Tag Archives: orphan drug

Putting the Newest Mesothelioma “Orphan Drug” Into Perspective

Last week, the FDA announced the approval of a new orphan drug for the treatment of malignant pleural mesothelioma. According to drug manufacturer MTG Biotherapeutics, MTG-201 is an “advanced biologic therapy” that targets a particular gene defect present in cancer cells. This defect prevents cells from producing a protein that is critical in apoptosis or programmed cell death. Without it, mesothelioma cells are free to replicate out of control and migrate to others parts of the body. MTG-201 also appears to trigger the immune system to produce mesothelioma-fighting T-cell lymphocytes that can target and destroy residual cancer cells. The two mechanisms together give it the potential to help combat mesothelioma in a new and potentially more effective way than conventional therapies. … Continue reading Putting the Newest Mesothelioma “Orphan Drug” Into Perspective »

Orphan Drugs Increase for Mesothelioma

There is some good news for mesothelioma sufferers in a recent report from Thomson Reuters on the orphan drug market. According to Reuters, rare diseases like mesothelioma are “winning unprecedented attention” from drug manufacturers. More than a quarter of the 39 new drugs approved by the FDA in 2012 were granted orphan drug status because they were designed to treat diseases (such as mesothelioma) that affect fewer than 200,000 people a year. Mesothelioma is an aggressive asbestos-linked cancer of internal membranes. It is considered an orphan disease because it claims the lives of about 2,500 Americans annually. The Reuters report on orphan drugs is good news for mesothelioma patients because few treatment options currently exist for them and their prognosis is … Continue reading Orphan Drugs Increase for Mesothelioma »

New Mesothelioma Drug Granted Special FDA Status

The Federal Drug Administration (FDA) has granted orphan drug status to a medication that may help boost the effectiveness of the standard cisplatin/pemetrexed chemotherapy combination for mesothelioma. CBP501, produced by the Japanese Drug company CanBas, is a novel synthetic peptide that seems to enhance the effectiveness of cisplatin by acting on multiple pathways that govern the lifecycle of cells and the natural repair of DNA damage. By modulating the production of a certain enzyme, it allows mesothelioma cells to become more susceptible to the damaging effects of platinum (cisplatin) build-up. At the same time, it prevents the cells from properly repairing themselves. The drug has also shown the ability to resensitize mesothelioma cells that have become resistant to cisplatin. The FDA’s … Continue reading New Mesothelioma Drug Granted Special FDA Status »

Orphan Drug May Help Shrink Mesothelioma Tumors

The developers of an FDA-designated ‘orphan drug’ for mesothelioma are recruiting patients for a new Phase II clinical trial on soft tissue sarcomas. The drug, called NGR-hTNF is a vascular targeting agent that appears to be able to seek out tumor cells in the body and disrupt their blood vessel formation. An orphan drug is a medicine that has been developed specifically to treat a rare medical condition, such as mesothelioma or liver cancer. The new clinical trial will test NGR-hTNF’s effectiveness against certain soft tissue sarcomas alone or in combination with the drug doxorubicin. In addition to the newly-announced clinical trial, the drug’s manufacturer, Italy-based MolMed S.p.A, has also been conducting Phase III clinical trials specifically in malignant pleural … Continue reading Orphan Drug May Help Shrink Mesothelioma Tumors »