet more childhood cancer heroes, here

December 1, 2017

by Adam Paris, ALSF

John Szigety was diagnosed at age 10 with Hodgkin lymphoma and underwent treatment at Memorial Sloan Kettering Hospital and Hackensack University Medical Center. After eight months of treatment, he suffered a relapse in early 2006, but completed treatment that June. Today, he is 11 years cancer-free. 

Amjad Shaikh was diagnosed just before his 9th birthday with leukemia and went through his entire treatment schedule at Children’s Hospital of Philadelphia (CHOP) for four years, before entering remission. Today, he is cancer-free. Amjad Shaikh

Both are childhood cancer survivors and medical school students who participated in Alex’s Lemonade Stand Foundation’s (ALSF) Pediatric Oncology Student Training (POST) grant program this past summer. They each felt the immense pride of being able to give back and help kids fight for their lives just like they did. They talked about the experience of beating cancer as kids and now, as researchers, searching for a cure.

Can you describe your work this summer as a POST student?
John Szigety (JS): I was looking at the effect of a drug on tumor progression and cell replication. The doctor I worked with at CHOP, Dr. Sarah Tasian, was investigating how to treat cancers with a specific mutation that makes the disease especially aggressive.
Amjad Shaikh (AS): I looked at CHOP and found an opportunity to study pediatric leukemia. I worked on studying mortalities associated with hospital characteristics and what a hospital can do to improve treatments or survival rates in kids fighting the same kind of cancer that I did.

 

Pictured: Amjad Shaikh

What was most meaningful about this experience?
JS: It was meaningful to see the different roles that a pediatric oncologist plays. As a patient, I only saw my oncologist, Dr. Steven Halpern, as a clinician. Dr. Tasian showed me how big of a role research plays in her life as a physician. That was new information to me.
AS: Part of it was that I got to see a lot of pediatric patients. The fact that I was on the other side and I understood from a clinical and medical level what was going on, I felt that maybe given a couple years I could be the person who was helping them out.

Was there a particular individual who inspired you to pursue pediatric oncology? 
JS: Dr. Halpern. When I was feeling miserable or having a bad day, he was receptive to me about how to change my regimen to make it more manageable. He would stop in my room during treatment to say hello or play board games. The cliché is that he treated me like a person and not an illness, but it's true. He's a big reason why I want to go into this field.
AS: My oncologist, Dr. Stephen Grupp. A few years after treatment, he told me he had been diagnosed with cancer and was undergoing treatment. It was a bit shocking because I had never seen an adult go through chemo. It left an impression that despite the fact he was going through chemo, and knowing what that was like, he still came in and treated kids. That meant a lot to me. It guided my philosophy in med school and framed my outlook for how a doctor should be. 

What did this POST opportunity and the chance to help other kids fight cancer mean to you?
JS: It was an honor. Since my diagnosis, I have wanted to help children affected by cancer. I've worked at Camp Kesem (summer camps hosted for kids whose parents have cancer), fundraised for various charities and pursued an education that brought me to medical school. The POST grant helped me contribute in another important way by investigating potential new treatments. While I didn't cure cancer or even come close, I am proud to say that I helped.
AS: It meant the world. Seeing Dr. Grupp and the team of physicians at CHOP, that’s my vision for the kind of doctor I’d like to be. 

As someone who survived childhood cancer, what does ALSF represent to you?
JS: In a word, ALSF represents hope. By supporting research across the country, ALSF represents the future of medical treatments and all the advances that are yet to come. 
AS: I think it’s a great organization for helping to develop a new generation of physicians and researchers who are going to continue these advancements. 

What are your goals and aspirations going forward?
JS: My goals are to become a pediatric oncologist and help treat children who have to fight for their lives. As a survivor, I have an understanding of how these pediatric cancer patients feel and I hope to help my future patients through the frightening and overwhelming experience of battling cancer.
AS: Especially after this summer I’m definitely set on a career in oncology. I’m not 100% sure how I want to go about it, since I'm still exploring all the specialties, but pediatric oncology is at the top of my list.

Each year, ALSF provides grants opportunities to medical students through the POST grant program. Read more about our POST grants here.