09 January 2018
TUESDAY, Jan. 9, 2018 (HealthDay News) — In a potential advance for medical research, scientists say they’ve created the first functioning human muscle from skin cells. The breakthrough could lead to better genetic or cell-based therapies, as well as furthering investigations into the causes and treatment of muscular disorders, the Duke University team said. “The prospect of studying rare diseases is especially exciting for us,” Nenad Bursac, professor of biomedical engineering, said in a university news release. “When a child’s muscles are already withering away from something like Duchenne muscular dystrophy, it would not be ethical to take muscle samples from them and do further damage,” he explained. “But with this technique, we can just take a small sample of non-muscle tissue — like skin or blood — revert the obtained cells to a pluripotent state, and eventually grow an endless amount of functioning muscle fibers to test,” Bursac said. According to the researchers, it might also be possible to fix genetic defects in pluripotent stem cells from a patient and then grow small patches of healthy muscle that could be used with other genetic treatments to heal or replace specific areas of diseased muscle. Of course, much more research is needed before any such therapies could be used in humans. In the new study, skin cells were reprogrammed in the lab to revert to what are called pluripotent stem cells — cells that can grow into any type of cell. The cells were then cultured while being exposed to a molecule called Pax7, which signaled the cells to start turning into muscle. The cells then grew into functioning skeletal muscle. According to Bursac’s team, the lab-grown cells were not as strong as those found in normal muscle tissue. However, after up to four weeks in the special lab culture, the newly formed muscle cells contracted and reacted to external stimuli much like regular muscle tissue. The lab-grown muscle fibers were also implanted into mice and appeared to integrate into the rodents’ natural muscle tissue, the investigators said. The study was published online Jan. 9 in the journal Nature Communications. More information The U.S. National Institute of Neurological Disorders and Stroke has more on muscle disorders.
03 January 2018
WEDNESDAY, Jan. 3, 2018 (HealthDay News) — 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 study’s 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. The study was scheduled for publication in the Jan. 4 issue of the New England Journal of Medicine. More information The American College of Rheumatology has more on scleroderma.