Nephrogenic systemic fibrosis (NSF) is a systemic fibrosing disorder seen exclusively in patients with renal insufficiency.  This severely disabling and painful fibrotic condition is associated with high morbidity and mortality. This disease has also been linked to the use of galolinium containing chemical dyes used prior to magnetic resonance imaging (MRI). Other than a successful renal transplantation, currently there are no effective treatment options available to reverse fibrosis in NSF. 

This new use takes advantage of the fact that in NSF a regulatory kinase called p70-s6 is expressed in phosphoinositol signaling cascade and that this kinase is the target of rapamycin.  This is the first evidence that the kinase is expressed in NSF fibroblasts and scleroderma fibroblasts.   Further this invention provides the first evidence that rapamycin is known to directly inhibit p70-s6 kinase resulting in rapid resolution of the disease and clinical improvement.   Clinical case studies report that rapamycin has been used on both NSF and scleroderma patients and there was a dramatic resolution of induration, swelling and pain within two weeks of therapy.  Rapamycin has been routinely used as an immunosuppressive medication in renal transplant patients.

08-23 Swaminathan et al

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