The new age of tailored medicine
Personalised medicine is all about treating an individual based on the specifics of his or her disease. However, both current EMA or FDA regulations and present clinical practice are stochastic, and thereby fundamentally in opposition. If we wish to truly usher in the era of personalised medicine, we need to change this environment. The TREAT1000 project is a good way to begin.
Imagine that you’ve taken your Ferrari to the mechanic because of a breakdown, and the mechanic consults a chart that tells him that a Ferrari with this problem needs a new fuel filter 30% of the time. So he changes your fuel filter. But the problem persists, so he replaces the the next item on the chart – the fuel injectors. This is how he continues until the problem is fixed. Wouldn’t you prefer to go to a mechanic who actually understands how the different parts of your car work together?
Unfortunately, this is still the current “state-of-the-art” in cancer medicine. Your doctor knows what type of cancer you have, and may also know a few additional things about it. Even so, physicians treat cancer patients based primarily on the stage and tissue of origin of their cancer, with no real understanding of what has gone wrong in their tumour cells. For a long time, this has been the only possible treatment, because establishing a cellular blueprint of an individual’s cancer was impossible, both in practical and economic terms.
However, as we approach the 10th anniversary of the UC Santa Cruz publication of the first working draft of the human genome, rapidly dropping sequencing costs will soon make full genome sequencing of all cancer patients a routine diagnostic.Along with the other dividends of the genome project – like the accumulated body of knowledge on the general molecular mechanisms of cancer – this will allow doctors to build a detailed blueprint of the workings an individual’s cancer cells. This very personalised information will allow physicians to identify the right therapy, greatly increasing the chance of successful treatment and reducing side-effects.
Substantial changes in regulatory and medical practice must take place for that to happen, and both the EMA and the FDA are well aware of the challenges. In a recent speech, former FDA Commissioner Andrew von Eschenbach touched on them, saying that “the transformation is not a linear extrapolation of the past. It’s a metamorphosis. The future will look no more like the past than a butterfly looks like a caterpillar.”
A good example for the next step in the development of true individualised medicine for cancer patients is a transatlantic EU-US pilot project called TREAT1000 (www.treat1000.org). Its goal is to identify the somatic changes in the genomes and transcriptomes of 1,000 cancer patients and to put their diagnoses, therapy recommendations, and clinical data into the public domain. This will allow us to begin a fact-based dialogue with regulatory agencies and medical professionals about the best way to ensure that von Eschenbach’s caterpillar turns into a butterfly.