Cord Blood Stem Cell Advances

A joint Israeli venture for a new cord blood stem cell product for leukemia and lymphoma patients has received the go-ahead for further study.

Tzvi Ben Gedalyahu , | updated: 2:37 PM

Gamida Cell logo
Gamida Cell logo
Israel news photo: Gamida Cell

A joint venture by Teva Pharmaceuticals and Gamida Cell for a new cord blood stem cell product for leukemia and lymphoma patients has received the go-ahead for further study by the independent Data Monitoring Committee (DMC).

The panel authorized continuing a Phase III Study of StemEx®, a cord blood stem Cell product, for Leukemia and Lymphoma.

The DMC said it has reviewed data collected from the first 70 patients enrolled in the pivotal Phase III clinical trial and has recommended to enroll more patients in the StemEx study. This marks the last protocol mandated DMC review of the product.

StemEx is being evaluated as a therapeutic treatment for adolescents and adults with blood cancers such as leukemia and lymphoma, who cannot find a family related matched bone marrow donor.

The DMC’s primary charge is to ensure that the study is conducted in an ethical manner that does not expose participants to undue risk. In addition, the Committee monitors the quality and overall conduct of the study and assesses whether to recommend that the sponsor modify or terminate an ongoing study, or whether to request further information for review.

The DMC had no major safety concerns and recommended that accrual on the study continue.

Dr. Yael Margolin, president and chief executive officer of Gamida Cell said, "The DMC’s recommendation to continue the StemEx study represents a very significant milestone. We are on track to complete enrollment of patients in a few months and to submit the regulatory files in the US and in Europe. The DMC has confirmed our resolve and confidence in the potential of StemEx to provide a good solution for patients with leukemia or lymphoma who need a bone marrow transplant but who do not have a family related matched bone marrow donor.”

StemEx is a graft of an expanded population of stem/progenitor cells, derived from part of a single unit of umbilical cord blood and transplanted by IV administration along with the remaining unit of non-manipulated cells.

The Gamida Cell-Teva Joint Venture expects to complete the enrollment of 100 patients in the StemEx study towards the end of the year and plans to submit a Biologic License Application to the FDA in 2012 as well as a Marketing Authorization Application.

A first market launch is planned for 2013.

Gamida Cell is a world leader in stem cell population expansion technologies and stem cell therapy products for transplantation and regenerative medicine. Its current shareholders include Elbit Imaging, Clal Biotechnology Industries, Israel Healthcare Venture, Teva Pharmaceuticals, Amgen, Denali Ventures and Auriga Ventures.