Hematopoietic stem cell (HSC) transplant is an essential component of cancer therapy for pediatric patients with aggressive leukemias that have relapsed or are at high risk of relapse. Donor HSCs regenerate blood and immune cells in the patient, and if the function of these donor cells is impaired, the recovery process after HSC transplant is delayed. The number of HSCs received from the donor is traditionally used to predict the success of the HSC transplant, but cell dose is not always the best predictor of transplant success.
Survival in pediatric cancer has improved dramatically, in part due to high patient enrollment in clinical trials. Whereas Hispanic children have higher incidence of certain cancers, they have poorer survival rates than Non-Hispanic Whites (NHW). Although it is projected that by 2050, Hispanics will comprise 29% of the U.S population, they have fewer opportunities to participate in research. Therefore outcomes data are based on information obtained mostly from NHWs.
Brain tumors are the most common solid tumor diagnosed in childhood and thus account for significant childhood mortality in the United States. Low-grade astrocytomas and gliomas (LGG) are the most common type of brain tumor of childhood (36% of childhood brain tumors). Radiotherapy is avoided or delayed in the treatment of LGG to prevent neurocognitive decline and to prevent transformation to more malignant grade of tumor (grade III and IV).
Testicular cancers are the most common solid malignancy in adolescents and young males. They are unusually responsive to platinum (Pt)-containing chemotherapeutic drugs even when widely metastatic. Nevertheless some patients do not respond to initial therapy, and others with a good initial response become resistant to treatment. Why this type of tumor is so responsive to initial Pt drug treatment, and how it becomes resistant during therapy remain major questions in the field.
Over 100 billion blood cells are generated every day from our bone marrow. Paradoxically, these vast numbers of cells derive from an exceedingly rare population of hematopoietic stem cells (HSCs) that both self-renew and generate all blood and immune cell types for life. The characteristics and functions of these remarkable cells are highly conserved in all vertebrate animals. Our laboratory utilizes the unique attributes of the zebrafish embryo to study the biology of HSCs in new ways.
Until last year, the majority of patients diagnosed with high risk neuroblastoma succumbed to the disease despite intensive chemotherapy and bone marrow transplantation. In 2010, the PI of this proposal reported a major improvement in survival from 46% to 66% using an antibody therapeutic. Unfortunately, we cannot predict who will respond to this treatment which is frequently accompanied by serious side effects.
Raymeer is the best thing that happened to his family and friends. Most know him as “Baby Bacon,” a nickname given to him by a nurse because his name is Raymeer Bacon-El. He was diagnosed at 4-months-old with undifferentiated soft tissue sarcoma.
