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The stakes are high, and time is of the essence. Every day, children worldwide are dying of cancer and other life-threatening diseases. Who has the intellect, the drive and the know-how to save them? The problem is too great for governments or international philanthropic organizations to handle alone.

So two years ago, St. Jude Children’s Research Hospital stepped into the gap. The hospital’s president and chief executive officer, James R. Downing, MD, announced a six-year, $7 billion strategy to accelerate progress toward curing childhood cancer.

Eager to accept that challenge, the hospital’s faculty and staff embraced the task. As they can attest, progress comes quickly at St. Jude

Race against time

The first task Downing faced was recruiting visionary leaders in areas such as financial operations, clinical efforts, strategic planning, information services, global outreach and the Comprehensive Cancer Center.

“It’s important to have people who understand the culture of St. Jude, who know our history and recognize that special magic that makes St. Jude what it is,” he explains. “We’ve been able to recruit seasoned leaders who bring new energy, offer new perspectives, and pose new ways of solving problems. We’re able to draw incredible people to this institution. That’s because of who we are—our workforce and our mission. People want to be a part of that.”

The leadership team immediately set to work, creating new programs and ensuring that employees know their roles in accomplishing the hospital’s mission. That dynamic has enabled Downing to begin checking tasks off the plan’s to-do list. Because, after all, there’s no time to lose. 

Phenomenal precision

One of the earliest accomplishments involved establishing the world’s first proton therapy center designed solely for children. The St. Jude Proton Therapy Center enables scientists to define the best way to use this treatment, which propels sub-atomic particles into tumors while sparing healthy tissue. More than 150 children have undergone proton therapy at St. Jude thus far.

Weiguang Yao, PhD examines the proton therapy machine.

In the world’s first proton therapy center designed solely for children, scientists such as Weiguang Yao, PhD, are defining the best way to harness this life-saving technology.

The hospital has also launched new precision medicine efforts, many of which have their genesis in the St. Jude – Washington University Pediatric Cancer Genome Project, which compared the genetic blueprint of cancerous and normal cells from more than 800 children. That project revealed important details about a host of childhood cancers.

During the past couple of years, the hospital has begun moving whole-genome sequencing to the clinic. A study called Genomes for Kids evaluated the feasibility, acceptance and impact of using genomic sequencing for children with cancer.

St. Jude created a Cancer Predisposition Program to work with families whose children may have genetic lesions in their normal tissues that significantly increase their risk of developing cancer.

The hospital’s clinical genomics service incorporates specialists from across the institution, all dedicated to using precision medicine to save lives. This service is already being used in front-line therapy through the hospital’s newest clinical trial for leukemia and lymphoma.

Another precision medicine effort includes a project to sequence the genes of 3,000 survivors as part of St. Jude LIFE, a long-term follow-up study. Accomplished in 18 months, the sequencing effort uncovered germline mutations in cancer predisposition genes in long-term survivors of cancer. The study also shed light on the risk for second malignancies in that population.

A similar initiative involved sequencing the genes of 800 patients with sickle cell disease. By evaluating results of that study, scientists hope to learn why some patients live long lives while others die early or suffer from severe complications. Discoveries will provide new targets for potential cures.

Patients first

St. Jude staff are always on the lookout for new ways to reduce the stress experienced by patient families. Last year, the hospital completed construction of state-of-the-art inpatient units designed to entice patients to leave their hospital rooms for exercise, fellowship and mental stimulation. 

Patient Mabry plays with an interactive wall installation in the halls of St. Jude.

The hospital’s new inpatient units entice patients such as Mabry Landstreet to leave their hospital rooms for exercise and exploration. 

“Those amazing facilities are providing a level of inpatient care and experience that are unparalleled in the United States,” Downing says.

Outpatients now have enhanced housing options, with an upgrade of Tri Delta Place for short-term stays.

The new Patient Experience Office is also creating a host of additional perks for families, including a babysitting service, Zipcars and a town-square concept that simulates a small-town environment.

