It is with great pleasure that I welcome you to The Medicine Maker’s Advancing Medicine supplement, focusing on advanced therapies. The EMA defines advanced therapy medicinal products as medicines based on genes or cells that offer “groundbreaking new opportunities for the treatment of disease and injury”.
When I first started out on my journalistic career in medicine and pharmaceuticals (many years ago), I made a mistake common to many young, inexperienced journalists – I went straight to the most dramatic headlines I could find, including miracle cures for cancer and Alzheimer’s, nano robots that could potentially patrol the inside of the human body, inventive ways to repair spinal cord injuries, and giving mice superhuman (or supermouse) strength. My very patient manager at the time explained that certain research topics, while fascinating, were a very long way from the clinic. Included on that list were cell and gene therapies. It was early days for the field and at the time there was uncertainty over whether such therapies could ever make it to a patient’s bedside. Now in the summer of 2017, things are very different. A handful of cell and gene therapy products have already been approved by US or EU regulators – and more are set to follow. There is much excitement around Novartis’ investigational CAR-T therapy, CTL019, which has recently been recommended by an FDA panel (1) for treating relapsed or refractory pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL), the most common childhood cancer in the US. CTL019 is likely to be the first CAR-T therapy to hit the market, although Kite Pharma isn’t far behind (2), and other companies are also focusing on the CAR-T field.
Cell and gene therapies are still a very long way from the hype of complete cures and revolutionary treatment options, but it is encouraging to see the field progressing. We know that cell therapies can have clinical effects; the questions now are what conditions can benefit? How do we maximize efficacy and safety? And how do we scale up manufacture to reach larger patient numbers? In this supplement, we unite experts from across the field to discuss the progress being made in advanced medicines, and how the industry is approaching the complex challenges posed by the supply chain and commercial manufacture.
Stephanie Sutton
Editor
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References
- Novartis, “Novartis CAR-T cell therapy CTL019 unanimously (10-0) recommended for approval by FDA advisory committee to treat pediatric, young adult r/r B-cell ALL,” (2017). Available at: http://bit.ly/2uTmQhC. Last accessed July 18, 2017. Kite Pharma, “Kite Receives U.S. Food and Drug Administration Priority Review for Axicabtagene Ciloleucel,” (2017). Available at: http://bit.ly/2ql22kc. Last accessed July 18, 2017.
