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all these hoops.” For example, ACT had to prove that it could deliver the cells through a fine-gauge needle without destroying them.
The cells ACT uses are differentiated, meaning they’ve already evolved into retinal cells. Still, the FDA instructed the company’s scientists to perform “spiking studies” to show in animals that nothing would go wrong if a few undifferentiated cells accidentally made it into the treatment. The reason for the agency’s concern, Lanza says, is that undifferentiated embryonic stem cells can turn into teratomas—a type of tumor. “We never got a teratoma” in the animal studies, Lanza says.
One advantage of working with eye cells, Lanza says, is that they can be easily observed with high-powered imaging tools. “We can see if there’s engraftment of the cells in the eye,” Lanza says. “We can see if anything adverse happens.” Although it’s too early to make any conclusions about the first two patients, the procedures, Lanza says, “couldn’t have gone more smoothly.”
ACT has reached this milestone with precious little financial resources. In 2005, ACT wanted to raise money through an initial public offering, but investment bankers were so antsy about embryonic stem cells that the company had to find another route onto the trading floor. So it reverse merged into a shell company that had once been a manufacturer of Hopi Indian dolls. From that less-than-glamorous Wall Street debut, ACT was able to take its new shares and raise $18 million. But the stock has taken a beating over the years, falling from a high of $7 in 2005 to just $0.18 today.
ACT is now surviving on about $13 million in cash, some of which was raised by selling preferred shares in private funding rounds. The company lost $54 million on just $725,044 in revenues from licensing fees in 2010.
The financial woes haven’t dampened Lanza’s spirits, though. Even though he won’t know until the end of the summer if ACT is cleared to move forward and treat more patients with RPE cells, he’s already dreaming of a day when the treatment will be able to be used as a sight-saver. “Our main goal now is to show the cells are safe,” Lanza says. “But ultimately we want to go to younger patients and prevent vision loss all together.”
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