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Recent progress in the discovery, development and evaluation of new drugs and combination regimens for drug-resistant tuberculosis through greater collaboration between industry, donors and academia provides renewed hope for overcoming the challenges in tuberculosis treatment.
Ongoing and upcoming clinical trials of metformin, mTOR inhibitors and senescent cell-killing drugs could prove that ageing and ageing-related diseases are viable drug discovery indications.
Penny Heaton, CEO of the Bill and Melinda Gates Medical Research Institute, discusses how the recently launched non-profit biotech will tackle the translational science of TB, malaria and diarrhoeal diseases.
This article discusses the market for oral anticoagulants, and how therapies in development and antidotes to existing drugs could affect the therapeutic landscape.
Dysregulation of the endocannabinoid system has been implicated in numerous diseases, particularly pain, psychiatric and neurological disorders, but therapeutic intervention in this complex system has proved challenging. In this Perspective article, Di Marzo discusses the lessons learned from the development of drugs that alter endocannabinoid signalling and highlights novel opportunities for pharmacologically manipulating the endocannabinoid system, such as the use of multi-target drugs.
The potential of adeno-associated viral (AAV)-mediated gene therapy for neurological disorders is rapidly emerging. Evidence of clinical efficacy and safety, as well as durable transgene expression, has now been reported in several central nervous system disorders. Here, Sah and colleagues discuss key considerations in the design and development of therapeutic AAV vectors, highlighting promising therapeutic targets and recent clinical trials.
Neurodegenerative disorders of ageing such as Alzheimer disease, Parkinson disease and Huntington disease are characterized by the presence of neurotoxic misfolded and aggregated proteins. One reason underlying the accumulation of these proteins is insufficient clearance by intracellular and extracellular pathways such as the autophagic–lysosomal network and the glymph system. This article reviews the potential for therapeutically enhancing the clearance of neurotoxic proteins to curtail the onset and slow the progression of neurodegenerative disorders of ageing.
