Just over a month ago I blogged about Gamida Cell's pivotal trial for their flagship product, StemExÂ®. Today I received news from Yael Margolin, CEO, Gamida Cell that the pivotal trial has completed its patient recruitment and clinical data is expected in Q4 of 2012.
In the lead up to the World Stem Cell & Regenerative Medicine Congress, we are looking to give our delegates an insight into what they can expect from this years elite speaker faculty and the industry shaping discussions that will be taking place across the 21st-23rd May 2012. Registration is open but the current early bird rate is only available until 13th April 2012.
Below is the full whitepaper released by Gamida Cell this week:
Each year, approximately 60,000 patients with leukemia or lymphoma need a bone marrow transplantation. Of these, only approximately 25,000 are transplanted, primarily because the other 35,000 are unable to find a fully matched bone marrow donor. Cord blood transplantation (CBT) does not require full tissue matching and is considered a good alternative for transplantation. However, CBT is limited by a low number of TNC and CD34+ cells, influencing the incidence and rate of hematopoietic recovery and risk of early transplant-related mortality. Ex-vivo expansion is a strategy employed to increase the number of stem/progenitor cells and improve clinical outcomes.
StemEx is a graft of stem/progenitor cells isolated and expanded from a portion of a single unit of umbilical cord blood (CBU) and transplanted in combination with non-expanded cells from the same unit. The portion of umbilical stem cells is expanded using Gamida Cell's proprietary copper chelator technology.
StemEx is poised to answer the dire unmet clinical need in the field of bone marrow transplantation by supplying alternative cell grafts to the 50,000 patients with hematological malignancies, who are indicated for transplantation but do not have a matched family related bone marrow donor. The market potential of StemEx is estimated at more than one billion USD.
StemEx has been studied as an alternative therapeutic treatment for hematological malignancies in an international, Phase III, pivotal registration clinical trial. This is an open study with a historical control cohort: the design is approved by the FDA under SPA. The pivotal study has completed recruitment. Safety and efficacy clinical outcomes of the study will be available in Q4/2012.
To date, more than 100 StemEx batches were manufactured in 3 centralized GMP facilities. Data available for the first 88 batches show a median fold expansion of TNC; CD34+ cells and CFU over culture input were 399 (range 52-764), 75 (6-280) and 107 (43-662), respectively. Since only a portion of the CBU was expanded, patients were infused with a median 8.4 fold increase (0.8-90.3) in the number of CD34+ cells infused over the number that would be infused from the entire CBU without expansion. The CFU potential of culture seeded CD133+ cells measured at day-0 of production indicates the expansion potential of the cryopreserved CB cells. In all six batches which failed to expand, day-0 CFU was low, while day-0 CFU of all successfully expanded batches was within specification ranges. This information, available before patient myeloablation, strengthens the clinical applicability of StemEx.
Gamida Cell has developed a robust logistical infrastructure to support the clinical implementation of StemEx. All StemEx batches were successfully delivered and infused to the patients.
A frozen StemEx product has also been developed. The added flexibility in the timing of transplantation allows for changes resulting from patient disease progression and/or complications. With the challenges of an ex-vivo expanded product being successfully met in the current registration trial, and the successful development of a frozen product, StemEx is demonstrating its feasibility as a quality alternative stem cell source for allogeneic HSCT.