Posted on Yahoo by worcester
NIH Stem Cell Definition Change 'Hugely Important'
BioWorld Today
23 February 2010
By Donna Young,, Washington Editor
[What follows is the full text of the news story.]
WASHINGTON - The National Institute of Health's proposal to revise its definition of human embryonic stem cells (hESC) to permit earlier stage embryos may at first glance appear to be a minor technical change. But to firms like Advanced Cell Technology Inc., which has sunk a "fortune" into developing the cell lines, it is "hugely important," said Robert Lanza, chief scientific officer for the Worcester, Mass.-based biotech.
The NIH late Friday afternoon proposed changing the definition of hESCs issued in guidelines last July from "cells that are derived from the inner cell mass of blastocyst stage human embryos" to "pluripotent cells that are derived from early stage human embryos, up to and including the blastocyst stage." (See BioWorld Today, July 7, 2009, and Feb. 22, 2010.)
That change would allow ACT's five single-blastomere lines currently under review at the NIH to receive federal funding for research, if approved by the agency. ACT currently has an investigational new drug application (IND) under review at the FDA for a Phase I/II trial using its MA09 single-blastomere line to treat Stargardt disease, a genetic condition and the leading cause of juvenile blindness in the U.S., Lanza told BioWorld Today.
The IND currently is on hold while ACT addresses some of the FDA's questions, but Lanza said the company is hoping to initiate the study by the end of the third quarter.
The hESC definition change also would affect ACT's four other NED, or "no embryo destruction," lines awaiting placement on the NIH registry: NED1, NED2, NED3 and NED4.
ACT's approach, Lanza said, is "simply plucking one cell out of the embryo in a way that doesn't harm the embryo and then you can create a line from that cell."
The MA09 line is the one the firm has been using for its "master bank" for its clinical trials, he noted. "What we have found, interestingly, is that these lines have opportunities superior to the other embryonic stem cell lines that we have been studying," he said. ACT's five lines affected by the NIH's proposed change were generated under good manufacturing practice conditions, "so that they are suitable for use in patients," Lanza said. "It turns out that when we look at embryonic stem cells side by side, that our best performers, the cells that do some of these tricks most efficiently, actually were the single-cell derived embryos," he said. "So if we want to make various endothelial cells or vascular cells to repair damage, we find that the single blastomere lines, at least one or two of them, are five times greater in efficiently generating the cells. That is huge when you talking about having to expand cells into very large numbers for medical therapy."
Lanza insisted that "it shouldn't really matter what cells" hESC lines are derived from.
"These are coming from the spirit of the law that these are leftover embryos with the proper consent forms that were discarded from IVF clinics or from leftover embryos that were not being used," he said. "So whether or not you generate your lines from this or that cell from the embryo really shouldn't matter," Lanza said.
And because the single blastomere method does not require the destruction of the embryo, "it could be strongly argued that these lines are far more ethical," he said.
Given the "many years" of data ACT has collected, including lifetime animal studies to show the cells do not form teratomas, it would have been "a real shame" if the NIH had ignored the pleas from the company and other researchers to revise the guidelines, Lanza said.
"You can imagine changing the official guidelines is not trivial," he said. "So my hat is off to them for correcting this. They are doing the right thing here. We are very pleased that they were willing to make this correction."
Lanza noted that his firm and its collaborators have applied for several NIH grants, which currently are on hold until ACT's lines are approved.
The company's efforts in winning federal funding, he said, "got caught" in President Bush's 2001 order that restricted federal funding for hESC research, which was lifted last March by President Obama. (See BioWorld Today, March 10, 2009, and March 11, 2009.)
The firm had a solicitation at the Department of Defense for $5.6 million to create universal blood, but none of the Bush lines were O negative, and ACT was unable to get the White House to sign off on permitting the use of the company's single blastomere technique.
"They let the clock run out on it," Lanza said. If the NIH revises it guidelines and approves ACT's five single-blastomere lines, it would open the possibility that the firm could potentially get the DoD to reconsider, he added.
Meanwhile, the company is in discussions with the Foundation for Fighting Blindness for appropriated funding from DoD to back ACT's clinical trial.
"There are many, many people who could greatly benefit from this technology," Lanza said. "It would be very sad for us not to be able to move ahead and try to help as many people as possible."
The NIH proposal is open for public comment for 30 days.
