Co-funded by the Dick Vitale Gala, and WWE in honor of Connor’s Cure
Dr. Jun Qi is a synthetic organic chemist and chemical biologist who has developed small molecules and pioneered a novel chemical strategy in which small molecule therapeutics can be designed to destroy specific proteins within a cell, as opposed to suppressing enzymatic function. Dr. Mariella Filbin is a physician scientist specializing in pediatric neuro-oncology with clinical and scientific interests converging upon pediatric brain cancers, in particular, diffuse intrinsic protein glioma (DIPG) which is universally fatal. Dr. Filbin has used patient-derived models to identify a potential DIPG-specific target for Dr. Qi’s protein degrader technology. They will work together to overcome challenges in childhood brain cancer treatment, such as toxicity and blood-brain-barrier (BBB) penetration. This exciting study has two broad objectives:
- To define the mechanism by which the cancer dependent protein is driving DIPG formation and growth;
- To yield optimized drug compounds suitable for preclinical study and translation to clinical trials in DIPG.
By working together as team, Drs. Qi and Filbin will cultivate a symmetrical relationship in which chemistry will be used to clarify the biology; and biology will be used to guide the small molecule design and development. By combining their complementary skill sets in chemistry, chemical biology and cancer biology, their joint efforts will result in the preclinical validation of eliminating the target genes and ideally the development of a clinical trial using this novel strategy for DIPG to achieve the bench-to-bedside translation of their research.