Children's Cancer Hospital Newsletter - Spring 2012

NEWSLETTER

• • • • • • SPRING 2013

New space, new services ...

It won’t be long before the sounds of children’s voices will be heard again from the PediDome on Floor 9 of The University of Texas MD Anderson Cancer Center’s Main Building.

With construction activities wrapping up, the renovated and expanded Children’s Cancer Hospital is set to open in May, and is designed specifically for children, adolescents and young adults with cancer and their families. Families and patients worked alongside architects and hospital staff designing the floor, which now brings treatment services together in one central location.

Positioned within the nation’s No. 1 cancer center, the Children’s Cancer Hospital brings young patients the latest in cancer therapies, groundbreaking surgeries, advanced technology and novel research. The Children’s Cancer Hospital’s new 25,664-square-foot home allows consolidation and expansion of services. In addition to two inpatient pods, the Pediatric Ambulatory Treatment Center and Patient Intensive Care Services will be relocated to the floor.

Having services provided in one place allows both outpatients and inpatients to interact in a secure environment with one another and participate more easily in the school program, Child Life activities and to access the Ronald McDonald Family Room areas.

MD Anderson Children’s Cancer Hospital cordially invites you to a Private Preview of our redesigned and expanded hospital Wednesday, May 1, 2013 • 5–8 p.m.

MD Anderson Main Building, The Park, Floor 2 CCHrsvp@mdanderson.org

New in the Children’s Cancer Hospital

Features of the redesigned pediatric floor include: • 35 private inpatient rooms with sleeper sofas and customizable lighting features; • nine rooms for intermediate and intensive care; • infusion suite with four treatment chairs and 11 private beds; • 24/7 welcome desk for enhanced customer service and security; • dedicated pediatric pharmacy; • laundry room for patients and families; • four Ronald McDonald Family Room locations, including a kitchen, family lounge and sleep rooms; • K-12 privately accredited school onsite; • PediDome recreation space with a basketball goal, reading nook, movie screen and adjoining playroom and teen room; and • décor complementing a theme of nature, healing and hope.

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the Children’s Cancer Hospital is ready for patients

The Children’s Cancer Hospital is a special place where young patients receive the latest in treatment options, personalized cancer therapy and opportunities to participate in clinical trials.

A unique place for pediatric cancer care

• Comprehensive care in a family-centered environment Multidisciplinary teams come together in partnership with the patient and family to decide on the best course of care. A dedicated chaplain, child life specialists, pediatric pharmacists, teachers, psychologists and medical interpreters work collaboratively with a patient’s medical team to provide comprehensive care.

• Primary focus is cancer Serving children since MD Anderson opened its doors, the Chil-

dren’s Cancer Hospital sees more types of cancer than any other children’s hospital in Texas, treating more rare cancers in a single day than most physicians see in a lifetime.

• A hospital within the No. 1 cancer center in America Pediatric and adult oncologists at MD Anderson collaborate to bring the latest cancer therapies to pediatric patients, including access to the nation’s largest Phase I clinical trial program and the largest stem cell transplantation and cell therapy program.

• Leading-edge technologies and therapies not found at most hospitals Patients have access to proton therapy, BrainSUITE®, robotic surgery, PET/CT fusion imaging as well as novel surgical procedures performed solely by MD Anderson pediatric surgeons.

• Programs tailored toward adolescents and young adults Often overlooked at many hospitals, the teen and young adult patient population has resources dedicated to serving their unique needs at MD Anderson, including a fertility clinic, teen and young adult patient advisory councils, vocational counseling and targeted clinical practices.

• Focus on survivorship The Children’s Cancer Hospital pioneered survivorship at

MD Anderson, forming its own Childhood Cancer Survivors Clinic that follows childhood survivors into adulthood. In addition, the ON (Optimizing Nutrition) to Life Program targets patients, survivors and the general public to understand nutrition’s relationship with cancer and treatment and to encourage healthier eating behaviors.

