Gamida Cell’s Flagship Product: StemEx®
Matched donors are located for approximately 50% of the patients in need of a bone marrow transplant. The other 50% are not as fortunate and cannot find a match. The best that can be done is to administer drugs that will palliate their situation. At present there are no existing cures.
Research shows that cells in umbilical cord blood offer a reasonable answer to the problem of finding a matched donor and have been used successfully to overcome the challenge of finding a matched donor for patients. Umbilical cord blood stem cell therapies in particular, offer a viable therapeutic option to leukemia patients without an appropriate matched donor, since cord blood is readily available, has a lower histocompatibility (matching) requirement, and reduces the risk of GVHD.
However, the limited volume of cord blood units poses a new challenge: the need for a large enough supply of cells to make a successful engraftment in large children and adults. The strategy for stem cell therapy is to supply the body with enough healthy stem cells to stimulate the regeneration of bone marrow or the tissue of a particular organ. Gamida Cell’s technology expands these populations of cord blood stem cells with limited differentiation to create a population large enough to treat larger children and adults. This expanded population of cord blood cells is called StemEx®. StemEx®, Gamida Cell’s flagship product was tested in a Phase I/II trial for the treatment of leukemia and lymphoma that successfully concluded with promising results in January 2005. The company is now planning for an international pivotal study of the product to commence in 2005. StemEx® was granted an orphan drug designation by the FDA in March 2006.