Scientists are very excited about the idea of studying stem cells. These primordial structures come in many different forms. While some are retrieved from embryos, others are more well-developed and embedded within adult organs. In the laboratory, researchers can even modify adult skin cells to look and behave similarly to embryonic stem cells. However, a certain type of stem cell may even be responsible for driving persistent malignant diseases, such as mesothelioma.
Fortunately, pharmaceutical companies such as Verastem are working to eradicate these cancer stem cells.
Malignancies get a boost from stem cells
Doctors take several approaches to cancer, including the surgical excision of diseased tissue, targeted radiation therapy and powerful chemotherapeutic drugs. However, even if a patient’s malignancy appears to be in remission, the possibility of it growing back at the same site or another part of the body is always an issue.
For years, experts have suspected that this is because some cancers have a population of cells that act similarly to stem cells. That is, they use certain pathways for cell migration, have a heightened resistance to dying and may be able to withstand any damage caused by medication. Over time, scientists have located pockets of these cancer stem cells in diseases such as breast cancer and leukemia.
Arresting cancer stem cells may stop illness
The discovery of cancer stem cells was very important because it provided researchers with another potential target for therapy. If they attack these cells, they may be able to not only shrink a malignancy, but also prevent it from growing back.
At Verastem, scientists developed a drug candidate known as VS-6063. This agent prevents cancer stem cells from producing a protein known as FAK. While both healthy and diseased cells make this protein, it is particularly abundant among cancer cells.
“If you can take away that driver, you may prevent [cancer cells from] spreading as well…and you may prevent them actually embedding to form new metastases,” Joanna Horobin, Verastem’s chief medical officer, told The Boston Globe.
Scientists apply for orphan drug status
Currently, Verastem is recruiting participants for a clinical trial to evaluate the performance of VS-6063 as a treatment for ovarian cancer, which research suggests can be characterized by high levels of FAK. Additionally, based on other experiments on a similar drug, Verastem has decided to apply for orphan drug status of VS-6063 as a regimen for malignant mesothelioma in the U.S. and the EU. The company expects to initiate an experiment on this disease in the middle of 2013.
Orphan drug designation is conferred upon drug candidates for diseases that affect fewer than 200,000 people in the U.S., or a maximum of five in 10,000 people in the EU. This status allows the company to have exclusive rights for their product for seven years following marketing approval by the U.S. Food and Drug Administration, or for the ensuing 10 years after approval in EU nations. Furthermore, orphan drug designation comes with development grants, tax credits, protocol development assistance and other benefits.
Although orphan diseases such as malignant mesothelioma are relatively rare, that does not take away from the fact that these illnesses are devastating for patients and their loved ones. At Mesothelioma Circle, we’re happy to hear about any medication that receives orphan drug status for the treatment of diseases caused by asbestos exposure, and we cannot wait to see the progress that VS-6063 makes.