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St. Jude Children's Research Hospital

by St. Jude Children's Research Hospital
St. Jude Children's Research Hospital
St. Jude Children's Research Hospital
St. Jude Children's Research Hospital
St. Jude Children's Research Hospital
St. Jude Children's Research Hospital
St. Jude Children's Research Hospital
St. Jude Children's Research Hospital
St. Jude Children's Research Hospital
St. Jude Children's Research Hospital
St. Jude Children's Research Hospital
St. Jude Children's Research Hospital
St. Jude Children's Research Hospital
St. Jude Children's Research Hospital
St. Jude Children's Research Hospital
Dr. Pui
Dr. Pui

St. Jude Children’s Research Hospital investigators report that measuring leukemia cells that persist in patient bone marrow in the early weeks of treatment helps identify high-risk patients who need intensive therapy.

Measuring the concentration of leukemia cells in patient bone marrow during the first 46 days of chemotherapy should help boost survival of young leukemia patients by better matching patients with the right intensity of chemotherapy. St. Jude Children’s Research Hospital investigators led the research, which appears in the March 20 edition of the journal Lancet Oncology.

The findings stem from a study of 498 children and adolescents with acute lymphoblastic leukemia (ALL) enrolled in a St. Jude-led protocol between 2000 and 2007. The clinical trial was the first to use measurement of residual leukemia cells – or minimal residual disease (MRD) – in bone marrow to help guide therapy. St. Jude pioneered MRD measurement as a tool to guide leukemia treatment.

“This analysis shows that MRD-directed therapy clearly contributed to the unprecedented high rates of long-term survival that patients in this study achieved,” said first and corresponding author Ching-Hon Pui, M.D., chair of the St.Jude Department of Oncology. Overall, 93.5 percent of patients were alive five years after their cancer was diagnosed. “MRD proved to be a powerful way to identify high-risk patients who needed more intensive therapy and helped us avoid over-treatment of low-risk patients by reducing their exposure to chemotherapy,” Pui said.

Researchers hope the findings will expand use of MRD measurements to guide leukemia treatment in children and adults.

The technique might also help identify patients who could be cured with less intensive chemotherapy, Pui said. Overall long-term survival was 97.9 percent or better for 244 patients in this study classified as low risk based on a variety of factors including their age at diagnosis and MRD of less than 1 percent on day 19 of treatment. “Given the excellent outcome, it will be important to determine if treatment can be further reduced in this subgroup of patients,” Pui said.

In countries with limited resources, Pui said the findings suggest that results of MRD on day 19 can be used to reduce treatment-related deaths by identifying patients who will likely be cured with low-intensity chemotherapy. “This study demonstrates these patients have an extremely low risk of relapse,” he said.

The study showed that measuring MRD just twice during remission induction therapy – at day 19 and day 46 – rather than multiple times during the more than two years of treatment was sufficient to guide treatment of most pediatric ALL patients. That will help save money and protect patients from the discomfort and risks associated with bone marrow aspiration for MRD testing. MRD measurements should continue, however, to guide treatment of patients with detectable MRD on day 46 of treatment. That is a level of 0.01 percent or more, which translates into one leukemia cell in 10,000 normal cells.

MRD was not a perfect predictor of relapse risk. Cancer returned in 26 of the 430 patients with undetectable MRD when treatment ended after 120 weeks. Researchers are working to develop even more sensitive methods for tracking treatment response in order to identify those at risk for having their cancer return.

Overall, researchers showed that regardless of other risk factors, including age at diagnosis or the initial white blood cell count, patients with an MRD level of 1 percent or more on day 19 of therapy were far less likely than other young leukemia patients to be alive and cancer-free 10 years later. Having detectable leukemia cells on day 46 of treatment was also associated with lower survival.

MRD levels on days 19 and 46 led to the reclassification of 50 patients from low risk to a higher risk leukemia that warranted more intensive therapy. Researchers credited the change with boosting survival.

The other authors are Deqing Pei, Sima Jeha, Cheng Cheng, John Sandlund, Raul Ribeiro, Jeffrey Rubnitz, Hiroto Inaba, Deepa Bhojwani, Tanja Gruber, Wing Leung, James R. Downing, William Evans and Mary Relling, all of St. Jude; Elaine Coustan-Smith and Dario Campana, both of the National University of Singapore; and W. Paul Bowman, of the University of North Texas Health Sciences Center, Fort Worth, Texas.

