Metabolism is described as the series of chemical reactions required to sustain life, and when it comes to drug metabolism no one does it better than the cytochrome P450 family of enzymes. Understanding of key enzymes within this superfamily – such as CYP2D6, CYP2C9 and CYP3A4 – is an important part of drug design. Were you expecting The Incredibles? Sorry, not that super family, but still super nonetheless!
Remember those ‘get out of jail free’ cards in the board game Monopoly, with somewhat dubious value (i.e. you’d sell them right before you landed in jail) that could get you back on track quicker? It turns out that this express ticket exists in the drug discovery world, and we’ll take you on this multi-million dollar ride.
Why does regulation matter in drug discovery? It may seem like a big obstacle in the path to delivering novel medicines to patients, delaying their treatment and prolonging discomfort. But how do we know that the drugs we take actually have their intended purpose? Are we aware of any potential side effects/adverse reactions? This is a true story of how a heroine dared to ask these questions, and saved a nation in the process.
The process of taking a synthesized compound to market as a drug takes years, even decades, with the monetary cost of such an endeavor averaging $2 billion per approved drug. With such high risk and no promise of reward, companies and institutions all around the world still work toward inventing, testing and manufacturing these drugs.
The past few months have been revolutionary in the history – and perhaps more so the future – of medicine. In August 2017, the US Food and Drug Administration (FDA) gave its first gene therapy approval to Kymriah for treatment of acute lymphoblastic leukemia. Just weeks later Yescarta was approved for non-Hodgkin lymphoma. By Christmas that year, Luxturna became the first ever in vivo gene therapy to be FDA approved. In March 2018, Luxturna was used successfully to treat a young patient, preventing him from going blind.