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Columbia University Medical Center

630 West 168th Street
Box 49
New York, NY 10032-3702
United States
Columbia University Medical Center

Diffuse Intrinsic Pontine Gliomas(DIPG) are aggressive brain tumorsin children that currently have no effective treatments. Most DIPG cases (over 70%) involve a mutation in a gene called H3K27M, which changes how genes are regulated and affects the tumor’s behavior. Although scientists now better understand these gene changes, new treatments are still needed to improve outcomes for children with DIPG. Cancer cells often change their metabolism to grow faster. These changes affect how enzymes work and influence gene regulation.

Mentor Name: Robert Wechsler-Reya

Background


Amplification of oncogene MYCN is a hallmark of neuroblastoma, and is associated with metastasis and very poor survival. However, despite the identification of MYCN-amplification as an adverse prognostic marker, no drugs that target MYCN have been developed.

FerrandoT-lineage acute lymphoblastic leukemia (T-ALL) is an aggressive hematologic cancer in which novel more effective antileukemic drugs are needed for the treatment of patients with chemotherapy resistant disease. In this context the identification of activating mutations of NOTCH1 in over 50% of T-ALL has brought great interest to the development of targeted anti-NOTCH1 therapies.