Botox is a household name when it comes to vanity and related calls for cosmetic surgery. Produced by bacteria (Clostridium botulinum), botulinum is a powerful neurotoxin—it seems strange to me that people have overcome such dangerous sounding words to repeatedly request that it be injected in their face. But then again, fans of tattoos and piercings are not phased by thick needles, pain (or disfigurement), and (off-topic but interesting) a British student recently had her tongue surgically lengthened to help improve her Korean pronunciation. We’re ok with body modification then. And why not?
Allergan’s Botox was in the news again recently, and this time not over a debate of whether a C-list (and unknown to me) celebrity had or hadn’t been for treatment, but because it received a positive opinion from 14 European countries for a new indication: urinary incontinence. The DIGNITY programme (Double-blind InvestiGation of purified Neurotoxin complex In neurogenic deTrusor overactivitY—this must be some new form of acronym creation technique… admittedly memorable and appropriate) consisted of two successful trials involving nearly 700 patients with either spinal cord injury or muscular schlerosis, according to an Allergan press release. The positive opinion from the Irish Medicines Board, under the Mutual Recognition Procedure, is a key step in securing national licenses in the 14 countries involved.
This got me thinking about indications on a wider scale. A recent BBC report discussed a study that claimed that ordinary painkillers could be more effective at treating dementia than the antipsychotic drugs often prescribed, which have powerful sedative effects and can sometimes make dementia worse. On the other side of the coin, the FDA recently withdrew approval for Roche’s Avastin for use in the treatment of metastatic breast cancer because there is not enough data to support the conclusion that the benefits outweigh the risks…
Clearly, research is ongoing in terms of New Drug Applications, and meanwhile, off-label use continues (and punishment off-label marketing continues also). But there is an issue here, and patients’ lives are potentially at risk (read our previous blog). In 2009, the FDA finalised guidance regarding the distribution of medical or scientific journal articles that involve unapproved uses of FDA-approved drugs, but perhaps this only increases the grey area rather than define it. Nevertheless and despite the risk, there is huge potential out there—potential for existing drugs to improve patients’ lives in new ways, especially when thorough research has proved the benefits and opened regulatory doors.