The true scale of osteoporosis is difficult to fathom. The progressive bone-weakening disease is estimated to affect as many as 500 million people worldwide, triggering up to 37 million fragility fractures a year.
That's a bone breaking in someone's body every single second.
While there's no cure for the condition, scientists say they may have found a kind of "living medication" for the disease.
In a first-in-human phase 1 trial, researchers gave patients with advanced osteoporosis a modified version of their own mesenchymal stem/stromal cells (MSCs), and it showed significant bone-strengthening effects. While the trial's primary aim was to investigate the safety of this experimental treatment, the researchers say their findings demonstrate much more.
Mesenchymal stem/stromal cells are a form of stem cell, but whereas embryonic stem cells and induced pluripotent stem cells ( iPSCs) can generate almost any cell type, MSCs are only multipotent – not pluripotent – meaning they're more limited in the kinds of cells they can become.
However, MSCs, which can be found in bone marrow (as well as other parts of the body), are the precursors to osteoblasts, cells that create and repair bone.
In theory, this makes MSCs a promising candidate for bone-strengthening therapies to treat osteoporosis, but once MSCs are cultured outside the body and then returned intravenously, their lack of a surface molecule called Sialylated Lewis X (sLeX) hampers their ability to find the bone marrow.
"In preclinical models, this deficit is correctable by MSC glycocalyx editing to enforce sialylated Lewis X (sLeX) expression, thereby programming osteotropism," the researchers explain.
Summary:
Mesenchymal stem/stromal cells (MSCs) are osteoregenerative; however, their therapeutic efficacy for skeletal conditions is hampered by poor bone-homing (“osteotropism”). In preclinical models, this deficit is correctable by MSC glycocalyx editing to enforce sialylated Lewis X (sLeX) expression, thereby programming osteotropism. We conducted a first-in-human clinical trial involving a single intravenous infusion of glycocalyx-edited autologous bone marrow-derived MSCs in ten women with advanced-stage osteoporosis. The protocol-mandated evaluation spanned 2 years and included clinical assessments, radiographic studies, and measurements of bone turnover markers (BTMs), bone tissue area (BTA), and bone mineral density (BMD). Thereafter, fracture and safety monitoring continued for >3 additional years for each patient. No serious adverse events occurred.
https://cdn.jwplayer.com/previews/xyka42tL
That's a bone breaking in someone's body every single second.
While there's no cure for the condition, scientists say they may have found a kind of "living medication" for the disease.
In a first-in-human phase 1 trial, researchers gave patients with advanced osteoporosis a modified version of their own mesenchymal stem/stromal cells (MSCs), and it showed significant bone-strengthening effects. While the trial's primary aim was to investigate the safety of this experimental treatment, the researchers say their findings demonstrate much more.
Mesenchymal stem/stromal cells are a form of stem cell, but whereas embryonic stem cells and induced pluripotent stem cells ( iPSCs) can generate almost any cell type, MSCs are only multipotent – not pluripotent – meaning they're more limited in the kinds of cells they can become.
However, MSCs, which can be found in bone marrow (as well as other parts of the body), are the precursors to osteoblasts, cells that create and repair bone.
In theory, this makes MSCs a promising candidate for bone-strengthening therapies to treat osteoporosis, but once MSCs are cultured outside the body and then returned intravenously, their lack of a surface molecule called Sialylated Lewis X (sLeX) hampers their ability to find the bone marrow.
"In preclinical models, this deficit is correctable by MSC glycocalyx editing to enforce sialylated Lewis X (sLeX) expression, thereby programming osteotropism," the researchers explain.
Summary:
Mesenchymal stem/stromal cells (MSCs) are osteoregenerative; however, their therapeutic efficacy for skeletal conditions is hampered by poor bone-homing (“osteotropism”). In preclinical models, this deficit is correctable by MSC glycocalyx editing to enforce sialylated Lewis X (sLeX) expression, thereby programming osteotropism. We conducted a first-in-human clinical trial involving a single intravenous infusion of glycocalyx-edited autologous bone marrow-derived MSCs in ten women with advanced-stage osteoporosis. The protocol-mandated evaluation spanned 2 years and included clinical assessments, radiographic studies, and measurements of bone turnover markers (BTMs), bone tissue area (BTA), and bone mineral density (BMD). Thereafter, fracture and safety monitoring continued for >3 additional years for each patient. No serious adverse events occurred.
https://cdn.jwplayer.com/previews/xyka42tL
