Mesothelioma patients and families who were counting on an important clinical trial were dealt a tough blow this past week when it was announced that the study was coming to a sudden end. Boston-based Verastem Inc., a small biotech company established in 2010, stopped enrollment in the middle of its study of the drug VS-6063, which they were hoping would bring major positive changes to mesothelioma treatment.

Lou Vaickus, Verastem’s Interim Chief Medical Officer, said in a company press release:

“Malignant pleural mesothelioma is among the most aggressive and lethal cancers with only one approved therapy. With the aggressiveness of this disease, the use of single agent VS-6063 as a maintenance treatment following chemotherapy where all patients had residual disease was not sufficient. There remains a significant unmet medical need for new treatment options for patients suffering from this very complex, difficult-to-treat cancer.”

The only known cause of mesothelioma, the “lethal cancer” that Verastem’s CMO is referring to, is exposure to asbestos. Verastem decided to stop enrollment following a Data Safety Monitoring Board review. The results of the review showed that while the drug did appear to be safe, there was no evidence that it was actually working.

Clinical trials are conducted before a new drug or procedure can be approved by the U.S. Food and Drug Administration (FDA) and made available to the general population. Usually when a drug reaches the clinical trial stage, it has already been researched extensively and has begun to show promise in effectiveness. There are 3 main phases of clinical trials, and Verastem stopped after Phase 1. Phase 1 determines how much of the medication can be given safely, or whether multiple treatments can be combined. The last 2 phases can take years to get through, so even if the VS-6063 trial still had the green light, it could have taken years for it to become available to mesothelioma patients.

This news is not only a great disappointment to the mesothelioma community, but Verastem as well. Heralded as Verastem’s biggest clinical trial to date, it was a chance for Verastem to truly stake their claim in the biotech world. Since the study’s abrupt end, investors are showing their discomfort with the news and shares have gone down almost 50% in pre-market trading. Verastem’s stock is currently only about $3 a share — when the company went public back in January 2012, it was at $10 a share. It is unclear exactly how much the company spent on developing VS-6063, but at the end of Q2 this year, Verastem had $132.1 Million in cash and cash equivalents.

In Verastem’s year-end 2014 update and outlook for 2015, the company said their goal was to achieve the first approval of VS-6063 in mesothelioma. Then, they hoped to broaden its use to major cancers like lung, ovarian, and breast cancer. They said their work with the drug since 2013 had been encouraging, which was hopeful news for the mesothelioma community as a whole. This turn of events is undoubtedly a sad and surprising end to what was thought to be a potential major breakthrough in the treatment of mesothelioma.

Other Mesothelioma Clinical Trials Show Promise

While the cancellation of Verastem’s trial is a disappointing setback, it is important to think of the glass as “half full.” Promising news is coming out of other clinical trials around the world and hopefully the many researchers working on behalf of mesothelioma patients will eventually find an effective treatment.

  • Researchers from the University of Pennsylvania found they were able to stop or reverse tumor growth in more than 75% of malignant pleural mesothelioma patients who took the skin cancer drug pembrolizumab.
  • MTG Biotherapeutics (MTG), an immuno-oncology company, secured orphan drug designation for MTG-201, a potential mesothelioma treatment. MTG-201 is an advanced biologic therapy that targets a gene defect in various cancers, including malignant mesothelioma. It is currently in Phase 1 of clinical trials.
  • In a presentation at the 2015 annual meeting of the American Society of Clinical Oncology (ASCO), The French Cooperative Thoracic Intergroup (IFCT) discussed their findings in studying the drug bevacizumab (Avastin®). The researchers discovered that the addition of bevacizumab to the current standard of treatment improved progression-free survival by 2 months and overall survival by 2.75 months.

Although Verastem’s clinical trial is no longer underway, mesothelioma patients may still have a number of options. If you are interested in learning more about clinical trials for mesothelioma, a good place to start is, which provides a registry of federally and privately sponsored clinical trials, in the U.S. as well as 190 other countries. A recent search for “mesothelioma” showed 280 clinical trials, 101 of which were currently recruiting for participants or planning to recruit soon.

The road to discovering an effective mesothelioma treatment has been an ongoing challenge, but there have been too many promising breakthroughs along the way to give up. We can only hope that one day, the hard work of researchers around the world will pay off and mesothelioma patients and their families will have viable treatment options to fight this rare and deadly form of cancer.