Blue-Sky Projects Accelerate Progress

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.”

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.”

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.”

From Promise, Autumn 2019