ACT’s single-cell blastomere technology (“blastomere technique”) is a patented, proprietary method for generating human embryonic stem cells (hESCs) without damage to the embryo, thus directly addressing the issue that has made embryonic stem cell research a controversial topic in some quarters. Documented in scientific journals Nature, Cell Stem Cell and elsewhere, the technique uses a single-cell biopsy approach to generate hESCs.
The technique involves removal of a single blastomere (cell) from an eight-cell stage embryo, which can then generate stem cell lines that have the same characteristics as the embryo. The biopsy procedure does not damage or destroy the embryo, nor does it interfere with the embryo’s developmental potential. In fact, the technique is very similar to pre-implantation genetic diagnosis (PGD). PGD is widely used during in vitro fertilization (IVF), and its safety and efficacy are amply demonstrated by the thousands of healthy people worldwide whose parents used PGD.
ACT’s single-blastomere technique is a viable alternative to other techniques that derive hESCs from the inner cell mass of later-stage embryos, thus destroying their potential for further development and raising ethical objections from various parties.
The U.S. National Institutes of Health (NIH) has called for “aggressively pursuing an assessment of the potential of alternative sources of pluripotent stem cell lines, including [...] single cell embryo biopsy” and has suggested that ACT’s blastomere program may qualify as an alternative method. Should the blastomere technique satisfy the NIH’s qualifications, ACT could qualify for federal funding. The NIH has also proposed expanding its definition of hESCs for funding purposes, in part to accommodate ACT’s lines derived using its single-cell blastomere technique, as as covered in the New York Times, here.
In February of this year ACT was issued a patent on its blastomere technique: Patent Number 7,893,315 broadly covers the technology.
For clinical applications, stem cells must first differentiate into a specific cell type, independent of the source from which they were derived, prior to transplantation into the body of a patient. In a controlled in vitro setting, stem cells can be differentiated into a variety of different cell types, insulin-producing cells, hepatocyte-like cells, cardiac myocytes, haematopoetic cells and many more. To read more about stem cell differentiation, please click here .
A 2009 publication demonstrates the long-term safety and function of retinal pigment epithelium (RPE) cells from human embryonic stem cells (hESCs). These cells were differentiated from single-blastomere hESC lines and cultured following current Good Manufacturing Practices (cGMPs) and current Good Tissue Practices (cGTPs) to produce consistently safe and effective products for clinical use.
Cellular reprogramming, also known as transdifferentiation or dedifferentiation, describes a process that enables the reprogramming of differentiated cells into a pluripotent state. This technology may be able to reprogram the patient’s own cells, such as skin cells, in the laboratory into pluripotent or multipotent cells. Pluripotent cells can differentiate into derivatives of all three germ layers, whereas multipotent cells can differentiate into multiple lineages that can give rise to a variety of tissues or differentiated cells. These cells will be histocompatible with the patient and may therefore be therapeutically relevant in treating diseased or destroyed tissues. To read more about cellular reprogramming, please click here.
This program is currently in preclinical development.