Bone cancer researchers discover how to block, potentially treat osteosarcoma


In research funded in part by The Princess Margaret Cancer Foundation, scientists at the Princess Margaret Cancer Centre have discovered that blocking the master regulator of bone renewal stops osteosarcoma, the most common primary bone cancer in children and teens. Osteosarcoma is the disease that proved fatal for Canadian icon Terry Fox. 
The findings, published online in Science Translational Medicine, establish the importance and function of the bone master regulator, a protein known as RANKL, in bone cancer and set the stage to develop targeted therapy for patients. “We now understand the molecular basis of how RANKL drives osteosarcoma and believe this new information could potentially be rapidly translated into the clinic as a new therapy for patients,” stated principal investigator Dr. Rama Khokha, Senior Scientist at the Princess Margaret Cancer Centre.
“There is already a clinically-approved RANKL-blocking drug being used to treat other bone diseases. The next step is to determine whether this particular drug could be adapted to treat osteosarcoma and improve outcomes for these patients. As there has been little improvement in treating this type of bone cancer for the past 20 years, we are eager to find out.”
Cancer statistics in Canada and the U.S. show that osteosarcoma occurs most often in males aged 10 to 30 years. If the cancer is localized, with treatment, the five-year survival rate ranges from 60% to 80%; however, if the cancer spreads, the survival rate drops to between 15% and 30%.
Dr. Khokha began researching osteosarcoma more than a dozen years ago, inspired by Terry Fox, who lost his life to the disease at the age of 22 in 1981 during his cross-Canada run to raise money and awareness for cancer research.