Childhood Cancer

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Osteosarcoma is the most common type of childhood bone cancer. It typically develops from osteoblasts, the cells that make growing bone. It most commonly diagnosed in adolescents who are having a growth spurt. Osteosarcoma is more common in boys than in girls. 

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Latest Osteosarcoma grants

Jie Song, PhD, Principal Investigator
University of Massachusetts Medical School
Innovation Grants, Awarded 2017
Beau Webber, PhD, Principal Investigator
University of Minnesota
Young Investigator Grants, Awarded 2017
Peter J. Murray, PhD, Principal Investigator
St. Jude Children's Research Hospital
Springboard Grants, Awarded 2013

Latest Osteosarcoma blog posts

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. 

February 7, 2018

When 14-year-old U.S. Junior National Cyclist Arnav, fell off his bike and shattered his helmet during a race, the race official suggested he drop out. 

Arnav shook off the fall and went on to finish 9th.

Then, just weeks later, Arnav’s leg began bothering him. He developed a noticeable limp—but it seemed typical for an athlete who was training four to six hours a day, six days a week. Rest and ice did nothing to curb the pain. Arnav kept racing, even though his leg ached. Then, Arnav had an MRI which revealed osteosarcoma, the most common type of bone cancer in children. Typically diagnosed in adolescence and more frequently in boys, osteosarcoma starts as pain around a joint. Often, like in Arnav’s case, the pain is attributed to a sports injury. 

A decade ago, Arnav’s diagnosis would have meant certain amputation.

You can’t ride a bike, if you don’t have a working leg. 

Lucky for Arnav and thanks to innovation in the field of orthopedic oncology, most children battling osteosarcoma today get a chance to save their leg and keep their mobility. 

For Arnav, who is now 15 years old, that meant he could hold on to his cycling dreams. Watch more of Arnav’s inspiring story of positivity and victory in the face near-certain defeat:


August 28, 2017

by Trish Adkins

Every year, over 250,000 new cases of cancer affect children around the world. Every day, 250 of these children will die from cancer. Childhood cancer is the leading cause of death by disease for children in the United States.  Despite these facts, childhood cancer research is consistently and vastly underfunded. There are dozens of types and hundreds of subtypes of childhood cancer—and many of these types have no known cures. 

Despite all these facts, there is so much hope for children battling cancer. Childhood cancer families, researchers, friends and communities have joined together with Alex’s Lemonade Stand Foundation to find cures for all types of childhood cancer. ALSF began with one little girl’s dream and has now grown into a worldwide movement to find safer, more effective treatments. Since 2005, ALSF has funded over 690 research grants, powering breakthroughs and getting closer to cures for all. 

Here are five videos that tell the story of Alex’s Lemonade Stand Foundation and will change the way you think about childhood cancer forever:

1. ALSF Founder and Original Lemonade Girl: Alex Scott

Alex Scott was just 3-years-old when she held her first lemonade stand in her front yard. It seemed to be just a lemonade stand, but it started a movement to cure childhood cancer. 

Before Alex died in 2005, she raised over $1 million for childhood cancer research. She left all of us with a legacy and a directive to continue the fight for cures, one cup at a time. 

2. Survivor Turned Oncology Nurse: Taylor

When Taylor was 11-years-old, she was diagnosed with osteosarcoma, a type of bone cancer. After nine months of treatment, Taylor was declared cancer-free. Eighteen months later, she relapsed. 

Her family was desperate for a cure. Taylor enrolled in a clinical trial at Texas Children’s Hospital. The trial was possible due to an infrastructure grant funded by ALSF. The trial worked. Today, Taylor is cancer-free and studying to become a pediatric oncology nurse. 

3. Breakthrough Treatment Leading to Cures: Edie 

When Edie was just 5-months-old, she started having a severe upset stomach and trouble sleeping. Edie’s parents had a gut feeling this was not just a typical bug or stage of infancy. 

Edie was diagnosed with stage IV neuroblastoma. Despite an aggressive treatment plan, Edie’s cancer continued to grow.  Testing showed that Edie carried the oncogene called anaplastic lymphoma kinase (ALK). ALSF funded-researcher Dr. Yael Mosse had a clinical trial for children with the ALK gene. The trial worked and today, Edie is cancer-free. 

4.  Living Proof that Research Works: Zach

Zach is a 12-year-old who loves playing baseball. 

When Zach was 5-years-old, he was diagnosed with anaplastic large cell lymphoma. While still in treatment, Zach relapsed. His condition worsened. Zach was getting sicker and sicker. It was a race against time to save his life. Since standard treatment was not working, Zach’s doctors told his parents about a clinical trial funded by ALSF. His parents took the risk. Within a few days, Zach was running down the halls of the hospital. His cancer disappeared. 

5. From Cups to Cures: What Lemonade Can Do 

ALSF-funded researcher Dr. Jeffrey Huo knows firsthand that cups of lemonade can add up to cures for childhood cancer. 

When you donate and supporters all around the world donate, your cups of lemonade add up to enough funding for an experiment in a pediatric oncology. Those experiments give scientists pieces of data which they analyze and eventually use to publish a journal article with their findings. Several journal articles lead researchers to breakthroughs and those breakthroughs become cures for childhood cancer.

Want to get involved in the fight against childhood cancer? Check out Alex's Million Mile, the largest Childhood Cancer Awareness Challenge! We are going 1 million miles and raising $1 million for childhood cancer research! Start or join a team, fundraise for a cure and help us go the distance for kids with cancer!

Subscribe to the Alex's Lemonade Stand Foundation YouTube channel for more inspiration.