The research was funded in part by grants (CA21765, CA36401, GM92666) from the National Institutes of Health and ALSAC.

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St. Jude Patient, Jamill
St. Jude Patient, Jamill

St. Jude wants to make sure that Jamill keeps a smile on his face. 

Jamill’s mom describes him as mellow and sweet. This 4-year-old loves riding his bike and going to basketball games. The first few years of his life have been difficult due to sickle cell disease, but St. Jude Children’s Research Hospital has been there every step of the way to ease his pain and keep him as healthy as possible.

When Jamill was born, tests revealed he suffered from sickle beta thalassemia disease, a subtype of sickle cell disease. Sickle cell disease is a genetic blood disorder that affects the hemoglobin within the red blood cells, causing the cells to become hard and form a sickle shape. The sickle shaped cells can cause pain, organ damage or stroke.

St. Jude has a deep and longstanding commitment to children with sickle cell disease. Scientists at the hospital have been researching the disease since the institution opened in 1962. With more than 750 patients in its sickle cell program, St. Jude remains one of the leaders in the crusade to spare children the suffering this chronic disease causes.

Little Jamill has experienced pain crises and high fevers associated with sickle cell disease, often needing to be hospitalized for days at a time. “As soon as I call and let St. Jude know we are on the way, they get Jamill’s room ready,” said his mom, Tanshika. “I know they are taking really good care of him.”

Jamill’s treatment at St. Jude includes daily penicillin and any additional care he might need to manage symptoms of the disease. He visits St. Jude every three months for routine checkups.

“Jamill’s pain crises have occurred less often as he gets older and continues treatment,” Tanshika explained. “I really like everyone at St. Jude. We’ve always been well taken care of thanks to the doctors and all of the staff.”

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Music Therapist Amy holds a session with patients
Music Therapist Amy holds a session with patients

At St. Jude, music therapy empowers children, helping them meet developmental goals while providing an outlet for expression.

With an elfin grin, Kyle approaches the piano, eager to tickle the ivories in preparation for his first recital.

Nervous?

“Oh, no!” he declares, dismissing the question with a quick shake of the head and flip of the hand.

“Him?” his mother, Chasity, teases. “He loves the attention.”

Weeks before the musical event in a St. Jude auditorium—invitation only, and everybody who knows and loves Kyle is invited—he is rehearsing his selections and preparing his wardrobe.

“He has a new blue shirt and tie,” announces his mom, eyes twinkling.

“And…tell what kind,” he prompts her.

“A big-boy tie; no clip-on.”

“Yep, that’s right!” he affirms, delighted.

At 7, Kyle possesses the confidence and tolerance he needs to take on not only the keyboard, but also the cancer treatment he’s undergoing at St. Jude Children’s Research Hospital.

He arrived in February from Louisiana and, to him, it was important to continue his music. Kyle had begun piano lessons in October 2013 and had started memorizing recital pieces.

But cancer interrupted the plan.

“He was upset,” Chasity says, “because he would miss his spring recital.”

When Kyle arrived at St. Jude, he met music therapist Amy. Kyle told her of his appreciation for piano; she immediately added an electronic keyboard to her collection of instruments. The more Kyle listened and learned and practiced, the more he hoped for a recital.


Hitting the right note

“Music is therapeutic,” Love explains. “Research shows that music is the only thing that lights up all of our brain at once. For kids like Kyle, music helps build fine motor skills, improves concentration and task orientation, and assists with reading and learning a language.

And music gives Kyle something to celebrate,” Love adds.

Kyle is being treated for a rare combination of germ cell tumors: germinoma and embryonal carcinoma. As part of his treatment, Kyle has undergone brain surgery, spinal surgery, chemotherapy and radiation therapy.

“He’s had minimal sickness. Chemo hasn’t slowed him down one little bit,” his mom reports, “but he did get upset thinking he was going to have to miss a year’s worth of piano.”

“I had already picked my song, ‘Swans on the Lake,’ for my recital,” Kyle adds.

“So they came up with the idea of a recital here,” Watson says. “While we’re in Memphis, Kyle particularly looks forward to seeing Miss Amy every week.”

