The day after Geron news: a realistic outlook for the stem cell field

Yesterday Geron announced it would be immediately stopping its stem cell research program.

What does this mean?

While this program has (or should I say “had”) a number of elements, at its heart was of course its hESC-based OPC drug (GRNOPC1) for spinal cord injury, which was in an FDA-approved Phase I Clinical Trial that had already enrolled 4 patients.

While the company will continue to follow these patients and keep a limited core staff of stem cell employees for a short period of time, no more patients will be treated with GRNOPC1, at least not via Geron.

Realistically, what does this mean for the stem cell field and the hopes of using stem cells to treat patients? Particularly for embryonic stem cell-based treatments?

I think there is no way to spin this as a good thing and in fact it is terrible news. 

Geron is a wonderful company full of smart, intelligent people. It is a mixture of great scientists and also business people who know what they are doing. Their pipeline of cancer drugs is quite intriguing and promising, and you can bet that their reasoning for focusing on the cancer programs is that financially they believed these programs had a better chance for rapidly become profitable.

It doesn’t mean their stem cell program was weak, but rather I think what it means is that the stem cell program, including the part based on the GRNOPC1 drug, required more patience and resources than they felt they could continue to invest in it. What makes this particularly surprising is that they had most of a $25 million loan from CIRM unspent. This event also illustrates the importance of federal support of stem cell research including embryonic stem cell research.

Overall, this illustrates a formidable roadblock in the area of trying to translate stem cell-based drugs to the bedside. It is a costly and time consuming process, perhaps from the perspective of biotech executives, too costly and too time consuming. If biotechs opt out of stem cell research, then we are in big trouble as a field in terms of making stem cell-based drugs a reality.

Most likely before their public announcement, Geron tried to sell GRNOPC1 either as a stand-alone or via a partnership, and could not find an interested party soon enough. That does not mean there are no interested parties (in fact I hear there are), but it is still a cause for concern.

Now the focus turns to Advanced Cell Technology (ACT). People are very worried that in the long run that ACT will not be able to sustain its stem cell program either due to financial pressures. However, I think there are two key reasons for hope that ACT will persist.

Why?

First of all, the leadership of ACT including stem cell superstar Bob Lanza are perhaps more deeply invested in making their stem cell product a reality. Second, I think for practical reasons, that ACT’s trials can realistically be predicted to proceed must faster than Geron’s would have. I think ACT is more battle hardened from their experiences over the years. However, let’s not kid ourselves, ACT faces a tough financial road as well to get their product to the clinic. Both ACT (symbol ACTC) and Geron (symbol GERN) stocks have lost almost 50% of their value just in 2011 so far. As scientists, we may not think that that matters, but realistically it does.

Still my gut says ACT will persist and ultimately succeed. Of course as scientists, we are taught not to go on gut feelings, but sometimes you have to.