Many readers may already have heard of Health Canada’s approval of Osiris’ Prochymal, a mesenchymal stem cell treatment for severe cases of pediatric Graft versus Host Disease (GvHD), with plenty of coverage circulating around the web. GvHD is a disease where transplanted bone marrow generates an immune system that attacks cells of the recipient. The approval comes with much fanfare; in fact, it’s being hailed with classic Canadian pride as the World’s First Approved Stem Cell Drug by the National Post.
Prochymal consists of human mesenchymal stem cells harvested from healthy adult donors that are expanded and immune suppressed. When infused intravenously, they usually don’t trigger host immune response and help to inhibit any existing inflammatory response and encourage repair of previously inflamed tissues. It’s this activity that yielded a positive change in about 60% of the children with GvHD in a small clinical trial.
This approval is a small, yet very significant, win that slowly increases the awareness of therapeutic stem cell technologies with regulators and the public. Prochymal failed two major clinical trials in the United States in 2009 and this Canadian approval for the most severe cases of childhood GvHD will lead the company to seek approval with the FDA.
Stem cell based technologies and treatments are maturing and living up to their promise, and for today at least the future looks bright.
Editor’s note: Rather fitting that this news should arrive just ahead of International Clinical Trials Day (which took place on May 20), as Prochymal aptly demonstrates the length of time it takes to move a therapy through clinical trials – phase I/II trials of Prochymal began in 2000 – 12 years ago. For those interested in reading more about the process of moving therapies from bench to bedside, a European consortium of stem cell researchers also released a graphic story over the weekend. Hope Beyond Hype describes the process to lay audiences and is currently available in English, but will be translated into French, Spanish, Italian and German with plans to also launch an interactive version.