Missing drugs

Idiopathic Pulmonary Fibrosis (IPF) is one of one of many diseases for which there is no Food and Drug Administration (FDA) approved drugs in USA. This does not mean, however, that there are no drugs of possible benefit for this disease. Based on the recommendation of Thoracic Society some pulmonary physicians prescribe glucocorticoids (e.g. prednisone) with or without immunosuppressants (e.g. azathioprine) . Only occasional benefit has been reported with this approach. Without FDA approval or recommendation of a professional society physicians are understandably reluctant to prescribe drugs that can conceivably be useful in the treatment of IPF, although they have right to prescribe drugs off-label. The reasons for this reluctancy include potential lawsuits and cost of malpractice insurance. There is also no readily available information source on the off-label use of drugs approved for other indications. Neither FDA, nor textbooks or databases provide such information and pharmaceutical companies are strictly prohibited to inform physicians about potential off-label indications for their drugs. One of the drugs used off-label in IPF is N-methyl-acetylcysteine (known also as NAC), a glutathione precursor and powerful antioxidant and hepatoprotective. In USA NAC is approved and commercially available (ACETADOTE) as an antidote for acetaminophen intoxication, but not other indications. Its usefulness in the therapy of IPF in combination with glucosteroids was suggested by a clinical trial (1), that led to still more ongoing trials. Since NAC's toxicity is very low and there is an urgent need for IPF therapeutics, NAC should be made available to all IPF patients. Fortunately, it is available also as a dietary supplement from Puritan's Pride, although this company cannot provide any information on its use in IPF. Even if additional clinical trial confirm effectiveness of NAC in IPF, it will probably never become a standard IPF therapeutic, since no pharmaceutical company will be capable of covering the cost of NAC development for this new indication without use patent coverage. NAC's availability for a very low cost as a dietary supplement will also further complicate its development as an IPF drug.