Childhood Cancer

Wilms' Tumor

Wilms’ tumor is the most common form of childhood kidney cancer. It most often occurs in one kidney; although it can be found in both kidneys at the same time. 

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Latest Wilms' Tumor grants

George Q. Daley, MD/PhD, Principal Investigator
Boston Children's Hospital
Innovation Grants, Awarded 2016
James Amatruda, MD/PhD, Principal Investigator
University of Texas Southwestern Medical Center at Dallas
Innovation Grants, Awarded 2016
Kenneth Chen, MD, Principal Investigator
University of Texas Southwestern Medical Center at Dallas
Young Investigator Grants, Awarded 2016

Latest Wilms' Tumor 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. 

January 13, 2018

The first signs of Wilms’ tumor—a painless swelling in the abdomen, blood in the urine, belly pain, high blood pressure or fevers—often seem to be symptoms of something much less scary. A pediatrician will recommend an abdominal ultrasound and then a diagnosis will be made. Treatment, which typically includes surgery, radiation and chemotherapy, begins. Most children, even those with higher stages of the disease can finish treatment knowing that they will most likely never battle Wilms’ tumor again.

However, being cured of Wilms’ tumor can come with a bevy of long-term side effects—loss of kidney function, bone and skeletal deformities, lung issues and more cancer.

With each year of remission, the risk of a secondary cancer increase, particularly for those who require radiation.

Dr. Julie Glade-Bender

Dr. Julia Glade-Bender

“No matter what you do, you are exposing a fair amount of a child’s body to radiation and every little bit of radiation counts, particularly in children who have already declared themselves as a ‘tumor-former’,” said Dr. Julia Glade Bender, a member of the ALSF Scientific Advisory Board and an oncologist at New York-Presbyterian Hospital/Morgan Stanley Children’s Hospital.

Now, oncologists are studying the biological drivers of Wilms’ tumor to understand how to reach a 100% cure rate, while minimizing long-term side effects.

The cures and the side effects. 
For years, the mainstay of treatment was surgery, chemotherapy and aggressive radiation. Clinical trials have shown that chemotherapy can be shortened and radiation can be eliminated for many children with Wilms’ tumor. For those who still require it, doses of radiation have been reduced, but not enough to completely avoid potential significant long-term health risks.

Radiation can leave a child susceptible to skeletal deformities—the radiated side of the spine can grow slower than the non-radiated side. Radiation can also cause restrictive lung disease later in life and hamper an individual’s ability to breathe. It also exposes a child to the risk of more cancer.

Every cancer is different.
Under the microscope, Wilms’ tumor cells look similar to other childhood cancer cells—round blue embryonic cells.

However, the microscope does not tell the whole story.

“Every cancer is different, because every child is different,” said Dr. Glade Bender. “The critical question is: which cure goes with which patient?”

The biology of Wilms’ tumor coupled with the biology of individual patients affected by disease are two areas of specific interest to researchers. They are working to understand the origins of the disease and also to understand why some cases of Wilms’ tumor are treatment resistant.

Researchers are performing retrospective studies and reviewing past cases of children who relapsed following frontline treatment. They are also working to study patterns of chromosomal changes that happen in children diagnosed with Wilms’ tumor.

Dr. Glade Bender and other ALSF-funded researchers see promise in the study of developmental therapeutics, which tests and examines new treatment agents in children after standard therapy has failed. These studies have the potential to identify new drugs that can help children who relapse and provide meaningful insights into offering safer treatments from the very beginning to future children affected by cancer.

“We won’t stop searching for cures, until we are at 100%,” said Dr. Glade Bender.

Learn more about Wilms’ tumor research and ALSF-funded projects, here.

November 8, 2017

“I want chocolate milk, IMMEDIATELY,” those were 4-year-old Sophia’s first words following a seven-hour surgery to remove a tumor on her kidney. 

It is no surprise that little Sophia, who is now 8 years old and a survivor of Wilms’ tumor, the most common type of kidney cancer in children, describes herself  as “one tough cookie.”

Wilms’ tumor typically presents itself as a painless swelling of the belly. In Sophia’s case, her pediatrician noticed it at her routine 4-year-old checkup. For other children, a parent might notice the swelling or even, rarely, blood in their child’s urine.

Treatment includes surgery to remove the tumor or the entire affected kidney, chemotherapy and radiation. The disease has a high cure rate—as high as 90% for most children—but high doses of radiation can cause long-term side effects. Children who have been treated for Wilms’ tumor have regular scans to check for both relapse and side effects. 

Sophia had surgery, followed by 18 months of chemotherapy and radiation at Wesley Medical Center in Wichita, Kansas. Treatment was hard—but her family worked just as hard to keep Sophia’s spirits high with special outings and lots of love. Sophia kept her spunky, tough cookie attitude throughout treatment. 

Sophia has been three years cancer-free and, together with her family, is a Hero Ambassador for Alex’s Lemonade Stand Foundation. Her family knows how important childhood cancer research is for children like their daughter.

“I’m so grateful that Sophia is well and that there’s a treatment plan for her, but she suffered,” said Tiffany Stepien, Sophia’s mom. 

Treatment for Wilms’ tumor has been largely unchanged for some time, because of its high cure rate. However, high doses of radiation to a child can come with several serious side effects, including scoliosis, restrictive lung disease and sarcoma, later in life. Now, research is studying how targeted, individualized treatments can help limit the use of high dose chemotherapy and radiation, so children can be cured without being harmed. Research is also looking to identify the highest risk patients—those who may be prone to relapse—so that this group can receive higher doses of frontline treatment and beat Wilms’ tumor without relapsing. 

Sophia is already planning to help search for safer, more effective treatments for kids like her. 

“Sophia always said she’s going to invent a chemo that only gets the cancer cells and not hair or stomach cells,” said Tiffany. “And then, “I am going to invent glasses that can just look at my belly and know the cancer is gone and I won’t need a CT scan.”

Read more about Sophia here.

ALSF funds several research projects studying Wilms’ tumor, as well as Beckwith-Wiedemann Syndrome, a genetic predisposition disorder that can lead to the development of Wilms’ tumor. Read more about that research here.