Potential Alternative to Bone Marrow Transplants Awarded FDA Breakthrough Therapy Status
Gamida Cell, a leader in cellular and immune therapies for the treatment of cancer and orphan genetic diseases, has been granted breakthrough therapy designation by the U.S. Food and Drug Administration (FDA) for the Company’s lead product candidate, NiCord®, in development as a novel graft modality for bone marrow transplantation in patients with high risk haematological malignancies (blood cancers) such as leukaemia and lymphoma.
NiCord® is cryopreserved (frozen) umbilical cord blood stem and progenitor cells, that were expanded (population increased) ex-vivo (outside of the body). NiCord® is derived from a single umbilical cord blood unit which has been expanded in culture and enriched with stem and progenitor cells using Gamida Cell’s proprietary NAM technology, and is in development as an investigational therapeutic treatment for blood cancers such as leukemia and lymphoma and for non-malignant haematological diseases.
NiCord® was originally transplanted as a “fresh” product that had to be infused into the patient within a limited number of hours from the moment the product was released from the manufacturing site. This timetable restricted the window for transplantation for the patient and limited the location of manufacturing sites to the vicinity of participating medical centers;it also increased the cost of shipping and logistical support required from manufacturing through delivery.
The first person was successfully transplanted with cryopreserved (frozen) NiCord in the company’s ongoing Phase I/II clinical study for blood cancer patients in January 2015.
The designation “breakthrough therapy” is granted to drugs that are intended to treat a serious or life-threatening condition, and where preliminary clinical evidence suggests substantial improvement over existing therapies. It is meant to expedite the development and review of new therapies by giving the company extensive FDA guidance for more efficient and accelerated drug development programs.
“We are very pleased the FDA has recognized the potential of NiCord to address the unmet clinical need in bone marrow transplantation,” Dr. Yael Margolin, president and CEO of Gamida Cell, said in a press release.
“The breakthrough therapy designation creates the foundation for a joint and concerted effort between the FDA and Gamida Cell to bring this important therapy faster to patients. We look forward to continuing our close cooperation with the FDA and other regulatory agencies to a positive conclusion as we prepare for commercialization,” Margolin said.
Each year, more than 60,000 patients with high-risk blood cancers should receive bone marrow transplants, which are currently the only potential cure for these patients.
But to qualify for the treatment, patients require a donor with fully matched tissue typing, which is often difficult to find. As a result, only about half of patients who are supposed to get bone marrow transplants actually receive one.
With NiCord, Gamida Cell is trying to make a difference for patients who can’t find matching donors. NiCord is an alternative to bone marrow transplants that instead uses cells collected from the blood of the umbilical cord. These cells are then cultured using a proprietary NAM platform technology, which enriches the cultured cells in highly functional stem and progenitor cells, as well as dendritic cells.
Gamida Cell believes that NiCord can provide a transplant option that is available to all patients in need, based on results from the pilot and Phase 1/2 studies of NiCord. Compared to unmanipulated umbilical cord blood, the therapy has improved time to neutrophil engraftment (incorporating the transplanted cells into the recipient tissue) from 21 days to 10 days; the number of patients achieving neutrophil engraftment (75% versus 18%); faster platelet engraftment (32 days vs. 46 days); and lower transplant-related mortality (19% vs. 39%).
Additionally, NiCord induced fewer infections and reduced the length of time spent in the hospital.
Gamida Cell is now waiting for positive feedback from the FDA and the European Medicines Agency (EMA) regarding the outline of the Phase 3 trial, after which it will soon begin assessing NiCord in patients with blood cancers.
Gamida Cell presented additional positive data of Nicord® in blood cancer patients at EBMT 2016 in an oral session titled Transplantation of Ex vivo Expanded Umbilical Cord Blood (NiCord®) Results in Decreased Infection Burden and Hospital Length of Stay in the First 100 Days by Sarah Anand, MD and Mitchell Horwitz, MD, both from the Duke University School of Medicine and the Duke Cancer Institute, at the 42nd Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT) in Valencia, Spain in April.
Dr. Horwitz said, “We saw a significant reduction in serious bacterial infections during the first 100 days in the NiCord group. This is encouraging because this type of infection is a major cause of early death following UCB transplantation. We also saw a significant reduction in hospitalization time in the NiCord group, indicating a faster recovery of these patients in comparison to those transplanted with standard umbilical cord blood. These results demonstrate that the rapid hematopoietic recovery from NiCord transplantation results in clinical benefit, in comparison to similar site controls.”
Posted on 16 November, 2016, in Blog, Cancer Treatments, Medical Treatments, Research and tagged cancer, cancer treatment, childhood cancer, Children with Cancer, Fighting Cancer, Gamida Cell, NiCord, paediatric cancer. Bookmark the permalink. Leave a comment.