Gee, I could have seen this one coming from a mile away…oh wait, I did see it coming. This week a company called Athersys reported that they missed both outcome endpoints in a trial using the company’s mass manufactured stem cells to treat strokes. The big story isn’t that the stem cells didn’t help stroke victims, but likely why they didn’t help.
Growing stem cells is a delicate thing, one that balances the final yield with stem cells that are grown so much that they’re no longer potent. I blogged on this issue way back in October of 2013. You see, anyone that has worked with culturing stem cells to greater numbers (like our cultured stem cell procedure that’s licensed down in Grand Cayman) knows that once you get past a certain point in growing cells, they don’t look so good and usually stop growing on their own. Hence, responsible cell culture only grows cells out a few passages (about 10-17 days max) to balance getting more with declining potency.
I picked up a few years ago that the numbers of cells that companies such as Athersys were reporting could be grown from a single donor strained credibility. For example, from a young donor, the best cell culture technicians could grow maybe 100 doses of stem cells. However, Athersys (and other companies) have been claiming that they can grow 10,000-100,000 doses or more from a single donor. Why? I don’t think it’s because the scientists at these companies don’t understand this concept, but it’s likely because of financial pressure applied by Wall-street. The FDA approval process has made cell therapy so much more expensive than it ever needed to be that it has forced companies to gain investment dollars by promising artificially low production costs. So while 100 doses from a donor has X cost, growing 100,000 from that donor costs a fraction of X per dose. However, recent research has shown that cells grown out even a little longer than a short time (beyond the 10 dose range) are dramatically less effective than fresh cells or those cultured for shorter periods (see link above for reference). In addition, the cells themselves get sick.
The upshot? Because other companies have traded common sense for stock price, expect to see many more failures of stem cell drug companies due to mass manufacturing techniques. What’s a biotech investor to do? If you see any company that claims to be able to grow more than 1,000 mesenchymal stem cell doses from a single donor…run. In addition, your biggest concern as an investor should be whether companies putting profit over potency will have a sentinel effect of destroying general investment in stem cells because of failed trial after failed trial.
The Regenexx-C procedure is not approved by the US FDA and is only offered in countries via license where culture expanded autologous cells are permitted via local regulations.