Rhyme, rhythm and song

Kyle is not the only patient who has fallen for Amy. She makes her rounds, guitar strapped to her back, pushcart in tow, piled with drums and ukuleles; little xylophones and jingly bells on bracelets; even a cylinder that, when shaken, sounds like a thunderstorm. Amy’s ever-present electronic tablet contains song lists.

As she passes through the corridors, children grin, parents nod and, by the dozens, she calls them by name.

Entering a patient’s room, she greets a dour little face and gently announces, “I’m glad to be with you today, and I’m really glad for all the music we will play.”

A silent boy reaches out a slim arm for the drum she holds, the mallets and noisemakers she offers.

“Which song would you like today?”


Voices and choices

Amy says she enjoys offering choices to children who may not feel that they have control over other aspects of their life.

“Music is a way of releasing a lot of tension in your body. People sing at weddings, funerals, graduations, on the radio, so why not in the hospital? You should have access to ways to express yourself—a music voice,” Amy says.

The boy in the bed chooses the drum, the song, and, as it turns out, the meter.

“Kids aren’t always feeling well, but sometimes they have a lot of energy they have to get out of their bodies,” Amy says.

As she sings, the boy beats out the rhythm—moderate, then slow; fast, then ridiculously fast; ridiculously slow, and back and forth. The patient grins at his own mischief with music. And the two musicians laugh together.


Heart, mind, spirit

“Sometimes we use music therapy to promote physical goals,” Amy explains. “You can use a steady pulse of music as a rhythmic cue for walking a specific pace or for learning a certain phrase with intonation, because the neuro-pathway for music is different than the one for speech.

“I may go into a patient’s room and the child is anxious,” Amy continues. “I match the tempo of my guitar picking to the child’s heart rate. As I slow the tempo, the child’s oxygen saturation may increase and the blood pressure may decrease.”

Music, she says, also has a place in legacy building.

“We’re rolling out heartbeat therapy,” Amy says. “We can put a microphone in a stethoscope, record the child’s heartbeat and create and perform a song over it, making that heartbeat into a beautiful memory.”

At regular sing- and play-a-longs in the Kay Kafe lobby for kids and adults who happen by, Amy is prepared when a little girl begins to cry: Another child has the instrument she wants. Amy pulls out jingly bell bracelets, and harmony is restored.

Harmony of spirit. Harmony of sound. Budding pianist Kyle understands.

“I love all the harmonies,” Kyle says. “When I watch a movie or listen to music, I notice the harmonies. I notice when the singing starts. I can pick out the instruments, especially the piano.

“You know, I’m having a recital in the auditorium. Would you like to come? I’m playing three songs: ‘Swans on the Lake,’ ‘Jesus Loves Me’ and ‘Let It Go.’ I can’t wait.”

What is music therapy?
A clinical and evidence-based practice, music therapy can be used to achieve both musical and non-musical goals. One musical goal for a patient such as Kyle might be learning to play piano. A non-musical goal might be using drums to improve motor skills. Music therapist Amy also helps patients attain emotional goals, such as learning to express themselves by writing and performing songs and discussing song lyrics. Creativity also helps patients cope with disease and its treatment.

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St. Jude - Finding Cures. Saving Children.
St. Jude - Finding Cures. Saving Children.

When St. Jude opened its doors in 1962, the medical community saw many childhood cancers as incurable.

We didn’t listen.

And today, thanks to the research of St. Jude scientists and the care of St. Jude doctors, there are thousands of families that are very glad we didn’t listen.

Our work at St. Jude has helped push the survival rates for childhood cancer from 20% to 80%. And that’s really good news.

But we want great news. And great news will be when we cure all kids.

In the next decade our goal is to increase the survival rate of childhood cancers to 90%, and we will keep pushing until we get to that incredible day when cancer doesn’t claim the life of any child.

You can help us get there. Make a gift today and you’ll be helping provide us with the needed tools to conquer childhood cancer.

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St. Jude surgeons are world renowned for innovative approaches that save the lives of children with cancer and other diseases.

Mick’el was 15 months old when his grandmother noticed that he had blood in his diaper and a firm, bloated belly. After examining the toddler, doctors discovered that Mick’el had advanced bilateral Wilms tumor, a cancer of both kidneys.

“I was shocked. Scared,” says Mick’el’s father, Tommie.

When chemotherapy and radiation failed to shrink the tumors, doctors at their local hospital announced there was nothing more they could do.

