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Foundation Funded Research Shows Promise for Lovastatin in Healing NF1 Bone Fractures

By March 5, 2010December 18th, 2023Awareness, NF1, Science & Research

Statin drugs, widely prescribed for cholesterol management, are well known in the neurofibromatosis community for the ongoing Phase II clinical trials of the drug Lovastatin for the treatment of NF1-related learning disabilities. However Lovastatin may also have promise for the treatment of NF1-related bone fractures.  Pseudarthrosis or ‘false joint’ can occur in children with NF1 when a long bone such as in the leg fractures but fails to heal because new bone is unable to be generated.  Pseudarthrosis is often untreatable and may require amputation.  A new study* (links to abstract) from the group of Florent Elefteriou (Vanderbilt University) uses a mouse model in which production of the NF1 protein neurofibromin is eliminated in osteoblasts (the cells that usually make new bone). The result is a mouse unable to heal fractures as fast as normal mice; and instead of the fracture healing, a weak callus remains at the site of injury, presenting (as in humans) a risk of re-breakage.  However, when these mice are treated with Lovastatin – which here, is delivered to the site of bone injury in tiny nanoparticles, to facilitate the drug’s rapid release – healing was markedly improved. Given that Lovastatin can safely be given to children, as evidenced by the NF1 learning disabilities trials, these exciting findings could quickly advance to Lovastatin trials to treat NF1-related bone dysplasia. This work was initiated with support of a Children’s Tumor Foundation Drug Discovery Initiative Award to Dr. Florent Elefteriou.