SOME EXCITING NEW PROJECTS ARE POPPING UP at St. Jude Children’s Research Hospital. They’re audacious in scope. Optimistic. Filled with wonder and hope, creativity and grit.
Since its founding in 1962, St. Jude clinicians and researchers have asked and answered “what-if” questions that have changed the landscape of childhood catastrophic diseases. Today, the hospital is guided by a strategic plan that outlines a bold strategy for the future. But other opportunities arise constantly. Technologies evolve. Priorities expand.
What if there were a way to identify and pursue bold new projects that could further transform science and medicine?
Now there is. It’s called the blue-sky process.
“We want to challenge people to come up with new ideas that have a game-changing impact,” explains James R. Downing, MD, St. Jude president and chief executive officer. “We want to look beyond the priorities of our strategic plan to identify unique opportunities that will accelerate our mission-critical objectives and test novel scientific and clinical approaches.”
All faculty and staff members can submit blue-sky ideas. Proposals that are deemed innovative and impactful receive funding, and the work begins. Below, you’ll get a glimpse of seven questions we’re working to answer.
What if … we could prevent adult cancers by vaccinating children?
DID YOU KNOW 1 out of every 4 people in America is infected with a virus that causes cancer? Each year, more than 31,000 individuals are diagnosed with cancer caused by the human papillomavirus (HPV).
But wait — There’s good news. A vaccine is available. When given to boys and girls in their early teens, the two-part vaccine helps them avoid infection and subsequent cancer development.
Despite that simple fix, HPV vaccination remains low. In fact, the nation’s lowest immunization rates and highest number of HPV-related cancers occur in the region where St. Jude is located.
“Pediatric cancers can’t be prevented, but we can reduce the risk of preventable cancers later in life,” says Charles Roberts, MD, PhD, director of the St. Jude Comprehensive Cancer Center. “Our goal is to reduce cancer deaths by establishing a premier HPV prevention initiative.”
This blue-sky project features collaborations with local, regional and national partners, as well as state and federal policy changes. Through the HPV cancer prevention blue-sky initiative, St. Jude will help lead the way in increasing vaccination rates.
“Together, we can help children eliminate their risk of HPV-associated cancers,” Roberts says.
What if … we could ease the transition to survivorship?
NO MORE CHEMO parties at St. Jude are joyful celebrations for patients completing treatment. But what happens after the confetti falls? For many families, the time of transition away from daily hospital visits is stressful, even scary.
“How will my child re-adjust to school?”
“Does a bruise or sniffle mean the cancer has returned?”
“What happens if I have a question in the months before our follow-up visit?”
The Transition Oncology Program (TOP) provides a safety net for patients who have finished treatment but are not yet eligible to enroll in the After Completion of Therapy (ACT) clinic, which generally occurs five years after diagnosis. Thanks to TOP, patients receive an extra layer of support from a multidisciplinary team.
“If you think of the primary oncology teams as the active cancer specialists, and the ACT team as the long-term survivor specialists, then you can think about the TOP team as being specialists for that in-between time of transition and uncertainty,” explains the program’s director, Emily Browne, DNP. “We have social workers, psychologists, nurse practitioners, school liaisons, and rehabilitation specialists ready to assess and address patients’ physical, cognitive and psychosocial concerns.”
What if … we could give children worldwide a fighting chance for survival?
ONE CHILD LIVES in the U.S., while another hails from a developing nation. Which of those children would be more likely to survive a bout with cancer? Too often, the answer depends on where the child lives. Worldwide, more than 80% of children with cancer live in low- and middle-income countries. Most of those children will die from their diseases.
A program called St. Jude Global has already been making strides to improve survival rates. But what if St. Jude teamed up with World Health Organization (WHO) to turbo-charge that effort?
Thanks to a blue-sky initiative, that’s what has happened. The result is the WHO Global Initiative for Childhood Cancer. As St. Jude works bottom-up with childhood cancer providers globally, WHO works top-down with governments, civil society and health-system leaders. St. Jude and WHO staff will analyze policies, medical workforce capacity and infrastructure. Together with other global stakeholders, they will develop and implement plans to improve the care for children with cancer around the world.
“This initiative unites health care providers, advocates and researchers from around the globe,” says Carlos Rodriguez-Galindo, MD, Global Pediatric Medicine chair. “It marks the start of a new era in the worldwide fight for the lives of children with cancer.”
