2002

Andrew Davidoff MD

St. Jude Children's Research Hospital, Memphis, TN Chair, Surgery St. Jude Endowed Chair in Surgical Research Division Chief, General Pediatric Surgery

Research Focus: Anti-angiogenesis

Cancer Type: Neuroblastoma

Award: Young Investigator

2002-2005 Research Grant:
Neuroblastoma is one of the most common childhood cancers. Angiogenesis, the formation of new blood vessels, is central to the progression of cancer, and it is known that inhibiting angiogenesis can literally starve a tumor to death. We hypothesize that delivering a particular gene called pigment epithelium derived factor (PEDF) to the liver has the potential to inhibit angiogenesis and restrict the growth of neuroblastoma. PEDF is known to be a highly potent angiogenesis inhibitor and also is capable of inducing neuroblastoma cells to mature, decreasing their malignant potential. My research is focused on using viruses as vectors to carry the gene for PEDF to the liver. The data being developed from these studies are likely also to be applicable to other types of cancer, and even to other diseases in which angiogenesis contributes to the disease phase.

Current Research:

Dr. Davidoff has continued his research on anti-angiogenesis in the treatment of neuroblastoma. He has since discovered a novel, gene-therapy mediated process of delivering the necessary angiogenesis inhibitor to a patient’s cells, likely a successful treatment for cancer. He has also expanded his research focus, applying gene therapy treatments beyond cancer to other monogenic disorders.

His studies have focused primarily on hemophilia and have found that gene therapy may be useful in treating this disease and others like it through long-term correction of the disease phenotype. This correction may be accomplished through the application of viral vectors (a tool used to deliver genetic material into cells), specifically adeno-associated virus (AAV) vectors. Dr. Davidoff’s research is likely to provide successful treatment of both neuroblastoma and other cancers, and expand the use of gene therapy to the treatment of many other types of diseases.

Dr. Davidoff will be recognized as a pioneer of basic gene therapy for hemophilia in Genetic Engineering News 2014 series “Perspectives”.

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