Our physicians and researchers hold leadership roles in the Children's Oncology Group (COG). The Children's Oncology Group is a national clinical research consortium made up of more than 200 pediatric cancer centers, which designs and evaluates cancer therapies through large clinical trials. Because we are a participating Center, all of our patients are eligible for treatment protocols used by this respected national research cooperative. Clinical trials allow our patients access to promising treatments still in development. The Center has an average of 75 active clinical trials available to eligible patients.
Our clinical research includes Phase I, II and III clinical trials investigating experimental therapies and new agents that reduce toxicity of chemotherapies. Our team also works closely with researchers at the Stanford School of Medicine in genetic profiling of tumors, new drug development and genetic targets for therapies.
Some of our clinical research highlights include:
Investigators at Lucile Packard Children's Hospital have been leaders in dissecting the immunology of acute lymphoblastic leukemia and in discerning the molecular events which underlie leukemogenesis. Packard investigators are nationally recognized for studies directed at reversing multi-drug resistance in leukemia and applying these findings to the treatment of children with refractory leukemia and more recently to the treatment of children with newly diagnosed acute non-lymphocytic leukemia.
Late Effects/Long Term Survivorship
The pediatric group at Lucile Packard Children's Hospital is part of a national study of 25,000 survivors of childhood cancer and place special emphasis on the assessment of cardiac, pulmonary and neurocognitive late effects.
Gene Therapy for Hemophilia
Researchers at Lucile Packard Children's Hospital and Stanford University Medical Center are testing a novel new treatment for hemophilia B. Patients with hemophilia B lack the ability to produce enough of a protein - called factor IX - essential for successful blood clotting. The researchers, led by Mark Kay, MD, PhD and Bertil Glader, MD, PhD, have genetically modified a virus to produce factor IX. In two of the three adult patients who participated in a small-scale study, the injected virus made enough factor IX to reduce the patients' dependence on external administration of the factor to treat bleeding episodes.
Kay and his colleagues are continuing their research into the gene therapy of hemophilia. They would also like to use the approach to treat the more common version of hemophilia, hemophilia A.
Our team is participating in some of the early clinical trials for a promising new treatment called non-myleoablative hematopoietic stem cell transplants.
Although researchers and clinicians have made much progress in improving the outcomes of hematopoietic stem cell transplants, the risk of developing complications after an allogeneic transplant (a transplant using bone marrow or peripheral blood stem cells provided by a donor) is still substantial.
The goal of a non-myeloablative allogeneic hematopoietic stem cell transplantation is to offer the benefit of a standard transplant, but with reduced toxicity. In a standard transplant procedure, a child undergoes very high dose chemotherapy -- with or without radiation therapy -- to destroy as many cancer cells as possible before the healthy donor stem cells are transplanted into the patient.
Because the treatment also destroys the patient's bone marrow and immune system, the donor's stem cells are not rejected, and grow to replace the patient's bone marrow. A non-myeloablative transplant uses much smaller and less toxic doses of chemotherapy and radiation, allowing the stem cells of both the patient and the donor to coexist (a condition known as immune tolerance). This partial replacement of the bone marrow function can cure many diseases while reducing the severity of treatment side effects.
Relapse After Transplant
Relapse remains a significant threat following a bone marrow transplant for leukemia. Our physicians are looking for alternative treatment approaches to further reduce the risk of relapse. One option they are researching is the use of leukocyte, or white blood cell, infusions to encourage the new bone marrow to graft and then attack the leukemia cells. Another study is investigating the use of a novel pre-transplant therapy prior to the transplant, which will attack and weaken the leukemia cells.
Our doctors are also developing new ways to process bone marrow in the laboratory to make it more acceptable to the recipient. This will allow doctors to broaden the availability of appropriate donors.
Graft-versus-host disease (GVHD) is a condition that occurs when the newly regenerated immune system arising from an allogeneic transplant attacks the patient's body and damages organs and tissues. The drugs that a recipient of an allogeneic transplant takes to suppress their immune system and reduce the chance of developing GVHD also make them susceptible to developing a serious infection. Lucile Packard Children's Hospital physicians are working on new drugs to diminish the risk of GVHD following hematopoietic stem cell transplant while minimizing the risk of infection.