Although pediatric cancer is rare, the cancers that develop in this younger age group are often very different from those seen in adults. Dissimilarities in tumor pathology appearance, signs and symptoms of the disease and response to treatment are common.

The MD Anderson Children’s Cancer Hospital is equipped to provide exceptional pediatric and adolescent cancer care.

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Children’s Cancer Hospital • Spring 2013

When discovery collides with reality:

The journey of an orphan drug

Notch pathway found to naturally slow growth of acute myelogenous leukemia in pre-clinical study • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • •

In 1982, Eugenie Kleinerman, M.D., division head of Pediatrics and head of the MD Anderson Children’s Cancer Hospital, began to study a medication named MEPACT (mifamurtide) and worked to get it to patients who had been newly diagnosed with resectable non-metastatic osteosarcoma.

She learned that the drug brought about the regression of melanoma lung metastases in mice.

Because osteosarcoma most often metastasizes to the lungs, and many of these metastases are resistant to chemotherapy, these findings caught her attention.

In collaboration with the Division of Cancer Medicine, Kleinerman and James L. Murray, M.D., professor in the Department of Breast Medical Oncology, led the first clinical trial that took MEPACT from preclinical testing to Phase I testing in humans. This trial determined the optimal biologic dose of the drug and defined side effects. Patients 18 years and older participated because children can’t be included in the first Phase I trials.

Kleinerman also was principal investigator on the Phase II clinical trial of the drug for pediatric patients with relapsed osteosarcoma. These trials showed how MEPACT stimulated human immune cells to react against osteosarcoma cells.

The research eventually led to a Phase III national trial of about 700 newly diagnosed osteosarcoma patients, conducted by the Children’s Oncology Group.

Patients were randomly assigned to receive chemotherapy alone or chemotherapy plus MEPACT. Scientists found that MEPACT, when given in conjunction with combination chemotherapy, resulted in a 30% reduction in the mortality rate at eight years after diagnosis, compared to the patients who received chemotherapy alone.

That number’s now increased to 15 years after diagnosis.

In 2007, with a new pharmaceutical company as their champion, the data from the Phase III trial was presented to the FDA, but the request for approval to use was denied. The FDA felt that another Phase III clinical trial, which would require approximately 900 patients with this rare disease, was needed.

Kleinerman believed that the odds were too great for this to occur, but the drug manufacturers pushed to take MEPACT to Europe. There, Kleinerman helped guide the drug through reviews by the European Medicines Agency. MEPACT was approved for pediatric patients with non-metastatic sarcoma in 2009, allowing it to be marketed in the 27 EU member states, as well as Iceland, Liechtenstein and Norway.

The National Institute for Clinical Excellence, the agency in the United Kingdom that determines which drugs will be covered by the government, approved MEPACT for newly diagnosed OS in 2011. Last September, MEPACT received a prestigious honor: the 2012 United Kingdom Prix Galien Orphan Drug award for international pharmaceutical research and development.

Kleinerman only wishes the news about MEPACT was so positive in the United States. But she believes that won’t happen without changes to the approval process for drugs used to treat diseases that primarily affect children.

Researchers at MD Anderson Cancer Center may have found a way to boost the body’s ability to kill acute myelogenous leukemia (AML). Notch signaling, a communication system between the body’s cells, was shown to play a role in limiting the growth of AML cells.

“These new findings can help us determine better ways to harness a patient’s own body to fight their leukemia,” said senior investigator Patrick Zweidler-McKay, M.D., Ph.D., associate professor at MD Anderson Children’s Cancer Hospital Researchers found that the body naturally slows down AML cell growth by expressing Notch ligands. Notch ligands turn on the Notch signaling pathway, which in turn causes AML cells to die.

However, there aren’t enough naturally occurring ligands to fully be effective against the cancer.

Results from the study indicated that strong activation of the Notch pathway in human AML cells led to a more than 25-fold decrease in leukemia cells compared to control group. In contrast, human AML cells in which Notch signaling was blocked grew much more aggressively than normal leukemia cells. Mice with the Notchinhibited cells died more quickly as well.

