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Crystal Ball: The Legacy of the PCGP

The Pediatric Cancer Genome Project provided transformational insights into how to approach research and treatment for childhood cancer.

For instance:

  • The project has yielded discoveries across 23 types of childhood cancers and even the non-cancerous degenerative disorder ALS, also known as Lou Gehrig’s disease.
  • Researchers found that about 10% of patients studied were born with genetic changes or mutations that increased their cancer risk.
  • The findings led to major National Cancer Institute initiatives to develop specific pediatric cancer therapies.
  • The project’s data and its analytic and data-visualization tools are freely shared worldwide through St. Jude Cloud, one of the largest online repositories of pediatric genomics data.

Based on these and other PCGP accomplishments, what’s next?

Where do we go from here?

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James R. Downing, MD

St. Jude president and CEO
Pediatric Cancer Genome Project architect

James Downing, MD

“We’ve gained new insights into every tumor subtype we’ve sequenced, but there are still many discoveries to be made from PCGP data. There are still genetic alterations that affect or drive cancer that we haven’t found.

“The legacy of the PCGP is that it has stimulated new research questions. What we continue to learn from this project will influence not only pediatric cancer, but adult cancer and many other diseases.”

Amar Gajjar, MD

Pediatric Medicine chair

Amar Gajjar, MD

“From the Pediatric Cancer Genome Project, we learned that medulloblastoma, the most common malignant brain tumor, is not a single entity. It has four major subgroups driven by specific mutations. We used to treat all these tumors the same, because they looked similar under the microscope. Now we know that molecularly they’re very different, so we’re developing specific therapies for each of the molecular subgroups.

“The PCGP has opened up a completely different concept of how to approach treatment now and in the coming years.”

William Evans, PharmD

Pharmaceutical Sciences
Former St. Jude president and CEO

William Evans, PharmD

“We thought sharing data from the Pediatric Cancer Genome Project was the best way to accelerate progress in curing childhood cancers. We thought that if we put that information in the hands of scientists and doctors around the world, it would advance their research and that would do the greatest good for the entire pediatric cancer research community. So, we announced at the start of the PCGP that we would share the data as soon as it passed our quality control—well before we actually used it for our own publications—and there are many examples of where this accelerated the research of other scientists.

“We think that’s really a legacy of this project—the impact it has had and continues to have on research globally, not just here at St. Jude.”

Ching-Hon Pui, MD

Oncology chair

Ching-Hon Pui, MD

“The Pediatric Cancer Genome Project has already revolutionized how we study cancer and find cures for previously incurable patients. This project has set the stage for true precision medicine.”

Kim Nichols, MD

Cancer Predisposition Division director

Kim Nichols, MD

“For me, one of the most surprising findings of the PCGP is that at least 1 in 10 children with cancer develops the disease due to an underlying disposition. These children may be at an increased risk to develop complications from the cancer treatment and to develop other cancers later in life. Some of these children may also pass the genetic condition to their future offspring.

“I predict that we will use the PCGP as a stepping stone to further explore the rest of the genome, to better understand why cancers form, and to use this information to improve upon the treatment of children with cancer.”

John Easton, PhD

Computational Biology Genomics Lab director

John Easton, PhD

“As part of this project, our validation laboratory confirmed hundreds of thousands of mutations. How many of those mutations directly drive cancer? As technology evolves, we’re asking those kinds of questions in a more detailed way.

“We’re already using PCGP data and applying it in ways we weren’t thinking about at the beginning of the project. In the coming years, we will continue to go back to this raw data to decipher perplexing samples from PCGP by employing more sophisticated analysis in combina-tion with new technologies.”

Charles Mullighan, MBBS, MD

Comprehensive Cancer Center deputy director

Charles Mullighan

“The Pediatric Cancer Genome Project has provided insights that will have a lasting impact for how we classify, diagnose and treat pediatric cancer. We’re now able to more precisely define cancer subtypes based on molecular genetics.

“The PCGP also changed the way testing is done, with less reliance on conventional pathology and more on molecular and sequencing approaches. We’ve developed tools and portals that allow anyone to mine the work of the PCGP—facilitating research worldwide. As we continue to mine the genome, our understanding of the genetic basis of cancer susceptibility will continue to grow—which will have a profound effect on diagnosis and management.”

Jinghui Zhang, PhD

Computational Biology chair

Jinghui Zhang, PhD

“PCGP has ushered in big-data science to pediatric cancer research. Discoveries made on the 23 cancer subtypes have helped set the direction for basic and translational research on pediatric cancer.

“The genomic data and the underlying analysis infrastructure provide a rich resource. That resource enables the design of pediatric-cancer precision medicine and continued data exploration.

“More than 400 research groups around the world have accessed the genomic data. So, PCGP serves as a role model for data sharing with the global research community. The goal is to engage everyone in finding cures for pediatric cancer.”

Michael Dyer, PhD

Developmental Neurobiology chair

Mike Dyer

“The thing that’s most amazing to me about the Pediatric Cancer Genome Project is the way it has exponentially expanded our research. For each of the discoveries from this project, there’s now a massive explosion in research into the basic mechanisms of cancer biology. Those discoveries will lead to new therapies that will improve the survival and quality of life for children with cancer.

“At St. Jude, children’s cancer genomes are now routinely sequenced. In some cases, that information is used to select a therapy for that particular child. So, the PCGP will continue to have an impact both in the laboratory and in the clinic.”

Suzanne Baker, PhD

Brain Tumor Research Division director

Suzanne Baker

“The Pediatric Cancer Genome Project opened exciting new avenues of research. We identified mutations, or mistakes in the DNA, in pediatric cancers that advanced understanding about how pediatric cancer forms and how we may develop more effective therapies.

“Our efforts mostly focused on the parts of the DNA that contain the code for making proteins in the cell. This is only a small proportion of all of the DNA in the genome. The data generated by the PCGP will be mined by scientists from St. Jude and around the world for many years to come as a rich resource to discover important disease-causing changes in parts of the pediatric cancer genome that we have not yet extensively explored.

“Importantly, we learned that epigenetic regulation, which does not change the sequence of our DNA, but changes the way that the code is packaged and read, is very important in pediatric cancer. This has resulted in an explosion of new research focused on rare pediatric cancers like childhood gliomas that were previously understudied.”

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