In Italy, a team of researchers developed a biocompatible scaffold seeded with molecule-releasing stem cells that, when implanted in mice with a rare but deadly bone disease called autosomal recessive osteopetrosis (ARO), showed potential to help these animals, and someday yield a treatment for humans afflicted by the same devastating condition.
One of every 250,000 babies born has ARO. ARO is caused by mutations in several genes involved in the formation, development and function of specialized cells called osteoclasts.
“Defining a cure for RANKL-ARO is currently an unmet medical need whose solution is unfairly complicated, owing to the extreme rarity of these patients,” Dr. Sobacchi said. In collaboration with Prof. Anna Tampieri, at ISTEC-CNR, the team turned to biomimetic scaffolds because, she explained, “They are extremely versatile in terms of chemical composition and physical properties, which can be defined to accomplish specific applications. One property that can be added is the production/release of bioactive soluble factors, either directly from the biomaterial or from cells embedded within the biomaterial. We reasoned that pursuing this strategy would be appropriate to set up a cell-based therapy for RANKL-deficient ARO.”