Therapies for rare diseases: therapeutic modalities, progress and challenges ahead

Tambuyzer, Erik; Vandendriessche, Benjamin; Austin, Christopher P.; Brooks, Philip J.; Larsson, Kristina; Needleman, Katherine I. Miller; Valentine, James; Davies, Kay E.; Groft, Stephen C.; Preti, Robert A.; Oprea, Tudor I.; Prunotto, Marco

doi:10.1038/s41573-019-0049-9

Cited by 229 publications

(205 citation statements)

References 153 publications

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“…[22][23][24] This provides excellent opportunities for drug repurposing. 25 Almost all drugs used in human therapy have the potential to address more than one target. 22,26 Thus, if the targets of these drugs are highly consistent with cancer, there is a high likelihood that those which share common targets could be therapeutic for other cancer patients.…”

Section: Introductionmentioning

confidence: 99%

Overcoming cancer therapeutic bottleneck by drug repurposing

Zhang

Zhou

Xie

et al. 2020

Sig Transduct Target Ther

364

315

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Ever present hurdles for the discovery of new drugs for cancer therapy have necessitated the development of the alternative strategy of drug repurposing, the development of old drugs for new therapeutic purposes. This strategy with a cost-effective way offers a rare opportunity for the treatment of human neoplastic disease, facilitating rapid clinical translation. With an increased understanding of the hallmarks of cancer and the development of various data-driven approaches, drug repurposing further promotes the holistic productivity of drug discovery and reasonably focuses on target-defined antineoplastic compounds. The "treasure trove" of non-oncology drugs should not be ignored since they could target not only known but also hitherto unknown vulnerabilities of cancer. Indeed, different from targeted drugs, these old generic drugs, usually used in a multi-target strategy may bring benefit to patients. In this review, aiming to demonstrate the full potential of drug repurposing, we present various promising repurposed non-oncology drugs for clinical cancer management and classify these candidates into their proposed administration for either mono-or drug combination therapy. We also summarize approaches used for drug repurposing and discuss the main barriers to its uptake.

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Section: Introductionmentioning

confidence: 99%

Overcoming cancer therapeutic bottleneck by drug repurposing

Zhang

Zhou

Xie

et al. 2020

Sig Transduct Target Ther

364

315

View full text Add to dashboard Cite

show abstract

“…In consideration of the involvement of microRNAs in the post-transcriptional regulation of IL-8 and other pro-inflammatory genes, both antagomiR and miRNA replacement approaches have been proposed ( 199 – 202 ). This confirmed that, in addition to relevance for the theoretical point of view, the studies on epigenetic regulation of chemokines (described in Chapter 5) might be important for the development of therapeutic protocols.…”

Section: Targeting Neutrophil Chemotaxis In Cf: Novel Molecules and Dmentioning

confidence: 99%

Role of Cystic Fibrosis Bronchial Epithelium in Neutrophil Chemotaxis

et al. 2020

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“…Key issues will be essential to approach as the field develops, including the engagement of different stakeholders, such as regulatory agencies, pharmaceutical industry and patients' groups, in order to bridge the gap between basic research and therapy development. Global collaborations will be essential to boost the progress of the field and make it a health priority [23].…”

Section: Future Perspectivesmentioning

confidence: 99%