Alex's Lemonade Stand Foundation for Childhood Cancer

Radiotherapy is the only standard of care for H3K27M mutant diffuse midline glioma. Unfortunately, following initial response tumors recur. Experimental therapeutics can synergize with radiotherapy to produce a greater therapeutic effect than either agent alone, a concept known as 'radiosensitization'. Our team has pioneered this concept both in the laboratory and clinic including in successful bench-to-bedside translational studies in patients with brain tumors.

Chromosomal translocations of the nucleoporin 98 (NUP98) genes, which lead to the expression of NUP98 fusion proteins, are often found in patients with various hematologic malignancies, but are particularly prevalent in pediatric acute leukemia, constituting ~10% of all childhood leukemia cases. The presence of NUP98 translocations in pediatric leukemia patients confers very poor clinical outcome, leading to only ~10% of event-free survival three years after diagnosis and <30% four years survival rate.

Mentor: Costas Lyssiotis