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In many ways, Mateo Kohler of San Jose is a typical 9-year-old. He likes to play soccer and practice tae kwon do, and he may be overly fond of video games. But he’s also a bit of mystery.

Diagnosed with a rare subtype of acute lymphoblastic leukemia just two weeks before his fifth birthday, Mateo did not respond to standard treatment, and a match for a stem cell transplant could not be found. Mateo spent weeks on and off as an inpatient, and then years going to weekly and biweekly clinics. He was put on one promising chemotherapy trial after another.

“The drugs that worked for other kids were just not working for him,” says his mom, Traci Kohler. “Mateo’s doctors at Packard Children’s are very well connected to the latest findings, so they were able to be more flexible in their thinking and pulled him off the trial instead of taking a ‘wait and see’ approach. I think we’re really lucky that they’re able to think outside the box and advocate for what’s best for him.”

That adaptable approach is the hallmark of the Bass Center for Childhood Cancer and Blood Diseases, where compassion, innovation, and collaboration are combined to produce the best outcomes for kids with cancer and other life-threatening illnesses.

“Children have their entire lives ahead of them, so it’s important that we are very aware of how we care for them,” says center director Kathleen Sakamoto, MD, PhD. “That’s the advantage of being located in a place committed to all aspects of understanding and treating pediatric cancer.”

Fresh Perceptions

Housed within an academic children’s hospital, the Bass Center can access a range of intellectual and technological resources. And because Packard Children’s is part of a larger but tight-knit medical community at Stanford, it’s easy for investigators from different specialties to work together and share unique perspectives. Together they are working to better understand important risk factors, as well as the biology and mechanisms of specific childhood cancers.

“That makes it easy for translational medicine to take place,” adds Sakamoto, the Shelagh Galligan Professor in the Stanford School of Medicine.

Translational medicine is the back-and-forth dynamic between basic and clinical scientists and patient care specialists, an interaction that speeds the process of applying discoveries to new technologies, treatments, diagnostics, or therapeutic targets.

Bass Center specialists collaborate with colleagues at Stanford Hospital & Clinics, as well as experts in non-medical specialties like engineering, physics, and chemistry. The center also has ties with Silicon Valley, where many pharmaceutical and high-tech companies stay closely affiliated with their Stanford roots.

Packard Children’s has an unmatched reputation for promoting new approaches to the development of medical therapeutics and diagnostics. Clinical boundaries are easy to cross, and it shares a culture of innovation and communication that attracts some of the world’s top scientists, physicians, and caregivers.

“Creating the right environment involves building a network that encourages leadership, communication, and teamwork,” says Michael Jeng, MD, section chief of pediatric hematology. “We have a close partnership with the adult hospital, and access to all the services patients may need. The result is better outcomes for patients and a more comprehensive approach to their quality of life.”

Under One Roof

Every physician in the Bass Center engages in clinical research, an important means of developing new protocols and cures. Packard Children’s is also deeply involved in the Children’s Oncology Group (COG), an international consortium that consolidates clinical research studies in pediatric cancer. Patients gain access to new protocols before they are widely available, and help others by contributing to medical research.

“Most cancer science focuses on adults, but cancer is different in kids,” says Neyessa Marina, MD, director of the outpatient hematology and oncology clinic and section chief of pediatric oncology. “We’re still learning what works and what pathways can be applied. For that we need to collaborate with other institutions to get the numbers of participants we need for meaningful clinical trials,” adds Marina, who is also principal institutional investigator of COG.

A leading researcher within COG, Clare Twist, MD, is refining treatment for neuroblastoma, the most common pediatric solid cancer outside the central nervous system. Twist recently led a study investigating relatively low-intensity therapy, a strategy for reducing toxicity and late effects of treatment. As director of the Bass Center’s neuroblastoma program, she also oversees studies to develop early trials of several promising new therapies.

More than 80 percent of Bass Center patients are involved in a clinical trial, and their participation makes a sizeable impact. Clinical trials are directly related to advances in surgery, chemotherapy, and radiation therapy, improving outcomes for cancer patients worldwide.

“Progress in the management of childhood cancer is one of the great success stories of modern medicine,” says Link, a specialist in lymphomas and sarcomas. “Pediatric oncology can serve as a model for conquering cancer in adults.”

Jeng, for example, was involved in a multi-site study to assess a more effective way to administer deferasirox, a drug used to remove iron from the body. Iron buildup can cause liver, heart, and endocrine system damage in patients who require regular blood transfusions, and previous medications could only be taken through a needle – which kids hate. Jeng’s research has helped to improve the quality of life for these patients by demonstrating the effectiveness of an oral medication that offers the same benefits.

Rajni Agarwal-Hashmi, MD, section chief and clinical director of the pediatric stem cell transplant program, is developing ways to counteract idiopathic pneumonia syndrome (IPS), a serious complication affecting the lungs after a stem cell transplant. A decade ago, 80 percent of children who developed IPS succumbed; today that number has dropped to 30 percent.

“This kind of progress takes place here because there is a lot of expertise and a very strong infrastructure under one rood,” Agarwal-Hashmi says. “It’s designed to develop novel protocols and breakthrough therapies.”

Growing Up Well

Many Bass Center physicians are also stem cell scientists working to understand how to use these cells' unique properties of regeneration. Packard Children’s performs more than 40 stem cell transplants each year to treat leukemia or other cancers, as well as some inherited immunodeficiency diseases, and was among the first to offer pediatric bone marrow transplants more than 20 years ago. Experience and ongoing refinements continue to expand on this life-saving procedure, steadily increasing survival rates.

That expertise helped Braden Fransham of San Jose, who was born with Wiskott-Aldrich syndrome, a rare condition that affects blood cells and cells of the immune system. Diagnosed at 6 months, it took more than a year before he matched with a bone marrow donor and received a successful transplant. The first five years of his life were filled with long hospital stays, clinic visits, and immunosuppressant drugs, says his mother, Sonya Palmer. Today, Braden is an energetic 8-year-old, and his family has happily donated blood and tissue so researchers can learn more about the genetic mutation behind the disease.

To expand the donor pool, Agarwal-Hashmi established a cord blood program at Packard Children’s. Cord blood, found in the placenta and umbilical cord after birth, is a rich source of stem cells, which can differentiate into various types of blood cells and rebuild a faulty immune system. “It expands the donor pool for any kind of stem cell transplant,” she says. “It means that every child has a donor.”

But with success come new challenges. Cancer treatments can also cause long-term side effects that can affect a child’s health and quality of life later on (see Life After Cancer). Packard Children’s provides long-term follow-up and ongoing supportive care to counteract any late-onset complications for childhood cancer survivors.

“Treatment now is less intense and more targeted, and outcomes are better across the board,” says Agarwal-Hashmi. “But there are consequences of therapies, and it’s vital that we make sure these children grow up well.”

That approach is something Mateo, Braden, and their families look forward to seeing through.

This article appeared in the Lucile Packard Children’s News publication in Fall 2012.

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