NiCord® was granted Breakthrough Therapy Designation by the FDA, as well orphan drug designation by the European Medicines Agency (EMA).
NiCord by Gamida Cell
Gamida Cell’s lead product, NiCord®, offers patients in need of hematopoietic stem cell transplantation (HSCT) an alternative to the existing approach of bone marrow transplant by using ex vivo expansion of cord blood. In clinical trials so far, NiCord® has demonstrated rapid engraftment and clinical outcomes that appear comparable to transplantation of peripheral blood.
Introducing a Paradigm Shift to HSCT with NiCord
Cord blood has many advantages over the use of peripheral blood for HSCT, including that it does not require a full tissue matching, it is widely available through a global network of public cord blood banks, and it exhibits low rates of graft versus host disease (GvHD). However, the small quantity of blood obtained from an umbilical cord (usually about 50-60mL) and the small number of cells results in delayed engraftment of neutrophils and platelets.
Therefore NiCord® has the potential to introduce a paradigm shift to HSCT by providing a transplantation option that could be available to practically all patients in need.
Gamida Cell Presents NiCord’s Phase 1/2 Results at ASCO
At ASCO 2016, Gamida Cell announced the presentation of results from its international, multi-center, Phase 1/2 study of NiCord at the 2016 American Society of Clinical Oncology (ASCO) Annual Meeting, results that demonstrate “statistically significant improvements across key measures, including time to engraftment of neutrophils and platelets.”
The key finding from the company’s ASCO presentation found patients treated with NiCord showed:
- Successful CD34+ expansion – A 101-fold increase in the number of CD34+ (stem and progenitor) cells in NiCord compared to unmanipulated cord blood.
- Improved engraftment – Faster neutrophil engraftment for NiCord recipients (p<0.0001) at a median of 10 days post-transplantation compared to 21 days in registry controls.
- Reduced morbidity
- Less transplant related mortality in comparison to controls
The results of this Phase 1/2 study will support commencement of Phase 3 Study. Click here to read the press announcement from Gamida Cell.
First Patient Transplanted in Phase 3 Study of NiCord for Blood Cancers
In February 2017, Gamida Cell announced it had treated it first patient in the Company’s Phase 3 study of NiCord®. As mentioned, the company is developing NiCord as a treatment option for patients with blood cancer who do not have a fully matched donor available to allow bone marrow transplantation.
This Phase 3 clinical trial is an “international, randomized, controlled, open-label, multicenter, registration study that is evaluating the safety and efficacy of transplanting NiCord® instead of an un-manipulated cord blood in patients with hematological malignancies (blood cancers) following a preparative conditioning regimen.”
The trial will include 120 patients across a wide range of ages (16 to 60 years old).
NiCord Update at BMT Tandem 2018
Gamida Cell also presented intriguing clinical data at BMT Tandem 2018. BMT Tandem is the combined annual meetings of the Center for International Blood & Marrow Transplant Research (CIBMTR) and the American Society for Blood and Marrow Transplantation (ASBMT).
In this presentation, Gamida Cell presented that:
- NiCord demonstrated rapid and durable engraftment in patients with high-risk hematologic malignancies
- 13 patients with sickle cell disease transplanted with NiCord and engrafted at median seven days
#BMTTandem18 Horwitz: immune reconstitution and summary of outcomes with Nicord pic.twitter.com/gQpcgJQiQo
— Aaron Logan, MD, PhD (@hemedoc) February 23, 2018
NiCord Clinical Trials
To view the clinical trials listing for NiCord, visit:
- Transplantation of NiCord®, Umbilical Cord Blood-derived Ex Vivo Expanded Cells, in Patients With HM
- Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies
- Phase I Trial of NAM NK Cells and IL-2 for Adult Pts With MM and NHL
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Up Next: Gamida Cell, President Dr. Yael Margolin Discusses the Unmet Need in HSCT