Sugar-Tagged Stem Cells Sent to Bone Marrow Show Early Promise Against Osteoporosis
In a small first-in-human trial, 10 women with advanced osteoporosis received their own stem cells modified with a sugar-based homing tag. The treatment appeared safe, and fractures dropped, though experts warn efficacy is unproven.
A small first-in-human trial found that stem cells given a sugar-based homing tag to guide them to bone marrow were safe in 10 women with advanced osteoporosis and were followed by fewer fractures, Chemical & Engineering News reported. The phase 1 results were published in the journal Cell.
A one-hour reaction adds a temporary homing signal
Mesenchymal stem cells can build bone, but when injected into the bloodstream they lack a way to find the skeleton. The team, led by bone marrow transplant physician Robert Sackstein of the Miami Veterans Affairs Medical Center, took each woman’s own stem cells and used an enzyme to add the sugar fucose to a surface protein called CD44. That created a temporary tag that docks onto receptors in bone marrow blood vessels.
Each participant received one infusion of her modified cells. Over roughly six years of follow-up there were no treatment-related serious adverse events. In the two years after infusion the women had one fragility fracture, compared with 16 in the two years before. Bone biopsies at four months showed more trabecular bone tissue.
“The efficacy signal was completely unanticipated.” — Robert Sackstein, bone marrow transplant physician, Miami Veterans Affairs Medical Center
Outside experts say it is too early to claim it works
Serge Ferrari of Geneva University Hospital called the study “clear progress” but noted that most participants also took bone drugs, which muddies the picture. Endocrinologist Marc Wein of Mass General Brigham said efficacy claims are “speculative at best,” pointing to the small size of the study and largely negative DXA bone scans.
Sackstein’s group plans larger, multicenter trials to confirm the effect. The paper is available in Cell.
The approach is part of wider stem cell research, such as work on growing salivary glands from stem cells to treat dry mouth.
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