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La Protein as a Novel Regulator of Osteoclastogenesis
NIH
Protein
Musculoskeletal disease
Hit To Lead or Lead Optimization
Abstract
Millions of patients in the United States are afflicted by a host of bone diseases caused by osteoclast (specialized calls arising from the macrophage/monocyte lineage) dysfunction. Diseases include Paget’s disease, osteoporosis, fibrous dysplasia and osteolytic bone metastasis. The current standard of care for these diseases uses broad-spectrum therapies that either coat the skeletal system or inhibit osteoclast development in an effort to modulate osteoclastogenesis. New therapies are needed that specifically target osteoclast fusion – allowing patients to forgo the off-target side effects caused by existing, broad-spectrum therapies. Researchers at the National Institute of Child Health and Human Development (NICHD) discovered that the Lupus autoantigen (La) protein is a master regulator of osteoclast fusion and osteoclastogenesis., The resorptive capacity of osteoclasts and the ability of osteoclasts to remodel bone can be modulated by :(1) administering an effective amount of a La protein or (2) an agent that modulates La protein expression or activity. This specific approach to regulating osteoclast fusion and osteoclastogenesis should bypass the off-target side effects associated with current therapies. It represents a major opportunity to improve the lives of millions who suffer from numerous bone diseases. NICHD seeks co-development partners and/or licensees for the further development of methods to target the La protein for the regulation of osteoclast fusion and osteoclastogenesis.
Commercial Advantages
Technology addresses most major forms of – and therefore the largest addressable markets for – bone disease Enables precise modulation of osteoclast fusion and function Potential to prevent off-target side-effects associated with current therapies.
Commercial Applications
Therapeutics targeting La protein to treat all the most common types of bone disease – including but not limited to: o Osteoporosis o Paget’s disease o Fibrous dysplasia o Osteolytic bone metastasis Therapeutics targeting La protein for relieving symptoms associated with: o Rheumatoid arthritis o Metastatic bone disease.
Leikina, Evgenya Chernomordik
PCT-US2022-018639 (2022.03.03)
Patent Family: PCT, US
"Cell surface-bound La protein regulates the cell fusion stage of osteoclastogenesis". Whitlock, J. M. et al. Nature Communications. 2023. 14(1). https://doi.org/10.1038/s41467-023-36168-x
