What can independent research for Mesothelioma achieve to treat this orphan disease?

Malignant pleural mesothelioma (MPM) is a rare neoplasm with a poor prognosis, as current therapies are ineffective. Despite the increased understanding of the molecular biology of mesothelioma, there is still a lack of drugs that dramatically enhance patient survival. The present review discusses recent and complete clinical trials supported by the NIH, other U.S. Federal agencies, universities and organizations found on clinicaltrials.gov. Firstly, chemotherapy-based trials are described, followed by immunotherapy and multitargeted therapy. Then drug repositioning and the use of drug docking as tools to find new interesting molecules are introduced. Finally, potential molecular pathways that may play a role in mesothelioma biology and therapy are highlighted. Numerous biases are present in the clinical trials due to a restricted number of cases, inappropriate endpoints and inaccurate stratification of patients which delay the finding of novel, successful treatments for MPM. The most crucial issue of independent research for MPM is the lack of more substantive funding to translate these findings to the clinical setting. Furthermore, many trials for mesothelioma adopt therapies utilised successfully in other cancers. However, this approach is not necessarily scientific due to the low mutational load of mesothelioma relative to other cancers. It is therefore clear that patients need a more solid rationale and bedrock of preclinical research to support these trials, to ultimately have a better chance of successful treatment.