Discovery of Novel Drugs for Promising Targets

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Therapeutic drugs have positively affected virtually all diseases, especially on a global scale, yet many challenges remain. In the spectrum from basic research on promising new therapeutic targets to robust clinical trial infrastructure to clinical practice, an area that is less discussed is the gap between a promising target and first-in-human study. The present review focused on how the gap between basic research and clinical research is bridged by novel drug discoveries.

Drug discoveries across therapeutic areas have many similarities. Although the techniques described in the present review can be applied broadly, many examples are from the oncology arena. Cancer is a complex disease that accounts for staggering mortality and morbidity, affecting all ranges of healthy and functional people as well as those with multiple comorbidities and poor functional status. The multidisciplinary aspect of cancer therapy has allowed substantial improvement in outcomes, including survival; however, for the many patients who die of cancer, their disease is often disseminated and not amenable to local therapies, leaving systemic therapy as the primary option for those with advanced cancer.

Stubborn problems remain in discovering and developing anticancer drugs. Probably the biggest issue is cancer's complexity, with multiple genetic drivers and intratumoral variation. It is often difficult to determine the appropriate target or targets. Means of identifying appropriate targets may have limitations. For example, RNA interference is prone to off-target outcomes and cell type-specific effects. 1 Once a potential driver for a malignancy is identified, it may be difficult, or even impossible, to develop drugs against that target. For example, a tumor suppressor, which is absent in the actual malignancy, cannot be directly restored, let alone targeted. Another issue is lack of drug discovery, which may result in off-target effects, or even anticancer activity for which the mechanism is unclear.

Thus, recent innovative advances in our ability to identify targets, understand their function, discover molecules that perturb targets, develop methods to select the best drug, and design approaches to bring new drugs into human testing have provided much promise for treatment of cancer.