Thu. Dec 3rd, 2020

MEMPHIS, Tenn.–Tiny Sebastian Perez lies in intensive care battling pneumonia, a big concern because the 8-month-old’s immune system is so weakened. Since he was born in California’s San Fernando Valley, Sebastian has hardly spent any time outside a hospital. The diagnosis came within hours once doctors saw the moles on his skin: acute lymphoblastic leukemia, the most common childhood cancer and typically one of the most curable. Except in infants. Within a month, Sebastian was receiving round after round of chemotherapy, to no avail.

So the family came here to St. Jude Children’s Research Hospital hoping that a cutting-edge experimental treatment would turn the tide. A peek inside his leukemia cells revealed a rearrangement in a gene called MLL that results in an abnormal protein that spurs cancer cells. “That’s the key driver,” says Sebastian’s oncologist, Tanja Gruber. She and her colleagues have discovered that several drugs primarily used to treat a different cancer, multiple myeloma, seem to target the aberrant MLL protein, stopping it from wreaking its havoc. So far, about 90 percent of children with this type of leukemia who have tried these drugs have had a positive response. Sebastian’s drug cocktail has him in remission for the first time in his young life.

“You want your miracle to happen,” says his mom, Jesenia Gonzalez. “And we’re just heading in the right direction.” She has quit her job as a supervisor for a financial institution in California to be here, while her husband comes back and forth with their 8-year-old daughter. If he can beat the pneumonia and all else goes well, Sebastian will undergo a bone marrow transplant when he is stronger, weeks or months from now.

Photos: St. Jude Children’s Research Hospital

Madison Burt, 4, from Texas is being treated for medulloblastoma. Here she is pictured with Child Life Specialist, Ashley Anderst, CCLS at St. Jude Children’s Research Hospital

Sebastian’s treatment plan represents one of the newest strands of hope in managing pediatric cancer. Researchers and clinicians at St. Jude – the only National Cancer Institute-designated comprehensive cancer center dedicated solely to children – see many of the rarest and most difficult cases, mostly as part of research trials. Besides being on the forefront of explorations into drug treatments targeted to mutations, they are applying the latest ultraprecision radiation and novel therapies that rev up the body’s immune system – or a combination of all three – to tackle a disease that, all told, affects about 1 in 285 children in the U.S. before age 20.

The hospital cares for about 7,800 patients per year, absorbing all expenses that insurance doesn’t cover. Families never receive a bill, and travel, food and lodging for up to four people are provided, too. U.S. News spent several days visiting St. Jude early this summer.

When the hospital was founded in 1962 by entertainer Danny Thomas, the overall survival rate for childhood cancer was less than 20 percent. Today, some 80 percent of children in the U.S. survive, but the rate for some cancers is still below 50 percent, and the disease remains the leading cause of death from illness for kids in the nation. St. Jude’s success rate mirrors the country’s, even though many of its patients are among the sickest, says President and CEO James Downing; still, the losses are too “staggering.” About three-quarters of the funding that sustains St. Jude, its research and the family support comes from donations; the hospital’s fundraising and awareness organization is one of the largest charities in the U.S., bringing in $851 million in public contributions in fiscal year 2013.

“We’ve been blessed with a lot,” says Jason Burt, of Meridian, Texas, who has lived with his wife Jessica at Target House, St. Jude housing a few miles from campus, while their 5-year-old daughter Maddy has been receiving treatment. In February, after Maddy had been pained for some time by worsening headaches, vomiting and vision problems, her family learned she had a medulloblastoma, a brain tumor. When Maddy suddenly stopped breathing and fell into a coma, she was rushed in for surgery to remove the tumor at a hospital in Texas. In March, she came to St. Jude for several rounds of chemo.

This June day, Maddy and her parents are painting a poster decorated with the word “faith” in her room, guided by Ashley Anderst, one of 17 “child life specialists” on the staff. Each patient is assigned a primary specialist who gets to know the families, answers their questions and assists them in navigating the hospital. The child life specialists also engage their charges in play, art and music to normalize their days and to help them socialize.

The way St. Jude cares for families as well as patients was a pleasant “culture shock,” says Tom Roenspie of Orland, California, whose son Ty, 3, is here for several weeks of radiation and chemo for an ependymoma, a different type of brain tumor. Ty also gets regular physical and speech therapy, as he, mom Megan and 1-year-old brother Lane stay in St. Jude housing a short drive from the hospital with shared kitchens, common areas and playrooms. Lane, too, is fixed up with chocolate milk after Ty has radiation. “They cater to both of them,” says Roenspie.

This kind of enveloping care has helped Maddy get through her fourth and final cycle of chemo. She is responding well, says Amar Gajjar, her doctor and director of neuro-oncology. Next, she will undergo proton therapy, an ultratargeted radiation treatment that minimizes the exposure of other tissue. That’s especially important in a case like Maddy’s, since her brain has a lot of growing to do, he notes.

Conventional photon therapy goes “all the way through you,” says Thomas Merchant, chair of radiation oncology. With proton therapy, clinicians can almost “paint the radiation dose in layer by layer, spot by spot” with a high degree of accuracy. A 2012 review in the journal Radiation Oncology by Mayo Clinic researchers showed that treating medulloblastoma with proton therapy entails a risk of radiation-induced cancer that is 8 to 15 times lower than that associated with photon treatment. St. Jude’s own $90 million proton therapy center, the world’s first specifically designed for children, is expected to open later this year or early next. Maddy will go for proton therapy sooner, at the hospital’s University of Florida partner facility. 

