An announcement made last week has the mesothelioma community abuzz over a clinical trial that brings the possibility of a cure one step closer to reality. Due to a surprise donation of $1.2M, the Asbestos Diseases Research Institute of Australia has fast-tracked the trial for a drug that could “stop mesothelioma tumors in their tracks.”
With the early start of the clinical trial, “we have jumped one-and-a-half years ahead,” said Professor Nico van Zandwijk, Director of the ADRI and project lead, when announcing the trial.
“It means that we might have a cure in a few years,” added Federal Health Minister Tanya Plibersek.
Three years of work spent determining the genetic characteristics of mesothelioma and how to best stop the tumor cells from growing and dividing culminated in this Phase I study that will focus on patients with malignant pleural mesothelioma. The trial, according to the ADRI’s press release, is scheduled to commence at the end of this year and run through the end of 2014.
World’s First Mesothelioma Drug Trial
Mesothelioma is an aggressive cancer that currently has no cure and limited treatment options. However, with a series of breakthroughs that make this trial “the world’s first mesothelioma drug trial,” Prof. van Zandwijk and his team believe TargomiRs will bring a much-needed new, effective treatment to mesothelioma patients.
When asked what sets this trial apart from others, making it the first of its kind, Prof. van Zandwijk responded to MesotheliomaHelp that the research team had multiple “firsts” that broke many of the previous approaches taken to researching mesothelioma. Prof. van Zandwijk said the unique study offers:
- A new concept through substitution of synthetic microRNAs for patients with malignant pleural mesothelioma.
- The drug delivery concept using a microRNA construct delivered through targeted mini-cells makes it a first study with this approach in malignant pleural mesothelioma.
- All of the team’s preclinical studies were carried out in mesothelioma models.
- The Phase I study will focus on patients with malignant pleural mesothelioma (failing on standard therapy), which is a deviation from the commonly used Phase I approach.
The Science Behind the Trial
In “Human Genetics: Concepts and Applications, Tenth Edition,” geneticist and author Ricki Lewis explains that certain microRNAs are either “more or less abundant in cancer cells than in healthy cells of the same type from which the cancer cells formed.” Thus, restoring levels of microRNAs to normal can impact the growth of the cancer cells.
In this case, the researchers at ADRI found that “a particular family of microRNAs” was significantly decreased in the mesothelioma tumors. The line, microRNA-16 (miR-16), has been associated with breast, colon, brain, prostate and other cancers, but this is the first time this microRNA has been linked to mesothelioma. In order to restore the levels, the ADRI team manipulated the genes by adding synthetic versions of the microRNAs to increase the count. However, they needed to deliver the microRNAs directly to the tumors, so in another first time breakthrough approach, the drug was delivered via a mini-cell delivery system “able to transport a drug to the tumor area guided by antibodies.”
The therapy, conducted in mice-models with human mesothelioma-derived tumors, not only inhibited the growth of the tumor cells, but also left the normal cells unaffected, resulting in minimal to no side-effects. The result, Prof. van Zandwijk concluded, showed “a remarkable inhibition of tumour growth, far greater than in studies of other agents in the same model.”
The clinical trial will be open to 20-30 malignant pleural mesothelioma patients. The goal of the trial is to determine the optimal and safe dose of the new drug. The funding for the trial was donated by the family of Andrew Lloyd who died of malignant mesothelioma in 2011.
For more information go to http://www.adri.org.au/.
Today, nearly 650 new cases of mesothelioma are diagnosed each year in Australia. According to reports, 40,000 more Australians could die from asbestos-related diseases in the next twenty years if a cure is not found. 3,000 mesothelioma cases are diagnosed in the U.S. annually.
This clinical trial focuses on personalized treatment by targeting the unique genetic characteristics of pleural mesothelioma. Personalized care optimizes the potential for success of the treatment and offers treatment options that may not otherwise have been considered.
About the Asbestos Diseases Research Institute
The Asbestos Diseases Research Institute aims to improve the prevention, the diagnosis and treatment of asbestos-related diseases and to provide a better future for all those Australians unfortunately exposed to asbestos.
The Asbestos Diseases Research Institute is the first stand-alone research institute tackling the still increasing epidemic of asbestos-related diseases. The ADRI was established by the Asbestos Diseases Research Foundation, a charitable, not-for-profit foundation. The ADRI is located on the Concord Hospital campus in the Bernie Banton Centre which was officially opened by the Prime Minister, the Hon Kevin Rudd in January 2009.