“I didn’t want to sit on the front row at a funeral,” recalls Mick’el’s mom, Tameka.

 The family requested a second opinion, and within days Mick’el was under the skilled care of Andrew Davidoff, MD, Surgery chair at St. Jude Children’s Research Hospital. The renowned surgeon is an expert in bilateral Wilms tumor.

Davidoff and his colleagues perform about 1,600 operations each year to diagnose and treat cancer and other life-threatening diseases. They partner with St. Jude oncologists, radiation therapists, pathologists, anesthesiologists, nurses and basic scientists to plan and deliver the best treatment for children with solid tumors. Their specific expertise includes complex surgical procedures to treat kidney, neuroendocrine, eye and bone cancers.

“In addition to the technical aspects of these procedures, understanding what is appropriate for each patient is even more important in children with cancer,” Davidoff says. “It means knowing when surgery is appropriate and when it is not. If surgery is appropriate, is the best timing before or after chemotherapy? How aggressive or conservative should we be with the surgery, and is surgery the only way to cure the cancer?”

Preserving kidney function

Bilateral Wilms tumor occurs in only 30 U.S. children each year. Nearly 20 percent of those children have their surgery performed at St. Jude.

“Because of our extensive experience surgically managing children with cancer, I feel we have a more aggressive willingness to perform complex surgical procedures rather than saying they can’t be done,” Davidoff says. “While most other surgeons would remove one or even both kidneys, our approach is to save as much normal kidney on both sides when possible. That’s where experience counts.”

 Not only does this approach, called nephron-sparing surgery, maintain excellent survival rates, but it also helps children with this rare cancer avoid kidney failure, dialysis and transplantation. The No. 1 goal is always cure, but improving quality of life is a priority as well.

Davidoff surgically removed a softball-sized mass from Mick’el’s left kidney and another the size of a tennis ball from his right kidney. Today, the active 9-year-old is five years cancer-free with normal kidney function.

Davidoff has performed more than 50 such surgeries and publishes the results so the medical community can learn from the St. Jude model. Early results from cases like Mick’el’s show promise not only for cures but for avoiding complications after surgery.

Experience and expertise

Neuroblastoma is the most common solid tumor in children next to brain tumors and accounts for 7 to 10 percent of all childhood cancers in the U.S. By the time neuroblastoma is diagnosed, the cancer has usually spread to other parts of the body. Outcomes for the disease vary widely.

“In some children, the disease can be very non-aggressive,” Davidoff says. “We remove the tumor with surgery, and the children are cured. Others with advanced disease may require surgery, chemotherapy, radiation and bone marrow transplantation and still the survival rate is only 30 percent.”

When venturing into this complex territory, St. Jude surgeons meticulously separate the tumor from delicate blood vessels that nourish the kidneys, liver, intestines and other structures. The surgery requires precision and skilled hands to prevent damage to vital organs.

“It’s a challenging and tedious surgery in which we have a lot of experience and expertise,” Davidoff says.

St. Jude surgeons also team with oncologists to incorporate new surgical techniques and approaches into clinical trials for neuroblastoma.

 

Improving care for children with eye cancer

The St. Jude retinoblastoma treatment team is one of the most recognized in the country and is a textbook example of how research in St. Jude laboratories improves outcomes for patients in the clinic.

“Our basic scientists are able to develop preclinical models and identify pathways we can exploit with novel targeted therapies that we hope will reduce the toxicity of treatment, preserve vision and make patients’ lives better,” says Matthew Wilson, MD, ocular oncologist. He and Rachel Brennan, MD, a pediatric oncologist and retinoblastoma expert, are part of the team that treats children with retinoblastoma.

“The ability to examine and observe the eye and understand the size and number of tumors in the eye is just as important as treatment,” Wilson says. “With aggressive surveillance and careful examination of the eye, we’re able to judiciously apply the laser and cryotherapy to treat tumors, save the eye and preserve as much vision as possible.”

 In addition to the retinoblastoma group, patients receive support from an an eye clinic and many other services both during and after therapy. Because of this teamwork, the hospital’s investigators recently achieved a 100 percent five-year survival rate for children with retinoblastoma in one or both eyes at diagnosis.

Researchers continue to create new treatment options for patients whose cancer has spread beyond the eye, as well as develop new routes for delivering drugs while avoiding radiation and minimizing long-term effects of therapy.

