Clapper, Patti, Schweizer and Mason

The results of a recent case study involving a highly experimental drug that could be used as a more effective mesothelioma treatment have many experts sitting up to take notice.  The results of the study were presented early November at the 24th EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics held in Dublin, Ireland.  The results show that a drug known only as GSK2256098 shows remarkable ability to slow and even stop the spread of mesothelioma within certain patient populations. 

The Phase I Trial was designed to assess the effectiveness of the new drug before it passed on to further testing which would subject human patients to varying doses in order to determine safety thresholds in correspondence with response levels.  Scientists discovered that the drug worked best in patients who did not display a certain biomarker.  During this research phase most of the focus was on the so-called “Merlin Gene” or NF2.

NF2 is a gene that essentially tells growing cells how they should look (shape, size, etc.) after the growth process is completed – in essence, a blueprint for the physical properties of a cell.  This Merlin Gene also effects how cells communicate with each other – a very important thing to keep in mind when attacking the mechanisms behind the spread of deadly cancers such as mesothelioma.  

Once a patient has been diagnosed with mesothelioma, the presence (or lack thereof) of the Merlin gene dramatically impacts how the disease spreads.  Research has shown that patients without the NF2 gene or with defective or “inactive” NF2 genes show much higher rates of cancer growth and metastasis.  What makes this NF2 deficiency doubly dangerous is the fact that when this gene isn’t present or shuts down, another protein compound called focal adhesion kinase (FAK) increases.  This FAK acts as a sort of accelerant for the growth and spread of cancers such as mesothelioma. 

Specifically, patients with healthy or active NF2 were shown to resist the spread of mesothelioma for 24 weeks on average.  Patients with the deficiency or inactive NF2 genes showed progression-free survival for only 11 weeks.

This new drug, GSK2256098, was designed to step in and work to counter the growth and spread of cancers and has been shown to work most effectively in patients with this Merlin Deficiency.  Moreover, doctors and mesothelioma experts can now test for this NF2 deficiency much earlier in the diagnosis process and determine not only the potential length of time a patient has before disease progression but also plot a more effective course of treatment with existing and experimental drugs and compounds such as GSK2256098.

However, before this new drug can be used in a “commercial” setting, it must first pass through at least two more rounds of testing to determine its safety in a human population.   Additionally, the results of these tests must be examined by the FDA and the drug itself must pass through an approval process. 

Scientists are actively recruiting willing patients for clinical trials involving this potentially powerful mesothelioma treatment.

Source:  Mesothelioma drug slows disease progression in patients with an inactive NF2 gene.