Patrick Gunning – University of Toronto, ON

Project Title: “Development of Selective Small Molecule Inhibitors as Treatments for Medulloblastoma”

Description of Project:

Brain cancers are exceptionally challenging to treat with chemotherapeutics, partly because the brain is protected by a ‘blood-brain-barrier’. This barrier serves to protect the brain from potentially harmful substances, but it can also block drugs that would be helpful. Therefore, we are developing drugs that have specific properties that allow them to cross the blood-brain-barrier. We have been developing drugs that engage a new target in medulloblastoma, one of the most frequently occurring pediatric brain cancers. This target is called HDAC6, and we have developed drugs that have exquisite selectivity for this protein. For example, current drugs in the clinic for HDAC6 have 5-6 fold selectivity, whereas our lead candidates are 800-fold selective. They have also shown the potential to cross the blood-brain-barrier in mouse models. We are working to expand our current chemical library to develop first-in-class HDAC6 inhibitors for medulloblastoma.

What receiving this award means:

“Cancer treatment has undergone many advancements in the past decade to improve patient quality of life and to provide new and improved treatment strategies. However, there is still a lot of work to be done, with current treatment strategies succumbing to resistance, and other more rare cancers remaining undruggable. We are extremely grateful for the support from the Brain Tumor Foundation of Canada which will help accelerate and improve the progress towards novel, safer therapeutics, as well as to provide unique cancer research opportunities for our trainees. This project is focused on developing blood-brain-barrier permeable molecules to target medulloblastoma, an aggressive pediatric brain cancer. We hope to discover new therapies, perform cutting edge science, and inspire and foster the next generation of top-tier cancer researchers in Canada.”