Saving lives and limbs

When Bhaskar Rao, MD, joined St. Jude in the 1980s, amputation was inevitable for children facing surgery for osteosarcoma and Ewing sarcoma, the most common bone cancers in children. St. Jude was one of the few centers that offered an alternative—saving their limbs as well as their lives.

Today, the offer still stands as St. Jude surgeons help children maintain function of their arms and legs.

“Approximately 90 to 95 percent of our patients with bone tumors will have some sort of limb salvage,” says Rao, a pioneer in limb-sparing procedures. “Our approach is chemotherapy to shrink the tumor to an operable size; then we perform the limb-sparing surgery.”

Rao and orthopedist Michael Neel, MD, remove the diseased bone and replace it with a custom-made, expandable prosthesis. Because bone cancer primarily affects adolescents, growing patients return to St. Jude for outpatient procedures to lengthen the prosthesis and to maintain the same limb length on either side. After surgery, children undergo extensive rehabilitation and support care to help them maintain normal function.

The local recurrence rate for St. Jude patients who have undergone surgery for Ewing sarcoma is below the 10 percent national average. Rao attributes the outcome to experience, supportive care and unmatched follow-up care.

“At some centers, the surgeon operates on patients and sends them back to the oncologist and then the surgeon never sees the patient again until they encounter complications,” Rao says. “Here, we provide continuity of care for patients until they are 18 years old or until 10 years after diagnosis, whichever comes later.”

 

Patients treated at St. Jude are eligible to receive comprehensive screenings and health assessments related to their cancer—for a lifetime. “This level of follow-up care is not available in many places,” adds Rao, who also serves as Surgery director for the St. Jude International Outreach Program. Looking ahead, surgeons are evaluating and exploring new limb-sparing techniques to improve survival and help children live normal lives after treatment.

Minimally invasive techniques

Minimally invasive surgery is an alternative approach to diagnose and treat solid tumors, depending on the size and location of the tumors. John Sandoval, MD, uses the tools of the trade to view and investigate tumors in the chest and abdomen. He makes small incisions to perform biopsies, sample lymph nodes and remove abdominal tumors and lung lesions.

“We are staying mainstream and contemporary and attempting to push the envelope in laparoscopic surgery in children with solid tumors,” Sandoval says.

There are advantages for patients who qualify for the technique: less pain, quicker recovery, a speedy return to therapy and fewer long-term functional complications.

In 93 percent of procedures performed between 1994 and 2004, patients were able to receive an accurate diagnosis relying solely on the minimally invasive techniques performed at St. Jude. A follow-up study will be published this year to identify improvements and trends from the past decade. Sandoval anticipates the success rate to rise to nearly 100 percent.

Minimally invasive surgery is relatively new to pediatric oncology when compared to traditional surgery. Sandoval and his colleagues are helping to establish consensus guidelines for the appropriate use of these techniques in children with cancer. This provides the ideal opportunity for St. Jude to help shape the future of minimally invasive surgery in pediatric oncology.

Because of you...St. Jude will soon have a new operating room and surgery complex that will double the size of the current space. The new facility is designed to provide optimal care to patients and families who look to St. Jude for treatment and to further the hospital’s commitment to education and training.

The facility will include:

  • Integrated Operating Rooms with imaging and pathology services to encourage real-time interaction during surgery
  • Adjacent Intensive Care Unit (ICU) to reduce transport time for critical patients for surgery
  • Spacious, connecting parent rooms in the ICU
  • High-tech observation rooms to train surgeons and fellows from around the globe, which will help improve outcomes for children worldwide. Nearly every new pediatric surgeon in the U.S. and Canada participates in training at St. Jude.
  • Air-filtration technology to maintain a germ-free environment
  • State-of-the art technology to better assess how cancer affects the eye in children with retinoblastoma and other diseases that secondarily affect the eye
  • A modular design to incorporate the latest surgical equipment as technology advances

Thank you

Mick'el today. A bright and energetic 9yr old.
Mick'el today. A bright and energetic 9yr old.

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Organization Information

St. Jude Children's Research Hospital

Location: Memphis, TN - USA
Website:
St. Jude Children's Research Hospital
Elizabeth Ashford
Project Leader:
Elizabeth Ashford
Memphis, TN United States

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