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Cord Blood Offers Hope for Rare Diseases and Ethnic Minorities

Oct08-Cord-Blood-for-Rare-Diseases-in-Ethnic-Minorities

A team of researchers from the University of Pittsburgh Medical Centre recently published details of a new method for transplanting stem cells. The technique has already been tested on patients and shown to be a safe and effective universal treatment for a variety of genetic disorders.

It is thought that combining this technique with umbilical cord blood will make it particularly useful for the treatment of minorities, a group that has historically had trouble accessing compatible bone marrow stem cells.

Details of the safety and efficacy of the treatment were published in the journal Blood Advances. The researchers used haematopoietic stem cells (HSCs) derived from umbilical cord blood to treat 44 children with a wide variety of non-cancerous diseases.

HSCs have the ability to differentiate into red and white blood cells. This gives them the capacity to treat a variety of blood disorders, metabolic disorders, immune disorders and cancer.

Lead author, Dr. Paul Szabolcs, said of the study: “We wanted to offer a uniform concept to a wide array of diseases. We successfully reduced the intensity of chemotherapy and nevertheless, we were still able to engraft all our patients.”

Less Intense Chemotherapy

In most cases, a patient will receive intense chemotherapy treatments before they have a stem cell transplant. The chemotherapy treatment will kill off the patient’s immune system, removing any mutated or cancerous cells which are responsible for their illness. A stem cell transplant is then used to restore the patient’s ability to produce healthy blood cells.

Unfortunately, chemotherapy is very hard on the body and can kill a large number of healthy cells. For patients who don’t have cancer and are only being treated to improve their quality of life, it can be an excessively dangerous process.

The University of Pittsburgh Medical Centre team decided to use a different combination of chemotherapy drugs which were less toxic and had less side effects. Instead of trying to kill all of a patient’s immune cells, they simply killed enough for the stem cell transplant to be effective.

As Dr. Szabolcs explained: “We successfully reduced the intensity of chemotherapy and we were still able to engraft all our patients using maximum intensity, you might be inadvertently killing patients and causing irreversible organ disease. [We used] use reduced intensity and have excellent survival.”

The researchers discovered that their new approach was safer yet still very effective, with an improved recovery rate compared to conventional chemotherapy treatments.

The fact that the treatment uses umbilical cord stem cells makes it more useful for treating patients in minority groups, as a perfect HLA (immune profile) match with the donor is not required.

Source: Cord blood offers hope for rare diseases and ethnic minorities

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