Modified bone-marrow cells nearly stop fractures in osteoporosis trial
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A single infusion of modified bone-marrow cells nearly eliminated low-impact fractures in ten older women with osteoporosis, according to a small clinical trial published in Cell. Before treatment, participants experienced fractures every one to two years on average; after treatment, the rate dropped to about once per decade. The results raise the possibility of a one-time cell therapy for the condition, though the study lacked a control group and most participants were taking conventional osteoporosis drugs.
Key Facts
- The trial involved ten older women who had experienced dozens of fractures before treatment.
- After a single infusion of modified bone-marrow cells, low-impact fracture rate dropped from every one to two years to about once per decade.
- The study was published in Cell and reported almost 100% efficacy sustained for several years with no side effects, according to an outside physician-scientist.
- The study had no control group, and most participants were taking conventional osteoporosis drugs before and during the trial.
- The researchers did not directly track the therapeutic cells in recipients' bodies, leaving unanswered whether enough cells reached the bones.
Trial Results
The ten participants had broken bones dozens of times, often after little more than a stumble, before receiving the cell therapy. After a single infusion of their own bone-marrow cells treated in the laboratory, fractures caused by low-impact trauma dropped to about once per decade. Ajit Varki, a physician-scientist at the University of California, San Diego, described the results as showing almost 100% efficacy sustained for several years with no side effects. The study was small, had no control group, and most participants were taking conventional osteoporosis drugs before and during the trial.
Cell Modification Mechanism
Robert Sackstein, a regenerative-medicine specialist at the Miami Veterans Affairs Medical Center, co-authored the study and has spent decades studying how to steer regenerative cells for therapeutic effect. Mesenchymal stromal cells (MSCs) injected into the bloodstream cannot usually reach bone tissue. In 2008, Sackstein and colleagues found that adding a sugar called fucose to MSCs enabled them to seek out and enter bones, leading to skeletal tissue generation in mice. The added sugars disappear within about two days and are thought to slow the cells by fostering interactions with blood-vessel walls, allowing them to squeeze into bone marrow. Few trials had tested MSCs for osteoporosis, and no trials in any disease had deliberately modified the cells to improve their trafficking ability, according to the authors.
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