Cord Blood Treatments Today – Thalassemia Major

Treating Thalassemia with Cord BloodWhen we think of cord blood, we often think of its potential to be used in medical treatments that are still being developed. And that potential is enormous. But it’s important to remember that cord blood already plays a critical role in treating—and in some cases curing—dozens of serious diseases and disorders.

One of these diseases is beta thalassemia major, also known as Cooley’s anemia. Beta thalassemia major is an inherited blood disorder that occurs when the genes governing the production of hemoglobin—the protein in red blood cells that binds to oxygen and carries it throughout the body—are flawed.

More specifically, hemoglobin contains two proteins, an alpha protein and a beta protein. In people afflicted with beta thalassemia major, the hemoglobin doesn’t contain enough of the beta protein, which means the red blood cells can’t carry sufficient oxygen from the lungs to the body’s cells and tissues.

Children with this condition are normal at birth but often develop severe anemia by age two. Beta thalassemia major can also slow a child’s growth, make bones brittle, and cause the spleen to become enlarged. 1

A Cord Blood Cure
The most common treatment is for a patient to receive regular blood transfusions. This keeps the body stocked with healthy red blood cells. But it can also cause unhealthy accumulations of iron, which can damage the heart and other organs. To prevent this, patients may also undergo chelation therapy, which uses drugs to remove excess iron. 2

The only known cure for beta thalassemia major is a stem cell transplant. It’s an arduous process that starts with using drugs and radiation to destroy a patient’s bone marrow. Then, stem cells from a healthy matched donor are infused into the patient’s bloodstream. The goal is for these cells to start manufacturing healthy red blood cells.

While the donated stem cells don’t have to come from cord blood, cord blood stem cells have been shown to be highly successful when used in transplants. A study published in the journal Blood looked at the outcomes of 44 patients given cord blood stem cell transplants for either beta thalassemia major or sickle cell disease. Of those patients, 36 were disease-free as a result of the transplant.3

This helps explain why, of the 200 units of cord blood ViaCord has released for stem cell transplants, 27 have been used to treat beta thalassemia major.

Help for Siblings
Patients who have beta thalassemia major can’t use their own cord blood for a transplant, since the stem cells it contains have the same genetic defects as their current stem cells. So patients need to find stem cells from a donor who is a genetic match. To do this, they can search through public banks, or they can receive stem cells from a family member. Patients who receive cord blood stem cells from family members have significantly lower risk of graft-vs.-host disease, and transplants using cord blood from family members are nearly twice as successful as transplants using cord blood from non-relatives. 

With this in mind, ViaCord believes that every family deserves to benefit from the potential opportunities presented by cord blood stem cells, regardless of their financial situation. That’s why we offer help to families with children who have an established diagnosis of a disease that is currently treatable with sibling cord blood. If you’re an expectant parent with a child who has beta thalassemia major or another disease that can be treated with a sibling’s cord blood stem cells and meet the other enrollment requirements of the program, ViaCord may collect, process, and store your baby’s cord blood for five years free of charge. It’s part of our Sibling Connection program—and it reflects our commitment to making cord blood stem cells available to all families.

We still have much to learn about how cord blood stem cells may be used in future medical applications; but their demonstrated value in medical treatments today is clear. That’s why when you bank cord blood you’re not only banking on the possibilities of tomorrow, you’re also banking on the possibilities of today.

 

 

REFERENCES
1. http://www.nhlbi.nih.gov/health/dci/Diseases/Thalassemia/Thalassemia_WhatIs.html
2. http://www.mayoclinic.com/health/thalassemia/DS00905
3  Franco Locatelli, F. et al. (2003). Related umbilical cord blood transplantation in patients
with thalassemia and sickle disease. Blood 101(6) 2137-2143 doi: 10.1182/blood-2002-07-2090
http://bloodjournal.hematologylibrary.org/content/101/6/2137.long
4. Gluckman, et al., New England Journal of Medicine 1997, pp. 373-381.

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