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

• Published in: Expert opinion on investigational drugs Vol. 28; no. 8; p. 719
• Main Authors: Guazzelli, Alice, Meysami, Parisa, Bakker, Emyr, Bonanni, Ezio, Demonacos, Constantinos, Krstic-Demonacos, Marija, Mutti, Luciano
• Format: Journal Article
• Language: English
• Published: England 03.08.2019
• Subjects: Animals; Antineoplastic Agents - administration & dosage; Antineoplastic Agents - pharmacology; Drug Repositioning; Humans; Immunotherapy - methods; Lung Neoplasms - drug therapy; Lung Neoplasms - pathology; Mesothelioma - drug therapy; Mesothelioma - pathology; Mesothelioma, Malignant; Molecular Docking Simulation; Molecular Targeted Therapy; Pleural Neoplasms - drug therapy; Pleural Neoplasms - pathology; Prognosis; Rare Diseases - drug therapy; Rare Diseases - pathology; Survival Rate; malignant pleural mesothelioma; targeted therapy; Chemotherapy; immunotherapy; independent research; drug repositioning; small molecules
• ISSN: 1744-7658
• DOI: 10.1080/13543784.2019.1638363

: 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. : This 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 we introduce drug repositioning and the use of drug docking as tools to find new interesting molecules. Finally, we highlight potential molecular pathways that may play a role in mesothelioma biology and therapy. : 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 a treatment 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. However, this approach is not necessarily scientific given the low mutational load of mesothelioma relative to other cancers, and therefore patients need a more solid rationale to have a good chance of successful treatment.