Risk taking is complicated. In business, ebullient entrepreneurs are lauded for putting their livelihood on the line and taking risks, while serial gamblers who do the same are frowned upon. When it comes to drug discovery, the picture is more nuanced. It is an inherently risky endeavour, but society relies upon new drug discoveries and innovations.
Even so, investors, funding bodies and regulators are increasingly eschewing higher risk discovery projects in favour of lower risk projects. This is a problem because we need a lively and dynamic drug discovery sector if we are to have a hope of developing cures and treatments for the biggest human health challenges facing our society. One solution is building confidence through platform-based discovery, which increases the opportunity for investment as we have seen recently with major pharma looking at a wide range of platform technologies to in-license or acquire.
Neurodegeneration is one of the areas of biggest unmet health need, affecting up to a billion people worldwide according to the World Health Organization. For all the diseases that come under its auspices, there are no treatments that reverse disease, or even genuinely halt progression and hence, patient deterioration. At best, many of the market-‘leading’ medicines are only able to reduce symptoms or slow their advance.
The lesson we can learn from witnessing drug development in neurodegeneration over the last few decades is a simple one: if we want big solutions to complex problems, we need to think big and embrace both risk and complexity.
The holy grail for those of us working in this field revolves around the three Ns: neuroprotection, neuroplasticity and neurorepair. The latter two in particular have had little focus in terms of meaningful drug discovery. Neurorepair refers to the ability of nerve cells and neuronal tissue to regenerate; in essence this refers to the ability to reverse disease. Neuroplasticity refers to the brain’s requirement to change and rewire and is necessary for learning, adapting and creating new memories and abilities.
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