What if … we freely share discoveries with even more scientists?
WHEN ST. JUDE and Washington University launched the Pediatric Cancer Genome Project in 2010, the goal was to share data from that project with researchers worldwide.
In 2018, St. Jude launched an online data-sharing and collaboration platform called St. Jude Cloud. Suddenly, researchers had access to the world’s largest public collection of childhood cancer genomics data. Researchers across the globe accessed the information and tools and performed computational analyses. But for many scientists, the process was cumbersome and time consuming — requiring significant computing resources and expertise to seamlessly download the data.
Thanks to the blue-sky process, St. Jude is enhancing the cloud infrastructure, tools and datasets. With more than 10,000 genomes, St. Jude Cloud holds more childhood cancer data than any other repository. The cloud is now user friendly for members of the scientific community.
“The design of St. Jude Cloud from its inception was to create a site that was not only useful for individuals with advanced computing skills, but that could be effectively used by clinicians and other researchers who might have limited computer skills,” says Jinghui Zhang, PhD, St. Jude Computational Biology chair. “We think we’ve been successful in developing such a site.”
What if … we find a new way to model pediatric diseases?
SCIENTISTS NOW HAVE a new way to model pediatric diseases — to study aspects of normal human biology and better determine how childhood diseases develop.
It starts when a patient or family member donates blood or skin cells. In the lab, scientists convert those cells into an immature state called induced pluripotent stem cells, or iPSCs. Researchers then guide the cells to differentiate into cell and organ-like structures that resemble various body tissues.
A blue-sky project will create a St. Jude core facility that uses samples donated by St. Jude patients to develop models for studying childhood cancers and neurological disorders. The models can also be used to assess how tumor-specific genetic mutations respond to new therapies. Scientists will use these models to assess the impact of gene-editing technologies on diseases caused by single gene defects, such as sickle cell disease.
“The generated iPSC cells and resulting models will be a shared resource for St. Jude investigators, but it’s really for researchers around the globe,” explains Michael Dyer, PhD, St. Jude Developmental Neurobiology chair and Howard Hughes Medical Institute investigator. “By freely sharing these models, we aim to accelerate research worldwide.”
What if … we could save the lives of kids with hemophilia worldwide?
CHILDREN WITH hemophilia B can have life-threatening bleeds because they lack a protein that causes the blood to clot normally. In the U.S., children with the severe form of the disease take protein-replacement therapy, which can cost about $1 million over their lifetimes.
In resource-challenged countries, most patients cannot afford this therapy, suffering severe bleeds that cause deformities and death.
How can we help those children?
At St. Jude, researchers have developed a gene therapy that can significantly decrease or eliminate patients’ need for replacement therapy.
What if we could use this single-injection therapy for children around the world? St. Jude researchers have a blue-sky plan. It involves a feasibility study in low- and middle-income countries. A positive outcome could lead to a public-private partnership that might save the lives of children around the globe.
“We need to develop the protocol and get it approved, make sure the countries chosen have the right regulatory infrastructures and that the institutions have adequate support to monitor the patients,” says Ulrike Reiss, MD, of St. Jude Hematology. “It’s an exciting project with far-reaching possibilities.”
What if … we could understand the biology of neurologic disorders?
FOR DECADES, St. Jude clinicians and researchers have sought cures for catastrophic diseases such as cancer, sickle cell and HIV. But what about children with neurologic disorders? About 1 in 6 children has some form of neurologic disability — ranging from neurodevelopmental or movement disorders to neuromuscular diseases. Early intervention can prevent or slow the progression of these diseases.
The Center for Translational Neuroscience will build on a strong foundation of discovery science in cell biology and neuroscience and add additional faculty, projects and research capabilities to push the frontier in neurological diseases. As scientists work with clinicians to move these discoveries into the clinic, they will accelerate the pace of clinical trials and implement new cutting-edge therapies.
“We are expanding the scope of catastrophic pediatric diseases to be addressed by St. Jude,” says J. Paul Taylor, MD, PhD, St. Jude Cell and Molecular Biology chair and Howard Hughes Medical Institute investigator. “Neurological diseases are among the greatest burden of catastrophic illness in children, and we are addressing this head on.”
From Promise, Autumn 2019