Clinical pharmacology and therapeutics
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The recent emergence of a multitude of synthetic cannabinoids (SCs) has generated a wealth of new information, suggesting the usefulness of state-of-the-art on lato sensu cannabinoids. By modulating a plurality of neurotransmission pathways, the endocannabinoid system is involved in many physiological processes that are increasingly explored. ⋯ Neurological and cardiovascular side effects observed after cannabinoid poisoning generally respond to conventional supportive care, but severe outcomes may occur in a minority of cases, mainly observed with SCs. The likelihood of severe abuse and addiction produced by SCs are of concern for the scientific community also interested in the potential therapeutic value of cannabinoids.
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Clin. Pharmacol. Ther. · Jan 2017
ReviewCan Cystic Fibrosis Patients Finally Catch a Breath With Lumacaftor/Ivacaftor?
Cystic fibrosis (CF) is a life-limiting disease caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) activity. The recent US Food and Drug Administration (FDA) approval of lumacaftor combined with ivacaftor (Orkambi) targets patients with the F508del-CFTR. The question remains: Is this breakthrough combination therapy the "magic-bullet" cure for the vast majority of patients with CF? This review covers the contemporary clinical and scientific knowledge-base for lumacaftor/ivacaftor and highlights the emerging issues from recent conflicting literature reports.
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This translational report on the current field of emerging cell therapy and tissue engineering therapies includes the challenges and opportunities to accelerate clinical translation in regenerative medicine. Translation of regenerative medicine refers to the transfer from bench (proof-of-concept) to bedside (clinical trials), and finally commercialization. Regenerative medicine therapies have the capacity to replace, repair, and regenerate cells, tissues, and organs to restore normal function in the body. These emerging therapies are shifting the paradigm from treatment-based to cure-based therapies.
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Clin. Pharmacol. Ther. · Dec 2016
Leveraging Industry-Academia Collaborations in Adaptive Biomedical Innovation.
Despite the rapid pace of biomedical innovation, research and development (R&D) productivity in the pharmaceutical industry has not improved broadly. Increasingly, firms need to leverage new approaches to product development and commercial execution, while maintaining adaptability to rapid changes in the marketplace and in biomedical science. Firms are also seeking ways to capture some of the talent, infrastructure, and innovation that depends on federal R&D investment. ⋯ One example of these external innovation initiatives is the Sanofi-MIT Partnership, which provided seed funding to MIT investigators to develop novel solutions and approaches in areas of interest to Sanofi. These projects were highly collaborative, with information and materials flowing both ways. The relatively small amount of funding and short time frame of the awards built an adaptable and flexible process to advance translational science.
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Clin. Pharmacol. Ther. · Dec 2016
Real World Data in Adaptive Biomedical Innovation: A Framework for Generating Evidence Fit for Decision-Making.
Analyses of healthcare databases (claims, electronic health records [EHRs]) are useful supplements to clinical trials for generating evidence on the effectiveness, harm, use, and value of medical products in routine care. A constant stream of data from the routine operation of modern healthcare systems, which can be analyzed in rapid cycles, enables incremental evidence development to support accelerated and appropriate access to innovative medicines. ⋯ Adaptive biomedical innovation (ABI) with rapid cycle database analytics is successfully enabled if evidence is meaningful, valid, expedited, and transparent. These principles will bring rigor and credibility to current efforts to increase research efficiency while upholding evidentiary standards required for effective decision-making in healthcare.