Stem cell transplant shows promise
Stem cell transplants could offer new hope for people with a severe form of scleroderma -- a debilitating and deadly condition that affects the immune system, a new study suggests. Scleroderma hardens the skin and connective tissues and, in its severe form, leads to fatal organ failure, most often the lungs, said the studys lead author, Dr. Keith Sullivan. He is a professor of medicine and cellular therapy at Duke University Medical Center.
In these severe cases, conventional drug therapies are not very effective long-term, so new approaches are a priority, Sullivan said in a hospital news release.
Drugs to suppress the immune system are the standard of care in the United States for scleroderma with internal organ involvement, according to the researchers. Their study tested the effectiveness of stem cell transplant along with high-dose chemotherapy and whole-body radiation to treat the disease. The study was funded by the U.S. National Institutes of Health.
For the study, 75 people with scleroderma were randomly assigned to receive one of two treatments. Roughly half of the group received a stem cell transplant designed to destroy their defective immune system and replace it with their own treated blood stem cells. The other half received 12 months of conventional immune-suppressing treatment.
After 10 years, survival was better among those who underwent chemotherapy, whole-body radiation and a stem cell transplant, the study found. People in this group also had less need for immune-suppressing drugs after their transplant.
These results show that individuals with poor-prognosis scleroderma can improve and live longer and that these advances appear durable, Sullivan said.
The researchers noted, though, that stem cell transplant was riskier. It was associated with more serious side effects, such as low blood counts, infections and death, the study found. Patients and their doctors should carefully weigh the pros and cons of intensive treatment with stem cell transplant, but this may hopefully set a new standard in this otherwise devastating autoimmune disease, Sullivan said.
These advances show the value of medical research and clinical trials in finding better therapies to advance health, he added.
A stem cell transplant aims to try and cure some types of blood cancer such as leukemia, lymphoma and myeloma. It is also called a peripheral blood stem cell transplant.
You have very high doses of chemotherapy, sometimes with whole body radiotherapy. This has a good chance of killing the cancer cells but also kills the stem cells in the bone marrow.
Stem cells are very early blood cells in the bone marrow that develop into red blood cells, white blood cells and platelets.
We need stem cells in order to survive. Doctors can collect stem cells from your blood or a donors. After high dose treatment, you have the stem cells into a vein through a drip to replace those that the cancer treatment has killed.
Stem cell transplant means that you can have higher doses of treatment. So there may be more chance of curing the cancer than with standard chemotherapy.
You might have your own stem cells given back to you after high dose treatment. This is called an autograft. You have growth factors before, and sometimes after, a stem cell transplant. Growth factors are natural substances that make the bone marrow produce more stem and blood cells. You have them as small injections under the skin.
You have daily injections of growth factor for between 5 and 10 days. Sometimes you may have low doses of a chemotherapy drug alongside the growth factor injections. The chemotherapy and growth factor injections help your bone marrow to make lots of stem cells. These stem cells then spill out of the bone marrow into the bloodstream.
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