International Patient Services

International Update Newsletter:
Division of Oncology

Our team of more than 40 full-time pediatric oncologists sees more than 500 new patients each year, finding the most effective treatments for each child’s cancer while working to minimize the side effects that can affect patients’ quality of life. Patients with relapsed or refractory cancers can find new treatment options through our Developmental Therapeutics program, which brings together physicians, nurse practitioners, nurses, social workers and research associates to provide access to the most innovative clinical trials — many of which were developed here at CHOP.

New Treatments, New Hope

Current strategies for treating childhood cancer, including intensive cytotoxic chemotherapy, surgery and radiation, cure approximately 80 percent of children with cancer. However, two-thirds of childhood cancer survivors will develop a chronic medical condition as a late effect of this curative therapy. Further improvements in survival for children with cancer and a decrease in long-term side effects of therapy require innovative therapies, including new drugs; immunotherapy with antibodies; or cells, vaccines or viruses that specifically target tumor cells.

The groundbreaking clinical trials led by Cancer Center investigators demonstrate the innovation and commitment of CHOP physician-scientists to discovering cures for cancer
and other serious medical conditions in children. We are pleased to highlight their work in this issue.

• Find a clinical trial at CHOP »
• Sign up for our “Featured Clinical Trials” enewsletter » 

CHOP Partners with the University of Pennsylvania on Promising New T Cell Immunotherapy Treatment

Stephan A. Grupp, MD, PhD, director of translational research for CHOP’s Center for Childhood Cancer Research, has long believed in the promise of T cell immunotherapy. Years ago, Grupp and other researchers reasoned that if they could genetically modify a patient’s T cells to make them better at finding and destroying cancer, they would have a promising new treatment option. Grupp believed that T cell immunotherapy would be even more effective than antibody immunotherapy, as well as gentler on patients.

But researchers who tried the T cell approach had trouble making enough of the modified cells. And the cells they did make didn’t work very well. “We had all the ideas,” says
Grupp, “but we didn’t really have the techniques to make it truly work.”

That changed when the Jeffrey Jay Weinberg Memorial Foundation provided funding that allowed Grupp and his colleagues at the University of Pennsylvania to refine the
techniques that would make the T cell approach feasible — and to start gathering the data they would need to begin a clinical trial.

Preliminary results of this trial, called CART19, gained widespread media attention when they were reported in The New England Journal of Medicine and Science Translational Medicine in August 2011. The researchers showed that a patient’s modified T cells can survive for many months after administration and that the modified cells can grow in the body in large quantities. Most significantly, the modified cells killed large quantities of cancer cells in all patients treated. When the results were published, three patients, all adults with chronic lymphocytic leukemia, had received the treatment; the trial is now enrolling children at CHOP.

Translational research like the T cell immunotherapy project is a priority at the Cancer Center. In 2010, Grupp and John M. Maris, MD, chief of the Division of Oncology and director of the Center for Childhood Cancer Research, were part of a group of researchers who found that a new type of antibody immunotherapy improved two-year survival rates in patients with high-risk neuroblastoma by 20 percent — the first significant increase in neuroblastoma cure rates in more than a decade. The study was published in The New England Journal of Medicine.

• The CART19 trial is now enrolling patients at CHOP, but it has limited openings. Get more information on CART19 »
• The CART19 trial was named one of the top 10 stories of 2011 by Discover magazine »

Blood and Marrow Transplantation (BMT) Team Takes Multidisciplinary Approach to Treating Nonmalignant Diseases

Marshaling CHOP’s vast expertise in blood and marrow transplantation and immunodeficiencies, as well as in clinical care and lab research, CHOP’s Cancer Center is well equipped to meet the needs of patients who receive a BMT to treat a nonmalignant disease.

“We’re developing more novel methods for patients who are difficult to transplant,” says Nancy J. Bunin, MD, director of Blood and Marrow Transplantation. In some cases, the team can use reduced-intensity conditioning, making the treatment less toxic. And, says Bunin, “For patients who don’t have a fully matched donor, we have the capability to transplant them while reducing the risk of severe graft-versus-host disease.”

Patients with genetic diseases can be among the most difficult to transplant. “Many kids with nonmalignant disorders have a genetic disease that brings unique concerns to the table,” says Edwin M. Horwitz, MD, PhD, a BMT physician. For example, children who receive a transplant to treat sickle cell anemia typically have an especially high risk of rejecting the transplanted marrow. And some kids with genetic diseases are at especially high risk for chemotherapy related toxicities.

The Cancer Center team partners with CHOP specialists in hematology, immunology, surgery, psychology and other areas to provide the multidisciplinary care these children require. “It really is a team approach,” says Horwitz. Clinical research is a major focus of the BMT program, which brings together clinicians and researchers with specific expertise in several areas, including:

• Bone disorders: Horwitz is the principal investigator of an NIH-funded trial testing the efficacy of mesenchymal stromal cell infusions in children with osteogenesis imperfecta. He and his team are also conducting laboratory research that they hope will lead to a BMT trial and make cell therapy an option for other bone disorders.
• Bone marrow failure disorders: Cancer Center investigators are collaborating with CHOP’s Pediatric and Adult Comprehensive Bone Marrow Failure Center to develop a protocol that will use investigational anti-CD45 antibodies and anti-CD52 as cytotoxic agentfreeconditioning for patients with dyskeratosis congenita or Shwachman-Diamond syndrome without evidence of myelodysplasia. In the future, the team hopes to use graft engineering techniques and personalized transplantation and/or cell therapy protocols to improve patient outcomes.
• Immune deficiencies: CHOP is building an integrated immunodeficiency service within its Division of Immunology, which will include a joint clinic and will conduct research on severe combined immunodeficiency disease.

CHOP’s BMT team is treating an increasing number of patients with nonmalignant diseases, says Bunin, and her team plans to develop even more protocols for these patients that are unique to CHOP.