When you support research, you are giving a gift of hope. You are sending a message that you care about the thousands of boys and young men who face Duchenne muscular dystrophy – the most common fatal genetic disease diagnosed in childhood.
For more than a decade, Dr. Patrick Gunning has been researching the use of small molecules to treat – and potentially cure – aggressive forms of cancer. He holds the Canada Research Chair in Medicinal Chemistry at the University of Toronto Mississauga (UTM). His accomplished team – known as the Gunning Group – has seen significant success in pre-clinical studies.
In 2016, they learned that two papers had been published the year before in high profile science journals indicating a possible link between the protein that the Gunning Group had been targeting and Duchenne muscular dystrophy.
Because the link between this protein – known as STAT3 – and Duchenne had only recently been proposed, it had not yet been fully explored. This created an incredible opportunity for the first study of its kind in the world to be launched at UTM in partnership with Jesse’s Journey.
What is STAT3?
It is a protein that is found in almost all cells in the human body. When we’re young, this protein is very active and plays a key part in growth. When we reach adulthood and stop growing, STAT3 slows down. However, in some people STAT3 stays hyper-activated causing overexpression of the protein. This is what is known to happen in certain forms of cancer. The Gunning Group has developed a molecule that blocks STAT3 activity. This molecule attaches to the protein and shuts it down, effectively halting the damage the hyper-activated protein is causing.
In Duchenne, it was discovered that activated STAT3 may be critical in determining the fate of the muscle stems cells: produce more stem cells or permanently convert into a mature muscle stem cell. Using the molecules we have created, the function of STAT3 could be blocked, thereby forcing the stem cells to reproduce and replenish the pool of the muscle cells lost to the disease.
In 2017, Jesse’s Journey committed $300,000 to the Gunning Group through UTM to study STAT3 inhibitor molecules in Duchenne. They are committed to finding a viable molecule that can eventually be used in human clinical trials. (Click here to watch a presentation given by Dr. Gunning to the Jesse’s Journey board of directors)
To make this project a reality, we launched the Journey to a Cure Campaign. The goal is to raise $300,000 to complete the funding commitment to the Gunning Group as they determine the effectiveness of STAT3 inhibitors, and other small molecules, in the treatment of Duchenne. The Gunning Group is already hard at work on this study, and we need your help. To date, donors have committed more than $270,000 meaning we are 90% of the way to our goal.
By making a gift to help support this innovative team of researchers, you can give hope to the thousands of boys and young men who face Duchenne muscular dystrophy.
Jesse Davidson once said, “When researchers work together, miracles can happen.” Today, in Mississauga, Ontario and all around the world, some of the brightest and most dedicated researchers are working together to find that miracle. Because parents should not outlive their children.
For information about other ways to give, please click here.