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Throughout history, human medicine in every form has had in principle a simple, yet extremely challenging mission: to cure. Although it might appear trivial in many cases, the ability to cure relies on knowledge of complex pathological processes, which allows the identification of therapeutic targets and solutions. Without a commitment to find solutions to the molecular causality of diseases, patients are likely to be treated long-term but not completely cured. Despite considerable progress in supportive care, life prolongation, and prevention of life-threatening complications, curability remains the only indicator of what we are able to truly cure and what we are able to only treat. Therefore, given the medical progress made to date, we should not mitigate our efforts to achieve the Holy Grail: to find safe, curative solutions without side effects or adverse events. Whether current research has a real or only an illusory prospect to accomplish this mission remains to be determined.

Despite the massive global spend on biology-driven drug discovery, tackling the issue of side effects and adverse events resulting from drug promiscuity represents a persistent challenge. Although delivering authentic medical innovations today is more complex than ever, minimization of off-target effects should be a priority.

According to a report by the Tufts Center for the Study of Drug Development, the average R&D cost of a new drug is currently estimated at US$2.6 billion, and drugs typically take 12 years to reach the market from the initial discovery stage. The juxtaposition of the growing knowledge of the molecular basis of biologically diverse diseases with rapid advances in material sciences is yielding an unprecedented promise for therapy innovations, which is evidenced by the more than 5000 drugs currently in development in the USA alone. Paraphrasing the words of Professor Richard L. Schilsky, former president of the American Society of Clinical Oncology: ‘Scientifically, we have never been in a better position to advance treatment as we have learned how to develop drugs that block key cellular pathways’. Given this perspective, it is plausible to expect that the extensive R&D effort will be rewarded by a qualitative shift in current treatment paradigms. However, despite worldwide promises to deliver authentic therapy innovations, reality lags behind expectation…

Razga F, Nemethova V. Selective therapeutic intervention: A challenge against off-target effects. Trends in Molecular Medicine. 2017; 23: 671-674. https://doi.org/10.1016/j.molmed.2017.06.007

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