“When you think about a town square, you envision an area that may have a school and a church nearby, as well as a little store where you can get cookies and coffee and ice cream,” Downing says. “We’re going to convert the second floor of our Patient Care Center into a town square for such amenities as the School Program, chapel, concierge desk, post office and bank. It will all be in one concentrated area for our families.”

Training tomorrow’s scientists today

This fall, St. Jude welcomes the inaugural class of the St. Jude Graduate School of Biomedical Sciences. The PhD-granting program will reflect the hospital’s mission and will provide a distinct educational experience for young scientists. Graduate students will inject energy and new ideas into clinics and labs across campus.

Miguela Caniza, MD, and Brennan Bergeron

Miguela Caniza, MD, of Global Pediatric Medicine, works with Brennan Bergeron, one of the first students in the St. Jude Graduate School of Biomedical Sciences.

“It fills a niche we think we can fill better than anybody,” Downing says. “In an institution where translational medicine has been at the core since Day One, these individuals will get a unique training experience that will develop a cohort of scientists who will go out and influence how science is done.”

Uniting the world’s top minds

St. Jude already collaborates on projects with institutions around the world. These studies generate discoveries that save untold lives worldwide. But time is of the essence. Cures must be accelerated. What if the hospital brought together the world’s top minds to create new, St. Jude–funded clinical trials and research projects to help find such cures?

The hospital is developing a clinical research consortium to generate clinical trials for pediatric cancers, non-malignant hematological diseases and other life-threatening disorders. Some of the planet’s top pediatric programs are being invited to join this effort.

 “These new clinical trials will not replace the collaborative protocols we develop and run,” Downing says. “We will continue to run our own protocols. But someone at another institution may have an idea that no one’s ever considered. The group will design the study, and it will be funded through this effort.”

Similarly, a St. Jude research collaborative will identify the best talent around the globe to work as part of an international team dedicated to answering previously unanswered scientific questions. The first project in that program is already underway. World-renowned scientists gathered in May 2017 to explore how cells organize specific contents into functional compartments called liquid organelles. By better understanding the biology of liquid organelles, scientists can make progress in eradicating diseases such as cancer, immune dysfunction and neurodegeneration.  

J. Paul Taylor, MD, PhD; Cliff Brangwynne, PhD; Rohit Pappu, PhD

As part of the hospital’s new research collaborative, J. Paul Taylor, MD, PhD (left), St. Jude Cell and Molecular Biology chair, confers with Cliff Brangwynne, PhD, of Princeton University and Rohit Pappu, PhD, of Washington University in St. Louis.

Mobilizing the world

Perhaps the most striking accomplishment thus far is the establishment of St. Jude Global, a major initiative to restructure and expand the hospital’s reach. The goal of St. Jude Global is to create a network of interactive programs and institutions worldwide so that every child with cancer has access to quality care.

“In the past, our international outreach efforts were humanitarian in nature, but it wasn’t a strategic, goal-oriented effort,” Downing explains.

“This is really a change in focus. It’s about building a workforce. It’s about establishing consortia of institutions, and then teaching them how to do clinical research so they can continually advance the level of care they provide to children within those regions. And then it’s about working across those regions around the globe so that we all learn from each other.”

This massive effort is moving quickly and generating excitement. For the first time ever, the Association of Pediatric Hematologists and Oncologists of Central America held its annual meeting in the U.S.—at St. Jude.

The St. Jude Global Academy also held its first course for clinicians who deal with pediatric cancer. The eight-week training course for infectious disease specialists from Latin America culminated in an intensive workshop held on the hospital’s campus. 

Progress expedited

More dramatic advances should occur during the next few years, as the plan continues to unfold. New buildings and programs will be added to support the research and clinical care necessary to reach the hospital’s goals.

Downing says St. Jude donors should take pride in the part they have already played in saving the lives of children around the globe. Continued support is needed to share the hospital’s life-saving mission worldwide.

“We owe a debt of gratitude to our dedicated donors who believe in our ambitious plan to eradicate cancer and other devastating diseases of childhood,” he says.

 “Who’s going to step up and accelerate progress? Who’s going to mobilize the world and get them to work together? We think St. Jude—with the support of our donors—can do it better than anybody.”

So the plan proceeds apace—because time is of the essence. 

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