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FDA Approves First Stem-Cell Therapy

Kate Traynor

BETHESDA, MD 11 Nov 2011—FDA on November 10 granted marketing approval to the New York Blood Center's allogeneic cord-blood product, Hemacord, the first U.S.-licensed hematopoietic progenitor cell therapy.

Hemacord is indicated for hematopoietic progenitor cell transplantation procedures in patients with inherited, acquired, or myeloablative-treatment-related diseases that affect the hematopoietic system, through which blood and blood cells are generated in the body.

Clinical trials of Hemacord involved patients with conditions including hematologic malignancies, inherited metabolic disorders, primary immunodeficiencies, and bone marrow failure, according to the product's labeling (PDF).

Hemacord contains CD34+ hematopoietic progenitor cells, monocytes, lymphocytes, and granulocytes recovered from human cord blood that has been partially depleted of erythrocytes and plasma. The cord blood is obtained and processed by the New York Blood Center's cord blood transplantation program at the Milstein National Cord Blood Center.

During a successful transplantation procedure, the immature cells in Hemacord migrate to the recipient's bone marrow, mature, and are released into the bloodstream, where they have the potential to partially or fully restore the recipient's blood-cell levels and function.

Because Hemacord is derived from human blood, recipients are at risk for infection by adventitious agents and transmission of leukemia and rare genetic diseases originating from the donor cells, according to the labeling.

A boxed warning in Hemacord's labeling warns that patients treated with the product are at risk for transfusion reactions, graft-versus-host disease, engraftment syndrome, and graft failure, any of which can be deadly.

About 25% of patients treated with Hemacord in clinical studies had died from any cause within 100 days of the transfusion, according to the labeling.

Hemacord should be administered under the supervision of a health care professional who is experienced in hematopoietic progenitor cell transplantation. Patients should be observed and assessed for transfusion reactions and other complications during the transfusion and for six hours afterward.

Patients must undergo human leukocyte antigen (HLA) typing before being treated with Hemacort. Matching procedures should ensure that potential recipients share at least four of six HLA-A antigens, HLA-B antigens, and HLA-DRB1 alleles with the batch of the product that is to be used for administration.

The labeling recommends premedicating patients with an "any or all" of a combination of antipyretic, histamine-blocking, or corticosteroid drugs 30–60 minutes before administering Hemacord.

The recommended starting dosage of Hemacord is at least 2.5 x 107 nucleated cells, at the time of cryopreservation, per kilogram of patient weight. Multiple units of Hemacord may need to be transfused to achieve the appropriate dose.

Each 25-mL cryopreserved bag of Hemacord contains a minimum of 1.25 x 106 viable CD34+ cells suspended in 10% dimethylsulfoxide (DMSO) and 1% Dextran 40. The product is contraindicated in patients with a known allergy to DMSO, Dextran 40, or plasma proteins.

The specific nucleated cell count at the time of cryopreservation, the CD34+ cell count, the ABO blood group, and the HLA typing results appear on the container's label and the accompanying paperwork. The product is considered viable for 48 months from the date of cryopreservation if stored at –196 °C or below. On receipt of the product, the transplant center must keep the bag at or below –150 °C, either inside the manufacturer's "Dry-Shipper" container or in a liquid nitrogen-cooled storage device.

Detailed instructions for preparing Hemacord for administration and performing the transfusion appear in the product's labeling.

 

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