On July 14, Advanced Cell Technology (ACT)—a long-struggling developer of therapies derived from human embryonic stem cells—started its first human trials, by treating two patients suffering from blinding eye diseases. The company’s chief scientific officer, Robert Lanza, was still on a high when reached by phone in his Marlborough, MA, office a few days later. “It’s vindication,” he says. “In the early days, we were called murderers. We almost went under a few times. This was not easy.”
That may be an understatement. Ever since human embryonic stem cells were first isolated 13 years ago, ACT (OTCBB: ACTC) has been laboring to capitalize on the cells’ talent for transforming themselves into any tissue in the body. Over the years, Lanza and his colleagues have been blamed for everything from overconfidence to unethical science—the latter coming from conservative politicians who wanted to ban such research because the cells are derived from unborn embryos. But ACT charged ahead, raising money from hedge funds and other risk-hungry investors, and concentrating on using embryonic stem cells to regenerate retinal pigment epithelial (RPE) cells, which form a tissue that protects the eye’s photoreceptors.
ACT was cleared by the FDA to test its RPE cells in two separate trials: one in patients suffering from Stargardt’s macular dystrophy and the other in those with dry age-related macular degeneration (AMD). Stargardt’s affects young people, and AMD strikes the elderly, but they are both marked by the degeneration of the RPE cell layer. And both are leading causes of blindness. ACT was the second company cleared by the FDA to test an embryonic stem cell-derived therapy, the first being Geron (NASDAQ: GERN), the Menlo Park, CA-based company that’s testing its cells in patients with spinal cord injuries.
ACT’s first two patients were treated at the David Geffen School of Medicine at UCLA, and are now being watched for six weeks to ensure the cells don’t cause any dangerous side effects. Lanza explains that each trial will include 12 patients, and the doses will escalate over time. If no adverse events are observed in the first set of patients—who will receive the smallest dose of 50,000 RPE cells—the next set of patients will receive 100,000 cells. Then they’ll be observed, and so on, until the highest dose of 200,000 cells is reached.
Lanza says he was both intimidated and impressed by the FDA’s thorough review of ACT’s trial regimen. “They know as much as I do about embryonic stem cells,” he says. “They put us through 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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