There was big news this week in the world of cellular drugs. One of the world’s largest pharma companies chose to drop it’s FDA approval pipeline for cell drugs. Let’s explore the top three reasons why.
Pfizer had partnered with a company called Athersys who has a culture expanded mesenchymal stem cell product called Multistem. This is an off the shelf stem cell therapy in a vial made from other people’s cells. IMHO, the top three reasons why this type of cell therapy didn’t make it were 1. profit over efficacy, 2. hyper-regulation, and 3. hype.
On the profit over efficacy front, I’ve blogged before on the problems with companies like Athersys. Caught up in trying to convince a company like Pfizer that cells could have pennies a dose manufacturing costs like chemical drugs, they participated and were one of the worst companies in the dosing arms race. This is where various companies each began claiming that they could grow more stem cells from a single donor than the last. At first it began in the thousands of doses, then it jumped to tens of thousands, then hundreds, and finally millions of doses. Athersys on their web-site still claims that Multistem has “Large scale production potential (e.g., millions of doses from each donor)”. Anyone who has ever been involved in the actual use of culture expanded stem cells clinically knows this number is crazy, in fact, a thousand doses is pushing reality. If Athersys or anyone else is actually getting 1,000,000 doses of cells from a donor, those cells are not helpful to patients. However, apparently Pfizer bought this hook line and sinker and realized it’s fatal error too late.
In the same category as ridiculous dosing numbers is the “allo” problem. Companies like Athersys, again to convince big pharma that cells could be made and used like drugs (rather than cells) have overlooked the mounting science that cells from another person still cause certain parts of the host’s immune system to activate, killing the transplanted cells. The company does seem to finally acknowledge this, but in code on their website in that they state “Not a permanent transplant – cells cleared from the body over time (like a drug)”. To the casual investor without a knowledge of how cells work, this statement might seem to make Multistem incredibly attractive. To anyone with more knowledge, it means, “we know that our cells are killed off by the host’s immune system and aren’t around for long, but we’re trying to make lemons look like lemonade”.
You can’t look at an autopsy of the death of the Athersys-Pfizer partnership without discussing regulation. The USFDA and now the EMA have both decided that many types of cell therapy should be regulated the same as chemical drugs. This has added a gigantic cost burden to the use of stem cells to help patients. While the cell therapy industry has argued until they’re blue in the face that this is the right move, many others have argued it’s the wrong move. In fact, without the financial muscle of huge pharma companies like Pfizer, the costs of FDA approval go from simply a market barrier to entry, to a crippling albatross.
Finally, the hype machine created around embryonic stem cells is partly to blame for the death of this partnership. Due to the religious right’s opposition to anything embryonic, to gain public support for their research, scientists created a marketing machine of professional groups second to none. They convinced the public and then investors that stem cells were magical and could cure everything known to man, rather than a great tool, useful to help patients in certain circumstances. Add in the pandering to big pharma as discussed above and the high cost of the regulatory burden and rather than cells being helpful tools, stem cells had to perform like magic fairy dust by making the blind see and the crippled walk to get insurance companies to want to pay the high price of admission. In the end, it looks like Pfizer finally realized that someone else’s stem cells in a vial weren’t performing like magic.
The upshot? As I’ve warned before, be very wary of investments in mass manufactured stem cell therapy drug companies. While a few will become the household names of the future and do quite well because they are focusing on low cost technologies or do have home run applications, most will fail miserably like Athersys for the reasons above.