Recent Advances in Stem Cell Expansion Technology
Success with cord blood stem cell expansion technology may allow cord blood units considered suboptimal by today’s standards, in terms of stem cell counts, to be used clinically in the future. For example, it may allow single cord blood units, which are currently too small, to be used for adult transplantation.
Gamida Cell, an Israeli company, is developing technology for the expansion of cord blood stem cells. Its product, NiCord®, is currently in Phase 1/2 clinical trials for blood cancers and sickle cell disease. NiCord® is derived from a single cord blood unit that has been expanded in culture and enriched with stem and progenitor cells using Gamida Cell’s proprietary NAM technology. In 2013, Gamida Cell announced results of a Phase 1/2 clinical trial using NiCord® in patients with blood cancers. In this trial, 11 patients were co-transplanted with NiCord® and an unmanipulated cord blood unit. Eight patients engrafted rapidly with NiCord® in a median time of 10.5 days while two patients engrafted with the unmanipulated CB unit in ~22 days. NiCord® also provided long-term engraftment (over two years) to the eight patients who experienced the rapid, early NiCord® engraftment. One patient experienced primary graft failure.
The results of this clinical trial paved the way to a second Phase 1/2 trial, which is currently ongoing. This trial will enroll up to 20 patients with blood cancers, and the patients will be transplanted with NiCord® alone instead of a co-transplant with NiCord® and an unmanipulated CB unit. Additionally, Gamida Cell recently announced that the first person has been successfully transplanted with cryopreserved (frozen) NiCord® in this study and that, after thaw, NiCord® maintained the advantage of “fresh”NiCord® in demonstrating very rapid engraftment.
Until now, NiCord® had always been transplanted as a “fresh” product that had to be administered into the patient within hours of its production. The use of a “fresh” product thus limited the location of production sites to the vicinity of participating transplant centres. Additionally, the production of NiCord® had to be strictly co-ordinated with the patient’s preparation for transplantation. The development of technology to cryopreserve NiCord® has several benefits, including the elimination of the need to have the production site located in close proximity to the medical centre. NiCord® can now be produced at one site and provided to transplant centres around the world, allowing more transplant centres to participate in their clinical trials. Also, cryopreserved NiCord® will allow greater flexibility in the timing of the patient transplant. Any delay in the transplant, if required, can now be accommodated since the frozen product is stable for prolonged periods of time in that state.
Results of Gamida Cell’s ongoing trials are eagerly anticipated in 2016.