Advancements in Cord Blood Expansion Technologies

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In Brief:  
  • Gamida Cell received FDA approval for its allogeneic cell therapy, Omisirge:  a new expanded cord blood cell source for stem cell transplantation. 
  • A Phase 3 clinical trial showed faster neutrophil engraftment and reduced bacterial and fungal infections as compared to standard cord blood transplants. 
  • Omisirge may increase access to stem cell transplants, including among patients from diverse backgrounds.

Expanding Cell Counts

Historically, usage of cord blood for transplants have been limited by the comparatively lower number of cells as compared to other sources (e.g. bone marrow, peripheral blood apheresis).1 Efforts to expand the number of therapeutic cells in cord blood has been researched for decades and now those efforts are set to bear fruit.

In April 2023, Gamida Cell received FDA approval for its allogeneic cell therapy, Omisirge:  a new expanded cord blood cell source for stem cell transplantation. A Phase 3 clinical trial showed faster neutrophil engraftment and reduced bacterial and fungal infections as compared to standard cord blood transplants. Omisirge may increase access to stem cell transplants, including among patients from diverse backgrounds

Gamida Cell investigators showed the expansion of the umbilical cord blood (UCB) stem cells and inhibition of differentiation of the stem cells using nicotinamide in cell culture.2 Median CD34+ cell expansion was 130-fold.3  The product is intended for use in adult and pediatric patients 12 years and older with hematologic malignancies who are undergoing umbilical cord blood transplantation following myeloablative conditioning., Omisirge demonstrated a median time to engraftment of 12 days, compared to 22 days for standard cord blood (p<0.001).3 Incidence of moderate to serious bacterial or fungal infections through 100 days following transplantation occurred in 39% of patients in the Omisirge arm versus 60% of patients in the standard cord blood arm.3

This is a significant milestone for Gamida Cell and for the field of cord blood cell therapy. This approval provides a much-needed new treatment option for patients with blood cancers and could potentially improve their outcomes.  

Cord blood expansion can greatly impact the therapeutic availability and usage of cord blood and may help the many patients in need of a stem cell transplant.

Excellthera Phase I/II Study

In other exciting cord blood expansion news, ExCellthera has recently published results of its Phase I/II study investigating its UM-171 product. Results from a phase I-II trial using UM171-expanded CB transplants showed prompt neutrophil engraftment at 18 days, low incidence of infectious complications, rapid resolution of febrile neutropenia, and shorter hospitalization after transplantation when compared to conventional cord blood transplants.4 Moreover, they achieved this expansion using  lower cell dose cord blood units than previous studies. The minimal TNC and CD34+ cell numbers that successfully expanded were 1.1 × 109 and 3.9 × 106. The median CD34+ cell expansion achieved has been 49.3-fold.4 UM171 CB expansion may improve usability of existing CB unit inventories and donor availability for patients of different races and ethnicities.4

 

Sources:
1. Sandhya R. Panch, James Szymanski, Bipin N. Savani, David F. Stroncek, Sources of Hematopoietic Stem and Progenitor Cells and Methods to Optimize Yields for Clinical Cell Therapy, Biology of Blood and Marrow Transplantation, Volume 23, Issue 8, 2017, Pages 1241-1249.
2. E. Shpall, K. Rezvani, Cord blood expansion has arrived, Comments on on Horwitz et al, page 1429: Results of a phase 3 randomized study. Blood, 138(16), 1381-1382. 

3. Horwitz, M. E., Stiff, P. J., Cutler, C., Brunstein, C., Hanna, R., Maziarz, R. T., Rezvani, A. R., Karris, N. A., McGuirk, J., Valcarcel, D., Schiller, G. J., Lindemans, C. A., Hwang, W. Y., Koh, L. P., Keating, A., Khaled, Y., Hamerschlak, N., Frankfurt, O., Peled, T., … Sanz, G. (2021). Omidubicel vs standard myeloablative umbilical cord blood transplantation: Results of a phase 3 randomized study. Blood, 138(16), 1429–1440. https://doi.org/10.1182/blood.2021011719
4. Maude Dumont-LagacĂ©, Albert Feghaly, Marie-Christine Meunier, Marcie Finney, Wouter Van’t Hof, Emeline Masson Frenet, Guy Sauvageau, Sandra Cohen, UM171 Expansion of Cord Blood Improves Donor Availability and HLA Matching For All Patients, Including Minorities, Transplantation and Cellular Therapy, Volume 28, Issue 7, 2022, Pages 410.e1-410.e5.

 

Last Updated on: 09/14/2023 by Oleg Mikulinsky