The Company Who Cried Clinical Trial,” blazoned a headline on Wired magazine’s blog network this summer, with the blogger calling attention to how often Geron has said it was on the verge of a first-of-its-kind clinical trial that entails transplanting a type of neural cell into spinal cord-injured patients. 

 

Even so, and with Geron researchers now saying the trial will begin in 2008, no one is tuning them out. Anticipation remains strong, not only because of what the test could portend for spinal cord repair, but because success would represent, according to Menlo Park, California-based Geron, the first time a product derived from embryonic stem cells (ES cells) has proved safe and effective for humans. This would be a boon for those who want to ease restrictions on federal funding for ES cell research. Should the trial fail, however, it could be a major setback for ES cells and the many labs hoping to channel them into countless therapies.

 

Such a product doesn’t come cheap. The investigational new drug application (IND) that Geron expects to file by year’s end will be some 20- to 30-thousand pages long. “Granted, this IND is larger than most,” notes Anthony Davies, Geron’s vice president of product development, “but that’s where the bar is, in terms of getting products into the clinic,” especially a product meant for the tricky confines of the spinal cord.

 

The research is part of Geron’s much larger, multiyear, human embryonic stem cell program, into which the company has sunk more than $100 million. Notes Davies, “This is biology; the raw materials are complex and expensive. This is the first time we”—or possibly anyone—“has tried to manufacture human embryonic stem cells under GMP [Good Manufacturing Practice Regulations] to meet FDA requirements.”

 

Geron has no products for patients yet, and the only return on investment to date comes from marketing its assay kits and selling cell lines through licensees. Still, Reni Benjamin, a research analyst at New York-based Rodman and Renshaw, remains optimistic about several prototypes inching through Geron’s pipeline. They include six different cell types that are slated for transplant therapies, respectively, for cardiovascular, central nervous system, pancreas, liver, and bone and joint disorders. Each cell type is derived from embryonic stem cells. 

 

“Geron is master of its domain,” observes Benjamin. “It’s been a leader in the stem cell space for quite some time, has been slowly making headway, and has been able to successfully raise money from investors repeatedly, such that now it has a cash hoard of about $217 million.” Benjamin projects that, on the heels of a net loss of around $31 million last year, Geron this year will burn through another $42 million. The company needs to spend money, he allows, if it’s to attain its goal of becoming a commercial drug company that manufactures off-the-shelf cell therapies for entire patient populations—no small undertaking when you consider that, in the area of spinal cord injury alone, there are as many as 50,000 new cases worldwide.