NIH Stem Cell Definition Change 'Hugely Important'
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.