For the first time ever at BIO International, the largest annual biotechnology conference in North America, stem cells and regenerative medicine are being showcased today in an all-day forum. This morning kicked off with a panel discussing how stem cells are shifting the paradigm in how we research disease, discover drugs and treat patients.
With a nearly full room, former CIRM president Alan Trounson led four panelists through a lively discussion of how stem cells are accelerating the discovery of the fundamental mechanisms of disease, while also helping to make drug discovery a much more targeted process and, most importantly, beginning to deliver life-changing therapies to patients.
Catriona Jamieson, a CIRM grantee from the University of California, San Diego, showed how she had uncovered how cancer cells develop stem cell-like properties in order to evade treatment. She has, in turn, found three molecular pathways the cells use to make this transformation and has drugs being tested to block those pathways. But not everyone responds to those various drugs the same way, so her goal is to analyze the genetics of each patient and deliver what she has dubbed “precision regenerative medicine.”
Next, Eric Michael David from Organovo talked about the company’s ability to 3-D print small portions of liver or kidney tissue and have it function like the real thing. The long-term goal is to create tissue of sufficient quality to implant in patients, but until that is perfected their system is being widely used to screen potential drugs to see how they affect the various tissues. Understanding this potential toxicity early stands to save drug companies millions—and potentially billions—of dollars by not taking drugs destined to fail into costly phase 3 large-scale clinical trials. Or, even worse, find out about toxicity through surveillance after the drug is on the market with the associated product liability costs.
David said that between 1990 and 2010 160 drugs failed in phase 3 or post marketing, and unfortunately all the drugs in a company’s development pipeline share the cost of those failures. This gives new stem cell technologies the ability to significantly drive down drug costs.
Then, Donna Skerret from Mesoblast, which may be the largest stem cell company in the world, made it clear that while mesenchymal stem cells, which form the basis of their products have the ability to impact several diseases, we still don’t know the full mechanism for all those effects. They can reduce scar formation and moderate an overactive immune response, but the exact method of action for each still needs to be worked out. She called them drug delivery devices because they secrete various proteins that affect the cells around them. Mesoblast currently has therapies in phase 3 clinical trials for heart disease and other therapies in late stage phase 2 trials.
Perry Karsen of Celgene talked about his company’s work with placental-derived stem cells. He too discussed ongoing and much needed work to understand why they are having some of the effects they are having in order to maximize their therapeutic potential. But even now, he said, “We see cell therapies as having the ability to completely change how medicine is practiced by the end of the decade.”
All the panelists talked about needing to use what Karsen called “the full research ecosystem,” forming collaborations between academic researchers and industry. Donna noted that Mesoblast is relying on academic partners to help define the mechanism of action of their cells.
Forming these partnerships to accelerate getting treatments to patients has always been a core part of CIRM’s function.
An outline of all five panels of the Regenerative Medicine Forum can be seen here. We organized the day along with the Alliance for Regenerative Medicine and the International Society for Stem Cell Research.