StemEx(R), A Gamida Cell-TEVA Joint Venture Cell Therapy Product,

StemEx(R), A Gamida Cell-TEVA Joint Venture Cell Therapy Product, Receives Orphan Medicinal Product Designation In The European Union


http://www.stemexstudy.com/index.html

What is the ExCell study?
ExCell is a clinical trial studying StemEx® as an alternative transplantation option for adolescents and adults with leukemia, lymphoma and other high risk hematological malignancies, who are unable to find  a matched, related bone marrow donor.

The ExCell study is sponsored by the Gamida Cell-Teva Joint Venture. The JV is committed to advancing the clinical development of StemEx® and improving the treatment of adolescents and adults with hematological malignancies. 

http://www.gamida-cell.com/stemex.asp

Hematopoeitic Stem Cell Transplantations
Bone marrow transplantation is a life saving treatment for many types of leukemia and lymphoma. However, matched bone marrow donors are identified for only approximately 30% of the patients in need of a bone marrow transplant. The other 70% are not as fortunate and cannot find a match. The problem is even more acute for minority population patients, whose chances of finding a matched, unrelated marrow donor are extremely small.

Research shows that cells in umbilical cord blood have been used successfully to overcome the challenge of finding a matched donor for patients. Umbilical cord blood stem cells offer a viable therapeutic option for leukemia and lymphoma patients without an appropriate matched donor. Umbilical cord blood presents two important advantages: it is readily available and has a lower histocompatibility (matching) requirement. Therefore almost all patients will be able to identify a matched cord blood donor.

However, the limited cell dose found in cord blood units poses a significant challenge: the need for a large enough supply of healthy stem cells to stimulate the regeneration of bone marrow or the tissue of a particular organ and to achieve successful engraftment in adult and adolescent patients. Gamida Cell’s technology expands populations of cord blood stem cells with limited differentiation to create a therapeutic dose to treat such patients.

Gamida Cell's Phase III Product: StemEx® for Leukemia and Lymphoma
Gamida Cell's phase III product, StemEx, offers a novel solution for patients with various critical hematological diseases such as leukemia and lymphoma who cannot find a matched bone marrow donor and are in need of a transplant. StemEx is composed of ex vivo expanded cord blood stem/progenitor cells which are transplanted with non-expanded cells from the same unit. StemEx production utilizes modification of the level of free copper in the cells, to allow large-scale, ex vivo self-renewal of stem/progenitor cells, with limited differentiation, in a reasonably short time. The StemEx product and its underlying technology are covered by two broad US patents: No. 6,887,704 and No. 09/463,320, and numerous other related patents granted worldwide.

Partnership with Teva Pharmaceuticals
StemEx is being developed by the Gamida-Cell-Teva Joint Venture which owns the commercialization and marketing rights for the product. The JV is now enrolling for the ExCell trial, multi-center, multi-national, historical cohort controlled, pivotal phase III study to evaluate efficacy and safety of transplantation of StemEx in subjects with hematologic malignancies following myeloablative therapy (http://stemexstudy.com/). The clinical protocol received a Special Protocol Assessment with the FDA, in October 2006. The same agency granted StemEx orphan drug designation in March 2005.

Andy and all others : my question is ,  Is this study have potential to cure Thalassemia ....

Best Regards
Take Care
Umair