“By understanding what causes AML to grow or die, we’ll be able to find more targeted ways using the body’s immune system to fight cancer while hopefully sparing some of the strong toxicities associated with systemic chemotherapy,” said Zweidler-McKay. AML accounts for approximately 20 percent of all childhood leukemias, which is the most common cancer in children according to the National Cancer Institute. The 5-year survival rate for these children is around 60 percent, much lower than the 80% survival rate.

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Division of Pediatrics - Unit 87 MD Anderson Cancer Center 1515 Holcombe Blvd Houston, Texas 77030-4009

The Children’s Cancer Hospital Newsletter

is an educational resource for physicians and others interested in the treatment, research and prevention of pediatric cancers, produced quarterly from the Division of Pediatrics at The University of Texas MD Anderson Cancer Center.

Non-Profit Org. U.S. Postage PAID Houston, TX Permit No. 7052

ADDRESS SERVICE REQUESTED

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Division Administrator: Karen Broussard Managing Editor: Gail Goodwin We welcome your questions and suggestions.

George Foreman Pediatric and Adolescent Inpatient Unit Robin Bush Child and Adolescent Clinic Brenda and J. Howard Johnson Pediatric Ambulatory Treatment Center Kim’s Place R.E. (Bob) Smith Research Facility

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Our Mission

To cure cancer in children and young adults within a caring, life-affirming environment.

Our Vision

We will offer children and young adults hope and an opportunity to lead full and productive lives. We will lead the efforts worldwide to cure cancers through the excellence and compassion of our people, research-driven innovative therapies, education programs and active collaboration with patients, families and communities.

• • • • • Contact us at 713-792-5410 8 a.m.–5 p.m. (M–F) and after hours at 713-792-7090.

Request the on-call pediatric oncology attending.

We’re on the web:

www.mdanderson.org/children www.mdanderson.org/cchnewsletter

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Change of address or other communication regarding this newsletter may be directed to Karen Broussard at 1515 Holcombe Blvd., Unit 087, Houston, TX 77030; 713-792-6620.

It’s all in the head

The new Pediatric Brain Tumor Program of The University of Texas MD Anderson Children’s Cancer Hospital and Children’s Memorial Hermann Hospital brings together the strengths of two leading medical institutions to treat brain and spinal cancer in children and young adults.

Primary in these strengths is the excellence in comprehensive care that both hospitals bring to the program.

• Leading expertise – Children receive care by some of the nation’s top specialists in neuro-oncology, neuro-surgery, radiation oncology, neuro-pathology and other sub-specialties.

• Support services – Patients have access to a variety of services and programs to ensure their emotional and social well-being during treatment and into survivorship. An in-hospital school, child life specialists, vocational counselors, psychologists, developmental specialists and school re-entry coordinators are just a few of the services provided to help patients maintain a normal life during treatment.

• Multidisciplinary approach – Each patient’s case is discussed together by pediatric neuro-oncologists, neuro-surgeons, radiation oncologists and neuro-radiologists to ensure a unified approach to care.

• Family-centered care – Parents partner with staff in planning the best treatment approach for their child. In addition, multiple advisory councils involving parents, patients and staff provide integral feedback in the daily decision-making process of the hospital.

• Innovative treatment – MD Anderson’s Brainsuite®, proton therapy, gamma knife radiosurgery, stereotactic radiotherapy, clinical trials, translational brain tumor research, advanced imaging technology and minimally invasive endoscopic tumor resection are new and advanced ways used to treat pediatric brain tumors.

• Clinical trial – A clinical trial is offered solely through this collaborative program for recurrent medulloblastoma, ependymoma and atypical teratoid rhabdoid tumors that uses the direct infusion of chemotherapy into the fourth ventricle.

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Your input will help us plan future issues, and your responses will be confidential.

You can access the survey at www.surveymonkey.com/s/cchnews.

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