Drugs like those that have put Sebastian Perez’s leukemia into remission are a key aim of the Pediatric Cancer Genome Project, a St. Jude initiative with Washington University in St. Louis that has so far entailed sequencing the entire genomes of some 700 young patients since 2010. While oncologists across the country are increasingly testing patients for certain specific mutations that might respond to targeted treatments, the St. Jude-Wash U effort provides a far wider window into what might have gone wrong in a patient’s DNA by exhaustively comparing the genetics of a child’s cancerous and healthy cells. Today, it’s an effort “unparalleled across the world,” Downing says. But the process “will ultimately become part of the routine” in cancer care. This summer, St. Jude begins sequencing the genomes of some cancer patients as they arrive, with the goal of eventually doing so for everyone.

The project has already borne fruit. In 2012, for example, researchers discovered the mechanism that causes the eye cancer retinoblastoma to develop rapidly by examining the genomes of several St. Jude patients. Work is underway to develop a treatment that has shown success in the lab at blocking that mechanism. Researchers also have identified 41 genes associated with medulloblastoma, some of which are already being investigated as targets for drug development.

Inside a playroom filled with puzzles, books and toys, Mack Ward, 6, practices changing the bandage on the chest of a bald medical doll. “You probably use something like that [bandage] on your line,” says child life specialist Ashley Wedderburn, referring to the catheter into his chest through which he receives IV chemotherapy. The Rogersville, Tennessee, native was diagnosed late last year with Ewing sarcoma, a type of bone cancer, after treatment for a misdiagnosed bone infection. He and his family arrived on Christmas Eve, with Mack’s kidneys and liver starting to fail. St. Jude doctors stabilized him and started chemotherapy. Now his tumor is shrinking. “He’s had a very, very good response,” says oncologist Sara Federico.

Once chemo is finished this summer, surgery will be scheduled to remove the tumor, which has invaded more than half of his shinbone. The family lives in Target House while his father, Ryan, takes a break from his job as a mechanic; each month, one of Mack’s three siblings, who live with friends or relatives, comes to visit. Two or three times a week, around treatments, Mack meets with a teacher in a classroom at the nearby Patient Care Center. St. Jude’s school program employs six K-12 teachers to help the children stay on track academically and developmentally as it coordinates lessons with their home schools. “We are not here to treat just a child’s illness,” says Laurie Leigh, the program’s director.

On the playground behind Target House, Ana Rogulic, 3, and her brother Branko, 8, play with their father, Jovan, as they prepare to say their goodbyes and head back home to Serbia. In early 2014, Ana was diagnosed with high-risk neuroblastoma, a cancer of the nerve cells that kills 50 percent to 60 percent of patients. Ana’s mother, Bojana, learned of an experimental treatment here, and the family arrived that March.

Along with chemotherapy and a bone marrow transplant, Ana’s treatment has involved a groundbreaking approach to battling tumors called immunotherapy. She received infusions of an antibody that prompts the body’s immune system to recognize and attack cancer cells. Earlier this year, the Food and Drug Administration approved an antibody drug called Unituxin after trials showed it to be effective against neuroblastoma. Ana’s particular antibody treatment has been engineered to reduce allergic reactions and pain, significant side effects, notes her oncologist, Wayne Furman. Ana has responded well: Her cancer and pain are gone.

Over the next year, Ana will return to St. Jude every few months for checkups, then at least once a year for the next five. Such visits are a key part of the hospital’s strategy to be ready with a new plan of attack should a cancer return, as well as to get a handle on the long-term effects of treatment. “Our patients have been our collaborators for years,” says oncologist Melissa Hudson, who directs the cancer survivorship division. Thousands of people treated for childhood cancer at St. Jude have returned over the years for comprehensive evaluations, paid for in part by the hospital.

“It’s like a homecoming for me,” says Jason Witt, 35, of Southaven, Mississippi, located about 12 miles away. Jason was treated for leukemia at St. Jude as a teenager. The high school finance and accounting teacher has returned several times, and he even met his wife, a St. Jude nurse, when he was volunteering at the hospital.

Josué Fernando Mendizábal is a fighter. An avid boxer in his native Guatemala, the 16-year-old “is a little bit tired today,” notes oncologist Raul Ribeiro as he examines him. Josué was diagnosed about a year ago with acute myeloid leukemia, a cancer of the blood cells. Standard chemotherapy treatment in his home country wasn’t proving effective, so Josué was referred to St. Jude and put on an experimental regimen involving several drugs, including one that appears to boost the action of tumor suppression proteins and stop cancer cells from growing.

When he arrived, about 80 percent of Josué’s white blood cells were myeloblasts, immature cells that form in the bone marrow and disrupt the normal production of healthy blood cells. After two weeks on the drugs, Josué’s blasts were down to about 28 percent, a marked improvement but not enough to safely perform a bone marrow transplant, his best shot at a cure. Ribeiro gave him another drug combination that eventually reduced Josué’s percentage of blasts to under 1 percent. “We can give very intense chemotherapy,” supporting the patient through it, Ribeiro says. (Josué eventually underwent a bone marrow transplant and will stay at St. Jude for several months to be sure it worked.) “When I was in Guatemala, I thought everything was over,” Josué says through an interpreter. “People should always fight.”

Paishence Joi Watkins has been fighting since 2012, when the then-8-year-old was diagnosed with osteosarcoma in her right femur. After several rounds of chemo and a surgery to replace cancerous bone with a titanium rod, she’s back from Lafayette, Indiana, for a limb-lengthening procedure. Clinicians surround her leg with a high-powered electromagnet that heats and expands the rod a few millimeters so her right leg can grow along with her left. Paishence, now 12, has been cancer-free for more than two years.

She comes back for the procedure about once a month, taking time to catch up with doctors and nurses and play air hockey with her mom or other kids. With the future looking bright, coming back is a treat. “I look forward to it,” she says.

Excerpted from U.S. News’ “Best Hospitals 2016,” the definitive consumer guidebook to U.S. hospitals. Order your copy now.

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