A new drug has recently become another treatment option for patients battling from malignant pleural mesothelioma. The drug, Amatuximab made by Morphotek, was recently granted orphan drug status by the FDA.
A drug may be designated an orphan drug if it is used to treat fewer than 200,000 people in the U.S., thanks to the Orphan Drug Act. If a disease affects fewer than 200,000 people, it is considered rare. Mesothelioma is amongst the rarest of rare diseases, affecting fewer than 3,000 Americans every year. The Orphan Drug Act provides incentive for companies like Morphotek so that they are able to work to develop drugs to treat rare diseases.
Mesothelioma may take up to decades to develop and often shows few symptoms until its advanced stages, so it is especially difficult to treat, making orphan drugs an especially critical option. The life expectancy for mesothelioma patients, even with such standard treatments as chemotherapy, radiation, and surgery, is poor. Researchers and mesothelioma doctors hope that studies on gene therapy and immunotherapy treatments, like Amatuximab, will offer more promising odds to mesothelioma patients.
The glycoprotein mesothelin is overexpressed in a number of types of cancer including pleural mesothelioma, and Amatuximab targets this protein. Experts believe that mesothelin is related to cell adhesion, allowing a tumor to adhere to a particular spot and encourage new tumor growth in other parts of the body. In a press release announcing Amatuximab’s new orphan drug status, Morphotek noted, “Researchers at the National cancer Institute (NCI) and Johns Hopkins University have independently validated mesothelin as a potential target of immune-based therapies.”
The National Cancer Institute states that Amatuximab binds to the mesothelin on cancer cells’ surfaces, triggering an immune response against the tumor cells that express mesothelin.