WASHINGTON, Oct. 19 (UPI) -- Novocell said Thursday it has determined the necessary conditions for turning human embryonic stem cells into insulin-producing cells, a finding that moves the prospect of using these cells to treat diabetes closer to the clinic.
Some work remains to be done before the cells are ready for patients, but the company anticipates phase 1/2 trials could begin as early as 2009.
"Our plan is to submit an IND towards the end of 2008 and get approval for phase 1/2 trials in the beginning of 2009," said Emmanuel Baetge, Novocell's chief scientific officer and senior author of the study, which Nature Biotechnology published online Thursday.
Baetge told United Press International the company has already met with the Food and Drug Administration about its human embryonic stem cell line, which was isolated under good manufacturing practices.
The company plans to conduct additional animal studies the rest of this year and into next year. This would put it on track to perform scale-up in 2007 and conduct safety and efficacy studies in 2008, Baetge said.
In the study, the researchers developed a process for differentiating human embryonic stem cells into insulin-producing pancreatic endocrine cells. The technique, which mimics the normal development that occurs in the body, could be used for generating insulin-producing cells that could be transplanted into type 1 diabetes patients.
Baetge said it's important to note that although the cells they generated produced insulin at nearly the levels found in adult pancreatic islet cells, they do not produce the hormone efficiently in response to glucose, or blood sugar, a critical requirement if they are to have clinical utility.
However, the cells appear to be at an earlier developmental stage, so the solution to getting them to respond to glucose may just be more development time.
"That's exactly what you find in normal human development," Baetge said. "Only after birth do the beta cells begin consistently responding to glucose."
Robert Lanza, vice president of research and scientific development at Advanced Cell Technology, which is also trying to develop therapies based on embryonic stem cells, told UPI the research was a major achievement.
"It's very impressive," Lanza said. But he noted that Novocell will have to make a few tweaks before the cells are ready for the clinic.
"They still need to get these cells to respond to glucose," he said. "Until they do that, they're not usable."
The efficiency of the technique also will need to be improved because only a small percent of the cells they started with actually produced insulin at the end.
"To expand these cells into sufficient numbers to create a practical therapy is going to be another hurdle," Lanza said.
Another potential problem is immune rejection if cells from one person are placed into another.
"Immune rejection is a problem that is going to plaque the entire field," Lanza said. "We need to very much work on solving that problem."
Novocell already may have found a way around that issue.
The company's cell encapsulation technology, which is designed to protect the cells from the body's immune system, is currently in clinical trials.
"We already have two patients implanted and they're completely off immunosuppression drugs," Baetge said. The patients have encapsulated human primary islet allografts implanted under their skin.
"So far there are no severe adverse events," he said, adding that the company plans to continue monitoring the patients without immunosuppressive drug administration, until loss of graft function.
