Noting that the result is preliminary and must be evaluated in clinical trials, Australian researchers working with the Asbestos Disease Research Institute published a case report announcing significant improvement for a pleural mesothelioma patient treated with microRNA therapy.  The results were reported in the American Journal of Respiratory and Critical Care Medicine.

Following in vitro testing and animal experiments, the researchers used a nanocell delivery platform to provide patients with synthetic mimics of microRNA that had been under expressed in mesothelial tumors.  One patient with progressive mesothelioma experienced “a complete metabolic response” as measured by PET scan as well as marked improvement in pulmonary function test results, with some minor side effects.  Other patients in the six-person cohort experienced stable disease with one reporting progressive disease.  A second cohort is being recruited to determine whether the result is repeatable, further study appropriate dose and investigate the causes for the variability in the results in the first group.

As the researchers report, microRNA therapy has the potential to be a paradigm shift in the management of treatment-resistant tumors generally and mesothelioma specifically.  Earlier this year, the U.S. Food and Drug Administration (FDA) granted a drug in development to treat malignant pleural mesothelioma “orphan drug” designation (for the related post click here).  For additional discussions about torts, science, and medicine, visit Kirk Hartley’s informative and thought-provoking blog, globaltort.com.