Childhood Cancer Research
Children’s Hospital of Philadelphia
3401 Civic Center Boulevard
Philadelphia, PA 19104
United States
Acute lymphoblastic leukemia (ALL) is cancer of the blood and bone marrow and is the most common cause of cancer in children. Different kinds of ALL are caused by different genetic changes within the leukemia cells, and these changes affect how well children respond to chemotherapy. Although most children with ALL can be cured with regular chemotherapy, the Philadelphia chromosome-like (Ph-like) type of ALL is known to be very difficult to cure and often comes back (relapses).
Children with high-risk neuroblastoma continue to have poor outcomes despite maximal intensification of multimodal treatments. Neuroblastoma tumors continue to express lineage-specific cell surface molecules that can be exploited using immune-based therapies, as exemplified by the unprecedented clinical success of anti-GD2 monoclonal antibodies. However, anti-GD2 immunotherapy is usually associated with unacceptable toxicities such as difficult-to-treat pain, as GD2 is also expressed on healthy tissues such as peripheral nerves.
Mentor: Dr. Richard Aplenc
Background
Current therapy of high-risk pediatric solid tumors like neuroblastoma (NB) and sarcomas requires extremely intense treatment. However, cure rates are <50%, and there are significant long-term side effects in survivors. Targeted delivery of anticancer agents using nanomedicines can dramatically improve efficacy and reduce systemic toxicity. We have packaged SN38, the active product of Irinotecan, in small packets called nanoparticles (NPs).
Lay Summary: Early T-cell precursor acute lymphoblastic leukemia (ETP ALL) is a type of T-cell acute lymphoblastic leukemia (T-ALL). Children with ETP ALL and non-ETP T-ALL have a similar chance to be cured. But, children with ETP ALL are more likely than children with non-ETP T-ALL not to respond to therapy. Children with non-ETP T-ALL are more likely to respond to therapy and then have the leukemia come back (relapse) than children with ETP ALL. We do not understand why some children with ETP ALL do not respond to treatment.
Background
High-grade pediatric gliomas (brain tumors) are a devastating disease. Mutation analysis on large cohorts of tumors has identified a number of commonly mutated genes in pediatric high-grade gliomas. However, it often remains unclear if these mutated genes play an important role in tumor formation and if they potentially confer a therapeutic sensitivity. In the current project, we are aiming to understand how mutations in two of these genes, the tumor suppressors NF1 and SETD2, cooperate to form tumors.
Pages
