On 17 April 2023, the FDA gave approval to the Gamida Cell product “Omisirge” (known also as omidubicel or NiCord)¹. This is the first FDA market approval of an “expanded” cord blood product, which means that the number of stem cells in cord blood is no longer it’s limitation.

Cord blood stem cells are used in medicine for treatment of over 80 different  diseases such as leukemias, lymphomas and sickle cell anemia for over 30 years. It has also been proven that in the field of regenerative medicine, the application of cord blood stem cells brings clinical benefit to patients. More than 60 000 patients have been treated with cord blood.

Stem cells are the hope of 21st century medicine. Over 3,000 studies in various fields are currently underway around the world to discover the potential of stem cells. Cord blood is the source of youngest, most powerful and easy to obtain stem cells. It can also be easily collected and banked at birth to be used later in life. Due to the fact that the number of stem cells isolated from umbilical cord blood is limited, critics pointed out that average cord blood unit is enough only  for patients weighing up to 40-50 kg. As result of that many people decided not to bank the cord blood – neither for family nor public use. The FDA's groundbreaking decision for approving expanded cord blood indicates that this objection is no longer valid.

The multiplication of hematopoietic cells was the Holy Grail of hematology - an idea that, despite many attempts over decades, no one managed to implement. The FDA decision shows that cord blood has reached the point where the number of cells no longer matters. What's more - the multiplied cells from umbilical cord blood can regenerate the hematopoietic system faster after transplantation. This may mean a wider introduction of this type of methods into clinical practice - says Tomasz Baran, MD, vicepresident of FamiCord Group.

What exactly is “Omisirge”²? it issubstantially modified allogeneic (donor) cord blood-based cell therapy to quicken the recovery of neutrophils (a subset of white blood cells) in the body and reduce the risk of infection. The product is intended for use in adults and pediatric patients 12 years and older with blood cancers planned for umbilical cord blood transplantation following a myeloablative conditioning regimen (treatment such as radiation and/or chemotherapy). Omisirge is intended to help patients who can’t find a suitable donor for a transplant. Currently, when transplant oncologists cannot find a fully matched bone marrow donor, they often choose a haploidentical, or half-matched, bone marrow donor over a cord blood unit. Now expanded cord blood is also an option.

In the context of FDA approvals for advanced cell therapy, expanded cord blood now joins the ranks of a half dozen CAR-T products and a handful of gene therapies for inherited disorders. I hope that in the near future expanded cord blood would be approved also by EMA in Europe comments Francisco Santos,PhD, Director of Production and Development, FamiCord Group.

Blood cancers are a form of cancer caused by uncontrolled growth of cells in the blood, disrupting the ability of blood cells to perform their normal functions. This abnormal cell growth often begins in the bone marrow, which is made up of stem cells that form into different types of blood cells with specific functions in the body. According to the WHO, there were around 320,000 new cases of people with blood cancer in Europe each year³. Blood cancers can be fatal, with varying survival rates based on multiple factors including the specific type of blood cancer diagnosed. This type of cancer can also cause serious and damaging effects to the body and lead to symptoms such as fatigue, bone and joint pain, night sweats, infections, weakness, weight loss and fever. 

Stem cell transplantation is a common treatment for blood cancers. It involves putting healthy stem cells into the body to help restore the normal production and function of blood cells. One source of healthy stem cells is umbilical cord blood. Generally, before receiving this kind of transplant, the patient will undergo a course of treatments to remove their own stem cells and prepare the body for the new stem cells. This process may include undergoing therapies such as radiation or chemotherapy, both of which may weaken an individual’s immune system. As a result, a frequent and serious risk of this treatment is the occurrence of severe and sometimes deadly infections.

FamiCord Group is the largest cord blood bank in Europe and 3rd largest cord blood bank in the world. We are offering services in 31 countries directly and indirectly. In our 15 laboratories, are stored more than 860 000 samples of cord blood and tissue. The quality of our service is proven by number of patients who received stem cells applications from our bank (incl. 149 cord blood transplantations). Our clients may be sure that the material entrusted to us are stored at a stable bank of international importance.

¹FDA Approves Cell Therapy for Patients with Blood Cancers to Reduce Risk of Infection Following Stem Cell Transplantation, www.fda.gov.com [April: 2023]

²FRANCES VERTER, Update on Omidubicel from Gamida Cell: Phase 3 Trial Outcomes. Parent's Guide to Cord Blood Foundation News published 2021-03  [April: 2023]

³SUSAN LANGTHORP, Blood cancer diagnosis: when mission impossible became possible, Horizon: Europe Research&Development Magazine, www.ec.europa.eu [April: 2023]