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Human Trials Planned for Promising New Mesothelioma Drug


The Australian Asbestos Diseases Research Institute says it is ready to begin human trials on what its lead researcher calls the first significant advance in mesothelioma treatment in a decade.

More than three years in development, TargomiRs utilizes a unique ‘minicell’ delivery system to insert a synthetic form of missing genetic material into mesothelioma cells. Like a number of other types of cancer, mesothelioma cells are missing a family of microRNAs critical to regulating the cellular life cycle. TargomiRs restores these microRNAs. In mice with human-derived mesothelioma, TargomiRs produced a “remarkable inhibition of tumour growth”, according to the Asbestos Diseases Research Institute.

“The last significant development in the treatment of mesothelioma occurred ten years ago,” ADRI director Nico van Zandwijk said in a press release. “While it is early days in the development of TargomiRs, in commencing this important world-first trial we hope to quickly find the optimal human dose and to enable us to take the trial to the next stage.”

The minicells that make up TargomiRs’ unique delivery system were developed by EnGeneIC, a biotech company in Sydney. The fact that TargomiRs was able to inhibit the growth of tumor cells in mice without affecting healthy cells suggests that the treatment may have fewer side effects than other types of mesothelioma therapies.

Mesothelioma is an asbestos-related disease that has traditionally occurred in people who have been exposed to asbestos. This aggressive cancer is now a major concern in Australia, where health experts are warning of a “third wave” of mesothelioma cases among homeowners exposed to asbestos during renovation projects.

The developers of TargomiRs say they will need an additional $750,000 in funding in order to conduct the planned clinical trial. The trial is expected to include 20 to 30 mesothelioma patients and is set to begin by the end of the year. “If the results are favourable (we’ll know in about 2 to 3 years), this may lead to a new form of treatment for patients with mesothelioma,” said Zandwijk.


“ADRI Announces a Phase I Clinical Trial of New Drug Designed for Malignant Mesothelioma”, July 31, 2013, ADRI website.
“World First Clinical Trial of New Cancer Drug by Australia’s Asbestos Diseases Research Institute”, July 31, 2013, PR Log, ADRI Press Release.

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