The US Food and Drug Administration (FDA) has approved the first product to use umbilical cord blood for stem cell transplantation. In a news release on Thursday, the agency said it has approved Hemacord, developed by the New York Blood Center, as “the first licensed hematopoietic progenitor cells-cord (HPC-C) cell therapy”.

Hemacord is “indicated for use in hematopoietic stem cell transplantation procedures in patients with disorders affecting the hematopoietic (blood forming) system”, the FDA said.

It covers certain blood cancers and inherited metabolic and immune system disorders, and includes bone marrow failure and beta-thalassemia.The product is also indicated for use with children.

Dr Karen Midthun, director of the FDA’s Center for Biologics Evaluation and Research, said:

“The use of cord blood hematopoietic progenitor cell therapy offers potentially life-saving treatment options for patients with these types of disorders.”

Cord blood is one of three sources of hematopoietic progenitor cells (HPCs) used in transplants, the other two being bone marrow and peripheral blood. Progenitor cells can turn into a variety of blood cells: they are like stem cells but a little further down the line in terms of specialization.

Once infused into patients, HPCs migrate to the bone marrow where they replicate and mature. When they move into the bloodstream, they can partially or fully restore the number and function of many blood cells, including immune function.

Dr Christopher D. Hillyer, President and CEO of New York Blood Center (NYBC), said:

“We are thrilled to be the first public cord blood bank with an FDA-licensed product for transplantation.”

The NYBC, a non-profit organization of community-based blood centers, has been providing blood and transfusion products to hospitals since 1964. In 1992 it established the National Cord Blood Program (NCBP) at the Howard P. Milstein National Cord Blood Center, where since 1996 it has provided cord blood units for transplantation under an Investigational New Drug (IND) exemption from the FDA.

In 2009 the FDA issued a guidance document to help manufacturers apply for licenses for their cord blood products. This was followed by a two-year phasing-in period for them to submit either a license application or an investigational new drug application. The period ended on 20 October, and these manufacturers now must submit such applications.

NYBC said to obtain their FDA license, all “collection and manufacturing conditions of the cord blood units met FDA standards for product potency, purity and efficacy, and patient safety”. The license application included “extensive documentation of the systems and controls used, and the validations performed to ensure compliance”, plus safety data from the transplantation of more than 4,000 cord blood grafts.

The FDA said that Hemacord will carry a boxed warning on the risks of Graft Versus Host Disease (GVHD), engraftment syndrome, graft failure, and infusion reactions, each of which can be fatal.

Patients who receive Hemacord should be monitored carefully, and a physician experienced in hematopoietic stem cell transplantation should oversee the risk benefit assessment, unit selection and administration of the product, noted the agency.

Written by Catharine Paddock PhD