A preliminary study published in Cell indicates that a novel cell therapy could offer significant relief for the millions of people worldwide living with osteoporosis. The trial involved ten older women with a history of severe, recurrent fractures who received a single infusion of their own bone-marrow-derived mesenchymal stromal cells (MSCs) that had been genetically modified to better penetrate bone tissue.
Before the treatment, the participants suffered fractures in their spines, hips, and arms roughly every one to two years, often from minor incidents like stumbling. Following the infusion, the rate of low-impact fractures dropped dramatically to approximately once per decade, with no reported side effects.
Osteoporosis is an age-related condition characterized by thin, porous bones that are highly susceptible to breaking. It currently affects an estimated 200 million women globally, particularly those post-menopause. Ajit Varki, a physician-scientist at the University of California, San Diego, who was not involved in the research, described the results as “quite remarkable,” noting the near-100% efficacy sustained over several years.
The therapeutic approach relies on work conducted by Robert Sackstein, a regenerative medicine specialist at the Miami Veterans Affairs Medical Center. In 2008, Sackstein and his team discovered that adding the sugar fucose to MSCs enabled the cells to navigate through the bloodstream and enter bone tissue, a feat they typically cannot accomplish. This modification appears to facilitate interactions with blood-vessel walls, slowing the cells down and allowing them to squeeze into the bone marrow.
While the method proved successful in mice, translating it to humans required years of refining manufacturing processes and generating preclinical data. In 2015, a clinical team led by José Moraleda, a bone-marrow transplant specialist at the University of Murcia in Spain, initiated the human trial. They treated women aged 51 to 72 with advanced osteoporosis using autologous MSCs enhanced with fucose.
Despite the encouraging outcomes, researchers caution that the findings come with significant limitations. The study was small and lacked a control group. Furthermore, most participants were continuing conventional osteoporosis medications throughout the trial, making it difficult to isolate the specific impact of the cell therapy. Additionally, the researchers did not directly track the therapeutic cells within the recipients’ bodies, leaving the question of whether sufficient cells reached the bones unanswered.
The study highlights a potential shift from managing osteoporosis symptoms to actively rebuilding skeletal structure, though larger, controlled trials are needed to confirm the therapy’s efficacy and mechanism.
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