Childhood Cancer

Acute Lymphoblastic Leukemia (ALL)

Childhood acute lymphoblastic leukemia (ALL) is cancer of the white blood cells, the cells that help fight infection. ALL is the most common form of childhood leukemia.

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Latest Acute Lymphoblastic Leukemia (ALL) grants

Takaya Moriyama, MD/PhD, Principal Investigator
St. Jude Children's Research Hospital
Young Investigator Grants, Awarded 2018
Christian Hurtz, PhD, Principal Investigator
University of Pennsylvania
Young Investigator Grants, Awarded 2018
Andrei Thomas-Tikhonenko, PhD, Principal Investigator
Children's Hospital of Philadelphia
Innovation Grants, Awarded 2017

Acute Lymphoblastic Leukemia (ALL) Heroes

Latest Acute Lymphoblastic Leukemia (ALL) 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. 

May 23, 2018

by Ruth Ciamarra, hero mom and host of “Anna’s Lemonade Stand”

While my daughter Anna was in treatment for T Cell acute lymphoblastic leukemia (ALL), we spent days confined to the house. Anna had a weakened immune system and as a result of our medically necessary confinement, she officially missed an entire school year. I was looking for ways to get our family involved in doing something—anything—to help other families avoid enduring the same outcome. 

Then I saw a social media post in a cancer moms group about Alex’s Lemonade Stand Foundation (ALSF). I immediately went to the ALSF site and I was happy to find ideas on how to set up a lemonade stand to raise money for better treatment options and cures for kids like Anna. 

It sounded like an easy way to have Anna and her sister host a fun event in our front yard. Anna would have easy access to our house if she might not feel well, her sister could get involved with her friends to help set up and sell, and our family could actively take part in making a difference.

As it goes with cancer, unexpected medical issues can pop up at any time. Anna spent the night before our first lemonade stand in the ER. She made it home in time for the stand and for the amazing day we had!

The materials sent to us from ALSF gave us everything we needed to get started. With the many signs, posters, donation materials, and so on, plus our own canopy tent, table, chairs, and some balloons, opening for business was easy! Neighbors, strangers, classmates, nurses from our hospital, and even an adult childhood cancer survivor stopped by our house not only to donate, but  to also ask about who Alex was, the little girl who started it all. What an amazing opportunity to share awareness about such an important cause!

The ripple effect of Anna’s Lemonade Stand spread throughout our community. Other children hosted their own stands and donated the proceeds to our event. Our school partnered with us to host a Lemonade Days stand outside of the school in a more visible location than our front yard. Additionally, the school and larger community have provided planning support, volunteers, donations of supplies and even a wooden lemonade stand! Last year, we held a classroom competition in the days leading up to the stand; we hope to continue to innovate and improve on how we can make this an annual tradition. 

For those interested in hosting a stand for the first time, here is some advice:

  • You do not need to feel overwhelmed by planning a grand event. Our own available tools and materials from ALSF was more than enough to simply and easily host our own stand and make a difference right away. If you feel comfortable, ask family or friends to help out the day of the stand. If you’re like us, you will keep running out of lemonade (a good thing!) and will need adults nearby to supervise while you run back inside to make more. 
  • Use your social media outlets and your online fundraising page well in advance of your stand if you can. So many people will want to help, even in small amounts, and this gives them an opportunity to support you remotely. 
  • Lastly, have fun! Our daughters have found so much joy, confidence, and pride in hosting these events each year. It gave them something to look forward to during one of the toughest stretches of treatment for our daughter, and it continues to be a highlight of our summer. We can’t wait to host again this year!  

Read more about Anna and her battle with ALL, here

Ready to get involved? You, too, can be a Lemonade Days stand host! Get the details here

 

 

 

December 2, 2017

by Trish Adkins, ALSF

Breakthroughs and cures for childhood cancer are only possible through comprehensive, cutting-edge research performed by the best minds in pediatric oncology. Each year, Alex’s Lemonade Stand Foundation (ALSF) provides grants to these oncology superstars—ensuring the future of research will mean safer treatments and more cures for children.

Our grants program funds all phases of research—from early career and student research to clinical trial support—through 13 different grant programs. Each potential project is given careful consideration and review by our Scientific Advisory Board, made up of leading scientists and clinicians. 

Two research grant areas: The ‘A’ Award and the Bio-Therapeutic Impact Grant programs address the full spectrum of research providing funding for early and late-career scientists who are studying hard-to-treat, high-risk cancers. The 'A' Award is designed specifically for the early independent career scientist who wants to establish a career in pediatric oncology research. The Bio-Therapeutic Impact Grant accelerates a project closer to clinical trial, bringing promising treatments to children waiting for cures. 

Meet three of our latest grant recipients and find out how they are powering breakthroughs, one research project at a time:

Fighting Neuroblastoma with Natural Killer T-Cells: Andras Heczey, MD, Baylor College of Medicine 
Dr. Heczey, a 2017 ALSF Bio-Therapeutics Impact Grantee, is developing a novel form of cancer immunotherapy for children with neuroblastoma. His therapeutic will use Natural Killer T cells (NKTs) which are part of the immune system. Their presence and/or absence could have implications in the development of cancer. Dr. Heczey altered NKTs in the lab with the hopes that these newly engineered cells could lead to the development of NKT cell-based immunotherapy for neuroblastoma, as well as have an application for other types of childhood cancer. His funded research will accelerate NKT immunotherapy closer to the clinical trial stage. 

 

 

Targeting Synovial Sarcoma: Cigall Kadoch, PhD, Dana-Farber Cancer Institute 
Dr. Kadoch, a recipient of The ‘A’ Award, is studying the genetics of synovial sarcoma, an aggressive soft-tissue malignancy that is largely resistant to conventional chemotherapy-based treatments. Synovial sarcomas all share a common chromosomal translocation—a genetic event in which two proteins abnormally fuse together in the DNA. The result is the development of abnormal cells. Dr. Kadoch will study this genetic phenomenon, work to understand the underlying mechanisms that cause it to happen and provide insights on how this translocation could be refused, with the aim of eventually developing highly-targeted drugs that can stop synovial sarcoma. 

 

 

Unpackng Leukemia: Andrew Lane, MD/PhD of Dana-Farber Cancer Institute 
Dr. Lane, a current 'A’ Award and past Young Investigator grantee, will study how DNA “unpacking” promotes acute myeloid leukemia (AML). Children diagnosed with AML have a poor survival rate. Dr. Lane believes that the physical structure of DNA could be important in leukemia development. He explained that if the DNA in a cell was stretched out, or unpacked, it would be six feet long. Yet to work properly, DNA needs to be tightly packed together into a space as small as the head of a pin. Under the microscope, leukemia DNA does not appear to be as tightly wound as it should, leading researchers to believe that problems in DNA packing can lead to the development of AML. Dr. Lane will study this phenomenon and will test if the drugs that target DNA packing can also kill leukemia cells. 

Read more about our grants programs here.