Icahn School of Medicine at Mount Sinai

Icahn School of Medicine at Mount Sinai

21 Claremont Avenue

Apt 94

NEW YORK, NY 10029

United States

Unraveling GATA1s dependency in down syndrome-associated leukemia

Acute Myeloid Leukemia (AML)

Mentor Name: Elvin Wagenblast

Dissecting lineage plasticity in mixed phenotype acute leukemias

Leukemia

Mentor Name: Elvin Wagenblast

Exploring the Immune Effects of Photodynamic Therapy Treatment on Diffuse Midline Glioma Progression

Brain Tumors

Mentor Name: Oren Becher

The project will explore a new therapeutic avenue for Diffuse Midline Gliomas called photodynamic therapy. This approach has not been explored in depth so far to treat Diffuse Midline Gliomas

IND-Enabling Studies for WNTinib, a Novel Selective Therapeutic for CTNNB1 Mutant Hepatoblastomas

Medulloblastoma, Wilms Tumor, Hepatoblastoma

Hepatoblastoma (HB) is the most frequent pediatric form of liver cancer, generally arising in young children (<3yo). Despite being a rare tumor (1.8 in 1,000,000 children/year), HB rates are on the rise and therapeutic options are limited to chemotherapy. Chemotherapy (i.e. cisplatin) is given to patients without any consideration to a specific mutation within tumors and toxicity is significant. Here we seek to advance a novel therapeutic candidate for HB that has been optimized to selectively kill cancer cells with specific mutations, while sparing normal hepatocites.

Modeling the Initiation and Progression of Down Syndrome Associated Leukemia Using CRISPR/Cas9 at Single Cell Resolution

Leukemia, Acute Myeloid Leukemia (AML)

Leukemia is the most common cancer in children, accounting for almost 1 out of 3 cancers. This is also true in children with Down syndrome, a chromosomal abnormality caused by a third copy of chromosome 21. In particular, children with Down syndrome have a 150-fold increased risk of developing acute myeloid leukemia during the first years of their childhood. In fact, the first mutation occurs in the gene GATA1 during the development of the fetus at pregnancy.

Identifying Therapeutic Targets to Prevent Progression of Familial RUNX1 Disorder to AML Using Novel iPSC Models

Acute Myeloid Leukemia (AML)

Background

Children with familial RUNX1 disorder have a high risk of developing leukemia, which in most cases is hard to treat with current chemotherapy. Finding ways to prevent progression of leukemia is the most promising strategy towards the effective management of this disorder.

Alex's Lemonade Stand Foundation for Childhood Cancer