Hemacord: Stem Cell Cure for Hemophilia Gains FDA Approval

Blood-forming disorders get new human cord cell therapy: Like a jackhammer on your back, the stomach, in the head and sometimes in the whole body that is relieved only by powerful, addictive painkillers. That’s how it feels to suffer from sickle-cell anemia.

The disease, caused by inheriting a gene that makes rigid, sickle-shaped cells, leads blood to clump and circulation to slow down, causing sudden episodes of extreme pain. It’s chronic and untreatable.

In turn, people who have hemophilia, a disorder in the blood-clotting system, suffer excessive bleeding. While small cuts don’t cause big problems, people with hemophilia risk deep internal bleeding.

They can also bleed painfully into joints and into muscles. Bleeding into muscles causes pain and swelling—and swelling can increase pressure on tissues and nerves, causing permanent damage and even deformity to muscles.

Hemophiliacs can also lose their sight from bleeding into the eye or from small bleeds in the brain, which can also cause psychiatric problems and even retardation. Most hemophiliac children die from bleeding in the brain—that can even occur spontaneously. Hemophilia is a lifelong disease.

Because people with beta-thalassemias produce inadequate amount of hemoglobin, the substance found inside all red blood cells that transports oxygen throughout the body, they suffer from severe anemia that doesn’t only make them weak and tired but may even require them to undergo frequent blood transfusions.

If untreated, severe anemia can result in bone thinning and heart failure. Lifelong transfusions lead to increased iron deposition, causing thyroid and liver failure—and even heart failure.

Sickle-cell anemia, hemophilia and beta-thalassemia are only some of the disabling disorders affecting the hematopoietic or blood-forming system—and people who suffer from these are apt to benefit for a new therapy that uses hematopoietic progenitor cells-cord (HPC-C) cells.

In November last year, the United States Food and Drug Authority approved for the first time the therapy that uses these self-recreating cells of human blood taken from cord blood.

After a baby is born and the umbilical cord is cut, some blood remains in the blood vessels of the placenta and the portion of the umbilical cord that remains attached to it. This blood, called “cord blood,” is no longer needed by the baby and in recent years, has been harvested—with the permission of birth mothers to produce stem cells and other cord-blood products like hematopoietic progenitor cells.

After being collected, cord blood is isolated, processed, frozen and stored in a cord blood bank for future use. The bank is regulated by the FDA.

The new product, called HEMACORD, is made from self-recreating “progenitor” cells that are similar to stem cells and will be used to treat patients with blood-forming system disorders, blood cancers, some inherited metabolic and immune system disorders. It is manufactured by the New York Blood Center, Inc., based in New York, NY.

“The use of cord blood hematopoietic progenitor cell therapy offers potentially life-saving treatment options for patients with these types of disorders,” says Dr. Karen Midthun, director of the U.S. Food and Drug Authority’s Center for Biologics Evaluation and Research in a statement released on Nov. 10, 2011 and is available on the agency’s website.

Hematopoietic progenitor cells (HPCs) are blood-forming cells used in transplants that can come from three sources—cord blood, bone marrow and peripheral blood. HEMACORD contains HPCs from human cord blood.

The FDA explains: Once these cells are infused into patients, the cells migrate into the bone marrow where they divide and mature. When the mature cells move into the bloodstream they can partially or fully restore the number and function of many blood cells, including immune function.

“We have been using cord blood for years,” a Reuters report quotes Dr. Machi Scaradavou, medical director of the NYBC’s National Cord Blood Program, as saying. “Recently, FDA decided that it needs to be licensed and this is the first cord blood product and stem cell product to be licensed.”

In 2009, the FDA guided manufacturers of such therapies to submit by October 20 applications either for a license or for an approval as an investigational new drug. National Cord Blood Program is the first to get FDA’s nod, Scaradavou said.

HEMACORD comes with a boxed warning, the FDA’s most serious warning associated that it reserves for serious side effects. The warning says HEMACORD is associated with possibly fatal risks of Graft Versus Host Disease, engraftment syndrome, graft failure and infusion reactions. These diseases may be fatal, the FDA said.

The agency also warned that patients who receive HEMACORD should be monitored carefully. A risk- benefit assessment, unit selection and administration of HEMACORD should be done under the direction of a physician experienced in hematopoietic stem cell transplantation, it said.


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