After decades of controversy and frustratingly slow progress, the stem cell field seems poised for breakthroughs in a range of diseases. One by one, obstacles such as tumorigenicity of embryonic stem cells and immune rejection of allogeneic transplants are giving way to focused study and innovation.
At the “World Stem Cells Regenerative Medicine Congress”, the regenerative medicine community will meet to exchange the latest news and progress in cell therapy. Topics include improvements in cord blood expansion, surprising new ways to harvest stem cells, progress in engraftment and vascularization of stem cells for heart patients, and groundbreaking research using embryonic stem cells to restore sight in macular degeneration.
Yael Margolin, Ph.D., president and CEO of Gamida Cell, offers an update to the company’s pivotal trial of its expanded umbilical cord stem cell product, StemEx, which was just completed, with expected market launch in 2013.
StemEx is made of hematopoietic progenitor cells isolated from a unit of umbilical cord blood. During expansion, the cells are treated with the copper chelator tetraethylenepentamine (TEPA), which has been shown to reduce the level of copper salts in the cells.
Copper deficiency has been shown to mimic refractory anemia, which is characterized by an excess of immature cells and a reduction in differentiated cells. Gamida uses TEPA to harness that effect, boosting the number of hematopoietic progenitor cells.
The company is now completing enrollment in a Phase III trial of StemEx. The trial is open-label with a historical control cohort. Its design was approved by the FDA under a special protocol assessment.
In order to meet the supply need for the trial, Gamida has manufactured more than 100 StemEx batches in three centralized GMP facilities.
“The data demonstrates a robust manufacturing process, providing patients with a significant increase in the dose of infused CD34+ cells,” says Dr. Margolin. Gamida has also developed an efficient logistical infrastructure to support the clinical implementation of StemEx.
Cryopreservation has also been a concern in the ramp up to Phase III trials. Gamida successfully developed a cryo-preservation method for the cells, which will make it possible to use a single, central manufacturing site for global distribution, significantly reducing costs.