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Stem Cells and Scleroderma

Results from a recent study performed by Duke University show that stem cell therapy may prove to be a highly effective treatment option for people with scleroderma.

According to the Scleroderma Foundation, approximately 300,000 people in the U.S. are living with the condition. Scleroderma is an autoimmune condition caused an overactive immune system. It affects the skin, blood vessels and organs, and causes pain, inflammation, debilitation, the thickening of connective tissue and organ failure.

There is no cure for scleroderma, and many conventional treatments, such as steroids and immunosuppressant drug therapy, are not effective long-term.

The Duke study found that patients with severe cases of scleroderma improved significantly through a combination of therapies that included chemotherapy, radiation and a stem cell transplant.

Before beginning the study, the Duke researchers developed a stem cell transplant conditioning regimen that combined stem cell therapy with high levels of chemotherapy and whole-body radiation to destroy the malfunctioning cells triggered by the overactive immune system. Thirty-six participants with scleroderma received a stem cell transplant at random after the chemotherapy and radiation portions of the study were completed.

The stem cells were taken from the patients' blood.

The goal of the study was to replace malfunctioning lymphocytes, a form of blood cell that plays a vital role in the body's ability to defend itself against illness, injury and infection.

"Stem cells have a powerful immunomodulatory effect, and can help reset the immune system," said Dr. Bill Johnson, M.D.

Johnson is a Dallas, Texas, physician who uses autologous adipose fat stem cell therapy to treat patients with scleroderma, lupus and other autoimmune diseases.

"Stem cells also have the additional benefits of having the infinite ability to regenerate themselves, which provides powerful healing," Johnson said.

In addition to their ability to treat malfunctioning immune systems, stem cells can also heal many different types of tissue.

"Stem cells are undifferentiated cells. Being undifferentiated means they can repair a wide range of tissues, including tissue damaged by scleroderma," Johnson said.

In addition to the 36 participants who received the stem cell transplant, 39 participants were given a year-long series of injections of the drug cyclophosphamide, a commonly used treatment for severe cases of scleroderma.

Over the next 10 years, researchers monitored patients to see if they improved more after receiving stem cell therapy or the cyclophosphamide. Fifty-four months into the study, participants were scored and compared regarding survival, quality of life, organ function and skin hardening. Findings showed that 67 percent of the participants saw a significant benefit from the stem cell transplant treatment, compared to 33 percent of those who received cyclophosphamide.

One benefit seen after the stem cell transplant treatment is that patients needed fewer immunosuppressant drugs. Only 9 percent of the participants who received the stem cell transplant went back to taking their anti-scleroderma medications, compared to 44 percent of those who received cyclophosphamide.

While the need to rely on drug therapy decreased, the survival rate of those receiving stem cell therapy went up. The survival rate at the six-year mark was 86 percent, compared to 51 percent of the patients who took cyclophosphamide.

"In many instances, stem cell therapy is beneficial for those individuals who do not respond to or cannot tolerate conventional medical treatments," Johnson said.

Previous studies of scleroderma and stem cell transplants have proved to be viable but did not include the intense radiation that the Duke University study included. As a result, the standard treatment of scleroderma is still immunosuppressant drug therapy.

"The ability to show the benefits of stem cell therapy such as increased longevity is significant to developing future treatments," Johnson said.


Sources:

Duke University Medical Center. "Stem cell transplant is better than drug therapy for scleroderma." ScienceDaily. ScienceDaily, 5 January 2018

Scleroderma Foundation. Patients and Newly Diagnosed. 2018.

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