Verastem Inc. to Begin Developing Novel Mesothelioma Drug

Male researcher examines sample under microscope

Nothing generates excitement in the cancer community quite like the development of a promising new therapy.

Such developments are especially exciting for mesothelioma patients. With only one FDA-approved mesothelioma drug on the market, more options are desperately needed. And while researchers have found ways to make existing drugs more applicable for mesothelioma treatment, nobody has recently created a new drug specifically for the disease.

Verastem, Inc. a Massachusetts-based biopharmaceutical company plans to change that.

“We feel that there is such a large medical need here, with so little approved,” Robert Forrester, Verastem’s President and Chief Operating Officer, told Asbestos.com.

They’re right about the void, and they’re moving quickly to fill it.

In summer 2012, they acquired the commercial rights to a compound that can inhibit cell growth. (For now, the compound is known as VS-6063.) Earlier this month, they revealed their plans to test its efficacy in mesothelioma patients at some point in 2013.

“Our lead drug, VS-6063, has been in development for a number of years. It’s been shown to be safe and have activity in a number of tumors, so we are now moving it into what we believe to be a pivotal trial, sometime around the middle of this year,” said Forrester.

Gearing Up for a Phase II Trial

“In late spring we’ll start to release more details about where the trial will be held. Right now, we’re planning on about 30 to 50 centers,” Forrester explained. “They’ll primarily be centers of excellence in big areas like Boston, New York, Los Angeles and San Francisco.”

International hospitals may also participate, as Verastem is in final negotiations with several European regulatory agencies.

Overall, they hope to recruit between 300 and 350 mesothelioma patients for the clinical trial. Ideal participants will have had a partial or complete response to first-line therapy.

Who will respond to the therapy?

Once the recruiting period is over, researchers will divide the patients into two categories: Merlin-positive and Merlin-negative.

Merlin is a protein that functions as a tumor suppressor. Verastem estimates that around half of the patients who lack the protein (Merlin-negative patients) may be especially responsive to treatments like VS-6063. Verastem’s partner LabCorp is working on a test to determine if the protein is present.

“They’re making a clinical test so when a mesothelioma patient presents to their doctor, they can conduct the test to see which drug [the patient] will respond to,” said Brian Sullivan, from Verastem’s corporate development department.

A 29-patient phase I study by researcher GlaxoSmithKline using a drug candidate in the same target class as VS-6063 found that merlin-negative patients had a much longer progression-free survival period after treatment. For merlin-positive patients, the median progression-free survival period was 11 weeks; for merlin-negative patients, it was 24 weeks. Since the GlaxoSmithKline drug candidate is also a FAK inhibitor, the study offered solid proof that FAK inhibitors could serve as a therapeutic agent for mesothelioma.

“They saw an average progression-free survival of 18 weeks [when Merlin-positive and Merlin-negative patients were combined]; a dramatic improvement,” Sullivan shared. Dramatic indeed, considering that Sullivan says, “Traditionally, you would expect to see a progression-free survival period of about six weeks.”

Zoning in on Cancer Stem Cells

So how does the drug itself work?

VS-6063 is a focal adhesion kinase (FAK) inhibitor. It regulates tumor growth by attacking the dividing cells that are ultimately responsible for metastasis.

While these cells are technically cancer stem cells, “we prefer to call them tumor-initiating cells,” Forrester clarified. This helps prevent confusion with the highly controversial embryonic stem cells.

“They’re the bad actors in the play of cancer. These are the cells that lead to occurrence and recurrence of the tumor,” Forrester explained. “We believe that for mesothelioma, they are highly implicated in the growth of the tumor. If we can kill them off, we can keep the tumor in check for a longer period.”

“Very luckily, the science around what this drug is has garnered attention from all the major mesothelioma leaders across the globe,” Forrester shared.

As they check off each step in the development process, they’ll certainly attract even more attention. And as we follow their progress, we’ll all be hoping for the best.

Check back with Asbestos.com later in the year for more updates about the trial, as well as information on enrollment.


After graduating with an English degree from Southeastern University in 2010, Faith Franz came on board as a health and wellness writer for The Mesothelioma Center.

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