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Drugs that block Janus kinases are showing promise in diseases ranging from myelofibrosis to rheumatoid arthritis, but with the leading agent in line for approval questions remain about their optimal selectivity.
Over a decade since gene therapy development came to a near standstill with the death of a clinical trial participant, the field is overcoming issues of immunogenicity, carcinogenicity, manufacturing and small patient populations.
Paul Brown, CEO of Roche Molecular Diagnostics, discusses the regulatory and reimbursement concerns that challenge the success of companion diagnostics.
Predictive biomarkers allow the identification of the subsets of patients who will benefit from a particular drug. However, the development of biomarker–drug combinations requires novel clinical trial designs. In their Perspective, Beckman and colleagues formulate guidelines for the adaptive integration of predictive biomarkers into Phase II/Phase III clinical trials and present strategies to achieve optimal efficiency of such trials.
In the past 15 years, it has become clear that physicochemical properties of drug candidates, such as lipophilicity and molecular mass, have an important influence on the likelihood of compound-related attrition during development. By analysing the properties of compounds described in patents from leading pharmaceutical companies between 2000 and 2010, this article indicates that a substantial part of the industry has not modified its drug design practices accordingly and is still producing compounds with suboptimal physicochemical profiles.
The ability of tumour cells to maintain a slightly alkaline intracellular pH and an acidic extracellular pH aids the growth of primary tumours and the formation of metastases. Inhibiting pH-regulating proteins in tumours represents a novel therapeutic strategy that is not exploited by the classical anticancer drugs.
Classical selective oestrogen receptor modulators are well established in the treatment of breast cancer and osteoporosis, but emerging knowledge on the oestrogen receptor subtypes α and β suggests that subtype-selective modulators may hold promise for more efficacious and safer treatments of many other diseases and symptoms.
