August 29th, 2012
Mesothelioma patients may have new hope with the introduction of a new drug in the United States. The drug, currently known as BAY 94-9343, is manufactured by Bayer HealthCare and is still in clinical development. However, the FDA has granted it orphan drug status to be used to treat mesothelioma patients.
The new mesothelioma drug contains an anti-cancer antibody-drug conjugate that targets mesothelin, which is a molecule released in high quantities by certain cancer tumors – including malignant mesothelioma tumors. Researchers hope the anti-cancer agent will work by combining antibodies with cytotoxic small molecules that attack and destroy mesothelioma cancer cells in the tumors.
Limited Options to Treat Rare Cancer
Right now, there is no cure for mesothelioma, and remains a large void for effective drugs used to combat the rapid spread of the disease. Mesothelioma is a malignant cancer that severely shortens the average life expectancy of the victims it affects. The cancer is caused by direct and secondary exposure to asbestos fibers, which were previously used in high quantities in U.S. manufacturing, despite warnings that the fibers could cause cancer and asbestosis. However, there is a long latency period associated with the cancer, meaning those who develop mesothelioma as a result of asbestos exposure may not be diagnosed for one to five decades following exposure.
The FDA provides orphan status to encourage drug makers to create potentially helpful drugs for diseases that are not particularly prevalent in the U.S. The FDA defines this as a disease that affects less than 200,000 people. Because mesothelioma is considered rare and affects only 3,000 people per year, it qualifies as an orphan disease. Under orphan status, drug makers are exempt from certain requirements, and may also be granted certain tax credits and other benefits to help fund clinical studies and drug development.
Clinical Studies Underway
For now, doctors are allowed to use BAY 94-9343 only in clinical studies for investigational purposes to determine its effectiveness in either slowing the progression of mesothelioma cancer cells, or otherwise extending the life expectancies of patients who take the drug. The study will not only examine the drug's possible effectiveness, but its safety too.
Right now, the trial is in clinical phase one, which aims to identify the maximum dosage of the drug that is found to be both safe and tolerable for patients. The initial phase will also determine its safety. Possible later clinical phases could be used to better identify the drug's effectiveness for treatment. The clinical trial – known as NCT01439152 – is being administered to patients at treatment centers in three U.S. states.
